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What changed in Moleculin Biotech, Inc.'s 10-K — 2023 vs 2024

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Paragraph-level year-over-year comparison of Moleculin Biotech, Inc.'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.

+485 added−505 removedSource: 10-K (2025-03-21) vs 10-K (2024-03-22)

Top changes in Moleculin Biotech, Inc.'s 2024 10-K

485 paragraphs added · 505 removed · 324 edited across 6 sections

Item 1. Business

Business — how the company describes what it does

174 edited+81 added−139 removed243 unchanged
Biggest changeWP1066 IND cleared for GBM 1 Open to an investigator to lead a study Trial has not begun Trial has not begun Trial has not begun WP1066 in combination with radiation IND allowed for Adult GBM / US 2 External Open to an investigator to lead a study Used as reference for next trial Trial has not begun Trial has not begun WP1066 in combination with radiation therapy IND allowed for IIT led Adult GBM / US 1B/2 External Investigator expected to begin recruiting in 2024 Trial has not begun Trial has not begun Trial has not begun WP1066 IIT / Pediatric Brain Tumors / US 1 External Concluded in February 2023 with a dose level at 8mg/kg Concluded 10 subjects enrolled and treated over 3 dose levels up to 8 mg/kg No drug related serious adverse events noted 1 DIPG subject had a temporary clinical response WP1220 MB-201 / CTCL / Poland 1B / Proof of Concept Concluded and CSR completed Believe results warrant a Phase 2 study Met safety endpoints 60% of subjects documented PR WP1122 MB-301 / COVID-19 / UK 1A Completed; Established RP2D Drug at RP2D was tolerable and safe Met safety endpoints N/A as in healthy volunteer subjects WP1122 IND approved for GBM 1B/2 External Exploring for an investigator to lead a study Trial has not begun Trial has not begun Trial has not begun Notes for Table 1 : 1) In MB-104 MLFS means "morphological leukemia-free state"; 2) In MB-105 we consider complete responses (CR) or complete response with incomplete recovery of the bone marrow (CRi) as where the bone marrow aspirate (BMAs) show leukemic blasts of less than 5%; 3) This is a summary of the detailed clinical discussion below and does not include compassionate use/right-to-try usage of our drug candidates; 4) Complete Response Composite (CRc) includes CRs and CRis; 5) Overall Response Rate (ORR) includes CRc and Partial Response (PR); 6) “Met safety endpoints” means that no drug-related serious and unexpected adverse events occurred as defined in the trial protocol 7) All data presented are preliminary unless a CSR or an investigator's final report has been issued for the trial referenced - specifically the data for MB-106 and MB-107 are preliminary and subject to change; 8) With regard to safety and human activity summaries please see the detailed discussion below; 9) MB-106 Phase 1 included “all-comers” or subjects with unlimited lines of prior therapy while Phase 2 included only subjects as 1 st thru 3 rd line of therapy, and 10) MB-107 Phase 2 has no ongoing treatment active and is following nine of fifteen subjects for overall survival (OS). 4 Table of Contents In the US and Europe, since our inception, we or independent investigators have approval to begin, are currently conducting or have completed thirteen internally or externally funded clinical trials for four of our drug candidates – Annamycin, WP1066, WP1220, and WP1122, as listed above.
Biggest changeConcluded 10 subjects enrolled and treated over 3 dose levels up to 8 mg/kg No drug related serious adverse events noted 1 DIPG subject had a temporary clinical response WP1220 MB-201 / CTCL / Poland 1B / Proof of Concept Concluded and CSR completed Believe results warrant a Phase 2 study Met safety endpoints 60% of subjects documented PR WP1122 MB-301 / COVID-19 / UK 1A Completed; Established RP2D Drug at RP2D was tolerable and safe Met safety endpoints N/A as in healthy volunteer subjects WP1122 IND approved for GBM 1B/2 External Exploring for an investigator to lead a study Trial has not begun Trial has not begun Trial has not begun 4 Table of Contents Notes for Table 1 : 1) This is a summary of the detailed clinical discussion below and does not include compassionate use/right-to-try usage of our drug candidates; 2) Complete Response Composite (CRc) includes CRs and CRi’s; 3) Overall Response Rate (ORR) includes CRc and Partial Response (PR); 4) “Met safety endpoints” means that no drug-related serious and no unexpected adverse event (only one serious in MB-105) occurred as defined in the trial protocol 5) All data presented are preliminary (and subject to change) unless a CSR or an investigator's final report has been issued for the trial referenced; 6) With regard to safety and human activity summaries please see the detailed discussion below; and, 7) MB-106 Phase 1 included “all-comers” or subjects with unlimited lines of prior therapy while Phase 2 included only subjects as 1 st thru 3 rd line of therapy.
While pancreatic cancer only accounts for 3.3% of all cancer diagnoses, it has the highest mortality rate of all cancers and is the third leading cause of cancer-related deaths in the US, behind lung and colon cancer. The most effective treatment for pancreatic cancer is surgery, but fewer than 20% of cases are eligible for a surgical approach.
While pancreatic cancer only accounts for 3% of all cancer diagnoses, it has the highest mortality rate of all cancers and is the third leading cause of cancer-related deaths in the US, behind lung and colon cancer. The most effective treatment for pancreatic cancer is surgery, but fewer than 20% of cases are eligible for a surgical approach.
The NCE exclusivity scheme is complicated and evolving; for that reason, although we believe that some of our products will qualify for five-year NCE exclusivity, we cannot be certain that will receive such exclusivity, or that if we do, the exclusivity will effectively protect our market position.
The NCE exclusivity scheme is complicated and evolving; for that reason, although we believe that some of our products will qualify for five-year NCE exclusivity, we cannot be certain we will receive such exclusivity, or that if we do, the exclusivity will effectively protect our market position.
In the United States, the FDA regulates pharmaceutical products such as our product candidates under the Federal Food, Drug, and Cosmetic Act and implementing regulations. Pharmaceutical products are also subject to other federal, state and local statutes and regulations. Obtaining regulatory approvals and complying with post-approval requirements generally is expensive, labor-intensive and time-consuming.
In the United States, the FDA regulates pharmaceutical products such as our product candidates under the Federal Food, Drug, and Cosmetic Act (FDCA) and implementing regulations. Pharmaceutical products are also subject to other federal, state and local statutes and regulations. Obtaining regulatory approvals and complying with post-approval requirements generally is expensive, labor-intensive and time-consuming.
Taken together, these factors suggest that Annamycin could represent an important treatment to help address a significant unmet need in patients with STS lung metastases. In February 2021, we announced that a preclinical study in animals had suggested a possible significant therapeutic benefit of Annamycin against metastatic osteosarcoma.
Taken together, these factors suggest that Annamycin could represent an important treatment to help address a significant unmet need in patients with STS lung metastases. In February 2021, we also announced that a preclinical study in animals had suggested a possible significant therapeutic benefit of Annamycin against metastatic osteosarcoma.
The FDA may approve the proposed product before the expiration of the regulatory stay if a court finds the patent invalid or not infringed or if the court shortens the period because the parties have failed to cooperate in expediting the litigation.
The FDA may approve the proposed product before the expiration of the regulatory stay if a court finds the patent invalid or not infringed or if the court shortens the period because the parties have failed to cooperate in expediting the litigation. Regulatory Exclusivities.
A portion of the patent term lost during product development and FDA review of an NDA is restored if approval of the application is the first permitted commercial marketing of a drug containing the active ingredient.
Patent Term Restoration. A portion of the patent term lost during product development and FDA review of an NDA is restored if approval of the application is the first permitted commercial marketing of a drug containing the active ingredient.
There was no limit on how many prior therapies a subject could have prior to entering this study. Most subjects were heavily treated with other therapies prior to entering our trial with our treatment representing the fourth median therapy for all subjects in the Phase 1B and Phase 2 portion of the trial (range of two to twelve ).
There was no limit on how many prior therapies a subject could have prior to entering this study. Most subjects were heavily treated with other therapies prior to entering our trial with our treatment representing the seventh median therapy for all subjects in the Phase 1B and Phase 2 portion of the trial (range of two to twelve).
We believe such compounds may provide an opportunity to cut off the fuel supply of tumors by taking advantage of their high degree of dependence on glucose in comparison to healthy cells, as well as viruses that also depend upon glycolysis and glycosylation to infect and replicate.
We believe such compounds may provide an opportunity to cut off the energy supply of tumors by taking advantage of their high degree of dependence on glucose in comparison to healthy cells, as well as viruses that also depend upon glycolysis and glycosylation to infect and replicate.
In July 2021, we formed Moleculin Amsterdam B.V., a wholly owned subsidiary, primarily to act as our legal representative for clinical trials in Europe for Moleculin Biotech, Inc. 19 Table of Contents Competition We operate in a highly competitive segment of the pharmaceutical market, which market is highly competitive as a whole.
In July 2021, we formed Moleculin Amsterdam B.V., a wholly owned subsidiary, primarily to act as our legal representative for clinical trials in Europe for Moleculin Biotech, Inc. 17 Table of Contents Competition We operate in a highly competitive segment of the pharmaceutical market, which market is highly competitive as a whole.
Any agency or judicial enforcement action could have a material adverse effect on us. 20 Table of Contents Development and Approval The process required by the FDA before a pharmaceutical product may be marketed in the US generally involves the following: • Completion of preclinical laboratory tests, animal studies and formulation studies; • Submission to the FDA of an Investigational New Drug application, or IND, which must become effective before human clinical trials may begin; • Performance of adequate and well-controlled human clinical trials according to the FDA’s regulations commonly referred to as good clinical practices (GCP) and any additional requirements for the protection of human research subjects and their health information, to establish the safety and efficacy of the proposed pharmaceutical product for its intended use; • Submission to the FDA of an NDA for marketing approval that includes substantial evidence of safety and effectiveness from results of clinical trials, as well as the results of preclinical testing, detailed information about the chemistry, manufacturing and controls, and proposed labeling and packaging for the product; • Review of the product candidate by an FDA advisory committee, if applicable; • Satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the pharmaceutical product is produced, to assess compliance with current good manufacturing practice, or cGMP, requirements, to assure that the facilities, methods and controls are adequate to preserve the pharmaceutical product’s identity, strength, quality and purity; • Potential FDA audit of the preclinical and clinical trial sites that generated the data in support of the NDA; and • FDA review and approval of the NDA, including agreement on post-marketing commitments, if applicable.
Any agency or judicial enforcement action could have a material adverse effect on us. 18 Table of Contents Development and Approval The process required by the FDA before a pharmaceutical product may be marketed in the US generally involves the following: • Completion of preclinical laboratory tests, animal studies and formulation studies; • Submission to the FDA of an Investigational New Drug application, or IND, which must become effective before human clinical trials may begin; • Performance of adequate and well-controlled human clinical trials according to the FDA’s regulations commonly referred to as good clinical practices (GCP) and additional requirements for the protection of human research subjects and their health information, to establish the safety and effectiveness of the proposed product; • Submission to the FDA of an NDA seeking marketing approval that includes substantial evidence of safety and effectiveness from results of clinical trials, as well as the results of preclinical testing, detailed information about the chemistry, manufacturing and controls, and proposed labeling and packaging for the product; • Review of the product candidate by an FDA advisory committee, if applicable; • Satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the pharmaceutical product is produced, to assess compliance with current good manufacturing practice, or cGMP, requirements, to assure that the facilities, methods and controls are adequate to preserve the pharmaceutical product’s identity, strength, quality and purity; • Potential FDA audit of the preclinical and clinical trial sites that generated the data in support of the NDA; and • FDA review and approval of the NDA, including agreement on post-marketing commitments, if applicable.
On August 7, 2023, we successfully completed the second cohort at 230 mg/m 2 of Annamycin in this combination study. Four subjects were treated in this cohort, one is believed to be relapsed from one or more prior therapies and three are believed to be refractory to up to three prior therapies.
In August 2023, we successfully completed the second cohort at 230 mg/m 2 of Annamycin in this combination study. Four subjects were treated in this cohort, one is believed to be relapsed from one or more prior therapies and three are believed to be refractory to up to three prior therapies.
Since 2021, we have been working on developing an appropriate IV formulation for WP1066 or its analogs. As a result of these studies, we believe we have now identified a candidate formulation that is worthy of IND-enabling preclinical testing, which is now underway.
Since 2020, we have been working on developing an appropriate IV formulation for WP1066 or its analogs. As a result of these studies, we believe we have now identified a candidate formulation that is worthy of IND-enabling preclinical testing, which is now underway.
The FDA usually will inspect the facilities at which the product candidate is manufactured, and will not approve the product candidate unless the agency determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent production of the product within required specifications.
The FDA usually inspects facilities at which the product candidate is manufactured, and will not approve the product candidate unless the agency determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent production of the product within required specifications.
We believe that the potential for such future incidences, however, does not outweigh the significant elimination of cardiotoxicity to date as reflected in the Expert’s reports. The Importance of the Unmet Need in 2 nd Line Therapies for AML There are approximately 160,000 people with AML worldwide with about 20,000 newly diagnosed patients annually in the U.S.
We believe that the potential for such future incidences, however, does not outweigh the significant elimination of cardiotoxicity to date as reflected in the Expert’s reports. 6 Table of Contents The Importance of the Unmet Need in 2 nd Line Therapies for AML There are approximately 160,000 people with AML worldwide with about 20,000 newly diagnosed patients annually in the U.S.
Although we periodically monitor the FDA compliance of our third-party manufacturers, we cannot be certain that our present or future third-party manufacturers will consistently comply with cGMP and other applicable FDA regulatory requirements. Discovery of problems with a product after approval may result in restrictions on a product, manufacturer or NDA sponsor, including withdrawal of the product from the market.
Although we periodically monitor the FDA compliance of our third-party manufacturers, we cannot be certain that our present or future third-party manufacturers will consistently comply with cGMP and other applicable FDA regulatory requirements. 22 Table of Contents Discovery of problems with a product after approval may result in restrictions on a product, manufacturer or NDA sponsor, including withdrawal of the product from the market.
Additionally, we will rely on external collaborations for testing other molecules in the WP1122 portfolio against other hard to treat viruses such as HIV, Dengue fever, and Zika. Funding Strategy By “internally funded” we mean that the primary costs of the preclinical activity and clinical trials are funded and sponsored by us.
Additionally, we will rely on external collaborations for testing other molecules in the WP1122 portfolio against other hard to treat viruses such as HIV, Dengue fever, and Zika. 13 Table of Contents Funding Strategy By “internally funded” we mean that the primary costs of the preclinical activity and clinical trials are funded and sponsored by us.
Preclinical testing demonstrated that WP1220, a synthetic compound, potently inhibits the activity of p-STAT3 and the growth of CTCL cell lines. This Phase 1 study was designed to demonstrate the safety and efficacy of WP1220 after topical treatment of CTCL. Of five subjects enrolled, eleven lesions were assessed according to the CAILS scoring system.
Preclinical testing demonstrated that WP1220, a synthetic compound, potently inhibits the activity of p-STAT3 and the growth of CTCL cell lines. This Phase 1 study was designed to demonstrate the safety and efficacy of WP1220 after topical treatment of CTCL. 12 Table of Contents Of five subjects enrolled, eleven lesions were assessed according to the CAILS scoring system.
Our Core Technologies Our core technologies consist of the following programs: a) Annamycin or L-Annamycin is a “next generation” anthracycline (one of the most common classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks and their susceptibility to multidrug resistance mechanisms.
Our core technologies consist of the following programs: a) Annamycin or L-Annamycin is a “next generation” anthracycline (one of the most widely used classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks and their susceptibility to multidrug resistance mechanisms.
In the case of acute leukemia, anthracyclines are typically used in “induction therapy,” where the goal is often to induce sufficient remission of patients’ blood-born tumor cells to allow for a potentially curative bone marrow transplant. Two key factors limit the safety and effectiveness of anthracyclines: cardiotoxicity and multidrug resistance.
In the case of acute leukemia, anthracyclines are typically used in “induction therapy,” where the goal is often to induce sufficient remission of patients’ bloodborne tumor cells to allow for a potentially curative bone marrow transplant. Two key factors limit the safety and effectiveness of anthracyclines: cardiotoxicity and multidrug resistance.
Clinical trials involve the administration of the product candidate to healthy volunteers or subjects with the targeted disease under the supervision of qualified investigators, generally physicians not employed by or under the clinical trial sponsor’s control.
Clinical trials involve the administration of the product candidate to healthy volunteers or subjects with the targeted disease under the supervision of qualified investigators, generally physicians not employed by or under the control of the clinical trial sponsor.
In February 2023, the Emory physician-sponsored clinical trial for the treatment of pediatric brain tumors with WP1066 concluded with treating a total of ten subjects in all three cohorts of the Phase 1 dose escalation portion of the trial. The third cohort dosing was deemed safe at 8mg/kg.
In February 2023, the Emory physician-sponsored clinical trial for the treatment of pediatric brain tumors with an oral formulation of WP1066 concluded with treating a total of ten subjects in all three cohorts of the Phase 1 dose escalation portion of the trial. The third cohort dosing was deemed safe at 8mg/kg.
In 2019, the FDA granted ODD for WP1066 for the treatment of glioblastoma, which means the agency believes we have established a medically plausible basis for using the drug to treat glioblastoma.
In 2019, the FDA granted ODD for WP1066 for the treatment of glioblastoma, which means the agency believes, in part, that we have established a medically plausible basis for using the drug to treat glioblastoma.
Failure to comply with the applicable requirements may subject an applicant to administrative or judicial enforcement action, which could include refusal to permit clinical trials to be conducted, refusal to approve an application, withdrawal of an approval, issuance of a warning letter, product recall, product seizure, suspension of production or distribution, fines, refusals of government contracts, and restitution, disgorgement or civil or criminal penalties.
Failure to comply with the applicable requirements may subject an applicant to administrative or judicial enforcement action, which could include refusal to permit clinical trials to be conducted, refusal to approve an application, placing a clinical trial on hold, withdrawal of an approval, issuance of a warning letter, product recall, product seizure, suspension of production or distribution, fines, refusals of government contracts, and restitution, disgorgement or civil or criminal penalties.
The maximum period of restoration is five years, and the patent cannot be extended to more than 14 years from the date of FDA approval of the product. Only one patent claiming each approved product is eligible for restoration and the patent holder must apply for restoration within 60 days of approval. The U.S.
The maximum period of restoration is five years, and the patent cannot be extended to more than 14 years from the date of FDA approval of the product. Only one patent claiming each approved product is eligible for restoration and the patent holder must apply for restoration within 60 days of approval. In consultation with the FDA, the U.S.
In addition, the Inflation Reduction Act (IRA), among other things, (1) directs HHS to negotiate the price of certain high-cost, single-source drugs and biologics covered under Medicare and (2) imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation.
Further, the Inflation Reduction Act (IRA), among other things, (1) directs HHS to negotiate the price of certain high-cost, single-source drugs and biologics covered under Medicare and (2) imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation.
Working Environment Our headquarters and laboratory are in Houston, Texas, and our workforce, as of year-end 2023, consisted of eighteen full and part-time employees, in the US which are leveraged with other service providers and contractors worldwide working in a primarily virtual environment. We do not have manufacturing facilities and all manufacturing activities are contracted out to third parties.
Working Environment Our headquarters and laboratory are in Houston, Texas, and our workforce, as of year-end 2024, consisted of 17 full and part-time employees, in the US which are leveraged with other service providers and contractors worldwide working in a primarily virtual environment. We do not have manufacturing facilities and all manufacturing activities are contracted out to third parties.
As discussed above, if a product is similar, but not identical, to an already approved product, it may be submitted for approval via an NDA under section 505(b)(2) of the FD&C Act. Unlike an ANDA, this does not excuse the sponsor from demonstrating the proposed product's safety and effectiveness.
As discussed above, if a product is similar, but not identical, to an already approved product, it may be submitted for approval via an NDA under section 505(b)(2) of the FDCA. Unlike an ANDA, this does not excuse the sponsor from demonstrating the proposed product's safety and effectiveness.
We believe WP1122 should be well suited as a treatment for highly glycolytic cancers such as GBM and pancreatic cancer. In addition to the market for GBM described above, pancreatic cancer is a rare and difficult to treat form of cancer.
We believe WP1122 should be well suited as a treatment for highly glycolytic cancers such as GBM and pancreatic cancer. 16 Table of Contents In addition to the market for GBM described above, pancreatic cancer is a rare and difficult to treat form of cancer.
Additionally, a manufacturer of an investigational drug for a serious disease or condition is required to make available, such as by posting on its website, its policy on evaluating and responding to requests for individual patient access to such investigational drug.
Additionally, a manufacturer of an investigational drug for a serious disease or condition is required to make available, such as by posting on its website, its policy on evaluating and responding to requests for individual patient access to such investigational drug. NDA Submission and Review.
To further explain this, we consider Annamycin to be a "next generation" anthracycline, unlike any currently approved anthracyclines, as it is designed to avoid multidrug resistance mechanisms and cardiotoxicity, recognizing that the efficacy of all currently approved anthracyclines is limited by both multidrug resistance and cardiotoxicity. Our preclinical studies and clinical trials support this intended design.
Annamycin Program Overview We consider Annamycin to be a "next generation" anthracycline, unlike any currently approved anthracyclines, as it is designed to avoid multidrug resistance mechanisms and cardiotoxicity, recognizing that the efficacy of all currently approved anthracyclines is limited by both multidrug resistance and cardiotoxicity. Our preclinical studies and clinical trials support this intended design.
In some instances, an NDA approval may be obtained based on Phase 2 clinical data, often with the understanding that the approved drug can be sold subject to a confirmatory trial to be conducted post-approval. Additionally, post-approval studies, also referred to as Phase 4 clinical trials, may be conducted after initial marketing approval.
In some instances, an NDA approval may be obtained based on Phase 2 clinical data, often with the understanding that the approved drug can be sold subject to a confirmatory trial to be conducted post-approval. 19 Table of Contents Additionally, post-approval studies, also referred to as Phase 4 clinical trials, may be conducted after initial marketing approval.
The IRB also approves the informed consent form that each study subject (or his or her legal representative) must sign, and is responsible for monitoring the conduct of the study until completed. 21 Table of Contents Clinical testing.
The IRB also approves the informed consent form that each study subject (or his or her legal representative) must sign, and is responsible for monitoring the conduct of the study until completed. Clinical testing.
An applicant receiving a complete response letter may either revise and resubmit the NDA or withdraw the application. FDA approval of an NDA may impose significant limitations that could weaken the commercial value of the product.
An applicant receiving a complete response letter may either revise and resubmit the NDA or withdraw the application. 20 Table of Contents FDA approval of an NDA may impose significant limitations that could weaken the commercial value of the product.
Drug Candidate FDA ODD - Indication FDA Fast Track - Designation (FTD) FDA Rare Pediatric Disease Priority Review Voucher (PRV) Program Annamycin Yes – AML, Soft Tissue Sarcoma Yes – AML, Soft Tissue Sarcoma No WP1066 Yes - GBM No Yes – Ependymoma, diffuse intrinsic pontine glioma (DIPG), medulloblastoma and atypical teratoid rhabdoid tumor WP1122 Yes - GBM Yes - GBM No 16 Table of Contents Overview of The Market for Our Oncology Drugs The American Cancer Society (https://www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/2024-cancer-facts-figures.ht ml) estimates that cancer continues to be the second most common cause of death in the US, after heart disease.
Drug Candidate FDA ODD - Indication FDA Fast Track - Designation (FTD) FDA Rare Pediatric Disease Priority Review Voucher (PRV) Program Annamycin Yes – AML, Soft Tissue Sarcoma Yes – AML, Soft Tissue Sarcoma No WP1066 Yes - GBM No Yes – Ependymoma, diffuse intrinsic pontine glioma (DIPG), medulloblastoma and atypical teratoid rhabdoid tumor WP1122 Yes - GBM Yes - GBM No Overview of The Market for Our Oncology Drugs The American Cancer Society (https://www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/2025-cancer-facts-figures.html ) estimates that cancer continues to be the second most common cause of death in the US, after heart disease.
We received ODD for WP1066 for the treatment of glioblastoma in 2019. If WP1066 is timely approved for the treatment of any of the following pediatric diseases, we may qualify for a Rare Pediatric Disease Priority Review Voucher: ependymoma, medulloblastoma, diffuse intrinsic pontine glioma, or atypical teratoid rhabdoid tumor, provided that related statutory sunset provisions are extended.
If WP1066 is timely approved for the treatment of any of the following pediatric diseases, we may qualify for a Rare Pediatric Disease Priority Review Voucher: ependymoma, medulloblastoma, diffuse intrinsic pontine glioma, or atypical teratoid rhabdoid tumor, provided that related statutory sunset provisions are extended.
These numbers also do not account for the effect the COVID-19 pandemic has likely had on cancer diagnoses and deaths because they are projections based on reported cases through 2019 and deaths through 2020.
These numbers also do not account for the effect the COVID-19 pandemic has likely had on cancer diagnoses and deaths because they are projections based on reported cases through 2021 and deaths through 2022.
Given this backdrop, we believe the best initial pathway for Annamycin is to pursue the second-line treatment of STS lung metastasis. Per the American Cancer Society, in 2024, an estimated 66,440 new cases of pancreatic cancer will be diagnosed in the US and 51,750 people will die from the disease.
Given this backdrop, we believe the best initial pathway for Annamycin is to pursue the second-line treatment of STS lung metastasis. Per the American Cancer Society, in 2025, an estimated 67,440 new cases of pancreatic cancer will be diagnosed in the US and 51,980 people will die from the disease.
But again, this means that only a small subset of the deemed “Un-Fit” patients, approximately 15% of all AML patients, achieve this positive outcome.
But again, this means that only a small subset of the deemed “Unfit” patients, approximately 15% of all AML patients, achieve this positive outcome.
The WP1122 Portfolio Program We have a license agreement with MD Anderson pursuant to which we have been granted a royalty-bearing, worldwide, exclusive license for the patent and technology rights related to our WP1122 Portfolio and similar molecules focused on inhibitors of glycolysis and glycosylation.
The WP1122 Portfolio Program We have agreements with MD Anderson pursuant to which we have the rights to a royalty-bearing, worldwide, exclusive license for the technology rights related to our WP1122 Portfolio and similar molecules focused on inhibitors of glycolysis and glycosylation.
Hatch-Waxman Act The Drug Price Competition and Patent Term Restoration Act of 1984 (the Hatch-Waxman Act) establishes two abbreviated approval pathways for pharmaceutical products that are in some way follow-on versions of already approved products. Generic Drugs.
Hatch-Waxman Act The Drug Price Competition and Patent Term Restoration Act of 1984 (the Hatch-Waxman Act) amended the FDCA to establish two abbreviated approval pathways for pharmaceutical products that are in some way follow-on versions of already approved products. Generic Drugs.
We now have independent assessments covering 63 subjects that have been treated with Annamycin in four different clinical trials in the U.S. and Europe with no evidence of cardiotoxicity.
We now have independent assessments covering 84 subjects that have been treated with Annamycin in five different clinical trials in the U.S. and Europe with no evidence of cardiotoxicity.
Among other things, a REMS can include restrictive conditions under which the product may be distributed, which may have a negative impact on the product’s commercial success. If the FDA concludes that a REMS is needed, the NDA sponsor must submit a proposed REMS, and the product will not be approved until FDA determines that the proposed REMS is adequate.
A REMS may include restrictions on the conditions under which the product is distributed, which may have a negative impact on the product’s commercial success. If the FDA concludes that a REMS is needed, the NDA sponsor must submit a proposed REMS, and the product will not be approved until FDA determines that the proposed REMS is adequate.
In addition, under the Pediatric Research Equity Act, as amended, an NDA or supplement to an NDA generally must contain data to assess the safety and effectiveness of the product candidate for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective.
In addition, an NDA or supplement to an NDA generally must contain data to assess the safety and effectiveness of the product candidate for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective.
The American Cancer Society has estimated 25,400 new cases of brain and other nervous system cancers will occur in the United States in 2024, resulting in 18,760 deaths (https://www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/2024-cancer-facts-figures.html). Despite the severity and poor prognosis of these tumors, there are few FDA-approved drugs on the market.
The American Cancer Society has estimated 24,820 new cases of brain and other nervous system cancers will occur in the United States in 2025, resulting in 18,330 deaths ( https://www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/2025-cancer-facts-figures.html ). Despite the severity and poor prognosis of these tumors, there are few FDA-approved drugs on the market.
Potential Clinical Trial for GBM with WP1122 On December 1, 2021, we announced that the FDA allowed our IND application to study WP1122 for the treatment of GBM to go forward.
Potential Clinical Trial for GBM with WP1122 In 2021, we announced that the FDA allowed our IND application to study WP1122 for the treatment of GBM to go forward.
This is based on the reality that effective treatment options are limited. We estimate that only around 40% of AML patients are afforded an opportunity to overcome their disease through a curative bone marrow transplant or through lasting remission. We believe this aligns with the published statistic that the 5-year survival rate for AML is only 29%.
We estimate that only around 40% of AML patients are afforded an opportunity to overcome their disease through a curative bone marrow transplant or through lasting remission. We believe this aligns with the published statistic that the 5-year survival rate for AML is only 29%.
As a result, we estimate around 50% of patients are deemed “Fit” for standard intensive first-line treatment and the other 50% are deemed “Un-Fit.” Those who are deemed “Fit” are most often treated with the “standard” induction therapy of three days of intravenous daunorubicin or equivalent anthracycline, and seven days of intravenous cytarabine, also known as Ara-C.
As a result, we estimate around 50% of patients are deemed “Fit” for standard intensive first-line treatment and the other 50% are deemed “UnFit.” Those who are deemed “Fit” are most often treated with the “standard” induction therapy of three days of intravenous daunorubicin or equivalent anthracycline, and seven days of intravenous cytarabine.
We believe this will advance future studies of WP1122 in antiviral and oncology indications. We have concluded and published the clinical study report for this trial. With an IND active for WP1122 for the treatment of glioblastoma, we have concluded that advancing WP1122 in these indications will occur only if external funds are available.
We have concluded and published the clinical study report for this trial. With an IND active for WP1122 for the treatment of glioblastoma, we have concluded that advancing WP1122 in these indications will occur only if external funds are available.
A total of 2.0 million new cancer cases and 611,720 deaths from cancer are expected to occur in the US in 2024, which is about 1,680 d eaths a day. These statistics do not include either basal cell or squamous cell skin cancers because US cancer registries are not required to collect information on these cancers.
A total of 2.0 million new cancer cases and 618,120 deaths from cancer are expected to occur in the US in 2025 , which is about 1,693 d eaths a day. These statistics do not include either basal cell or squamous cell skin cancers because US cancer registries are not required to collect information on these cancers.
Market for Annamycin Per the American Cancer Society, digestive, reproductive, breast and respiratory cancers comprise most of expected cancer diagnoses in 2024, while cancers like leukemia and brain tumors are considered “rare diseases.” Leukemia in particular, can be divided into acute, chronic and other, with acute lymphoblastic leukemia (ALL) and AML comprising 27,350 of the estimated 62,770 new cases expected in the United States in 2024.
Market for Annamycin Per the American Cancer Society, digestive, reproductive, breast and respiratory cancers comprise most of expected cancer diagnoses in 2024, while cancers like leukemia and brain tumors are considered “rare diseases.” Leukemia in particular, can be divided into acute, chronic and other, with acute lymphoblastic leukemia (ALL) and AML comprising 28,110 of the estimated 66,890 new cases expected in the United States in 2025 .
Therefore, our focus is primarily on securing an accelerated approval pathway for the treatment of 2 nd line subjects (those who were relapsed from or refractory to a 1st line AML therapy, regardless of whether the subject was deemed “fit” or “unfit”).
Therefore, our focus is primarily on securing an accelerated approval pathway for the treatment of 2 nd line subjects (those who were relapsed from or refractory to a 1st line AML therapy, regardless of whether the subject was deemed “fit” or “unfit”). Later in 2024, we closed all recruitment.
It has been the subject of over 50 peer-reviewed articles and its activity against p-STAT3 has now been validated in independent labs around the world. This discovery was inspired by a naturally occurring compound (caffeic acid) in propolis (from honeybees).
WP1066 WP1066 is our flagship Immune/Transcription Modulator. It has been the subject of over 50 peer-reviewed articles and its activity against p-STAT3 has now been validated in independent labs around the world. This discovery was inspired by a naturally occurring compound (caffeic acid) in propolis (from honeybees).
Table 1 - Clinical Summary as of this filing Unless CSR Completed – Data are as of March 19, 2024 - preliminary and subject to change Drug Candidate Trial / Indication / Location Phase (Funding Source: Internal unless noted as External) Status Comments Safety Summary Human Activity Summary Annamycin MB-104 / R/R AML / US 1 P1 Concluded; P2 replaced with MB-105 Maximum Dose allowed per protocol 120 mg/m 2 Met safety endpoints; no cardiotoxicity reported 14.3% MLFS (subtherapeutic dose level when compared to MB-105) Annamycin MB-105 / R/R AML / Poland 1/2 P1 concluded; P2 replaced with MB-106 Maximum Dose and RP2D 240 mg/m 2 Met safety endpoints; no cardiotoxicity reported 80% ORR in last cohort (3 CRi and 1 PR) Annamycin in Combination with Cytarabine MB-106 / R/R AML / Europe 1B/2 20 subjects recruited to date.
Table 1 - Clinical Summary as of this filing Unless CSR Completed – Data are as of March 1, 2025 - preliminary and subject to change Drug Candidate Trial / Indication / Location Phase (Funding Source: Internal unless noted as External) Status Comments Safety Summary Human Activity Summary Annamycin MB-104 / R/R AML / US 1 P1 Concluded; P2 replaced with MB-105 Maximum Dose allowed per protocol 120 mg/m 2 Met safety endpoints; no cardiotoxicity reported 14.3% MLFS (subtherapeutic dose level when compared to MB-105) Annamycin MB-105 / R/R AML / Poland 1/2 P1 concluded; P2 replaced with MB-106 Maximum Dose and RP2D 240 mg/m 2 Met safety endpoints; no cardiotoxicity reported 80% ORR in last cohort Annamycin in Combination with Cytarabine MB-106 / R/R AML / Europe 1B/2 22 subjects intent to treat. 2 subjects had an allergic reaction and did not receive full dose.
Under the Prescription Drug User Fee Act (PDUFA), as amended, the submission of an NDA is subject to the payment of a substantial fee, although the fee may be waived under certain circumstances, which may or may not be applicable to us or our partners for any of our product candidates.
The submission of an NDA is subject to the payment of a substantial fee, although the fee may be waived under certain circumstances, which may or may not be applicable to us or our partners for any of our product candidates.
ODD and ODE are also available from the European Union (EU). ODD in the EU is generally available for drug products intended to treat life-threatening or chronically debilitating conditions affecting not more than five in 10,000 persons in the EU when the application is made.
ODD in the EU is generally available for drug products intended to treat life-threatening or chronically debilitating conditions affecting not more than five in 10,000 persons in the EU when the application is made.
While having operations in neighboring Poland, we have no operations directly in Russia or Ukraine, but we do not and cannot know if the current uncertainties in these geopolitical areas, which are unfolding in real-time, will escalate and result in broad economic and security conditions or rationing of medical supplies or production facilities, which could limit our ability to conduct clinical trials or result in material implications for our business.
We do not and cannot know if the current uncertainties in these geopolitical areas, which are unfolding in real-time, will escalate and result in broad economic and security conditions or rationing of medical supplies or production facilities, which could limit our ability to conduct clinical trials or result in material implications for our business.
During that period, the FDA generally may not approve any other application for product with the same active moiety for the same use, although there are exceptions, most notably when the later product is shown to be clinically superior to the product with orphan drug exclusivity.
During that period, the FDA generally may not approve any other application for product with the same active moiety for the same use, although there are exceptions, most notably when the later product is shown to be clinically superior to the product with orphan drug exclusivity. ODD and ODE are also available from the European Union (EU).
We have three core technologies, each of which have had one or more drugs successfully complete a Phase 1 clinical trial, based substantially on discoveries made at and licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas.
Each of our three core technologies is based substantially on discoveries made at and licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas, and features one or more drugs that have successfully completed a Phase 1 clinical trial.
Along with the results in STS lung metastases, our animal models have shown activity in other lung metastases, including osteosarcoma, colorectal and triple negative breast cancer, as well as meaningful concentration levels of Annamycin in the liver, spleen and pancreas.
The data are preliminary and subject to change. 10 Table of Contents Along with the results in STS lung metastases, our animal models have shown activity in other lung metastases, including osteosarcoma, colorectal and triple negative breast cancer, as well as meaningful concentration levels of Annamycin in the liver, spleen and pancreas.
Within 90 days of receiving the applications and assessment report, each member state must decide whether to recognize approval. In addition to regulations in Europe and the United States, we will be subject to a variety of foreign regulations governing clinical trials and commercial distribution of our future drugs. Access to Information Our website is at www.moleculin.com.
Within 90 days of receiving the applications and assessment report, each member state must decide whether to recognize approval. 23 Table of Contents In addition to regulations in Europe and the United States, we will be subject to a variety of foreign regulations governing clinical trials and commercial distribution of our future drugs.
In the event of prolonged business interruptions due to geopolitical events, we could incur significant losses, require substantial recovery time and experience significant expenditures in order to resume our business or clinical operations.
In the event of prolonged business interruptions due to geopolitical events, we could incur significant losses, require substantial recovery time and experience significant expenditures in order to resume our business or clinical operations. While having operations in neighboring Poland, we have no operations directly in Russia.
Our reference to the URL for our website is intended to be an inactive textual reference only. 26 Table of Contents
Our reference to the URL for our website is intended to be an inactive textual reference only.
The subjects treated to date have included those who were initially deemed unfit for intensive chemotherapy and the initial, preliminary data suggest that Annamycin’s safety and tolerability profile may make the product suitable for those patients, too. Annamycin Clinical Trials – AML We have studied Annamycin in three internally funded AML clinical trials, one of which is still under way.
The subjects treated to date have included those who were initially deemed unfit for intensive chemotherapy and the initial, preliminary data suggest that Annamycin’s safety and tolerability profile may make the product suitable for those patients, too. Annamycin Clinical Trials – AML We have studied Annamycin in three internally funded AML clinical trials. These trials are MB-104, MB-105, and MB-106.
To the extent other drugs or therapies are found to be more effective than our products, payers may elect to cover such therapies in lieu of our products and/or reimburse our products at a lower rate. 25 Table of Contents Different pricing and reimbursement schemes exist in other countries.
Our pharmaceutical product candidates may not be considered medically necessary or cost-effective. To the extent other drugs or therapies are found to be more effective than our products, payers may elect to cover such therapies in lieu of our products and/or reimburse our products at a lower rate. Different pricing and reimbursement schemes exist in other countries.
According to our estimates, the highest market size of STS with lung metastases was estimated in the United States, followed by Germany. The market of STS with lung metastases is categorized into first-line and second-line therapies. The therapies in first-line treatment involve surgery, off-label treatment, and stereotactic radiation therapy (SBRT).
According to our estimates, the highest market size of STS with lung metastases was estimated in the United States, followed by Germany (https://finance.yahoo.com/news/2025-research-soft-tissue-sarcoma-124900301.html). The market of STS with lung metastases is categorized into first-line and second-line therapies. The therapies in first-line treatment involve surgery, off-label treatment, and stereotactic radiation therapy (SBRT).
They include: • Priority Review, under which FDA aims to complete the NDA review within eight months of the date of submission; • Accelerated Approval, where a product may be approved on the basis of data demonstrating an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality (IMM) that is reasonably likely to predict an effect on IMM or other clinical benefit; • Fast Track, which may provide for FDA review of sections of the NDA on a rolling basis, before the complete application is submitted; and • Breakthrough Therapy, which also provides for rolling review and other actions to expedite review of the NDA. 22 Table of Contents The availability of these programs is determined by the facts surrounding each specific product candidate, the disease or condition it is intended to treat, and the availability and characteristics of alternative treatments.
They include: • Priority Review, under which the FDA aims to complete the NDA review within eight months of the submission; • Accelerated Approval, where a product may be approved on the basis of data demonstrating an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality (IMM) that is reasonably likely to predict an effect on IMM or other clinical benefit; • Fast Track, which may provide for FDA review of sections of the NDA on a rolling basis, before the complete application is submitted; and • Breakthrough Therapy, which also provides for rolling review and other actions to expedite review of the NDA.
Patent and Trademark Office (USPTO) in consultation with the FDA, reviews and approves the application for patent term restoration.
Patent and Trademark Office (USPTO) reviews and approves the application for patent term restoration.
Pursuant to goals established under PDUFA, the FDA aims to complete the review within 10 months of the 60-day filing date, which would be within 12 months of the date of submission, but that deadline is extended in certain circumstances, including by FDA requests for additional information or clarification.
Pursuant to goals established in statute, the FDA aims to complete the review within 12 months of the date of NDA submission, but that deadline is extended in certain circumstances, including by FDA requests for additional information or clarification.
In February 2023, the externally funded Phase 1 clinical trial with WP1066 for the treatment of pediatric brain tumors concluded. We expect one externally funded Phase 1B/2 clinical trial for WP1066 in combination with radiation for the treatment of GBM and other brain tumors in 2024.
In February 2023, the externally funded Phase 1 clinical trial with WP1066 for the treatment of pediatric brain tumors concluded. An externally funded Phase 1B/2 clinical trial for WP1066 in combination with radiation for the treatment of GBM began treating subjects in 2024.
Once the FDA accepts for filing an ANDA or 505(b)(2) application containing a Paragraph IV certification, the applicant must within 20 days provide notice to the RLD or listed drug NDA holder and patent owner that the application has been submitted, and provide the factual and legal basis for the applicant's assertion that the patent is invalid or not infringed.
A "Paragraph IV" certification is an assertion that the patent does not block approval of the later product, either because the patent is invalid or unenforceable or because the patent, even if valid, is not infringed by the new product. 21 Table of Contents Once the FDA accepts for filing an ANDA or 505(b)(2) application containing a Paragraph IV certification, the applicant must within 20 days provide notice to the RLD or listed drug NDA holder and patent owner that the application has been submitted, and provide the factual and legal basis for the applicant's assertion that the patent is invalid or not infringed.
Three of our six drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2 or Phase 2 clinical trials. Since our inception, our drugs have completed, are currently in, or have been permitted to proceed in thirteen clinical trials.
Three of our six drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2 or Phase 2 clinical trials. One is currently beginning a Phase 2B/3 trial. Since our inception, our drug candidates have completed, are currently in, or have been permitted to proceed in, fourteen clinical trials.
In December 2018, we began treatment at the final dose of MB-104 of 120 mg/m 2 . In February 2022, we successfully concluded the Phase 1 portion of that trial and established the RP2D of 240 mg/m 2 . A total of 20 subjects were enrolled in this trial.
In February 2022, we successfully concluded the Phase 1 portion of that trial and established the RP2D of 240 mg/m 2 . A total of 20 subjects were enrolled in this trial.
Clinical data where a CSR or its equivalent has not been published are considered preliminary and subject to change. Our Expert has issued and will continue to issue periodic reports as additional data are provided in batches of subject data. To date, we have received a several independent assessments for the absence of cardiotoxicity in subjects treated with Annamycin.
Our Expert has issued and will continue to issue periodic reports as additional data are provided in batches of subject data. To date, we have received several independent assessments for the absence of cardiotoxicity in subjects treated with Annamycin.
Another important attribute of WP1066 (unlike some of our other Immune/Transcription Modulators) is its apparent ability in pre-clinical testing to cross the blood brain barrier, which we believe makes it a good candidate for potentially treating brain tumors and other malignancies of the central nervous system.
We believe this overcomes the limitations of many other drugs designed to inhibit STAT3 activity by blocking upstream receptors. 11 Table of Contents Another important attribute of WP1066 (unlike some of our other Immune/Transcription Modulators) is its apparent ability in pre-clinical testing to cross the blood brain barrier, which we believe makes it a good candidate for potentially treating brain tumors and other malignancies of the central nervous system.
This would be especially valuable in the case of pediatric acute leukemia (both AML and ALL) because of the potential impact of cardiotoxicity on long-term survival. 6 Table of Contents In addition, the effectiveness of currently approved anthracyclines is limited by their propensity for succumbing to “multidrug resistance.” This can occur where, as a natural defense mechanism, transmembrane proteins acting as transporters (one type of which is referred to as a “P-glycoprotein pump” or an “ABCB1 transporter”; otherwise referred to as “MDR1 mechanisms”) develop on the outer surface of cells to expel perceived threats like anthracyclines.
In addition, the effectiveness of currently approved anthracyclines is limited by their propensity for succumbing to “multidrug resistance.” This can occur where, as a natural defense mechanism, transmembrane proteins acting as transporters (one type of which is referred to as a “P-glycoprotein pump” or an “ABCB1 transporter”; otherwise referred to as “MDR1 mechanisms”) develop on the outer surface of cells to expel perceived threats like anthracyclines.
Such activity may or may not be repeated in future clinical trials, including potentially pivotal and/or confirmatory clinical trials. While we believe such data are encouraging, the FDA or its foreign counterpart will ultimately determine if such data and future data are individually conclusive and supports future clinical trials or approval.
While we believe such data are encouraging, the FDA or its foreign counterpart will ultimately determine if such data and future data are individually conclusive and supports future clinical trials or approval.
Royalty payments can range in the single digits as a percent of net sales on drug products or flat fees as high as $0.6 million, depending upon certain terms and conditions. Not all of these payments are applicable to every drug. Total expenses under these agreements we re $0.3 million for the years ended December 31, 2023 and 2022, respectively.
Royalty payments can range in the single digits as a percent of net sales on drug products or flat fees as high as $0.6 million, depending upon certain terms and conditions. Not all of these payments are applicable to every drug.
Accordingly, we also intend to pursue approval for pediatric use in these conditions when practicable. Soft tissue sarcoma is a broad term for cancers that start in soft tissues (muscle, tendons, fat, lymph and blood vessels, and nerves). These cancers can develop anywhere in the body but are found mostly in the arms, legs, chest, and abdomen.
Accordingly, we also intend to pursue approval for pediatric use in these conditions when practicable. Soft tissue sarcoma is a broad term for cancers that start in soft tissues (muscle, tendons, fat, bone, lymph and blood vessels, and nerves).
The FDA granted ODD to Annamycin for the treatment of AML and for soft tissue sarcoma patients, which means the agency believes we have established a medically plausible basis for using the drug for those indications. The FDA also granted Fast Track-Designation (FTD) for Annamycin for both the treatment of AML and Soft Tissue Sarcoma.
Additionally, the FDA has granted ODD for Annamycin for the treatment of soft tissue sarcoma. Such designation means, in part, these agencies believe we have established a medically plausible basis for using the drug for those indications. The FDA also granted Fast Track-Designation (FTD) for Annamycin for both the treatment of AML and Soft Tissue Sarcoma.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeFast Track designation may also be rescinded if the FDA believes the designation is no longer supported by data from our clinical development program. • Interim or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data. • We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities. • We, or our third-party manufacturers, may be unable to successfully scale-up manufacturing of our product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing approved products, if any. • Recently enacted legislation, future legislation and healthcare reform measures may increase the difficulty and cost for us to obtain marketing approval for and commercialize Annamycin and any future product candidates and may affect the prices we may set.
Biggest changeFast Track designation may also be rescinded if the FDA believes the designation is no longer supported by data from our clinical development program. • Interim or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data. • We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities. • We, or our third-party manufacturers, may be unable to successfully scale-up manufacturing of our product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing approved products, if any. • Even if we obtain regulatory approvals for our product candidates, they will remain subject to ongoing regulatory oversight. • Recently enacted legislation, future legislation and healthcare reform measures may increase the difficulty and cost for us to obtain marketing approval for and commercialize Annamycin and any future product candidates and may affect the prices we may set. 24 Table of Contents Risks Related to Our Intellectual Property • The composition of matter patent for Annamycin has expired, and other patents have not yet been issued, and may not be issued. • The intellectual property rights we have licensed from MD Anderson are subject to the rights of the US government. • We may incur substantial costs as a result of litigation or other proceedings relating to patent and other intellectual property rights. • We may be subject to claims that our employees have wrongfully used or disclosed alleged trade secrets of their former employers. • If we are not able to adequately prevent disclosure of trade secrets and other proprietary information, the value of our technology and products could be significantly diminished. • If we breach any of the agreements under which we license patent rights or if we fail to meet certain development deadlines, pay certain fees including extension fees or exercise certain rights to technology, we could lose or fail to obtain license rights that are important to our business. • We will not be able to protect our intellectual property rights throughout the world.
We may not experience a faster development or regulatory review or approval process with Fast Track designation compared to conventional FDA procedures. In addition, the FDA may withdraw Fast Track designation if it believes that the designation is no longer supported by data from our clinical development program.
We may not experience a faster development or regulatory review or approval process with Fast Track designation compared to conventional FDA review procedures. In addition, the FDA may withdraw Fast Track designation if it believes that the designation is no longer supported by data from our clinical development program.
Risks Related to Regulatory Approval and the Development and Commercialization of our Drug Candidates • We are developing our drugs to treat patients who are extremely or terminally ill, and patient deaths that occur in our clinical trials could negatively impact our business even if such outcomes are not shown to be related to our drugs. • We are conducting important clinical trials in the US and Europe, and studies for additional countries in which to perform preclinical studies and clinical trials and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business. • There are limited suppliers for active pharmaceutical ingredients (API) used in in our drug candidates and we utilize a single source for such API for certain of our drug candidates.
Risks Related to Regulatory Approval and the Development and Commercialization of our Drug Candidates • We are developing our drugs to treat patients who are extremely or terminally ill, and patient deaths that occur in our clinical trials could negatively impact our business even if such outcomes are not shown to be related to our drugs. • We are conducting important clinical trials in the US and Europe, and assessing additional countries in which to perform preclinical studies and clinical trials and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business. • There are limited suppliers for active pharmaceutical ingredients (API) used in in our drug candidates and we utilize a single source for such API for certain of our drug candidates.
The amount and timing of our future funding requirements will depend on many factors, including but not limited to: • whether our plan for clinical trials will be completed on a timely basis and, if completed, whether we will be able to publicly announce results from our phase I/II clinical trials in accordance with our announced milestones; • whether the results of our clinical trials will be announced on a timely basis and, when announced, whether such results are in accordance with our expectations or our announced milestones; • whether we are successful in obtaining the benefits of FDA’s expedited development and review programs related to Annamycin or our other drug candidates; • the progress, costs, results of and timing of our clinical trials and also of our preclinical studies; • the outcome, costs and timing of seeking and obtaining FDA and any other regulatory approvals; • the costs associated with securing and establishing commercialization and manufacturing capabilities; • market acceptance of our product candidates; • the costs of acquiring, licensing or investing in businesses, products, product candidates and technologies; • our ability to maintain, expand and enforce the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make, or that we may receive, in connection with the licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights; • our need and ability to hire additional management and scientific and medical personnel; • the effect of competing drug candidates and new product approvals; • our need to implement additional internal systems and infrastructure, including financial and reporting systems; and • the economic and other terms, timing of and success of our existing licensing arrangements and any collaboration, licensing or other arrangements into which we may enter in the future. 38 Table of Contents Some of these factors are outside of our control.
The amount and timing of our future funding requirements will depend on many factors, including but not limited to: • whether our plan for clinical trials will be completed on a timely basis and, if completed, whether we will be able to publicly announce results from our phase I/II clinical trials in accordance with our announced milestones; • whether the results of our clinical trials will be announced on a timely basis and, when announced, whether such results are in accordance with our expectations or our announced milestones; • whether we are successful in obtaining the benefits of FDA’s expedited development and review programs related to Annamycin or our other drug candidates; • the progress, costs, results of and timing of our clinical trials and also of our preclinical studies; • the outcome, costs and timing of seeking and obtaining FDA and any other regulatory approvals; • the costs associated with securing and establishing commercialization and manufacturing capabilities; • market acceptance of our product candidates; • the costs of acquiring, licensing or investing in businesses, products, product candidates and technologies; • our ability to maintain, expand and enforce the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make, or that we may receive, in connection with the licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights; • our need and ability to hire additional management and scientific and medical personnel; • the effect of competing drug candidates and new product approvals; • our need to implement additional internal systems and infrastructure, including financial and reporting systems; and • the economic and other terms, timing of and success of our existing licensing arrangements and any collaboration, licensing or other arrangements into which we may enter in the future. 36 Table of Contents Some of these factors are outside of our control.
We may not have the financial resources to continue development of, or to enter into collaborations for, a product candidate if we experience any problems or other unforeseen events that delay or prevent regulatory approval of, or our ability to commercialize, product candidates, including: • inability to obtain sufficient funds required for a clinical trial; • inability to reach agreements on acceptable terms with prospective CROs and trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; • clinical sites dropping out of a clinical trial; • time required to add new clinical sites; • negative or inconclusive results from our clinical trials or the clinical trials of others for product candidates similar to ours, leading to a decision or requirement to conduct additional preclinical testing or clinical trials or abandon a program; • serious and unexpected drug-related side effects experienced by subjects in our clinical trials or by individuals using drugs similar to our product candidates; • conditions imposed by the FDA or comparable foreign authorities regarding the scope or design of our clinical trials; 31 Table of Contents • delays in or inability to enroll research subjects in sufficient numbers or at the expected rate; • high drop-out rates and high fail rates of research subjects; • imposition of a clinical hold following an inspection of our clinical trial operations or trial sites by the FDA or foreign regulatory authorities; • delays or failures in obtaining required IRB approval; • inadequate supply or quality of product candidate components or materials or other supplies necessary for the conduct of our clinical trials; failure to comply with GLP, GCP, cGMP or similar foreign regulatory requirements that affect the conduct of pre-clinical and clinical studies and the manufacturing of product candidates; • greater than anticipated clinical trial costs; • poor efficacy of our product candidates during clinical trials; • requests by regulatory authorities for additional data or clinical trials; • governmental or regulatory agency assessments of pre-clinical or clinical testing that differ from our interpretations or conclusions; • governmental or regulatory delays, or changes in approval policies or regulations; or • unfavorable FDA or other regulatory agency inspection and review of a clinical trial site or vendor.
We may not have the financial resources to continue development of, or to enter into collaborations for, a product candidate if we experience any problems or other unforeseen events that delay or prevent regulatory approval of, or our ability to commercialize, product candidates, including: • inability to obtain sufficient funds required for a clinical trial; • inability to reach agreements on acceptable terms with prospective CROs and trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; • clinical sites dropping out of a clinical trial; • time required to add new clinical sites; • negative or inconclusive results from our clinical trials or the clinical trials of others for product candidates similar to ours, leading to a decision or requirement to conduct additional preclinical testing or clinical trials or abandon a program; • serious and unexpected drug-related side effects experienced by subjects in our clinical trials or by individuals using drugs similar to our product candidates; • conditions imposed by the FDA or comparable foreign authorities regarding the scope or design of our clinical trials; • delays in or inability to enroll research subjects in sufficient numbers or at the expected rate; • high drop-out rates and high fail rates of research subjects; • imposition of a clinical hold following an inspection of our clinical trial operations or trial sites by the FDA or foreign regulatory authorities; • delays or failures in obtaining required IRB approval; • inadequate supply or quality of product candidate components or materials or other supplies necessary for the conduct of our clinical trials; failure to comply with GLP, GCP, cGMP or similar foreign regulatory requirements that affect the conduct of pre-clinical and clinical studies and the manufacturing of product candidates; • greater than anticipated clinical trial costs; • poor efficacy of our product candidates during clinical trials; • requests by regulatory authorities for additional data or clinical trials; • governmental or regulatory agency assessments of pre-clinical or clinical testing that differ from our interpretations or conclusions; • governmental or regulatory delays, or changes in approval policies or regulations; or • unfavorable FDA or other regulatory agency inspection and review of a clinical trial site or vendor.
NDAs must also include significant information regarding the chemistry, manufacturing and controls for the product. Obtaining approval of a NDA is a lengthy, expensive and uncertain process, and we may not be successful in obtaining approval. The FDA review processes can take years to complete and approval is never guaranteed.
NDAs must also include significant information regarding the chemistry, manufacturing and controls for the product. Obtaining approval of an NDA is a lengthy, expensive and uncertain process, and we may not be successful in obtaining approval. The FDA review processes can take years to complete and approval is never guaranteed.
Because we have limited financial and managerial resources, we focus on research programs and product candidates for specific indications. Specifically, with regard to Annamycin, we are initially focusing our efforts on the treatment of AML and STS lung mets.
Because we have limited financial and managerial resources, we focus on research programs and product candidates for specific indications. Specifically, with regard to Annamycin, we are focusing our efforts on the treatment of AML and STS lung mets.
ODD may enable market exclusivity of 7 years from the date of approval of a NDA in the United States. During that period the FDA generally could not approve another product containing the same drug for the same designated indication.
However, ODD may enable market exclusivity of 7 years from the date of approval of a NDA in the United States. During that period the FDA generally could not approve another product containing the same drug for the same designated indication.
If our third-party drug suppliers fail to achieve and maintain high manufacturing standards in compliance with cGMP regulations, we could be subject to certain product liability claims in the event such failure to comply resulted in defective products that caused injury or harm. 30 Table of Contents We cannot guarantee how long it will take regulatory agencies to review our applications for product candidates, and we may fail to obtain the necessary regulatory approvals to market our product candidates.
If our third-party drug suppliers fail to achieve and maintain high manufacturing standards in compliance with cGMP regulations, we could be subject to certain product liability claims in the event such failure to comply resulted in defective products that caused injury or harm. 27 Table of Contents We cannot guarantee how long it will take regulatory agencies to review our applications for product candidates, and we may fail to obtain the necessary regulatory approvals to market our product candidates.
In trials, both with prior developers and with ours using Annamycin, subjects have experienced adverse events. There can be no assurance that other adverse events may not emerge related to our drug.
In clinical trials, both with prior developers and with ours using Annamycin, subjects have experienced adverse events. There can be no assurance that other adverse events may not emerge related to our drug.
The commencement and completion of future clinical trials could be substantially delayed or prevented by several factors, including, but not limited to: • failure to reach agreement with the FDA or other regulatory agency requirements for clinical trial design or scope of the development program; a limited number of, and competition for, suitable subjects with particular types of cancer and viruses for enrollment in our clinical trials; 32 Table of Contents • delays or failures in reaching acceptable clinical trial agreement terms with CROs or clinical trial sites; • delays or inability to attract clinical investigators for trials; • clinical sites dropping out of a clinical trial; • time required to add new clinical sites; • failure of subjects to complete the clinical trial or inability to follow subjects adequately after treatment; failures by, changes in our relationship with, or other issues at, CROs, vendors and investigators responsible for pre-clinical testing and clinical trials; imposition of a clinical hold; and • unforeseen safety issues.
The commencement and completion of future clinical trials could be substantially delayed or prevented by several factors, including, but not limited to: • • failure to reach agreement with the FDA or other regulatory agency requirements for clinical trial design or scope of the development program; a limited number of, and competition for, suitable subjects with particular types of cancer and viruses for enrollment in our clinical trials; • delays or failures in reaching acceptable clinical trial agreement terms with CROs or clinical trial sites; • delays or inability to attract clinical investigators for trials; • clinical sites dropping out of a clinical trial; • time required to add new clinical sites; • • • failure of subjects to complete the clinical trial or inability to follow subjects adequately after treatment; failures by, changes in our relationship with, or other issues at, CROs, vendors and investigators responsible for pre-clinical testing and clinical trials; imposition of a clinical hold; and • unforeseen safety issues.
If we are not able to obtain required regulatory approvals, we will not be able to commercialize our product candidates and our ability to generate revenue will be materially impaired. • Delays in the commencement, enrollment and completion of clinical trials could result in increased costs to us and delay or limit our ability to obtain regulatory approval for any of our product candidates. • As an organization, we have never conducted pivotal clinical trials, and we may be unable to do so for any product candidate we may develop. • We may expend significant resources to pursue certain product candidates for specific indications and fail to capitalize on the potential of such product candidates for the potential treatment of other indications that may be more profitable or for which there is a greater likelihood of success. • We have commenced clinical trials and have never submitted an NDA, and any product candidate we advance through clinical trials may not have favorable results in later clinical trials or receive regulatory approval. • We may find it difficult to enroll subjects in our clinical trials, which could delay or prevent clinical trials of our product candidates. • A portion of our clinical development plan relies on physician-sponsored trials, which we do not control, and which may encounter delays for reasons outside of our control. • If any of our drug product candidates are found to be unsafe or lack sufficient efficacy, we will not be able to obtain regulatory approval for it and our business would be harmed. • Our product candidates may have undesirable side effects that may delay or prevent marketing approval, or, if approval is received, require them to be taken off the market, require them to include safety warnings or otherwise limit their sales. • Even if our product candidates otherwise qualify for approval from the FDA, if the FDA does not find the manufacturing facilities of our future contract manufacturers acceptable for commercial production, we may not be able to commercialize any of our product candidates. • We received ODD for Annamycin, WP1066, and WP1122, but even if either product candidate is approved and receives ODE, ODE may not effectively prevent approval of a competing product. • The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and even if we obtain approval for a product candidate in one country or jurisdiction, we may never obtain approval for or commercialize it in any other jurisdiction, which would limit our ability to realize our full market potential. • We have received Fast Track designation for two of our product candidates and may seek the same designation for one of more of our other product candidates.
If we are not able to obtain required regulatory approvals, we will not be able to commercialize our product candidates and our ability to generate revenue will be materially impaired. • Delays in the commencement, enrollment and completion of clinical trials could result in increased costs to us and delay or limit our ability to obtain regulatory approval for any of our product candidates. • As an organization, we have never before conducted pivotal clinical trials, and we may be unable to do so for any product candidate we may develop. • We may expend significant resources to pursue certain product candidates for specific indications and fail to capitalize on the potential of such product candidates for the potential treatment of other indications that may be more profitable or for which there is a greater likelihood of success. • We have commenced clinical trials and have never submitted an NDA, and any product candidate we advance through clinical trials may not have favorable results in later clinical trials or receive regulatory approval. • We may find it difficult to enroll subjects in our clinical trials, which could delay or prevent clinical trials of our product candidates. • A portion of our clinical development plan relies on physician-sponsored trials, which we do not control, and which may encounter delays for reasons outside of our control. • If any of our drug product candidates are found to be unsafe or lack sufficient efficacy, we will not be able to obtain regulatory approval for it and our business would be harmed. • Our product candidates may have undesirable side effects that may delay or prevent marketing approval, or, if approval is received, require them to be taken off the market, require them to include safety warnings or otherwise limit their sales. • Even if our product candidates otherwise qualify for approval from the FDA, if the FDA does not find the manufacturing facilities of our future contract manufacturers acceptable for commercial production, we may not be able to commercialize any of our product candidates. • We received ODD for Annamycin, WP1066, and WP1122, but even if either product candidate is approved and receives ODE, ODE may not effectively prevent approval of a competing product. • We may not be able to obtain or maintain rare pediatric disease designation or exclusivity for our product candidates, which could limit the potential profitability of our product candidates. • The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and even if we obtain approval for a product candidate in one country or jurisdiction, we may never obtain approval for or commercialize it in any other jurisdiction, which would limit our ability to realize our full market potential. • We have received Fast Track designation for two of our product candidates and may seek the same designation for one of more of our other product candidates.
If any of our product candidates receives marketing approval and we or others later identify undesirable or unacceptable side effects caused by such products: • regulatory authorities may require the addition of labeling statements, specific warnings, a contraindication or field alerts to physicians and pharmacies; • we may be required to change instructions regarding the way the product is administered, conduct additional clinical trials or change the labeling of the product; • we may be subject to limitations on how we may promote the product; • sales of the product may decrease significantly; • regulatory authorities may require us to take our approved product off the market; • we may be subject to litigation or product liability claims; and • our reputation may suffer.
If any of our product candidates receives marketing approval and we or others later identify undesirable or unacceptable side effects caused by such products: • regulatory authorities may require the addition of labeling statements, specific warnings, a contraindication or field alerts to physicians and pharmacies; • we may be required to change instructions regarding the way the product is administered, conduct additional clinical trials or change the labeling of the product; • we may be subject to limitations on how we may promote the product; • sales of the product may decrease significantly; • regulatory authorities may require us to take our approved product off the market; 31 Table of Contents • we may be subject to litigation or product liability claims; and • our reputation may suffer.
At the state level, individual states in the United States have also 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.
At the state level, individual states in the United States have also increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price 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.
We are not permitted to market our product candidates in the United States until we receive approval of a NDA from the FDA. We have not submitted any marketing applications for any of our product candidates. NDAs must include extensive preclinical and clinical data and supporting information to establish the product candidate’s safety and effectiveness for each desired indication.
We are not permitted to market our product candidates in the United States until we receive approval of an NDA from the FDA. We have not submitted any marketing applications for any of our product candidates. NDAs must include extensive preclinical and clinical data and supporting information to establish the product candidate’s safety and effectiveness for each desired indication.
If we are unable to do so, the institution may offer the intellectual property rights to other parties, potentially blocking our ability to pursue a program based on that technology. 37 Table of Contents We will not be able to protect our intellectual property rights throughout the world. We are dependent on patents licensed with MD Anderson.
If we are unable to do so, the institution may offer the intellectual property rights to other parties, potentially blocking our ability to pursue a program based on that technology. 35 Table of Contents We will not be able to protect our intellectual property rights throughout the world. We are dependent on patents licensed with MD Anderson.
These and other risks associated with our international operations may materially adversely affect our ability to attain or maintain profitable operations. 29 Table of Contents There are limited suppliers for active pharmaceutical ingredients (API) used in in our drug candidates and we utilize a single source for such API for certain of our drug candidates.
These and other risks associated with our international operations may materially adversely affect our ability to attain or maintain profitable operations. 26 Table of Contents There are limited suppliers for active pharmaceutical ingredients (API) used in our drug candidates and we utilize a single source for such API for certain of our drug candidates.
As a result of the accounting for our acquisition of Moleculin, LLC and the agreement we, on Moleculin, LLC’s behalf, entered into with Houston Pharmaceuticals, Inc., we have carried on our balance sheet within intangible assets in-process research and development (IPR&D) of $11.1 million as of December 31, 2023.
As a result of the accounting for our acquisition of Moleculin, LLC and the agreement we, on Moleculin, LLC’s behalf, entered into with Houston Pharmaceuticals, Inc., we have carried on our balance sheet within intangible assets in-process research and development (IPR&D) of $11.1 million as of December 31, 2024.
We expect that the Affordable Care Act and other healthcare reform measures, including those that may be adopted in the future may result in additional reductions in Medicare and other healthcare funding, more rigorous coverage criteria, new payment methodologies and additional downward pressure on the price that we receive for any approved product.
We expect that the Affordable Care Act, Inflation Reduction Act, and other healthcare reform measures, including those that may be adopted in the future may result in additional reductions in Medicare and other healthcare funding, more rigorous coverage criteria, new payment methodologies and additional downward pressure on the price that we receive for any approved product.
Accordingly, the results of any historical quarterly or annual periods should not be relied upon as indications of future operating performance. 39 Table of Contents We have in the past completed related party transactions that were not conducted on an arm’s length basis.
Accordingly, the results of any historical quarterly or annual periods should not be relied upon as indications of future operating performance. 37 Table of Contents We have in the past completed related party transactions that were not conducted on an arm’s length basis.
Off-label sales would limit our ability to generate revenue from the sale of Annamycin, if approved for commercial sale. 36 Table of Contents The intellectual property rights we have licensed from MD Anderson are subject to the rights of the US government.
Off-label sales would limit our ability to generate revenue from the sale of Annamycin, if approved for commercial sale. 34 Table of Contents The intellectual property rights we have licensed from MD Anderson are subject to the rights of the US government.
Any claims for indemnification made by our directors or officers could impact our cash resources and our ability to fund the business. 46 Table of Contents We have no intention of declaring dividends in the foreseeable future.
Any claims for indemnification made by our directors or officers could impact our cash resources and our ability to fund the business. 44 Table of Contents We have no intention of declaring dividends in the foreseeable future.
We can provide no assurance that we will not encounter future delays with our physician-sponsored trials. 33 Table of Contents If any of our drug product candidates are found to be unsafe or lack sufficient efficacy, we will not be able to obtain regulatory approval for it and our business would be harmed.
We can provide no assurance that we will not encounter future delays with our physician-sponsored trials. If any of our drug product candidates are found to be unsafe or lack sufficient efficacy, we will not be able to obtain regulatory approval for it and our business would be harmed.
If we cannot provide reliable financial reports or prevent fraud, our business and results of operations could be harmed, and investors could lose confidence in our reported financial information. 45 Table of Contents Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price.
If we cannot provide reliable financial reports or prevent fraud, our business and results of operations could be harmed, and investors could lose confidence in our reported financial information. Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price.
We are conducting important clinical trials in the US and Europe, and we are performing studies for additional countries in which to perform preclinical studies and clinical trials and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business. We have approved Clinical Trial Authorizations in Poland and Italy.
We are conducting important clinical trials in the US and Europe, and assessing additional countries in which to perform preclinical studies and clinical trials, and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business. We have approved Clinical Trial Authorizations in Poland and Italy.
The exercise of the options and warrants will dilute the voting interest of the owners of presently outstanding shares by adding a substantial number of additional shares of our common stock. As a biotechnology company, we are at increased risk of securities class action litigation.
The exercise of the options and warrants will dilute the voting interest of the owners of presently outstanding shares by adding a substantial number of additional shares of our common stock. 42 Table of Contents As a biotechnology company, we are at increased risk of securities class action litigation.
Finally, a data breach affecting sensitive personal information, including health information, could result in significant legal and financial exposure and reputational damages that could potentially have an adverse effect on our business. EU Member States, Switzerland and other countries have also adopted data protection laws and regulations, which impose significant compliance obligations.
Finally, a data breach affecting sensitive personal information, including health information, could result in significant legal and financial exposure and reputational damages that could potentially have an adverse effect on our business. 40 Table of Contents EU Member States, Switzerland and other countries have also adopted data protection laws and regulations, which impose significant compliance obligations.
Our failure to comply with these laws, or changes in the way in which these laws are implemented, could lead to government enforcement actions and significant penalties against us, and adversely impact our business. 43 Table of Contents We depend on our information technology and infrastructure so compromises could materially harm our ability to conduct business or delay our financial reporting.
Our failure to comply with these laws, or changes in the way in which these laws are implemented, could lead to government enforcement actions and significant penalties against us, and adversely impact our business. We depend on our information technology and infrastructure so compromises could materially harm our ability to conduct business or delay our financial reporting.
Our operations to date have been limited to acquiring our technology portfolio, preparing several drugs for authorization to conduct clinical trials and conducting Phase 1 and 2 clinical trials. We have yet to receive regulatory approvals for any of our drug candidates.
Our operations to date have been limited to acquiring our technology portfolio, preparing several drugs for authorization to conduct clinical trials and conducting Phase 1 through Phase 3 clinical trials. We have yet to receive regulatory approvals for any of our drug candidates.
In addition, the Inflation Reduction Act (IRA), among other things, (1) directs HHS to negotiate the price of certain high-cost, single-source drugs and biologics covered under Medicare and (2) imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation.
In addition, the Inflation Reduction Act (IRA), among other things, (1) directs the Department of Health and Human Services (HHS) to negotiate the price of certain high-cost, single-source drugs and biologics covered under Medicare and (2) imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation.
Subject enrollment is affected by a variety factors including, among others: • severity of the disease under investigation; • design of the trial protocol and size of the patient population required for analysis of the trial’s primary endpoints; • size of the patient population; • eligibility criteria for the trial in question; • perceived risks and benefits of the product candidate being tested; • willingness or availability of subjects to participate in our clinical trials (including due to the COVID-19 pandemic); • proximity and availability of clinical trial sites for prospective subjects; • our ability to recruit clinical trial investigators with the appropriate competencies and experience; • availability of competing vaccines and/or therapies and related clinical trials; • efforts to facilitate timely enrollment in clinical trials; • our ability to obtain and maintain subject consents; • the risk that subjects enrolled in clinical trials will drop out of the trials before completion; • subject referral practices of physicians; and • ability to monitor subjects adequately during and after treatment.
Subject enrollment is affected by a variety factors including, among others: • severity of the disease under investigation; • design of the trial protocol and size of the patient population required for analysis of the trial’s primary endpoints; • size of the patient population; • eligibility criteria for the trial in question; • perceived risks and benefits of the product candidate being tested; • willingness or availability of subjects to participate in our clinical trials; • willingness of the investigators to accept the trial design, including the control arm, of the study; • proximity and availability of clinical trial sites for prospective subjects; • our ability to recruit clinical trial investigators with the appropriate competencies and experience; • availability of competing vaccines and/or therapies and related clinical trials; 30 Table of Contents • efforts to facilitate timely enrollment in clinical trials; • our ability to obtain and maintain subject consents; • the risk that subjects enrolled in clinical trials will drop out of the trials before completion; • subject referral practices of physicians; and • ability to monitor subjects adequately during and after treatment.
We are currently utilizing contract manufacturers for the production of the active pharmaceutical ingredients and the formulation of drug product candidates for our clinical trials. Additionally, even if our product candidates otherwise qualify for approval from the FDA, we do not intend to manufacture the approved pharmaceutical products.
We are currently utilizing contract manufacturers for the production of the active pharmaceutical ingredients and the formulation of drug product candidates for our clinical trials. Additionally, even if our product candidates would otherwise qualify for approval from the FDA based on results from preclinical and clinical trials, we do not intend to manufacture the approved pharmaceutical products.
Responding to proxy contests and other actions by such activist investors or others in the future could be costly and time-consuming, disrupt our operations and divert the attention of our Board of Directors and senior management from the pursuit of our business strategies, which could adversely affect our results of operations and financial condition.
Responding to proxy contests and other actions by such activist investors or others in the future could be costly and time-consuming, disrupt our operations and divert the attention of our Board of Directors and senior management from the pursuit of our business strategies, which could adversely affect our results of operations and financial condition. ITEM 1B. UNRESOLVED STAFF COMMENTS None.
As a result, our ability to reduce our losses and reach profitability is unproven, and we may never achieve or sustain profitability. • Our financial condition would be adversely impacted if our intangible assets become impaired. • We have no sales, marketing or distribution experience and we will have to invest significant resources to develop those capabilities or enter into acceptable third-party sales and marketing arrangements. • We may not be successful in establishing and maintaining development and commercialization collaborations, which could adversely affect our ability to develop certain of our product candidates and our financial condition and operating results. • We face competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete effectively. • We will need to expand our operations and increase the size of our company, and we may experience difficulties in managing growth. • We may not be able to manage our business effectively if we are unable to attract and retain key personnel and consultants. • We do not expect that our insurance policies will cover all of our business exposures thus leaving us exposed to significant uninsured liabilities. • We may incur penalties if we fail to comply with healthcare regulations. • We may not be able to recover from any catastrophic event affecting our suppliers. • Our business and operations would suffer in the event of third-party computer system failures, cyber-attacks on third-party systems or deficiency in our cyber security. • The COVID-19 outbreak delayed recruitment in our clinical trials in the past and may return, may affect the business of the FDA, EMA or other health authorities, which could result in delays in meetings related to our planned clinical trials and ultimately of reviews and approvals of our product candidates. • Our failure to comply with data protection laws and regulations could lead to government enforcement actions and significant penalties against us, and adversely impact our operating results. • We depend on our information technology and infrastructure so compromises could materially harm our ability to conduct business or delay our financial reporting. • We may be required to make significant payments under our license agreements with MD Anderson. • New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified, or applied adversely to us or our customers may have a material adverse effect on our business and financial condition.
As a result, our ability to reduce our losses and reach profitability is unproven, and we may never achieve or sustain profitability. • Our financial condition would be adversely impacted if our intangible assets become impaired. • We have no sales, marketing or distribution experience and we will have to invest significant resources to develop those capabilities or enter into acceptable third-party sales and marketing arrangements. • We may not be successful in establishing and maintaining development and commercialization collaborations, which could adversely affect our ability to develop certain of our product candidates and our financial condition and operating results. • We face competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete effectively. • We will need to expand our operations and increase the size of our company, and we may experience difficulties in managing growth. • We may not be able to manage our business effectively if we are unable to attract and retain key personnel and consultants. • We do not expect that our insurance policies will cover all of our business exposures thus leaving us exposed to significant uninsured liabilities. • We may incur penalties if we fail to comply with healthcare regulations. • We may not be able to recover from any catastrophic event affecting our suppliers. • Our business and operations would suffer in the event of third-party computer system failures, cyber-attacks on third-party systems or deficiency in our cyber security. • Our failure to comply with data protection laws and regulations could lead to government enforcement actions and significant penalties against us, and adversely impact our operating results. • We depend on our information technology and infrastructure so compromises could materially harm our ability to conduct business or delay our financial reporting. • We may be required to make significant payments under our license agreements with MD Anderson. • New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified, or applied adversely to us or our customers may have a material adverse effect on our business and financial condition. • We must comply with indirect tax laws in multiple jurisdictions, as well as complex customs duty regimes worldwide.
If subjects are unwilling to participate in our trials because of the COVID-19 pandemic and restrictions on travel or healthcare institution policies, negative publicity from adverse events in the biotechnology industries, public perception of vaccine safety issues or for other reasons, including competitive clinical trials for similar patient populations, the timeline for recruiting subjects, conducting studies and obtaining regulatory approval of potential products may be delayed.
If subjects are unwilling to participate in our trials because of negative publicity from adverse events in the biotechnology industries, public perception of vaccine safety issues or for other reasons, including competitive clinical trials for similar patient populations, the timeline for recruiting subjects, conducting studies and obtaining regulatory approval of potential products may be delayed.
We have received Fast Track designation for two of our product candidates and may seek the same designation for one of more of our other product candidates. Such designation may not actually lead to a faster development or regulatory review or approval process.
Such regulatory requirements may differ from country to country depending on where we have received regulatory approval. We have received Fast Track designation for two of our product candidates and may seek the same designation for one of more of our other product candidates. Such designation may not actually lead to a faster development or regulatory review or approval process.
In addition, the supply chain for the manufacturing of our product candidates is complicated and can involve several parties. If we were to experience any supply chain issues, including as a result of the COVID-19 pandemic, our product supply could be seriously disrupted.
In addition, the supply chain for the manufacturing of our product candidates is complicated and can involve several parties. If we were to experience any supply chain issues, our product supply could be seriously disrupted.
Competition to hire and retain employees and consultants from this limited pool is intense, and we may be unable to hire, train, retain or motivate these additional key personnel and consultants. Our failure to retain key personnel or consultants could materially harm our business.
Competition to hire and retain employees and consultants from this limited pool is intense, and we may be unable to hire, train, retain or motivate these additional key personnel and consultants.
Biotechnology companies have experienced greater than average stock price volatility in recent years, and our common stock price has been particularly volatile ranging from a high o f $862.20 to a low of $5.07 (taking into a ccount the reverse stock splits we have completed).
Biotechnology companies have experienced greater than average stock price volatility in recent years, and our common stock price has been particularly volatile ranging from a high o f $30.00 to a low of $0.42 over the past two years (taking into a ccount the reverse stock splits we have completed).
Rates of subject enrollment are affected by many factors, including the size of the patient population, the eligibility criteria for the clinical trial, that include the age and condition of the subjects and the stage and severity of disease, the nature of the protocol, the proximity of subjects to clinical sites and the availability of effective treatments and/or availability of investigational treatment options for the relevant disease.
Rates of subject enrollment are affected by many factors, including the size of the patient population, the eligibility criteria for the clinical trial, that include the age and condition of the subjects and the stage and severity of disease, the nature of the protocol, the proximity of subjects to clinical sites and the availability of effective treatments and/or availability of investigational treatment options for the relevant disease. 28 Table of Contents A product candidate can unexpectedly fail at any stage of preclinical and clinical development.
In February 2019, we received notice that the FDA granted ODD for WP1066 for the treatment of glioblastoma and later on for WP1122, as well. ODD from the FDA is available for drugs targeting diseases with less than 200,000 cases per year.
In February 2019, we received notice that the FDA granted ODD for WP1066 for the treatment of glioblastoma and later on for WP1122, as well. ODD from the FDA is available for drugs targeting diseases with less than 200,000 cases per year. ODD does not convey any advantage in, or shorten the duration of, the regulatory review and approval process.
Reliance on third-party manufacturers entails risks to which we would not be subject if we manufactured our product candidates, including: • the possibility that we are unable to enter into a manufacturing agreement with a third party to manufacture our product candidates; • the possible breach of the manufacturing agreements by the third parties because of factors beyond our control; and • the possibility of termination or nonrenewal of the agreements by the third parties before we are able to arrange for a qualified replacement third-party manufacturer. 34 Table of Contents Any of these factors could prevent or cause the delay of approval or commercialization of our product candidates, cause us to incur higher costs or prevent us from commercializing our product candidates successfully.
Reliance on third-party manufacturers entails risks to which we would not be subject if we manufactured our product candidates, including: • the possibility that we are unable to enter into a manufacturing agreement with a third party to manufacture our product candidates; • the possible breach of the manufacturing agreements by the third parties because of factors beyond our control; and • the possibility of termination or nonrenewal of the agreements by the third parties before we are able to arrange for a qualified replacement third-party manufacturer.
Fast Track designation alone does not guarantee qualification for the FDA’s priority review procedures. Interim or preliminary data from our clinical trials that we announce or publish from time to time may change as more subject data become available and are subject to audit and verification procedures that could result in material changes in the final data.
Interim or preliminary data from our clinical trials that we announce or publish from time to time may change as more subject data become available and are subject to audit and verification procedures that could result in material changes in the final data.
Since our IPO in June 2016, our stock price has ranged from a high o f $862.20 to a low of $5.07 (t aking into account the reverse stock splits we have completed), and the market price of our common stock is likely to continue to be highly volatile and could fluctuate widely in response to various factors, many of which are beyond our control.
Over the past two years, our stock price has ranged from a high o f $30.00 to a low of $0.42 (t aking into account the reverse stock splits we have completed), and the market price of our common stock is likely to continue to be highly volatile and could fluctuate widely in response to various factors, many of which are beyond our control.
In addition, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not guarantee regulatory approval in any other country.
Approval by the FDA does not ensure approval by regulatory authorities in any other country or jurisdiction outside the United States. In addition, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not guarantee regulatory approval in any other country.
As of December 31, 2023, we had warrants and options outstanding to purchase an aggregate of 1,621,576 shares of common stock at an average exercise price of $28.65 per share (taking into account the reverse stock splits we have completed).
As of December 31, 2024, we had warrants and options outstanding to purchase an aggregate of 8,594,561 shares of common stock at an average exercise price of $4.33 per share (taking into account the reverse stock splits we have completed).
We may incur penalties if we fail to comply with healthcare regulations. We are exposed to the risk of employee fraud or other illegal activity by our employees, independent contractors, consultants, commercial partners and vendors.
We are exposed to the risk of employee fraud or other illegal activity by our employees, independent contractors, consultants, commercial partners and vendors.
If we are not able to attract and retain necessary personnel and consultants to accomplish our business objectives, we may experience constraints that will significantly impede the achievement of our development objectives, our ability to raise additional capital and our ability to implement our business strategy. 41 Table of Contents We are highly dependent on the development, regulatory, commercialization and business development expertise of our management team, key employees, and consultants.
If we are not able to attract and retain necessary personnel and consultants to accomplish our business objectives, we may experience constraints that will significantly impede the achievement of our development objectives, our ability to raise additional capital and our ability to implement our business strategy.
Further, disclosure of preliminary or interim data by us could result in volatility in the price of our common stock. 35 Table of Contents In addition, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the approvability of the particular drug candidate and our business in general.
In addition, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the approvability of the particular drug candidate and our business in general.
If the interim data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for and commercialize our current or any our future drug candidate, our business, operating results, prospects or financial condition may be materially harmed.
If the interim data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for and commercialize our current or any our future drug candidate, our business, operating results, prospects or financial condition may be materially harmed. 33 Table of Contents We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities.
We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities. Certain laws and regulations relating to drug development require us to test our product candidates on animals before initiating clinical trials involving humans. Animal testing activities have been the subject of controversy and adverse publicity.
Certain laws and regulations relating to drug development require us to test our product candidates on animals before initiating clinical trials involving humans. Animal testing activities have been the subject of controversy and adverse publicity.
Artificial intelligence presents risks and challenges that can impact our business, including by posing security risks to our confidential information , proprietary information and personal data . Issues in the development and use of artificial intelligence, combined with an uncertain regulatory environment, may result in reputational harm, liability, or other adverse consequences to our business operations.
Issues in the development and use of artificial intelligence, combined with an uncertain regulatory environment, may result in reputational harm, liability, or other adverse consequences to our business operations. As with many technological innovations, artificial intelligence presents risks and challenges that could impact our business.
Prior to obtaining approval to commercialize a product candidate in any jurisdiction, we and our collaborators must demonstrate with substantial evidence from well controlled clinical trials, and to the satisfaction of the FDA or comparable foreign regulatory agencies, that such product candidates are safe and effective for their intended uses.
Before obtaining approval to commercialize a product candidate in any jurisdiction, we and our collaborators must demonstrate to the satisfaction of the FDA or comparable foreign regulatory agencies that such product candidates are safe and effective for their intended uses. The FDA requires “substantial evidence” to make a finding of effectiveness for any approval.
We have no exposure to SVB. If other banks and financial institutions enter receivership or become insolvent in the future in response to financial conditions affecting the banking system and financial markets, our ability to access our existing cash, cash equivalents and investments may be threatened and could have a material adverse effect on our business and financial condition.
If other banks and financial institutions enter receivership or become insolvent in the future in response to financial conditions affecting the banking system and financial markets, our ability to access our existing cash, cash equivalents and investments may be threatened and could have a material adverse effect on our business and financial condition. 43 Table of Contents We cannot predict the effect that our reverse stock split will have on the market price for shares of our common stock .
We have no products approved for commercial sale and may never achieve or maintain profitability, which could have an impact on finding additional financing. • Shares issuable upon the exercise of outstanding options or warrants may substantially increase the number of shares available for sale in the public market and depress the price of our common stock. • As a biotechnology company, we are at increased risk of securities class action litigation. • If we are unable to maintain compliance with the listing requirements of The Nasdaq Capital Market, our common stock may be delisted from The Nasdaq Capital Market which could have a material adverse effect on our financial condition and could make it more difficult for you to sell your shares. • Failure to maintain our accounting systems and controls could impair our ability to comply with the financial reporting and internal controls requirements for publicly traded companies. • Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price. • We cannot predict the effect that our reverse stock split will have on the market price for shares of our common stock.
Risks Relating to Our Common Stock • Our stock price has been and may continue to be volatile, which could result in substantial losses for investors. • Shares issuable upon the exercise of outstanding options or warrants may substantially increase the number of shares available for sale in the public market and depress the price of our common stock. • As a biotechnology company, we are at increased risk of securities class action litigation. • If we are unable to maintain compliance with the listing requirements of The Nasdaq Capital Market, our common stock may be delisted from The Nasdaq Capital Market which could have a material adverse effect on our financial condition and could make it more difficult for you to sell your shares. • Failure to maintain our accounting systems and controls could impair our ability to comply with the financial reporting and internal controls requirements for publicly traded companies. • Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price. • We cannot predict the effect that our reverse stock split will have on the market price for shares of our common stock. 25 Table of Contents General Risks • Your ownership may be diluted if additional capital stock is issued to raise capital, to finance acquisitions or in connection with strategic transactions. • Negative research about our business published by analysts or journalists could cause our stock price to decline.
The reverse stock split was effected in accordance with the authorization adopted by our stockholders at a special meeting of stockholders held in October 2023.
On March 22, 2024, we completed a one-for-fifteen reverse stock split of our shares of common stock. The reverse stock split was effected in accordance with the authorization adopted by our stockholders at a special meeting of stockholders held in October 2023.
We may expend significant resources to pursue certain product candidates for specific indications, and fail to capitalize on the potential of such product candidates for the potential treatment of other indications that may be more profitable or for which there is a greater likelihood of success.
Failure to commence or complete, or delays in, our planned clinical trials, could prevent us from or delay us in commercializing our product candidates. 29 Table of Contents We may expend significant resources to pursue certain product candidates for specific indications and fail to capitalize on the potential of such product candidates for the potential treatment of other indications that may be more profitable or for which there is a greater likelihood of success.
We intend to seek to raise additional funds, finance acquisitions or develop strategic relationships by issuing equity or convertible debt securities, which would reduce the percentage ownership of our existing stockholders.
General Risks Your ownership may be diluted if additional capital stock is issued to raise capital, to finance acquisitions or in connection with strategic transactions. We intend to seek to raise additional funds, finance acquisitions or develop strategic relationships by issuing equity or convertible debt securities, which would reduce the percentage ownership of our existing stockholders.
The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and even if we obtain approval for a product candidate in one country or jurisdiction, we may never obtain approval for or commercialize it in any other jurisdiction, which would limit our ability to realize our full market potential.
Failure of Congress to reauthorize the program will limit our ability to obtain an RPDPRV if WP1066 is approved for the treatment any of the four pediatric cancer indications for which WP1066 received rare pediatric disease designation and may limit our ability to obtain future rare pediatric disease designations for our product candidates. 32 Table of Contents The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and even if we obtain approval for a product candidate in one country or jurisdiction, we may never obtain approval for or commercialize it in any other jurisdiction, which would limit our ability to realize our full market potential.
As with many technological innovations, artificial intelligence presents risks and challenges that could impact our business. We may adopt and integrate generative artificial intelligence tools into our systems for specific use cases reviewed by legal and information security.
We may adopt and integrate generative artificial intelligence tools into our systems for specific use cases reviewed by legal and information security.
In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates. As an organization, we have never conducted pivotal clinical trials, and we may be unable to do so for any product candidates we may develop.
In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates.
We are subject to US data protection laws and regulations (i.e., laws and regulations that address privacy and data security) at both the federal and state levels. The legislative and regulatory landscape for data protection continues to evolve, and in recent years there has been an increasing focus on privacy and data security issues.
The legislative and regulatory landscape for data protection continues to evolve, and in recent years there has been an increasing focus on privacy and data security issues.
When we collaborate with a third party for development and commercialization of a product candidate, we can expect to relinquish some or all of the control over the future success of that product candidate to the third party.
We may not be successful in our efforts to establish and implement collaborations or other alternative arrangements for the development of our product candidates. 38 Table of Contents When we collaborate with a third party for development and commercialization of a product candidate, we can expect to relinquish some or all of the control over the future success of that product candidate to the third party.
In order to market any products in any particular jurisdiction, we must establish and comply with numerous and varying regulatory requirements on a country-by-country basis regarding safety and efficacy. Approval by the FDA does not ensure approval by regulatory authorities in any other country or jurisdiction outside the United States.
Even if we believe the nonclinical or clinical data for a product candidate are promising, such data may not be sufficient to support approval by the FDA and other regulatory authorities. In order to market any products in any particular jurisdiction, we must establish and comply with numerous and varying regulatory requirements on a country-by-country basis regarding safety and efficacy.
An investment in our securities is speculative in nature, involves a high degree of risk and should not be made by an investor who cannot bear the economic risk of its investment for an indefinite period of time and who cannot afford the loss of its entire investment. 28 Table of Contents Risks Related to Regulatory Approval and the Development and Commercialization of our Drug Candidates We are developing our drug candidates to treat patients who are extremely or terminally ill, and severe adverse outcomes, including patient deaths, that occur in our clinical trials could negatively impact our business even if such outcomes are not shown to be related to our drugs.
Risks Related to Regulatory Approval and the Development and Commercialization of our Drug Candidates We are developing our drug candidates to treat patients who are extremely or terminally ill, and severe adverse outcomes, including patient deaths, that occur in our clinical trials could negatively impact our business even if such outcomes are not shown to be related to our drugs.
Risks Related to Our Intellectual Property • The composition of matter patent for Annamycin has expired, and other patents have not yet been issued, and may not be issued. • The intellectual property rights we have licensed from MD Anderson are subject to the rights of the US government. • We may incur substantial costs as a result of litigation or other proceedings relating to patent and other intellectual property rights. • We may be subject to claims that our employees have wrongfully used or disclosed alleged trade secrets of their former employers. • If we are not able to adequately prevent disclosure of trade secrets and other proprietary information, the value of our technology and products could be significantly diminished. • If we breach any of the agreements under which we license patent rights or if we fail to meet certain development deadlines, pay certain fees including extension fees or exercise certain rights to technology, we could lose or fail to obtain license rights that are important to our business. • We will not be able to protect our intellectual property rights throughout the world. 27 Table of Contents Risks Relating to Our Business and Financial Condition • We will require additional funding, which may not be available to us on acceptable terms, or at all, and, if not so available, may require us to delay, limit, reduce or cease our operations. • Because successful development of our product candidates is uncertain, we are unable to estimate the actual amount of funding we will require to complete research and development and commercialize our products under development. • We have commenced clinical trials, have a limited operating history and we expect a number of factors to cause our operating results to fluctuate on an annual basis, which may make it difficult to predict our future performance. • We have in the past completed related party transactions that were not conducted on an arm’s length basis. • We have never been profitable, we have no products approved for commercial sale, and to date we have not generated any revenue from product sales.
Risks Relating to Our Business and Financial Condition • We will require additional funding, which may not be available to us on acceptable terms, or at all, and, if not so available, may require us to delay, limit, reduce or cease our operations. • Because successful development of our product candidates is uncertain, we are unable to estimate the actual amount of funding we will require to complete research and development and commercialize our products under development. • We have commenced clinical trials, have a limited operating history and we expect a number of factors to cause our operating results to fluctuate on an annual basis, which may make it difficult to predict our future performance. • We have in the past completed related party transactions that were not conducted on an arm’s length basis. • We have never been profitable, we have no products approved for commercial sale, and to date we have not generated any revenue from product sales.
New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified or applied adversely to us or our customers may have a material adverse effect on our business and financial condition.
If we fail to meet our payment obligations, our license agreements could be terminated, which would materially and adversely affect our business operations and financial condition. 41 Table of Contents New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified or applied adversely to us or our customers may have a material adverse effect on our business and financial condition.
Investors in our common stock should not expect to receive dividend income on their investment, and investors will be dependent on the appreciation of our common stock to earn a return on their investment.
Investors in our common stock should not expect to receive dividend income on their investment, and investors will be dependent on the appreciation of our common stock to earn a return on their investment. Artificial intelligence presents risks and challenges that can impact our business, including by posing security risks to our confidential information, proprietary information and personal data .
In addition, we have scientific and clinical advisors and consultants who assist us in formulating our research, development, and clinical strategies. These advisors are not our employees and may have commitments to, or consulting or advisory contracts with, other entities that may limit their availability to us and typically they will not enter into non-compete agreements with us.
These advisors are not our employees and may have commitments to, or consulting or advisory contracts with, other entities that may limit their availability to us and typically they will not enter into non-compete agreements with us. If a conflict of interest arises between their work for us and their work for another entity, we may lose their services.
There is no assurance that the FDA will accept the results from these clinical trials, which could require us to redo such trials in order to receive approval of our product candidates in the United States. Approval processes vary among countries and can involve additional product testing and validation, as well as additional administrative review periods.
There is no assurance that the FDA will accept the results from these clinical trials, which could require us to conduct additional preclinical studies or clinical trials, either prior to or post-approval, in order to receive approval of our product candidates in the United States.
For the year ended December 31, 2023, we incurred a net loss o f $29.8 million. We had an accumulated deficit of $131.6 million as of December 31, 2023. To date, we have devoted most of our financial resources to research and development, including our drug discovery research, preclinical development activities and clinical trial preparation, as well as corporate overhead.
We had an accumulated deficit of $153.4 million as of December 31, 2024. To date, we have devoted most of our financial resources to research and development, including our drug discovery research, preclinical development activities and clinical trial preparation, as well as corporate overhead. We have not generated any revenues from product sales.
There can be no assurance that we will secure adequate insurance coverage or that any such insurance coverage will be sufficient to protect our operations to significant potential liability in the future. Any significant uninsured liability may require us to pay substantial amounts, which would adversely affect our financial position and results of operations.
We do not carry insurance for all categories of risk that our business may encounter. There can be no assurance that we will secure adequate insurance coverage or that any such insurance coverage will be sufficient to protect our operations to significant potential liability in the future.
To the extent that any disruption or security breach results in a loss of or damage to our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur liability and development of our product candidates could be delayed or could fail. 42 Table of Contents The COVID-19 outbreak delayed recruitment in our clinical trials in the past and may return, may affect the business of the FDA, EMA or other health authorities, which could result in delays in meetings related to our planned clinical trials and ultimately of reviews and approvals of our product candidates.
To the extent that any disruption or security breach results in a loss of or damage to our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur liability and development of our product candidates could be delayed or could fail.
If we lose one or more of our executive officers or key employees or consultants, our ability to implement our business strategy successfully could be seriously harmed. Any of our executive officers or key employees or consultants may terminate their employment at any time.
We are highly dependent on the development, regulatory, commercialization and business development expertise of our management team, key employees, and consultants. If we lose one or more of our executive officers or key employees or consultants, our ability to implement our business strategy successfully could be seriously harmed.
As a result, our ability to reduce our losses and reach profitability is unproven, and we may never achieve or sustain profitability. We have never been profitable and do not expect to be profitable in the foreseeable future. We have not yet submitted any drug candidates for approval by regulatory authorities in the United States or elsewhere.
We have never been profitable and do not expect to be profitable in the foreseeable future. We have not yet submitted any drug candidates for approval by regulatory authorities in the United States or elsewhere. For the year ended December 31, 2024, we incurred a net loss of $21.8 million.
Seeking regulatory approval could result in difficulties and costs for us and require additional nonclinical studies or clinical trials, which could be costly and time consuming. Regulatory requirements can vary widely from country to country and could delay or prevent the introduction of our products in those countries.
Seeking regulatory approval could result in difficulties and costs for us and require additional nonclinical studies or clinical trials, which could be costly and time consuming and could impair our ability to generate revenue from future drug sales or other sources.
A product candidate can unexpectedly fail at any stage of preclinical and clinical development. The historical failure rate for product candidates is high due to scientific feasibility, safety, efficacy, changing standards of medical care and other variables.
The historical failure rate for product candidates is high due to scientific feasibility, safety, efficacy, changing standards of medical care and other variables. The results from preclinical testing or early clinical trials of a product candidate may not predict the results that will be obtained in later phase clinical trials of the product candidate.
While we have obtained orphan drug designation for Annamycin, if we seek additional indications, or fail to maintain our orphan drug status, we may become subject to the price negotiation process. This could reduce the ultimate price that we receive for Annamycin, which could negatively affect our business, results of operations, financial conditions, and prospects.
This could reduce the ultimate price that we receive for Annamycin, which could negatively affect our business, results of operations, financial conditions, and prospects.
We will need to successfully complete pivotal clinical trials in order to obtain the approval of the FDA, EMA or other regulatory agencies to market our product candidates. Carrying out pivotal clinical trials is a complicated process.
As an organization, we have never before conducted pivotal clinical trials, and we may be unable to do so for any product candidates we may develop. We will need to successfully complete pivotal clinical trials in order to obtain the approval of the FDA, EMA or other regulatory agencies to market our product candidates.
We also expect to continue to rely on third parties to conduct our pivotal clinical trials. Consequently, we may be unable to successfully and efficiently execute and complete necessary clinical trials in a way that leads to NDA submission and approval of our product candidates.
Consequently, we may be unable to successfully and efficiently execute and complete necessary clinical trials in a way that leads to NDA submission and approval of our product candidates. We may require more time and incur greater costs than our competitors and may not succeed in obtaining regulatory approvals of product candidates that we develop.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeFor additional information regarding risks from cybersecurity threats, please refer to Item 1A, “Risk Factors,” in this Annual report on Form 10-K. Governance One of the key responsibilities of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats.
Biggest changeFor additional information regarding risks from cybersecurity threats, please refer to Item 1A, “Risk Factors,” in this Annual report on Form 10-K. 45 Table of Contents Governance One of the key responsibilities of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats.
As of December 31, 2023, and through the date of the filing of this report, we are not aware of any cybersecurity incidents that have materially affected or are reasonably likely to materially affect us, including our business strategy, results of operations or financial condition.
As of December 31, 2024, and through the date of the filing of this report, we are not aware of any cybersecurity incidents that have materially affected or are reasonably likely to materially affect us, including our business strategy, results of operations or financial condition.
Our Head of IT and Cybersecurity has served as an information technology professional for over eight years and has held senior IT positions at multiple companies, where his primary responsibilities included maintaining direct oversight over his companies’ cybersecurity risks.
Our Head of IT and Cybersecurity has served as an information technology professional for over nine years and has held senior IT positions at multiple companies, where his primary responsibilities included maintaining direct oversight over his companies’ cybersecurity risks.
The audit committee, in turn, provides periodic reports to our board of directors regarding our cybersecurity processes, including the results of cybersecurity risk assessments. 48 Table of Contents
The audit committee, in turn, provides periodic reports to our board of directors regarding our cybersecurity processes, including the results of cybersecurity risk assessments.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeWe have insurance policies covering potential losses where such coverage is cost effective. We are not at this time involved in any legal proceedings that we believe could have a material effect on our business, financial condition, results of operations or cash flows.
Biggest changeWe have insurance policies covering potential losses where such coverage is cost effective. We are not at this time involved in any legal proceedings that we believe could have a material effect on our business, financial condition, results of operations or cash flows. ITEM 4. MINE SAFETY DISCLOSURE Not applicable. PART II

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed on the NASDAQ Capital Market under the symbol “MBRX”. Holders As of Marc h 14, 2024, there were approximately 138 hol ders of record of our common stock.
Biggest changeITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed on the NASDAQ Capital Market under the symbol “MBRX”. Holders As o f March 13, 2025, there were approximately 139 holders of record of our common stock.
Recent Sales of Unregistered Securities All information related to equity securities sold by us during the period covered by this report that were not registered under the Securities Act have been included in our Form 10-Q filings or in a Form 8-K filing.
Recent Sales of Unregistered Securities Except as set forth below, all information related to equity securities sold by us during the period covered by this report that were not registered under the Securities Act have been included in our Form 10-Q filings or in a Form 8-K filing.
In addition, we believe that a significant number of beneficial owners of our common stock hold their shares in nominee or in “street name” accounts through brokers. Dividends We have never paid any dividends on our common stock.
I n addition, we believe that a significant number of beneficial owners of our common stock hold their shares in nominee or in “street name” accounts through brokers. 46 Table of Contents Dividends We have never paid any dividends on our common stock.
Purchases of Equity Securities by the Issuer and Affiliated Purchasers We did not repurchase any of our equity securities during the year ended December 31, 2023.
Purchases of Equity Securities by the Issuer and Affiliated Purchasers We did not repurchase any of our equity securities during the year ended December 31, 2024. ITEM 6. [RESERVED]
Added
During the quarter ended December 31, 2024, we issued ten-year warrants to purchase an aggregate of 36,000 shares of our common stock at an exercise price of $2.36 per share to four entities that provided services to the Company.
Added
The warrants and the shares issuable upon exercise of the warrants are being sold and issued without registration under the Securities Act in reliance on the exemptions provided by Section 4(a)(2) of the Securities Act as transactions not involving a public offering, and in reliance on similar exemptions under applicable state laws.

Item 7. Management's Discussion & Analysis

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

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Biggest changeResults of Operations for the Year Ended December 31, 2023 as Compared to the Year Ended December 31, 2022 The following table is data derived from the Consolidated Statement of Operations (in thousands) and the discussions that follow are in approximate amounts: Year ended December 31, 2023 2022 Revenue $ — $ — Operating expenses: Research and development 19,487 18,968 General and administrative 10,017 11,542 Depreciation and amortization 127 130 Total operating expense 29,631 30,640 Loss from operations (29,631 ) (30,640 ) Other income: (Loss) gain from change in fair value of warrant liability (1,044 ) 1,335 Other income, net 48 40 Interest income, net 1,368 240 Transaction costs allocated to warrant liabilities (510 ) Net loss $ (29,769 ) $ (29,025 ) Research and Development Expense Research and development (R&D) expense w as $19.5 million an d $19.0 million for the years ended December 31, 2023 and 2022, respectively.
Biggest changeResults of Operations for the Year Ended December 31, 2024 as Compared to the Year Ended December 31, 2023 The following table is data derived from the Consolidated Statement of Operations (in thousands) and the discussions that follow are in approximate amounts: Year ended December 31, 2024 2023 Revenue $ — $ — Operating expenses: Research and development 17,729 19,487 General and administrative 8,786 10,017 Depreciation and amortization 126 127 Total operating expense 26,641 29,631 Loss from operations (26,641 ) (29,631 ) Other income: Gain (loss) from change in fair value of warrant liability 6,125 (1,044 ) Transaction costs allocated to warrant liabilities (993 ) (510 ) Loss on issuance of warrant liabilities (847 ) — Other income, net 43 48 Interest income, net 550 1,368 Net loss $ (21,763 ) $ (29,769 ) 48 Table of Contents Research and Development Expense Research and development (R&D) expense w as $17.7 million an d $19.5 million for the years ended December 31, 2024 and 2023, respectively.
We estimate leasehold improvements to have a estimated useful life over the term of the lease or the estimated useful life, whichever is shorter; computer equipment to have a 2-year life; software to have a 3-year life, machinery and equipment to have a 2 to 5 year life and furniture and office equipment to have a 2 to 7 year life.
We estimate leasehold improvements to have an estimated useful life over the term of the lease or the estimated useful life, whichever is shorter; computer equipment to have a 2-year life; software to have a 3-year life, machinery and equipment to have a 2 to 5 year life and furniture and office equipment to have a 2 to 7 year life.
We believe such compounds may provide an opportunity to cut off the fuel supply of tumors by taking advantage of their high degree of dependence on glucose in comparison to healthy cells, as well as viruses that also depend upon glycolysis and glycosylation to infect and replicate.
We believe such compounds may provide an opportunity to cut off the energy supply of tumors by taking advantage of their high degree of dependence on glucose in comparison to healthy cells, as well as viruses that also depend upon glycolysis and glycosylation to infect and replicate.
Impairment of Long-Lived Assets We evaluate the recoverability of our property and equipment and amortizable intangible assets for possible impairment whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable or at a minimum annually during the fourth quarter of the year.
Impairment of Long-Lived Assets We evaluate the recoverability of our property and equipment and amortizable intangible assets for possible impairment whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable or at a minimum annually during the third quarter of the year.
We account for our warrants issued in accordance with Accounting Standards Codification (ASC) Topic 815, Derivatives and Hedging, guidance applicable to derivative instruments, which requires every derivative instrument within its scope to be recorded on the balance sheet as either an asset or liability measured at its fair value, with changes in fair value recognized in earnings for liability classified warrants.
We account for our warrants issued in accordance with Accounting Standards Codification (ASC) Topic 815, Derivatives and Hedging, which requires every derivative instrument within its scope to be recorded on the balance sheet as either an asset or liability measured at its fair value, with changes in fair value recognized in earnings for liability classified warrants.
ASC 740 Income Taxes is an asset and liability approach that requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in our financial statements or tax returns.
ASC 740 is an asset and liability approach that requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in our financial statements or tax returns.
Based on this guidance, we determined that certain of our warrants to purchase shares of common stock related to equity transactions in 2017, 2018, 2019, 2020 , and 2023 meet the criteria for classification as a liability. Accordingly, the warrants were classified as a warrant liability and are subject to fair value remeasurement at each transaction and balance sheet date.
Based on this guidance, we determined that certain of our warrants to purchase shares of common stock related to equity transactions in 2020 , and 2023 meet the criteria for classification as a liability. Accordingly, the warrants were classified as a warrant liability and are subject to fair value remeasurement at each transaction and balance sheet date.
This takes into account cash outlays for preparations for clinical trials beyond the current active trials. The continuation of our Company as a going concern is dependent upon our ability to obtain necessary financing to continue operations and the attainment of profitable operations.
This takes into account cash outlays for preparations for clinical trials beyond the current active trials. The continuation of our Company as a going concern is depe ndent upon our ability to obtain necessary financing to continue operations and the attainment of profitable operations.
We also had $2.5 million of accounts payable and $4.3 million of accrued expenses and other current liabilities. A significant portion of the accounts payable and accrued expenses are due to work performed in relation to our preclinical activities and our clinical trials.
We also had $2.0 million of accounts payable and $3.3 million of accrued expenses and other current liabilities. A significant portion of the accounts payable and accrued expenses are due to work performed in relation to our preclinical activities and our clinical trials.
Our Core Technologies Our core technologies consist of the following: a) Annamycin or L-Annamycin is a “next generation” anthracycline (one of the most common classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks and their susceptibility to multidrug resistance mechanisms.
Our core technologies consist of the following programs: a) Annamycin or L-Annamycin is a “next generation” anthracycline (one of the most widely used classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks and their susceptibility to multidrug resistance mechanisms.
Beginning in 2020, only the volatility of our stock was used in the BSM as we now have sufficient historic data in our stock price. Income taxes We account for income taxes using ASC 740, Income Taxes.
Beginning in 2020, only the volatility of our stock was used in the BSM as we now have sufficient historic data in our stock price. 52 Table of Contents Income taxes We account for income taxes using ASC 740, Income Taxes.
Warrants are classified as liabilities when the Company may be required to settle a warrant exercise in cash and classified as equity when the Company settles a warrant exercise in shares of its common stock. We issued warrants to purchase shares of common stock related to equity transactions in 2017, 2018, 2019, 2020, 2021, 2022, and 2023.
Warrants are classified as liabilities when the Company may be required to settle a warrant exercise in cash and classified as equity when the Company settles a warrant exercise in shares of its common stock. We issued warrants to purchase shares of common stock related to equity transactions in 2020, 2021, 2022, 2023, and 2024.
We cannot predict when this matter will be resolved or what, if any, action the SEC may take following the conclusion of the investigation. During the year ended December 31, 2023 and 2022, we have expensed approximately $1.5 and $2.4 million, respectively, in related legal fees and expenses, which has impacted and may continue to impact our liquidity.
We cannot predict when this matter will be resolved or what, if any, action the SEC may take following the conclusion of the investigation. During the years ended December 31, 2024 and 2023, we have expensed approximately $0.2 million and $1.5 million, respectively, in related legal fees and expenses, which has impacted and may continue to impact our liquidity.
Cash provided by financing activities Net cash provided by financing activities was $4.7 million f or the year ended December 31, 2023, consisting of the December 2023 stock offering, as well as shares issued utilizing the Lincoln Park Equity Line. Off-Balance Sheet Transactions We do not engage in off-balance sheet transactions.
Net cash provided by financing activities was $4.1 million for the year ended December 31, 2023, consisting of the December 2023 stock offering, as well as shares issued utilizing the Lincoln Park Equity Line. Off-Balance Sheet Transactions We do not engage in off-balance sheet transactions.
The fair value of this warrant liability associated with the February 2017, February 2018, June 2018, March 2019, April 2019, February 2020, and December 2023 Off erings (Offerings) are included in long-term liabilities on the accompanying financial statements as of December 31, 2023 and 2022 respectively.
The fair value of this warrant liability associated with the February 2017, February 2018, June 2018, March 2019, April 2019, February 2020, D ecember 2023, and August 2024 Off erings (Offerings) are included in long-term liabilities on the accompanying financial statements as of December 31, 2024 and 2023 respectively.
(Loss) Gain from Change in Fair Value of Warrant Liability We recorded a loss of $1.0 million durin g the year ended December 31, 2023 as compared to a gain of $1.3 million, during the year ended December 31, 2022, for the change in fair value on revaluation of our warrant liability associated with our warrants issued in conjunction with our stock offerings.
Gain (Loss) from Change in Fair Value of Warrant Liability We recorded a gain o f $6.1 million durin g the year ended December 31, 2024 as compared to a loss of $1.0 million, during the year ended December 31, 2023, for the change in fair value on revaluation of our warrant liability associated with our warrants issued in conjunction with our stock offerings.
General and Administrative Expense General and administrative (G&A) expense was $10.0 million a nd $11.5 million for the years ended December 31, 2023 and 2022, respectively. The decrease in G&A of $1.5 million wa s mainly attributable to a decrease in regulatory and legal services, and consulting & advisory fees.
General and Administrative Expense General and administrative (G&A) expense was $8.8 million a nd $10.0 million for the years ended December 31, 2024 and 2023, respectively. Th e decrease in G&A of $1.2 million wa s mainly attributable to a decrease in regulatory and legal services, and consulting & advisory fees.
We have three core technologies, each of which have had one or more drugs successfully complete a Phase 1 clinical trial, based substantially on discoveries made at and licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas.
Each of our three core technologies is based substantially on discoveries made at and licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas, and features one or more drugs that have successfully completed a Phase 1 clinical trial.
Critical Accounting Policies and Significant Judgments and Estimates The accompanying consolidated financial statements and related notes have been prepared in accordance with accounting principles generally accepted in the United States of America (US GAAP) for financial information, and in accordance with the rules and regulations of the SEC.
Refer to Note 2 for additional discussion. 50 Table of Contents Critical Accounting Policies and Significant Judgments and Estimates The accompanying consolidated financial statements and related notes have been prepared in accordance with accounting principles generally accepted in the United States of America (US GAAP) for financial information, and in accordance with the rules and regulations of the SEC.
We expect our general and administrative expense to increase due to the anticipated growth of our business and related infrastructure as well as accounting, insurance, investor relations and other costs associated with being a public company. Depreciation and Amortization. Depreciation and amortization expense consists of depreciation on our property and equipment. We depreciate our assets over their estimated useful life.
We expect our general and administrative expense to increase due to the anticipated growth of our business and related infrastructure as well as accounting, insurance, investor relations and other costs associated with being a public company. 51 Table of Contents Depreciation and Amortization. Depreciation and amortization expense consists of depreciation on our property and equipment.
Three of our six drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2 or Phase 2 clinical trials. Since our inception, our drugs have completed, are currently in, or have been permitted to proceed in thirteen clinical trials.
Three of our six drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2 or Phase 2 clinical trials. One is currently beginning a Phase 2B/3 trial. Since our inception, our drugs have completed, are currently in, or have been permitted to proceed in, fourteen clinical trials.
The fair value hierarchy gives the highest priority to quoted prices in active markets for identical assets and liabilities (Level 1) and lowest priority to unobservable inputs (Level 3). 55 Table of Contents Assets and liabilities recorded in the balance sheets at fair value are categorized based on a hierarchy of inputs as follows: Level 1 - Unadjusted quoted prices in active markets of identical assets or liabilities.
Assets and liabilities recorded in the balance sheets at fair value are categorized based on a hierarchy of inputs as follows: Level 1 - Unadjusted quoted prices in active markets of identical assets or liabilities.
Annamycin is our lead molecule and is in three Phase 1B/2 clinical trials - one for treating Acute Myeloid Leukemia (AML) and two for treating Soft Tissue Sarcoma metastasized to the lungs (STS lung metastases, STS lung mets, or Advanced STS).
Annamycin is also in two Phase 1B/2 clinical trials for treating Soft Tissue Sarcoma metastasized to the lungs (STS lung metastases, STS lung mets, or Advanced STS).
Proceeds of the December 2023 Offering were allocated between common shares and warrants first by allocating proceeds to the warrants classified as a liability based on their fair value and then allocating the residual to the equity instruments, which includes the Pre-Funded Warrants. Our financial instruments consist primarily of non-trade receivables, account payables, accrued expenses, and a warrant liability.
Proceeds of the December 2023 and August 2024 Offerings were allocated between common shares and warrants first by allocating proceeds to the warrants classified as a liability based on their fair value and then allocating the residual to the equity instruments, which includes the Pre-Funded Warrants.
The forward-looking statements included in this discussion and elsewhere in this Form 10-K involve risks and uncertainties, including those set forth under “Cautionary Statement About Forward-Looking Statements.” Actual results and experience could differ materially from the anticipated results and other expectations expressed in our forward-looking statements as a result of a number of factors, including but not limited to those discussed in this Item and in Item 1A - “Risk Factors.” Our Business Summary We are a clinical stage pharmaceutical company with a growing pipeline, including Phase 2 clinical programs for hard-to-treat cancers and viruses.
The forward-looking statements included in this discussion and elsewhere in this Form 10-K involve risks and uncertainties, including those set forth under “Cautionary Statement About Forward-Looking Statements.” Actual results and experience could differ materially from the anticipated results and other expectations expressed in our forward-looking statements as a result of a number of factors, including but not limited to those discussed in this Item and in Item 1A - “Risk Factors.” Our Business Summary We are a late-stage pharmaceutical development company currently conducting a pivotal Phase 3 trial evaluating Annamycin, a non-cardiotoxic anthracycline, in combination with Cytarabine for the treatment of subjects with relapsed/refractory acute myeloid leukemia (AML).
Generally, a gain results principally from a decline in our share price during the period and a loss results principally from an increase in our share price. 52 Table of Contents Net Loss The net loss for the year ended December 31, 2023 wa s $29.8 million, which included a non-cash loss of $1.0 million on wa rrants in 2023 as compared to a gain of $1.3 million in the prior year and approximatel y $2.0 million o f stock-based compensation expense in 2023 as compared to $2.3 million in 2022.
Net Loss The net loss for the year ended December 31, 2024 wa s $21.8 million, which included a non-cash gain of $6.1 million on wa rrants in 2024 as compared to a loss of $1.0 million in the prior year and approximatel y $1.7 million o f stock-based compensation expense in 2024 as compared to $2.0 million in 2023.
In the December 2023 Offering, we agreed not to utilize the Lincoln Park Agreement or any such extension thereof, until after June 26, 2024. 53 Table of Contents The following table sets forth the primary sources and uses of cash for the years indicated (in thousands): For the Year Ended December 31, 2023 2022 Net cash used in operating activities $ (24,101 ) $ (27,639 ) Net cash used in investing activities (124 ) (67 ) Net cash provided by (used in) financing activities 4,651 (23 ) Effect of exchange rate changes on cash and cash equivalents (21 ) (29 ) Net change in cash and cash equivalents $ (19,595 ) $ (27,758 ) As of December 31, 2023, there was $0.3 million of cas h on hand in a bank account in Australia and we know of no related limitations impacting our liquidity in Australia.
The following table sets forth the primary sources and uses of cash for the years indicated (in thousands): For the Year Ended December 31, 2024 2023 Net cash used in operating activities $ (23,862 ) $ (23,591 ) Net cash used in investing activities (13 ) (124 ) Net cash provided by financing activities 4,635 4,141 Effect of exchange rate changes on cash and cash equivalents (32 ) (21 ) Net change in cash and cash equivalents $ (19,272 ) $ (19,595 ) As of December 31, 2024, there was $0.6 million of cas h on hand in a bank account in Australia and we know of no related limitations impacting our liquidity in Australia.
For the years ended December 31, 2023 and 2022, we used approxima tely $24.1 million and $27.6 million of cash in operating activities, respectively, which represents cash outlays for research and development and general and administrative expenses in such periods.
For the years ended December 31, 2024 and 2023, we used approxima tely $23.9 million and $23.6 million of cash in operating activities, respectively, which represents cash outlays for research and development and general and administrative expenses in such periods. The slightly increased cash outflows in 2024 was primarily due to timing of payments for sponsored research and other expenses.
Interest income, net Interest income, net increased by approximately $1.1 million for the comparable period due to rising interest rates during the past year. Liquidity and Capital Resources As of December 31, 2023 , we had cash and cash equivalents of $23.6 million and prepaid expenses and other current assets of $2.7 million.
Interest income, net Interest income, net decreased by approximately $0.8 million for the comparable period due to a decreasing cash balance, coupled with decreasing interest rates during the past year. Liquidity and Capital Resources As of December 31, 2024 , we had cash and cash equivalents of $4.3 million and prepaid expenses and other current assets of $0.9 million.
The increase in R&D of $0.5 million is mainly rela ted to the $1.5 million WPD sublicense termination, which enabled the reacquisition of our intellectual property rights in certain territories, including parts of the European Union. This was offset by $1.0 million in costs related to the timing of costs incurred for clinical trials and sponsored research.
The decrease in R&D of $1.8 million is mainly related to the $1.5 million WPD sublicense termination in 2023, which enabled the reacquisition of our intellectual property rights in certain territories, including parts of the European Union.
The carrying amount of non-trade receivables, accounts payables, and accrued expenses approximates their fair value because of the short-term maturity of such. We have categorized our assets and liabilities that are valued at fair value on a recurring basis into three-level fair value hierarchy in accordance with GAAP.
We have categorized our assets and liabilities that are valued at fair value on a recurring basis into a three-level fair value hierarchy in accordance with GAAP.
Such a formulation will require additional preclinical work prior to a clinical trial. We have established an RP2D for WP1122 to potentially enable future externally funded oncology and virology trials. Beyond this, we support development of our core technologies through several externally funded clinical trials and primarily externally funded non-clinical research, with the potential for further studies in the future.
Beyond this, we support development of our core technologies through several externally funded clinical trials and primarily externally funded non-clinical research, with the potential for further studies in the future.
We evaluate the recoverability of our IPR&D assets for possible impairment annually during the fourth quarter or whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Recoverability of IPR&D assets is measured by a comparison of the carrying amounts its fair value.
Acquired in-process research and development (IPR&D) assets are considered indefinite lived until the completion or abandonment of the associated research and development efforts. We evaluate the recoverability of our IPR&D assets for possible impairment annually during the fourth quarter or whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable.
Cash used in investing activities Net cash used in investing activities w as de minimis f or the years ended December 31, 2023 and December 31, 2022, respectively.
Cash used in investing activities Net cash used in investing activities w as de minimis f or the years ended December 31, 2024 and December 31, 2023, respectively. Cash provided by financing activities Net cash provided by financing activities was $4.6 million f or the year ended December 31, 2024, consisting of the proceeds from the August 2024 stock offering.
Recoverability of these assets is measured by a comparison of the carrying amounts to the future undiscounted cash flows the assets are expected to generate.
Recoverability of these assets is measured by a comparison of the carrying amounts to the future undiscounted cash flows the assets are expected to generate. If such review indicates that the carrying amount of property and equipment and amortizable intangible assets is not recoverable, the carrying amount of such asset is reduced to fair value.
Cash used in operating activities Net cash used in operating activities was$24.1 million for the year ended December 31, 2023 compared to $27.6 million for the year ended December 31, 2022. This decrease in use of cash for operations was mainly due to an overall decrease in expenses, timing of payments, as well as license rights settled in stock.
Cash used in operating activities Net cash used in operating activities was $23.9 million for the year ended December 31, 2024 compared to $23.6 million for the year ended December 31, 2023. This slight increase in use of cash for operations was mainly due to timing of payments related to sponsored research and other expenses.
If such review indicates that the carrying amount of IPR&D assets is not recoverable, the carrying amount of such asset is reduced to fair value. Components of our Results of Operations, Net Loss and Financial Condition Operating expenses We classify our operating expenses into three categories: research and development, general and administrative and depreciation. Research and development .
Components of our Results of Operations, Net Loss and Financial Condition Operating expenses We classify our operating expenses into three categories: research and development, general and administrative and depreciation. Research and development .
Cash used in investing activities for the years ended December 31, 2023 and 2022 was approximately$0.1 million.
F or the year ended December 31, 2024, there were $4.6 million in net proceeds from financing activities. In 2023, there were $4.1 million in net proceeds from financing activities. Cash used in investing activities for the years ended December 31, 2024 and 2023 was approximatel y $0.0 million, and $0.1 million, respectively.
WP1066 and WP1220 have both independently successfully completed Phase 1 clinical trials and have demonstrated efficacy as described further below. c) Our WP1122 Portfolio contains compounds (including WP1122, WP1096, and WP1097) designed to exploit the potential uses of inhibitors of glycolysis such as 2-deoxy-D-glucose (2-DG).
These also stimulate a natural immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs). c) Our WP1122 Portfolio contains compounds (including WP1122, WP1096, and WP1097) designed to exploit the potential uses of inhibitors of glycolysis such as 2-deoxy-D-glucose (2-DG).
In the case of Annamycin, our unique use of lipid technology enables improved tissue/organ distribution and as demonstrated in multiple clinical trials, dramatically reduced toxicity, including cardiotoxicity. 51 Table of Contents b) Our WP1066 Portfolio includes, WP1066, WP1193 and WP1220, three of several Immune/Transcription Modulators in the portfolio designed to inhibit p-STAT3 (phosphorylated signal transducer and activator of transcription) among other transcription factors associated with tumor activity.
Furthermore, we have demonstrated safe dosing significantly beyond the dose limitations imposed by regulatory authorities upon commonly prescribed anthracyclines due to their inherent cardiotoxicity. b) Our WP1066 Portfolio includes WP1066, WP1193 and WP1220, three of several Immune/Transcription Modulators in the portfolio designed to inhibit p-STAT3 (phosphorylated signal transducer and activator of transcription) among other transcription factors associated with tumor activity.
Our Focus We are focused on internally funded development of: 1) Annamycin in combination with Cytarabine (also known as Ara-C, the combination with Annamycin of which is referred to as AnnAraC) for the treatment of AML. 2) Annamycin for the treatment of STS metastasized to the lungs . 3) A better formulation for delivery of a molecule from the WP1066 portfolio to possibly further support future externally funded oncology clinical trials.
In combination with Cytarabine (also known as Ara-C, the combination with Annamycin of which is referred to as AnnAraC) for the treatment of R/R AML, and b. For the treatment of STS metastasized to the lungs. 2.
Annamycin was designed to avoid multidrug resistance and to be non-cardiotoxic and has shown no cardiotoxicity in subjects treated in clinical trials to date. Furthermore, we have demonstrated safe dosing beyond the dose limitations imposed by regulatory authorities upon currently prescribed anthracyclines due to their inherent cardiotoxicity.
Annamycin was designed to avoid multidrug resistance and to be non-cardiotoxic and, with intensive cardiac monitoring, has shown no cardiotoxicity in subjects treated in our five Annamycin clinical trials to date.
We believe that our cash resources as of December 31, 2023, will be sufficient to fund our planned operations, which include our current Phase 1B/2 clinical programs and preparations for future clinical trials, into the fourth quarter of 2024, without the issuance of additional equity for cash.
We believe that our cash resources as of December 31, 2024, along with $9.3 m illion in gross proceeds received via our financing activities in the first quarter of 2025 (see Recent Stock Offerings below) will be sufficient to fund our planned operations into the third quarter of 2025, without the issuance of additional equity for cash.
We calculated the fair value of the warrants outstanding using the Black-Scholes model.
We calculated the fair value of the warrants outstanding using the Black-Scholes model. Generally, a gain results principally from a decline in our share price during the period and a loss results principally from an increase in our share price.
Subject to certain ownership limitations, each Pre-Funded Warrant is exercisable into one share of common stock at a price per share of $0.001 (as adjusted from time to time in accordance with the terms thereof).
The combined purchase price for the securities was $1.07 per share of common stock (or pre-funded warrant in lieu thereof) and accompanying common warrant. Each pre-funded warrant is exercisable for one share of common stock at an exercise price of $0.001 per share.
The Company received gross proceeds of $4.5 million, before deducting the placement agent's fees and other offering expenses payable by the Company. Lincoln Park Equity Lines In June 2021, we entered into a Purchase Agreement with Lincoln Park Capital Fund (Lincoln Park Agreement).
In October 2024, the Company’s shareholders approved the issuance of both the August 2024 warrants, as well as the warrant amendment. The Company received gross proceeds of $5.5 million, before deducting the placement agent's fees and other offering expenses payable by the Company.
If such review indicates that the carrying amount of property and equipment and amortizable intangible assets is not recoverable, the carrying amount of such asset is reduced to fair value. 54 Table of Contents Acquired in-process research and development (IPR&D) assets are considered indefinite lived until the completion or abandonment of the associated research and development efforts.
Recoverability of IPR&D assets is measured by a comparison of the carrying amounts its fair value. If such review indicates that the carrying amount of IPR&D assets is not recoverable, the carrying amount of such asset is reduced to fair value.
One of our core management beliefs is that anthracyclines represent the most important treatment for AML and Advanced STS, and we believe Annamycin may, for the first time ever, allow a majority of these patients to benefit from this treatment. This belief leads us to currently focus mainly on the development of Annamycin.
We believe that such a benefit would be disruptive to the competitive landscape for these markets. This belief, coupled with our limited resources, leads us to currently focus mainly on the development of Annamycin.
Stock Offerings In December 2023, the Company entered into a Securities Purchase Agreement with an institutional investor and certain of the Company's executive officers, employees, advisors and a member of its board of directors for the sale by the Company of 240,151 shares (taking into account the reverse stock splits we have completed) of the Company's common stock, and pre-funded warrants to purchase 229,506 shares of common stock (taking into account the reverse stock splits we have completed) in lieu thereof in a registered direct offering.
Recent Stock Offerings In February 2025, the Company entered into a securities purchase agreement with an institutional investor for the sale by the Company of 1,150,000 shares of common stock, and 2,121,029 pre-funded warrants to purchase shares of common stock, and common warrants to purchase up to 6,543,058 shares of common stock.
Removed
Annamycin is demonstrating efficacy in two of its Phase 1B/2 trials as described further below in subjects with AML and Advanced STS. We believe that Annamycin has potential to fill an unmet need as a second line therapy (2nd line or 2L) in AML and potentially as first line therapy in Advanced STS.
Added
This Phase 3 trial should have an interim unblinding of data by the end of 2025, less than a year from its commencement, and an additional unblinding in the first half of 2026.
Removed
As part of our Annamycin clinical trials, we have engaged an independent expert in assessing cardiotoxicity associated with chemotherapy at the Cleveland Clinic (Expert or Independent Expert). The data made available to the Expert includes left ventricular ejection fraction (LVEF) as determined by echocardiograms, and ECHO strain imaging, as well as Troponin levels (a biochemical marker of acute heart damage).
Added
We believe such early visibility for a pivotal registration-enabling trial is highly unique in that stakeholders will receive preliminary safety and efficacy data in the “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) within one year of dosing the first subject.
Removed
“ECHO strain imaging” is a method in echocardiography (medical ultrasound) for measuring regional or global deformation (contraction or beating) of the myocardium (heart muscle). By strain rate imaging, the simultaneous function of different regions can be displayed and measured. Cardiac health biomarkers such as blood Troponin levels are considered an indicator of potential long-term heart damage.
Added
Additionally, we have two portfolios of technologies for hard-to-treat cancers and viruses with clinical and preclinical research funded by investigators at academic institutions.
Removed
The Expert has issued and will continue to issue periodic reports as additional data are provided to him in batches of subject data. Such data include some data which are preliminary and subject to change. In our discussions regarding the lack of Annamycin’s cardiotoxicity, we rely on the Expert’s assessment .
Added
Annamycin is in a class of drugs referred to as Anthracyclines, which are an inhibitor of topoisomerase II, enabling them to cause DNA damage in rapidly replicating tumor cells.
Removed
Annamycin benefits from a promising advancement in lipid enabled drug delivery developed in collaboration with and exclusively licensed from MD Anderson. The unique patent-pending lipid composition allows us to combine a new concept in chemotherapeutic agents within a lipid structure that helps target the delivery of the payload and reduce the potential for toxicity.
Added
Annamycin, in a unique multilamellar lipid formulation, is our lead molecule and we have recently concluded one Phase 1B/2 clinical trial for treating Acute Myeloid Leukemia (AML) and are embarking on a Phase 3 clinical trial for the treatment of AML, which we believe will be pivotal.
Removed
These also stimulate a natural immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs). WP1066, in oral formulation, has been in two clinical trials, including compassionate use cases. WP1066 and WP1193 are being tested in preclinical programs in intravenous (IV) formulations. WP1066 and WP1220 have been in clinical trials in a topical formulation.
Added
We believe that our lead drug candidate Annamycin has summarily: ● Demonstrated significant efficacy in Phase 1 and 2 cancer clinical trials – specifically relapsed and refractory (R/R) AML with complete remission rates significantly above currently approved second line therapies and STS lung mets with overall survival as a median seventh line therapy comparable to overall survival seen with approved first line monotherapies; ● Shown encouraging activity in five different clinical trials (most of which are now complete), funded both internally and externally (through investigator initiated trials); ● Shown in its clinical trials to be non-cardiotoxic (N=84) with some patients being safely dosed at five times the typical lifetime maximum allowed anthracycline dose, which potentially enables repeated dosing and consolidation; ● Demonstrated in preclinical models, to be more potent than and to avoid cross resistance with some of the most common drugs used to treat AML (as well as many other cancers), including currently approved anthracyclines, Cytarabine and Venetoclax; ● Composition of matter patent protection thru 2040 and has orphan drug and fast track status; ● No vesicant activity, making it safer to handle and administer as compared to other anthracyclines; and, ● Shown a complete response (CR) rate of 50%, a composite complete response (CRc) rate of 60%, durability of CRc’s of approximately 9 months (and climbing), and an overall survival (OS) rate of approximately 11 months (and climbing) (N=10) in combination with Cytarabine for the treatment of R/R AML as second line therapy to date, which is higher than other currently approved second line therapies for R/R AML One of our core management beliefs is that anthracyclines represent one of the most important treatments for AML and Advanced STS, and we believe Annamycin may, for the first time ever, allow a majority of these patients to benefit from this treatment.
Removed
WP1122 has completed a Phase 1 clinical study, successfully establishing a Recommended Phase 2 Dose or RP2D.
Added
We intend to advance our other drug candidates via investigator led studies – both clinically and preclinically. 47 Table of Contents Focus and Core Technologies We are focused on internally funded (“internally” and “externally” funded trials are defined in the Funding Strategy section below) development of our core technologies: 1. Annamycin: a.
Removed
The decreased cash outflows in 2023 was primarily due to an overall decrease in expenses, timing of payments, as well as license rights settled in stock . For the year ended December 31, 2023, there were $4.7 million in net proceeds from financing activities. In 2022, there were no net proceeds from financing activities.
Added
WP1066 IV: A better formulation for delivery intravenously of a molecule from the WP1066 portfolio to possibly further support future externally funded oncology clinical trials. Such a formulation will require additional preclinical work prior to a clinical trial. We have established a Recommended Phase 2 Dose for WP1122 to potentially enable future externally funded oncology and virology trials.
Removed
In a concurrent private placement, the Company also sold to the investors unregistered warrants to purchase up to an aggregate of 939,312 shares of common stock ( t aking into account the reverse stock splits we have completed).
Added
In all of our discussions, clinical data, where a CSR or its equivalent has not been published, are considered preliminary and subject to change. Clinical Trials Summary We have multiple active INDs/CTAs (Investigational New Drug authorization in the US or Clinical Trial Authorization in Europe).
Removed
Subject to certain ownership limitations, each Common Warrant has an exercise price of $9.60 per share ( t aking into account the reverse stock splits we have completed), and expires five years from the date of stockholder approval of the Common Warrants (which occurred February 14,2024).
Added
These INDs/CTAs are under development, approved, in progress, or completed and total fourteen clinical trials, internally and externally funded.
Removed
The combined purchase price of one share of common stock (or pre-funded warrant in lieu thereof) and accompanying Common Warrant was $9.60 ( t aking into account the reverse stock splits we have completed) for the institutional investor, and $10.35 ( t aking into account the reverse stock splits we have completed) for the executive officers, employees, advisors and the member of the Company's board of directors who participated in the offering.
Added
With Annamycin, we currently have active two AML clinical trials MB-106 which is a Phase 1B/2 treating AML with AnnAraC (recruitment is closed and is in subject follow-up) and MB-108 which is a Phase 2B/3 pivotal clinical trial treating R/R AML as 2 nd line therapy and is just starting.
Removed
Pursuant to the terms of the Purchase Agreement, Lincoln Park agreed to purchase from us up to $20.0 million of common stock (subject to certain limitations) from time to time during the term of the Purchase Agreement.
Added
Additionally, there are one externally funded Phase 1B/2 trial treating STS Lung Mets with Annamycin as monotherapy. This trial is closed and is in follow-up on the trial’s subjects. With WP1066, we have an externally funded phase 1B/2 in combination with radiation treating GBM at Northwestern University that is actively recruiting. Moleculin Biotech, Inc.
Removed
Pursuant to the terms of the Purchase Agreement, at the time we signed the Purchase Agreement, we issued 7,186 shares of common stock ( t aking into account the reverse stock splits we have completed) to Lincoln Park as an initial fee for its commitment to purchase shares of our common stock under the Purchase Agreement, and have agreed to issue Lincoln Park up to an additional 3,593 shares of common stock ( t aking into account the reverse stock splits we have completed) as commitment shares pro-rata when and if Lincoln Park purchases (at our discretion) the $20.0 million aggregate commitment.
Added
The pre-funded warrants are exercisable immediately and may be exercised at any time until all of the pre-funded warrants are exercised in full, subject to the beneficial ownership limitation. Each common warrant will be exercisable upon the receipt of shareholder approval, will have an exercise price of $1.07 per share, and expire five years from the initial exercise date.
Removed
During the year ended December 31, 2023, utilizing the Lincoln Park Equity Line, we issued 15,038 shares of common stock ( t aking into account the reverse stock splits we have completed) (including commitment shares), at an average price of $14.10 per share ( t aking into account the reverse stock splits we have completed), resulting in gross proceeds of $0.2 million.
Added
The Company received gross proceeds of $3.5 million. 49 Table of Contents In February 2025, the Company entered into a warrant exercise inducement offer letter with a holder of certain existing warrants to receive new warrants to purchase up to a number of shares of common stock equal to 200% of the number of warrant shares issued pursuant to the exercise of such existing warrants to purchase up to 5,828,570 shares of common stock pursuant to which the warrant holder agreed to exercise for cash their existing warrants at a reduced exercise price of $1.00 in exchange for the Company's agreement to issue the inducement warrants to purchase up to 11,657,140 shares of the Company's common stock.
Added
Each inducement warrant has an exercise price of $0.75, and was immediately exercisable as of the date of issuance and may be exercised for a period of five years therefrom. The Company received gross proceeds of $5.8 million. This brings the total gross proceeds received in February 2025 to $9.3 million.
Added
In August 2024, the Company entered into a securities purchase agreement with an institutional investor for the sale by the Company of 283,000 shares of common stock, and 2,183,368 pre-funded warrants to purchase shares of common stock, series A warrants to purchase up to 2,466,368 shares of common stock, series B warrants to purchase up to 2,466,368 shares of common stock, and placement agent warrants.
Added
The combined purchase price for the securities was $2.23 per share of common stock (or pre-funded warrant in lieu thereof) and accompanying warrants. Each pre-funded warrant is exercisable for one share of common stock at an exercise price of $0.001 per share.

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