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What changed in Traws Pharma, Inc.'s 10-K — 2023 vs 2024

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Paragraph-level year-over-year comparison of Traws Pharma, 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.

+552 added−371 removedSource: 10-K (2025-03-31) vs 10-K (2024-04-01)

Top changes in Traws Pharma, Inc.'s 2024 10-K

552 paragraphs added · 371 removed · 242 edited across 6 sections

Item 1. Business

Business — how the company describes what it does

126 edited+107 added−83 removed195 unchanged
Biggest changeThese sanctions could include the imposition by the FDA or an institutional review board (IRB), or Independent Ethics Committee (IEC) of a hold on clinical trials, refusal to approve pending marketing applications or supplements, withdrawal of approval, warning letters, untitled letters, cyber letters, product recalls, product seizures or detention, prohibition on importing or exporting, total or partial suspension of production or distribution, injunctions, fines, civil penalties, adverse publicity, disgorgement, restitution, FDA debarment, debarment from government contracting or refusal of future orders under existing contracts, exclusion from Federal healthcare programs, corporate integrity agreements, consent decrees, or criminal prosecution. 14 Table of Contents The steps required before a new drug may be marketed in the United States generally include: ● Completion of preclinical or nonclinical laboratory tests, animal studies and formulation studies in compliance with the FDA’s good laboratory practice (GLP), regulations and other applicable laws and regulations; ● Submission to the FDA of an IND to support human clinical testing; ● Approval by an IRB at each clinical site or centrally before each trial may be initiated; ● Performance of adequate and well-controlled clinical trials in accordance with federal regulations and with current good clinical practices (GCPs) to establish the safety and efficacy of the investigational drug product for each targeted indication; ● Submission of an NDA to the FDA; ● Satisfactory completion of an FDA Advisory Committee review, if applicable; ● Satisfactory completion of an FDA inspection of the manufacturing facilities at which the investigational product is produced to assess compliance with cGMPs, and to assure that the facilities, methods and controls are adequate, as well as satisfactory completion of FDA inspections of selected clinical trial sites to ensure that clinical trials were conducted in accordance with GCPs; and ● FDA review and approval of the NDA.
Biggest changeThe steps required before a new drug may be marketed in the United States generally include: ● Completion of preclinical or nonclinical laboratory tests, animal studies and formulation studies in compliance 16 Table of Contents with the FDA’s good laboratory practice (“GLP”), regulations and other applicable laws and regulations; ● Submission to the FDA of an IND to support human clinical testing; ● Approval by an IRB at each clinical site or centrally before each trial may be initiated; ● Performance of adequate and well-controlled clinical trials in accordance with federal regulations and with current good clinical practices (“GCPs”) to establish the safety and efficacy of the investigational drug product for each targeted indication; ● Submission of an NDA to the FDA; ● Satisfactory completion of an FDA Advisory Committee review, if applicable; ● Satisfactory completion of an FDA inspection of the manufacturing facilities at which the investigational product is produced to assess compliance with cGMPs, and to assure that the facilities, methods and controls are adequate, as well as satisfactory completion of FDA inspections of selected clinical trial sites to ensure that clinical trials were conducted in accordance with GCPs; and ● FDA review and approval of the NDA.
We believe, based on data from preclinical studies, that narazaciclib has the potential to overcome the limitations of the current generation of approved cyclin dependent kinase (CDK) 4/6 inhibitors. The below table depicts the half-maximal in vitro inhibitory concentration (IC 50 ) of narazaciclib palbociclib, ribociclib and abemaciclib.
We believe, based on data from preclinical studies, that narazaciclib has the potential to overcome the limitations of the current generation of approved cyclin dependent kinase (CDK) 4/6 inhibitors. The table below depicts the half-maximal in vitro inhibitory concentration (IC 50 ) of narazaciclib palbociclib, ribociclib and abemaciclib.
FDA’s regulations, provide flexibility in meeting approval standards for new therapies intended to treat persons with life-threatening and severely-debilitating illnesses, especially where no satisfactory alternative therapy exists , such that FDA may exercise scientific judgment in determining the kind and quantity of data required for approval and during development programs.
The FDA’s regulations, provide flexibility in meeting approval standards for new therapies intended to treat persons with life-threatening and severely-debilitating illnesses, especially where no satisfactory alternative therapy exists , such that FDA may exercise scientific judgment in determining the kind and quantity of data required for approval and during development programs.
If we or CMOs fail to comply with applicable cGMP requirements and conditions of product approval, the FDA may seek sanctions, including fines, civil penalties, injunctions, suspension of manufacturing operations, operating restrictions, withdrawal of FDA approval, refusal to approve applications, seizure or recall of products and criminal prosecution.
If we or our CMOs fail to comply with applicable cGMP requirements and conditions of product approval, the FDA may seek sanctions, including fines, civil penalties, injunctions, suspension of manufacturing operations, operating restrictions, withdrawal of FDA approval, refusal to approve applications, seizure or recall of products and criminal prosecution.
If we choose to build a commercial infrastructure to support marketing in the United States, such commercial infrastructure could be expected to include a targeted, oncology sales force supported by sales management, internal sales support, an internal marketing group and distribution support. To develop the appropriate commercial infrastructure internally, we would have to invest significant financial and management resources.
If we choose to build a commercial infrastructure to support marketing in the United States, such commercial infrastructure could be expected to include a targeted, sales force supported by sales management, internal sales support, an internal marketing group and distribution support. To develop the appropriate commercial infrastructure internally, we would have to invest significant financial and management resources.
For instance, in 2023, FDA took a few actions with respect to advertising and promotion, including issuing a final rule and a guidance on risk and efficacy disclosures in direct to consumer advertising, and a guidance on communication of off-label scientific information about approved products.
For instance, in 2023, the FDA took a few actions with respect to advertising and promotion, including issuing a final rule and a guidance on risk and efficacy disclosures in direct-to-consumer advertising, and a guidance on communication of off-label scientific information about approved products.
Moreover, under the provisions of the Food and Drug Administration Safety and Innovation Act, or FDASIA, enacted in 2012, a sponsor can request designation of a product candidate as a “breakthrough therapy.” A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
Moreover, under the provisions of the Food and Drug Administration Safety and Innovation Act (“FDASIA”) enacted in 2012, a sponsor can request designation of a product candidate as a “breakthrough therapy.” A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
United States Government Regulation The FDA is the main regulatory body that controls pharmaceuticals in the United States, and its regulatory authority is based in the FDC Act. Pharmaceutical products are also subject to other federal, state and local statutes.
United States Government Regulation The FDA is the main regulatory body that controls biopharmaceuticals and pharmaceuticals in the United States, and its regulatory authority is based in the FDC Act. Biopharmaceuticals and pharmaceutical products are also subject to other federal, state and local statutes.
Europe and Other International Government Regulation In addition to regulations in the United States, we will be subject to a variety of regulations in other jurisdictions governing, among other things, clinical trials and any commercial sales and distribution of our products.
International Government Regulation In addition to regulations in the United States, we will be subject to a variety of regulations in other jurisdictions governing, among other things, clinical trials and any commercial sales and distribution of our products.
Per guidance issued by FDA in 2023 with respect to rare diseases, “[t]his flexibility extends from the early stages of development to the design of adequate and well-controlled clinical investigations required to demonstrate effectiveness to support marketing approval and to establish safety data needed for the intended use.” FDA states that it “is committed to helping sponsors create successful drug development programs that address the particular challenges posed by each disease….” If a product receives the first FDA approval for the indication for which it has orphan designation, the product is entitled to seven years of market exclusivity, which means the FDA may not approve any other application for the same drug for the same indication for a period of seven years, except in limited circumstances, such as a showing of clinical superiority over the product with orphan exclusivity.
Per guidance issued by FDA in 2023 with respect to rare diseases, “[t]his flexibility extends from the early stages of development to the design of adequate and well-controlled clinical investigations required to demonstrate effectiveness to support marketing approval and to establish safety data needed for the intended use.” FDA states that it “is committed to helping sponsors create successful drug development programs that address the particular challenges posed by each disease….” 25 Table of Contents If a product receives the first FDA approval for the indication for which it has orphan designation, the product is entitled to seven years of market exclusivity, which means the FDA may not approve any other application for the same drug for the same indication for a period of seven years, except in limited circumstances, such as a showing of clinical superiority over the product with orphan exclusivity.
With the departure of the United Kingdom (UK) from the EU, trials in the UK have to be approved through the portal and a separate application will need to be made to the UK Medicines and Healthcare products Regulatory Agency.
With the departure of the United Kingdom (“UK”) from the EU, trials in the UK have to be approved through the portal and a separate application will need to be made to the UK Medicines and Healthcare products Regulatory Agency.
Generally, our activities in other countries will be subject to regulation that is similar in nature and scope as that imposed in the United States, although there can be important differences.
Generally, our activities in other countries are and will be subject to regulation that is similar in nature and scope as that imposed in the United States, although there can be important differences.
These sanctions could include the imposition by the FDA or an institutional review board, or IRB, of a hold on clinical trials, refusal to approve pending marketing applications or supplements, withdrawal of approval, warning letters, untitled letters, cyber letters, product recalls, product seizures or detention, prohibition on importing or exporting, total or partial suspension of production or distribution, injunctions, fines, civil penalties, adverse publicity, disgorgement, restitution, FDA debarment, debarment from government contracting or refusal of future orders under existing contracts, exclusion from Federal healthcare programs, corporate integrity agreements, consent decrees, or criminal prosecution.
These sanctions could include the imposition by the FDA or an IRB of a hold on clinical trials, refusal to approve pending marketing applications or supplements, withdrawal of approval, warning letters, untitled letters, cyber letters, product recalls, product seizures or detention, prohibition on importing or exporting, total or partial suspension of production or distribution, injunctions, fines, civil penalties, adverse publicity, disgorgement, restitution, FDA debarment, debarment from government contracting or refusal of future orders under existing contracts, exclusion from Federal healthcare programs, corporate integrity agreements, consent decrees, or criminal prosecution.
We may rely on licensing and co-promotion agreements with strategic partners for the commercialization of our products in the United States and other territories.
In the future, we may rely on licensing and co-promotion agreements with strategic partners for the commercialization of our products in the United States and other territories.
Real world evidence may be used to assist in clinical trial design or support an NDA for already approved products. The decision to terminate development of an investigational drug product may be made by either a health authority body, such as the FDA or IRB/independent ethics committees (IECs), or by a company for various reasons.
Real world evidence may be used to assist in clinical trial design or support an NDA for already approved products. The decision to terminate development of an investigational drug product may be made by either a health authority body, such as the FDA or IRB/independent ethics committees (“IECs”), or by a company for various reasons.
Some countries outside of the United States have a similar process that requires the submission of a clinical trial application, or CTA, much like the IND prior to the commencement of human clinical trials. In Europe, for example, a CTA must be submitted to each country’s national health authority and an IEC, much like the FDA and IRB, respectively.
Some countries outside of the United States have a similar process that requires the submission of a clinical trial application (“CTA”), much like the IND prior to the commencement of human clinical trials. In Europe, for example, a CTA must be submitted to each country’s national health authority and an IEC, much like the FDA and IRB, respectively.
The FDA may grant full or partial waivers, or deferrals, for submission of data under PREA and this requirement. The Best Pharmaceuticals for Children Act, or BPCA, provides NDA holders a six-month extension of any exclusivity — Orange Book listed patent or non-patent exclusivity — for a drug if certain conditions are met.
The FDA may grant full or partial waivers, or deferrals, for submission of data under PREA and this requirement. The Best Pharmaceuticals for Children Act (“BPCA”) provides NDA holders a six-month extension of any exclusivity — Orange Book listed patent or non-patent exclusivity — for a drug if certain conditions are met.
These requirements include permissable fees, rebates, discounts and payment reductions, required price reporting and pricing transparency, caps on price increases, requirements around price negotiation, and requirements to enter into agreements with government agencies and certain healthcare entities that may result in significant limits on the prices we may charge for our products.
These requirements include permissible fees, rebates, discounts and payment reductions, required price reporting and pricing transparency, caps on price increases, requirements around price negotiation, and requirements to enter into agreements with government agencies and certain healthcare entities that may result in significant limits on the prices we may charge for our products.
In addition, the Affordable Care Act provides that the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a per se false or fraudulent claim for purposes of the civil False Claims Act (discussed below), which imposes penalties against any person who is determined to have presented or caused to be presented a claim to a federal healthcare program that the person knows or should know is for an item or service that was not provided as claimed or is false or fraudulent.
In addition, the Affordable Care Act provides that the 29 Table of Contents government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a per se false or fraudulent claim for purposes of the civil False Claims Act (discussed below), which imposes penalties against any person who is determined to have presented or caused to be presented a claim to a federal healthcare program that the person knows or should know is for an item or service that was not provided as claimed or is false or fraudulent.
Patent and Trademark Office. For example, we have received adjustments of 1,139 days extension to the patent term for the rigosertib composition of matter patent (US 7,598,232), 1,155 days extension for the patent covering the process for making rigosertib (US 8,143,453) and 751 days extension for rigosertib formulation patent (US 8,063,109) under the provisions of 35 U.S.C. §154.
For example, we have received adjustments of 1,139 days extension to the patent term for the rigosertib composition of matter patent (US 7,598,232), 1,155 days extension for the patent covering the process for making rigosertib (US 8,143,453) and 751 days extension for rigosertib formulation patent (US 8,063,109) under the provisions of 35 U.S.C. §154.
The STA License Agreement is for a term of 15 years from the launch on a country-by-country basis in the STA Territory and contains customary provisions for termination by either party in the event of breach of the STA License Agreement by the other party (subject to a cure period), bankruptcy of the other party, or challenges to the patents by any sublicensee or assignee.
The STA License Agreement has a term of 15 years from the launch, on a country-by-country basis, in the STA Territory and contains customary provisions for termination by either party in the event of breach of the STA License Agreement by the other party (subject to a cure period), bankruptcy of the other party, or challenges to the patents by any sublicensee or assignee.
We believe our CDK inhibitor is differentiated from other agents in the market or in development due to its multi-targeted kinase inhibition profile. ​ ​ ​ ​ ​ ​ ​ Narazaciclib Palbociclib Ribociclib Abemaciclib Sponsor Onconova Pfizer Novartis Lilly ​ ​ CDK Family ​ ​ CDK4/cyclin D1 2 2 3 0.8 CDK6/cyclin D1 0.6 0.8 6.0 0.6 CDK1/cyclin A 2190 >10,000 >10,000 270 CDK2/cyclin E 69 2300 >10,000 130 CDK9/T1 48 630 390 7 ​ ​ Other Kinases ​ ​ CSF1R 0.7 >10,000 >10,000 >10,000 ARK 5/NUAK 1 5 1,400 1,540 773 FLT3 6.0 496 753 72 ​ Source: Reaction Biology 2021 In addition to CDK 4/6, narazaciclib also inhibits ARK5 (NUAK1) with a 50% inhibitory concentration (IC50) of 4.95 nM (Report EPR-123300-001 and Reddy 2014) while palbociclib, ribociclib, and abemaciclib do not.
We believe our CDK inhibitor is differentiated from other agents in the market or in development due to its multi-targeted kinase inhibition profile. ​ ​ ​ ​ ​ ​ ​ Narazaciclib Palbociclib Ribociclib Abemaciclib Sponsor Traws Pfizer Novartis Lilly ​ ​ CDK Family ​ ​ CDK4/cyclin D1 2 2 3 0.8 CDK6/cyclin D1 0.6 0.8 6.0 0.6 CDK1/cyclin A 2190 >10,000 >10,000 270 CDK2/cyclin E 69 2300 >10,000 130 CDK9/T1 48 630 390 7 ​ ​ Other Kinases ​ ​ CSF1R 0.7 >10,000 >10,000 >10,000 ARK 5/NUAK 1 5 1,400 1,540 773 FLT3 6.0 496 753 72 ​ Source: Reaction Biology 2021 Narazaciclib also inhibits ARK5 (“NUAK1”) with a 50% inhibitory concentration (IC50) of 4.95 nM (Report EPR-123300-001 and Reddy 2014) while palbociclib, ribociclib, and abemaciclib do not.
The Health Insurance Portability and Accountability Act of 1996 (HIPAA) created new federal criminal statutes that prohibit, among other things, knowingly and willfully executing a scheme to defraud any healthcare benefit program, including private third-party payors, and knowingly and willfully falsifying, concealing or covering up a material fact or 27 Table of Contents making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services.
The Health Insurance Portability and Accountability Act of 1996 (HIPAA) created new federal criminal statutes that prohibit, among other things, knowingly and willfully executing a scheme to defraud any healthcare benefit program, including private third-party payors, and knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services.
The Foreign Corrupt Practices Act The Foreign Corrupt Practices Act (FCPA), prohibits any U.S. individual or business from paying, offering, or authorizing payment or offering of anything of value, directly or indirectly, to any foreign official, political party or candidate for the purpose of influencing any act or decision of the foreign entity in order to assist the individual or business in obtaining or retaining business.
The Foreign Corrupt Practices Act The Foreign Corrupt Practices Act (the “FCPA”), prohibits any U.S. individual or business from paying, offering, or authorizing payment or offering of anything of value, directly or indirectly, to any foreign official, political party or candidate for the purpose of influencing any act or decision of the foreign entity in order to assist the individual or business in obtaining or retaining business.
In the European Union, the EMA’s Committee for Orphan Medicinal Products, or COMP, grants Orphan Drug Designation to promote the development of products that are intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions affecting not more than five in 10,000 persons in the European Union.
In the European Union, the EMA’s Committee for Orphan Medicinal Products (“COMP”) grants Orphan Drug Designation to promote the development of products that are intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions affecting not more than five in 10,000 persons in the European Union.
Pediatric Information Under the Pediatric Research Equity Act (the PREA), NDAs or certain supplements to NDAs must contain data to assess the safety and effectiveness of the drug for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the drug is safe and effective.
Pediatric Information Under the Pediatric Research Equity Act (the “PREA”), NDAs or certain supplements to NDAs must contain data to assess the safety and effectiveness of the drug for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the drug is safe and effective.
Under the terms of the Pint License Agreement, we granted Pint an exclusive, royalty-bearing license, with the right to sublicense, under certain Company patent rights and know-how to develop and commercialize any pharmaceutical product (the Pint Licensed Product) containing rigosertib in all uses of rigosertib or the Product in humans in Latin American countries (the Pint Territory, including Argentina, Belize, Bolivia, Brazil, Chile, Colombia, Costa Rica, Cuba, Dominican Republic, Ecuador, El Salvador, French Guiana, British Guiana, Suriname, Guatemala, Haiti, Honduras, Mexico, Nicaragua, Panama, Paraguay, Peru, Uruguay and Venezuela).
Under the terms of the Pint License Agreement, we granted Pint an exclusive, royalty-bearing license, with the right to sublicense, under certain Company patent rights and know-how to develop and commercialize any pharmaceutical product (the “Pint Licensed Product”) containing rigosertib in all uses of the Pint Licensed Product in humans in Latin American countries (including Argentina, Belize, Bolivia, Brazil, Chile, Colombia, Costa Rica, Cuba, Dominican Republic, Ecuador, El Salvador, French Guiana, British Guiana, Suriname, Guatemala, Haiti, Honduras, Mexico, Nicaragua, Panama, Paraguay, Peru, Uruguay and Venezuela).
Certain changes to a drug, such as the addition of a new indication to the package insert, may be associated with a three-year period of exclusivity during which the FDA cannot approve an ANDA for a generic drug or a 505(b)(2) application that includes the change, if the applicant conducted clinical trials essential to the approval of the application, which are not bioavailability or bioequivalence studies.
Certain changes to a drug, such as the addition of a new indication to the package insert, may be associated with a three-year period of exclusivity during which the FDA cannot approve an ANDA for a generic drug or a 505(b)(2) application that includes the change, if the applicant conducted clinical trials essential to the approval of the application, which are not bioavailability or 22 Table of Contents bioequivalence studies.
In partnership with HanX, a Phase 1 dose escalation study (Study HX301-I-01) for patients with advanced relapsed/refractory cancer has been initiated in China at three sites and the first patient was enrolled on September 15, 2020. In this study HX301 (narazaciclib) is dosed every day for 21 days followed by 7 days off therapy in each 28 -day cycle.
In partnership with HanX, a Phase 1 dose escalation study (Study HX301-I-01) for patients with advanced relapsed/refractory cancer has been initiated in China at three sites and the first patient was enrolled on September 15, 2020. In this study HX301 (narazaciclib) was dosed daily for 21 days followed by 7 days off therapy in each 28-day cycle.
Rigosertib Patents As of March 2024, we owned or exclusively licensed issued patents and pending patent applications covering composition-of-matter, process, formulation and various indications for method-of-use for rigosertib filed worldwide, including in the United States. The U.S. composition-of-matter patent for rigosertib, which we in-licensed pursuant to the license agreement with Temple, currently expires in 2026.
Rigosertib Patents As of March 2024, we owned or exclusively licensed issued patents and pending patent applications covering composition-of-matter, process, formulation and various indications for method-of-use for rigosertib filed worldwide, 13 Table of Contents including in the United States. The U.S. composition-of-matter patent for rigosertib, which we in-licensed pursuant to the license agreement with Temple, currently expires in 2026.
New Drug Applications In order to obtain approval to market a drug in the United States, a marketing application must be submitted to the FDA that provides data establishing the safety and effectiveness of the drug product for the proposed indication.
New Drug Applications In order to obtain approval to market a drug in the United States, a marketing application must be submitted to the FDA, which provides data establishing the safety and effectiveness of the drug product for the proposed indication.
The benefit to public health must outweigh the risk due to the limited availability of clinical data at the time of marketing authorization. The EMA has recently been conducting a pilot on ‘adaptive pathways’ — an iterative process building on existing regulatory processes involving gathering evidence through real-life use to supplement clinical trial data.
The benefit to public health must outweigh the risk due to the limited availability of clinical data at the time of marketing authorization. 27 Table of Contents The EMA has recently been conducting a pilot on ‘adaptive pathways’ — an iterative process building on existing regulatory processes involving gathering evidence through real-life use to supplement clinical trial data.
Failure to meet all the requirements of a particular applicable statutory exception or regulatory safe harbor does not 26 Table of Contents make the conduct per se illegal under the Anti-Kickback Statute. Instead, the legality of the arrangement will be evaluated on a case-by-case basis based on a cumulative review of all its facts and circumstances.
Failure to meet all the requirements of a particular applicable statutory exception or regulatory safe harbor does not make the conduct per se illegal under the Anti-Kickback Statute. Instead, the legality of the arrangement will be evaluated on a case-by-case basis based on a cumulative review of all its facts and circumstances.
This could subject a company to a range of penalties that could have a significant commercial impact, including civil and criminal fines and agreements that materially restrict the manner in which a company promotes or distributes drug products. 18 Table of Contents Post-Approval Regulations After regulatory approval of a drug is obtained, a company is required to comply with a number of post-approval requirements.
This could subject a company to a range of penalties that could have a significant commercial impact, including civil and criminal fines and agreements that materially restrict the manner in which a company promotes or distributes drug products. Post-Approval Regulations After regulatory approval of a drug is obtained, a company is required to comply with a number of post-approval requirements.
The Company has established a Compensation Committee of the Board of Directors which considers the impact of our corporate performance in 29 Table of Contents determining compensation for named executive officers, as well as each named executive officer’s individual performance, macroeconomic conditions generally, and data from peer group companies. Corporate Information We were incorporated in Delaware in December 1998.
The Company has established a Compensation Committee of the Board of Directors, which considers the impact of our corporate performance in determining compensation for named executive officers, as well as each named executive officer’s individual performance, macroeconomic conditions generally, and data from peer group companies. Corporate Information We were incorporated in Delaware in December 1998.
Moreover, under the 21st Century Cures Act, manufacturers or distributors of investigational drugs for the diagnosis, monitoring, or treatment of one or more serious diseases or conditions must have a publicly available policy concerning expanded access to investigational drugs. 16 Table of Contents Further, the manufacture of investigational drugs for the conduct of human clinical trials is subject to cGMP requirements.
Moreover, under the 21st Century Cures Act, manufacturers or distributors of investigational drugs for the diagnosis, monitoring, or treatment of one or more serious diseases or conditions must have a publicly available policy concerning expanded access to investigational drugs. Further, the manufacture of investigational drugs for the conduct of human clinical trials is subject to cGMP requirements.
Special clinical trial ethical considerations also must be taken into account if a study involves children. The clinical investigation of an 15 Table of Contents investigational drug is generally divided into three phases. Although the phases are usually conducted sequentially, they may overlap or be combined. The three phases of an investigation are as follows: ● Phase 1.
Special clinical trial ethical considerations also must be taken into account if a study involves children. The clinical investigation of an investigational drug is generally divided into three phases. Although the phases are usually conducted sequentially, they may overlap or be combined. The three phases of an investigation are as follows: ● Phase 1.
In all cases, again, the clinical trials are conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki. Expanded Access Under certain circumstances, regulators may permit unapproved drugs to be used by patients outside of clinical trials.
In all cases, again, the clinical trials are conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki. 23 Table of Contents Expanded Access Under certain circumstances, regulators may permit unapproved drugs to be used by patients outside of clinical trials.
Manufacturing Our product candidates are synthetic small molecules. Manufacturing activities must comply with FDA current good manufacturing practices (cGMP), regulations commensurate with the product candidates’ stage of development. We conduct our manufacturing activities under individual purchase orders with third-party contract manufacturers (CMOs). We have quality agreements in place with our key CMOs.
Manufacturing Our product candidates are synthetic small molecules. Manufacturing activities must comply with FDA current good manufacturing practices (“cGMP”), regulations commensurate with the product candidates’ stage of development. We conduct our manufacturing activities under individual purchase orders with third-party contract manufacturers (“CMOs”). We have quality agreements in place with our key CMOs.
Our principal executive offices are located at 12 Penns Trail, Newtown, PA 18940 and our telephone number is (267) 759-3680. Our website address is www.onconova.com. The information contained in, or that can be accessed through, our website is not part of this report.
Our principal executive offices are located at 12 Penns Trail, Newtown, PA 18940 and our telephone number is (267) 759-3680. Our website address is www.trawspharma.com. The information contained in, or that can be accessed through, our website is not part of this Annual Report.
The License Agreement is for a term of 15 years from the launch on a country-by-country basis in the Territory and contains customary provisions for termination by either party in the event of breach of the License Agreement by the other party (subject to a cure period), bankruptcy of the other party, or challenges to the patents by any sublicensee or assignee.
The License Agreement has a term of 15 years from the launch, on a country-by-country basis in the Knight Territory, and contains customary provisions for termination by either party in the event of breach of the Knight License Agreement by the other party (subject to a cure period), bankruptcy of the other party, or challenges to the patents by any sublicensee or assignee.
Additionally, before approving an NDA, the FDA will typically inspect or 17 Table of Contents conduct a remote regulatory assessment of one or more clinical sites to assure compliance with GCP. After the FDA evaluates the NDA and the manufacturing facilities, it issues either an approval letter or a complete response letter.
Additionally, before approving an NDA, the FDA will typically inspect or conduct a remote regulatory assessment of one or more clinical sites to assure compliance with GCP. After the FDA evaluates the NDA and the manufacturing facilities, it issues either an approval letter or a complete response letter.
Government Regulation As a pharmaceutical company that operates in the United States, we are subject to extensive regulation by the FDA, and other federal, state, and local regulatory agencies.
Government Regulation As a biopharmaceutical company that operates in the United States, we are subject to extensive regulation by the FDA, and other federal, state, and local regulatory agencies.
Drugs listed in the Orange Book can, in turn, be cited by potential competitors in support of approval of an abbreviated new drug application, or ANDA or 505(b)(2) application.
Drugs listed in the Orange Book can, in turn, be cited by potential competitors in support of approval of an abbreviated new drug application (“ANDA”) or 505(b)(2) application.
As an apoptotic and antiproliferative agent, narazaciclib modulates the levels and activities of regulatory proteins of the cell cycle, including cyclin D1 and inhibits retinoblastoma (Rb) protein binding. Narazaciclib is believed to inhibit cancer cell growth and suppresses deoxyribonucleic acid (DNA) synthesis by preventing CDK-mediated G1-S phase transition, followed by tumor cell death by induction of mitochondria-mediated apoptosis.
As an apoptotic and antiproliferative agent, narazaciclib modulates the levels and activities of regulatory proteins of the cell cycle, including cyclin D1 and inhibits retinoblastoma (“Rb”) protein binding. Narazaciclib is believed to inhibit cancer cell growth and suppresses deoxyribonucleic acid (“DNA”) synthesis by preventing CDK-mediated G1-S phase transition, followed by tumor cell death by induction of mitochondria-mediated apoptosis.
Preclinical investigations demonstrated overexpression of polo like kinase 1 (PLK1) in RDEB-associated SCC tumor cells. These tumors show a highly aggressive, early metastasizing course, making them the primary cause of death for these patients, with a cumulative risk of death of 70% and 78.7% by age 45 and 55, 7 Table of Contents respectively (Mellerio, 2016), (Fine, 2016).
Preclinical investigations demonstrated overexpression of polo like kinase 1 (“PLK1”) in RDEB-associated SCC tumor cells. These tumors show a highly aggressive, early metastasizing course, making them the primary cause of death for these patients, with a cumulative risk of death of 70% and 78.7% by age 45 and 55, respectively (Mellerio, 2016), (Fine, 2016).
The key feature of the 2017 collaboration was that HanX provided all funding required for the Chinese Investigational New Drug Application (IND) thereby enabling the studies necessary in order to seek IND approval by the National Medical Products Administration (the Chinese FDA).
The key feature of the 2017 collaboration was that HanX provided all funding required for the Chinese Investigational New Drug Application (“IND”), thereby enabling the studies necessary in order to seek IND approval by the National Medical Products Administration (the “NMPA”), the Chinese equivalent of the FDA.
The FDA has agreed to certain performance goals in the review of NDAs. For new molecular entities, or NMEs, the FDA has the goal of completing its review within 10 months of the application’s acceptance for filing. This, however, is just a goal, and the review time may take longer.
The FDA has agreed to certain performance goals in the review of NDAs. For new molecular entities (“NMEs”), the FDA has the goal of completing its review within 10 months of the application’s acceptance for filing. This, however, is just a goal, and the review time may take longer.
Under the terms of the License Agreement, we granted STA (i) a non-exclusive, royalty-bearing license, with the right to sublicense, under certain Company patent rights and know-how to develop and manufacture any product (the STA Licensed Product) containing rigosertib for Australia and New Zealand (the STA Territory) and in human uses (the STA Licensed Field), and (ii) an exclusive, royalty-bearing license, with the right to sublicense, under certain Company patent rights and know-how to commercialize the STA Licensed Product in the STA Territory and in the STA Licensed Field.
Under the terms of the STA License Agreement, we granted STA (i) a non-exclusive, royalty-bearing license, with the right to sublicense, under certain Company patent rights and know-how to develop and manufacture any product (the “STA Licensed Product”) containing rigosertib for Australia and New Zealand (the “STA Territory”) and in human uses (the “STA Licensed Field”), and (ii) an exclusive, royalty-bearing license, with the right to sublicense, under certain Company patent rights and know-how to commercialize the STA Licensed Product in the STA Territory and in the STA Licensed Field.
Other Special Regulatory Procedures Orphan Drug Designation The FDA may grant Orphan Drug Designation to drugs intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the United States, or, if the disease or condition affects more than 200,000 individuals in the United States, there is no reasonable expectation that the cost of developing and making the drug would be recovered from sales in the United States.
Orphan Drug Designation The FDA may grant Orphan Drug Designation to drugs intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the United States, or, if the disease or condition affects more than 200,000 individuals in the United States, there is no reasonable expectation that the cost of developing and making the drug would be recovered from sales in the United States.
These studies are designed to evaluate the safety, metabolism, pharmacokinetics and pharmacologic actions of the investigational drug in humans, the side effects associated with increasing doses, and if possible, to gain early evidence on effectiveness.
These studies are designed to evaluate the safety, metabolism, pharmacokinetics and pharmacologic actions of the investigational 17 Table of Contents drug in humans, the side effects associated with increasing doses, and if possible, to gain early evidence on effectiveness.
These efforts may result in a decrease in the amount of reimbursement we receive for our drugs from Medicare or other government programs for our drugs, once approved, and any reduction in reimbursement from Medicare and other government programs may result in a similar reduction in payments from private payors.
These efforts may result in a decrease in the amount of reimbursement we receive for our drugs from Medicare or other government programs for our drugs, once approved, and any reduction in reimbursement from Medicare and other government programs may result in a similar 28 Table of Contents reduction in payments from private payors.
Participation under the VHCA requires submission of pricing data and calculation of discounts and rebates 28 Table of Contents pursuant to complex statutory formulas, as well as the entry into government procurement contracts governed by the Federal Acquisition Regulation.
Participation under the VHCA requires submission of pricing data and calculation of discounts and rebates pursuant to complex statutory formulas, as well as the entry into government procurement contracts governed by the Federal Acquisition Regulation.
Pint International SA In March 2018, we entered into a License, Development and Commercialization Agreement (the Pint License Agreement) with Pint International SA (which, together with its affiliate Pint Pharma GmbH, are collectively referred to as Pint).
Pint International SA In March 2018, we entered into a License, Development and Commercialization Agreement (the “Pint License Agreement”) with Pint International SA (which, together with its affiliate Pint Pharma GmbH, are collectively referred to as “Pint”).
Failure to comply with applicable FDA requirements and restrictions in this area may subject a company to adverse publicity and enforcement action by the FDA, the Department of Justice (the DOJ), or the Office of the Inspector General of the Department of Health and Human Services (HHS), as well as state authorities.
Failure to comply with applicable FDA requirements and restrictions in this area may subject a company to adverse publicity and enforcement action by the FDA, the Department of Justice (the “DOJ”), or the Office of the Inspector General of the Department of Health and Human Services (“HHS”), as well as state authorities.
Upon approval of a drug, each of the patents listed in the application for the drug is then published in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations, commonly known as the Orange Book.
Upon approval of a drug, each of the patents listed in the application for the drug is then published in the FDA’s Approved Drug Products with Therapeutic Equivalence 21 Table of Contents Evaluations, commonly known as the Orange Book.
Congress passed the Coronavirus Aid, Relief, and Economic Security Act, or CARES Act, which includes various provisions regarding FDA drug shortage and manufacturing volume reporting requirements, as well as provisions regarding supply chain security, such as risk management plan requirements, and the promotion of supply chain redundancy and domestic manufacturing.
Congress passed the Coronavirus Aid, Relief, and Economic Security Act (the “CARES Act”), which includes various provisions regarding FDA drug shortage and manufacturing volume reporting requirements, as well as provisions regarding supply chain security, such as risk management plan requirements, and the promotion of supply chain redundancy and domestic manufacturing.
If manufacturing 13 Table of Contents challenges occur, they are thoroughly reviewed and, as may be required, reported to health authorities to determine whether the product can be used for clinical trials. The FDA regulates and inspects or conducts remote regulatory assessments of equipment, facilities and processes used in manufacturing pharmaceutical products prior to approval.
If manufacturing challenges occur, they are thoroughly reviewed and, as may be required, reported to health authorities to determine whether the product can be used for clinical trials. The FDA regulates and inspects or conducts remote regulatory assessments of equipment, facilities and processes used in manufacturing biopharmaceutical products prior to approval.
Competition The pharmaceutical industry is highly competitive and subject to rapid and significant technological change. While we believe that our development experience and scientific knowledge provide us with competitive advantages, we face competition from both large and small pharmaceutical and biotechnology companies.
Competition The biotechnology and pharmaceutical industries are highly competitive and subject to rapid and significant technological change. While we believe that our development experience and scientific knowledge provide us with competitive advantages, we face competition from both large and small pharmaceutical and biotechnology companies.
Regulatory approval of oncology products often requires that patients in clinical trials be followed for long periods to determine the overall survival benefit of the drug.
Regulatory approval of oncology products often requires that patients in clinical trials be 20 Table of Contents followed for long periods to determine the overall survival benefit of the drug.
In addition to patents, we rely upon unpatented trade secrets, know-how and continuing technological innovation to develop and maintain a competitive position. We seek to protect our proprietary information, in part, through confidentiality agreements with our employees, collaborators, contractors and consultants, and invention assignment agreements with our employees.
In addition to patents and other licensed intellectual property rights, we rely upon unpatented trade secrets, know-how and continuing technological innovation to develop and maintain a competitive position. We seek to protect our proprietary information, in part, through confidentiality agreements with our employees, collaborators, contractors and consultants, and invention assignment agreements with our employees.
Our success will be based in part on our ability to identify, develop and manage a portfolio of drugs that are safer and more effective than competing products in the treatment of cancer patients.
Our success will be based in part on our ability to identify, develop and manage a portfolio of drugs that are safer and more effective than competing products.
We have also established an internal quality management organization, which audits and qualifies CMOs in the United States and abroad. We believe that the manufacturing processes for the active pharmaceutical ingredient and finished drug products for our products are being developed to adequately support future development and commercial demands.
We have also established an internal quality management organization, which audits and qualifies CMOs in the United States and abroad. We believe that the manufacturing processes for the active biopharmaceutical ingredients and finished drug products for our product candidates are being developed to adequately support future development and commercial demands.
In November 2019, we entered into a Distribution, License and Supply Agreement (the Knight License Agreement) with Knight Therapeutics Inc. (Knight).
Knight Therapeutics, Inc. In November 2019, we entered into a Distribution, License and Supply Agreement (the “Knight License Agreement”) with Knight Therapeutics Inc. (“Knight”).
Medicines authorized across the EU with the results of studies from a pediatric investigation plan included in the product information are eligible for an extension of their supplementary protection certificate by six months. This is the case even when the studies’ results are negative.
Medicines authorized across the EU with the results of studies from a pediatric investigation plan included in the product information are eligible for an extension of their supplementary protection certificate by six months. This is the case even when the studies’ results are negative. For orphan medicines, the incentive is an additional two years of market exclusivity.
Cancer cells can lose Rb function through mutation and become resistant or insensitive to palbociclib. Generally, second generation agents have not been shown to be suitable for single agent therapy and must typically be used in combination with hormonal therapy in the treatment of HR+/HER2- mBC.
Cancer cells can also lose Rb function through mutation and become resistant or insensitive to palbociclib. Generally, second generation CDK 4/6 inhibitors agents have not been shown to be suitable for single agent therapy and 7 Table of Contents must typically be used in combination with hormonal therapy in the treatment of HR+/HER2- mBC.
The Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Act, permits a patent term extension of up to five years beyond the expiration of the patent.
The Drug Price Competition and Patent Term Restoration Act of 1984 (the “Hatch-Waxman Act”) permits a patent term extension of up to five years beyond the expiration of the patent.
In December 2017, we entered into a license and collaboration agreement with HanX Biopharmaceuticals, Inc. (HanX), a company focused on development of novel oncology products, for the manufacturing, clinical development, 4 Table of Contents registration and commercialization in China of narazaciclib (the HanX License Agreement).
In December 2017, we entered into a license and collaboration agreement with HanX Biopharmaceuticals, Inc. (“HanX”), a company focused on development of novel oncology products, for the manufacturing, clinical development, registration and commercialization in China of narazaciclib (the “HanX License Agreement”).
Narazaciclib is a multi-targeted kinase inhibitor targeting multiple cyclin-dependent kinases, (CDK's), AMP-activated protein kinase (AMPK) related protein kinase 5 (ARK5), and colony-stimulating factor 1 receptor (CSF1R) at low nM concentrations as well as other tyrosine kinases believed to drive tumor cell proliferation, survival and metastasis.
Narazaciclib is a multi-targeted kinase inhibitor targeting multiple cyclin-dependent kinases (“CDK's”), AMP-activated protein kinase (“AMPK”) related protein kinase 5 (“ARK5”), and colony-stimulating factor 1 receptor (“CSF1R”) at low nM concentrations as well as other tyrosine kinases believed to drive tumor cell proliferation, survival and metastasis.
We have recently filed new patent applications covering methods of treatment in target indications that are projected to extend to 2044 before any possible patent term extensions. Patent term extensions may be available, depending on various provisions in the law.
The novel formulation patent for rigosertib expires in 2037. We have recently filed new patent applications covering methods of treatment in target indications that are projected to extend to 2042-2044 before any possible patent term extensions. Patent term extensions may be available, depending on various provisions in the law.
Specialised Therapeutics Asia Pte. Ltd. In December 2019, we entered into a Distribution, License and Supply Agreement (the STA License Agreement) with Specialised Therapeutics Asia Pte. Ltd. (STA).
Specialised Therapeutics Asia Pte. Ltd. In December 2019, we entered into a Distribution, License and Supply Agreement (the “STA License Agreement”) with Specialised Therapeutics Asia Pte. Ltd. (“STA”).
The MAA is comparable to the NDA. 21 Table of Contents For other countries outside of the European Union, such as countries in Eastern Europe, Latin America or Asia, the requirements governing the conduct of clinical trials, product licensing, pricing and reimbursement vary from country to country.
For other countries outside of the European Union, such as countries in Eastern Europe, Latin America or Asia, the requirements governing the conduct of clinical trials, product licensing, pricing and reimbursement vary from country to country.
The study is ongoing. Our IND submission to the US FDA was submitted in November 2020 and the FDA Study May Proceed letter was issued in December 2020. Enrollment into the complementary US phase 1 study (Study 19-01) with narazaciclib commenced in May 2021. In Study 19-01 in the US, narazaciclib is dosed on a continuous daily schedule.
Our IND submission to the US FDA was submitted in November 2020 and the FDA Study May Proceed letter was issued in December 2020. Enrollment into the complementary US phase 1 study (Study 19-01) with narazaciclib commenced in May 2021.
Research and Development Since commencing operations, we have dedicated a significant portion of our resources to the development of our clinical-stage product candidates, particularly rigosertib and more recently narazacicib. We incurred research and development expenses of $11.4 million and $11.4 million during the years ended December 31, 2023 and 2022, respectively.
Research and Development Since commencing operations, we have dedicated a significant portion of our resources to the development of our clinical-stage product candidates, particularly rigosertib, narazaciclib and, more recently, t ivoxavir marboxil and ratutrelvir . We incurred research and development expenses of $12.8 million and $11.4 million during the years ended December 31, 2024 and 2023, respectively.
The Kd value of narazaciclib binding to CSF1R was determined to be 0.7 nM. The ability of narazaciclib to bind and inhibit CSF1R at low nanomolar values, in both in vitro and cell-based assays suggests that this compound may have an impact in cancers with a dependence on CSF1R signaling.
The ability of narazaciclib to bind and inhibit CSF1R at low nanomolar values, in both in vitro and cell-based assays suggests that this compound may have an impact in cancers with a dependence on CSF1R signaling.
The Federal Food, Drug, and Cosmetic Act (the FDC Act), and its implementing regulations set forth, among other things, requirements for the research, testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record keeping, reporting, distribution, import, export, advertising, marketing, and promotion of our products.
The Federal Food, Drug and Cosmetic Act (the “FDC Act”), and its implementing regulations, as well as various other federal and state statutes and regulations, set forth, among other things, requirements for the research, testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record keeping, reporting, distribution, import, export, advertising, marketing, and promotion of our product candidates.
Human Capital We believe that our success is largely dependent upon our ability to attract and retain qualified employees. As of December 31, 2023, we had 17 employees (16 of which were full-time employees), in addition to several consultants or independent contractors working for us.
Human Capital We believe that our success is largely dependent upon our ability to attract and retain qualified employees. As of December 31, 2024, we had 7 employees (6 of which were full-time employees), in addition to various independent contractors working for us.
In the fourth quarter of 2019, HanX filed an IND with the Chinese FDA which was approved on January 6, 2020. We and HanX also intended for these studies underlying the Chinese IND approval, to meet the US Food and Drug Administration (FDA) standards for IND approval.
In the fourth quarter of 2019, HanX filed an IND with the NMPA, which was approved on January 6, 2020. We and HanX also intended for these studies underlying the Chinese IND approval, to meet the FDA standards for IND approval. Accordingly, such studies were used by us for an IND filing with the US FDA.
Although we periodically monitor the FDA compliance of our third-party CMOs, we cannot be certain that our present or future third-party CMOs will consistently comply with cGMP and other applicable FDA regulatory requirements. Commercial Operations We do not currently have an organization for the sales, marketing and distribution of pharmaceutical products.
Although we periodically monitor the FDA compliance of our third-party CMOs, we cannot be certain that our present or future third-party CMOs will consistently comply with cGMP and other applicable FDA regulatory requirements. 15 Table of Contents Commercial Operations We do not currently have any relationships with organizations for the sale, marketing and distribution of biopharmaceutical products.
To obtain regulatory approval to commercialize a new drug under European Union regulatory systems, we must submit a marketing authorization application, or MAA.
To obtain regulatory approval to commercialize a new drug under European Union regulatory systems, we must submit a marketing authorization application (“MAA”). The MAA is comparable to the NDA.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeAny product candidate we advance into clinical trials may not have favorable results in later-stage clinical trials or receive regulatory approval. ● Clinical drug development involves a lengthy and expensive process with an uncertain outcome. ● Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following any marketing approval. ● Failure to follow the FDA’s applicable regulatory requirements may result in enforcement action. ● Changes in product candidate manufacturing or formulation may result in additional costs or delay. ● Healthcare legislation, including potentially unfavorable pricing regulations or other healthcare reform initiatives, may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates and affect the prices we may obtain. ● Our employees may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could cause significant liability for us and harm our reputation. ● We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do. ● If we breach our license agreements or fail to negotiate new agreements pertaining to our product candidates, we could lose the ability to continue the development and potential commercialization of these product candidates. ● Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of any products that we may develop. ● We may engage in future business combinations or collaborations that could disrupt our business, cause dilution to our stockholders and harm our financial condition and operating results. ● We depend on information technology and computer systems to operate our business; our business and operations would suffer in the event of any failures or interruptions of our computer system, such as a data breach or cybersecurity incident. 30 Table of Contents ● Climate change, environmental, social and governance and sustainability initiatives may result in regulatory or structural industry changes that could require significant operational changes and expenditures, reduce demand for the Company’s products and adversely affect our business, financial condition, and results of operations. ● Business disruptions could seriously harm our future revenues and financial condition and increase our costs and expenses. ● Our future success depends on our ability to retain our executive officers and to attract, retain and motivate qualified personnel. ● The recent COVID-19 pandemic, or the widespread outbreak of any other communicable disease, could adversely impact our business, including our clinical trials, drug manufacturing and nonclinical activities. ● Our recurring operating losses, negative cash flows from operations, and accumulated deficit raise substantial doubt about our ability to continue as a going concern absent obtaining adequate new financings. ● The report of our independent registered accounting firm on our audited financial statements for the fiscal year ended December 31, 2023 contains an explanatory paragraph relating to our ability to continue as a going concern. ● We need to obtain additional funding to continue as a going concern; if we are unable to meet our needs for additional funding in the future, we will be required to limit, scale back or cease operations. ● We have incurred significant losses since our inception and anticipate that we will continue to incur losses in the future. ● We currently have no source of product revenue and may never become profitable. ● We need to obtain substantial additional funding to further develop our products in future clinical trials and through regulatory processes; if we are unable to meet our needs for additional funding in the future, we will be required to limit, scale back or cease operations. ● Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates. ● Adverse developments affecting financial institutions, companies in the financial services industry or the financial services industry generally, such as actual events or concerns involving liquidity, defaults, or non-performance could adversely affect our operations and liquidity. ● Changes in United States and China relations, as well as relations with other countries, and/or regulations may adversely impact our business, our operating results, our ability to raise capital and the market price of our shares. ● We rely on third parties to conduct our preclinical and clinical trials.
Biggest changeAny product candidate we advance into clinical trials may not have favorable results in later-stage clinical trials or receive regulatory approval. ● Clinical drug development involves a lengthy and expensive process with an uncertain outcome. ● Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following any marketing approval. ● Failure to follow the FDA’s applicable regulatory requirements may result in enforcement action. ● Changes in product candidate manufacturing or formulation may result in additional costs or delay. ● Healthcare legislation, including potentially unfavorable pricing regulations or other healthcare reform initiatives, may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates and affect the prices we may obtain. ● Our employees may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could cause significant liability for us and harm our reputation. ● We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do. ● If we breach our license agreements or fail to negotiate new agreements pertaining to our product candidates, we could lose the ability to continue the development and potential commercialization of these product candidates. ● Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of any products that we may develop. ● There is no guarantee that the Merger will increase stockholder value, and stockholders may not realize a benefit from the Merger commensurate with the ownership dilution they will experience in connection with the Merger, including the issuance of our common stock upon conversion of the shares of Series C Preferred issued in the Merger and the concurrent financing. ● We expect to incur substantial expenses related to the integration of Trawsfynydd. ● We may engage in future business combinations or collaborations that could disrupt our business, cause dilution to our stockholders and harm our financial condition and operating results. ● We depend on information technology and computer systems to operate our business; our business and operations would suffer in the event of any failures or interruptions of our computer system, such as a data breach or cybersecurity incident. ● Climate change, environmental, social and governance and sustainability initiatives may result in regulatory or structural industry changes that could require significant operational changes and expenditures, reduce demand for the Company’s products and adversely affect our business, financial condition, and results of operations. 33 Table of Contents ● Business disruptions could seriously harm our future revenues and financial condition and increase our costs and expenses. ● Our future success depends on our ability to retain our executive officers and to attract, retain and motivate qualified personnel. ● Our business, financial condition and results of operations could be materially adversely affected by any negative impact on the global economy and capital markets resulting from international conflicts, international trade disputes and geopolitical tensions. ● Changes in United States and China relations, as well as relations with other countries, and/or regulations may adversely impact our business, our operating results, our ability to raise capital and the market price of our shares. ● We currently conduct clinical trials, and may in the future choose to conduct additional clinical trials, of our product candidates in sites outside the US, and the FDA may not accept data from trials conducted in foreign locations. ● Disruptions at the FDA and foreign regulatory authorities caused by funding shortages, staffing limitations or global health concerns could hinder their ability to hire, retain or deploy key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved or commercialized in a timely manner or at all, which could negatively impact our business. ● We rely on third parties to conduct our preclinical and clinical trials.
ITEM 1A. RISK FACTORS You should carefully consider the following risk factors together with the other information contained in this Annual Report, including our financial statements, the related notes and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” appearing in this report.
ITEM 1A. RISK FACTORS You should carefully consider the following risk factors together with the other information contained in this Annual Report, including our financial statements, the related notes and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” appearing in this Annual Report.
Any provision of our amended and restated certificate of incorporation or amended and restated bylaws or Delaware law that has the effect of delaying or deterring a change in control could limit the opportunity for our stockholders to receive a premium for their shares of our common stock, and could also affect the price that some investors are willing to pay for our common stock.
Any provision of our Certificate of Incorporation or Amended and Restated Bylaws or Delaware law that has the effect of delaying or deterring a change in control could limit the opportunity for our stockholders to receive a premium for their shares of our common stock, and could also affect the price that some investors are willing to pay for our common stock.
Clinical trials may be delayed, suspended or prematurely terminated and development programs may not be successful for a variety of reasons, including: ● delay or failure in reaching identifying, contracting with, and retaining contract research organizations, or CROs, and clinical trial sites; ● delay or failure in recruiting and enrolling suitable subjects to participate in a trial and/or retaining subjects; ● failure to follow the study procedures or applicable regulatory requirements; ● change in standards of care which may necessitate the modification of our clinical trials or the conduct of new trials; ● negative or ambiguous study results; ● manufacturing or product quality issues; ● the need to conduct additional development work, including clinical trials; ● unanticipated clinical trial costs or insufficient funding, including paying substantial application user fees; ● changes in governmental laws, regulations, policies, or administrative actions; and ● regulatory authority disagreements regarding the design or implementation of our clinical trials.
Clinical trials may be delayed, suspended or prematurely terminated and development programs may not be successful for a variety of reasons, including: ● delay or failure in reaching identifying, contracting with, and retaining contract research organizations (“CROs”) and clinical trial sites; ● delay or failure in recruiting and enrolling suitable subjects to participate in a trial and/or retaining subjects; ● failure to follow the study procedures or applicable regulatory requirements; ● change in standards of care, which may necessitate the modification of our clinical trials or the conduct of new trials; ● negative or ambiguous study results; ● manufacturing or product quality issues; ● the need to conduct additional development work, including clinical trials; ● unanticipated clinical trial costs or insufficient funding, including paying substantial application user fees; ● changes in governmental laws, regulations, policies, or administrative actions; and ● regulatory authority disagreements regarding the design or implementation of our clinical trials.
Even if we are able to complete the development and regulatory process for any product candidates, we anticipate incurring significant costs associated with commercializing these products. Even if we are able to generate revenues from the sale of our products, we may not become profitable and may need to obtain additional funding to continue operations.
Even if we are able to complete the development and regulatory process for any product candidates, we anticipate incurring significant costs associated with commercializing these products. Additionally, even if we are able to generate revenues from the sale of our products, we may not become profitable and may need to obtain additional funding to continue operations.
If we or our third-party contractors are not able to follow the FDA’s or comparable foreign regulatory authorities’ regulatory requirements, we or they may face enforcement actions that may materially harm our business, including, but not limited to: 37 Table of Contents ● warning letters, untitled letters, cyber letters or otherwise unacceptable inspectional findings; ● injunctions, penalties, fines, restitution, consent decrees, corporate integrity agreements, suspension or debarment; ● suspension or termination of any ongoing clinical studies, imposition of a clinical hold, or regulatory authority refusal to approve pending marketing applications; ● modification of promotional materials or labeling, provision of corrective information, imposition of post-market requirements including the need for additional testing; ● restrictions on operations, product seizure or detention, refusal to permit the import or export of products, or product recalls; or ● adverse publicity.
If we or our third-party contractors are not able to follow the FDA’s or comparable foreign regulatory authorities’ regulatory requirements, we or they may face enforcement actions that may materially harm our business, including, but not limited to: ● warning letters, untitled letters, cyber letters or otherwise unacceptable inspectional findings; ● injunctions, penalties, fines, restitution, consent decrees, corporate integrity agreements, suspension or debarment; ● suspension or termination of any ongoing clinical studies, imposition of a clinical hold, or regulatory authority refusal to approve pending marketing applications; ● modification of promotional materials or labeling, provision of corrective information, imposition of post-market requirements including the need for additional testing; ● restrictions on operations, product seizure or detention, refusal to permit the import or export of products, or product recalls; or ● adverse publicity.
Even if patents are granted that cover commercially valuable compounds, we may decide to allow such patents to lapse, or if in-licensed, return the patents to the licensor. The effects of doing so are uncertain.
Even if patents are granted that cover commercially valuable molecules or compounds, we may decide to allow such patents to lapse, or if in-licensed, return the patents to the licensor. The effects of doing so are uncertain.
If adequate funds are unavailable, we may be required, among other things, to: 33 Table of Contents ● delay, reduce the scope of or eliminate one or more of our research or development programs; ● license rights to technologies, product candidates or products at an earlier stage or for indications or territories than otherwise would be desirable, or on terms that are less favorable to us than might otherwise be available; ● obtain funds through arrangements that may require us to relinquish rights to product candidates or products that we would otherwise seek to develop or commercialize by ourselves; or ● further reduce or cease operations.
If adequate funds are unavailable, we may be required, among other things, to: ● delay, reduce the scope of or eliminate one or more of our research or development programs; ● license rights to technologies, product candidates or products at an earlier stage or for indications or territories than otherwise would be desirable, or on terms that are less favorable to us than might otherwise be available; ● obtain funds through arrangements that may require us to relinquish rights to product candidates or products that we would otherwise seek to develop or commercialize by ourselves; or ● further reduce or cease operations.
Climate change, environmental, social and governance and sustainability initiatives may result in regulatory or structural industry changes that could require significant operational changes and expenditures, reduce demand for the Company’s products and adversely affect our business, financial condition, and results of operations. Climate change, environmental, social and governance (ESG) and sustainability are a growing global movement.
Climate change, environmental, social and governance and sustainability initiatives may result in regulatory or structural industry changes that could require significant operational changes and expenditures, reduce demand for the Company’s products and adversely affect our business, financial condition, and results of operations. Climate change, environmental, social and governance (“ESG”) and sustainability are a growing global movement.
Provisions in our Tenth Amended and Restated Certificate of Incorporation, as amended, or Certificate of Incorporation, and Amended and Restated Bylaws, as well as provisions of Delaware law, could make it more difficult for a third party to acquire us or increase the cost of acquiring us, even if doing so would benefit our stockholders, or remove our current management.
Provisions in our Tenth Amended and Restated Certificate of Incorporation, as amended (“Certificate of Incorporation”) and Amended and Restated Bylaws, as well as provisions of Delaware law, could make it more difficult for a third party to acquire us or increase the cost of acquiring us, even if doing so would benefit our stockholders, or remove our current management.
We have adopted a code of conduct for our directors, officers and employees, but it is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to be in compliance with such laws or regulations.
We have adopted a code of 44 Table of Contents conduct for our directors, officers and employees, but it is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to be in compliance with such laws or regulations.
If any new legislation, executive orders, tariffs, laws and/or regulations are implemented, if existing trade agreements are renegotiated or if the U.S. or Chinese governments take retaliatory actions due to the recent U.S.-China tension, such changes could have an adverse effect on our business, financial condition and results of operations, our ability to raise capital and the market price of our shares.
If any new legislation, executive orders, tariffs, laws and/or regulations are implemented, if existing trade agreements are renegotiated or if the U.S. or Chinese governments take retaliatory actions due to the recent U.S.-China 39 Table of Contents tension, such changes could have an adverse effect on our business, financial condition and results of operations, our ability to raise capital and the market price of our shares.
If CROs or sites do not successfully carry out their contractual duties or obligations or meet expected deadlines or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, our clinical trials may be extended, delayed or terminated and we may not be able to obtain regulatory approval for or successfully commercialize our product candidates.
If CROs or sites do not successfully carry out their 49 Table of Contents contractual duties or obligations or meet expected deadlines or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, our clinical trials may be extended, delayed or terminated and we may not be able to obtain regulatory approval for or successfully commercialize our product candidates.
Our future capital requirements will depend on many factors, including: ● timing and success of our clinical trials; ● continued progress of and increased spending related to our research and development activities; ● conditions in the capital markets and the biopharmaceutical industry, particularly with respect to raising capital or entering into strategic arrangements; ● progress with preclinical experiments and clinical trials, including regulatory approvals necessary for advancement and continuation of our development programs; ● changes in regulatory requirements and guidance of the FDA and other regulatory authorities, which may require additional cl inical trials to evaluate safety and/or efficacy, and thus have significant impacts on our timelines, cost projections, and financial requirements; ● ongoing general and administrative expenses related to our reporting obligations under the Exchange Act; ● cost, timing, and results of regulatory reviews and approvals; ● costs of any legal proceedings, claims, lawsuits and investigations; ● success, timing, and financial consequences of any existing or future collaborative, licensing and other arrangements t hat we may establish, including potential granting of licenses to one or more of our programs in various territories, or otherwise monetizing one or more of our programs; ● cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; ● costs of commercializing any of our product candidates; ● technological and market developments; ● compliance with Nasdaq's continued listing requirements; ● cost of manufacturing development; and ● timing and volume of sales of products for which we obtain marketing approval.
Our future capital requirements will depend on many factors, including: ● timing and success of our clinical trials; ● continued progress of, and increased spending related to, our research and development activities; ● conditions in the capital markets and the biopharmaceutical industry, particularly with respect to raising capital or entering into strategic arrangements; 35 Table of Contents ● progress with preclinical experiments and clinical trials, including regulatory approvals necessary for advancement and continuation of our development programs; ● changes in regulatory requirements and guidance of the FDA and other regulatory authorities, which may require additional clinical trials to evaluate safety and/or efficacy, and thus have significant impacts on our timelines, cost projections, and financial requirements; ● ongoing general and administrative expenses related to our reporting obligations under the Exchange Act; ● cost, timing, and results of regulatory reviews and approvals; ● costs of any legal proceedings, claims, lawsuits and investigations; ● success, timing, and financial consequences of any existing or future collaborative, licensing and other arrangements that we may establish, including potential granting of licenses to one or more of our programs in various territories, or otherwise monetizing one or more of our programs; ● cost of filing, prosecuting, defending and enforcing any patent ● claims and other intellectual property rights; ● costs of commercializing any of our product candidates; ● technological and market developments; ● compliance with Nasdaq's continued listing requirements; ● cost of manufacturing development; and ● timing and volume of sales of products for which we obtain marketing approval.
The report of our independent registered accounting firm on our audited financial statements for the fiscal year ended December 31, 2023 contains an explanatory paragraph relating to our ability to continue as a going concern.
The report of our independent registered accounting firm on our audited financial statements for the fiscal year ended December 31, 2024 contains an explanatory paragraph relating to our ability to continue as a going concern.
Any uncured, material breach under the license agreement could result in our loss of exclusive rights and may lead to a complete termination of our product development and any commercialization efforts for the applicable product candidates. Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of any products that we may develop.
Any uncured, material breach under the license agreement could result in our loss of exclusive rights and may lead to a complete termination of our product development and any commercialization efforts for the applicable product candidates. 45 Table of Contents Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of any products that we may develop.
There can be no assurance that the FDA, an IRB, or a comparable foreign regulatory authority will permit our clinical trials to commence and will not put clinical trials of any of our product candidates on clinical hold in the future. Study results may also cause us to 36 Table of Contents discontinue trials.
There can be no assurance that the FDA, an IRB, or a comparable foreign regulatory authority will permit our clinical trials to commence and will not put clinical trials of any of our product candidates on clinical hold in the future. Study results may also cause us to discontinue trials.
There can be no assurance that our efforts, or the efforts of our partners and vendors, will prevent service interruptions, or identify breaches in our systems, that could adversely affect our business and operations and/or result in the loss of critical or sensitive information, which could result in financial, legal, business or reputational harm to us.
There can be no assurance that our efforts, or the efforts of our partners and vendors, will prevent service interruptions, or identify breaches in our systems, that could adversely affect our business and operations and/or result in the loss of critical or sensitive information, which could 46 Table of Contents result in financial, legal, business or reputational harm to us.
Failure to comply with these regulations may require us to repeat preclinical and clinical trials, which would delay the regulatory approval process. We may also face liability 42 Table of Contents and/or regulatory enforcement action should any of the third parties that we rely upon fail to comply with legal and/or regulatory requirements.
Failure to comply with these regulations may require us to repeat preclinical and clinical trials, which would delay the regulatory approval process. We may also face liability and/or regulatory enforcement action should any of the third parties that we rely upon fail to comply with legal and/or regulatory requirements.
In addition, regulatory authorities enforce cGMP through periodic inspections and remote regulatory assessments of active pharmaceutical ingredient (API) and drug product manufacturing sites, quality control contract laboratories and distribution centers.
In addition, regulatory authorities enforce cGMP through periodic inspections and remote regulatory assessments of active pharmaceutical ingredient (“API”) and drug product manufacturing sites, quality control contract laboratories and distribution centers.
While we have not experienced any such material system failure, accident or security breach to date, if such an event were to occur and cause interruptions in our operations, it could result in a material disruption of our drug development 40 Table of Contents programs.
While we have not experienced any such material system failure, accident or security breach to date, if such an event were to occur and cause interruptions in our operations, it could result in a material disruption of our drug development programs.
In such an event, our trials could be suspended or terminated, and the FDA or comparable foreign regulatory authorities could order us to cease further development or deny approval of our product candidates for any or all targeted indications.
In such an event, our trials could be suspended or terminated, and the FDA or comparable foreign regulatory authorities could order us to cease further development or deny approval of our product candidates for any or all targeted 41 Table of Contents indications.
To the extent that we raise additional capital through the sale of equity or 34 Table of Contents convertible debt securities, the ownership interests of existing stockholders will be diluted, and the terms may include liquidation or other preferences that adversely affect the rights of existing stockholders.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interests of existing stockholders will be diluted, and the terms may include liquidation or other preferences that adversely affect the rights of existing stockholders.
We may become party to, or threatened with, future adversarial proceedings or litigation regarding intellectual property rights with respect to our products and technology, including interference or derivation proceedings 45 Table of Contents before the USPTO. Third parties may assert infringement claims against us based on existing patents or patents that may be granted in the future.
We may become party to, or threatened with, future adversarial proceedings or litigation regarding intellectual property rights with respect to our products and technology, including interference or derivation proceedings before the USPTO. Third parties may assert infringement claims against us based on existing patents or patents that may be granted in the future.
Although we may take measures to mitigate the effects of inflation, if these measures are not effective, our business, financial condition, results of operations and liquidity could 35 Table of Contents be materially adversely affected.
Although we may take measures to mitigate the effects of inflation, if these measures are not effective, our business, financial condition, results of operations and liquidity could be materially adversely affected.
The 44 Table of Contents rights already granted under any of our currently issued patents and those that may be granted under future issued patents may not provide us with the proprietary protection or competitive advantages we are seeking.
The rights already granted under any of our currently issued patents and those that may be granted under future issued patents may not provide us with the proprietary protection or competitive advantages we are seeking.
The auditor’s opinion on our audited financial statements for the year ended December 31, 2023 includes an explanatory paragraph stating that we have incurred recurring losses from operations that raise substantial doubt about our ability to continue as a going concern for the next twelve months from the date of the financial statements included in this Annual Report on Form 10-K.
The auditor’s opinion on our audited financial statements for the year ended December 31, 2024 includes an explanatory paragraph stating that we have incurred recurring losses from operations that raise substantial doubt about our ability to continue as a going concern for the next twelve months from the date of the financial statements included in this Annual Report.
We, our clinical trial sites, and our CROs are required to comply with federal regulations and current GCPs, which are international standards meant to protect the rights and health of patients that are enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area and comparable foreign regulatory authorities for all of our products in clinical development.
We, our clinical trial sites, and our CROs are required to comply with federal regulations and current GCPs, which are international standards meant to protect the rights and health of patients that are enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area, the Australian Human Research Ethics Committee and comparable foreign regulatory authorities for all of our products in clinical development.
If we or our CMO fail to comply with applicable cGMPs, the manufacturing data generated and subsequent API lots and drug product batches in 43 Table of Contents our supply chain may be deemed unreliable. Clinical trials using the product candidate may also be deemed to be unreliable.
If we or our CMO fail to comply with applicable cGMPs, the manufacturing data generated and subsequent API lots and drug product batches in our supply chain may be deemed unreliable. Clinical trials using the product candidate may also be deemed to be unreliable.
Where we have the right to do so under our license agreements, we seek to protect our proprietary position by filing patent applications in the United States and abroad related to our novel technologies and products that are important to our business.
Where we 51 Table of Contents have the right to do so under our license agreements, we seek to protect our proprietary position by filing patent applications in the United States and abroad related to our novel technologies and products that are important to our business.
We also rely on third parties to assist in conducting our preclinical studies in accordance with Good Laboratory Practices, or GLP, and the Animal Welfare Act requirements.
We also rely on third parties to assist in conducting our preclinical studies in accordance with Good Laboratory Practices (“GLP”) and the Animal Welfare Act requirements.
These include provisions that will: ● permit our board of directors to issue up to 5,000,000 shares of preferred stock, with any rights, preferences and privileges as they may designate (as of February 29, 2024, we had no shares of preferred stock issued and outstanding); ● provide that all vacancies on our board of directors, including as a result of newly created directorships, may, except as otherwise required by law, be filled by the affirmative vote of a majority of directors then in office, even if less than a quorum; ● require that any action to be taken by our stockholders must be effected at a duly called annual or special meeting of stockholders and not be taken by written consent; ● provide that stockholders seeking to present proposals before a meeting of stockholders or to nominate candidates for election as directors at a meeting of stockholders must provide advance notice in writing, and also specify requirements as to the form and content of a stockholder’s notice; ● not provide for cumulative voting rights, thereby allowing the holders of a majority of the shares of common stock entitled to vote in any election of directors to elect all of the directors standing for election; and ● provide that special meetings of our stockholders may be called only by the board of directors or by such person 47 Table of Contents or persons requested by a majority of the board of directors to call such meetings.
These include provisions that will: ​ ● permit our board of directors to issue up to 5,000,000 shares of preferred stock, with any rights, preferences and privileges as they may designate (as of March 26, 2025, we had 7,397.893 shares of Series C Preferred stock issued and outstanding); ● provide that all vacancies on our board of directors, including as a result of newly created directorships, may, except as otherwise required by law, be filled by the affirmative vote of a majority of directors then in office, even if less than a quorum; ● require that any action to be taken by our stockholders must be effected at a duly called annual or special meeting of stockholders and not be taken by written consent; ● provide that stockholders seeking to present proposals before a meeting of stockholders or to nominate candidates for election as directors at a meeting of stockholders must provide advance notice in writing, and also specify requirements as to the form and content of a stockholder’s notice; ● not provide for cumulative voting rights, thereby allowing the holders of a majority of the shares of common 55 Table of Contents stock entitled to vote in any election of directors to elect all of the directors standing for election; and ● provide that special meetings of our stockholders may be called only by the board of directors or by such person or persons requested by a majority of the board of directors to call such meetings.
The foregoing may require that we consult with relevant review and ethics committees, IRBs, and the FDA. The foregoing may also impact the integrity of our study data, which may not become evident until later in our development programs.
The foregoing may require that we consult with relevant review and ethics committees, IRBs, and the FDA or foreign regulatory authorities. The foregoing may also impact the integrity of our study data, which may not become evident until later in our development programs.
If later-stage clinical trials do not produce favorable results, our ability to achieve regulatory approval for any of our product candidates may be adversely impacted. Clinical drug development involves a lengthy and expensive process with an uncertain outcome.
If our clinical trials do not produce favorable results for our product candidates, our ability to achieve regulatory approval for any of our product candidates may be adversely impacted. 40 Table of Contents Clinical drug development involves a lengthy and expensive process with an uncertain outcome.
Although we have employment agreements with the persons named above, these agreements are at-will and do not prevent such persons from terminating their employment with us at any time. We do not maintain "key person" insurance for any of our executives or other employees.
Although we have employment agreements with our executive officers, these agreements are at-will and do not prevent such persons from terminating their employment with us at any time. We do not maintain "key person" insurance for any of our executives or other employees.
Our ability to generate revenue from product sales and achieve profitability will depend upon our ability to successfully commercialize products, including any of our current product candidates, or other product candidates that we may in-license or acquire in the future.
To date, we have not generated any revenues from commercial product sales. Our ability to generate revenue from product sales and achieve profitability will depend upon our ability to successfully commercialize products, including any of our current product candidates, or other product candidates that we may in-license or acquire in the future.
We are dependent on third-party manufacturers for the manufacture of our product candidates for clinical trials as well as on third parties for our supply chain, and if we experience problems with any third parties, the manufacturing of our product candidates or products could be delayed. ● We could be required to incur significant expenses to perfect our intellectual property rights, and our intellectual property rights may be inadequate to protect our competitive position.
We are dependent on third-party manufacturers for the manufacture of our product candidates for clinical trials as well as on third parties for our supply chain, and if we experience problems with any third parties, the manufacturing of our product candidates or products could be delayed. ● We have entered into certain related party transactions and may continue to rely on related parties for certain development and support activities. ● We could be required to incur significant expenses to perfect our intellectual property rights, and our intellectual property rights may be inadequate to protect our competitive position.
The loss of the services of any of these persons could impede the achievement of our research, development and commercialization objectives. The recent COVID-19 pandemic, or the widespread outbreak of any other communicable disease, could adversely impact our business, including our clinical trials, drug manufacturing and nonclinical activities.
The loss of the services of any of these persons could impede the achievement of our research, development and commercialization objectives. 47 Table of Contents The widespread outbreak of a communicable disease, such as the recent COVID-19 pandemic, could adversely impact our business, including our clinical trials, drug manufacturing and nonclinical activities.
We or our contractors may also face enforcement actions. We have not yet qualified alternate suppliers in the event the current CMOs we utilize are unable to scale production, or if we otherwise experience any problems with them.
We or our contractors may also face enforcement actions. We have not yet qualified alternate suppliers in the event the current CMO we utilize is unable to 50 Table of Contents scale production, or if we otherwise experience any problems with them.
States, in the US, have also enacted laws requiring pharmaceutical companies to, among other things, establish marketing compliance programs, file periodic reports with the state, make periodic public disclosures on sales, marketing, pricing, clinical trials and other activities, cap or regulate price increases, negotiate or pay increased supplemental rebates and/or register their sales representatives, as well as to prohibit pharmacies and other healthcare entities from providing specified physician prescribing data to pharmaceutical companies for use in sales and marketing, and to prohibit other specified sales and marketing practices. 38 Table of Contents Some countries require approval of the sale price of a drug before it can be marketed.
Certain states, in the US, have also enacted laws requiring pharmaceutical companies to, among other things, establish marketing compliance programs, file periodic reports with the state, make periodic public disclosures on sales, marketing, pricing, clinical trials and other activities, cap or regulate price increases, negotiate or pay increased supplemental rebates and/or register their sales representatives, as well as to prohibit pharmacies and other healthcare entities from providing specified physician prescribing data to pharmaceutical companies for use in sales and marketing, and to prohibit other specified sales and marketing practices.
This can be expensive and time consuming. Many of our current and potential competitors have the ability to dedicate substantially greater resources to defend their intellectual property rights than we can. Accordingly, despite our efforts, we may not be able to prevent third parties from infringing upon or misappropriating our intellectual property.
Many of our current and potential competitors have the ability to dedicate substantially greater resources to defend their intellectual property rights than we can. Accordingly, despite our efforts, we may not be able to prevent third parties from infringing upon or misappropriating our intellectual property. Litigation could result in substantial costs and diversion of management resources.
Competitors may infringe our patents or misappropriate or otherwise violate our intellectual property rights. To counter infringement or unauthorized use, litigation may be necessary in the future to enforce or defend our intellectual property rights, to protect our trade secrets or to determine the validity and scope of our own intellectual property rights or the proprietary rights of others.
To counter infringement or unauthorized use, litigation may be necessary in the future to enforce or defend our intellectual property rights, to protect our trade secrets or to determine the validity and scope of our own intellectual property rights or the proprietary rights of others. This can be expensive and time consuming.
In many countries, the pricing review period begins after marketing or product licensing approval is granted. In some foreign markets, prescription pharmaceutical pricing remains subject to continuing governmental control even after initial approval is granted.
Some countries require approval of the sale price of a drug before it can be marketed. In many countries, the pricing review period begins after marketing or product licensing approval is granted. In some foreign markets, prescription pharmaceutical pricing remains subject to continuing governmental control even after initial approval is granted.
We do not have the funding resources necessary to carry out all of our proposed operating activities. We will need to obtain additional financing in the future in order to fully fund narazaciclib, rigosertib or any other product candidates through the regulatory approval process. Accordingly, we may delay or pause our planned clinical trials, until we secure adequate additional funding.
We will need to obtain additional financing in the future in order to fully fund tivoxavir marboxil, ratutrelvir, narazaciclib, rigosertib or any other product candidates through the regulatory approval process. Accordingly, we may be required to delay or pause our planned clinical trials until we secure adequate additional funding.
Clinical and non-clinical development is expensive, time-consuming, and uncertain as to the outcome. The focus of our development efforts is currently on narazaciclib and oral rigosertib.
Clinical and non-clinical development is expensive, time-consuming, and uncertain as to the outcome. The focus of our development efforts is currently on tivoxavir marboxil and ratutrelvir, while we consider strategic options for narazaciclib and rigosertib.
There is no guarantee that a similar event will not occur again and that our liquidity will not be adversely affected. Changes in United States and China relations, as well as relations with other countries, and/or regulations may adversely impact our business, our operating results, our ability to raise capital and the market price of our shares.
Changes in United States and China relations, as well as relations with other countries, and/or regulations may adversely impact our business, our operating results, our ability to raise capital and the market price of our shares.
Delisting could also impair our ability to raise capital. ​ Our share price and the liquidity of our stock may be volatile and result in substantial losses to our stockholders. The trading price of our common stock is highly volatile and could be subject to wide fluctuations in response to various factors, some of which are beyond our control.
The trading price of our common stock is highly volatile and could be subject to wide fluctuations in response to various factors, some of which are beyond our control.
To alleviate the conditions that raise substantial doubt about our ability to continue as a going concern, we plan to explore various dilutive and non-dilutive opportunities, including equity financings, strategic alliances, business development and/or combinations, and other transactions. The future success of the Company is dependent upon our ability to obtain additional funding.
We will require substantial additional financing to fund our ongoing clinical trials and operations, and to continue to execute our strategy. To alleviate the conditions that raise substantial doubt about our ability to continue as a going concern, we plan to explore various dilutive and non-dilutive opportunities, including equity financings, strategic alliances, business development and/or combinations, and other transactions.
We do not have any products approved by regulatory authorities for marketing and have not generated any revenue from product sales to date, and we continue to incur significant research, development and other expenses related to our ongoing operations. As a result, we are not profitable and have incurred losses in every reporting period since our inception in 1998.
We do not have any products approved by regulatory authorities for marketing and have not generated any revenue from product sales to date, and we continue to 36 Table of Contents incur significant research, development and other expenses related to our ongoing operations.
These losses may increase as we continue the research and development of, and seek regulatory approvals for, our product candidates, and potentially begin to commercialize any products that may achieve regulatory approval. We may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect our business.
We expect to continue to incur significant expenses and operating losses for the foreseeable future. These losses may increase as we continue the research and development of, and seek regulatory approvals for, our product candidates, and potentially begin to commercialize any products that may achieve regulatory approval.
If we are unable to comply with the continued listing requirements of the Nasdaq Capital Market, our Common Stock could be delisted, which could affect our Common Stock's market price and liquidity and reduce our ability to raise capital. 31 Table of Contents ● Our share price and the liquidity of our stock may be volatile and result in substantial losses to our stockholders. ● We may be subject to securities litigation, which is expensive and could divert management attention. ● Some provisions of our charter documents and Delaware law may have anti-takeover effects that could discourage an acquisition of us by others, even if an acquisition would be beneficial to our stockholders and may prevent attempts by our stockholders to replace or remove our current management.
If we are unable to protect our intellectual property rights, our competitive position could be harmed. ● We may become involved in lawsuits to protect or enforce our intellectual property, which could be expensive, time consuming and unsuccessful. ● Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business. ● We may be subject to claims that our employees have wrongfully used or disclosed alleged trade secrets of their former employers. ● If we are unable to maintain compliance with the continued listing requirements of the Nasdaq Capital Market, our common stock could be delisted, which could affect our common stock's market price and liquidity and reduce our ability to raise capital. ● Our share price and the liquidity of our stock may be volatile and result in substantial losses to our stockholders. ● We may be subject to securities litigation, which is expensive and could divert management attention. ● Some provisions of our charter documents and Delaware law may have anti-takeover effects that could discourage an acquisition of us by others, even if an acquisition would be beneficial to our stockholders and may prevent attempts by our stockholders to replace or remove our current management.
Compared to us, many of our competitors may have significantly greater financial, technical and human resources. 39 Table of Contents If we breach our license agreements or fail to negotiate new agreements pertaining to our product candidates, we could lose the ability to continue the development and potential commercialization of these product candidates.
If we breach our license agreements or fail to negotiate new agreements pertaining to our product candidates, we could lose the ability to continue the development and potential commercialization of these product candidates.
Risks Related to Our Financial Position and Capital Needs Our recurring operating losses, negative cash flows from operations, and accumulated deficit raise substantial doubt about our ability to continue as a going concern absent obtaining adequate new financings.
Risks Related to Our Financial Position and Capital Needs Our recurring operating losses, negative cash flows from operations, and accumulated deficit raise substantial doubt about our ability to continue as a going concern absent obtaining adequate new financings. 34 Table of Contents Management has concluded that substantial doubt exists about our ability to continue as a going concern for the next twelve months from the date of the financial statements included in this Annual Report.
There can be no assurance that our focus on narazaciclib and oral rigosertib will be successful, and that we will be able to successfully develop a product candidate or, even if we do, that we will be able to successfully commercialize such candidate. Our future success is dependent primarily on the regulatory approval and commercialization of our product candidates.
There can be no assurance that our focus on tivoxavir marboxil and ratutrelvir and the strategic options available for narazaciclib and rigosertib will be successful, and that we will be able to successfully develop a product candidate or, even if we do, that we will be able to successfully commercialize such candidate.
Accordingly, we have concluded that substantial doubt exists with respect to our ability to continue as a going concern within one year after the date that these financial statements are issued.
The failure to obtain sufficient capital on acceptable terms when needed would have a material adverse effect on our business, results of operations, and financial condition. Accordingly, we have concluded that substantial doubt exists with respect to our ability to continue as a going concern within one year after the date that these financial statements are issued.
This could delay completion of clinical trials; require the conduct of bridging clinical trials or studies, or the repetition of one or more clinical trials; increase clinical trial costs; delay approval of our product candidates; and jeopardize our ability to commence product sales and generate revenue.
This could delay completion of clinical trials; require the conduct of bridging clinical trials or studies, or the repetition of one or more clinical trials; increase clinical trial costs; delay approval of our product candidates; and jeopardize our ability to commence product sales and generate revenue. 42 Table of Contents We currently conduct clinical trials, and may in the future choose to conduct additional clinical trials, of our product candidates in sites outside the US, and the FDA may not accept data from trials conducted in foreign locations.
Our commercial success will depend in large part on our ability to obtain and maintain patent protection in the United States and other countries with respect to our proprietary technology and products.
We rely on trade secret, patent, copyright and trademark laws, and confidentiality, licensing and other agreements with employees and third parties, all of which offer only limited protection. Our commercial success will depend in large part on our ability to obtain and maintain patent protection in the United States and other countries with respect to our proprietary technology and products.
The size of our future net losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenues. If any of our product candidates fail in clinical trials or do not gain regulatory approval, or if approved, fail to achieve market acceptance, we may never become profitable.
If any of our product candidates fail in clinical trials or do not gain regulatory approval, or if approved, fail to achieve market acceptance, we may never become profitable. Even if we achieve profitability in the future, we may not be able to sustain profitability in subsequent periods.
We need to obtain additional funding to continue as a going concern; if we are unable to meet our needs for additional funding in the future, we will be required to limit, scale back or cease operations.
We need to obtain additional funding to execute our business plans; if we are unable to meet our needs for additional funding in the future, we will be required to limit, scale back or cease operations. We do not currently have the funding resources necessary to carry out all of our proposed operating activities.
There can be no assurance, however, that we will be successful in obtaining such funding in sufficient amounts, on terms acceptable to us, or at all. The failure to obtain sufficient capital on acceptable terms when needed would have a material adverse effect on our business, results of operations, and financial condition.
The future success of the Company is dependent upon our ability to obtain additional funding. There can be no assurance, however, that we will be successful in obtaining such funding in sufficient amounts, on terms acceptable to us, or at all.
Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. 52 Table of Contents Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business.
An adverse result in any litigation proceeding could put one or more of our patents at risk of being invalidated, held unenforceable or interpreted narrowly. Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation.
An adverse result in any litigation proceeding could put one or more of our patents at risk of being invalidated, held unenforceable or interpreted narrowly.
Our future success depends on our ability to retain our executive officers and to attract, retain and motivate qualified personnel. 41 Table of Contents We are highly dependent upon Steven Fruchtman, M.D., President and Chief Executive Officer, Mark Guerin, C.P.A., Chief Operating and Chief Financial Officer, and Victor Moyo, M.D., Chief Medical Officer, and our other executive officers.
Our future success depends on our ability to retain our executive officers and to attract, retain and motivate qualified personnel. We are highly dependent upon members of our executive management team and other employees.
These manufacturing facilities will further be subject to continuing regulatory oversight and inspection, and, thus, they must continue to expend time and resources to maintain regulatory compliance. Risks Related to Our Intellectual Property We could be required to incur significant expenses to perfect our intellectual property rights, and our intellectual property rights may be inadequate to protect our competitive position.
These manufacturing facilities will further be subject to continuing regulatory oversight and inspection, and, thus, they must continue to expend time and resources to maintain regulatory compliance. We have entered into certain related party transactions and may continue to rely on related parties for certain development and support activities.
This may make it difficult for us to achieve market acceptance at desired levels in a timely manner to ensure viability of our business. More established companies may have a competitive advantage over us due to their greater size, cash flows and institutional experience.
More established companies may have a competitive advantage over us due to their greater size, cash flows and institutional experience. Compared to us, many of our competitors may have significantly greater financial, technical and human resources.
The approved CDK 4/6 inhibitor drugs palbociclib, ribociclib and abemaciclib are well established therapies or products and are widely accepted by physicians, patients and third-party payors. By the time narazaciclib possibly is approved in the future, insurers and other third-party payors may also encourage the use of generic products.
More recently, G1 Therapeutics secured FDA approval of the CDK 4/6 triaciclib for the prevention of myelosuppression following chemotherapy. The approved antiviral drugs baloxavir marboxil, oseltamivir and CDK 4/6 inhibitor drugs palbociclib, ribociclib and abemaciclib are well established therapies or products and are widely accepted by physicians, patients and third-party payors.
At that time, we may appeal the Nasdaq staff’s determination to a Nasdaq Hearings Panel.​ ​ We intend to monitor the closing bid price of the Company’s common stock and continue to consider our available options to resolve the noncompliance with the minimum bid price requirement. 46 Table of Contents ​ There can be no assurance that we will be able to regain compliance with the minimum bid price requirement or will otherwise be in compliance with other Nasdaq listing criteria. ​ If we are unable to maintain compliance with the continued listing requirements of Nasdaq, our common stock could be delisted, making it could be more difficult to buy or sell our securities and to obtain accurate quotations, and the price of our securities could suffer a material decline.
Instead, the Staff will issue a Delist Determination Letter and we will have an opportunity to request a new hearing with the initial Hearings Panel or a newly convened Hearings Panel if the initial Hearings Panel is unavailable. ​ If we are unable to maintain compliance with the continued listing requirements of Nasdaq, our common stock could be delisted, making it could be more difficult to buy or sell our securities and to obtain accurate quotations, and the price of our securities could suffer a material decline.
For example, large pharmaceutical companies such as Pfizer, Novartis, Eli Lilly successfully market commercialized CDK 4/6 inhibitors palbociclib, ribociclib and abemaciclib and have done so for a number of years. More recently, G1 Therapeutics secured FDA approval of the CDK 4/6 triaciclib for the prevention of myelosuppression following chemotherapy.
Gilead, Pfizer and Merck have commercialized drugs for the management of COVID-19 in certain populations, including remdesivir and, nirmatrelvir + ritonavir, or molnupiravir, respectively. Furthermore, other companies such as Pfizer, Novartis, Eli Lilly successfully market commercialized CDK 4/6 inhibitors palbociclib, ribociclib and abemaciclib and have done so for a number of years.
In the first quarter of 2023, we returned a portion of the patents that we in-licensed from Temple University back to Temple. The long-term economic effects of doing so are uncertain. We may become involved in lawsuits to protect or enforce our intellectual property, which could be expensive, time consuming and unsuccessful.
We may become involved in lawsuits to protect or enforce our intellectual property, which could be expensive, time consuming and unsuccessful. Competitors may infringe our patents or misappropriate or otherwise violate our intellectual property rights.
Summary of Principal Risk Factors ● Our product development efforts may not be successful. ● Our future success is dependent primarily on the regulatory approval and commercialization of our product candidates. ● The results of preclinical testing or earlier clinical studies are not necessarily predictive of future results.
Our future success is dependent primarily on the regulatory approval and commercialization of our product candidates.
We do not have sufficient cash and cash equivalents as of the date of this Annual Report on Form 10-K to support our operations for at least the 12 months following the date that the financial statements are issued. We will require substantial additional financing to fund our ongoing clinical trials and operations, and to continue to execute our strategy.
As of December 31, 2024, we had cash and cash equivalents of $21.3 million and current liabilities of $11.5 million. Based on current projections, we do not have sufficient cash and cash equivalents as of the date of this Annual Report to support our operations for more than one year following the date that the financial statements are issued.
Even if our clinical development programs are successful, we may not be able to successfully commercialize any product.
Although we believe that there are opportunities for us to develop our drug candidates in various indications, clinical drug development is expensive, can take many years to complete, and its outcome is inherently uncertain. Even if our clinical development programs are successful, we may not be able to successfully commercialize any product.
If we are unable to comply with the continued listing requirements of the Nasdaq Capital Market, our Common Stock could be delisted, which could affect our common stock's market price and liquidity and reduce our ability to raise capital. ​ We are required to meet certain qualitative and financial tests to maintain the listing of our securities on the Nasdaq Capital Market (Nasdaq).
If we raise additional funds by issuing debt securities, the holders of these debt securities would similarly have some rights senior to those of our existing securityholders, and the terms of these debt securities could impose restrictions on operations and create a significant interest expense for us which could have a materially adverse effect on our business. ​ If we are unable to comply with the continued listing requirements of the Nasdaq Capital Market, our common stock could be delisted, which could affect our common stock's market price and liquidity and reduce our ability to raise capital. ​ Our common stock is listed on the Nasdaq Capital Market (“Nasdaq”), a national securities exchange, which imposes continued listing requirements with respect to issuers whose securities are listed on Nasdaq.
If we are unable to protect our intellectual property rights, our competitive position could be harmed. We depend on our ability to protect our proprietary technology. We rely on trade secret, patent, copyright and trademark laws, and confidentiality, licensing and other agreements with employees and third parties, all of which offer only limited protection.
Risks Related to Our Intellectual Property We could be required to incur significant expenses to perfect our intellectual property rights, and our intellectual property rights may be inadequate to protect our competitive position. If we are unable to protect our intellectual property rights, our competitive position could be harmed. We depend on our ability to protect our proprietary technology.
The effects of a health crisis may also increase the need for clinical trial patient monitoring and regulatory reporting of adverse effects. ​ Risks Related to Our Dependence on Third Parties We rely on third parties to conduct our preclinical and clinical trials.
The effects of a health crisis may also increase the need for clinical trial patient monitoring and regulatory reporting of adverse effects. ​ Risk Factors Relating to the Merger There is no guarantee that the Merger will increase stockholder value. As discussed elsewhere in this Annual Report, in April 2024, we merged with Trawsfynydd.
For the years ended December 31, 2023, and 2022, we reported net losses of $18.9 million and $19.0 million, respectively, and we had an accumulated deficit of $482.6 million at December 31, 2023. We expect to continue to incur significant expenses and operating losses for the foreseeable future.
As a result, we are not profitable and have incurred losses in every reporting period since our inception in 1998. For the years ended December 31, 2024, and 2023, we reported net losses of $166.5 million and $18.9 million, respectively, and we had an accumulated deficit of $649.2 million as of December 31, 2024.
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If we are unable to protect our intellectual property rights, our competitive position could be harmed. ● We may become involved in lawsuits to protect or enforce our intellectual property, which could be expensive, time consuming and unsuccessful. ● Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business. ● We may be subject to claims that our employees have wrongfully used or disclosed alleged trade secrets of their former employers. ● We may not comply with the Nasdaq continued listing requirements.
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Summary of Principal Risk Factors ● Our recurring operating losses, negative cash flows from operations, and accumulated deficit raise substantial doubt about our ability to continue as a going concern absent obtaining adequate new financings. 32 Table of Contents ● The report of our independent registered accounting firm on our audited financial statements for the fiscal year ended December 31, 2024 contains an explanatory paragraph relating to our ability to continue as a going concern. ● We have identified material weaknesses in our internal control over financial reporting; if we fail to develop and maintain an effective system of disclosure controls and internal control over financial reporting, our ability to produce timely and accurate financial statements or comply with applicable laws and regulations could be impaired. ● We need to obtain additional funding to continue as a going concern; if we are unable to meet our needs for additional funding in the future, we will be required to limit, scale back or cease operations. ● Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates. ● Our product development efforts may not be successful. ● Our future success is dependent primarily on the regulatory approval and commercialization of our product candidates. ● The results of preclinical testing or earlier clinical studies are not necessarily predictive of future results.
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Management has concluded that substantial doubt exists about our ability to continue as a going concern for the next twelve months from the date of the financial statements included in this Annual Report on Form 10-K. As of December 31, 2023, we had cash and cash equivalents of $20.8 million and current liabilities of $9.2 million.
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We may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect our business. The size of our future net losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenues.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeThird-party vendor cybersecurity risks and procedures are evaluated by our Quality Assurance and Regulatory Department during our prequalification process and during subsequent periodic reviews. We do not specifically utilize assessors, consultants, auditors, or other third parties to evaluate or enhance our cybersecurity programs.
Biggest changeWe do not currently have a formal process in place for evaluating or reviewing third party vendor cybersecurity risks and procedures and rely on such third parties to implement adequate protectionary and detection measures. We do not specifically utilize assessors, consultants, auditors, or other third parties to evaluate or enhance our cybersecurity programs.
We also alert employees to significant new cybersecurity issues on a regular basis. We rely heavily on third party service providers for our clinical development activities and cloud-based documentation and communications. A cybersecurity incident at a vendor or other third-party service provider could have a material and adverse impact on our business, results of operations and financial condition.
We rely heavily on third party service providers for our clinical development activities and cloud-based documentation and communications. A cybersecurity incident at a vendor or other third-party service provider could have a material and adverse impact on our business, results of operations and financial condition.
The Director- Information Technology reviews cybersecurity risks with and reports cybersecurity incidents to the Chief Executive Officer, Chief Operating Officer/Chief Financial Officer, and Senior Director – Financial Reporting. 48 Table of Contents To date, we are not aware of any cybersecurity incident that has had or is reasonably likely to have a material impact on our business or operations; however, because of the frequently changing attack techniques, along with the increased volume and sophistication of the attacks, there is the potential for us to be adversely impacted.
To date, we are not aware of any cybersecurity incident that has had or is reasonably likely to have a material impact on our business or operations; however, because of the frequently changing attack techniques, along with the increased volume and sophistication of the attacks, there is the potential for us to be adversely impacted.
On an annual basis, our information technology risks, controls and procedures are reviewed by third-party experts as part of our Sarbanes-Oxley review and testing. Board Governance and Management Our board of directors has oversight responsibility for risk management, which it administers directly and with assistance from its committees, primarily the audit committee.
On an annual basis, our information technology risks, controls and procedures are reviewed by third-party experts as part of our Sarbanes-Oxley review and testing. Board Governance and Management Our management team is primarily responsible for assessing and managing our strategic risk exposures, including material risks from cybersecurity threats, with assistance from third-party service providers.
Significant changes are reported to the audit committee quarterly. Cybersecurity incidents which are determined by management to be material will be reported immediately to the audit committee. We take a risk-based approach to cybersecurity and have implemented cybersecurity procedures designed to address cybersecurity threats and risks.
Significant changes are reported to the audit committee from time to time. Cybersecurity incidents which are determined by management to be material will be reported immediately to the audit committee.
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ITEM 1C. CYBERSECURITY Risk Management and Strategy We monitor our information systems for potential cybersecurity issues and assess risks from cybersecurity threats as an element of our overall business risks. To protect our information systems from cybersecurity threats, we use various security tools supporting protection, detection, and response/mitigation capabilities.
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ITEM 1C. CYBERSECURITY Risk Management and Strategy Due to the size of our company, we have not yet developed robust policies and processes for assessing, identifying, and managing material risk from cybersecurity threats. We have implemented access controls with respect to our systems, which we monitor regularly.
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The Director – Information Technology, who has a combination of 15 years of professional experience and education, as well as certifications in cybersecurity, leads the Company’s cybersecurity program and is responsible for assessing and managing cybersecurity risk.
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We currently rely heavily on products and services provided by third-party suppliers to operate certain critical business systems, including without limitation, cloud-based infrastructure, encryption and authentication technology, email, and other functions. We rely on third party providers and outsourced IT services to protect, detect, monitor, mitigate and address cybersecurity related risks, including installing software for threat protection and malware.
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Such third party providers are tasked with notifying management of any material risks or cybersecurity concerns that they identify, which management then assesses and may bring to our audit committee or board of directors to discuss if deemed necessary or appropriate. We also alert employees to significant new cybersecurity issues on a regular basis.
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Management oversees our cybersecurity process on a day-to-day basis, including those described in “Risk Management and Strategy,” above. 56 Table of Contents Our board of directors has oversight responsibility for risk management, which it administers directly and with assistance from its committees, primarily the audit committee.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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ITEM 3. LEGAL PROCEEDINGS We are not a party to any legal proceedings and we are not aware of any such proceedings contemplated by government authorities. ITEM 4. MINE SAFETY DISCLOSURES Not applicable. ​ 49 Table of Contents PART II
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ITEM 3. LEGAL PROCEEDINGS For a description of our material pending legal proceedings, please see Note 5, Commitments and Contingencies, to our consolidated financial statements included in Part IV in this Annual Report.
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We may, in the ordinary course of business, face various claims brought by third parties, and we may, from time to time, make claims or take legal actions to assert our rights, including intellectual property rights as well as claims relating to employment matters and the safety or efficacy of our products.
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Any of these claims could subject us to costly litigation. If this were to happen, the payment of any such awards could have a material adverse effect on our business, financial condition and results of operations. Additionally, any such claims, whether or not successful, could damage our reputation and business. ITEM 4.
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MINE SAFETY DISCLOSURES Not applicable. ​ 57 Table of Contents PART II

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeThis does not reflect beneficial stockholders who held their common stock in “street” or nominee name through brokerage firms. Securities Authorized for Issuance Under Equity Compensation Plans Information regarding securities authorized for issuance under the Company’s equity compensation plans is contained in Part III, Item 11 of this Annual Report.
Biggest changeThis does not reflect beneficial stockholders who held their common stock in “street” or nominee name through brokerage firms. Securities Authorized for Issuance Under Equity Compensation Plans Information regarding securities authorized for issuance under the Company’s equity compensation plans is contained in Part III, Item 11 of this Annual Report, and incorporated by reference herein.
Dividend Policy We have never declared or paid any cash dividends on our capital stock. We currently intend to retain all available funds and any future earnings to support our operations and finance the growth and development of our business. We do not intend to pay cash dividends on our common stock for the foreseeable future. ITEM 6. RESERVED
Dividend Policy We have never declared or paid any cash dividends on our capital stock. We currently intend to retain all available funds and any future earnings to support our operations and finance the growth and development of our business. We do not intend to pay cash dividends on our common stock for the foreseeable future.
ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed under the symbol ONTX on the Nasdaq Capital Market. Stockholders As of February 29, 2024, there were approximately 96 holders of record for shares of our common stock.
ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed under the symbol TRAW on the Nasdaq Capital Market. Stockholders As of March 26, 2025, there were approximately 105 holders of record for shares of our common stock.
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Recent Sales of Unregistered Securities; Use of Proceeds from Registered Securities During the fiscal year ended December 31, 2024, there were no unregistered sales of our securities that were not reported in a Current Report on Form 8-K or our Quarterly Reports on Form 10-Q.
Added
Issuer Repurchases of Equity Securities There were no repurchases of our capital stock during the fourth quarter of 2024. ​ ITEM 6. RESERVED

Item 7. Management's Discussion & Analysis

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

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Biggest changeThe accompanying financial statements have been prepared on a going concern basis, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business, and do not include any adjustments relating to recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might be necessary should we be unable to continue as a going concern. 55 Table of Contents Cash Flows The following table summarizes our cash flows for the year ended December 31, 2023 and 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ 2023 2022 Net cash (used in) provided by: ​ ​ Operating activities ​ $ (17,932,000) ​ $ (16,294,000) Investing activities ​ (14,000) ​ — Financing activities ​ — ​ — Effect of foreign currency translation ​ 10,000 ​ (19,000) Net decrease in cash and cash equivalents ​ $ (17,936,000) ​ $ (16,313,000) ​ Net cash used in operating activities Net cash used in operating activities was $17.9 million for the year ended December 31, 2023 and consisted primarily of a net loss of $18.9 million, including $1.3 million of noncash stock-based compensation and depreciation expense.
Biggest changeAccordingly, we have concluded that substantial doubt exists with respect to our ability to continue as a going concern within one year after the date that these financial statements are issued. 71 Table of Contents The accompanying financial statements have been prepared on a going concern basis, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business, and do not include any adjustments relating to recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might be necessary should we be unable to continue as a going concern.
These conditions raise substantial doubt about our ability to continue as a going concern through the one-year period after the date that the financial statements are issued. We are exploring various sources of funding for continued development of narazaciclib and any potential in-licensed compounds as well as our ongoing operations.
These conditions raise substantial doubt about our ability to continue as a going concern through the one-year period after the date that the financial statements are issued. We are exploring various sources of funding for continued development and any potential in-licensed compounds as well as our ongoing operations.
We may rely on licensing and co-promotion agreements with strategic or collaborative partners for the commercialization of our products in the United States and other territories.
In the future, we may rely on licensing and co-promotion agreements with strategic or collaborative partners for the commercialization of our products in the United States and other territories.
We are recognizing the $7.5 million upfront payment received in 2011 under the SymBio collaboration agreement as revenue on a straight-line basis through December 2037, reflecting our estimate of when we will complete our obligations under the agreement. For the years ended December 31, 2023 and 2022, we recognized revenues of $226,000 and $226,000, respectively, under the SymBio collaboration agreement.
We are recognizing the $7.5 million upfront payment received in 2011 under the SymBio collaboration agreement as revenue on a straight-line basis through December 2037, reflecting our estimate of when we will complete our obligations under the agreement. For the years ended December 31, 2024 and 2023, we recognized revenues of $226,000, under the SymBio collaboration agreement.
The financial terms of these contracts are subject to negotiations, which vary from contract to contract and may result in payment flows that do not match the periods over which materials or services are provided to us under such contracts.
The financial terms of these contracts are subject to negotiations, which vary from contract to contract and may result in 73 Table of Contents payment flows that do not match the periods over which materials or services are provided to us under such contracts.
If we obtain regulatory approval for any of our product candidates, we expect to incur significant NDA preparation and commercialization expenses. We do not currently have an organization for the sales, marketing and distribution of pharmaceutical products.
If we obtain regulatory approval for any of our product candidates, we expect to incur significant NDA preparation and commercialization expenses. We do not currently have a relationship with an organization for the sales, marketing and distribution of pharmaceutical products.
We expect net cash expended in 2024 to be higher than 2023 due to clinical trials with narazaciclib and increased headcount in our clinical and regulatory groups. We also expect an increase in costs for potential in-licensing, the timing of which will be determined by the timing of any potential in-licensing.
We expect net cash expended in 2025 to be higher than 2024 due to clinical trials and increased headcount in our clinical and regulatory groups. We also expect an increase in costs for potential in-licensing, the timing of which will be determined by the timing of any potential in-licensing.
Research and Development Expenses Our research and development expenses consist primarily of costs incurred for the development of our product candidates, which include: ● employee-related expenses, including salaries, benefits, travel and stock-based compensation expense; ● expenses incurred under agreements with CROs and investigative sites that conduct our clinical trials and preclinical studies; ● the cost of acquiring, developing and manufacturing clinical trial materials; ● direct expenses for maintenance of research equipment, clinical trial insurance and other supplies; and ● costs associated with preclinical activities and regulatory operations.
As such, and since our inception, the purchase price of licenses acquired is classified as acquired in-process research and development expenses in the statements of operations. ​ Research and Development Expenses Our research and development expenses consist primarily of costs incurred for the development of our product candidates, which include: ● employee-related expenses, including salaries, benefits, travel and stock-based compensation expense; ● expenses incurred under agreements with CROs and investigative sites that conduct our clinical trials and preclinical studies; ● the cost of acquiring, developing and manufacturing clinical trial materials; ● direct expenses for maintenance of research equipment, clinical trial insurance and other supplies; and ● costs associated with preclinical activities and regulatory operations.
Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. Our research and development expenses are related to narazaciclib, oral rigosertib, other candidates in our pipeline, and potentially in-licensed products.
Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. 68 Table of Contents Our research and development expenses are related to tivoxavir marboxil, ratutrelvir, narazaciclib, rigosertib, and potentially in-licensed products.
We expect to incur significant expenses and operating losses for the foreseeable future as we continue the development and clinical trials of, and seek regulatory approval for, our product candidates, even if milestones under our license and collaboration agreements may be met. As of December 31, 2023 we had $20.8 million in cash and cash equivalents.
We expect 58 Table of Contents to incur significant expenses and operating losses for the foreseeable future as we continue the development of, and seek regulatory approval for, our product candidates, even if milestones under our license and collaboration agreements may be met. ​ As of December 31, 2024, the Company had $21.3 million in cash and cash equivalents.
Due to the inherent uncertainty involved in making estimates and the risks associated with the research, development, and commercialization of biotechnology products, we may have based this estimate on assumptions that may prove to be wrong, and our operating plan may change as a result of many factors currently unknown to us.
Due to the inherent uncertainty involved in making estimates and the risks associated with the research, development, and commercialization of biotechnology products, we may have based this estimate on assumptions that may prove to be wrong, and our operating plan may change as a result of many factors currently unknown to us. ​ We will require substantial additional financing to fund our ongoing clinical trials and operations, and to continue to execute our strategy.
To alleviate the conditions that raise substantial doubt about our ability to continue as a going concern, we plan to explore various dilutive and non-dilutive sources of funding, including equity financings, strategic alliances, business development and/or combinations, and other sources. The future success of the Company is dependent upon our ability to obtain additional funding.
To alleviate the conditions that raise substantial doubt about our ability to continue as a going concern, we plan to explore various dilutive and non-dilutive sources of funding, including debt and equity financings (including pursuant to the ATM Agreement), strategic alliances, business development and/or combinations, and other sources.
Our operating activities used $17.9 million and $16.3 million of net cash during the year ended December 31, 2023 and 2022, respectively. At December 31, 2023, we had an accumulated deficit of $482.6 million, working capital of $13.4 million, and cash and cash equivalents of $20.8 million.
Our operating activities used $29.8 million and $17.9 million of net cash during the year ended December 31, 2024 and 2023, respectively. As of December 31, 2024, we had an accumulated deficit of $649.2 million, working capital of $13.4 million, and cash and cash equivalents of $21.3 million.
Other general and administrative expenses include facility-related costs, communication expenses, insurance, board of directors expenses and professional fees for legal, patent review, consulting and accounting services.
General and Administrative Expenses General and administrative expenses consist principally of salaries and related costs for executive and other administrative personnel, including stock-based compensation and travel expenses. Other general and administrative expenses include facility-related costs, communication expenses, insurance, board of directors expenses and professional fees for legal, patent review, consulting and accounting services.
Liquidity and Capital Resources Since our inception, we have incurred net losses and experienced negative cash flows from our operations. We incurred net losses of $18.9 million and $19.0 million for the years ended December 31, 2023 and 2022, respectively.
The change was caused by lower interest income due to lower cash balances. Liquidity and Capital Resources Since our inception, we have incurred net losses and experienced negative cash flows from our operations. We incurred net losses of $166.5 million and $18.9 million for the years ended December 31, 2024 and 2023, respectively.
Deferred revenue decreased $0.2 million due to recognition of the unamortized portion of the upfront payment under our collaboration agreement with SymBio. Net cash used in operating activities was $16.3 million for the year ended December 31, 2022 and consisted primarily of a net loss of $19.0 million, including $1.2 million of noncash stock-based compensation and depreciation expense.
Deferred revenue decreased $0.2 million due to recognition of the unamortized portion of the upfront payment under our collaboration agreement with SymBio. Net cash used in investing activities Net cash used in investing activities was $3.6 million for the year ended December 31, 2024 and primarily related to the transaction costs of $3.6 million in connection with the Merger.
Net cash provided by financing activities There was no net cash used in or provided by financing activities in the 2023 or 2022 periods. Material Cash Requirements We have not achieved profitability since our inception and we expect to continue to incur net losses for the foreseeable future.
There were no financing activities during the year ended December 31, 2023. 72 Table of Contents Material Cash Requirements We have not achieved profitability since our inception and we expect to continue to incur net losses for the foreseeable future.
Our net losses were $18.9 million and $19.0 million for the years ended December 31, 2023 and 2022, respectively. As of December 31, 2023, we had an accumulated deficit of $482.6 million.
Overview The Company’s net losses were $166.5 million and $18.9 million for the years ended December 31, 2024 and 2023, respectively. As of December 31, 2024, the Company had an accumulated deficit of $649.2 million.
We believe that our cash and cash equivalents will be sufficient to fund our ongoing trials and operations into the third quarter of 2024; however, based on current projections, we do not have sufficient cash and cash equivalents as of the date of this Annual Report on Form 10-K to support our operations for at least the 12 months following the date that the financial statements are issued.
Based on current projections, we believe that we do not have sufficient cash and cash equivalents to support our operations for more than one year following the date that these financial statements from our Annual Report on Form 10-K are issued.
We anticipate that our general and administrative expenses will remain consistent in the short-term, but would increase in the future with the continued research and development and potential commercialization of our product 51 Table of Contents candidates.
We anticipate that our general and administrative expenses will remain consistent in the short-term, but would increase in the future with the continued research and development and potential commercialization of our product candidates. These increases will likely include increased costs for insurance, costs related to the hiring of additional personnel and payments to outside consultants among other expenses.
There can be no assurance, however, that we will be successful in obtaining such funding in sufficient amounts, on terms acceptable to us, or at all. The failure to obtain sufficient capital on acceptable terms when needed would have a material adverse effect on our business, results of operations, and financial condition.
The failure to obtain sufficient capital on acceptable terms when needed would have a material adverse effect on our business, results of operations, and financial condition.
The details of our general and administrative expenses are: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ 2023 2022 Professional & consulting fees ​ $ 2,162,000 ​ $ 1,824,000 ​ Stock based compensation ​ ​ 715,000 ​ ​ 538,000 ​ Personnel related ​ 3,264,000 ​ 3,408,000 ​ Public company costs ​ 1,968,000 ​ 1,382,000 ​ Insurance & other ​ ​ 985,000 ​ ​ 1,295,000 ​ ​ ​ $ 9,094,000 ​ $ 8,447,000 ​ ​ 54 Table of Contents Research and development expenses Research and development expenses were flat in 2023 compared to 2022 at $11.4 million.
The increases were partially offset by a $3.2 million decrease in oncology costs and a $0.4 million decrease in stock based compensation. 70 Table of Contents General and administrative expenses The details of our general and administrative expenses are: ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ 2024 2023 Professional & consulting fees ​ $ 5,954,000 ​ $ 2,162,000 Stock based compensation ​ ​ 1,209,000 ​ ​ 715,000 Personnel related ​ 3,035,000 ​ 3,264,000 Public company costs ​ 1,231,000 ​ 1,968,000 Insurance & other ​ ​ 860,000 ​ ​ 985,000 ​ ​ $ 12,289,000 ​ $ 9,094,000 ​ General and administrative expenses increased by $3.2 million, or 35.1%, to $12.3 million for the year ended December 31, 2024, from $9.1 million for the year ended December 31, 2023.
These increases were partially offset by $0.3 million lower insurance and other costs and lower personnel related costs of $0.1 million in the 2023 period due to lower bonus accrual and headcount.
These increases were partially offset by a decrease of $0.7 million in public company costs and a decrease in personnel related costs of $0.2 million due to a decrease in headcount.
Other Income, Net Other income, net consists principally of interest income earned on cash and cash equivalent balances and foreign exchange gains and losses. 52 Table of Contents Critical Accounting Policies and Estimates This management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which have been prepared in accordance with US generally accepted accounting principles (GAAP).
Please see “Risk Factors” for additional risks associated with our substantial capital requirements. Critical Accounting Policies and Estimates This management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which have been prepared in accordance with US generally accepted accounting principles (“GAAP”).
Results of Operations Comparison of the Years Ended December 31, 2023 and 2022 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ ​ 2023 2022 Change Revenue ​ $ 226,000 ​ $ 226,000 ​ $ — Operating expenses: ​ ​ ​ ​ General and administrative ​ ​ 9,094,000 ​ 8,447,000 ​ (647,000) Research and development ​ ​ 11,430,000 ​ 11,406,000 ​ (24,000) Total operating expenses ​ 20,524,000 ​ 19,853,000 ​ (671,000) Loss from operations ​ (20,298,000) ​ (19,627,000) ​ (671,000) Other income, net ​ 1,350,000 ​ 663,000 ​ 687,000 Net loss ​ $ (18,948,000) ​ $ (18,964,000) ​ $ 16,000 ​ Revenues Revenues for 2023 were consistent with 2022 and were due to the recognition of deferred revenue from our collaboration with SymBio.
Other Income, Net Other income, net consists principally of interest income earned on cash and cash equivalent balances and foreign exchange gains and losses. 69 Table of Contents Results of Operations Comparison of the Years Ended December 31, 2024 and 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ ​ 2024 2023 Change Revenue ​ $ 226,000 ​ $ 226,000 ​ $ — Operating expenses: ​ ​ ​ ​ Acquired in-process research and development ​ ​ 117,464,000 ​ — ​ 117,464,000 Research and development ​ ​ 12,847,000 ​ ​ 11,430,000 ​ ​ 1,417,000 General and administrative ​ ​ 12,289,000 ​ 9,094,000 ​ 3,195,000 Total operating expenses ​ 142,600,000 ​ 20,524,000 ​ 122,076,000 Loss from operations ​ (142,374,000) ​ (20,298,000) ​ (122,076,000) Series A warrant and prefunded warrant expense ​ (24,438,000) ​ — ​ (24,438,000) Other income, net ​ 289,000 ​ 1,350,000 ​ (1,061,000) Net loss ​ $ (166,523,000) ​ $ (18,948,000) ​ $ (147,575,000) ​ Revenues Revenues for the years ended December 31, 2024 were consistent with the year ended December 31, 2023 and were due to the recognition of deferred revenue from our collaboration with SymBio.
Accordingly, we have concluded that substantial doubt exists with respect to our ability to continue as a going concern within one year after the date that these financial statements are issued.
Based on current projections, we believe that we do not have sufficient cash and cash equivalents to support our operations for more than one year following the date that these financial statements are issued. As a result of these conditions, substantial doubt exists about our ability to continue as a going concern.
We believe that our cash and cash equivalents will be sufficient to fund our ongoing trials and business operations into the third quarter of 2024; however, based on current projections, we do not have sufficient cash and cash equivalents as of the date of this Annual Report on Form 10-K to support our operations for at least the 12 months following the date that the financial statements are issued.
Based on current projections, we believe that we do not have sufficient cash and cash equivalents to support our operations for more than one year following the date that these financial statements are issued. ​ We are exploring various sources of funding for development and applying for regulatory approval of our research compounds as well as for our ongoing operations.
The details of our research and development expenses are: ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2023 2022 Preclinical & clinical development ​ $ 4,060,000 ​ $ 4,206,000 Personnel related ​ 2,400,000 ​ 2,399,000 Manufacturing, formulation & development ​ 2,798,000 ​ 2,851,000 Stock based compensation ​ 586,000 ​ 617,000 Consulting fees ​ 1,586,000 ​ 1,333,000 ​ ​ $ 11,430,000 ​ $ 11,406,000 ​ Other income, net Other income, net, increased by $0.7 million for the year ended December 31, 2023 compared to the year ended December 31, 2022 due primarily to $0.7 million higher interest income in the 2023 period, partially offset by $49,000 higher foreign currency exchange loss in the 2023 period.
Research and development expenses The details of our research and development expenses are: ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 Virology ​ $ 4,589,000 ​ $ - Oncology ​ ​ 5,290,000 ​ ​ 8,444,000 Personnel related ​ 2,787,000 ​ 2,400,000 Stock based compensation ​ 181,000 ​ 586,000 ​ ​ $ 12,847,000 ​ $ 11,430,000 ​ Research and development expenses increased by $1.4 million, or 12.4%, to $12.8 million for the year ended December 31, 2024, from $11.4 million for the year ended December 31, 2023.
Significant changes in operating assets and liabilities included an increase in accounts payable and accrued liabilities of $1.9 million as a result of the timing of clinical trial and other accruals, and receipt and payment of vendor invoices, and an increase of $0.2 million in prepaid expenses and other current assets.
Significant changes in operating assets and liabilities included a net decrease in accounts payable and accrued expenses of $4.5 million due to timing of invoices and payments to our vendors.
Changes in operating assets and liabilities resulted in a net decrease in cash of $0.3 million.
Net cash used in operating activities was $17.9 million for the year ended December 31, 2023 and consisted primarily of a net loss of $18.9 million, including $1.3 million of noncash stock-based compensation and depreciation expense. Changes in operating assets and liabilities resulted in a net decrease in cash of $0.3 million.
Accordingly, substantial doubt exists with respect to our ability to continue as a going concern within one year after the date that these financial statements are issued. Financial Overview Revenue During the years ended December 31, 2023 and 2022, our revenues were derived exclusively from activities conducted in accordance with our collaboration arrangement with SymBio.
Cautreels will continue to serve as a director on the Board after the Termination Date, and will also continue to provide certain consulting services to the Company for a period of time after the Termination Date. ​ Iain Dukes, the Company’s Executive Chairman, has been appointed as Interim Chief Executive Officer, effective as of the Termination Date. ​ ​ 67 Table of Contents Financial Overview Revenue During the years ended December 31, 2024 and 2023, our revenues were derived exclusively from activities conducted in accordance with our collaboration arrangement with SymBio.
The increase was attributable primarily to $0.6 million higher expenses for investor relations, proxy solicitation, and fees related to our shareholder meetings in the 2023 period, $0.3 million higher professional and consulting fees, and $0.2 million higher stock-based compensation costs in 2023.
The increase was primarily attributable to a $3.8 million increase in consulting fees in connection with seeking strategic alternatives for our investors and a $0.5 million increase in stock based compensation as a result of the options assumed in connection with the Merger.
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Overview We are a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. We have proprietary molecularly targeted agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.
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On December 29, 2024, the Company entered into the Securities Purchase Agreement described below (the “December 2024 Purchase Agreement”), for the sale of shares of Company common stock, pre-funded warrants, and Series A Warrants and raised gross proceeds of $20.0 million at closing on December 31, 2024.
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We believe that the product candidates in our pipeline have the potential to be efficacious in a variety of cancers with unmet medical need.
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In addition, on April 1, 2024, in connection with the Merger, the Company entered into a Securities Purchase Agreement (the “April 2024 Securities Purchase Agreement”) for the sale of common and preferred stock to TPAV, LLC (“TPAV”), an affiliate of Torrey Pines, and OrbiMed Private Investments VIII, LP (“OrbiMed”), an affiliate of OrbiMed Advisors (together, the “Investors”) and raised gross proceeds of $14.0 million.
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We have the following two clinical-stage programs: (1) narazaciclib (ON 123300), a multi-targeted kinase inhibitor in solid tumors and hematological malignancies as a single agent or in combination with other anti-cancer therapies; and (2) rigosertib administered alone or in combination for the treatment of various cancers. We are currently evaluating potential compounds for in-licensing opportunities.
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If we raise additional funds through strategic collaborations and alliances or licensing arrangements with third parties, which may include existing collaboration partners, we may have to relinquish valuable rights to our technologies or product candidates or grant licenses on terms that are not favorable to us.
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We were incorporated in Delaware in December 1998 and commenced operations in January 1999.
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There can be no assurance, however, that we will be successful in obtaining such financing in sufficient amounts, on terms acceptable to us, or at all. In addition, there can be no assurance that we will obtain approvals necessary to market our product candidates or achieve profitability or sustainable, positive cash flow.
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Our operations to date have included our organization and staffing, business planning, raising capital, in-licensing technology from research institutions, identifying potential product candidates, developing product candidates and building strategic alliances, as well as undertaking preclinical studies and clinical trials of our product candidates. 50 Table of Contents Since commencing operations, we have dedicated a significant portion of our resources to the development of our clinical-stage product candidates, particularly narazaciclib and rigosertib.
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If we are unable to successfully raise sufficient additional capital, through future financings or through strategic and collaborative arrangements, we will not have sufficient cash to fund our ongoing trials and operations. ​ Our Portfolio/ Product Candidates/ Compounds ​ We are a clinical-stage biopharmaceutical company aiming to address unmet medical needs in respiratory viral diseases and cancer.
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We incurred research and development expenses of $11.4 million and $11.4 million during the years ended December 31, 2023 and 2022, respectively. We anticipate that a significant portion of our operating expenses will continue to be related to research and development as we continue to advance narazaciclib and our other programs.
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Following the closing of the Merger in which we acquired Trawsfynydd Therapeutics, Inc. on April 1, 2024, we are advancing the development of four clinical programs: ​ ● Tivoxavir marboxil, which we acquired as part of the Merger, is a small molecule cap-dependent endonuclease inhibitor. Cap-dependent endonuclease (“CEN”) is an enzyme that is important for influenza virus replication.
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In addition, we recognized revenues of $0 and $0 for the years ended December 31, 2023 and 2022, respectively, related to the supply agreement.
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Tivoxavir marboxil is intended to inhibit CEN and, thus, is intended to impede influenza virus replication including, the influenza A or B viral strains and bird flu viral strains.
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Operating Expenses The following table summarizes our operating expenses for the years ended December 31, 2023 and 2022: ​ ​ ​ ​ ​ ​ ​ ​ 2023 2022 General and administrative ​ $ 9,094,000 ​ $ 8,447,000 Research and development ​ 11,430,000 ​ 11,406,000 Total operating expenses ​ $ 20,524,000 ​ $ 19,853,000 ​ General and Administrative Expenses General and administrative expenses consist principally of salaries and related costs for executive and other administrative personnel, including stock-based compensation and travel expenses.
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It is our intention to develop tivoxavir marboxil as an oral dose given only once for potential treatment and prophylaxis of bird flu and seasonal influenza. ​ The first-in-man clinical study of tivoxavir marboxil (designated AV5124 in a previous study) was performed from May to September of 2023 in Russia. The study sponsor was Pharmasyntez, JSC.
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These increases will likely include increased costs for insurance, costs related to the hiring of additional personnel and payments to outside consultants among other expenses.
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We have the right to use the data resulting from the study outside of Russia and the Eurasian Economic Community countries. The trial was a single ascending dose study, and, as such, each study participant only received one dose of tivoxavir marboxil.
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Stock-Based Compensation We account for stock-based payments to employees and non-employees using an option pricing model for estimating fair value. Accordingly, stock-based compensation expense is measured based on the estimated fair value of the awards on the date of grant, net of forfeitures.
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The study consisted of four dose cohorts that received 20, 40, 80 or 120 mg tivoxavir marboxil delivered as 20 mg strength tablets, or placebo. The study enrolled 28 healthy males ages 18-45 years who received either the study drug or placebo. The primary study endpoint was measurement of the safety and tolerability of single drug doses in healthy volunteers.
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Compensation expense is recognized for the portion that is ultimately expected to vest over the period during which the recipient renders the required services, using the straight-line single option method. We record stock-based compensation expense as a component of research and development expenses or general and administrative expenses, depending on the function performed by the optionee.
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The secondary endpoint was the measurement of pharmacokinetic parameters of single drug doses in healthy volunteers on an empty stomach or after a meal. In the study, one subject who received a single 40 mg dose of the study drug, experienced two adverse events (“AEs”).
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For the years ended December 31, 2023 and 2022, we allocated stock-based compensation as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2023 2022 General and administrative ​ $ 586,000 $ 538,000 Research and development ​ 715,000 ​ 617,000 ​ ​ $ 1,301,000 ​ $ 1,155,000 53 Table of Contents ​ Fair Value Estimates We estimate the fair value of share-based awards to employees and non-employees using the Black-Scholes option pricing model.
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This subject experienced hyperglycemia, which was deemed to be mild and believed probably related to tivoxavir marboxil, and erosive gastritis with complications in the form of severe iron deficiency anemia, which was considered to be a serious adverse event (“SAE”) believed unlikely to be related (doubtful per the protocol) to the study drug. 59 Table of Contents There were no other AEs in the trial, including at higher doses.
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The Black-Scholes model requires the input of highly complex and subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of the expected term of the award, (c) the risk free interest rate and (d) expected dividends. Expected volatility is based on the historical volatility of the Company’s common stock.
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The pharmacokinetic measurements indicated a small food effect for tivoxavir marboxil, with increased exposure when drug was taken after a meal but otherwise showed increasing exposure with increasing dose.
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We estimate the expected life of our employee stock options using the “simplified” method, whereby, the expected life equals the arithmetic average of the vesting term and the original contractual term of the option. The risk-free interest rates for periods within the expected life of the option are based on the U.S.
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We are further advancing the development of tivoxavir marboxil with a Traws Pharma sponsored Phase 1 randomized, blinded, and placebo-controlled study in Australia that was approved by the Human Research Ethics Committee. To date, this study enrolled four cohorts of 8 participants, with 6 participants randomized to receive study drug and 2 participants assigned to receive placebo in each cohort.
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Treasury yield curve in effect during the period when the options were granted. We have never paid, and do not expect to pay dividends in the foreseeable future.
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Participants were required to be healthy males or females ages 18-64 years. Participants took either one dose of the study drug or one dose of placebo, depending on the group they were assigned to. The dose levels that were evaluated included 80, 120, 240 mg, and 480 mg taken, via oral capsules.
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General and administrative expenses General and administrative expenses increased by $0.6 million or 7.7%, to $9.1 million for the year ended December 31, 2023 from $8.4 million for the year ended December 31, 2022.
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The primary endpoint of the study was the measurement of safety and tolerability, and the secondary and other endpoints included the determination of the drug pharmacokinetic profile. Topline data showed good overall tolerability and a pharmacokinetic profile that appears to support the potential use of tivoxavir marboxil as a one-time treatment for influenza.
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There was an increase of $0.3 million in consulting fees, which was offset by decreases of $0.1 million preclinical and clinical development, as well as $0.1 million lower manufacturing and formulation costs.
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Sixteen AEs were recorded, of which three were reported as possibly related to study drug during the study; all were mild headaches. Topline data from this study showed that a single dose of tivoxavir marboxil maintained plasma drug levels consistently above the EC90 and within the predicted therapeutic window for more than 23 days.
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These conditions raise substantial doubt about our ability to continue as a going concern through the one-year period after the date that the financial statements are issued.
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We plan to meet with the FDA in the first half of 2025 to align on a path forward, including to seek guidance regarding the potential for accelerated approval utilizing the “Animal Rule” for further development of tivoxavir marboxil in the treatment of H5N1 bird flu.
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We will require substantial additional financing to fund our ongoing clinical trials and operations, and to continue to execute our strategy.
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The FDA “Animal Rule” allows approval of therapeutic interventions in cases where there is a risk of severe disease and a controlled human trial would be unethical or infeasible. ​ ● Ratutrelvir (“TRX01”), which we acquired as part of the Merger, is an inhibitor of the main protease (also known as 3CL protease) of the SAR-CoV-2 virus, the causative agent in COVID19.
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Changes in operating assets and liabilities resulted in a net increase in cash of $1.5 million.
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The main protease is an essential component in the mechanism for SARS-CoV-2 replication. TRX01 is intended to inhibit this protease and reduce SARS-CoV-2 virus replication.
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Deferred revenue decreased $0.2 million due to recognition of the unamortized portion of the upfront payment under our collaboration agreement with SymBio. Net cash used in investing activities Net cash used in investing activities was $14,000 in the 2023 period related to information technology asset purchases. There was no net cash used in investing activities in the 2022 period.
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In vitro laboratory tests that measured the impact of TXR01 on SARS-CoV-2 replication, demonstrated that TRX01 inhibited the replication of viral isolates of the original SARS-CoV-2 isolates, and viral variants in the delta and omicron types.
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We believe that our cash and cash equivalents will be sufficient to fund our ongoing trials and operations into the third quarter of 2024; however, based on current projections, we do not have sufficient cash and cash equivalents as of the date of this Annual Report on Form 10-K to support our operations for at least the 12 months 56 Table of Contents following the date that the financial statements are issued.
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An animal study using the widely adopted K18 transgenic mouse model, demonstrated non-inferiority between TRX01 and the combination of nirmatrelvir + ritonavir, in terms of time to death and lung virus burden in this highly lethal model with neurological manifestations.
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Please see “Risk Factors” for additional risks associated with our substantial capital requirements. Segment Reporting We view our operations and manage our business in one segment, which is the identification and development of oncology therapeutics. Recent Accounting Pronouncements In June 2016, the Financial Accounting Standards Board (FASB) issued new guidance on the accounting for credit losses on financial instruments.
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Based on preclinical pharmacokinetic studies in multiple animal species, we intend to develop TRX01 for use without co-administration of a human cytochrome P450 (“CYP”) inhibitor such as ritonavir. ​ TRX01 w as studied in a Phase 1 clinical trial that included single and multiple ascending dose phases. Participants were required to be healthy males or females ages 18-64 years.
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The guidance was amended in November 2019. The new guidance introduces an expected loss model for estimating credit losses, replacing the incurred loss model. The new guidance also changes the impairment model for available-for-sale debt securities, requiring the use of an allowance to record estimated credit losses (and subsequent recoveries).
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The primary endpoint of the study was the measurement of safety and tolerability, and the secondary endpoint included the determination of the drug pharmacokinetic and pharmacodynamic profiles. The Phase 1 trial was conducted in Australia. It was sponsored by the Company and was approved by the Human Research Ethics Committee.

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