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What changed in GENELUX Corp's 10-K — 2023 vs 2024

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Paragraph-level year-over-year comparison of GENELUX Corp's 2023 and 2024 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2024 report.

+530 added−499 removedSource: 10-K (2024-12-31) vs 10-K (2024-03-29)

Top changes in GENELUX Corp's 2024 10-K

530 paragraphs added · 499 removed · 402 edited across 8 sections

Item 1. Business

Business — how the company describes what it does

73 edited+45 added−14 removed216 unchanged
Biggest changeNSCLC We are also aware of other companies either marketing or focused on developing competing therapies for the treatment of NSCLC, which generally fall into the following treatment groups: ● Chemotherapies which include carboplatin, vinorelbine tartrate, paclitaxel, taxotere, and doxorubicin hydrochloride, each of which is manufactured by multiple companies, along with Celgene’s ABRAXANE, Eli Lilly’s GEMZAR and Eli Lilly’s ALIMTA. ● BRAF (v-Raf murine sarcoma viral oncogene homolog B) kinase inhibitors which include Novartis’s TAFINLAR and Novartis’s MEKINIST. ● ALK (anaplastic lymphoma kinase) inhibitors which include Pfizer’s XALKORI, Novartis’s ZYKADIA, Genentech’s ALECENSA, Takeda Pharmaceutical Company’s ALUNBRIG and Pfizer’s LORBRENA. ● EGFR (epidermal growth factor receptor) inhibitors which include AstraZeneca’s TAGRISSO, AstraZeneca/Teva Pharmaceutical Industries Ltd.’s IRESSA, Astellas Pharma Inc./Chugai Pharmaceutical Inc./Roche/Genentech’s TARCEVA, Boehringer Ingelheim Pharmaceutical’s GILOTRIF, Pfizer’s VIZIMPRO and Eli Lilly’s PORTRAZZA. ● TRK (tropomyosin receptor kinase) inhibitors which include Genentech’s ROZLYTREK, Bayer AG’s VITRAKVI and Novartis’s TABRECTA. ● RET (rearranged during transfection) kinase inhibitors which include Eli Lilly’s RETEVMO and Blueprint Medicines/Roche’s GAVRETO. ● Anti-angiogenesis medications which include Genentech’s Avastin and Amgen Inc.’s MVASI (in combination with cisplatin and paclitaxel) and Eli Lilly’s CYRAMZA (in combination with docetaxel and erlotinib).
Biggest changeNSCLC We are also aware of other companies either marketing or focused on developing competing therapies for the treatment of NSCLC, which generally fall into the following treatment groups: ● Chemotherapies which include cisplatin (manufactured by 7 companies), carboplatin (manufactured by 12 companies), carboplatin (manufactured by 12 companies), vinorelbine tartrate (manufactured by 4 companies), and paclitaxel (manufactured by 11 companies), along with the following brand products (and generic manufacturers) Sanofi-Aventis’s Taxotere (16 manufacturers), Celgene’s ABRAXANE, Baxter International’s Doxil (8 manufacturer), Eli Lilly’s GEMZAR (14 companies) and Eli Lilly’s ALIMTA (18 manufacturers). 17 ● BRAF (v-Raf murine sarcoma viral oncogene homolog B) kinase inhibitors which include Roche’s ZELBORAF, GSK’s TAFINLAR and Novartis’s BRAFTOVI. ● ALK (anaplastic lymphoma kinase) inhibitors which include Pfizer’s XALKORI, Novartis’s ZYKADIA, Roche/Genentech’s ALECENSA, Takeda Pharmaceutical Company’s ALUNBRIG and Pfizer’s LORBRENA. ● EGFR (epidermal growth factor receptor) inhibitors which include AstraZeneca’s TAGRISSO, AstraZeneca/Teva Pharmaceutical Industries Ltd.’s IRESSA, Astellas Pharma Inc./Chugai Pharmaceutical Inc./Roche/Genentech’s TARCEVA, Boehringer Ingelheim Pharmaceutical’s GILOTRIF, Pfizer’s VIZIMPRO, Janssen’s LAZCLUZE and RYBREVANT, and Eli Lilly’s PORTRAZZA. ● TRK (tropomyosin receptor kinase) inhibitors which include Genentech’s ROZLYTREK, Bayer AG’s VITRAKVI, Eli Lilly’s TEPMETKO and Novartis’s MEKINIST and TABRECTA. ● RET (rearranged during transfection) kinase inhibitors which include AstraZeneca’s CAPRELSA, Eli Lilly’s RETEVMO and Rigel Pharmaceuticals’ GAVRETO. ● HER2/ErbB2 (human epidermal growth factor receptor 2 and receptor tyrosine-protein kinase) inhibitors which includes AstraZeneca’s ENHERTU. ● ROS1 (receptor tyrosine kinase) inhibitors which includes BMS’ AUGTYRO.
Such restrictions under applicable federal and state healthcare laws and regulations include the following: ● The federal Anti-Kickback Statute, which prohibits, among other things, individuals and entities from knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe, or rebate), directly or indirectly, overtly or covertly, in cash or in kind, to induce, or in return for, either the referral of an individual for, or the purchase, lease, order or recommendation of, any good, facility, item or service for which payment may be made, in whole or in part, under a federal healthcare program, such as the Medicare and Medicaid programs. 23 ● The federal civil and criminal false claims laws, including, without limitation, the civil False Claims Act, and the federal Civil Monetary Penalties Law, which prohibit individuals or entities from, among other things, knowingly presenting, or causing to be presented, false or fraudulent claims for payment of federal funds, and knowingly making, or causing to be made, a false record or statement material to a false or fraudulent claim to avoid, decrease or conceal an obligation to pay money to the federal government. ● The Health Insurance Portability and Accountability Act (HIPAA), which prohibits, among other things, knowingly and willfully executing, or attempting to execute, a scheme or artifice to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private), willfully obstructing a criminal investigation of a healthcare offense, and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false, fictitious or fraudulent statements in connection with the delivery of, or payment for, healthcare benefits, items or services relating to healthcare matters. ● The FDCA, which prohibits, among other things, the adulteration or misbranding of drugs, biological products and medical devices. ● The federal physician payment transparency requirements, sometimes referred to as the Physician Payments Sunshine Act, created under the Patient Protection and Affordable Care Act as amended by the Health Care and Education Reconciliation Act of 2010 (collectively, the ACA) and its implementing regulations, which require certain manufacturers of drugs, devices, biological products and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to CMS information related to payments or other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members. ● Analogous state and foreign anti-kickback and false claims laws that may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state and local laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; state laws that require the reporting of information related to drug pricing; and state and local laws requiring the registration of pharmaceutical sales representatives. 24 Efforts to ensure that our business arrangements with third parties comply with applicable healthcare laws and regulations will involve substantial costs.
Such restrictions under applicable federal and state healthcare laws and regulations include the following: ● The federal Anti-Kickback Statute, which prohibits, among other things, individuals and entities from knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe, or rebate), directly or indirectly, overtly or covertly, in cash or in kind, to induce, or in return for, either the referral of an individual for, or the purchase, lease, order or recommendation of, any good, facility, item or service for which payment may be made, in whole or in part, under a federal healthcare program, such as the Medicare and Medicaid programs. 27 ● The federal civil and criminal false claims laws, including, without limitation, the civil False Claims Act, and the federal Civil Monetary Penalties Law, which prohibit individuals or entities from, among other things, knowingly presenting, or causing to be presented, false or fraudulent claims for payment of federal funds, and knowingly making, or causing to be made, a false record or statement material to a false or fraudulent claim to avoid, decrease or conceal an obligation to pay money to the federal government. ● The Health Insurance Portability and Accountability Act (HIPAA), which prohibits, among other things, knowingly and willfully executing, or attempting to execute, a scheme or artifice to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private), willfully obstructing a criminal investigation of a healthcare offense, and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false, fictitious or fraudulent statements in connection with the delivery of, or payment for, healthcare benefits, items or services relating to healthcare matters. ● The FDCA, which prohibits, among other things, the adulteration or misbranding of drugs, biological products and medical devices. ● The federal physician payment transparency requirements, sometimes referred to as the Physician Payments Sunshine Act, created under the Patient Protection and Affordable Care Act as amended by the Health Care and Education Reconciliation Act of 2010 (collectively, the ACA) and its implementing regulations, which require certain manufacturers of drugs, devices, biological products and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to CMS information related to payments or other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members. ● Analogous state and foreign anti-kickback and false claims laws that may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state and local laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; state laws that require the reporting of information related to drug pricing; and state and local laws requiring the registration of pharmaceutical sales representatives. 28 Efforts to ensure that our business arrangements with third parties comply with applicable healthcare laws and regulations will involve substantial costs.
We recently leased a second building in the same location which, when upgrades are completed, will provide laboratory capabilities and administrative offices. 1 PIPELINE Our pipeline is summarized below: OUR STRATEGY Our strategy is to leverage our deep internal capabilities in the clinical development of oncolytic viruses to create a leading immunotherapy company, discovering, developing and commercializing next-generation products for the treatment of a broad range of cancers, including solid tumors, many of which are among the most difficult cancers to treat.
We leased a second building in the same location which, when upgrades are completed, will provide laboratory capabilities and administrative offices. 1 PIPELINE Our pipeline is summarized below: OUR STRATEGY Our strategy is to leverage our deep internal capabilities in the clinical development of oncolytic viruses to create a leading immunotherapy company, discovering, developing and commercializing next-generation products for the treatment of a broad range of cancers, including solid tumors, many of which are among the most difficult cancers to treat.
DEVELOPMENT PROGRAMS Platinum Resistant/Refractory Ovarian Cancer (PRROC) We envision that Olvi-Vec-primed immunochemotherapy may overcome chemotherapy resistance for patients with end-stage ovarian cancer that would otherwise consider palliative care or use of drugs with historically poor response rates. We initiated a Phase 3 registration trial in PRROC in the third quarter of 2022.
DEVELOPMENT PROGRAMS Platinum Resistant/Refractory Ovarian Cancer We envision that Olvi-Vec-primed immunochemotherapy may overcome chemotherapy resistance for patients with end-stage ovarian cancer that would otherwise consider palliative care or use of drugs with historically poor response rates. We initiated a Phase 3 OnPrime registration trial in PRROC in the third quarter of 2022.
We have strong in-house pharmaceutical development and manufacturing capabilities and have established, equipped and are operating our own cGMP manufacturing facility in San Diego, California for multi-product cGMP manufacturing. 2 ● Leverage our CHOICE discovery platform to build a portfolio of oncology product candidates that target a range of immune mechanisms and progress these product candidates into clinical development.
We have strong in-house pharmaceutical development and manufacturing capabilities and have established, equipped and are operating our own cGMP manufacturing facility in San Diego, California for multi-product cGMP manufacturing. ● Leverage our CHOICE discovery platform to build a portfolio of oncology product candidates that target a range of immune mechanisms and progress these product candidates into clinical development.
Under the Newsoara License, Newsoara also granted to us an exclusive and royalty bearing license to develop, commercialize and exploit outside the territory any derived products developed by Newsoara. 10 Under the terms of the Newsoara License and to date, we have received from Newsoara an aggregate of $11.0 million ($5.0 million as an upfront payment and $6.0 million as a milestone payment).
Under the Newsoara License, Newsoara also granted to us an exclusive and royalty bearing license to develop, commercialize and exploit outside the territory any derived products developed by Newsoara. 11 Under the terms of the Newsoara License and to date, we have received from Newsoara an aggregate of $11.0 million ($5.0 million as an upfront payment and $6.0 million as a milestone payment).
To date, Olvi-Vec has been studied in multiple early- and mid-phase clinical trials via regional, local and systemic deliveries, as a monotherapy and in combination with other therapies, in approximately 150 patients in seven clinical trials with a variety of cancer types.
To date, Olvi-Vec has been studied in multiple early- and mid-phase clinical trials via regional, local and systemic deliveries, as a monotherapy and in combination with other therapies, in approximately 150 patients in seven completed clinical trials with a variety of cancer types.
Accordingly, we may be subject to numerous data privacy and security obligations, including federal, state, local, and foreign laws, regulations, guidance, industry standards, external and internal privacy and security policies, contractual requirements and other obligations related to data privacy and security. These frameworks are evolving and may impose potentially conflicting obligations.
Accordingly, we are subject to numerous data privacy and security obligations, including federal, state, local, and foreign laws, regulations, guidance, industry standards, external and internal privacy and security policies, contractual requirements and other obligations related to data privacy and security. These frameworks are evolving and may impose potentially conflicting obligations.
The conduct of the preclinical tests must comply with federal regulations and requirements including GLPs. 17 Concurrent with clinical trials, companies usually are required to complete some long-term preclinical testing, such as animal tests of reproductive adverse events and carcinogenicity, and must also develop additional information about the chemistry and physical characteristics of the drug and finalize a process for manufacturing the drug in commercial quantities in accordance with cGMP requirements.
The conduct of the preclinical tests must comply with federal regulations and requirements including GLPs. 19 Concurrent with clinical trials, companies usually are required to complete some long-term preclinical testing, such as animal tests of reproductive adverse events and carcinogenicity, and must also develop additional information about the chemistry and physical characteristics of the drug and finalize a process for manufacturing the drug in commercial quantities in accordance with cGMP requirements.
In addition, changes to the manufacturing process or facility generally require prior FDA approval or notification before being implemented, and other types of changes to the approved product, such as adding new indications and additional labeling claims, are also subject to further FDA review and approval. 22 Moreover, the Drug Quality and Security Act imposes obligations on manufacturers of biopharmaceutical products related to product tracking and tracing.
In addition, changes to the manufacturing process or facility generally require prior FDA approval or notification before being implemented, and other types of changes to the approved product, such as adding new indications and additional labeling claims, are also subject to further FDA review and approval. 26 Moreover, the Drug Quality and Security Act imposes obligations on manufacturers of biopharmaceutical products related to product tracking and tracing.
Orphan products are also exempt from the PREA requirements. 20 The FDA reviews a BLA within 60 days of submission to determine if it is substantially complete before the agency accepts it for filing. The FDA may refuse to file any BLA that it deems incomplete or not properly reviewable at the time of submission and may request additional information.
Orphan products are also exempt from the PREA requirements. 22 The FDA reviews a BLA within 60 days of submission to determine if it is substantially complete before the agency accepts it for filing. The FDA may refuse to file any BLA that it deems incomplete or not properly reviewable at the time of submission and may request additional information.
The FDA may also require post-marketing clinical trials, sometimes referred to as Phase 4 clinical trials, designed to further assess a biologic product’s safety and effectiveness, and testing and surveillance programs to monitor the safety of approved products that have been commercialized. 21 Every five years, the FDA agrees to specified performance goals in the review of BLAs under the PDUFA.
The FDA may also require post-marketing clinical trials, sometimes referred to as Phase 4 clinical trials, designed to further assess a biologic product’s safety and effectiveness, and testing and surveillance programs to monitor the safety of approved products that have been commercialized. 23 Every five years, the FDA agrees to specified performance goals in the review of BLAs under the PDUFA.
We are focused on developing next-generation viral immunotherapies for the treatment of cancer. Any viral immunotherapies that we successfully develop and commercialize will compete with existing therapies and new therapies that may become available in the future. 14 Competition in cancer therapeutics comes in many forms, where different technologies are employed against different molecular targets or biological systems.
We are focused on developing next-generation viral immunotherapies for the treatment of cancer. Any viral immunotherapies that we successfully develop and commercialize will compete with existing therapies and new therapies that may become available in the future. 15 Competition in cancer therapeutics comes in many forms, where different technologies are employed against different molecular targets or biological systems.
We cannot provide any assurances that we will be able to obtain and maintain third-party coverage or adequate reimbursement for our product candidates in whole or in part. 25 In the EU, pricing and reimbursement schemes vary widely from country to country. Some countries provide that products may be marketed only after a reimbursement price has been agreed.
We cannot provide any assurances that we will be able to obtain and maintain third-party coverage or adequate reimbursement for our product candidates in whole or in part. 29 In the EU, pricing and reimbursement schemes vary widely from country to country. Some countries provide that products may be marketed only after a reimbursement price has been agreed.
This clinical trial is not yet scheduled to be initiated. 9 Additional Potential Indications for Olvi-Vec We believe our preclinical and clinical data support the broad development of Olvi-Vec in patients with liquid or (metastatic) solid tumors, as a monotherapy or in combination with other therapies.
This clinical trial is not yet scheduled to be initiated. 10 Additional Potential Indications for Olvi-Vec We believe our preclinical and clinical data support the broad development of Olvi-Vec in patients with liquid or (metastatic) solid tumors, as a monotherapy or in combination with other therapies.
Clinical trial sponsors may also choose to discontinue clinical trials as a result of risks to subjects, a lack of favorable results, or changing business priorities. 19 Compliance with cGMP Requirements Manufacturers of biological products must comply with applicable cGMP regulations, including quality control and quality assurance and maintenance of records and documentation.
Clinical trial sponsors may also choose to discontinue clinical trials as a result of risks to subjects, a lack of favorable results, or changing business priorities. 21 Compliance with cGMP Requirements Manufacturers of biological products must comply with applicable cGMP regulations, including quality control and quality assurance and maintenance of records and documentation.
In the case of some product candidates for severe or life-threatening diseases, especially when the product candidate may be too inherently toxic to ethically administer to healthy volunteers, the initial human testing is often conducted in patients. 18 ● Phase 2.
In the case of some product candidates for severe or life-threatening diseases, especially when the product candidate may be too inherently toxic to ethically administer to healthy volunteers, the initial human testing is often conducted in patients. 20 ● Phase 2.
In that regard, we have observed the potential benefits of combining Olvi-Vec with platinum compounds in preclinical studies, and in a completed Phase 1 clinical trial combining Olvi-Vec with cisplatin and radiation as front-line therapy in newly diagnosed head and neck cancer patients. Olvi-Vec was well tolerated and demonstrated favorable trends in PFS and OS.
Moroever, we have observed the potential benefits of combining Olvi-Vec with platinum compounds in preclinical studies, and in a completed Phase 1 clinical trial combining Olvi-Vec with cisplatin and radiation as front-line therapy in newly diagnosed head and neck cancer patients. Olvi-Vec was well tolerated and demonstrated favorable trends in PFS and OS.
In addition, in clinical trials in which Olvi-Vec was systemically administered, Olvi-Vec was: ● Shown to likely overcome pre-existing anti-vaccinia antibody levels by high and condensed dosing; ● Detectable in the active state as live virus in blood circulation even at two hours after infusion, which we believe is ample time for the virus to reach distal metastases; and ● Capable of infecting tumor tissues and reducing circulating tumor cells.
In addition, in clinical trials in which Olvi-Vec was systemically administered, Olvi-Vec was: ● Shown to likely overcome pre-existing and/or induced anti-vaccinia antibody levels by high and/or extended dosing; ● Detectable in the active state as live virus in blood circulation even at two hours after infusion, which we believe is ample time for the virus to reach distal metastases; and ● Capable of infecting tumor tissues and reducing circulating tumor cells.
We do not have long-term supply arrangements in place with our raw material and component suppliers. 12 Sales and Marketing None of our product candidates has been approved for sale.
We do not have long-term supply arrangements in place with our raw material and component suppliers. 13 Sales and Marketing None of our product candidates has been approved for sale.
As shown in the following figure, this was documented by multiple efficacy evaluation endpoints (based on pre-chemotherapy baseline), such as overall response rate (ORR), as determined by RECIST 1.1 Criteria by CT scans and GCIG CA-125 Response Criteria, and durability of responses as determined by duration of response, progression free survival (PFS) and overall survival (OS).
As shown in the following figure, this was documented by multiple efficacy evaluation endpoints (based on pre-chemotherapy baseline), such as overall response rate (ORR), as determined by RECIST 1.1 Criteria by CT scans and GCIG CA-125 Response Criteria, and durability of responses as determined by duration of response, PFS and OS.
(SillaJen), Targovax USA, Theriva Biologics, Inc., Transgene SA, Turnstone Biologics Corp. and Vyriad, Inc.; ● Approved immunotherapy antibodies and immunotherapy agents in clinical development, including antibody agents, bispecific T cell engagers, including those in development by Amgen, and immuno-oncology companies focused on IL-12, such as Ziopharm Oncology Inc.; ● Cancer vaccines, including personalized vaccines and those targeting tumor neoantigens, including neoantigen therapies in development by companies such as Advaxis, Inc., Agenus Inc., AstraZeneca, Bavarian Nordic A/S, BioNTech SE, Genocea Biosciences, Inc., Gritstone Oncology, Inc., Heat Biologics, Inc., ImmunityBio, Inc., IMV Inc., Moderna, Inc., SOTIO a.s., Transgene SA, and VBI Vaccines Inc.; ● Cell-based therapies, including TILs in development by Iovance Biotherapeutics, Inc., TVax Biomedical, Inc. and Turnstone Biologics, Inc. and approved and in-development CAR T cell therapies, including those commercialized by Bristol-Myers Squibb Company (BMS), Gilead Sciences Inc. and Novartis AG, T cell receptor and NK cell therapies; ● Therapies aimed at activating innate immunity such as those targeting stimulator of interferon genes protein and toll-like receptors including those in development by BMS, Checkmate Pharmaceuticals Inc., Chinook Therapeutics Inc., GlaxoSmithKline plc (GSK), Idera Pharmaceuticals, Inc., Merck, Mologen AG, Nektar Therapeutics, TriSalus Life Sciences, and UroGen Pharma Inc.; and ● Traditional cancer therapies, including chemotherapy, surgery, radiation and targeted therapies.
(SillaJen), Circio Holding ASA, Theriva Biologics, Inc., Transgene SA (Transgene) and Vyriad, Inc.; ● Approved immunotherapy antibodies and immunotherapy agents in clinical development, including antibody agents, bispecific T cell engagers, including those in development by Amgen, and immuno-oncology companies focused on IL-12, such as Ziopharm Oncology Inc.; ● Cancer vaccines, including personalized vaccines and those targeting tumor neoantigens, including neoantigen therapies in development by companies such as Advaxis, Inc., Agenus Inc., AstraZeneca, Bavarian Nordic A/S, BioNTech SE, Genocea Biosciences, Inc., Gritstone Oncology, Inc., Heat Biologics, Inc., ImmunityBio, Inc., IMV Inc., Moderna, Inc., SOTIO a.s., Transgene, and VBI Vaccines Inc.; ● Cell-based therapies, including tumor infiltrating lymphocytes in development by Iovance Biotherapeutics, Inc., TVAX and Turnstone Biologics, Corp. and approved and in-development CAR T cell therapies, including those commercialized by Bristol Myers Squibb Company (BMS), Gilead Sciences Inc. and Novartis AG, T cell receptor and NK cell therapies; ● Therapies aimed at activating innate immunity such as those targeting stimulator of interferon genes protein (and toll-like receptors including those in development by BMS, Checkmate Pharmaceuticals Inc., Chinook Therapeutics Inc., GlaxoSmithKline plc, Idera Pharmaceuticals, Inc., Merck, Mologen AG, Nektar Therapeutics, TriSalus Life Sciences, and UroGen Pharma Inc.; and ● Traditional cancer therapies, including chemotherapy, surgery, radiation and targeted therapies.
Our actual or perceived failure to comply with such obligations could lead to regulatory investigations or actions; litigation (including class claims) and mass arbitration demands; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; loss of customers or sales; and other adverse business consequences ” for additional information about the laws and regulations to which we are or may become subject and about the risks to our business associated with such laws and regulations.
Our (or the third parties with whom we work) actual or perceived failure to comply with such obligations could lead to regulatory investigations or actions; litigation (including class-action claims) and mass arbitration demands; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; loss of customers or sales; and other adverse business consequences ” for additional information about the laws and regulations to which we are or may become subject and about the risks to our business associated with such laws and regulations.
Because of the extensive time required for clinical development and regulatory review of any drug we may develop from our product candidates, it is possible that, before any of our drugs can be commercialized, any related patent may expire or remain in force for only a short period following commercialization, thereby reducing any advantage of any such patent. 13 As of December 31, 2023, our patent portfolio consisted of 19 issued U.S. patents, one pending U.S. patent application, 14 issued foreign patents, six pending foreign patent applications and one PCT application, which relate generally to the composition of our current and potential future products, and their methods of use.
Because of the extensive time required for clinical development and regulatory review of any drug we may develop from our product candidates, it is possible that, before any of our drugs can be commercialized, any related patent may expire or remain in force for only a short period following commercialization, thereby reducing any advantage of any such patent. 14 As of December 31, 2024, our patent portfolio consisted of 11 issued U.S. patents, 1 pending U.S. patent application, 9 issued foreign patents, 7 pending foreign patent applications and one PCT application, which relate generally to the composition of our current and potential future products, and their methods of use.
To facilitate talent attraction and retention, we strive to make our company a safe and rewarding workplace, with opportunities for our employees to grow and develop in their careers, supported by strong compensation, benefits and health and wellness programs, and by programs that build connections between our employees. 29 As of December 31, 2023, we had 23 full-time and part-time employees, including 15 employees engaged in research and development and manufacturing and eight engaged in management and administrative functions.
To facilitate talent attraction and retention, we strive to make our company a safe and rewarding workplace, with opportunities for our employees to grow and develop in their careers, supported by strong compensation, benefits and health and wellness programs, and by programs that build connections between our employees. 33 As of December 31, 2024, we had 24 full-time and part-time employees, including 15 employees engaged in research and development and manufacturing and 9 engaged in management and administrative functions.
In addition, in August 2022, President Biden signed the Inflation Reduction Act of 2022 (IRA) into law, which among other things, extends enhanced subsidies for individuals purchasing coverage in ACA marketplaces through plan year 2025.
For example, in August 2022, the Inflation Reduction Act of 2022 (IRA) was signed into law, which among other things, extends enhanced subsidies for individuals purchasing coverage in ACA marketplaces through plan year 2025.
In addition to lysing cancer cells, Olvi-Vec induces an acute inflammatory response within cancer tissue that modulates the immune microenvironment in a way that would be anticipated to enhance the effects of adoptively transferred neoantigen-specific effector T cells.
Reducing cancer tissue associated immunosuppression could increase the anti-cancer effects of adoptively transferred neoantigen- specific effector T cells. In addition to lysing cancer cells, Olvi-Vec induces an acute inflammatory response within cancer tissue that modulates the immune microenvironment in a way that would be anticipated to enhance the effects of adoptively transferred neoantigen-specific effector T cells.
We believe intravenous delivery of Olvi-Vec to the lung, unlike other viruses that are administered intra-tumorally and that are less amenable to repeat injections, is particularly compelling because of the ‘first pass effect’ (i.e., after administration the virus reaches the heart and is then first transported to the lungs).
We believe intravenous delivery of Olvi-Vec to the lung, unlike other viruses that are administered locally, is particularly compelling because of the ‘first pass effect’ (i.e., after administration the virus reaches the heart and is then first transported to the lungs).
We believe that the potential to induce immune responses may represent an important mechanism to control tumor growth, prevent the spread of tumors, improve the ability to surgically remove tumors and perhaps reduce the need for surgery, and reduce or delay the onset of relapse. We may also pursue additional indications via regional delivery.
We believe that the potential to induce immune responses may represent an important mechanism to control tumor growth, prevent the spread of tumors, improve the ability to surgically remove tumors and perhaps reduce the need for surgery, and reduce or delay the onset of relapse.
Information contained in our website is not a part of, nor incorporated by reference into, this Annual Report on Form 10-K or our other filings with the SEC, and should not be relied upon. 30
Information contained in our website is not a part of, nor incorporated by reference into, this Annual Report or our other filings with the SEC, and should not be relied upon. 34
Our Phase 3 registration trial of Olvi-Vec in platinum resistant/refractory ovarian cancer (PRROC) initiated enrollment in the third quarter of 2022 and we began regulatory study start-up in the United States of a Phase 2, open-label, randomized, and controlled clinical trial of Olvi-Vec in patients with recurrent non-small cell lung cancer (NSCLC) (VIRO-25) in 2023. ● Support the clinical and commercial development of Olvi-Vec with our strategic partner, Newsoara, advise and coordinate the design and initiation of clinical trials in China and provide product supply and technology transfer .
Our Phase 3 OnPrime registration trial of Olvi-Vec in platinum resistant/refractory ovarian cancer (PRROC) initiated enrollment in the third quarter of 2022 and our Phase 2 VIRO-25 trial, an open-label, randomized and controlled clinical trial of Olvi-Vec in patients with recurrent non-small cell lung cancer (NSCLC), initiated enrollment in the United States in the fourth quarter of 2024. ● Support the clinical and commercial development of Olvi-Vec with our strategic partner, Newsoara, advise and coordinate the design and initiation of clinical trials in China and provide product supply and technology transfer .
We completed our initial public offering in January 2023, and our common stock is listed on the Nasdaq Capital Market under the symbol “GNLX.” We are an “emerging growth company” as defined in the Tax Cuts and Jobs Act of 2017.
We completed our initial public offering in January 2023, and our common stock is listed on the Nasdaq Capital Market under the symbol “GNLX.” We are an “emerging growth company” as defined in the Jumpstart Our Business Startups Act of 2012 (JOBS Act).
The topline data of the Phase 2 portion was published in JAMA Oncology in May 2023 In the Phase 1b portion of the clinical trial, no virus- related severe organ toxicity was observed by clinical or serologic parameters and a maximum tolerated dose (MTD) was not reached. Olvi-Vec treatment was observed to be well tolerated.
In the Phase 1b portion of the clinical trial, no virus- related severe organ toxicity was observed by clinical or serologic parameters and a maximum tolerated dose (MTD) was not reached. Olvi-Vec treatment was observed to be well tolerated.
Those clinical trials have yielded data that has informed our current and future clinical strategy and trial design involving multiple indications and methods of delivery. 3 In our clinical trials, irrespective of the route of administration, dosing regimen or cancer type, Olvi-Vec was: ● Observed to be well tolerated, and whether administered in a single dose or multiple doses per cycle, no MTD was reached in any of the trials and there were no significant issues with virus shedding into the environment; ● Shown to infect and selectively kill tumor cells, initiate an anti-tumoral response and modulate the tumor microenvironment, including re-sensitizing certain tumors to chemotherapy; and ● Shown to enhance chemotherapeutic activities in a combination therapy setting.
In our clinical trials, irrespective of the route of administration, dosing regimen or cancer type, Olvi-Vec was: ● Observed to be well tolerated, and whether administered in a single dose or multiple doses per cycle, no MTD was reached in any of the trials and there were no significant issues with virus shedding into the environment; ● Shown to infect and selectively kill tumor cells, initiate an anti-tumoral response and modulate the tumor microenvironment, including re-sensitizing certain tumors to chemotherapy; and ● Shown to enhance chemotherapeutic activities in a combination therapy setting.
We expect to report topline results in the second half of 2025. The following graphic summarizes the study design for the Phase 3 registration trial. 5 Previously, we conducted a Phase 1b/2 clinical trial of Olvi-Vec, which was administered intraperitoneally in a single round of treatment consisting of a bolus infusion on two consecutive days.
The following graphic summarizes the study design for the Phase 3 OnPrime registration trial. 5 Previously, we conducted a Phase 1b/2 clinical trial of Olvi-Vec, which was administered intraperitoneally in a single round of treatment consisting of a bolus infusion on two consecutive days.
In June 2021, we entered into a License Agreement with V2ACT (V2ACT License), pursuant to which we granted V2ACT a worldwide, non-exclusive, fully paid, royalty free license for our proprietary oncolytic virus (Licensed Virus(es)) to research, develop and commercialize any product, procedure or method for the treatment of cancer that combines (a) Licensed Virus(es), and (b) autologous or allogeneic cancer-specific T lymphocytes (T-Cell Therapeutic(s)) for the diagnosis, prevention and treatment of cancer in humans (Products).
In June 2021, TVAX entered into a License Agreement with V2ACT (TVAX License), pursuant to which TVAX granted V2ACT a worldwide, non-exclusive, fully paid, royalty free license for its proprietary T-Cell Therapeutics (Licensed T-Cell Therapeutic(s)) to research, develop and commercialize any product, procedure or method for the treatment of cancer that combines (a) any virus-based cancer therapeutics, and (b) Licensed T-Cell Therapeutic(s) for the diagnosis, prevention and treatment of cancer in humans (TVAX Products).
By way of example, in March 2010, the ACA was signed into law, intended to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add transparency requirements for the healthcare and health insurance industries, impose taxes and fees on the healthcare industry and impose additional health policy reforms.
By way of example, in March 2010, the ACA was signed into law, intended to broaden access to health insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add transparency requirements for the healthcare and health insurance industries, impose taxes and fees on the healthcare industry and impose additional health policy reforms. 30 There have been executive, judicial and Congressional challenges and amendments to certain aspects of the ACA.
Importantly, relative to historical comparisons, patients receiving Olvi-Vec-primed immunochemotherapy generally showed marked clinical benefits, particularly with respect to ORR per RECIST 1.1 (54%) with durable response, median PFS (11.0 months) and median OS (15.7 months).
Importantly, relative to historical comparisons, the heavily pretreated patients with median 4 prior lines receiving Olvi-Vec-primed immunochemotherapy generally showed marked clinical benefits, particularly with respect to ORR per RECIST 1.1 (54%: 19% CRs; 35% PRs) with durable response, median PFS (11.0 months) and median OS (15.7 months).
Additionally, the CCPA applies to personal information of consumers, business representative, and employees who are California residents, imposes specific requirements on covered businesses, provides for administrative fines of up to $7,500 per violation and allows private litigants affected by certain data breaches to recover significant statutory damages. In addition, the CPRA expanded the CCPA’s requirements.
Additionally, the CCPA applies to personal information of consumers, business representative, and employees who are California residents, imposes specific requirements on covered businesses, provides for fines and allows private litigants affected by certain data breaches to recover significant statutory damages.
Our Phase 1b/2 clinical trial in patients with recurrent small cell lung cancer (SCLC) in China is ongoing. Upon successful completion, Newsoara intends to seek approval from the Chinese Centre for Drug Evaluation to join us in the VIRO-25 clinical trial and initiate further trials in SCLC and ovarian cancer. ● Prepare for US Launch in Ovarian Cancer.
Our co-sponsored Phase 1b/2 clinical trial in patients with recurrent small cell lung cancer (SCLC) in China is ongoing. Subject to regulatory authorization from the Chinese Centre for Drug Evaluation, Newsoara intends to initiate a Phase 2 VIRO-25 clinical trial in China and initiate further trials in SCLC and ovarian cancer. 2 ● Prepare for US Launch in ovarian cancer.
In other words, while we administer the same virus product to different patients, the cellular immune response generated is expected to be specific to the unique neoantigens in that patient.
Importantly, these product candidates are “off-the-shelf” personalized immunotherapies. In other words, while we administer the same virus product to different patients, the cellular immune response generated is expected to be specific to the unique neoantigens in that patient.
We expect that these initiatives, as well as other healthcare reform measures that may be adopted in the future, as well as the trend toward managed healthcare and increasing influence of managed care organizations, may result in more rigorous coverage criteria and lower reimbursement, and in additional downward pressure on the price that we receive for any approved product.
Presidential and Congressional elections, as well as the trend toward managed healthcare and increasing influence of managed care organizations, may result in more rigorous coverage criteria and lower reimbursement, and in additional downward pressure on the price that we receive for any approved product.
As Genelux’ clinical trial program progresses and diversifies, the company intends to expand its preliminary commercial strategy initiatives and support corporate development efforts. ● Broaden and strengthen our internal manufacturing capabilities, utilizing our in-house manufacturing facility.
As our clinical trial program progresses in ovarian cancer, we intend to expand our preliminary commercial strategy initiatives and support corporate development efforts. ● Broaden and strengthen our internal manufacturing capabilities, utilizing our in-house manufacturing facility.
Patients enrolled into the trial were heavily pretreated (with a median of four prior lines of therapy), with confirmed progressive disease (PD) at the time of enrollment, and had PRROC, with poor responses to conventional chemotherapies.
Patients enrolled into the trial were heavily pretreated (with a median of four prior lines of therapy), with confirmed progressive disease (PD) at the time of enrollment, and had PRROC, with poor responses to conventional chemotherapies. The topline data of the Phase 2 portion was published in JAMA Oncology in May 2023.
V2ACT Immunotherapy is designed to combine the benefits of agents from four of the five subcategories of immunotherapies. Neoantigen-specific adoptive T cell therapy and Olvi-Vec employ different and potentially synergistic mechanisms for cancer cell killing and prolonging patient survival.
V2ACT Immunotherapy is designed to combine the benefits of agents from four of the five subcategories of immunotherapies. Neoantigen-specific adoptive T cell therapy and Olvi-Vec employ different and potentially synergistic mechanisms for cancer cell killing and prolonging patient survival. In January 2024, Elias Animal Health, a companion animal cancer therapeutics company developing V2ACT immunotherapy in dogs, announced that the U.S.
Pancreatic Cancer V2ACT Immunotherapy is a proprietary, indication-agnostic personalized immunotherapy designed to maximize the number and effect of cancer neoantigen-specific effector T cells within cancer tissues.
We expect to provide an interim readout (updated data) in the second half of 2025. Pancreatic Cancer V2ACT Immunotherapy is a proprietary, indication-agnostic personalized immunotherapy designed to maximize the number and effect of cancer neoantigen-specific effector T cells within cancer tissues.
In addition, the IRA directs the Secretary of HHS to establish a Drug Price Negotiation Program (the Program) to lower prices for certain single-source prescription drugs and biologics covered under Medicare Parts B and D, based on criteria established under the IRA.
For example, the IRA directs the Secretary of HHS to establish a Drug Price Negotiation Program (the Program) to lower prices for certain high-expenditure, single-source biologics that have been on the market for at least 11 years covered under Medicare Parts B and D, based on criteria established under the IRA.
In August 2019, SillaJen announced the discontinuation of its Phase 3 PHOCUS trial of Pexa-Vec in advanced liver cancer for futility. 15 PRROC We are also aware of other companies either marketing or focused on developing competing therapies for the treatment of ovarian cancer, including PRROC: ● Currently marketed products for ovarian cancer include generic products cisplatin (manufactured by 18 companies), carboplatin (manufactured by 22 companies) and paclitaxel (manufactured by 19 companies), along with the following brand products (and generic manufacturers): AbbVie’s ELAHERE, Sanofi-Aventis’s TAXOTERE (17 manufacturers), Celgene Corp.’s ABRAXANE (one manufacturer), Esai Inc.’s HEXALEN, Roche Holding AG’s (Roche) XELODA, Roche/Genentech, Inc.’s AVASTIN (four manufacturers), Baxter Healthcare’s CYTOXAN and LFEX, Etoposide (10 manufacturers), Eli Lilly and Company’s GEMZAR (15 manufacturers) and ALIMTA (14 manufacturers), Pfizer Inc.’s CAMPTOSAR (19 manufacturers), Janssen Pharmaceutical’s DOXIL (one manufacturer), Aspen Pharmacare’s ALKERAN, Meitheal Pharmaceuticals’s TOPOTECAN, Laboratoires Pierre Fabre’s NAVELBINE (four manufacturers), GSK’s ZEJULA, AstraZeneca’s LYNPARZA, and Clovis Oncology’s RUBRACA. ● Product candidates in registration trials or later development for PRROC include: ○ Nemvaleukin alfa, an engineered interleukin-2, by Mural Oncology; ○ Relacorilant, an anti-glucocorticoid, by Corcept Therapeutics Inc.; and ○ Luveltamab tazevibulin, an anti-folate receptor alpha (FolRα) antibody drug conjugate (ADC), by Sutro Biopharma.
In August 2019, SillaJen announced the discontinuation of its Phase 3 PHOCUS trial of Pexa-Vec in advanced liver cancer for futility. 16 PRROC We are also aware of other companies either marketing or focused on developing competing therapies for the treatment of ovarian cancer, including PRROC: ● Currently marketed products for ovarian cancer include generic products cisplatin (manufactured by 7 companies), carboplatin (manufactured by 12 companies), melphalan (manufactured by 17 companies) topotecan hydrochloride (8 manufacturers) and paclitaxel (manufactured by 11 companies), along with the following brand products (and generic manufacturers): Abbvie’s ELAHERE, Sanofi-Aventis’s TAXOTERE (16 manufacturers), Celgene Corp.’s ABRAXANE (one manufacturer), Esai Inc.’s HEXALEN, Roche Holding AG’s (Roche) XELODA, Roche/Genentech, Inc.’s AVASTIN (5 manufacturers), Baxter Healthcare’s CYTOXAN and LFEX, Etoposide (6 manufacturers), Eli Lilly and Company’s GEMZAR (14 manufacturers) and ALIMTA (18 manufacturers), Pfizer Inc.’s CAMPTOSAR (16 manufacturers), Baxter International’s DOXIL (8 manufacturer), Laboratoires Pierre Fabre’s NAVELBINE (four manufacturers), GSK’s ZEJULA, AstraZeneca’s LYNPARZA, and Pharma& and Tolmar’s RUBRACA. ● Product candidates in registration trials or later development for PRROC include: ○ Relacorilant, an anti-glucocorticoid, by Corcept Therapeutics Inc.; ○ Raludotatug deruxtecan, a CDH6 directed DXd ADC, developed by Daichii Sankyo; ○ Anlotinib, a receptor tyrosine kinase inhibitor, developed by Advenchen Laboratories; and ○ Afureserib, an ATP-competitive AKT inhibitor, developed by Laekna Therapeutics.
We are aware of a number of companies developing competing therapies for the treatment of cancer which generally fall into the following treatment groups: ● Oncolytic viral immunotherapies, including Amgen’s IMLYGIC (talimogene laherparepvec), the only FDA-approved oncolytic immunotherapy, which is approved for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery and is in development for several other indications, and other oncolytic viruses in development by companies such as AstraZeneca PLC (AstraZeneca), Boehringer Ingelheim, CG Oncology, Inc., Candel Therapeutics, Inc., Daiichi Sankyo Company, Limited, DNAtrix Inc., Imugene Limited, Johnson & Johnson, Merck & Co., Inc.
We are aware of a number of companies developing competing therapies for the treatment of cancer which generally fall into the following treatment groups: ● Oncolytic viral immunotherapies, including Amgen’s IMLYGIC (talimogene laherparepvec), the only FDA-approved oncolytic immunotherapy, which is approved for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery and is in development for several other indications and Daiichi Sankyo Company, Limited’s DELYTACT (teserpaturev/G47∆), which received conditional and time-limited marketing approval in Japan as a regenerative medical product for treatment of malignant glioma.
The trial is an open-label, randomized control design (2:1 randomization), enrolling patients who are platinum resistant/refractory by standard definitions and received their last platinum within 24 months from enrollment.
The trial is an open-label, randomized control design (2:1 randomization), enrolling patients who are platinum resistant/refractory by standard definitions and received a minimum of 3 prior lines of therapy.
Small Cell Lung Cancer (SCLC) We co-sponsor with Newsoara an ongoing Phase 1b/2 clinical trial of Olvi-Vec in patients with recurrent small cell lung cancer that we initiated in China in April 2023.
Newsoara is generally obligated under our collaboration agreement to fund this trial. Small Cell Lung Cancer We co-sponsor with Newsoara an ongoing Phase 1b/2 clinical trial of Olvi-Vec in patients with recurrent SCLC in China.
The EU GDPR, UK GDPR, and CCPA are examples of the increasingly stringent and evolving regulatory frameworks related to personal information processing that may increase our compliance obligations and exposure for any noncompliance.
In addition, numerous US states have enacted comprehensive data privacy laws, and similar laws are being considered at the federal, state, and local levels. The EU GDPR, UK GDPR, and CCPA are examples of the increasingly stringent and evolving regulatory frameworks related to personal information processing that may increase our compliance obligations and exposure for any noncompliance.
To help reduce the increased risk of the introduction of adventitious agents, the Public Health Service Act (PHSA) emphasizes the importance of manufacturing controls for products whose attributes cannot be precisely defined.
Manufacturers are required to comply with applicable requirements in the cGMP regulations, including quality control and quality assurance and maintenance of records and documentation. To help reduce the increased risk of the introduction of adventitious agents, the Public Health Service Act (PHSA) emphasizes the importance of manufacturing controls for products whose attributes cannot be precisely defined.
Furthermore, U.S. federal and state consumer protection laws may require us to publish statements that accurately and fairly describe how we handle personal information and choices individuals may have about the way we handle their personal information. 28 See the section titled “Risk Factors – We are subject to stringent and evolving U.S. and foreign laws, regulations, and rules, contractual obligations, policies and other obligations related to data privacy and security.
Furthermore, U.S. federal and state consumer protection laws require us to publish statements that accurately and fairly describe how we handle personal information and choices individuals may have about the way we handle their personal information.
We have generated an extensive portfolio of oncolytic vaccinia immunotherapy clinical candidates, of which, Olvi-Vec is the furthest along in clinical development. In addition to Olvi-Vec, we have over 500 different versions of the VACV armed with greater than 110 transgenes, having a variety of engineered attributes, including immune modulatory and tumor cell killing properties.
In addition to Olvi-Vec, we have over 500 different versions of the VACV armed with greater than 110 transgenes, having a variety of engineered attributes, including immune modulatory and tumor cell killing properties. 3 Our oncolytic immunotherapy product candidates are intended to selectively kill tumor cells and induce a robust immune response against a patient’s tumor neoantigens.
Post-Approval Requirements After approval, there also are continuing annual program user fee requirements for approved products, excluding, under certain circumstances, orphan products. Additionally, products approved under accelerated approval regulations may require a “confirmatory” study which is designed to verify the clinical benefits of the product. FDA may also require other post approval commitments as a condition of approval.
Additionally, products approved under accelerated approval regulations may require a “confirmatory” study which is designed to verify the clinical benefits of the product. FDA may also require other post approval commitments as a condition of approval. Rigorous and extensive FDA regulation of biological products continues after approval, particularly with respect to cGMP requirements.
While march-in rights have not previously been exercised, it is uncertain if that will continue under the new framework. 27 At the state level, individual states in the United States have increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
Congress may introduce and ultimately pass health care related legislation that could impact the drug approval process and make changes to the Medicare Drug Price Negotiation Program created under the IRA. 31 At the state level, individual states in the United States have increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
The trial was designed to address a broad and underserved pool of patients and the inclusion criteria allows patients to enroll regardless of (i) tumor biomarkers, (ii) platinum refractory tumors, or (iii) number of prior lines of treatments (i.e., no cap on previous treatments) The experimental arm patients will receive a single cycle (two doses) of Olvi-Vec administered intraperitoneally and, approximately four weeks later, a regimen of a platinum-based doublet plus bevacizumab followed by maintenance therapy.
The trial is designed to address a broad and underserved pool of patients and the inclusion criteria allows patients to enroll regardless of (i) tumor biomarkers, (ii) platinum refractory tumors or (iii) the maximum number of prior lines of treatments (i.e., no cap on previous treatments).
Some third-party payors also require pre-approval of coverage for new or innovative devices or therapies before they will reimburse healthcare providers that use such therapies.
Some third-party payors also require pre-approval of coverage for new or innovative devices or therapies before they will reimburse healthcare providers that use such therapies. We expect that these initiatives, as well as other healthcare reform measures that may be adopted in the future, particularly in light of the recent U.S.
Additionally, 20% of patients were long-term survivors, which is generally regarded as a hallmark of clinically beneficial immunotherapies. 8 Non-Small Cell Lung Cancer (NSCLC) We selected recurrent NSCLC as our first registration-path indication for intravenous delivery of Olvi-Vec-primed immunochemotherapy because of the promising preclinical and clinical data generated in patients with lung disease (primary or metastatic) in our prior clinical trials.
Systemic Administration We selected recurrent lung cancers (recurrent SCLC and recurrent NSCLC) as our initial registration-path indications for intravenous delivery of Olvi-Vec-primed immunochemotherapy because of the promising preclinical and clinical data generated in patients with lung disease (primary or metastatic) in our prior clinical trials.
Further, the IRA imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation. These provisions take effect progressively starting in fiscal year 2023.
Further, the IRA imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation. These provisions began to take effect progressively in fiscal year 2023. On August 15, 2024, HHS announced the agreed-upon price of the first ten drugs that were subject to price negotiations, although the Program is currently subject to legal challenges.
Historically, the expected ORR per RECIST 1.1 would be 6 With 13 objective responders per RECIST 1.1 out of 24 evaluable patients, the trial results exceeded the pre-defined threshold of 43%, and after our discussions with the U.S. Food and Drug Administration (FDA), supported moving into a Phase 3 trial.
Historically, the expected ORR per RECIST 1.1 would be With 13 objective responders per RECIST 1.1 out of 24 evaluable patients, the trial results exceeded the pre-defined threshold of 43%, and after our discussions with the FDA, supported moving into a Phase 3 trial. 6 In the following graphic, we show the results of three exemplary heavily pre-treated platinum-refractory (i.e., progression while on last platinum) patients, presenting at time of enrollment with progressive disease and projected short life expectancy.
Potential competitors also include academic institutions, government agencies, and other public and private research organizations that conduct research, seek patent protection, and establish collaborative arrangements for research, development, manufacturing, and commercialization of NSCLC therapies. 16 GOVERNMENT REGULATION AND PRODUCT APPROVAL In the United States, the FDA regulates biological products under the Federal Food, Drug, and Cosmetic Act (FDCA), the Public Health Service Act (PHSA), and regulations and guidance documents implementing these laws.
GOVERNMENT REGULATION AND PRODUCT APPROVAL In the United States, the FDA regulates biological products under the Federal Food, Drug, and Cosmetic Act (FDCA), the Public Health Service Act (PHSA), and regulations and guidance documents implementing these laws.
The joint venture is governed by an Amended and Restated Limited Liability Company Agreement entered into in June 2021 which provides each of us and TVAX with 50% ownership interests, identical voting and management rights and responsibilities, equal representation on the governing four-member management committee, and equal sharing of profits and losses of V2ACT.
The joint venture is governed by an Amended and Restated Limited Liability Company Agreement entered into in June 2021 which provides each of us and TVAX with 50% ownership interests, identical voting and management rights and responsibilities, equal representation on the governing four-member management committee, and equal sharing of profits and losses of V2ACT. 12 In June 2021, we entered into a License Agreement with V2ACT (V2ACT License), pursuant to which we granted V2ACT a worldwide, non-exclusive, fully paid, royalty free license for our proprietary oncolytic virus (Licensed Virus(es)) to research, develop and commercialize any product, procedure or method for the treatment of cancer that combines (a) Licensed Virus(es), and (b) autologous or allogeneic cancer-specific T lymphocytes (T-Cell Therapeutic(s)) for the diagnosis, prevention and treatment of cancer in humans (Products).
In the second half of 2023, we began regulatory study start-up in the United States of a Phase 2, open-label, randomized, and controlled clinical trial of Olvi-Vec in patients with recurrent NSCLC (after progression on a front-line maintenance Immune Checkpoint Inhibitor-based regimen) (VIRO-25). We expect to begin enrolling patients in VIRO-25 in the first half of 2024.
Each of these trials is designed to enroll and re-challenge patients who have failed prior platinum therapy (and, in the case of the NSCLC re-challenge patients who also failed a prior immune checkpoint inhibitor). 8 Non-Small Cell Lung Cancer In the fourth quarter of 2024, we initiated enrollment in the United States in our Phase 2 VIRO-25 trial in the United States, open-label, randomized, and controlled clinical trial of Olvi-Vec in patients with recurrent NSCLC (after progression on a front-line maintenance Immune Checkpoint Inhibitor-based regimen).
On September 26, 2021, we and V2ACT entered into a First Amendment to the License Agreement, whereby the territory was defined as worldwide except for Greater China (i.e., Mainland China, Hong Kong, Macau and Taiwan). 11 In June 2021, TVAX entered into a License Agreement with V2ACT (TVAX License), pursuant to which TVAX granted V2ACT a worldwide, non-exclusive, fully paid, royalty free license for its proprietary T-Cell Therapeutics (Licensed T-Cell Therapeutic(s)) to research, develop and commercialize any product, procedure or method for the treatment of cancer that combines (a) any virus-based cancer therapeutics, and (b) Licensed T-Cell Therapeutic(s) for the diagnosis, prevention and treatment of cancer in humans (TVAX Products).
On September 26, 2021, we and V2ACT entered into a First Amendment to the License Agreement, whereby the territory was defined as worldwide except for Greater China (i.e., Mainland China, Hong Kong, Macau and Taiwan).
The median overall survival of patients exceeded the historical survival rates of earlier lines of therapy.
The median overall survival of patients exceeded the historical survival rates of earlier lines of therapy. Additionally, 20% of patients were long-term survivors, which is generally regarded as a hallmark of clinically beneficial immunotherapies.
The scientific rationale for V2ACT Immunotherapy is that adoptive transfer of cancer neoantigen- specific effector T cells has proven to be an effective treatment for multiple cancers. Reducing cancer tissue associated immunosuppression could increase the anti-cancer effects of adoptively transferred neoantigen- specific effector T cells.
Department of Agriculture Center for Veterinary Biologics determined that data from the company’s ECI-OSA-04 pivotal combined safety and efficacy study demonstrated a reasonable expectation of efficacy, a critical milestone in the licensure pathway. The scientific rationale for V2ACT Immunotherapy is that adoptive transfer of cancer neoantigen- specific effector T cells has proven to be an effective treatment for multiple cancers.
VIRO-25 is expected to become a multi-regional clinical trial with Newsoara adding clinical trial sites and patients in China. Newsoara is generally obligated under our collaboration agreement to fund this trial.
A readout of interim results of this trial is expected in the second half of 2025. Subject to regulatory authorization from the Chinese Centre for Drug Evaluation, we anticipate Newsoara initiating our Phase 2 VIRO-25 trial. We also expect the trial to become a multi-regional clinical trial with Newsoara adding clinical trial sites and patients in China.
Potential indications include appendiceal, colorectal and gastric cancers, other gynecologic malignancies, and peritoneal mesothelioma. TERMS OF CERTAIN LICENSE AGREEMENTS Newsoara Biopharma Co., Ltd.
TERMS OF CERTAIN LICENSE AGREEMENTS Newsoara Biopharma Co., Ltd.
The active comparator arm patients will receive a regimen of single agent chemotherapy with optional platinum, plus bevacizumab followed by maintenance therapy. We recently amended the protocol with respect to certain design criteria and expect to begin enrolling patients upon receipt of institutional review board (IRB) approvals. The enrollment will be approximately 186 patients.
The experimental arm patients will receive a single cycle (two doses) of Olvi-Vec administered intraperitoneally and, approximately four weeks later, a regimen of a platinum-based doublet plus bevacizumab followed by maintenance therapy. The active comparator arm patients will receive a regimen of single agent chemotherapy with optional platinum, plus bevacizumab followed by maintenance therapy. After discussions with the U.S.
(Merck), Oncolytics Biotech Inc., Otsuka Holdings Co. Ltd., Pfizer Inc., PsiOxus Therapeutics, Ltd., Regeneron Pharmaceuticals, Inc., Replimune Group, Inc., SillaJen, Inc.
Other oncolytic viral immunotherapies in development include those by companies such as AstraZeneca PLC (AstraZeneca), Boehringer Ingelheim, CG Oncology, Inc., Candel Therapeutics, Inc., Imugene Limited, Johnson & Johnson, Merck & Co., Inc. (Merck), Oncolytics Biotech Inc., Otsuka Holdings Co. Ltd., Pfizer Inc., Akamis Bio., Regeneron Pharmaceuticals, Inc., Replimune Group, Inc., SillaJen, Inc.
A readout of interim results in the Phase 1b portion of this trial is expected in the second half of 2024, after which we will move into the Phase 2 portion of the trial. The following graphic summarizes the study design for the Phase 1b/2 trial.
The following graphic summarizes the study design for the Phase 1b/2 trial. 9 A readout of interim results in the Phase 1b portion of this trial was disclosed in the first quarter of 2025. Data are supportive of Olvi-Vec being a platinum resensitizing agent beyond ovarian cancer and underscore the current clinical development strategy.
Removed
Our oncolytic immunotherapy product candidates are intended to selectively kill tumor cells and induce a robust immune response against a patient’s tumor neoantigens. Importantly, these product candidates are “off-the-shelf” personalized immunotherapies.
Added
We have generated an extensive portfolio of oncolytic vaccinia immunotherapy clinical candidates, of which, Olvi-Vec is the furthest along in clinical development.
Removed
The median PFS of the patients’ immediately preceding line of therapy was approximately 4.5 months versus 11.0 months achieved after treatment with Olvi-Vec. In the following graphic, we show the results of three exemplary heavily pre-treated platinum-refractory (i.e., progression while on last platinum) patients, presenting at time of enrollment with progressive disease and projected short life expectancy.
Added
Those clinical trials have yielded data that has informed our current and future clinical strategy and trial design involving multiple indications and methods of delivery.
Removed
Rigorous and extensive FDA regulation of biological products continues after approval, particularly with respect to cGMP requirements. Manufacturers are required to comply with applicable requirements in the cGMP regulations, including quality control and quality assurance and maintenance of records and documentation.
Added
Food and Drug Administration (the FDA), we amended the protocol with respect to certain design criteria including removal of an eligibility criterion requiring patients to have received their last platinum within 24 months of enrollment. We anticipate enrolling patients eligible under the amended protocol upon receipt of institutional review board (IRB) approvals.
Removed
The ACA, among other things, increased the minimum level of Medicaid rebates payable by manufacturers of brand name drugs; required collection of rebates for drugs paid by Medicaid managed care organizations; required manufacturers to participate in a coverage gap discount program, under which they must agree to offer point-of-sale discounts (increased to 70 percent, effective as of January 1, 2019) off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D; imposed a non-deductible annual fee on pharmaceutical manufacturers or importers who sell certain “branded prescription drugs” to specified federal government programs, implemented a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted, or injected expanded the types of entities eligible for the 340B drug discount program; expanded eligibility criteria for Medicaid programs; created a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research; and established a Center for Medicare Innovation at CMS to test innovative payment and service delivery models to lower Medicare and Medicaid spending, potentially including prescription drug spending. 26 There have been executive, judicial and Congressional challenges to certain aspects of the ACA.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

266 edited+61 added−50 removed564 unchanged
Biggest changeBecause the design and outcome of our ongoing, anticipated and any future clinical trials is highly uncertain, we cannot reasonably estimate the actual amounts necessary to successfully complete the development and commercialization of any product candidate we develop. 32 Our future capital requirements depend on many factors, including: ● the scope, progress, results and costs of researching and developing Olvi-Vec and our other product candidates and programs, and of conducting preclinical studies and clinical trials; ● the timing of, and the costs involved in, obtaining marketing approvals for Olvi-Vec and future product candidates we develop if clinical trials are successful; ● the success of any future collaborations; ● the cost of commercialization activities for any approved product, including marketing, sales and distribution costs; ● the cost and timing of establishing, equipping, and operating our current and planned manufacturing activities; ● the cost of manufacturing Olvi-Vec and future product candidates for clinical trials in preparation for marketing approval and commercialization; ● our ability to establish and maintain strategic licensing or other arrangements and the financial terms of such agreements; ● the cost, timing and outcome of seeking FDA and any other regulatory approvals for any future product candidates; ● the costs involved in preparing, filing, prosecuting, maintaining, expanding, defending and enforcing patent claims, including litigation costs and the outcome of such litigation; ● our ability to establish and maintain healthcare coverage and adequate reimbursement for our future products, if any; ● the timing, receipt, and amount of sales of, or royalties on, our future products, if any; ● the emergence of competing cancer therapies and other adverse market developments; ● our efforts to enhance operational systems and our ability to attract, hire and retain qualified personnel, including personnel to support the development of our product candidates; ● the costs associated with being a public company; ● our need and ability to retain key management and hire scientific, technical, medical and business personnel; ● the costs associated with expanding our facilities or building out our laboratory space; and ● the impact of geopolitical and macroeconomic events, including future bank failures, increased geopolitical tensions between the United States and China, the Russia/Ukraine conflict, the war in the Middle East and global pandemics on U.S. and global economic conditions. 33 Two investors from our private placements (the Private Placements) were contractually obligated to fund $30.0 million on or before November 15, 2023, of which we have received $6.0 million to date.
Biggest changeFood and Drug Administration (FDA) and any other regulatory approvals for any future product candidates; ● the costs involved in preparing, filing, prosecuting, maintaining, expanding, defending and enforcing patent claims, including litigation costs and the outcome of such litigation; ● our ability to establish and maintain healthcare coverage and adequate reimbursement for our future products, if any; ● the timing, receipt, and amount of sales of, or royalties on, our future products, if any; ● the emergence of competing cancer therapies and other adverse market developments; ● our efforts to enhance operational systems and our ability to attract, hire and retain qualified personnel, including personnel to support the development of our product candidates; ● the costs associated with being a public company; 37 ● our need and ability to retain key management and hire scientific, technical, medical and business personnel; ● the costs associated with expanding our facilities or building out our laboratory space; and ● the impact of geopolitical and macroeconomic events, including future bank failures, tariffs, increased geopolitical tensions between the United States and China, the Russia/Ukraine conflict, the conflicts in the Middle East and global pandemics on U.S. and global economic conditions.
If we elect to fund and undertake development or commercialization activities on our own, we may need to obtain additional expertise and additional capital, which may not be available to us on acceptable terms, or at all.
If we elect to fund and undertake development or commercialization activities on our own, we may need to obtain additional expertise and additional capital, which may not be available to us on acceptable terms, or at all.
Europe and other jurisdictions have enacted laws requiring data to be localized or limiting the transfer of personal data to other countries. In particular, the EEA and the UK have significantly restricted the transfer of personal data to the United States and other countries whose privacy laws it believes are generally inadequate.
Europe and other jurisdictions have enacted laws requiring data to be localized or limiting the transfer of personal data to other countries. In particular, the EEA and the UK have significantly restricted the transfer of personal data to the United States and other countries whose privacy laws it generally believes are inadequate.
Data Privacy Framework and the UK extension thereto (which allows for transfers for relevant U.S.-based organizations who self-certify compliance and participate in the Framework), these mechanisms are subject to legal challenges, and there is no assurance that we can satisfy or rely on these measures to lawfully transfer personal data to the United States.
Data Privacy Framework and the UK extension thereto (which allows for transfers to relevant U.S.-based organizations who self-certify compliance and participate in the Framework), these mechanisms are subject to legal challenges, and there is no assurance that we can satisfy or rely on these measures to lawfully transfer personal data to the United States.
Security incidents and attendant consequences may cause customers to stop or prevent customers from using our products, deter new customers from using our products, and negatively impact our ability to grow and operate our business.
Security incidents and attendant consequences may prevent or cause customers to stop using our products, deter new customers from using our products, and negatively impact our ability to grow and operate our business.
Our current collaborations with TVAX and Newsoara, and potential future collaborations we might enter into for Olvi-Vec or our other product candidates, may pose a number of risks, including the following: ● collaborators may not perform their obligations as expected; ● collaborators may not pursue development and commercialization of product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create competing priorities; ● collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; ● collaborators could fail to make timely regulatory submissions for a product candidate; ● collaborators may not comply with all applicable regulatory requirements or may fail to report safety data in accordance with all applicable regulatory requirements, which could subject them or us to regulatory enforcement actions; ● collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; ● product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; ● a collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of such product candidate or product; ● disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred course of development, might cause delays or termination of the research, development or commercialization of product candidates, might lead to additional responsibilities for us with respect to product candidates, or might result in litigation or arbitration, any of which would be time consuming and expensive; ● collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation; and ● collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability.
Our current collaborations with TVAX and Newsoara, and potential future collaborations we might enter into for Olvi-Vec or our other product candidates, may pose a number of risks, including the following: ● collaborators may not perform their obligations as expected; ● collaborators may not pursue development and commercialization of product candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create competing priorities; 72 ● collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; ● collaborators could fail to make timely regulatory submissions for a product candidate; ● collaborators may not comply with all applicable regulatory requirements or may fail to report safety data in accordance with all applicable regulatory requirements, which could subject them or us to regulatory enforcement actions; ● collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; ● product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; ● a collaborator with marketing and distribution rights to one or more of our product candidates that achieve regulatory approval may not commit sufficient resources to the marketing and distribution of such product candidate or product; ● disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred course of development, might cause delays or termination of the research, development or commercialization of product candidates, might lead to additional responsibilities for us with respect to product candidates, or might result in litigation or arbitration, any of which would be time consuming and expensive; ● collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation; and ● collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability.
If we, or our collaboration partners, are successful in obtaining marketing approval from applicable regulatory authorities for Olvi-Vec or any other product candidate, our ability to generate revenues from any such products will depend on our success in: ● launching commercial sales of such products, whether alone or in collaboration with others; ● receiving approved labels with claims that are necessary or desirable for successful marketing, and that do not contain safety or other limitations that would impede our ability to market such products; ● creating market demand for such products through marketing, sales and promotion activities; ● hiring, training, and deploying a sales force or contracting with third parties to commercialize such products in the United States; ● creating partnerships with, or offering licenses to, third parties to promote and sell such products in foreign markets where we receive marketing approval; ● manufacturing such products in sufficient quantities and at acceptable quality and cost to meet commercial demand at launch and thereafter; ● establishing and maintaining agreements with wholesalers, distributors, and group purchasing organizations on commercially reasonable terms; ● maintaining patent and trade secret protection and regulatory exclusivity for such products; ● achieving market acceptance of such products by patients, the medical community, and third-party payors; ● achieving coverage and adequate reimbursement from third-party payors for such products; ● achieving patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement from third-party payors; ● competing effectively with other therapies; and ● maintaining a continued acceptable safety profile of such products following launch.
If we, or our collaboration partners, are successful in obtaining marketing approval from applicable regulatory authorities for Olvi-Vec or any other product candidate, our ability to generate revenues from any such products will depend on our success in: ● launching commercial sales of such products, whether alone or in collaboration with others; ● receiving approved labels with claims that are necessary or desirable for successful marketing, and that do not contain safety or other limitations that would impede our ability to market such products; ● creating market demand for such products through marketing, sales and promotion activities; ● hiring, training, and deploying a sales force or contracting with third parties to commercialize such products in the United States; ● creating partnerships with, or offering licenses to, third parties to promote and sell such products in foreign markets where we receive marketing approval; ● manufacturing such products in sufficient quantities and at acceptable quality and cost to meet commercial demand at launch and thereafter; ● establishing and maintaining agreements with wholesalers, distributors, and group purchasing organizations on commercially reasonable terms; 75 ● maintaining patent and trade secret protection and regulatory exclusivity for such products; ● achieving market acceptance of such products by patients, the medical community, and third-party payors; ● achieving coverage and adequate reimbursement from third-party payors for such products; ● achieving patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement from third-party payors; ● competing effectively with other therapies; and ● maintaining a continued acceptable safety profile of such products following launch.
Federal Food, Drug and Cosmetic Act, which prohibits, among other things, the adulteration or misbranding of drugs, biological products and medical devices. ● The federal physician payment transparency requirements, sometimes referred to as the Physician Payments Sunshine Act, created under the ACA and its implementing regulations, which require certain manufacturers of drugs, devices, biological products and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to the Centers for Medicare & Medicaid Services (CMS) information related to payments or other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members. ● Analogous state and foreign anti-kickback and false claims laws that may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state and local laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; state laws that require the reporting of information related to drug pricing; and state and local laws requiring the registration of pharmaceutical sales representatives. 86 If we or our operations are found to be in violation of any federal or state healthcare law, or any other governmental laws or regulations that apply to us, we may be subject to penalties, including significant civil, criminal, and administrative penalties, damages, monetary fines, disgorgement, imprisonment, suspension and debarment from government contracts, and refusal of orders under existing government contracts, exclusion from participation in U.S. federal or state health care programs, additional reporting requirements and/or oversight if we become subject to corporate integrity agreements or similar agreement to resolve allegations of non-compliance, contractual damages, reputational harm, diminished profits and future earnings, and the curtailment or restructuring of our operations, any of which could materially adversely affect our ability to operate our business and our financial results.
Federal Food, Drug and Cosmetic Act, which prohibits, among other things, the adulteration or misbranding of drugs, biological products and medical devices. ● The federal physician payment transparency requirements, sometimes referred to as the Physician Payments Sunshine Act, created under the ACA and its implementing regulations, which require certain manufacturers of drugs, devices, biological products and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to the Centers for Medicare & Medicaid Services (CMS) information related to payments or other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members. ● Analogous state and foreign anti-kickback and false claims laws that may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state and local laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; state laws that require the reporting of information related to drug pricing; and state and local laws requiring the registration of pharmaceutical sales representatives. 95 If we or our operations are found to be in violation of any federal or state healthcare law, or any other governmental laws or regulations that apply to us, we may be subject to penalties, including significant civil, criminal, and administrative penalties, damages, monetary fines, disgorgement, imprisonment, suspension and debarment from government contracts, and refusal of orders under existing government contracts, exclusion from participation in U.S. federal or state health care programs, additional reporting requirements and/or oversight if we become subject to corporate integrity agreements or similar agreement to resolve allegations of non-compliance, contractual damages, reputational harm, diminished profits and future earnings, and the curtailment or restructuring of our operations, any of which could materially adversely affect our ability to operate our business and our financial results.
In addition, if one or more of our product candidates receives marketing approval, and we or others later identify undesirable side effects caused by such products, a number of potentially significant negative consequences could result, including: ● regulatory authorities may withdraw approvals of such product; 49 ● regulatory authorities may require additional warnings on the label; ● we may be required to create a medication guide outlining the risks of such side effects for distribution to patients; ● we may be forced to suspend marketing of that product, or decide to remove the product from the marketplace; ● we may be required to change the way the product is administered; ● we could be subject to fines, injunctions, or the imposition of criminal or civil penalties; ● we could be sued and held liable for harm caused to patients; and ● the product may become less competitive, and our reputation may suffer.
In addition, if one or more of our product candidates receives marketing approval, and we or others later identify undesirable side effects caused by such products, a number of potentially significant negative consequences could result, including: ● regulatory authorities may withdraw approvals of such product; ● regulatory authorities may require additional warnings on the label; ● we may be required to create a medication guide outlining the risks of such side effects for distribution to patients; ● we may be forced to suspend marketing of that product, or decide to remove the product from the marketplace; ● we may be required to change the way the product is administered; ● we could be subject to fines, injunctions, or the imposition of criminal or civil penalties; ● we could be sued and held liable for harm caused to patients; and ● the product may become less competitive, and our reputation may suffer.
We also may experience numerous unforeseen events during, or as a result of, any ongoing or future clinical trials that we could conduct that could delay or prevent our ability to receive marketing approval or commercialize Olvi-Vec or any future product candidates, including: ● delays or failures, which may result in clinical site closures, delays to patient enrollment, patients withdrawing prior to receiving treatment (e.g., catheter implantation failure), patients discontinuing their treatment or follow-up visits or changes to trial protocols; ● regulators or institutional review boards (IRBs), may not authorize us or our investigators to commence a clinical trial, conduct a clinical trial at a prospective trial site, or amend trial protocols, or may require that we modify or amend our clinical trial protocols; ● we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites and/or CROs; ● clinical trials of our product candidates may produce negative or inconclusive results, or our studies may fail to reach the necessary level of statistical significance, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs; ● the unsuccessful implantation of catheters used to deliver Olvi-Vec; ● the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or participants may drop out of these clinical trials or be lost to follow-up at a higher rate than we anticipate, or may elect to participate in alternative clinical trials sponsored by our competitors with product candidates that treat the same indications as our product candidates; 41 ● third-party contractors may fail to comply with regulatory requirements or the clinical trial protocol, or meet their contractual obligations to us in a timely manner, or at all, or we may be required to engage in additional clinical trial site monitoring; ● manufacturing delays; ● we, regulators, or IRBs may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, emergent drug-drug interactions between Olvi-Vec and any of the other therapeutic agents given to the clinical trial subjects or other unexpected characteristics of the product candidate, or due to findings of undesirable effects caused by a biologically, chemically or mechanistically similar therapeutic or therapeutic candidate, or flaws in the design of the trial; ● changes could be adopted in marketing approval policies during the development period, rendering our data insufficient to obtain marketing approval; ● statutes or regulations could be amended, or new ones could be adopted; ● changes could be adopted in the regulatory review process for submitted product applications; ● the cost of clinical trials of our product candidates may be greater than we anticipate, or we may have insufficient funds for a clinical trial or product manufacture or to pay the substantial user fees required by the FDA upon the submission of a BLA or equivalent authorizations from comparable foreign regulatory authorities; ● the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; 42 ● the FDA or comparable foreign regulatory authorities may fail to approve the existing or future manufacturing processes or facilities of our company or of third-party manufacturers with which we contract for clinical and commercial supplies; ● we may decide, or regulators may require us, to conduct or gather, as applicable, additional clinical trials, analyses, reports, data, or preclinical trials, or we may abandon product development programs; ● we may fail to reach an agreement with regulators or IRBs regarding the scope, design, or implementation of our clinical trials, and the FDA or comparable foreign regulatory authorities may require changes to our study designs that make further study impractical or not financially prudent; ● regulators may ultimately disagree with the design or our conduct of our preclinical studies or clinical trials, finding that they do not support product candidate approval; ● we may have delays in adding new investigators or clinical trial sites, or we may experience a withdrawal of clinical trial sites; ● patients that enroll in our studies may misrepresent their eligibility or may otherwise not comply with the clinical trial protocol, resulting in the need to drop the patients from the study or clinical trial, increase the needed enrollment size for the clinical trial or extend its duration; ● there may be regulatory questions or disagreements regarding interpretations of data and results, or new information may emerge regarding our product candidates; ● the FDA or comparable foreign regulatory authorities may disagree with our trial design, including endpoints, or our interpretation of data from preclinical studies and clinical trials or find that a product candidate’s benefits do not outweigh its safety risks; ● the FDA or comparable foreign regulatory authorities may not accept data from studies with clinical trial sites in foreign countries; ● the FDA or comparable foreign regulatory authorities may disagree with our intended indications; ● the FDA or comparable foreign regulatory authorities may fail to approve or subsequently find fault with the manufacturing processes or our manufacturing facilities for clinical and future commercial supplies; ● the data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of the FDA or comparable foreign regulatory authorities to support the submission of a BLA or other comparable submission in foreign jurisdictions or to obtain regulatory approval in the United States or elsewhere; ● the FDA or comparable foreign regulatory authorities may take longer than we anticipate to make a decision on our product candidates; and ● we may not be able to demonstrate that a product candidate provides an advantage over current standards of care or current or future competitive therapies in development, including, for example, due to a longer-and/or-higher-than-expected response rate determination in the active comparator group or a shorter-and/or-lower-than-expected response rate determination in the experimental drug group.
We also may experience numerous unforeseen events during, or as a result of, any ongoing or future clinical trials that we could conduct that could delay or prevent our ability to receive marketing approval or commercialize Olvi-Vec or any future product candidates, including: ● delays or failures, which may result in clinical site closures, delays to patient enrollment, patients withdrawing prior to receiving treatment (e.g., catheter implantation failure), patients discontinuing their treatment or follow-up visits or changes to trial protocols; ● regulators or institutional review boards (IRBs), may not authorize us or our investigators to commence a clinical trial, conduct a clinical trial at a prospective trial site, or amend trial protocols, or may require that we modify or amend our clinical trial protocols; ● we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites and/or CROs; ● clinical trials of our product candidates may produce negative or inconclusive results, or our studies may fail to reach the necessary level of statistical significance, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs; 45 ● the unsuccessful implantation of catheters used to deliver Olvi-Vec; ● the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or participants may drop out of these clinical trials or be lost to follow-up at a higher rate than we anticipate, or may elect to participate in alternative clinical trials sponsored by our competitors with product candidates that treat the same indications as our product candidates; ● third-party contractors may fail to comply with regulatory requirements or the clinical trial protocol, or meet their contractual obligations to us in a timely manner, or at all, or we may be required to engage in additional clinical trial site monitoring; ● manufacturing delays; ● we, regulators, or IRBs may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, emergent drug-drug interactions between Olvi-Vec and any of the other therapeutic agents given to the clinical trial subjects or other unexpected characteristics of the product candidate, or due to findings of undesirable effects caused by a biologically, chemically or mechanistically similar therapeutic or therapeutic candidate, or flaws in the design of the trial; ● changes could be adopted in marketing approval policies during the development period, rendering our data insufficient to obtain marketing approval; ● statutes or regulations could be amended, or new ones could be adopted; ● changes could be adopted in the regulatory review process for submitted product applications; ● the cost of clinical trials of our product candidates may be greater than we anticipate, or we may have insufficient funds for a clinical trial or product manufacture or to pay the substantial user fees required by the FDA upon the submission of a BLA or equivalent authorizations from comparable foreign regulatory authorities; ● the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; ● the FDA or comparable foreign regulatory authorities may fail to approve the existing or future manufacturing processes or facilities of our company or of third-party manufacturers with which we contract for clinical and commercial supplies; ● we may decide, or regulatory authorities may require us, to conduct or gather, as applicable, additional clinical trials, analyses, reports, data, or preclinical studies, or we may abandon product development programs; 46 ● we may fail to reach an agreement with regulatory authorities or IRBs regarding the scope, design, or implementation of our clinical trials, and the FDA or comparable foreign regulatory authorities may require changes to our study designs that make further study impractical or not financially prudent; ● Regulatory authorities may ultimately disagree with the design or our conduct of our preclinical studies or clinical trials, finding that they do not support product candidate approval; ● we may have delays in adding new investigators or clinical trial sites, or we may experience a withdrawal of clinical trial sites; ● patients that enroll in our studies may misrepresent their eligibility or may otherwise not comply with the clinical trial protocol, resulting in the need to drop the patients from the study or clinical trial, increase the needed enrollment size for the clinical trial or extend its duration; ● there may be regulatory questions or disagreements regarding interpretations of data and results, or new information may emerge regarding our product candidates; ● the FDA or comparable foreign regulatory authorities may disagree with our trial design, including endpoints, or our interpretation of data from preclinical studies and clinical trials or find that a product candidate’s benefits do not outweigh its safety risks; ● the FDA or comparable foreign regulatory authorities may not accept data from studies with clinical trial sites in foreign countries; ● the FDA or comparable foreign regulatory authorities may disagree with our intended indications; ● the FDA or comparable foreign regulatory authorities may fail to approve or subsequently find fault with the manufacturing processes or our manufacturing facilities for clinical and future commercial supplies; ● the data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of the FDA or comparable foreign regulatory authorities to support the submission of a BLA or other comparable submission in foreign jurisdictions or to obtain regulatory approval in the United States or elsewhere; ● the FDA or comparable foreign regulatory authorities may take longer than we anticipate to make a decision on our product candidates; and ● we may not be able to demonstrate that a product candidate provides an advantage over current standards of care or current or future competitive therapies in development, including, for example, due to a longer-and/or-higher-than-expected response rate determination in the active comparator group or a shorter-and/or-lower-than-expected response rate determination in the experimental drug group.
Regardless of merit or eventual outcome, liability claims may result in: ● loss of revenue from decreased demand for our products and/or product candidates; ● impairment of our business reputation or financial stability; ● costs of related litigation; ● substantial monetary awards to patients or other claimants; ● diversion of management attention; ● withdrawal of clinical trial participants and potential termination of clinical trial sites or entire clinical programs; ● the inability to commercialize our product candidates; ● significant negative media attention; ● decreases in our stock price; ● initiation of investigations and enforcement actions by regulators; and ● product recalls, withdrawals or labeling, marketing or promotional restrictions, including withdrawal of marketing approval.
Regardless of merit or eventual outcome, liability claims may result in: ● loss of revenue from decreased demand for our products and/or product candidates; 111 ● impairment of our business reputation or financial stability; ● costs of related litigation; ● substantial monetary awards to patients or other claimants; ● diversion of management attention; ● withdrawal of clinical trial participants and potential termination of clinical trial sites or entire clinical programs; ● the inability to commercialize our product candidates; ● significant negative media attention; ● decreases in our stock price; ● initiation of investigations and enforcement actions by regulators; and ● product recalls, withdrawals or labeling, marketing or promotional restrictions, including withdrawal of marketing approval.
Our ability to generate revenue and achieve profitability depends heavily on our success in achieving a number of goals, including: ● completing research regarding, and preclinical and clinical development of, product candidates and programs, including Olvi-Vec, and identifying and developing new product candidates; 34 ● obtaining marketing approvals for any product candidates for which we complete clinical trials; ● obtaining regulatory approval to use and sell products generated by our existing or future manufacturing processes for Olvi-Vec and future product candidates, including at our existing manufacturing facility and/or by establishing and maintaining supply and manufacturing relationships with third parties; ● launching and commercializing product candidates for which we obtain marketing approvals, either directly by establishing a sales force and marketing, medical affairs and distribution infrastructure or, alternatively, with a collaborator or distributor; ● establishing and maintaining healthcare coverage and adequate reimbursement for our future products, if any; ● obtaining market acceptance of product candidates that we develop as viable treatment options; ● addressing any competing technological and market developments; ● identifying, assessing, acquiring and developing new product candidates; ● negotiating favorable terms in any collaboration, licensing, or other arrangements into which we may enter and performing our obligations in such collaborations; ● maintaining, protecting, and expanding our portfolio of intellectual property rights, including patents, trade secrets, and know-how; and ● attracting, hiring, and retaining qualified personnel.
Our ability to generate revenue and achieve profitability depends heavily on our success in achieving a number of goals, including: ● completing research regarding, and preclinical and clinical development of, product candidates and programs, including Olvi-Vec, and identifying and developing new product candidates; 38 ● obtaining marketing approvals for any product candidates for which we complete clinical trials; ● obtaining regulatory approval to use and sell products generated by our existing or future manufacturing processes for Olvi-Vec and future product candidates, including at our existing manufacturing facility and/or by establishing and maintaining supply and manufacturing relationships with third parties; ● launching and commercializing product candidates for which we obtain marketing approvals, either directly by establishing a sales force and marketing, medical affairs and distribution infrastructure or, alternatively, with a collaborator or distributor; ● establishing and maintaining healthcare coverage and adequate reimbursement for our future products, if any; ● obtaining market acceptance of product candidates that we develop as viable treatment options; ● addressing any competing technological and market developments; ● identifying, assessing, acquiring and developing new product candidates; ● negotiating favorable terms in any collaboration, licensing, or other arrangements into which we may enter and performing our obligations in such collaborations; ● maintaining, protecting, and expanding our portfolio of intellectual property rights, including patents, trade secrets, and know-how; and ● attracting, hiring, and retaining qualified personnel.
The degree of market acceptance of any product for which we receive marketing approval will depend on a number of factors, including: ● the efficacy of our product, including in combination with other cancer therapies; ● the commercial success of any cancer therapies with which our product may be co-administered; ● the prevalence and severity of adverse events associated with our product or those products with which it is co-administered; ● the clinical indications for which our product is approved and the approved claims that we may make with respect to the product; ● limitations or warnings contained in the FDA-approved labeling of the product or the labeling approved by comparable foreign regulatory authorities, including potential limitations or warnings for our product that may be more restrictive than other competitive products; ● changes in the standard of care for the targeted indications for our product, which could reduce the marketing impact of any claims that we could make following FDA approval or approval by comparable foreign regulatory authorities, if obtained; ● the relative convenience and ease of administration of our product and any products with which it is co-administered; ● the cost of treatment compared with the economic and clinical benefit of alternative treatments or therapies; ● the availability of coverage and adequate reimbursement by third-party payors, such as private insurance companies and government healthcare programs, including Medicare and Medicaid; ● the ability to have our product placed on approved formularies; ● patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement from third-party payors; ● the price concessions required by third-party payors to obtain coverage and adequate reimbursement; ● the extent and strength of our marketing and distribution of our product; ● the safety, efficacy, and other potential advantages over, and availability of, alternative treatments already used or that may later be approved; ● distribution and use restrictions imposed by the FDA or comparable foreign regulatory authorities with respect to our product or to which we agree as part of a REMS or voluntary risk management plan; ● the timing of market introduction of our product, as well as competitive products; ● our ability to offer our product for sale at competitive prices; ● the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; ● the extent and strength of our raw material supplier and service provider support; ● the actions of companies that market any products with which our product is co-administered; ● the approval of other new products; ● adverse publicity about our product or any products with which it is co-administered, or favorable publicity about competitive products; and ● potential product liability claims. 73 The size of the potential market for our product candidates is difficult to estimate and, if any of our assumptions are inaccurate, the actual markets for our product candidates may be smaller than our estimates.
The degree of market acceptance of any product for which we receive marketing approval will depend on a number of factors, including: ● the efficacy of our product, including in combination with other cancer therapies; ● the commercial success of any cancer therapies with which our product may be co-administered; ● the prevalence and severity of adverse events associated with our product or those products with which it is co-administered; ● the clinical indications for which our product is approved and the approved claims that we may make with respect to the product; ● limitations or warnings contained in the FDA-approved labeling of the product or the labeling approved by comparable foreign regulatory authorities, including potential limitations or warnings for our product that may be more restrictive than other competitive products; ● changes in the standard of care for the targeted indications for our product, which could reduce the marketing impact of any claims that we could make following FDA approval or approval by comparable foreign regulatory authorities, if obtained; ● the relative convenience and ease of administration of our product and any products with which it is co-administered; ● the cost of treatment compared with the economic and clinical benefit of alternative treatments or therapies; 81 ● the availability of coverage and adequate reimbursement by third-party payors, such as private insurance companies and government healthcare programs, including Medicare and Medicaid; ● the ability to have our product placed on approved formularies; ● patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement from third-party payors; ● the price concessions required by third-party payors to obtain coverage and adequate reimbursement; ● the extent and strength of our marketing and distribution of our product; ● the safety, efficacy, and other potential advantages over, and availability of, alternative treatments already used or that may later be approved; ● distribution and use restrictions imposed by the FDA or comparable foreign regulatory authorities with respect to our product or to which we agree as part of a REMS or voluntary risk management plan; ● the timing of market introduction of our product, as well as competitive products; ● our ability to offer our product for sale at competitive prices; ● the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; ● the extent and strength of our raw material supplier and service provider support; ● the actions of companies that market any products with which our product is co-administered; ● the approval of other new products; ● adverse publicity about our product or any products with which it is co-administered, or favorable publicity about competitive products; and ● potential product liability claims. 82 The size of the potential market for our product candidates is difficult to estimate and, if any of our assumptions are inaccurate, the actual markets for our product candidates may be smaller than our estimates.
The success of our current and future product candidates will depend on several factors, including the following: ● successful completion of preclinical studies and clinical trials; 37 ● sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; ● acceptance of INDs/IND amendments for our planned clinical trials or future clinical trials; ● successful enrollment and completion of clinical trials; ● successful data from our clinical trials that support FDA conclusion of an acceptable risk-benefit profile of our product candidates in the intended populations; ● receipt of regulatory and marketing approvals from applicable regulatory authorities; ● obtaining and maintaining patent and trade secret protection or regulatory exclusivity for our product candidates; ● obtaining regulatory approval to use our existing or future manufacturing processes for the clinical and commercial manufacture of our product candidates at our existing or future manufacturing facilities or at the facilities of one or more third-party manufacturers with whom we would need to establish supply arrangements; ● successfully launching commercial sales of our product candidates, if and when approved, whether alone or in collaboration with others; ● acceptance of any products we develop and their benefits and uses, if and when approved, by patients, the medical community and third-party payors; ● effectively competing with other therapies; ● obtaining and maintaining healthcare coverage and adequate reimbursement from third-party payors; and ● maintaining a continued acceptable safety profile of the products following approval.
The success of our current and future product candidates will depend on several factors, including the following: ● successful completion of preclinical studies and clinical trials; ● sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; ● acceptance of INDs or IND amendments for our planned clinical trials or future clinical trials; ● successful enrollment and completion of clinical trials; ● successful data from our clinical trials that support FDA conclusion of an acceptable risk-benefit profile of our product candidates in the intended populations; ● receipt of regulatory and marketing approvals from applicable regulatory authorities; ● obtaining and maintaining patent and trade secret protection or regulatory exclusivity for our product candidates; ● obtaining regulatory approval to use our existing or future manufacturing processes for the clinical and commercial manufacture of our product candidates at our existing or future manufacturing facilities or at the facilities of one or more third-party manufacturers with whom we would need to establish supply arrangements; ● successfully launching commercial sales of our product candidates, if and when approved, whether alone or in collaboration with others; ● acceptance of any products we develop and their benefits and uses, if and when approved, by patients, the medical community and third-party payors; 42 ● effectively competing with other therapies; ● obtaining and maintaining healthcare coverage and adequate reimbursement from third-party payors; and ● maintaining a continued acceptable safety profile of the products following approval.
We cannot be certain that we will be able to: ● complete IND-enabling preclinical studies or develop manufacturing processes and associated analytical methods that meet current good manufacturing practice (cGMP) requirements in time to initiate or to complete our anticipated or future clinical trials in the timeframes we announce; ● obtain sufficient clinical supply of our product candidates to support our anticipated or future clinical trials; ● initiate clinical trials within the timeframes we announce; ● enroll and maintain a sufficient number of subjects to complete or timely complete any clinical trials; or ● collect and analyze the data from any completed clinical trials in the timeframes we announce.
We cannot be certain that we will be able to: ● complete IND-enabling preclinical studies or develop manufacturing processes and associated analytical methods that meet current good manufacturing practice (cGMP) requirements in time to initiate or to complete our anticipated or future clinical trials in the timeframes we announce; ● obtain sufficient clinical supply of our product candidates to support our anticipated or future clinical trials; 56 ● initiate clinical trials within the timeframes we announce; ● enroll and maintain a sufficient number of subjects to complete or timely complete any clinical trials; or ● collect and analyze the data from any completed clinical trials in the timeframes we announce.
These factors include, without limitation: ● “short squeezes”; ● comments by securities analysts or other third parties, including blogs, articles, message boards and social and other media; ● large stockholders exiting their position in our common stock or an increase or decrease in the short interest in our common stock; ● actual or anticipated fluctuations in our financial and operating results; ● negative public perception of us, our competitors, or the biopharmaceutical and biotechnology industries; and ● overall general market fluctuations.
These factors include, without limitation: ● “short squeezes”; ● comments by securities analysts or other third parties, including blogs, articles, message boards and social and other media; 116 ● large stockholders exiting their position in our common stock or an increase or decrease in the short interest in our common stock; ● actual or anticipated fluctuations in our financial and operating results; ● negative public perception of us, our competitors, or the biopharmaceutical and biotechnology industries; and ● overall general market fluctuations.
The enrollment of patients depends on many factors, including: ● availability and efficacy of approved therapies for the disease under investigation; 44 ● patient eligibility criteria for the trial in question; ● risks that enrolled subjects will drop out before completion of the trial, including as a result of emergent drug-drug interactions between Olvi-Vec and any of the other therapeutic agents given to the clinical trial subjects or contracting health conditions; ● risks of excessive catheter implantation failures leading to elimination of particular study sites from the trial in question; ● perceived risks and benefits of the product candidate under study; ● the timely initiation of clinical trial sites; ● efforts to facilitate timely enrollment in clinical trials; ● patient referral practices of physicians; ● the ability to monitor patients adequately during and after treatment; ● proximity and availability of clinical trial sites for prospective patients; ● withdrawal of consent for any reason; ● imbalance in withdrawals between the comparator and treatment arms; ● unforeseen limitations of protocol design; and ● protocol amendment by the sponsor and/or as requested by applicable regulatory authorities.
The enrollment of patients depends on many factors, including: ● availability and efficacy of approved therapies for the disease under investigation; 48 ● patient eligibility criteria for the trial in question; ● risks that enrolled subjects will drop out before completion of the trial, including as a result of emergent drug-drug interactions between Olvi-Vec and any of the other therapeutic agents given to the clinical trial subjects or contracting health conditions; ● risks of excessive catheter implantation failures leading to elimination of particular study sites from the trial in question; ● perceived risks and benefits of the product candidate under study; ● the timely initiation of clinical trial sites; ● efforts to facilitate timely enrollment in clinical trials; ● patient referral practices of physicians; ● the ability to monitor patients adequately during and after treatment; ● proximity and availability of clinical trial sites for prospective patients; ● withdrawal of consent for any reason; ● imbalance in withdrawals between the comparator and treatment arms; ● unforeseen limitations of protocol design; and ● protocol amendment by the sponsor and/or as requested by applicable regulatory authorities.
As product candidates are developed through preclinical studies to later-stage clinical trials towards approval and commercialization, it is common that various aspects of the development program, such as manufacturing methods and formulation, are altered along the way in an effort to optimize processes and results. 59 Changes in product candidate manufacturing or formulation may result in additional costs or delay.
As product candidates are developed through preclinical studies to later-stage clinical trials towards approval and commercialization, it is common that various aspects of the development program, such as manufacturing methods and formulation, are altered along the way in an effort to optimize processes and results. Changes in product candidate manufacturing or formulation may result in additional costs or delay.
Manufacturers of therapeutics often encounter difficulties in production, particularly in scaling up initial production, with such risks including: ● quality control, including stability of the product candidate and quality assurance testing; ● shortages of qualified personnel or key raw materials or components; ● product loss during the manufacturing process, including loss caused by contamination, equipment failure or improper installation or operation of equipment, or operator error.
Manufacturers of therapeutics often encounter difficulties in production, particularly in scaling up initial production, with such risks including: ● quality control, including stability of the product candidate and quality assurance testing; ● shortages of qualified personnel or key raw materials or components; 64 ● product loss during the manufacturing process, including loss caused by contamination, equipment failure or improper installation or operation of equipment, or operator error.
We previously engaged a third-party contract manufacturing organization (CMO) that specializes in the manufacture of vaccines to produce clinical-grade Olvi-Vec for all of our prior clinical trials. We have leased a building in San Diego, California and have established and equipped our own manufacturing facility in order to secure supplies for pivotal studies and commercial launch.
We previously engaged a third-party contract manufacturing organization (CMO) that specializes in the manufacture of vaccines to produce clinical-grade Olvi-Vec for all of our prior clinical trials. We have leased a building in San Diego, California and have established and equipped our own cGMP manufacturing facility in order to secure supplies for pivotal studies and commercial launch.
For example, physicians are often reluctant to switch their patients and patients may be reluctant to switch from existing therapies even when new and potentially more effective or safer treatments enter the market. 72 Efforts to educate the medical community and third-party payors on the benefits of our product candidates may require significant resources and may not be successful.
For example, physicians are often reluctant to switch their patients and patients may be reluctant to switch from existing therapies even when new and potentially more effective or safer treatments enter the market. Efforts to educate the medical community and third-party payors on the benefits of our product candidates may require significant resources and may not be successful.
The success of our business depends primarily upon our ability to identify novel product candidates based on our CHOICE platform and to successfully develop and commercialize those product candidates. While we have had promising preclinical and clinical study results for Olvi-Vec, to date, it remains our only product candidate that has moved into clinical trials.
The success of our business depends primarily upon our ability to identify novel product candidates based on our CHOICE platform and to successfully develop and commercialize those product candidates. While we have had promising preclinical study and clinical trial results for Olvi-Vec, to date, it remains our only product candidate that has moved into clinical trials.
Even if we succeed in developing our product candidates, our product candidates may not have a therapeutic effect in a broad patient population. Future negative developments in the field of immuno-oncology or the biopharmaceutical industry could also result in greater governmental regulation, stricter labeling requirements and potential regulatory delays in the testing or approvals of our products.
Even if we succeed in developing our product candidates, our product candidates may not have a therapeutic effect in a broad patient population. 83 Future negative developments in the field of immuno-oncology or the biopharmaceutical industry could also result in greater governmental regulation, stricter labeling requirements and potential regulatory delays in the testing or approvals of our products.
Adequate third-party coverage and reimbursement might not be available to enable us to maintain price levels sufficient to realize an appropriate return on investment in product development. In addition, in the United States, no uniform policy of coverage and reimbursement for products exists among third-party payors. Therefore, coverage and reimbursement for products can differ significantly from payor to payor.
Adequate third-party coverage and reimbursement might not be available to enable us to maintain price levels sufficient to realize an appropriate return on investment in product development. 97 In addition, in the United States, no uniform policy of coverage and reimbursement for products exists among third-party payors. Therefore, coverage and reimbursement for products can differ significantly from payor to payor.
Commencing with our fiscal year ending December 31, 2024, we must perform system and process evaluation and testing of our internal controls over financial reporting to allow management to report on the effectiveness of our internal controls over financial reporting in our Form 10-K filing for that year, as required by Section 404 of the Sarbanes-Oxley Act.
Commencing with our fiscal year ending December 31, 2024, we must perform system and process evaluation and testing of our internal controls over financial reporting to allow management to report on the effectiveness of our internal controls over financial reporting in our Annual Report on Form 10-K filing for that year, as required by Section 404 of the Sarbanes-Oxley Act.
In addition, government funding of other government agencies that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable. 67 Disruptions at the FDA and other agencies may also slow the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business.
In addition, government funding of other government agencies that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable. Disruptions at the FDA and other agencies may also slow the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business.
Conducting clinical trials outside the United States exposes us to additional risks, including risks associated with: ● additional foreign regulatory requirements; ● compliance with foreign manufacturing, customs, shipment and storage requirements; ● cultural differences in medical practice and clinical research; and ● diminished protection of intellectual property in some countries. 54 Approval by the FDA or comparable foreign regulatory authorities to market a product candidate will be limited to those specific indications and conditions for which approval has been granted, and we may be subject to substantial fines, criminal penalties, injunctions, or other enforcement actions if we are determined to be promoting the use of any products for unapproved or “off-label” uses, resulting in damage to our reputation and business.
Conducting clinical trials outside the United States exposes us to additional risks, including risks associated with: ● additional foreign regulatory requirements; ● compliance with foreign manufacturing, customs, shipment and storage requirements; ● cultural differences in medical practice and clinical research; and ● diminished protection of intellectual property in some countries. 59 Approval by the FDA or comparable foreign regulatory authorities to market a product candidate will be limited to those specific indications and conditions for which approval has been granted, and we may be subject to substantial fines, criminal penalties, injunctions, or other enforcement actions if we are determined to be promoting the use of any products for unapproved or “off-label” uses, resulting in damage to our reputation and business.
This could delay completion of clinical trials, require the conduct of bridging clinical trials or studies, require the repetition of one or more clinical trials, increase clinical trial costs, delay approval of our product candidates and/or jeopardize our ability to commence product sales and generate revenue. 60 We may rely on CMOs to conduct large-scale manufacture of Olvi-Vec in the future.
This could delay completion of clinical trials, require the conduct of bridging clinical trials or studies, require the repetition of one or more clinical trials, increase clinical trial costs, delay approval of our product candidates and/or jeopardize our ability to commence product sales and generate revenue. We may rely on CMOs to conduct large-scale manufacture of Olvi-Vec in the future.
Defense of these claims, regardless of their merit, would involve substantial litigation expense and would be a substantial diversion of employee resources from our business. 78 Parties making claims against us may be able to sustain the costs of complex patent litigation more effectively than we can because they have substantially greater resources.
Defense of these claims, regardless of their merit, would involve substantial litigation expense and would be a substantial diversion of employee resources from our business. Parties making claims against us may be able to sustain the costs of complex patent litigation more effectively than we can because they have substantially greater resources.
Should we encounter development problems, including unfavorable preclinical or clinical trial results, the FDA or foreign regulatory authorities may place all, or part, of our clinical development on hold or refuse to approve our product candidates, or may require additional information, tests, or trials, which could significantly delay product development and significantly increase our development costs.
Should we encounter development problems, including unfavorable preclinical study or clinical trial results, the FDA or foreign regulatory authorities may place all, or part, of our clinical development on hold or refuse to approve our product candidates, or may require additional information, tests, or trials, which could significantly delay product development and significantly increase our development costs.
Therefore, even if favorable coverage and reimbursement status is attained, less favorable coverage policies and reimbursement rates may be implemented in the future. There is significant uncertainty related to third-party payor coverage and reimbursement of newly approved therapeutics. Marketing approvals, pricing, and reimbursement for new therapeutic products vary widely from country to country.
Therefore, even if favorable coverage and reimbursement status is attained, less favorable coverage policies and reimbursement rates may be implemented in the future. 96 There is significant uncertainty related to third-party payor coverage and reimbursement of newly approved therapeutics. Marketing approvals, pricing, and reimbursement for new therapeutic products vary widely from country to country.
Any requests from the FDA or comparable foreign regulatory authority for additional data or information could also result in substantial delays in the approval of our product candidates. Drug-related side effects could also affect subject recruitment or the ability of enrolled subjects to complete the trial or result in potential product liability claims.
Any requests from the FDA or comparable foreign regulatory authority for additional data or information could also result in substantial delays in the approval of our product candidates. 53 Drug-related side effects could also affect subject recruitment or the ability of enrolled subjects to complete the trial or result in potential product liability claims.
Some of these events could be the basis for FDA or other regulatory authority action, including injunction, recall, seizure or total or partial suspension of product manufacture. We rely, and expect to continue to rely, on third parties to conduct, supervise, and monitor our preclinical studies and clinical trials.
Some of these events could be the basis for FDA or other regulatory authority action, including injunction, recall, seizure or total or partial suspension of product manufacture. 68 We rely, and expect to continue to rely, on third parties to conduct, supervise, and monitor our preclinical studies and clinical trials.
In such a case, we could be required to obtain a license from the other company or institution to use the required or desired package labeling, which may not be available on commercially reasonable terms, or at all. We may not be able to protect our intellectual property and proprietary rights throughout the world.
In such a case, we could be required to obtain a license from the other company or institution to use the required or desired package labeling, which may not be available on commercially reasonable terms, or at all. 87 We may not be able to protect our intellectual property and proprietary rights throughout the world.
If any of these occur, our business, financial condition, results of operations, stock price and prospects may be materially harmed. 50 Moreover, the development of product candidates for use in combination with another product or product candidate may present challenges that are not faced for single agent product candidates.
If any of these occur, our business, financial condition, results of operations, stock price and prospects may be materially harmed. Moreover, the development of product candidates for use in combination with another product or product candidate may present challenges that are not faced for single agent product candidates.
We can provide no assurance that we would be successful at developing other product candidates based on an alternative therapeutic approach. Our product candidates are based on a novel approach to the treatment of cancer, which makes it difficult to predict the time and cost of product candidate development.
We can provide no assurance that we would be successful at developing other product candidates based on an alternative therapeutic approach. 43 Our product candidates are based on a novel approach to the treatment of cancer, which makes it difficult to predict the time and cost of product candidate development.
Even if we receive accelerated approval from the FDA, if our confirmatory trials do not verify clinical benefit, or if we do not comply with rigorous post-marketing requirements, the FDA may seek to withdraw any accelerated approval we have obtained. 47 We may in the future seek an accelerated approval for Olvi-Vec or our future product candidates.
Even if we receive accelerated approval from the FDA, if our confirmatory trials do not verify clinical benefit, or if we do not comply with rigorous post-marketing requirements, the FDA may seek to withdraw any accelerated approval we have obtained. We may in the future seek an accelerated approval for Olvi-Vec or our future product candidates.
For example, the ACA was passed in March 2010 and substantially changed the way healthcare is financed by both governmental and private insurers and continues to significantly impact the U.S. pharmaceutical industry. There have been executive, judicial and congressional challenges to certain aspects of the ACA.
For example, the ACA was passed in March 2010 and substantially changed the way healthcare is financed by both governmental and private insurers and continues to significantly impact the U.S. pharmaceutical industry. There have been executive, judicial and congressional challenges and amendments to certain aspects of the ACA.
Although there are currently various mechanisms that may be used to transfer personal data from the EEA and UK to the United States in compliance with law, such as the EEA’s standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S.
Although there are currently various mechanisms that may be used to transfer personal data from the EEA and UK to the United States in compliance with law, such as the EEA standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S.
Enrollment delays in our clinical trials may result in increased development costs for our product candidates, which could have an adverse effect on our business, financial condition, results of operations, and prospects. Results of preclinical studies and early clinical trials may not be predictive of results of future clinical trials.
Enrollment delays in our clinical trials may result in increased development costs for our product candidates, which could have an adverse effect on our business, financial condition, results of operations, and prospects. 49 Results of preclinical studies and early clinical trials may not be predictive of results of future clinical trials.
Even if our product candidates are approved, they may: ● be subject to limitations on the indicated uses or patient populations for which they may be marketed, distribution restrictions, or other conditions of approval; 52 ● contain significant safety warnings, including boxed warnings; ● contain significant contraindications, and precautions which could reduce the size of the patient population; ● not be approved with label statements necessary or desirable for successful commercialization; ● contain requirements for costly post-market testing and surveillance, or other requirements, including the submission of a REMS to monitor the safety or efficacy of the products; or ● be withdrawn from the market because a serious safety issue becomes known after approval is granted.
Even if our product candidates are approved, they may: ● be subject to limitations on the indicated uses or patient populations for which they may be marketed, distribution restrictions, or other conditions of approval; ● contain significant safety warnings, including boxed warnings; 57 ● contain significant contraindications, and precautions which could reduce the size of the patient population; ● not be approved with label statements necessary or desirable for successful commercialization; ● contain requirements for costly post-market testing and surveillance, or other requirements, including the submission of a REMS to monitor the safety or efficacy of the products; or ● be withdrawn from the market because a serious safety issue becomes known after approval is granted.
For example, we will remain responsible for ensuring that each of our trials is conducted in accordance with the general investigational plan and protocols for the trial. We must also ensure that our preclinical trials are conducted in accordance with the FDA’s Good Laboratory Practice regulations, as appropriate.
For example, we will remain responsible for ensuring that each of our trials is conducted in accordance with the general investigational plan and protocols for the trial. We must also ensure that our preclinical studies are conducted in accordance with the FDA’s Good Laboratory Practice regulations, as appropriate.
The FDA’s analysis and interpretation of the data may also differ from ours. 45 The results of previous clinical trials of Olvi-Vec and results of preclinical studies or early clinical trials of any other product candidate we develop, may not be predictive of the results of subsequent and later-stage clinical trials.
The FDA’s analysis and interpretation of the data may also differ from ours. The results of previous clinical trials of Olvi-Vec and results of preclinical studies or early clinical trials of any other product candidate we develop, may not be predictive of the results of subsequent and later-stage clinical trials.
We may need to devote significant time and resources to compliance with these requirements. 39 Preclinical and clinical development involve a lengthy and expensive process with an uncertain outcome and stringent regulations, and delays can occur for a variety of reasons.
We may need to devote significant time and resources to compliance with these requirements. Preclinical and clinical development involve a lengthy and expensive process with an uncertain outcome and stringent regulations, and delays can occur for a variety of reasons.
A successful product liability claim or series of claims brought against us could cause our stock price to fall and, if judgments exceed our insurance coverage, could decrease our cash, and materially harm our business, financial condition, results of operations, stock price and prospects. 101 Our employees, independent contractors, consultants, commercial partners, principal investigators, CMOs, or CROs may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements and insider trading, which could have a material adverse effect on our business.
A successful product liability claim or series of claims brought against us could cause our stock price to fall and, if judgments exceed our insurance coverage, could decrease our cash, and materially harm our business, financial condition, results of operations, stock price and prospects. 112 Our employees, independent contractors, consultants, commercial partners, principal investigators, CMOs, or CROs may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements and insider trading, which could have a material adverse effect on our business.
If we are not able to effectively expand our organization by hiring qualified new employees and expanding our groups of consultants and contractors, we may not be able to successfully implement the tasks necessary to further develop and commercialize Olvi-Vec and our other product candidates and, accordingly, may not achieve our research, development and commercialization goals. 96 If we engage in future acquisitions or strategic partnerships, this may increase our capital requirements, dilute our stockholders, cause us to incur debt or assume contingent liabilities, and subject us to other risks.
If we are not able to effectively expand our organization by hiring qualified new employees and expanding our groups of consultants and contractors, we may not be able to successfully implement the tasks necessary to further develop and commercialize Olvi-Vec and our other product candidates and, accordingly, may not achieve our research, development and commercialization goals. 106 If we engage in future acquisitions or strategic partnerships, this may increase our capital requirements, dilute our stockholders, cause us to incur debt or assume contingent liabilities, and subject us to other risks.
Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates, which could have a material adverse effect on our business, financial conditions, results of operations, and prospects. 77 If we are unable to protect the confidentiality of our proprietary information and know-how, the value of our technology and products could be adversely affected.
Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates, which could have a material adverse effect on our business, financial conditions, results of operations, and prospects. 86 If we are unable to protect the confidentiality of our proprietary information and know-how, the value of our technology and products could be adversely affected.
It is also possible that we will fail to identify patentable aspects of inventions made in the course of our development and commercialization activities before it is too late to obtain patent protection on them. 75 In addition, the patent prosecution process is expensive, time-consuming and complex, and we may not be able to file, prosecute, maintain, enforce or license all necessary or desirable patent applications at a reasonable cost or in a timely manner.
It is also possible that we will fail to identify patentable aspects of inventions made in the course of our development and commercialization activities before it is too late to obtain patent protection on them. 84 In addition, the patent prosecution process is expensive, time-consuming and complex, and we may not be able to file, prosecute, maintain, enforce or license all necessary or desirable patent applications at a reasonable cost or in a timely manner.
If we are unable to obtain, maintain, and protect our intellectual property, our competitive advantage could be harmed, and it could result in a material adverse effect on our business, financial condition, results of operations, stock price and prospects. 76 If we fail to comply with our obligations in the agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose intellectual property rights that are important to our business.
If we are unable to obtain, maintain, and protect our intellectual property, our competitive advantage could be harmed, and it could result in a material adverse effect on our business, financial condition, results of operations, stock price and prospects. 85 If we fail to comply with our obligations in the agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose intellectual property rights that are important to our business.
For example, we previously modified our manufacturing process and had to demonstrate comparability to the FDA in order to use Olvi-Vec manufactured by this process in our ongoing Phase 3 PRROC trial.
For example, we previously modified our manufacturing process and had to demonstrate analytical comparability to the FDA in order to use Olvi-Vec manufactured by this process in our ongoing Phase 3 PRROC trial.
For example, we previously modified our manufacturing process and had to demonstrate comparability to the FDA in order to use Olvi-Vec manufactured by this process in our ongoing Phase 3 PRROC trial.
For example, we previously modified our manufacturing process and had to demonstrate analytical comparability to the FDA in order to use Olvi-Vec manufactured by this process in our ongoing Phase 3 PRROC trial.
We also could incur significant costs associated with civil or criminal fines and penalties, as well as our curtailment of the use of these materials or even shutting down our facilities and operations. 94 Although we maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities.
We also could incur significant costs associated with civil or criminal fines and penalties, as well as our curtailment of the use of these materials or even shutting down our facilities and operations. 104 Although we maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities.
Many product candidates that have received fast track designation have ultimately failed to obtain approval. We may attempt to secure approval from the FDA through the use of the accelerated approval pathway.
Many product candidates that have received fast track designation have ultimately failed to obtain approval. 51 We may attempt to secure approval from the FDA through the use of the accelerated approval pathway.
If Newsoara is unable or unwilling to provide this funding and/or reimbursement in a timely manner or at all, we would need to obtain the funding on our own and/or scale back or discontinue these clinical development activities. 66 In addition, all of the risks relating to product development, regulatory approval and commercialization described in this Annual Report also apply to the activities of any of our current or future collaborators.
If Newsoara is unable or unwilling to provide this funding and/or reimbursement in a timely manner or at all, we would need to obtain the funding on our own and/or scale back or discontinue these clinical development activities. 73 In addition, all of the risks relating to product development, regulatory approval and commercialization described in this Annual Report also apply to the activities of any of our current or future collaborators.
These False Claims Act (FCA) lawsuits against manufacturers of drugs and biological products have increased significantly in volume and breadth, leading to several substantial civil and criminal settlements, up to $3.0 billion, pertaining to certain sales practices and promoting off-label uses. In addition, FCA lawsuits may expose manufacturers to follow-on claims by private payors based on fraudulent marketing practices.
These False Claims Act (the “FCA”) lawsuits against manufacturers of drugs and biological products have increased significantly in volume and breadth, leading to several substantial civil and criminal settlements, up to $3.0 billion, pertaining to certain sales practices and promoting off-label uses. In addition, FCA lawsuits may expose manufacturers to follow-on claims by private payors based on fraudulent marketing practices.
If we cannot provide reliable financial reports or prevent fraud, our business and results of operations could be harmed, investors could lose confidence in our reported financial information and we could be subject to sanctions or investigations by Nasdaq, the SEC or other regulatory authorities. 112 Future changes in financial accounting standards or practices may cause adverse and unexpected revenue fluctuations and adversely affect our reported results of operations.
If we cannot provide reliable financial reports or prevent fraud, our business and results of operations could be harmed, investors could lose confidence in our reported financial information and we could be subject to sanctions or investigations by Nasdaq, the SEC or other regulatory authorities. 124 Future changes in financial accounting standards or practices may cause adverse and unexpected revenue fluctuations and adversely affect our reported results of operations.
If we become subject to regulatory and enforcement actions, our business, financial condition, results of operations, stock price and prospects will be materially harmed. 55 Engaging in the impermissible promotion of our products, in the United States, following approval, for off-label uses can also subject us to false claims and other litigation under federal and state statutes.
If we become subject to regulatory and enforcement actions, our business, financial condition, results of operations, stock price and prospects will be materially harmed. 60 Engaging in the impermissible promotion of our products, in the United States, following approval, for off-label uses can also subject us to false claims and other litigation under federal and state statutes.
Any of the foregoing may have a material adverse effect on our business, financial condition, results of operations, stock price and prospects. 82 We may be subject to damages resulting from claims that we or our employees have wrongfully used or disclosed confidential information of third parties or are in breach of non-competition or non-solicitation agreements with our competitors.
Any of the foregoing may have a material adverse effect on our business, financial condition, results of operations, stock price and prospects. 90 We may be subject to damages resulting from claims that we or our employees have wrongfully used or disclosed confidential information of third parties or are in breach of non-competition or non-solicitation agreements with our competitors.
In addition, shares of common stock that are either subject to outstanding options or reserved for future issuance under our employee benefit plans will become eligible for sale in the public market to the extent permitted by the provisions of various vesting schedules and Rule 144 and Rule 701 under the Securities Act of 1933, as amended (Securities Act).
In addition, shares of common stock that are either subject to outstanding options or reserved for future issuance under our employee benefit plans will become eligible for sale in the public market to the extent permitted by the provisions of various vesting schedules and Rule 144 and Rule 701 under the Securities Act of 1933, as amended (the “Securities Act”).
In this case, we could ultimately be forced to cease use of such trademarks. 85 Risks Related to Government Regulation If we fail to comply with federal and state healthcare laws, including fraud and abuse laws, we could face substantial penalties and our business, financial condition, results of operations, stock price and prospects will be materially harmed.
In this case, we could ultimately be forced to cease use of such trademarks. 94 Risks Related to Government Regulation If we fail to comply with federal and state healthcare laws, including fraud and abuse laws, we could face substantial penalties and our business, financial condition, results of operations, stock price and prospects will be materially harmed.
If these additional shares of common stock are sold, or if it is perceived that they will be sold, in the public market, the trading price of our common stock could decline. 107 Future sales and issuances of our common stock or rights to purchase common stock, including pursuant to our equity incentive plans, could result in additional dilution of the percentage ownership of our stockholders and could cause our stock price to fall.
If these additional shares of common stock are sold, or if it is perceived that they will be sold, in the public market, the trading price of our common stock could decline. 119 Future sales and issuances of our common stock or rights to purchase common stock, including pursuant to our equity incentive plans, could result in additional dilution of the percentage ownership of our stockholders and could cause our stock price to fall.
(SillaJen), Targovax USA, Theriva Biologics, Inc., Transgene SA (Transgene), Turnstone Biologics Corp. and Vyriad, Inc.; ● Approved immunotherapy antibodies and immunotherapy agents in clinical development, including antibody agents, bispecific T cell engagers, including those in development by Amgen, and immuno-oncology companies focused on IL-12, such as Ziopharm Oncology Inc.; ● Cancer vaccines, including personalized vaccines and those targeting tumor neoantigens, including neoantigen therapies in development by companies such as Advaxis, Inc., Agenus Inc., AstraZeneca, Bavarian Nordic A/S, BioNTech SE, Genocea Biosciences, Inc., Gritstone Oncology, Inc., Heat Biologics, Inc., ImmunityBio, Inc., IMV Inc., Moderna, Inc., SOTIO a.s., Transgene, and VBI Vaccines Inc.; ● Cell-based therapies, including tumor infiltrating lymphocytes in development by Iovance Biotherapeutics, Inc., TVaxBiomedical, Inc. and Turnstone Biologics, Corp. and approved and in-development CAR T cell therapies, including those commercialized by BMS, Gilead Sciences Inc. and Novartis AG, T cell receptor and NK cell therapies; ● Therapies aimed at activating innate immunity such as those targeting stimulator of interferon genes protein (and toll-like receptors including those in development by Bristol-Myers Squibb Company, Checkmate Pharmaceuticals Inc., Chinook Therapeutics Inc., GlaxoSmithKline plc, Idera Pharmaceuticals, Inc., Merck, Mologen AG, Nektar Therapeutics, TriSalus Life Sciences, and UroGen Pharma Inc.; and ● Traditional cancer therapies, including chemotherapy, surgery, radiation and targeted therapies.
(SillaJen), Theriva Biologics, Inc., Transgene SA (Transgene) and Vyriad, Inc.; 76 ● Approved immunotherapy antibodies and immunotherapy agents in clinical development, including antibody agents, bispecific T cell engagers, including those in development by Amgen, and immuno-oncology companies focused on IL-12, such as Ziopharm Oncology Inc.; ● Cancer vaccines, including personalized vaccines and those targeting tumor neoantigens, including neoantigen therapies in development by companies such as Advaxis, Inc., Agenus Inc., AstraZeneca, Bavarian Nordic A/S, BioNTech SE, Genocea Biosciences, Inc., Gritstone Oncology, Inc., Heat Biologics, Inc., ImmunityBio, Inc., IMV Inc., Moderna, Inc., SOTIO a.s., Transgene, and VBI Vaccines Inc.; ● Cell-based therapies, including tumor infiltrating lymphocytes in development by Iovance Biotherapeutics, Inc., TVAX and Turnstone Biologics, Corp. and approved and in-development CAR T cell therapies, including those commercialized by BMS, Gilead Sciences Inc. and Novartis AG, T cell receptor and NK cell therapies; ● Therapies aimed at activating innate immunity such as those targeting stimulator of interferon genes protein (and toll-like receptors including those in development by Bristol-Myers Squibb Company, Checkmate Pharmaceuticals Inc., Chinook Therapeutics Inc., GlaxoSmithKline plc, Idera Pharmaceuticals, Inc., Merck, Mologen AG, Nektar Therapeutics, TriSalus Life Sciences, and UroGen Pharma Inc.; and ● Traditional cancer therapies, including chemotherapy, surgery, radiation and targeted therapies.
This will require us to be successful in a range of challenging activities, including completing preclinical testing and clinical trials, obtaining regulatory approval for product candidates and manufacturing, marketing and selling products for which we may obtain marketing approval and satisfying any post-marketing requirements. We are only in the development stages of most of these activities.
This will require us to be successful in a range of challenging activities, including completing preclinical studies and clinical trials, obtaining regulatory approval for product candidates and manufacturing, marketing and selling products for which we may obtain marketing approval and satisfying any post-marketing requirements. We are only in the development stages of most of these activities.
We may be required to obtain additional funds to complete clinical trials and prepare for possible commercialization of our product candidates. We do not know whether any preclinical tests or clinical trials beyond what we currently have planned will be required, will begin as planned, will need to be restructured, or will be completed on schedule or at all.
We may be required to obtain additional funds to complete clinical trials and prepare for possible commercialization of our product candidates. We do not know whether any preclinical studies or clinical trials beyond what we currently have planned will be required, will begin as planned, will need to be restructured, or will be completed on schedule or at all.
Further, in July 2010, the Dodd-Frank Wall Street Reform and Consumer Protection Act (the Dodd-Frank Act) was enacted. There are significant corporate governance and executive compensation related provisions in the Dodd-Frank Act that require the SEC to adopt additional rules and regulations in these areas such as “say on pay” and proxy access.
Further, in July 2010, the Dodd-Frank Wall Street Reform and Consumer Protection Act (the “Dodd-Frank Act”) was enacted. There are significant corporate governance and executive compensation related provisions in the Dodd-Frank Act that require the SEC to adopt additional rules and regulations in these areas such as “say on pay” and proxy access.
If we are not able to generate revenue from the sale of any approved products, we could be prevented from or significantly delayed in achieving profitability. 35 Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
If we are not able to generate revenue from the sale of any approved products, we could be prevented from or significantly delayed in achieving profitability. 39 Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and instances of fraud will be detected. 103 These inherent limitations include the realities that judgments in decision-making can be faulty, and that breakdowns can occur because of simple error or mistake.
Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and instances of fraud will be detected. 114 These inherent limitations include the realities that judgments in decision-making can be faulty, and that breakdowns can occur because of simple error or mistake.
If microbial, viral or other contaminations are discovered in our products or in the manufacturing facilities in which our products are made, such manufacturing facilities may need to be closed for an extended period of time to investigate and remedy the contamination; ● the manufacturing facilities in which our product candidates are made could be adversely affected by equipment failures, labor and raw material shortages, natural disasters, power failures and numerous other factors; and ● any adverse developments affecting manufacturing operations for our products may result in shipment delays, inventory shortages, lot failures, product withdrawals or recalls, or other interruptions in the supply of our product candidates.
If microbial, viral or other contaminations are discovered in our products or in the manufacturing facilities in which our products are made, such manufacturing facilities may need to be closed for an extended period of time to investigate and remedy the contamination; ● the manufacturing facilities in which our product candidates are made could be adversely affected by equipment failures, natural disasters, power failures and numerous other factors; and ● any adverse developments affecting manufacturing operations for our products may result in shipment delays, inventory shortages, lot failures, product withdrawals or recalls, or other interruptions in the supply of our product candidates.
We do not maintain “key person” insurance policies on the lives of all of these individuals or the lives of any of our other employees. 95 We will need to continue to expand the size of our organization, and we may experience difficulties in managing this growth, which could disrupt our operations.
We do not maintain “key person” insurance policies on the lives of all of these individuals or the lives of any of our other employees. 105 We will need to continue to expand the size of our organization, and we may experience difficulties in managing this growth, which could disrupt our operations.
Any of the foregoing could harm our business and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could adversely impact our business. 97 Public health crises such as pandemics could materially and adversely affect our preclinical studies and clinical trials, business, financial condition and results of operations.
Any of the foregoing could harm our business and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could adversely impact our business. 107 Public health crises such as pandemics could materially and adversely affect our preclinical studies and clinical trials, business, financial condition and results of operations.
Patent reform legislation in the United States and other countries, including the Leahy-Smith America Invents Act (the Leahy-Smith Act) signed into law in the United States on September 16, 2011, could increase those uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.
Patent reform legislation in the United States and other countries, including the Leahy-Smith America Invents Act (the “Leahy-Smith Act”) signed into law in the United States on September 16, 2011, could increase those uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.
Such threats are prevalent and continue to increase, are becoming increasingly difficult to detect, and come from a variety of sources, including traditional computer “hackers,” “hacktivists,” organized criminal threat actors, personnel (such as through theft or misuse), sophisticated nation states, and nation-state-supported actors.
Such threats are prevalent and continue to rise, are becoming increasingly difficult to detect, and come from a variety of sources, including traditional computer “hackers,” threat actors, “hacktivists,” organized criminal threat actors, personnel (such as through theft or misuse), sophisticated nation states, and nation-state-supported actors.
The report of our independent registered public accounting firm on our financial statements as of and for the years ended December 31, 2023 and 2022 included an explanatory paragraph indicating that there was substantial doubt about our ability to continue as a going concern.
The report of our independent registered public accounting firm on our financial statements as of and for the years ended December 31, 2024 and 2023 included an explanatory paragraph indicating that there was substantial doubt about our ability to continue as a going concern.
Any future changes to our manufacturing process may similarly require comparability assessments by the FDA and could delay clinical trials or, if the modified manufacturing process is not comparable, result in inconsistencies in trial results that may be difficult to explain.
Any future changes to our manufacturing process may similarly require comparability assessments by the FDA and could delay clinical trials or, if the product of the modified manufacturing process is not comparable, result in inconsistencies in trial results that may be difficult to explain.
We anticipate being awarded market exclusivity for each of our biological product candidates that is subject to its own BLA for 12 years in the United States, 10 years in Europe and significant durations in other markets.
We anticipate being awarded data exclusivity for each of our biological product candidates that is subject to its own BLA for 12 years in the United States, 10 years in Europe and significant durations in other markets.
The commencement and rate of completion of preclinical studies and clinical trials for a product candidate may be delayed by many factors, including, for example: ● inability to generate sufficient preclinical or other in vivo or in vitro data to support the initiation of clinical trials; ● unexpected toxicities observed in preclinical IND-enabling studies precluding the identification of a safe dose to move forward in human clinical trials; ● delays in obtaining regulatory approval for, and production or manufacturing of, clinical supply; ● delays in reaching a consensus with regulatory agencies on study or trial design; and ● regulatory authorities not allowing us to rely on previous findings of safety and efficacy for other similar but approved products and published scientific literature. 40 We may experience delays in initiating or completing clinical trials.
The commencement and rate of completion of preclinical studies and clinical trials for a product candidate may be delayed by many factors, including, for example: ● inability to generate sufficient preclinical or other in vivo or in vitro data to support the initiation of clinical trials; ● unexpected toxicities observed in preclinical IND-enabling studies precluding the identification of a safe dose to move forward in human clinical trials; ● delays in obtaining regulatory approval for, and production or manufacturing of, clinical supply; ● delays in reaching a consensus with regulatory agencies on study or trial design; and ● regulatory authorities not allowing us to rely on previous findings of safety and efficacy for other similar but approved products and published scientific literature.
Before obtaining marketing approval for the commercial distribution of our product candidates, we, or a future collaborator, must conduct extensive preclinical tests and clinical trials to demonstrate the safety and efficacy in humans of our product candidates.
Before obtaining marketing approval for the commercial distribution of our product candidates, we, or a future collaborator, must conduct extensive preclinical studies and clinical trials to demonstrate the safety and efficacy in humans of our product candidates.
Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud. We are subject to the periodic reporting requirements of the Securities Exchange Act of 1934, as amended (the Exchange Act).
Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud. We are subject to the periodic reporting requirements of the Securities Exchange Act of 1934, as amended (the “Exchange Act”).
In addition, pursuant to our ESPP, the number of shares of our common stock reserved for issuance will automatically increase on January 1 of each calendar year, beginning on January 1, 2024 through January 1, 2032, by the lesser of (i) 1% of the total number of shares of our common stock outstanding on December 31 of the preceding calendar year, and (ii) 2,100,000 shares of common stock; provided that before the date of any such increase, our board of directors may determine that such increase will be less than the amount set forth in clauses (i) and (ii).
In addition, pursuant to our Employee Stock Purchase Plan, the number of shares of our common stock reserved for issuance will automatically increase on January 1 of each calendar year, beginning on January 1, 2024 through January 1, 2032, by the lesser of (i) 1% of the total number of shares of our common stock outstanding on December 31 of the preceding calendar year, and (ii) 2,100,000 shares of common stock; provided that before the date of any such increase, our board of directors may determine that such increase will be less than the amount set forth in clauses (i) and (ii).
Newsoara has also agreed to reimburse us for the costs and expenses of a CRO to conduct certain startup activities for the NSCLC trial in the United States only, but is permitted to defer such reimbursement payments until the completion of its next round of financing, which Newsoara expects to occur in 2024.
Newsoara has also agreed to reimburse us for the costs and expenses of a CRO to conduct certain startup activities for the NSCLC trial in the United States only, but is permitted to defer such reimbursement payments until the completion of its next round of financing, which Newsoara expects to occur in late 2025.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeRisk Factors in this Annual Report on Form 10-K, including “ If our information technology systems or data, or those of third parties upon which we rely, are or were compromised, we could experience adverse consequences resulting from such compromise, including but not limited to regulatory investigations or actions; litigation and mass arbitration demands; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; loss of customers or sales; and other adverse consequences .” Governance Our board of directors addresses the Company’s cybersecurity risk management as part of its general oversight function.
Biggest changeRisk Factors in this Annual Report on Form 10-K, including “ If our information technology systems or those third parties with whom we work or our data are or were compromised, we could experience adverse consequences resulting from such compromise, including but not limited to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; loss of customers or sales; and other adverse consequences .” Governance Our Board of Directors addresses the Company’s cybersecurity risk management as part of its general oversight function.
Cybersecurity Risk management and strategy We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, confidential information that is proprietary, strategic or competitive in nature, and clinical trial data (“Information Systems and Data”).
Cybersecurity Risk management and strategy We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third-party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, confidential information that is proprietary, strategic or competitive in nature, and clinical trial data (Information Systems and Data).
We use third-party service providers to perform a variety of functions throughout our business, such as application providers, hosting companies, contract research organizations, contract manufacturing organizations, and distributors. We have a vendor management program to manage cybersecurity risks associated with our use of these providers.
We also use third-party service providers to perform a variety of other functions throughout our business, such as application providers, hosting companies, contract research organizations, contract manufacturing organizations, and distributors. We have a vendor management program to manage cybersecurity risks associated with our use of these providers.
Our CEO is responsible for hiring appropriate personnel, helping to integrate cybersecurity risk considerations into the Company’s overall risk management strategy, and communicating key priorities to relevant personnel. The Company’s Cybersecurity Risk Management Team is responsible for developing budgets, helping prepare for cybersecurity incidents, approving cybersecurity processes, and reviewing security assessments and other security-related reports.
Our Chief Executive Officer (CEO) is responsible for hiring appropriate personnel, helping to integrate cybersecurity risk considerations into the Company’s overall risk management strategy, and communicating key priorities to relevant personnel. The Company’s Cybersecurity Risk Management Team is responsible for developing budgets, helping prepare for cybersecurity incidents, approving cybersecurity processes, and reviewing security assessments and other security-related reports.
The program includes a review of certain vendor’s written security program, a risk assessment for certain vendors, and imposition of information security contractual obligations on such vendors. 114 For a description of the risks from cybersecurity threats that may materially affect the Company and how they may do so, see our risk factors under Part 1. Item 1A.
The program includes a review of certain vendors’ written security program, a risk assessment for certain vendors, and imposition of information security contractual obligations on certain vendors. 126 For a description of the risks from cybersecurity threats that may materially affect the Company and how they may do so, see our risk factors under Part 1. Item 1A.
Our cybersecurity risk assessment and management processes are implemented and maintained by our Cybersecurity Risk Management Team, including Sean Ryder (General Counsel) and an external cybersecurity consultant with over 15 years of experience providing cybersecurity services and training (who is also a member of the Company’s Cybersecurity Risk Management Team) and an external information technology consultant.
Our cybersecurity risk assessment and management processes are implemented and maintained by our Cybersecurity Risk Management Team, (which includes our General Counsel) an external cybersecurity consultant with over 15 years of experience providing cybersecurity services and training (who is also a member of the Company’s Cybersecurity Risk Management Team) and an external information technology consultant.
Our cybersecurity incident response and vulnerability management processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our CFO and CEO. Our CFO and CEO work with the Company’s Incident Response Team to help the Company mitigate and remediate cybersecurity incidents of which they are notified.
Our cybersecurity incident response and vulnerability management processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Chief Financial Officer (CFO) and CEO. Our CFO and CEO work with the Company’s Incident Response Team to help the Company triage, contain, mitigate and remediate cybersecurity incidents of which they are notified.
The board of directors and Audit Committee also receives various reports, summaries or presentations related to cybersecurity threats, risk and mitigation.
The Board of Directors and Audit Committee also receive various reports, summaries and presentations related to cybersecurity threats, risk and mitigation.
The Company engages two external cybersecurity and information technology consultants to work with the Company, including the general counsel, to help identify, assess and manage the Company’s cybersecurity threats and risks.
The Company engages two external cybersecurity and information technology consultants to work with the Company, including the general counsel, to help identify, assess and manage the Company’s cybersecurity threats and risks, including through the use of the Company’s information security risk register.

Item 2. Properties

Properties — owned and leased real estate

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Biggest changeItem 2. Properties. Our current corporate headquarters are located in Westlake Village, California, consisting of 4,050 square feet of office space. The lease for this facility expires in July 2027. Additionally, we lease 6,880 square feet in San Diego, California for research and development and pharmaceutical development laboratory and office space; the lease expires in December 2024.
Biggest changeItem 2. Properties. Our current corporate headquarters are located in Westlake Village, California, consisting of 4,050 square feet of office space. The lease for this facility expires in July 2027.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Item 3. Legal Proceedings. As of December 31, 2023, we were involved in one pending litigation. On November 6, 2023, the Los Angeles County Superior Court granted the Company’s motion for summary judgment and issued an order and final judgment dismissing all claims against the Company with prejudice.
Removed
Although the plaintiff filed a notice of appeal of the dismissal order with the California Court of Appeal, the plaintiff subsequently filed a request for dismissal of his appeal, which was dismissed by the appellate court on February 23, 2024. Accordingly, the order and final judgment dismissing all claims against the Company with prejudice is now final.

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeSecurities Authorized for Issuance under Equity Compensation Plans See Item 12 of Part III of this Annual Report for information about our equity compensation plans, which is incorporated by reference herein . Recent Sales of Unregistered Securities None.
Biggest changeSecurities Authorized for Issuance under Equity Compensation Plans See Item 12 of Part III of this Annual Report on Form 10-K (the Annual Report) for information about our equity compensation plans, which is incorporated by reference herein. Recent Sales of Unregistered Securities None. Purchases of Equity Securities by the Issuer and Affiliated Purchasers Not applicable.
Dividend Policy We have never declared or paid a cash dividend on our common stock. We currently intend to retain all available funds and any future earnings, if any, to fund the development and expansion of our business and we do not anticipate paying any cash dividends in the foreseeable future.
Dividend Policy We have never declared or paid cash dividends on our common stock. We currently intend to retain all available funds and any future earnings, if any, to fund the development and expansion of our business and we do not anticipate paying any cash dividends in the foreseeable future.
Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information Our common stock, par value $0.001 per share, is traded on The Nasdaq Capital Market under the symbol “GNLX.” Trading of our common stock commenced on January 26, 2023, following the completion of our IPO.
Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information Our common stock, par value $0.001 per share, is traded on The Nasdaq Capital Market under the symbol “GNLX.” Trading of our common stock commenced on January 26, 2023, following the completion of our initial public offering (IPO).
Prior to that time, there was no established public trading market for our common stock. Holders of Record As of March 26, 2024, there were approximately 1101 stockholders of record of our common stock. Certain shares are held in street name and accordingly, the number of beneficial owners of such shares is not known or included in the foregoing number.
Prior to that time, there was no established public trading market for our common stock. Holders of Record As of March 13, 2025, there were approximately 1,026 stockholders of record of our common stock. Certain shares are held in street name and accordingly, the number of beneficial owners of such shares is not known or included in the foregoing number.
Removed
Use of Proceeds On January 25, 2023, our Registration Statement on Form S-1, as amended (File No. 333-265828) was declared effective in connection with the IPO of our common stock, pursuant to which we registered an aggregate of 2,500,000 shares of our common stock, of which we sold 2,653,000 shares, including the partial exercise of the underwriters’ option to purchase additional shares, at a price to the public of $6.00 per share, for aggregate gross proceeds of $15.9 million.
Removed
The offering closed on January 30, 2023. The underwriting discounts and commissions for the IPO totaled approximately $1.4 million. We incurred additional costs of approximately $2.1 million in offering expenses, which when added to the underwriting discounts and commissions paid by us, amounts to total fees and costs of approximately $3.5 million.
Removed
Thus, estimated net offering proceeds to us, after deducting underwriting discounts, commissions and offering expenses, were approximately $12.4 million, including the partial exercise of the underwriters’ overallotment option.
Removed
No offering expenses were paid directly or indirectly to any of our directors or officers (or their associates) or persons owning 10 percent or more of any class of our equity securities or to any other affiliates. The Benchmark Company, LLC and Brookline Capital Markets, a division of Arcadia Securities, LLC, acted as joint book-running managers for the IPO.
Removed
The net proceeds from our initial public offering are being held in cash, cash equivalents and investments securities , primarily in money market funds invested in U.S. government agency securities and U.S. treasury securities.
Removed
These investments are made pursuant to our investment policy and we may further invest these funds in high-quality marketable debt instruments of corporations and government sponsored enterprises with contractual maturity dates of generally less than two years until needed to fund our operations.
Removed
There has been no material change in the use of proceeds from our initial public offering as described in our final prospectus filed with the SEC pursuant to Rule 424(b)(4) on January 26, 2023 . As of December 31, 2023, we had used all of the net proceeds received from our initial public offering to support our operations.
Removed
Purchases of Equity Securities by the Issuer and Affiliated Purchasers Not applicable.

Item 7. Management's Discussion & Analysis

Management's Discussion & Analysis (MD&A) — revenue / margin commentary

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Biggest changeIn addition, if we obtain regulatory approval for any of our product candidates and do not enter into a third-party commercialization collaboration, we expect to incur significant expenses related to building a sales and marketing team to support product sales, marketing and distribution activities. 120 Results of Operations Comparison of the Years Ended December 31, 2023 and 2022 The following table summarizes our results of operations for the years ended December 31, 2023 and 2022 (in thousands): December 31, December 31, 2023 2022 Revenues $ 170 $ 11,068 Operating Expenses: Research and development 12,767 9,078 General and administrative 11,568 5,003 Total operating expenses 24,335 14,081 Loss from operations (24,165 ) (3,013 ) Other income (expenses): Interest income 244 — Interest expense (173 ) (1,150 ) Debt discount amortization (649 ) (258 ) Debt extinguishment costs (402 ) — Financing costs (3,152 ) — Gain on the forgiveness of PPP loan payable — 314 Total other expenses, net (4,132 ) (1,094 ) Loss before provision for foreign income taxes (28,297 ) (4,107 ) Provision for foreign income taxes — (1,100 ) Net loss $ (28,297 ) $ (5,207 ) Research and Development Expenses The table below summarizes our research and development expenses for the years ended December 31, 2023 and 2022 (in thousands): Research and Development Expenses: December 31, 2023 December 31, 2022 Employee compensation and related expenses $ 2,538 $ 1,531 Stock compensation 1,876 368 Manufacturing and laboratory materials and other expenses 1,502 937 Outsourced manufacturing services 1,145 908 Clinical and regulatory expenses 3,698 3,252 Facility-related expenses, including depreciation 1,356 1,278 Consulting expenses 595 746 Other expenses 57 58 Total research and development expenses $ 12,767 $ 9,078 Research and development expenses were $12.8 million and $9.1 million for the years ended December 31, 2023 and 2022, respectively, an increase of $3.7 million.
Biggest changeIn addition, if we obtain regulatory approval for any of our product candidates and do not enter into a third-party commercialization collaboration, we expect to incur significant expenses related to building a sales and marketing team to support product sales, marketing and distribution activities. 132 Results of Operations Comparison of the Years Ended December 31, 2024 and 2023 The following table summarizes our results of operations for the years ended December 31, 2024 and 2023 (in thousands): December 31, December 31, 2024 2023 Revenues $ 8 $ 170 Operating Expenses: Research and development 18,998 12,767 General and administrative 12,706 11,568 Total operating expenses 31,704 24,335 Loss from operations (31,696 ) (24,165 ) Other income (expenses): Interest income 1,457 244 Gain on extinguishment of accounts payable 370 — Interest expense — (173 ) Debt discount amortization — (649 ) Financing costs — (3,152 ) Debt extinguishment costs — (402 ) Total other income (expenses), net 1,827 (4,132 ) Net loss $ (29,869 ) $ (28,297 ) Research and Development Expenses The table below summarizes our research and development expenses for the years ended December 31, 2024 and 2023 (in thousands): December 31, December 31, Research and Development Expenses: 2024 2023 Employee compensation and related expenses $ 3,766 $ 2,538 Stock compensation, including the cost of stock options and restricted stock grants 3,090 1,876 Manufacturing and laboratory materials and other expenses 556 1,502 Outsourced manufacturing services 1,602 1,145 Clinical and regulatory expenses 8,204 3,698 Facility-related expenses, including depreciation 1,320 1,356 Consulting expenses and contract labor 414 595 Other expenses 46 57 Total research and development expenses $ 18,998 $ 12,767 Research and development expenses were $19.0 million and $12.8 million for the years ended December 31, 2024 and 2023, respectively, an increase of approximately $6.2 million.
In addition, if we obtain regulatory approval for our product candidates and do not enter into a third-party commercialization partnership, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing, manufacturing, and distribution activities. 117 As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy.
In addition, if we obtain regulatory approval for our product candidates and do not enter into a third-party commercialization partnership, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing, manufacturing, and distribution activities. As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy.
Operating Expenses Our operating expenses consist of (i) research and development expenses and (ii) general and administrative expenses. 118 Research and Development Expenses Research and development expenses consist primarily of costs incurred for our research and development activities, including our product candidate discovery efforts and preclinical and clinical studies under our research programs, which include: ● employee-related expenses, including salaries, benefits and stock-based compensation expense for our research and development personnel; ● costs of funding research performed by third parties that conduct research and development and preclinical and clinical activities on our behalf; ● costs of manufacturing drug product and drug supply related to our current or future product candidates; ● costs of conducting preclinical studies and clinical trials of our product candidates; ● consulting and professional fees related to research and development activities, including equity-based compensation to non-employees; ● costs of maintaining our laboratory, including purchasing laboratory supplies and non-capital equipment used in our preclinical studies; ● costs related to compliance with clinical regulatory requirements; and ● facility costs and other allocated expenses, which include expenses for rent and maintenance of facilities, insurance, depreciation and other supplies.
Operating Expenses Our operating expenses consist of (i) research and development expenses and (ii) general and administrative expenses. 130 Research and Development Expenses Research and development expenses consist primarily of costs incurred for our research and development activities, including our product candidate discovery efforts and preclinical and clinical studies under our research programs, which include: ● employee-related expenses, including salaries, benefits and stock-based compensation expense for our research and development personnel; ● costs of funding research performed by third parties that conduct research and development and preclinical and clinical activities on our behalf; ● costs of manufacturing drug product and drug supply related to our current or future product candidates; ● costs of conducting preclinical studies and clinical trials of our product candidates; ● consulting and professional fees related to research and development activities, including equity-based compensation to non-employees; ● costs of maintaining our laboratory, including purchasing laboratory supplies and non-capital equipment used in our preclinical studies; ● costs related to compliance with clinical regulatory requirements; and ● facility costs and other allocated expenses, which include expenses for rent and maintenance of facilities, insurance, depreciation and other supplies.
In November 2023, we agreed with Newsoara that Genelux would directly engage a contract research organization on mutually agreeable terms to conduct certain startup activities for the NSCLC trial in the U.S. only, with Newsoara reimbursing Genelux for the costs and expenses of such agreed-upon startup activities.
In November 2023, we agreed with Newsoara that Genelux would directly engage a contract research organization on mutually agreeable terms to conduct certain startup activities for the NSCLC trial in the U.S. only, with Newsoara reimbursing us for the costs and expenses of such agreed-upon startup activities.
Even if we are able to obtain additional financing, it may contain undue restrictions on our operations, in the case of debt financing, cause substantial dilution to our stockholders, in the case of equity financing, or grant unfavorable terms in future licensing agreements.
Even if we are able to obtain additional financing, it may contain undue restrictions on our operations, in the case of debt financing, or cause substantial dilution for our stockholders, in case of equity financing, or grant unfavorable terms in future licensing agreements.
Please also see the section entitled “Special Note Regarding Forward- Looking Statements.” Overview Genelux is a late clinical-stage biopharmaceutical company focused on developing a pipeline of next-generation oncolytic viral immunotherapies for patients suffering from aggressive and/or difficult-to-treat solid tumor types.
Please also see the section titled “Special Note Regarding Forward- Looking Statements.” Overview Genelux is a late clinical-stage biopharmaceutical company focused on developing a pipeline of next-generation oncolytic viral immunotherapies for patients suffering from aggressive and/or difficult-to-treat solid tumor types.
Significant judgment and estimates are made in determining the accrued expense balances at the end of any reporting period. The successful development of our product candidates is highly uncertain. We cannot reasonably estimate or know the nature, timing, and estimated costs of the efforts that will be necessary to complete development of our current or future product candidates.
Significant judgement and estimates are made in determining the accrued expense balances at the end of any reporting period. The successful development of our product candidates is highly uncertain. We cannot reasonably estimate or know the nature, timing, and estimated costs of the efforts that will be necessary to complete development of our current or future product candidates.
Our ability to raise additional funds also may be adversely impacted by potential worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from geopolitical and macroeconomic events such as actual or anticipated changes in interest rates and economic inflation, current and future bank failures, global pandemics, geopolitical tensions between the U.S. and China and the impact of the Russia/Ukraine conflict and the war in the Middle East.
Our ability to raise additional funds also may be adversely impacted by potential worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from geopolitical and macroeconomic events such as actual or anticipated changes in interest rates and economic inflation, current and future bank failures, tariffs, global pandemics, geopolitical tensions between the United States and China and the impact of the Russia/Ukraine conflict and the conflicts in the Middle East.
Investing Activities Net cash used in investing activities for the year ended December 31, 2023 was $14.8 million, consisting of the purchase of short-term investments of $13.7 million, and purchases of property and equipment for construction-in-progress of $1.0 million.
Net cash used in investing activities for the year ended December 31, 2023 was $14.7 million, consisting of purchases of short-term investments of $13.7 million, and the purchase of property and equipment of $1.0 million.
We also anticipate increased expenses associated with being a public company, including costs for audit, legal, regulatory and tax-related services related to compliance with the rules and regulations of the SEC, and listing standards applicable to companies listed on a national securities exchange, director and officer insurance premiums, and investor relations costs.
We also anticipate increased expenses associated with being a public company, including costs for audit, legal, regulatory and tax-related services related to compliance with the rules and regulations of the U.S. Securities and Exchange Commission (the “SEC”), and listing standards applicable to companies listed on a national securities exchange, director and officer insurance premiums, and investor relations costs.
Our ability to continue as a going concern is dependent upon our ability to raise additional funds and implement our strategies. The financial statements do not include any adjustments that might be necessary if we are unable to continue as a going concern.
These factors raise substantial doubt about our ability to continue as a going concern. Our ability to continue as a going concern is dependent upon our ability to raise additional funds and implement our strategies. The financial statements do not include any adjustments that might be necessary if we are unable to continue as a going concern.
This is due to the numerous risks and uncertainties associated with developing product candidates, including the uncertainty of: ● the scope, rate of progress, and expenses of our ongoing research activities as well as any preclinical studies and clinical trials and other research and development activities; ● establishing an appropriate safety profile; ● successful enrollment in and completion of clinical trials; ● whether our product candidates show safety and efficacy in our clinical trials; ● receipt of marketing approvals from applicable regulatory authorities; ● establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; ● obtaining and maintaining patent and trade secret protection and regulatory exclusivity for our product candidates; ● commercializing product candidates, if and when approved, whether alone or in collaboration with others; and ● continued acceptable safety profile of the products following any regulatory approval.
This is due to the numerous risks and uncertainties associated with developing product candidates, including the uncertainty of: ● the scope, rate of progress, and expenses of our ongoing research activities as well as any preclinical studies and clinical trials and other research and development activities; ● establishing an appropriate safety profile; ● successful enrollment in and completion of clinical trials; ● whether our product candidates show safety and efficacy in our clinical trials; ● receipt of marketing approvals from applicable regulatory authorities; ● establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; ● obtaining and maintaining patent and trade secret protection and regulatory exclusivity for our product candidates; ● commercializing product candidates, if and when approved, whether alone or in collaboration with others; and ● continued acceptable safety profile of the products following any regulatory approval. 131 A change in the outcome of any of these variables with respect to the development of our current and future product candidates would significantly change the costs and timing associated with the development of those product candidates.
The primary use of cash during the year ended December 31, 2023 was the decrease in accounts payable and accrued expenses of $2.4 million. The primary use of cash during the year ended December 31, 2022 was the decrease in deferred revenue of $4.3 million.
The primary use of cash during the year ended December 31, 2023 was the decrease in accounts payable and accrued expenses of $2.4 million.
If we are unable to raise capital when needed or on acceptable terms, we would be forced to delay, reduce or eliminate our research and development programs or future commercialization efforts. 125 We believe that our existing cash will enable us to fund our operating expenses and capital expenditure requirements until at least 12 months from the date of filing of this Annual Report.
If we are unable to raise capital when needed or on acceptable terms, we would be forced to delay, reduce or eliminate our research and development programs or future commercialization efforts. 136 We believe that our existing cash, cash equivalents and short-term investments may not enable us to fund our operating expenses and capital expenditure requirements for at least the next 12 months from the date of filing of this Annual Report.
Our future capital requirements will depend on a number of factors, including: ● the costs of conducting preclinical studies and clinical trials; ● the costs of manufacturing; ● the scope, progress, results and costs of discovery, preclinical development, laboratory testing, and clinical trials for product candidates we may develop, if any; ● the costs, timing, and outcome of regulatory review of our product candidates; ● our ability to establish and maintain collaborations on favorable terms, if at all; ● the achievement of milestones or occurrence of other developments that trigger payments under any license or collaboration agreements we might have at such time; ● the costs and timing of future commercialization activities, including product sales, marketing, manufacturing and distribution, for any of our product candidates for which we receive marketing approval; ● the amount of revenue, if any, received from commercial sales of our product candidates, should any of our product candidates receive marketing approval; ● the costs of preparing, filing and prosecuting patent applications, obtaining, maintaining and enforcing our intellectual property rights, and defending intellectual property-related claims; ● our headcount growth and associated costs as we expand our business operations and research and development activities; and ● the costs of operating as a public company.
Our future capital requirements will depend on a number of factors, including: ● the costs of conducting preclinical studies and clinical trials; ● the costs of manufacturing; ● the scope, progress, results and costs of discovery, preclinical development, laboratory testing, and clinical trials for product candidates we may develop, if any; ● the costs, timing, and outcome of regulatory review of our product candidates; ● our ability to establish and maintain collaborations on favorable terms, if at all; ● the achievement of milestones or occurrence of other developments that trigger payments under any license or collaboration agreements we might have at such time; ● the costs and timing of future commercialization activities, including product sales, marketing, manufacturing and distribution, for any of our product candidates for which we receive marketing approval; ● the amount of revenue, if any, received from commercial sales of our product candidates, should any of our product candidates receive marketing approval; ● the costs of preparing, filing and prosecuting patent applications, obtaining, maintaining and enforcing our intellectual property rights, and defending intellectual property-related claims; ● our headcount growth and associated costs as we expand our business operations and research and development activities; ● the costs of operating as a public company; and ● the impact of geopolitical and macroeconomic events, including future bank failures, tariffs, increased geopolitical tensions between the U.S. and China, the Russia/Ukraine conflict, the Israel-Hamas war and global pandemics on U.S. and global economic conditions that may affect our ability to access capital on acceptable terms, if at all.
There are numerous factors associated with the successful commercialization of any of our product candidates, including future trial design and various regulatory requirements, many of which cannot be determined with accuracy at this time based on our stage of development.
There are numerous factors associated with the successful commercialization of any of our product candidates, including future trial design and various regulatory requirements, many of which cannot be determined with accuracy at this time based on our stage of development. Additionally, future commercial and regulatory factors beyond our control will impact our clinical development programs and plans.
Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through public or private equity offerings and debt financings or other sources, such as potential collaboration agreements, strategic alliances and licensing arrangements.
We anticipate needing to obtain further funding to achieve our business objectives beyond such date. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through public or private equity offerings and debt financings or other sources, such as potential collaboration agreements, strategic alliances and licensing arrangements.
Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.
Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.
During the year ended December 31, 2023, we incurred a net loss of $28.3 million and had non-cash expenses of $11.3 million, compared to a net loss of $5.2 million and non-cash expenses of $3.3 million during the year ended December 31, 2022.
During the year ended December 31, 2024, we incurred a net loss of $29.9 million and had non-cash expenses of $7.9 million, compared to a net loss of $28.3 million and non-cash expenses of $11.3 million during the year ended December 31, 2023.
Since inception, we have incurred significant operating losses. Our net losses were $28.3 million and $5.2 million for the years ended December 31, 2023 and 2022, respectively. As of December 31, 2023, we had an accumulated deficit of $221.5 million.
Since inception, we have incurred significant operating losses. Our net losses were $29.9 million and $28.3 million, respectively, for the years ended December 31, 2024 and 2023. As of December 31, 2024, we had an accumulated deficit of $251.4 million.
For the year ended December 31, 2023, cash provided by financing activities consisted of proceeds from the issuance of notes payable totaling $0.9 million, proceeds from the exercise of stock options of $1.5 million, proceeds from the exercise of stock warrants of $3.0 million, and gross proceeds from the sale of common stock related to our IPO and the Private Placements totaling $39.6 million, excluding offering costs paid by us.
For the year ended December 31, 2024, cash provided by financing activities consisted of proceeds from the sale of common stock of $27.7 million, proceeds from the exercise of stock warrants of $0.7 million, and proceeds from our company’s equity awards programs of $0.1 million. 135 For the year ended December 31, 2023, cash provided by financing activities consisted of proceeds from the issuance of notes payable totaling $0.9 million, proceeds from the sale of common stock related to our IPO and private placements totaling $39.6 million, the exercise of stock options of $1.5 million and the exercise of stock warrants of $3.0 million.
Cash Flows The table below summarizes our cash flow activities for the years ended December 31, 2023 and 2022 (in thousands): December 31, December 30, Net cash provided by (used in): 2023 2022 Operating activities $ (20,275 ) $ (3,571 ) Investing activities (14,724 ) (49 ) Financing activities 44,020 (478 ) Net increase (decrease) in cash $ 9,021 $ (4,098 ) 123 Operating Activities During the year ended December 31, 2023, we used $20.3 million cash in operating activities, compared to $3.6 million used during the year ended December 31, 2022.
Cash Flows The table below summarizes our cash flow activities for the years ended December 31, 2024 and 2023 (in thousands): December 31, December 31, Net cash provided by (used in): 2024 2023 Operating activities $ (21,228 ) $ (20,275 ) Investing activities (8,131 ) (14,724 ) Financing activities 28,506 44,020 Net increase (decrease) in cash $ (853 ) $ 9,021 Operating Activities During the year ended December 31, 2024, we used cash from operating activities of $21.2 million, compared to $20.3 million used during the year ended December 31, 2023.
During the year ended December 31, 2023, other expenses consisted of interest expense of $0.2 million, debt discount amortization of $0.6 million, debt extinguishment costs of $0.4 million and financing costs of $3.2 million, while during the same period in 2022, other expenses consisted of interest expense of $1.1 million and debt discount amortization of $0.3 million.
In 2024, other income also includes a gain on the extinguishment of accounts payable of $0.4 million. Other expenses during the year ended December 31, 2023, consisted of $0.2 million of interest expense, $0.6 of debt discount amortization, $0.4 of debt extinguishment costs, and $3.2 million of financing costs.
Additionally, future commercial and regulatory factors beyond our control will impact our clinical development programs and plans. 119 General and Administrative Expenses General and administrative expenses include salaries and other compensation-related costs, including stock-based compensation, for personnel in executive, finance and accounting, business development, operations and administrative roles.
General and Administrative Expenses General and administrative expenses include salaries and other compensation-related costs, including stock-based compensation, for personnel in executive, finance and accounting, business development, operations and administrative roles.
Although we do not expect our estimates to be materially different from amounts incurred, if our estimates of the status and timing of services performed differ from the actual status and timing of services performed, it could result in us reporting amounts that are too high or too low in any particular period.
Non-refundable advance payments for goods and services that will be used in future research and development activities are expensed when the activity has been performed or when the goods have been received rather than when the payment is made. 138 Although we do not expect our estimates to be materially different from amounts incurred, if our estimates of the status and timing of services performed differ from the actual status and timing of services performed, it could result in us reporting amounts that are too high or too low in any particular period.
During the year ended December 31, 2023, other income consisted of interest income on investments in money market funds and other short-term investments of $0.2 million, and during the same period in 2022, other income consisted of a gain on the forgiveness of a PPP loan payable of $0.3 million.
During the year ended December 31, 2024, other income consisted of interest income of $1.4 million from the investment into money market funds and short and long-term investments, while during the same period in 2023, other income consisted of interest income of $0.2 million.
If we raise funds through potential collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates, or to grant licenses on terms that may not be favorable to us.
Additional debt financing, if available, may involve agreements that include restrictive covenants that limit our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends, that could adversely impact our ability to conduct our business. 137 If we raise funds through potential collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates, or to grant licenses on terms that may not be favorable to us.
Recent Accounting Pronouncements For a description of recently issued accounting standards that may have a material impact on our financial statements or will otherwise apply to our operations, please see Note 2 to our audited financial statements appearing elsewhere in this Annual Report. 128 Emerging Growth Company Status As an “emerging growth company,” the Jumpstart Our Business Startups Act of 2012 permits us to take advantage of an extended transition period to comply with new or revised accounting standards applicable to public companies until those standards would otherwise apply to private companies.
Emerging Growth Company Status As an “emerging growth company,” the Jumpstart Our Business Startups Act of 2012 permits us to take advantage of an extended transition period to comply with new or revised accounting standards applicable to public companies until those standards would otherwise apply to private companies.
We estimate accruals for legal expenses when incurred as of each balance sheet date based on the facts and circumstances known to us at that time. Off-Balance Sheet Arrangements During the years ended December 31, 2023 and 2022, we did not have, and we do not currently have, any off-balance sheet arrangements (as defined under SEC rules).
We estimate accruals for legal expenses when incurred as of each balance sheet date based on the facts and circumstances known to us at that time.
Common Stock Issued for Cash Upon Closing of the Company’s Private Placements On May 12, 2023, we entered into a securities purchase agreement (the PIPE 1 SPA) with certain investors (the PIPE 1 Purchasers), pursuant to which we agreed to sell and issue 1,665,213 shares of our common stock in a private placement transaction (the First Private Placement).
The warrants expire five years from the date of grant. Common Stock Issued for Cash Upon Closing of the Company’s Private Placements In May and June 2023, we entered into securities purchase agreements (the “Purchase Agreements”) with certain investors pursuant to which we agreed to sell and issue shares of our common stock in two private placement transactions.
Generally, we issue stock options with only service-based vesting conditions and record the expense for these awards using the straight-line method over the requisite service period. 127 We classify equity-based compensation expense in our statements of operations in the same manner in which the award recipient’s salary and related costs are classified or in which the award recipient’s service payments are classified.
We classify equity-based compensation expense in our statements of operations in the same manner in which the award recipient’s salary and related costs are classified or in which the award recipient’s service payments are classified.
We may be unable to raise additional funds or enter into such other agreements or arrangements when needed on acceptable terms, or at all. Our failure to raise capital or enter into such agreements as, and when needed, could have a material adverse effect on our business, results of operations and financial condition.
We may be unable to raise additional funds or enter into such other agreements or arrangements when needed on acceptable terms, or at all.
The reversal of compensation cost previously recognized for an award that is forfeited because of a failure to satisfy a service or performance condition is recognized in the period of the forfeiture.
The reversal of compensation cost previously recognized for an award that is forfeited because of a failure to satisfy a service or performance condition is recognized in the period of the forfeiture. Generally, we issue stock options with only service-based vesting conditions and record the expense for these awards using the straight-line method over the requisite service period.
Net cash used in investing activities for the year ended December 31, 2022 was $0.05 million, consisting of the purchase of property and equipment. Financing Activities During the year ended December 31, 2023, cash provided from financing activities was $44.0 million, compared to $0.5 million used during the year ended December 31, 2022.
Financing Activities During the year ended December 31, 2024, we provided cash from financing activities of $28.5 million, compared to $44.0 million provided during the year ended December 31, 2023.
Recent Developments The Company currently is engaged in regulatory study start-up activities of a Phase 2, open-label, randomized, and controlled clinical trial designed to evaluate the efficacy and safety of intravenously delivered Olvi-Vec oncolytic VACV for patients with recurrent NSCLC in the United States.
US-Based Phase 2 Trial in NSCLC In October 2024, we announced that the first patient had been dosed in a Phase 2, open-label, randomized, and controlled clinical trial designed to evaluate the efficacy and safety of intravenously delivered Olvi-Vec oncolytic VACV for patients with recurrent non-small cell lung cancer (NSCLC) in the United States.
During the year ended December 31, 2022, under the Newsoara License Agreement, we invoiced and collected $0.2 million relating to supplying product for Newsoara to use in its clinical trials. As the product did not ship during the year ended December 31, 2022, we recorded the cash received as deferred revenue until the product was shipped.
Components of Results of Operations Net Sales During the year ended December 30, 2023, under our license agreement with Newsoara, we invoiced and collected $0.2 million relating to supplying product for Newsoara to use in its clinical trials.
In accordance with our licensing agreement, the Phase 2 clinical trial will be funded in its entirety by our partner in China, Newsoara.
In accordance with our license agreement with our partner in China, Newsoara BioPharma Co. Ltd. (Newsoara), Newsoara is generally obligated to fund the Phase 2 clinical trial in its entirety.
Newsoara is permitted to defer such reimbursement payments until the completion of its next round of financing, which Newsoara expects to occur in 2024. Components of Results of Operations Net sales During the year ended December 31, 2022, under the License Agreement with Newsoara BioPharma Co.
Newsoara is permitted to defer such reimbursement payments until the completion of its next round of financing, which Newsoara expects to occur in 2025.
Significant variations between periods are a result of a $1.0 million increase in employee compensation and related expenses in 2023, primarily related to new employee hires in 2023, and a $1.5 million increase in stock compensation in 2023, primarily related to increased stock grants in 2023 and the cost of stock option repricing in 2023. 121 General and Administrative Expenses The table below summarizes our general and administrative expenses for the years ended December 31, 2023 and 2022 (in thousands): General and Administrative Expenses: December 31, 2023 December 31, 2022 Employee compensation and related expenses $ 2,502 $ 1,520 Stock compensation, including the cost of stock option modifications 4,270 2,047 Professional services 2,781 290 Facility-related expenses 371 319 Insurance expenses 1,078 334 Consulting and contract labor expenses 418 305 Other expenses 148 188 Total general and administrative expenses $ 11,568 $ 5,003 General and administrative expenses were $11.6 million and $5.0 million for the years ended December 31, 2023 and 2022, respectively, an increase of $6.6 million.
Significant variations between periods are primarily a result of a $1.2 million increase in employee compensation in 2024, primarily related to new employee hires in 2024; a $1.2 million increase in stock-related compensation in 2024, relating to the increased cost of stock options and restricted stock units in 2024, and a $4.5 million increase in clinical and regulatory expenses relating to increased clinical trial costs associated with our Phase 3 On Prime Registration trial in 2024 and Phase 2 clinical trial for non-small cell lung cancer, which Newsoara is obligated to fully reimburse per the terms of our agreement; and partially offset by a $0.9 million decrease in manufacturing and laboratory materials in 2024. 133 General and Administrative Expenses The table below summarizes our general and administrative expenses for the years ended December 31, 2024 and 2023 (in thousands): December 31, December 31, General and Administrative Expenses: 2024 2023 Employee compensation and related expenses $ 2,705 $ 2,502 Stock compensation, including the cost of stock options and restricted stock grants 5,024 4,270 Professional services 2,278 2,781 Facility-related expenses 457 371 Insurance expenses 966 1,078 Consulting and contract labor expenses 831 418 Other expenses 445 148 Total general and administrative expenses $ 12,706 $ 11,568 General and administrative expenses were $12.7 million and $11.6 million for the years ended December 31, 2024 and 2023, respectively, an increase of approximately $1.1 million.
Equity Financings Common Stock Issued for Cash Upon Closing of the Company’s IPO On January 30, 2023, we completed our IPO, in which we issued and sold 2,500,000 shares of our common stock at a public offering price of $6.00 per share.
Common Stock Issued for Cash Upon Closing of the Company’s Second Public Offering In May 2024, we completed an underwritten offering of our common stock, in which we issued and sold 7,500,000 shares of our common stock at a price of $4.00 per share, which included 625,000 shares of common stock at $4.00 per share pursuant to the underwriters’ partial exercise of their option to purchase additional shares of common stock.
If we are unable to raise additional funds when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves. 126 Critical Accounting Policies and Significant Judgments and Estimates This Management’s Discussion and Analysis of Financial Condition and Results of Operations is based on our financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States (GAAP).
If we are unable to raise additional funds when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
The total gross proceeds of the IPO were $15.9 million, and we raised $12.6 million in net proceeds after deducting underwriting discounts and commissions and offering expenses payable by us.
The total gross proceeds received from the offering were $30.0 million and we raised $27.7 million in net proceeds after deducting underwriting discounts and commissions and offering expenses payable by us. Included in the offering were accompanying warrants to purchase 7,500,000 shares of common stock with an exercise price of $5.25 per share.
Significant variations between periods are a result of a $1.0 million increase in employee related costs in 2023, primarily due to new employee hires in 2023; a $2.2 million increase in stock compensation expense in 2023, primarily due to increased stock grants in 2023 and the cost of stock option repricing in 2023; a $2.5 million increase in professional service expenses in 2023, primarily resulting from increased corporate legal costs and other professional services related to costs of being a newly publicly-traded company; and a $0.7 million increase in insurance expenses, primarily due to increased D&O insurance costs. 122 Other Income (Expenses) Other income (expenses), net, were $4.1 million and $1.1 million for the years ended December 31, 2023 and 2022, respectively.
Significant variations between periods are primarily a result of a $0.2 million increase in employee compensation in 2024, a $0.8 million increase in stock compensation expense in 2024, due to the increase in the cost of stock options and restricted stock units in 2024, a $0.4 million increase in consulting and contract labor expenses in 2024, primarily resulting from increased accounting and finance costs in 2024, partially offset by a $0.5 million decrease in professional services, primarily resulting from the decrease in legal expenses in 2024.
We expect our existing cash and cash equivalents, and short-term investments, will last for at least the next 12 months. Due to the funds received through these offerings, and the conversion of preferred stock and convertible notes payable upon the closing of the IPO, we had shareholders’ equity of $19.5 million at December 31, 2023.
During the year ended December 31, 2024, we closed a second public offering and received $27.7 million of net proceeds from that offering. Due to the funds received through these offerings, and the conversion of preferred stock and convertible notes payable upon the closing of the IPO, we have shareholders’ equity of $27.9 million at December 31, 2024.
The primary non-cash expense during both periods was equity-related expenses, totaling $9.3 million and $2.4 million during the years ended December 31, 2023 and 2022, respectively. We had a $3.3 million decrease in operating assets and liabilities during the year ended December 31, 2023, compared to $1.7 million decrease during the year ended December 31, 2022.
The primary non-cash expense during both periods was stock-related compensation totaling $8.1 million and $6.1 million during the years ended December 31, 2024 and 2023, respectively, a gain on the extinguishment of accounts payable of $0.4 million in 2024; and the fair value of warrants issued in connection with the conversion of convertible notes of $3.2 million in 2023.
As reflected in the accompanying financial statements, we experienced recurring losses from operations since inception and incurred a net loss of $28.3 million and used cash in operations of $20.3 million during the year ended December 31, 2023. These factors raise substantial doubt about our ability to continue as a going concern.
There were no other expenses during the year ended December 31, 2024. Liquidity and Capital Resources We have experienced recurring losses from operations since inception and incurred a net loss of $29.9 million and used cash in operations of $21.2 million during the year ended December 31, 2024.
At December 31, 2023, we had cash and cash equivalents, and short-term investments, on hand in the amount of $23.2 million. During the year ended December 31, 2023, we closed our initial public offering (IPO) and two private placements (Private Placements) and received $37.8 million of aggregate net proceeds from these offerings.
Our failure to raise capital or enter into such agreements as and when needed, could have a material adverse effect on our business, results of operations and financial condition. 129 During the year ended December 31, 2023, we closed our IPO and two private placements (the Private Placements) and received $37.8 million of net proceeds from these offerings.
Since inception, we have funded our operations primarily through equity and debt financings and licensing income, and we expect to continue to rely on these sources of capital in the future. No assurance can be given that any future financing will be available or, if available, that it will be on terms that are satisfactory to us.
The failure to receive all or some of the committed proceeds would exhaust our available capital resources sooner than expected and will require us to obtain further funding to achieve our business objectives. 134 No assurance can be given that any future financing will be available or, if available, that it will be on terms that are satisfactory to the Company.
Removed
We also received commitments through the Private Placements for the funding of an additional $24.0 million that were due by November 15, 2023. In November 2023, we agreed to extend the funding deadline for $2.0 million of the remaining committed investment amounts to March 31, 2024.
Added
We expect our cash, cash equivalents and short-term investments, totaling $30.9 million at December 31, 2024, to last until the first quarter of 2026. Recent Developments Underwritten Public Offering On March 26, 2025, we completed an underwritten offering of 3,000,000 shares of our common stock at an offering price of $3.50 per share.
Removed
The investor who was obligated to fund $22.0 million of the remaining committed investment amounts has not made such payments and has indicated that he does not intend to comply with his investment commitments through the Private Placements. We are currently evaluating our potential remedies with respect to this investor’s non-compliance with his contractual obligations to us.
Added
The gross proceeds received from the offering were $10.5 million before deducting underwriting discounts and commissions and estimated offering expenses payable by the Company.
Removed
Ltd., dated September 27, 2021 (the Newsoara License Agreement) and the License Agreement with ELIAS Animal Health LLC, dated November 15, 2021, as amended, we recognized revenue of $11.1 million, with a 10% foreign income tax of $1.1 million being recorded as a provision for foreign income taxes relating to the Newsoara License Agreement.
Added
During the quarter ended September 30, 2024, the Company entered into a Clinical Trial Services Agreement with Hong Kong Tigermed Consulting Co., Ltd., to provide regulatory and development support services for the NSCLC trial in the United States.
Removed
During the year ended December 31, 2023, we shipped the product to Newsoara and thus recognized the revenue.
Added
During the year ended December 31, 2024, we recognized revenue of $0.01 million relating to the Company’s license agreement with ELIAS Animal Health, LLC.
Removed
A change in the outcome of any of these variables with respect to the development of our current and future product candidates would significantly change the costs and timing associated with the development of those product candidates. Research and development activities are central to our business model.
Added
Other Income (Expenses), net Other income (expenses), net, were $1.8 million and $(4.1) million for the years ended December 31, 2024 and 2023, respectively.
Removed
Liquidity and Capital Resources Going Concern The accompanying financial statements have been prepared on a going concern basis, which contemplates the realization of assets and the settlement of liabilities and commitments in the normal course of business.
Added
As of December 31, 2024, we had cash and short-term investments of $30.9 million, however we do not have any committed external source of funds or other support for our development efforts.
Removed
At December 31, 2023, we had cash and cash equivalents, and short-term investments, in the amount of $23.2 million. The ability to continue as a going concern is dependent on us attaining and maintaining profitable operations in the future and raising additional capital to meet our obligations and repay our liabilities arising from normal business operations when they come due.
Added
Until we can generate sufficient product revenue to finance our cash requirements, which we may never do, we expect to finance our future cash needs through a combination of public or private equity offerings and debt financings, or other capital sources such as potential collaborations, strategic alliances, licensing arrangements and other arrangements.
Removed
Net cash provided by financing activities during the year ended December 31, 2022 consisted of proceeds from the issuance of notes payable totaling $1.1 million and the exercise of stock warrants of $0.1 million, while cash used in financing activities for the year ended December 31, 2022 was $0.1 million related to the repayment of convertible notes payable and $1.6 million for the payment of deferred offering costs.
Added
Based on our research and development plans, we expect that our existing cash balance may not enable us to fund our planned operating expenses and capital expenditure requirements for at least the next 12 months from the date of filing of this Annual Report.
Removed
In February 2023, we sold an additional 153,000 shares of common stock at $6.00 per share pursuant to the underwriters’ partial exercise of their option to purchase additional shares of common stock.
Added
We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.
Removed
The purchase price per share of common stock was $20.00 per share. The initial closing of the First Private Placement occurred in May 2023 (the PIPE 1 Initial Closing) subject to customary closing conditions.
Added
In addition, because the design and outcome of our anticipated and any future clinical trials is highly uncertain, we cannot reasonably estimate the actual amounts necessary to successfully complete the development and commercialization of Olvi-Vec or any future product candidates. Our existing cash balance may not be sufficient to complete the development of Olvi-Vec or any other product candidate.
Removed
The total gross proceeds to us at the PIPE 1 Initial Closing from the First Private Placement are expected to be approximately $33.3 million, including $1.5 million from the cancellation of certain of our bridge loans and accrued interest.
Added
Additionally, although we have commitments from investors to fund the remaining aggregate investment amounts in connection with our Private Placements, we may not receive some or all of the committed proceeds, due to ongoing liquidity constraints or other factors.
Removed
Two of the PIPE 1 Purchasers were contractually obligated to fund up to $17.5 million of such PIPE 1 Purchasers’ investment amounts following the PIPE 1 Initial Closing but no later than November 15, 2023. During the year ended December 31, 2023, we received $6.0 million of the committed amount of $17.5 million.
Added
The net change in operating assets and liabilities during the year ended December 31, 2024, used cash of $0.8 million, compared to $3.3 million used during the year ended December 31, 2023.
Removed
As of December 31, 2023, we had sold 1,017,079 shares of our common stock under the PIPE 1 SPA resulting in gross and net proceeds to us of $20.3 million and $19.8 million, respectively. 124 On June 9, 2023, we entered into another securities purchase agreement (the PIPE 2 SPA, and, together with the PIPE 1 SPA, the Purchase Agreements) with certain investors (the PIPE 2 Purchasers), pursuant to which we agreed to sell and issue 900,000 shares of our common stock in a private placement transaction (the Second Private Placement, and, together with the First Private Placement, the Private Placements).
Added
The primary source of cash relating to operating assets and liabilities during the year ended December 31, 2024 was the increase in accounts payable and accrued expenses of $2.2 million; and the primary uses of cash were the decrease in accrued payroll and payroll taxes of $1.1 million, and the increase in prepaid expenses and other assets of $0.5 million.
Removed
The purchase price per share of common stock was $20.00 per share. The initial closing of this Second Private Placement occurred in June 2023 (the PIPE 2 Initial Closing), subject to customary closing conditions. The total gross proceeds to us from the Second Private Placement are expected to be approximately $18.0 million.
Added
Investing Activities Net cash used in investing activities for the years ended December 31, 2024 was $8.1 million, consisting of the net purchases of short and long-term investments of $7.7 million, and the purchase of property and equipment of $0.4 million.
Removed
One of the PIPE 2 Purchasers was contractually obligated to fund up to $12.5 million of such PIPE 2 Purchaser’s investment amounts following the PIPE 2 Initial Closing, but no later than November 15, 2023. We did not receive any part of such PIPE 2 Purchaser’s committed investment amount during the year ended December 31, 2023.
Added
Equity Financings Common Stock Issued for Cash Upon Closing of the Company’s Third Public Offering On March 26, 2025, the Company completed an underwritten offering of 3,000,000 shares of its common stock at an offering price of $3.50 per share.
Removed
As of December 31, 2023, we had sold 275,000 shares of our common stock under the PIPE 2 SPA resulting in gross and net proceeds to us of $5.5 million and $5.3 million, respectively.
Added
The gross proceeds received from the offering were $10.5 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by the Company.

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Item 7A. Quantitative and Qualitative Disclosures About Market Risk

Market Risk — interest-rate, FX, commodity exposure

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Biggest changeInflation generally affects us by increasing our cost of labor. We do not believe that inflation had a material effect on our business, financial condition or results of operations during the years ended December 31, 2023 and 2022. Item 8.
Biggest changeInflation generally affects us by increasing our cost of labor. We do not believe that inflation had a material effect on our business, financial condition or results of operations during the years ended December 31, 2024 and 2023. Item 8.

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