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What changed in Bio Green Med Solution, Inc.'s 10-K — 2023 vs 2024

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Paragraph-level year-over-year comparison of Bio Green Med Solution, 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.

+457 added−452 removedSource: 10-K (2025-04-02) vs 10-K (2024-03-21)

Top changes in Bio Green Med Solution, Inc.'s 2024 10-K

457 paragraphs added · 452 removed · 320 edited across 6 sections

Item 1. Business

Business — how the company describes what it does

75 edited+24 added−87 removed150 unchanged
Biggest changeThe aim of the current streamlined studies is to assess safety and identify signals of clinical activity which may lead to registration-enabling outcomes. 5 Table of Contents The following table summarizes our current development programs: PROGRAM INDICATION PHASE Transcriptional Regulation Fadraciclib CDK inhibitor (oral) Solid tumors – multiple cohorts defined by tumor histology and a basket cohort Phase 1/2 to achieve proof of concept Mitosis Regulation Plogosertib PLK inhibitor (oral) Solid tumors – multiple cohorts defined by tumor histology and a basket cohort Phase 1/2 to achieve proof of concept # CDK: cyclin-dependent kinase; PLK: polo-like kinase. # Study to resume recruitment following introduction of new oral formulation.
Biggest changeThe aim of the current streamlined studies is to assess safety and identify signals of clinical activity which may lead to registration-enabling outcomes. ​ 5 Table of Contents The following table summarizes our development programs: PROGRAM INDICATION PHASE Mitosis Regulation Plogosertib PLK* inhibitor (oral) Solid tumors – multiple cohorts defined by tumor histology and a basket cohort Phase 1/2 to achieve proof of concept # ​ * PLK: polo-like kinase. # Study to resume recruitment following introduction of new oral formulation. ​ We currently retain all global marketing rights to the compounds associated with our clinical-stage drug program. ​ Mitosis Regulation Program Polo-Like-Kinase inhibitor — Plogosertib In our Polo-like Kinase, or PLK, inhibitor program, we have discovered potent and selective small molecule inhibitors of PLK1.
In the future, we intend to apply for restorations of patent term for some of our currently owned or licensed patents to add patent life beyond their current expiration date, depending on the expected length of clinical trials and and other factors involved in the submission of the relevant NDA.
In the future, we intend to apply for restorations of patent term for some of our currently owned or licensed patents to add patent life beyond their current expiration date, depending on the expected length of clinical trials and other factors involved in the submission of the relevant NDA.
Accelerated assessment might be granted by the CHMP in exceptional cases, when a medicinal product is expected to be of a major public health interest, particularly from the point of view of therapeutic innovation. The timeframe for the evaluation of an MAA under the accelerated assessment procedure is of 150 days, excluding stop-clocks.
Accelerated assessment might be granted by the CHMP in exceptional cases, when a medicinal product is expected to be of a major public health interest, particularly from the point of view of therapeutic innovation. The timeframe for the evaluation of an MAA under the accelerated assessment procedure is 150 days, excluding stop-clocks.
These changes include aggregate reductions to Medicare payments to providers of up to 2% per fiscal year pursuant to the Budget Control Act of 2011, which began in 2013 and was extended by the Consolidated Appropriations Act for 2023, and will remain in effect through 2032 unless additional Congressional action is taken. 24 Table of Contents Moreover, there has been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products.
These changes include aggregate reductions to Medicare payments to providers of up to 2% per fiscal year pursuant to the Budget Control Act of 2011, which began in 2013 and was extended by the Consolidated Appropriations Act for 2023 and will remain in effect through 2032 unless additional Congressional action is taken. 20 Table of Contents Moreover, there has been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products.
Item 1. Busin ess The following Business Section contains forward-looking statements. Our actual results could differ materially from those anticipated in these forward-looking statements as a result of certain risks, uncertainties and other factors including the risk factors set forth in Part I, Item 1A of this Annual Report on Form 10-K.
Item 1. Business The following Business Section contains forward-looking statements. Our actual results could differ materially from those anticipated in these forward-looking statements as a result of certain risks, uncertainties and other factors including the risk factors set forth in Part I, Item 1A of this Annual Report on Form 10-K.
Such conditional approvals may be granted for product candidates (including medicines designated as orphan medicinal products) if (1) the risk-benefit balance of the product candidate is positive, (2) it is likely that the applicant will be in a position to provide the required comprehensive clinical trial data, (3) the product fulfills unmet medical needs and (4) the benefit to public health of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required.
Such conditional approvals may be granted for product candidates (including medicines 18 Table of Contents designated as orphan medicinal products) if (1) the risk-benefit balance of the product candidate is positive, (2) it is likely that the applicant will be in a position to provide the required comprehensive clinical trial data, (3) the product fulfills unmet medical needs and (4) the benefit to public health of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required.
Plogosertib is a novel, small molecule, selective, PLK1 inhibitor which has demonstrated an epigenetic mechanism, potent and selective target inhibition (PLK1 IC50 approximately 3 nM) and impressive efficacy in human tumor xenografts at non-toxic doses. Plogosertib has improved pharmaceutical properties over earlier, clinical stage, PLK inhibitors.
Plogo is a novel, small molecule, selective, PLK1 inhibitor which has demonstrated an epigenetic mechanism, potent and selective target inhibition (PLK1 IC50 approximately 3 nM) and impressive efficacy in human tumor xenografts at non-toxic doses. Plogo has improved pharmaceutical properties over earlier, clinical stage, PLK inhibitors.
In a cell line panel derived from esophageal cancer and various non-malignant solid tissues, plogosertib was preferentially cytotoxic to malignant cells. Malignant cells which are sensitive to plogosertib undergo complete growth inhibition and induction of cell death in response to treatment. In contrast, non-malignant cells are only temporarily arrested and normal cell cycle transit is restored.
In a cell line panel derived from esophageal cancer and various non-malignant solid tissues, plogo was preferentially cytotoxic to malignant cells. Malignant cells which are sensitive to plogo undergo complete growth inhibition and induction of cell death in response to treatment. In contrast, non-malignant cells are only temporarily arrested and normal cell cycle transit is restored.
Regulatory approval in one country or jurisdiction does not ensure regulatory approval in another, but a failure or delay in obtaining regulatory approval in one country or jurisdiction may negatively impact the regulatory process in others. European Union drug development, review and approval In the European Union, our product candidates also may be subject to extensive regulatory requirements.
Regulatory approval in one country or jurisdiction does not ensure regulatory approval in another, but a failure or delay in obtaining regulatory approval in one country or jurisdiction may negatively impact the regulatory process in others. European Union drug development, review and approval In the European Union, our product candidate also may be subject to extensive regulatory requirements.
The ten-year market exclusivity in the European Union may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, for example, if the product is sufficiently profitable not to justify maintenance of market exclusivity.
The ten-year market exclusivity in the European Union may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, for example, if the product is sufficiently profitable not justifying maintenance of market exclusivity.
The PREA requires a sponsor that is planning to submit a marketing application for a product that includes a new active ingredient, new indication, new dosage form, new dosing regimen or new route of administration to submit an initial Pediatric Study Plan, or PSP, within sixty days of an end-of-Phase 2 meeting or, if there is no such meeting, as early as 16 Table of Contents practicable before the initiation of the Phase 3 or Phase 2/3 clinical trial.
The PREA requires a sponsor that is planning to submit a marketing application for a product that includes a new active ingredient, new indication, new dosage form, new dosing regimen or new route of administration to submit an initial Pediatric Study Plan, or PSP, within sixty days of an end-of-Phase 2 meeting or, if there is no such meeting, as early as practicable before the initiation of the Phase 3 or Phase 2/3 clinical trial.
The process required by the FDA before our drug candidates may be marketed in the United States generally involves the following: ● completion of extensive nonclinical laboratory tests, which may include animal studies and formulation studies, all performed in accordance with the FDA’s good laboratory practice, or GLP, regulations; ● submission to the FDA of an investigational new drug application, or IND, which must become effective before clinical trials may begin and must be updated annually or when significant changes are made; ● approval by an IRB or ethics committee at each clinical site before the trial is initiated at such sites; ● performance of adequate and well-controlled clinical trials in accordance with good clinical practice, or GCP, and other clinical-trial related regulations to establish the safety and efficacy of the drug candidate for each proposed indication; ● preparation and submission of a new drug application, or NDA, to the FDA; ● a determination by the FDA within 60 days of its receipt of an NDA to file the application for review; ● satisfactory completion of an FDA Advisory Committee review, if applicable; ● satisfactory completion of an FDA pre-approval inspection of the manufacturing facilities at which the product is produced to assess compliance with current good manufacturing practice requirements, or cGMP, regulations; ● potential audit of selected clinical trial sites to assess compliance with GCP and the integrity of the clinical data submitted in support of the NDA; and ● FDA review and approval of the NDA to permit commercial marketing of the drug product for particular approved indications for use in the United States.
The process required by the FDA before our drug candidates may be marketed in the United States generally involves the following: · completion of extensive nonclinical laboratory tests, which may include animal studies and formulation studies, all performed in accordance with the FDA’s good laboratory practice, or GLP, regulations; · submission to the FDA of an investigational new drug application, or IND, which must become effective before clinical trials may begin and must be updated annually or when significant changes are made; · approval by an Institutional Review Board (“IRB”) or ethics committee at each clinical site before the trial is initiated at such sites; · performance of adequate and well-controlled clinical trials in accordance with good clinical practice, or GCP, and other clinical-trial related regulations to establish the safety and efficacy of the drug candidate for each proposed indication; ​ · preparation and submission of a new drug application (“NDA”), to the FDA; · a determination by the FDA within 60 days of its receipt of an NDA to file the application for review; 9 Table of Contents · satisfactory completion of an FDA Advisory Committee review, if applicable; · satisfactory completion of an FDA pre-approval inspection of the manufacturing facilities at which the product is produced to assess compliance with current good manufacturing practice requirements, or cGMP, regulations; · potential audit of selected clinical trial sites to assess compliance with GCP and the integrity of the clinical data submitted in support of the NDA; and · FDA review and approval of the NDA to permit commercial marketing of the drug product for particular approved indications for use in the United States.
The data demonstrated that plogosertib is a selective PLK1 inhibitor which is highly active against both solid and liquid cancer models, preferentially induces growth inhibition and cell death in malignant versus non-malignant cells.
The data demonstrated that plogo is a selective PLK1 inhibitor which is highly active against both solid and liquid cancer models, preferentially induces growth inhibition and cell death in malignant versus non-malignant cells.
Breakthrough therapy designation provides all the features of fast track 17 Table of Contents designation in addition to intensive guidance on an efficient development program beginning as early as Phase 1, and FDA organizational commitment to expedited development, including involvement of senior managers and experienced review and regulatory staff in a proactive, collaborative, cross-disciplinary review, where appropriate.
Breakthrough therapy designation provides all the features of fast track designation in addition to intensive guidance on an efficient development program beginning as early as Phase 1, and FDA organizational commitment to expedited development, including involvement of senior managers and experienced review and regulatory staff in a proactive, collaborative, cross-disciplinary review, where appropriate.
There is fierce competition both within our industry and in the geographic locations in which we have offices for highly skilled talent, and we offer a robust set of benefits, career-enhancing learning experiences and initiatives aligned with our mission, vision, and values in order to attract qualified prospective employees and to retain and motivate our employees.
There is fierce competition both within our industry and in the geographic locations in which we have offices for highly skilled talent, and we offer a robust set of benefits, career-enhancing learning experiences and initiatives aligned with our 22 Table of Contents mission, vision, and values in order to attract qualified prospective employees and to retain and motivate our employees.
Treatment of proliferating cells with plogosertib resulted in reduced phosphorylation of the PLK1 substrate phospho-nucleophosmin, accumulation of cells in mitosis and an increase in the proportion of mitotic cells with monopolar spindles, which are all features consistent with PLK1 inhibition.
Treatment of proliferating cells with plogo resulted in reduced phosphorylation of the PLK1 substrate phospho-nucleophosmin, accumulation of cells in mitosis and an increase in the proportion of mitotic cells with monopolar spindles, which are all features consistent with PLK1 inhibition.
The manufacturing facilities for our product candidates must meet applicable cGMP requirements to the FDA's or comparable foreign regulatory authorities' satisfaction before any product is approved and our commercial products can be manufactured.
The manufacturing facilities for our product candidate must meet applicable cGMP requirements to the FDA's or comparable foreign regulatory authorities' satisfaction before any product is approved and our commercial products can be manufactured.
This testing and approval process requires substantial time, effort and financial resources, and we cannot be certain that any approvals for our drug candidates will be granted on a timely basis, if at all. Preclinical development Before testing any drug product candidate, including our product candidates, in humans, the product candidate must undergo rigorous preclinical testing.
This testing and approval process requires substantial time, effort and financial resources, and we cannot be certain that any approvals for our drug candidates will be granted on a timely basis, if at all. Preclinical development Before testing any drug product candidate, including plogo, our sole current product candidate, in humans, the product candidate must undergo rigorous preclinical testing.
Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors. 25 Table of Contents There have been, and likely will continue to be, legislative and regulatory proposals at the foreign, federal and state levels directed at broadening the availability of healthcare and containing or lowering the cost of healthcare.
Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors. There have been, and likely will continue to be, legislative and regulatory proposals at the foreign, federal and state levels directed at broadening the availability of healthcare and containing or lowering the cost of healthcare.
Health Care Reform The FDA’s and other regulatory authorities’ policies may change, and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidates.
Health Care Reform The FDA’s and other regulatory authorities’ policies may change, and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidate.
Some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines, or the relevant compliance guidance promulgated by the 20 Table of Contents federal government, in addition to requiring drug manufacturers to report information related to payments to physicians and other health care providers or marketing expenditures to the extent that those laws impose requirements that are more stringent than the Physician Payments Sunshine Act.
Some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines, or the relevant compliance guidance promulgated by the federal government, in addition to requiring drug manufacturers to report information related to payments to physicians and other health care providers or marketing expenditures to the extent that those laws impose requirements that are more stringent than the Physician Payments Sunshine Act.
We are aware of several published patent applications, and understand that others may exist, that could support claims that, if granted and held valid, would cover various aspects of our developmental programs, including in some cases particular uses of our drug candidates fadraciclib and plogosertib, or other therapeutic candidates, or substances, processes and techniques that we use in the course of our research and development and manufacturing operations.
We are aware of several published patent applications, and understand that others may exist, that could support claims that, if granted and held valid, would cover various aspects of our developmental programs, including in some cases particular uses of our drug candidate plogo, or other therapeutic candidates, or substances, processes and techniques that we use in the course of our research and development and manufacturing operations.
In the case of some products for severe or life-threatening diseases, such as cancer, especially when the product may be too inherently toxic to ethically administer to healthy volunteers, the initial human testing is often conducted in patients.
In the case of some 10 Table of Contents products for severe or life-threatening diseases, such as cancer, especially when the product may be too inherently toxic to ethically administer to healthy volunteers, the initial human testing is often conducted in patients.
An adverse outcome in litigation could subject us to significant liabilities to third parties and require us to seek licenses of the disputed rights from third parties or to cease using the technology, even a therapeutic product, if such licenses are unavailable or too expensive. Issued patents for fadraciclib cover the United States, EPO and eleven other countries.
An adverse outcome in litigation could subject us to significant liabilities to third parties and require us to seek licenses of the disputed rights from third parties or to cease using the technology, even a therapeutic product, if such licenses are unavailable or too expensive. Issued patents for plogo cover the United States, EPO and six other countries.
Drugs and biologics granted accelerated approval must meet the same statutory standards for safety and effectiveness as those granted traditional approval. The accelerated approval pathway is usually contingent on a sponsor’s agreement to conduct, in a diligent manner, additional post-approval confirmatory studies to verify and describe the product candidate’s clinical benefit.
Drugs and biologics granted accelerated approval must meet the same statutory standards for safety and effectiveness as those granted traditional approval. 13 Table of Contents The accelerated approval pathway is usually contingent on a sponsor’s agreement to conduct, in a diligent manner, additional post-approval confirmatory studies to verify and describe the product candidate’s clinical benefit.
Such reforms could have an adverse effect on anticipated revenue from product candidates that we may successfully develop and for which we may obtain regulatory approval and may affect our overall financial condition and ability to develop product candidates. Competition The biotechnology and biopharmaceutical industries are rapidly changing and highly competitive.
Such reforms could have an adverse effect on anticipated revenue from plogo and any of our future product candidates that we may successfully develop and for which we may obtain regulatory approval and may affect our overall financial condition and ability to develop product candidates. Competition The biotechnology and biopharmaceutical industries are rapidly changing and highly competitive.
If competitors prepare and file patent applications in the United States that claim technology that we also claim, we may have to participate in interference proceedings in the United States Patent and Trademark Office to determine which invention has priority. These proceedings could result in substantial costs, even if the eventual outcome is favorable to us.
If 8 Table of Contents competitors prepare and file patent applications in the United States that claim technology that we also claim, we may have to participate in interference proceedings in the United States Patent and Trademark Office to determine which invention has priority. These proceedings could result in substantial costs, even if the eventual outcome is favorable to us.
Along with our third-party contractors, we will be required to navigate the various preclinical, clinical and commercial approval requirements of the governing regulatory agencies of the countries in which we wish to conduct studies or seek approval or licensure of its current or future product candidates.
Along with our third-party contractors, we will be required to navigate the various preclinical, clinical and commercial approval requirements of the governing regulatory agencies of the countries in which we wish to conduct studies or seek approval or licensure of plogo or any of its product candidates.
In addition, we understand that other applications and patents exist relating to potential uses of fadraciclib and plogosertib which are not part of our current clinical programs for those compounds.
In addition, we understand that other applications and patents exist relating to potential uses of plogo which are not part of our current clinical programs for those compounds.
We rely, and expect to continue to rely, on third parties for the production of clinical (and 19 Table of Contents ultimately commercial) quantities of our products in accordance with cGMP regulations.
We rely, and expect to continue to rely, on third parties for the production of clinical (and ultimately commercial) quantities of our products in accordance with cGMP regulations.
Part II, which contains the national and patient-level documentation, will be assessed individually by each EU Member State. Strict deadlines have been established for the assessment of clinical trial applications. The role of the relevant ethics committees in the assessment procedure will continue to be governed by the national law of the concerned EU Member State.
Part II, which contains the national and patient-level documentation, will be assessed individually by each EU Member State. Strict deadlines have been established for the assessment of clinical trial applications. The role of the relevant ethics committees in the assessment procedure will continue to be governed by the national law of 17 Table of Contents the competent EU Member State.
According to the amended language, a sponsor may fulfill nonclinical testing requirements by completing various in 14 Table of Contents vitro assays (e.g., cell-based assays, organ chips, or microphysiological systems), in silico studies (i.e., computer modeling), other human or nonhuman biology-based tests (e.g., bioprinting), or in vivo animal tests.
According to the amended language, a sponsor may fulfill nonclinical testing requirements by completing various in vitro assays (e.g., cell-based assays, organ chips, or micro-physiological systems), in silico studies (i.e., computer modeling), other human or nonhuman biology-based tests (e.g., bioprinting), or in vivo animal tests.
Our translational biology program supports the development of plogosertib in solid tumor and hematological malignancy indications. Clinical development Phase 1/2 Study in advanced solid tumors and lymphomas (CYC140-101, orally dosed) Similar to fadraciclib, this ongoing open-label Phase 1/2 registration-directed trial uses a streamlined design and seeks to first determine in a dose escalation stage the RP2D for single-agent plogosertib.
Our translational biology program supports the development of plogo in solid tumor and hematological malignancy indications. ​ Clinical development Phase 1/2 Study in advanced solid tumors and lymphomas (CYC140-101, orally dosed) This open-label Phase 1/2 registration-directed study uses a streamlined design and initially seeks to determine the RP2D for single-agent oralplogo in a dose escalation stage.
We are a pioneer company in the field of cancer cell cycle biology with a vision to improve patient healthcare by translating insights in cancer biology into medicines that can overcome resistance and ultimately increase a patient’s overall survival. The transcriptional regulation program is evaluating fadraciclib, a CDK2/9 inhibitor, in solid tumors and hematological malignancies.
We are a pioneer company in the field of cancer cell cycle biology with a vision to improve patient healthcare by translating insights in cancer biology into medicines that can overcome resistance and ultimately increase a patient’s overall survival. The epigenetic/anti-mitotic program is evaluating plogo, a PLK1 inhibitor, in solid tumors and hematological malignancies.
Phase 1 clinical trials can be designed to evaluate the impact of the drug candidate in combination with currently approved drugs. ● Phase 2: These clinical trials are generally conducted in a limited patient population to identify possible adverse effects and safety risks, to evaluate preliminary efficacy of the drug candidate for specific targeted indications and to determine dose tolerance and optimal dosage.
Phase 2: These clinical trials are generally conducted in a limited patient population to identify possible adverse effects and safety risks, to evaluate preliminary efficacy of the drug candidate for specific targeted indications and to determine dose tolerance and optimal dosage.
The protocol allows for expansion of individual cohorts based on response which may allow acceleration of the clinical development and registration plan for plogosertib. Fifteen patients have been treated at the first five dose escalation levels with no dose limiting toxicities observed.
The protocol allows for expansion of individual cohorts based on response which may allow acceleration of the clinical development and registration plan for plogo. ​ Fifteen patients have been treated at the first five dose escalation levels with no dose limiting toxicities observed. Stable disease has been observed in pretreated patients with gastrointestinal, lung, and ovarian cancers.
We have no direct experience in manufacturing commercial quantities of any of our products, and we currently lack the resources or capability to manufacture any of our products on a clinical or commercial scale.
Manufacturing We have no in-house manufacturing capabilities and have no current plans to establish manufacturing facilities for significant clinical or commercial production. We have no direct experience in manufacturing commercial quantities of any of our products, and we currently lack the resources or capability to manufacture any of our products on a clinical or commercial scale.
Other potential consequences include, among other things: ● restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls; ● fines, warning letters or other enforcement-related letters or clinical holds on post-approval clinical trials; ● refusal of the FDA to approve pending marketing applications or supplements to approved marketing authorizations, or suspension or revocation of product approvals; ● product seizure or detention, or refusal to permit the import or export of products; ● injunctions or the imposition of civil or criminal penalties; and ● consent decrees, corporate integrity agreements, debarment, or exclusion from federal health care programs; or mandated modification of promotional materials and labeling and the issuance of corrective information.
Other potential consequences include, among other things: · restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls; · fines, warning letters or other enforcement-related letters or clinical holds on post-approval clinical trials; · refusal of the FDA to approve pending marketing applications or supplements to approved marketing authorizations, or suspension or revocation of product approvals; · product seizure or detention, or refusal to permit the import or export of products; · injunctions or the imposition of civil or criminal penalties; and · consent decrees, corporate integrity agreements, debarment, or exclusion from federal health care programs; or mandated modification of promotional materials and labeling and the issuance of corrective information. ​ In addition, the distribution of prescription pharmaceutical products is subject to the Prescription Drug Marketing Act, or PDMA, which regulates the distribution of drugs and drug samples at the federal level and sets minimum standards for the registration and regulation of drug distributors by the states.
We expect that these and other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we receive for any approved drug, which could have an adverse effect on customers for our product candidates.
These measures could reduce the ultimate demand for our products, once approved, or put pressure on our product pricing. 21 Table of Contents We expect that these and other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we receive for any approved drug, which could have an adverse effect on customers for our product candidate.
In August 2022, the Inflation Reduction Act of 2022, or the IRA, was signed into law. Among other things, the IRA has multiple provisions that may impact the prices of drug products that are both sold into the Medicare program and throughout the United States.
Among other things, the IRA has multiple provisions that may impact the prices of drug products that are both sold into the Medicare program and throughout the United States.
In addition, the discovery of conditions that violate these rules, including failure to conform to cGMPs, could result in enforcement actions, and the discovery of problems with a product after approval may result in restrictions on a product, manufacturer or holder of an approved NDA, including voluntary recall and regulatory sanctions as described below.
In addition, the discovery of conditions that violate these rules, including failure to conform to cGMPs, could result in enforcement actions, and the discovery of problems with a product after approval may result in restrictions on a product, manufacturer or holder of an approved NDA, including voluntary recall and regulatory sanctions as described below. 15 Table of Contents Once an approval or clearance of a drug is granted, the FDA may withdraw the approval if compliance with regulatory requirements and standards is not maintained or if problems occur after the product reaches the market.
All suspected unexpected serious adverse reactions to the investigated drug that occur during the clinical trial have to be reported to the competent national authority and the Ethics Committee of the Member State where they occurred. 21 Table of Contents Under the EU’s new Clinical Trials Regulation, which took effect in January 2022, there will be a centralized application procedure where one EU Member State’s competent authority takes the lead in reviewing part I of the application, which contains scientific and medicinal product documentation, and the other national authorities only have limited involvement.
Under the EU’s new Clinical Trials Regulation, which took effect in January 2022, there will be a centralized application procedure where one EU Member State’s competent authority takes the lead in reviewing part I of the application, which contains scientific and medicinal product documentation, and the other national authorities only have limited involvement.
Published preclinical data Preclinical data presented at the 2016 28th EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium and at the 2017 Annual Meeting of the American Association of Cancer Research demonstrated the therapeutic potential of plogosertib as a targeted anti-cancer agent.
A new, alternative salt, oral formulation of plogo with improved bioavailability is under development. ​ 6 Table of Contents Published preclinical data ​ Preclinical data presented at the 2016 28th EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium and at the 2017 Annual Meeting of the American Association of Cancer Research demonstrated the therapeutic potential of plogo as a targeted anti-cancer agent.
We offer competitive compensation for our employees and strongly embrace a pay for performance philosophy in setting and adjusting compensation. 26 Table of Contents Our codes of conduct clearly outline our commitment to diversity and inclusion, where all employees are welcomed in an environment designed to make them feel comfortable, respected, and accepted regardless of their age, race, national origin, gender, religion, disability or sexual orientation.
Our codes of conduct clearly outline our commitment to diversity and inclusion, where all employees are welcomed in an environment designed to make them feel comfortable, respected, and accepted regardless of their age, race, national origin, gender, religion, disability or sexual orientation. We have a set of policies explicitly setting forth our expectations for nondiscrimination and a harassment-free work environment.
Cardiff Oncology has a PLK1 inhibitor in clinical trials and we believe that Arbutus, Boehringer Ingelheim, GlaxoSmithKline, Merck, Onconova, and Takeda have been and may continue to be evaluating PLK inhibitors for hemato-oncology indications. Several companies are pursuing discovery and research activities in each of the other areas that are the subject of our research and drug development programs.
Cardiff Oncology has a PLK1 inhibitor in clinical trials and we believe that Arbutus, Boehringer Ingelheim, GlaxoSmithKline, Merck, Onconova, and Takeda have been and may continue to be evaluating PLK inhibitors for hemato-oncology indications.
In addition to composition of matter claims, we seek 12 Table of Contents coverage for solid state forms, polymorphic and crystalline forms, medical uses, combination therapies, specific regimens, pharmaceutical forms of our compounds and synthetic routes where available and appropriate.
We prefer composition of matter claims because they provide us with rights to the compounds themselves, and not merely a particular use. In addition to composition of matter claims, we seek coverage for solid state forms, polymorphic and crystalline forms, medical uses, combination therapies, specific regimens, pharmaceutical forms of our compounds and synthetic routes where available and appropriate.
The FDA reviews all NDAs submitted to determine if they are substantially complete before it accepts them for filing and may request additional information rather than accepting a submission for filing.
FDA approval of an NDA must be obtained before the corresponding drug may be marketed in the United States. 11 Table of Contents The FDA reviews all NDAs submitted to determine if they are substantially complete before it accepts them for filing and may request additional information rather than accepting a submission for filing.
Data can come from company-sponsored clinical studies intended to test the safety and effectiveness of a use of the product, or from a number of alternative sources, including studies initiated by investigators. FDA approval of an NDA must be obtained before the corresponding drug may be marketed in the United States.
Data can come from company-sponsored clinical studies intended to test the safety and effectiveness of a use of the product, or from a number of alternative sources, including studies initiated by investigators.
In addition, regional healthcare authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other healthcare programs. These measures could reduce the ultimate demand for our products, once approved, or put pressure on our product pricing.
In addition, regional healthcare authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other healthcare programs.
Even if an SPA is agreed to, approval of the NDA is not guaranteed because a final determination that an agreed-upon protocol satisfies a specific objective, such as the demonstration of efficacy, or supports an approval decision, will be based on a complete review of all the data in the NDA. 18 Table of Contents Orphan Drugs Under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug intended to treat a rare disease or condition, defined as a disease or condition with a patient population of fewer than 200,000 individuals in the United States, or a patient population greater than 200,000 individuals in the United States and when there is no reasonable expectation that the cost of developing and making available the drug in the United States will be recovered from sales in the United States for that drug.
Orphan Drugs Under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug intended to treat a rare disease or condition, defined as a disease or condition with a patient population of fewer than 200,000 individuals in the United States, or a patient population greater than 200,000 individuals in the United States and when there is no reasonable expectation that the cost of developing and making available the drug in the United States will be recovered from sales in the United States for that drug.
Under the above described procedures, before granting the marketing authorization, the EMA or the competent authorities of the Member States of the EEA make an assessment of the risk-benefit balance of the product on the basis of scientific criteria concerning its quality, safety and efficacy. 22 Table of Contents Conditional approval In specific circumstances, E.U. legislation (Article 14(7) Regulation (EC) No 726/2004 and Regulation (EC) No 507/2006 on Conditional Marketing Authorizations for Medicinal Products for Human Use) enables applicants to obtain a conditional marketing authorization prior to obtaining the comprehensive clinical data required for an application for a full marketing authorization.
Conditional approval In specific circumstances, E.U. legislation (Article 14(7) Regulation (EC) No 726/2004 and Regulation (EC) No 507/2006 on Conditional Marketing Authorizations for Medicinal Products for Human Use) enables applicants to obtain a conditional marketing authorization prior to obtaining the comprehensive clinical data required for an application for a full marketing authorization.
Additionally, marketing authorization may be granted to a similar product for the same indication at any time if: ● the second applicant can establish that its product, although similar, is safer, more effective or otherwise clinically superior; 23 Table of Contents ● the applicant consents to a second orphan medicinal product application; or ● the applicant cannot supply enough orphan medicinal product.
Additionally, marketing authorization may be granted to a similar product for the same indication at any time if: · the second applicant can establish that its product, although similar, is safer, more effective or otherwise clinically superior; · the applicant consents to a second orphan medicinal product application; or · the applicant cannot supply enough orphan medicinal product. 19 Table of Contents PRIME designation The EMA grants access to the Priority Medicines, or PRIME, program to investigational medicines for which it determines there to be preliminary data available showing the potential to address an unmet medical need and bring a major therapeutic advantage to patients.
Patent term restoration Depending upon the timing, duration and specifics of FDA approval of the use of our product candidates, some of our United States patents may be eligible for limited patent term extension under the Hatch-Waxman Act.
Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the NDA application user fee. 14 Table of Contents Patent term restoration Depending upon the timing, duration and specifics of FDA approval of the use of our product candidate, some of our United States patents may be eligible for limited patent term extension under the Hatch-Waxman Act.
As a result, we are dependent on corporate partners, licensees or other third parties for the manufacturing of clinical (and eventually commercial) scale quantities of all of our products.
As a result, we are dependent on corporate partners, licensees or other third parties for the manufacturing of clinical (and eventually commercial) scale quantities of all of our products. We believe that this strategy will enable us to direct operational and financial resources to the development of our product candidate rather than diverting resources to establishing a manufacturing infrastructure.
Clinical Development Pipeline Our pipeline of innovative medicines aims to provide safe and effective anticancer treatment options to patients combined with the convenience of oral administration. Our preclinical and clinical studies suggests that daily dosing by the oral route is a preferred strategy for both our drugs. We also conducted certain early clinical studies using i.v. administration.
Our preclinical and clinical studies suggests that daily dosing by the oral route is a preferred strategy for both our drugs. We also conducted certain early clinical studies using intravenous (“i.v.”) administration.
In addition, our senior management team has extensive experience in research, preclinical and clinical development and sales and marketing. The novel, mechanism-targeted cell cycle drugs we are developing are designed to be highly selective in comparison to conventional chemotherapies, potentially inducing death in cancer cells while sparing most normal cells which may give rise to fewer side-effects.
The novel, mechanism-targeted cell cycle drugs we are developing are designed to be highly selective in comparison to conventional chemotherapies, potentially inducing death in cancer cells while sparing most normal cells which may give rise to fewer side-effects. ​ Thus, we believe that we are well placed to exploit the significant opportunities that this area offers for new drug discovery and development.
In some cases, the FDA may condition approval of an NDA for a drug candidate on the sponsor’s agreement to conduct a Phase 4, which includes additional clinical trials to further assess the drug’s safety and effectiveness after NDA approval. 15 Table of Contents In the Consolidated Appropriations Act for 2023, Congress amended the FDCA to require sponsors of a Phase 3 clinical trial, or other “pivotal study” of a new drug to support marketing authorization, to submit a diversity action plan for such clinical trial.
In some cases, the FDA may condition approval of an NDA for a drug candidate on the sponsor’s agreement to conduct a Phase 4, which includes additional clinical trials to further assess the drug’s safety and effectiveness after NDA approval.
In this stage plogosertib will be administered to patients in up to seven mechanistically relevant cohorts including patients with bladder, breast, colorectal (including KRAS mutant), hepatocellular and biliary tract, and lung cancers (both small cell and non-small cell), as well as lymphomas. An additional basket cohort will enroll patients with biomarkers relevant to the drug’s mechanism, including MYC amplified tumors.
Once RP2D has been established, the study will enter into proof-of-concept, cohort stage, using a Simon 2-stage design. In this stage plogo will be administered to patients in up to seven mechanistically relevant cohorts including patients with bladder, breast, colorectal (including KRAS mutant), hepatocellular and biliary tract, and lung cancers (both small cell and non-small cell), as well as lymphomas.
Fast Track, Priority Review, and Breakthrough Therapy Designations A sponsor may seek approval of its product candidate under programs designed to accelerate FDA’s review and approval of new drugs and biological products that meet certain criteria.
In addition, new government requirements, including those resulting from new legislation, may be established, or the FDA’s policies may change, which could delay or prevent regulatory approval of our products under development. 12 Table of Contents Fast Track, Priority Review, and Breakthrough Therapy Designations A sponsor may seek approval of its product candidate under programs designed to accelerate FDA’s review and approval of new drugs and biological products that meet certain criteria.
State and foreign laws also govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
State and foreign laws also govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts. 16 Table of Contents Because of the breadth of these laws and the narrowness of their exceptions and safe harbors, it is possible that business activities can be subject to challenge under one or more of such laws.
As we do not operate laboratories or manufacture products, we believe that our environmental impact is relatively small. We are involved in office waste reduction practices. Our mostly remote workforce has further reduced our carbon footprint. We strive to offer excellent benefits and long-term incentives to help retain our workforce.
We support professional development at all levels. We also take reports of suspected violations of our codes of conduct and take seriously appropriate action. As we do not operate laboratories or manufacture products, we believe that our environmental impact is relatively small. We are involved in office waste reduction practices. Our mostly remote workforce has further reduced our carbon footprint.
Our human capital resources and objectives include identifying, recruiting, retaining and incentivizing our existing and additional employees.
We strive to offer excellent benefits and long-term incentives to help retain our workforce. Our human capital resources and objectives include identifying, recruiting, retaining and incentivizing our existing and additional employees.
Our breach of an existing license or failure to obtain a license to technology required to develop, test and commercialize our products may seriously harm our business. In March 2023 we terminated our license agreement with Daiichi Sankyo Co., Ltd. for patents and patent applications covering sapacitabine for commercial reasons.
Our breach of an existing license or failure to obtain a license to technology required to develop, test and commercialize our products may seriously harm our business.
Orphan product exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition. Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the NDA application user fee.
Orphan product exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition.
In the mutual recognition procedure, a medicine is first authorized in one EU Member State, in accordance with the national procedures of that country. Following this, further marketing authorizations can be sought from other EU countries in a procedure whereby the countries concerned agree to recognize the validity of the original, national marketing authorization.
In the mutual recognition procedure, a medicine is first authorized in one EU Member State, in accordance with the national procedures of that country.
Environmental Social and Government (“ESG”) Matters We recognize the importance of ESG matters, with a specific focus on Human Capital Management, as integral to creating a sustainable foundation for our long-term business strategy. We support professional development at all levels. We also take reports of suspected violations of our codes of conduct and take seriously appropriate action.
Several companies are pursuing discovery and research activities in each of the other areas that are the subject of our research and drug development program. ​ Environmental Social and Government (“ESG”) Matters We recognize the importance of ESG matters, with a specific focus on Human Capital Management, as integral to creating a sustainable foundation for our long-term business strategy.
We believe that this strategy will enable us to direct operational and financial resources to the development of our product candidates rather than diverting resources to establishing a manufacturing infrastructure. 13 Table of Contents Government Regulation The FDA and comparable regulatory agencies in state and local jurisdictions, as well as in foreign countries, impose substantial regulatory requirements upon the clinical development, manufacture, marketing and distribution of drugs.
Government Regulation The FDA and comparable regulatory agencies in state and local jurisdictions, as well as in foreign countries, impose substantial regulatory requirements upon the clinical development, manufacture, marketing and distribution of drugs.
Polo Kinases and other mitotic kinases were first discovered in fruit flies by our former Chief Scientist, Professor David Glover, PhD. PLK1 is a serine/threonine kinase playing a central role in cell division, or mitosis. In particular, PLK1 regulates mitotic entry, spindle formation, mitotic exit, cytokinesis and is an important regulator of the DNA damage checkpoint.
PLK1 is a serine/threonine kinase playing a central role in cell division, or mitosis. In particular, PLK1 regulates mitotic entry, spindle formation, mitotic exit and cytokinesis and is an important regulator of the DNA damage checkpoint. Cancer cells are much more sensitive to PLK1 depletion than normal cells with intact cell cycle checkpoints.
Notably, on December 20, 2019, the Further Consolidated Appropriations Act for 2020 was signed into law (P.L. 116-94) that includes a piece of bipartisan legislation called the Creating and Restoring Equal Access to Equivalent Samples Act of 2019 (the CREATES Act).
Notably, on December 20, 2019, the Further Consolidated Appropriations Act for 2020 was signed into law (P.L. 116-94) that includes a piece of bipartisan legislation called the Creating and Restoring Equal Access to Equivalent Samples Act of 2019 (the “CREATES Act”), which provides a legislatively defined private right of action under which eligible product developers can bring suit against companies who refuse to sell sufficient quantities of their branded products on commercially reasonable, market-based terms to support such eligible product developers’ marketing applications.
Cancer cells are much more sensitive to PLK1 depletion than normal cells with intact cell cycle checkpoints. Inhibiting PLK1 blocks proliferation by prolonged mitotic arrest followed by onset of cancer cell death. The lead drug in our anti-mitotic program is plogosertib (formerly known as CYC140).
Inhibiting PLK1 blocks proliferation by prolonged mitotic arrest followed by onset of cancer cell death. The lead drug in our anti-mitotic program is plogosertib, or plogo (formerly known as CYC140). Clinical Development Pipeline Our pipeline of innovative medicines aims to provide safe and effective anticancer treatment options to patients combined with the convenience of oral administration.
Intellectual Property Strategy We consider intellectual property rights to be vital and use a variety of methods to secure, protect and evaluate these rights. These methods include ownership and enforcement of patent rights, patent applications, license agreements with third parties, invention assignment, confidentiality and non-compete agreements with key employees and consultants, material transfer agreements, and trademark protection.
These methods include ownership and enforcement of patent rights, patent applications, license agreements with third parties, invention assignment, confidentiality and non-compete agreements with key employees and consultants, material transfer agreements, and trademark protection. We give priority to obtaining substance of matter claims in the United States, the EPO, Japan and other important markets if such protection is available.
The DSCSA mandates phased-in and resource-intensive obligations for pharmaceutical manufacturers, wholesale distributors, and dispensers over a 10-year period that is expected to culminate in November 2023. From time to time, new legislation and regulations may be implemented that could significantly change the statutory provisions governing the approval, manufacturing and marketing of products regulated by the FDA.
Since then, the deadline has been pushed further with an additional year into 2025. The DSCSA’s new 2025 deadline for manufacturers and repackagers is May 27, 2025. From time to time, new legislation and regulations may be implemented that could significantly change the statutory provisions governing the approval, manufacturing and marketing of products regulated by the FDA.
Patents and Proprietary Technology Patents and Proprietary Rights We own 14 patents granted in the United States, 5 granted by the European Patent Office, or EPO, and 30 granted in other countries worldwide. In addition, we have a license to 11 patents granted in the US, by the EPO or worldwide.
We owned 2 patent applications filed in the United States, 2 filed at the EPO and 16 filed in other countries worldwide. As of March 26, 2025, we have 2 patents granted in the United States, 1 granted by the European Patent Office, or EPO, and 6 granted in other countries worldwide.
We have retained rights to commercialize our clinical development candidates and our business objective is to enter into selective partnership arrangements with these programs. Substantially all our efforts to date have been devoted to performing research and development, conducting clinical trials, developing and acquiring intellectual property, raising capital and recruiting and training personnel.
Our strategy is to build a diversified biopharmaceutical business based on a pipeline of novel drug candidates addressing oncology and hematology indications. We have retained rights to commercialize our clinical development candidates and our business objective is to enter into selective partnership arrangements with these programs.
In this report, “Cyclacel,” the “Company,” “we,” “us,” and “our” refer to Cyclacel Pharmaceuticals, Inc. General We are a clinical-stage biopharmaceutical company working to develop innovative cancer medicines based on cell cycle, transcriptional regulation, epigenetics and mitosis biology.
The deconsolidation, which is anticipated to increase stockholders’ equity by approximately $5.0 million, will be reported in the Company’s Form 10-Q for the three months ended March 31, 2025. General We are a clinical-stage biopharmaceutical company working to develop innovative cancer medicines based on cell cycle, epigenetics and mitosis biology.
Removed
The epigenetic/anti-mitotic program is evaluating plogosertib, a PLK1 inhibitor, in solid 4 Table of Contents tumors and hematological malignancies. Our strategy is to build a diversified biopharmaceutical business based on a pipeline of novel drug candidates addressing oncology and hematology indications.
Added
In this report, “Cyclacel,” the “Company,” “we,” “us,” and “our” refer to Cyclacel Pharmaceuticals, Inc. ​ Recent Developments ​ In December 2024 the Company announced that it was in the process of exploring and reviewing strategic alternatives on an expedited basis in order to preserve the Company’s cash, including a potential transaction with investor David Lazar of Activist Investing, LLC, or “Lazar”.
Removed
Cell Cycle Control Biology Loss of control of the cell cycle, the process by which cells grow and divide, lies at the heart of cancer. In normal cells, a complex set of interacting proteins tightly regulates progression through the phases of the cell cycle by which a cell grows, replicates its DNA and divides.
Added
The Company’s Board of Directors reviewed a range of appropriate strategies to realize value from its assets. The Board directed management to reduce operating costs, which included the liquidation of the Company’s wholly owned United Kingdom subsidiary Cyclacel Limited, or Subsidiary, while such alternatives were being explored.
Removed
This process also includes mechanisms known as cell cycle checkpoints, to ensure all necessary events of each cell cycle phase are completed before beginning the next phase. Specific isoforms of cyclin dependent kinases, or CDKs, and Polo-like Kinases, or PLKs, are some of the key regulators among the numerous genes and proteins involved in cell cycle checkpoints.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeFailure can occur at any stage of the testing and we may experience numerous unforeseen events during, or as a result of, the 27 Table of Contents clinical trial process that could delay or prevent commercialization of our current or future drug candidates, including, but not limited to: ● delays in securing clinical investigators or trial sites for our clinical trials; ● delays in obtaining institutional review board, or IRB, and regulatory approvals to commence a clinical trial; ● failure to obtain regulatory authority permission to conduct a clinical trial, after review of an investigational new drug or equivalent foreign application or amendment; ● slower than anticipated rates of subject recruitment and enrollment, or not reaching the targeted number of subjects because of competition for patients from other trials; ● negative or inconclusive results from clinical trials, as demonstrated by our announcement on February 24, 2017 that our SEAMLESS Phase 3 study failed to reach its primary endpoint; ● inability to generate satisfactory preclinical or other nonclinical data, including, toxicology, or other in vivo or in vitro data or diagnostics to support the initiation or continuation of clinical trials; ● unforeseen safety issues; ● failure by clinical sites or contract research organizations, or CROs, or other third parties to adhere to clinical trial requirements, GCP, or other applicable regulatory requirements; ● subjects discontinuing participating in our clinical trials at a greater than expected rate; ● imposition by the FDA of a clinical hold or the requirement by other similar regulatory agencies that one or more clinical trials be delayed or halted; ● uncertain dosing issues that may or may not be related to incompletely explored pharmacokinetic and pharmacodynamics behaviors; ● approval and introduction of new therapies or changes in standards of practice or regulatory guidance that render our clinical trial endpoints or the targeting of our proposed indications less attractive; ● inability to monitor patients adequately during or after treatment or problems with investigator or patient compliance with the trial protocols; ● inability to replicate in large, controlled studies safety and efficacy data obtained from a limited number of patients in uncontrolled trials; ● the ultimate affordability of the cost of clinical trials of our product candidates; ● changes in regulatory requirements and guidance that require amending or submitting new clinical protocols or performing additional nonclinical studies; and ● unavailability of clinical trial supplies.
Biggest changeThe success of our product candidate will depend on several factors, including, but not limited to: ● delays in securing clinical investigators or trial sites for our clinical trials; ● delays in obtaining institutional review board, or IRB, and regulatory approvals to commence a clinical trial; ● failure to obtain regulatory authority permission to conduct a clinical trial, after review of an investigational new drug or equivalent foreign application or amendment; ● slower than anticipated rates of subject recruitment and enrollment, or not reaching the targeted number of subjects because of competition for patients from other trials; ● negative or inconclusive results from clinical trials, as demonstrated by our announcement on February 24, 2017 that our SEAMLESS Phase 3 study failed to reach its primary endpoint; ● inability to generate satisfactory preclinical or other nonclinical data, including, toxicology, or other in vivo or in vitro data or diagnostics to support the initiation or continuation of clinical trials; ● unforeseen safety issues; ● failure by clinical sites or contract research organizations, or CROs, or other third parties to adhere to clinical trial requirements, GCP, or other applicable regulatory requirements; 23 Table of Contents ● subjects discontinuing participating in our clinical trials at a greater than expected rate; ● imposition by the FDA of a clinical hold or the requirement by other similar regulatory agencies that one or more clinical trials be delayed or halted; ● uncertain dosing issues that may or may not be related to incompletely explored pharmacokinetic and pharmacodynamics behaviors; ● approval and introduction of new therapies or changes in standards of practice or regulatory guidance that render our clinical trial endpoints or the targeting of our proposed indications less attractive; ● inability to monitor patients adequately during or after treatment or problems with investigator or patient compliance with the trial protocols; ● inability to replicate in large, controlled studies safety and efficacy data obtained from a limited number of patients in uncontrolled trials; ● the ultimate affordability of the cost of clinical trials of our product candidate; ● effectively launching commercial sales of our product candidate, if approved, whether alone or in collaboration with others; ● achieving acceptance of our product candidate, if approved, by patients, the medical community and third-party payors; ● effectively competing with other therapies; ● if our product candidate is approved, obtaining and maintaining coverage and adequate reimbursement by third-party payors, including government payors, for our product candidate; ● complying with all applicable regulatory requirements, including FDA current Good Clinical Practices (“GCP”), current Good Manufacturing Practices (“cGMP”), and standards, rules and regulations governing promotional and other marketing activities; ● changes in regulatory requirements and guidance that require amending or submitting new clinical protocols or performing additional nonclinical studies; and ● unavailability of clinical trial supplies.
Although lawsuits have been filed under the CREATES Act since its enactment, those lawsuits have settled privately; therefore, to date, no federal court has reviewed or opined on the statutory language and there continues to be uncertainty regarding the scope and application of the law.
Although lawsuits have been filed under the CREATES Act since its enactment, those lawsuits have been settled privately; therefore, to date, no federal court has reviewed or opined on the statutory language and there continues to be uncertainty regarding the scope and application of the law.
Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is possible that some of our business activities could be subject to challenge under one or more of such laws. In addition, recent health care reform legislation has strengthened these laws.
Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is possible that some of our business activities could be subject to challenge under one or more such laws. In addition, recent health care reform legislation has strengthened these laws.
Any such delay or rejection could prevent us from commercializing our clinical-stage product candidate or any future therapeutic product candidates it may develop. We rely on third-party supply and manufacturing partners for drug supplies for our late-stage clinical activities and may do the same for any commercial supplies of our product candidates.
Any such delay or rejection could prevent us from commercializing our clinical-stage product candidate or any future therapeutic product candidates it may develop. We rely on third-party supply and manufacturing partners for drug supplies for our late-stage clinical activities and may do the same for any commercial supplies of our product candidate.
The laws that may affect our ability to operate include: ● The federal Anti-Kickback Statute, which prohibits, among other things, persons from knowingly and willfully soliciting, receiving, offering or paying remuneration, directly or indirectly, to induce, or in return for, the purchase or recommendation of an item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs; ● Federal civil and criminal false claims laws and civil monetary penalty laws, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment from Medicare, Medicaid, or other government payors that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government; ● The federal Health Insurance Portability and Accountability Act of 1996 (HIPAA), which created new federal criminal statutes that prohibit executing a scheme to defraud any healthcare benefit program and making false statements relating to healthcare matters; 41 Table of Contents ● HIPAA, as amended by the Health Information Technology and Clinical Health Act, and its implementing regulations, which imposes specified requirements relating to the privacy, security, and transmission of individually identifiable health information; ● The federal physician payments sunshine requirements under the ACA require manufacturers of drugs, devices, biologics, and medical supplies to report annually to the CMS information related to payments and other transfers of value to physicians, certain advanced non-physician healthcare providers, and teaching hospitals, and ownership and investment interests held by physicians and other healthcare providers and their immediate family members; and ● State law equivalents of each of the above federal laws, such as anti-kickback and false claims laws that may apply to items or services reimbursed by any third-party payor, including governmental and private payors, to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, or otherwise restrict payments that may be made to healthcare providers and other potential referral sources; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures, and state laws governing the privacy and security of health information in specified circumstances, many of which differ from each other in significant ways and may not have the same effect, thus complicating compliance efforts.
The laws that may affect our ability to operate include: ● The federal Anti-Kickback Statute, which prohibits, among other things, persons from knowingly and willfully soliciting, receiving, offering or paying remuneration, directly or indirectly, to induce, or in return for, the purchase or recommendation of an item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs; ● Federal civil and criminal false claims laws and civil monetary penalty laws, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment from Medicare, Medicaid, or other government payors that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government; ● The federal Health Insurance Portability and Accountability Act of 1996 (HIPAA), which created new federal criminal statutes that prohibit executing a scheme to defraud any healthcare benefit program and making false statements relating to healthcare matters; ● HIPAA, as amended by the Health Information Technology and Clinical Health Act, and its implementing regulations, which imposes specified requirements relating to the privacy, security, and transmission of individually identifiable health information; ● The federal physician payments sunshine requirements under the ACA require manufacturers of drugs, devices, biologics, and medical supplies to report annually to the CMS information related to payments and other transfers of value to physicians, certain advanced non-physician healthcare providers, and teaching hospitals, and ownership and investment interests held by physicians and other healthcare providers and their immediate family members; and ● State law equivalents of each of the above federal laws, such as anti-kickback and false claims laws that may apply to items or services reimbursed by any third-party payor, including governmental and private payors, to 39 Table of Contents comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, or otherwise restrict payments that may be made to healthcare providers and other potential referral sources; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures, and state laws governing the privacy and security of health information in specified circumstances, many of which differ from each other in significant ways and may not have the same effect, thus complicating compliance efforts.
As a result of intellectual property infringement claims, or to avoid potential claims, we might: ● be prohibited from selling or licensing any product that we may develop unless the patent holder licenses the patent to us, which it is not required to do; ● be required to pay substantial royalties or grant a cross license to our patents to another patent holder; decide to locate some of our research, development or manufacturing operations outside of Europe or the United States; ● be required to pay substantial damages for past infringement, which we may have to pay if a court determines that our product candidates or technologies infringe a competitor’s patent or other proprietary rights; or ● be required to redesign the manufacturing process or formulation of a drug candidate so it does not infringe, which may not be possible or could require substantial funds and time.
As a result of intellectual property infringement claims, or to avoid potential claims, we might: ● be prohibited from selling or licensing any product that we may develop unless the patent holder licenses the patent to us, which it is not required to do; ● be required to pay substantial royalties or grant a cross license to our patents to another patent holder; decide to locate some of our research, development or manufacturing operations outside of Europe or the United States; ● be required to pay substantial damages for past infringement, which we may have to pay if a court determines that our product candidate or technologies infringe a competitor’s patent or other proprietary rights; or ● be required to redesign the manufacturing process or formulation of a drug candidate so it does not infringe, which may not be possible or could require substantial funds and time.
In addition, our clinical trials will compete with other clinical trials for product candidates that are in the same therapeutic areas as our product candidates, and this competition will reduce the number and types of patients available to us, because some patients who might have opted to enroll in our trials may instead opt to enroll in a trial being conducted by one of our competitors.
In addition, our clinical trials will compete with other clinical trials for product candidates that are in the same therapeutic areas as our product candidate, and this competition will reduce the number and types of patients available to us, because some patients who might have opted to enroll in our trials may instead opt to enroll in a trial being conducted by one of our competitors.
We anticipate that we will face increased competition in the future as new companies enter the markets and as scientific developments progress. If our drug candidates obtain regulatory approvals, but do not compete effectively in the marketplace, our business will suffer. Our future product candidates for which we obtain approval may face competition sooner than anticipated.
We anticipate that we will face increased competition in the future as new companies enter the markets and as scientific developments progress. If our drug candidates obtain regulatory approvals, but do not compete effectively in the marketplace, our business will suffer. Our future product candidates, if any, for which we obtain approval may face competition sooner than anticipated.
We rely on third-party contract manufacturing organizations, or CMOs, for our preclinical and future clinical trial product materials and commercial supplies. We do not intend to produce any meaningful quantity of our future product candidates for preclinical and clinical development through our internal resources, and we do not currently own manufacturing facilities for producing such supplies.
We rely on third-party contract manufacturing organizations, or CMOs, for our preclinical and future clinical trial product materials and commercial supplies. We do not intend to produce any meaningful quantity of plogo or any of our future product candidates for preclinical and clinical development through our internal resources, and we do not currently own manufacturing facilities for producing such supplies.
Any of the foregoing circumstances could materially harm the commercial prospects for our drug candidates. Our product candidates may cause undesirable side effects that could delay or prevent their marketing approval, limit their commercial potential, or result in significant negative consequences following marketing approval, if marketing approval is obtained.
Any of the foregoing circumstances could materially harm the commercial prospects for our drug candidates. Our product candidate may cause undesirable side effects that could delay or prevent their marketing approval, limit their commercial potential, or result in significant negative consequences following marketing approval, if marketing approval is obtained.
Furthermore, the CREATES Act established a private cause of action that permits a generic product developer to sue the brand manufacturer to compel it to furnish necessary samples of an RLD on “commercially reasonable, market-based terms.” If generic developers request samples of any product candidates for which we receive marketing approval in order to conduct comparative testing to support one or more ANDAs for a generic version of our products, and we refuse any such request, we may be subject to litigation under the CREATES Act.
Furthermore, the CREATES Act established a private cause of action that permits a generic product developer to sue the brand manufacturer to compel it to furnish necessary samples of an RLD on “commercially reasonable, market-based terms.” If generic developers request samples of any product candidate for which we receive marketing approval in order to conduct comparative testing to support one or more ANDAs for a generic version of our products, and we refuse any such request, we may be subject to litigation under the CREATES Act.
Any such failure by us or any of our CMOs would significantly impact our ability to develop, obtain regulatory approval for or, if approved, market our product candidates. We may rely on third-party manufacturers if we receive regulatory approval for any product candidate.
Any such failure by us or any of our CMOs would significantly impact our ability to develop, obtain regulatory approval for or, if approved, market our product candidate. We may rely on third-party manufacturers if we receive regulatory approval for any product candidate.
Dependence on collaborative arrangements or strategic alliances will subject us to a number of risks, including the risks that: ● we may not be able to control the amount and timing of resources that our collaborators may devote to the drug candidates; ● our collaborators may experience financial difficulties; ● we may be required to relinquish important rights such as marketing and distribution rights; ● business combinations or significant changes in a collaborator’s business strategy may also adversely affect a collaborator’s willingness or ability to complete its obligations under any arrangement; ● a collaborator could independently move forward with a competing drug candidate developed either independently or in collaboration with others, including our competitors; and ● collaborative arrangements are often terminated or allowed to expire, which would delay development and may increase the cost of developing our drug candidates.
Dependence on collaborative arrangements or strategic alliances will subject us to a number of risks, including the risks that: ● we may not be able to control the amount and timing of resources that our collaborators may devote to the drug 51 Table of Contents candidates; ● our collaborators may experience financial difficulties; ● we may be required to relinquish important rights such as marketing and distribution rights; ● business combinations or significant changes in a collaborator’s business strategy may also adversely affect a collaborator’s willingness or ability to complete its obligations under any arrangement; ● a collaborator could independently move forward with a competing drug candidate developed either independently or in collaboration with others, including our competitors; and ● collaborative arrangements are often terminated or allowed to expire, which would delay development and may increase the cost of developing our drug candidates.
If laboratories, CROs or clinical investigators do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our preclinical or clinical protocols, regulatory requirements or for other reasons, our preclinical studies or 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 laboratories, CROs or clinical investigators do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our preclinical or clinical protocols, regulatory requirements or for other reasons, our preclinical studies or clinical trials may be extended, delayed or terminated and we may not be able to obtain regulatory approval for or successfully commercialize our product candidate.
If the FDA approves generic versions of any of our products in the future, should they be approved for commercial marketing, such competitive products may be able to immediately compete with us in each indication for which our product has received approval, which could negatively impact our future revenue, profitability and cash flows and substantially limit our ability to obtain a return on our investments.
If the FDA approves generic versions of any of our products in the future, should they be approved for commercial marketing, such competitive products may be able to immediately compete with us in each indication for which our product has received approval, which could negatively impact our future revenue, profitability and cash flow and substantially limit our ability to obtain a return on our investments.
In the United States, various federal and state regulators, including governmental agencies like the Federal Trade Commission, have promulgated, or are considering promulgating, regulations concerning personal information and data securityIn addition to fines and penalties imposed upon violators, some of these state laws also afford private rights of action to individuals who believe their personal information has been misused.
In the United States, various federal and state regulators, including governmental agencies like the Federal Trade Commission, have promulgated, or are considering promulgating, regulations concerning personal information and data security. In addition to fines and penalties imposed upon violators, some of these state laws also afford private rights of action to individuals who believe their personal information has been misused.
Significant additional research, preclinical testing and clinical testing is required before we can file applications with the FDA or EMA for approval of our drug candidates. In addition, to compete effectively, our drugs must be easy to administer, cost-effective and economical to manufacture on a commercial scale. We may not achieve any of these objectives.
Significant additional research, preclinical testing and clinical testing is required before we can file applications with the FDA or EMA for approval of plogo or any of our future drug candidates. In addition, to compete effectively, our drugs must be easy to administer, cost-effective and economical to manufacture on a commercial scale. We may not achieve any of these objectives.
Such products, if approved and depending upon the scope of the changes made to the reference drug, may also compete with any product candidates for which we receive approval. The FDA is prohibited by statute from approving an ANDA or 505(b)(2) NDA when certain marketing or data exclusivity protections apply to the reference listed drug.
Such products, if approved and depending upon the scope of the changes made to the reference drug, may also compete with any product candidate for which we receive approval. The FDA is prohibited by statute from approving an ANDA or 505(b)(2) NDA when certain marketing or data exclusivity protections apply to the reference listed drug.
Other potential consequences include, among other things: 33 Table of Contents ● restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls; ● fines, warning letters or other enforcement-related letters or clinical holds on post-approval clinical trials; ● refusal of the FDA to approve pending NDAs or supplements to approved NDAs, or suspension or revocation of product approvals; ● product seizure or detention, or refusal to permit the import or export of products; ● injunctions or the imposition of civil or criminal penalties; and ● consent decrees, corporate integrity agreements, debarment, or exclusion from federal health care programs; or mandated modification of promotional materials and labeling and the issuance of corrective information.
Other potential consequences include, among other things: ● restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls; ● fines, warning letters or other enforcement-related letters or clinical holds on post-approval clinical trials; ● refusal of the FDA to approve pending NDAs or supplements to approved NDAs, or suspension or revocation of product approvals; ● product seizure or detention, or refusal to permit the import or export of products; ● injunctions or the imposition of civil or criminal penalties; and ● consent decrees, corporate integrity agreements, debarment, or exclusion from federal health care programs; or mandated modification of promotional materials and labeling and the issuance of corrective information.
With a limited number of patients, rare and severe side effects of our product candidates may only be uncovered with a significantly larger number of patients exposed to the product candidate.
With a limited number of patients, rare and severe side effects of our product candidate may only be uncovered with a significantly larger number of patients exposed to the product candidate.
The FDA may also require a REMS program as a condition of approval of our product candidates, which could entail requirements for long-term patient follow-up, a medication guide, physician communication plans or additional elements to ensure safe use, such as restricted distribution methods, patient registries and other risk minimization tools.
The FDA may also require a REMS program as a condition of approval of our product candidate, which could entail requirements for long-term patient follow-up, a medication guide, physician communication plans or additional elements to ensure safe use, such as restricted distribution methods, patient registries and other risk minimization tools.
In addition, if the FDA or a comparable foreign regulatory authority approves our product candidates, we will have to comply with requirements including submissions of safety and other post-marketing information and reports, registration, as well as continued compliance with cGMP and GCP for any clinical trials that we conduct post-approval.
In addition, if the FDA or a comparable foreign regulatory authority approves our product candidate, we will have to comply with requirements including submissions of safety and other post-marketing information and reports, registration, as well as continued compliance with cGMP and GCP for any clinical trials that we conduct post-approval.
To the extent that any disruption or security breach were to result in a loss of or damage to our data, or inappropriate disclosure of confidential or proprietary information, we could suffer material legal claims and liability, damage to our reputation, suffer loss or harm to our intellectual property rights and the further research, development and commercial efforts of our products and product candidates could be delayed.
To the extent that any disruption or security breach were to result in a loss of or damage to our data, or inappropriate disclosure of confidential or proprietary information, we could suffer material legal claims and liability, damage to our reputation, suffer loss or harm to our intellectual property rights and the further research, development and commercial efforts of our products and product candidate could be delayed.
The degree of market acceptance of any of our approved drugs will depend on a variety of factors, including: ● timing of market introduction, number and clinical profile of competitive drugs; ● our ability to provide acceptable evidence of safety and efficacy; ● relative convenience and ease of administration; ● pricing and cost-effectiveness, which may be subject to regulatory control; ● availability of coverage, reimbursement and adequate payment from health maintenance organizations and other third-party payors; and ● prevalence and severity of adverse side effects; and other potential advantages over alternative treatment methods.
The degree of market acceptance of any of our approved drugs will depend on a variety of factors, including: ● timing of market introduction, number and clinical profile of competitive drugs; ● our ability to provide acceptable evidence of safety and efficacy; ● relative convenience and ease of administration; ● pricing and cost-effectiveness, which may be subject to regulatory control; ● availability of coverage, reimbursement and adequate payment from health maintenance organizations and 33 Table of Contents other third-party payors; and ● prevalence and severity of adverse side effects; and other potential advantages over alternative treatment methods.
Our future funding requirements will depend on many factors, including: ● the scope, rate of progress and cost of our clinical trials and other research and development activities; ● the costs and timing of seeking and obtaining regulatory approvals; ● the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; ● the costs associated with establishing sales and marketing capabilities; ● the costs of acquiring or investing in businesses, products and technologies; ● the effect of competing technological and market developments; and 48 Table of Contents ● the payment, other terms and timing of any strategic alliance, licensing or other arrangements that we may establish.
Our future funding requirements will depend on many factors, including: ● the scope, rate of progress and cost of our clinical trials and other research and development activities; ● the costs and timing of seeking and obtaining regulatory approvals; ● the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; ● the costs associated with establishing sales and marketing capabilities; ● the costs of acquiring or investing in businesses, products and technologies; ● the effect of competing technological and market developments; and ● the payment, other terms and timing of any strategic alliance, licensing or other arrangements that we may establish.
Our product candidates may not be effective, may be only moderately effective or may prove to have undesirable or unintended side effects, toxicities or other characteristics that may preclude our obtaining marketing approval or prevent or limit commercial use, or there may be deficiencies in cGMP compliance by us or by our contract manufacturers that could result in the candidate not being approved.
Our product candidate, plogo, may not be effective, may be only moderately effective or may prove to have undesirable or unintended side effects, toxicities or other characteristics that may preclude our obtaining marketing approval or prevent or limit commercial use, or there may be deficiencies in cGMP compliance by us or by our contract manufacturers that could result in the candidate not being approved.
If we elect to fund drug development or research programs on our own, we will have to increase our expenditures and will need to obtain additional funding, which may be unavailable or available only on unfavorable terms. To the extent we are able to enter into strategic transactions, we will be exposed to risks related to those collaborations and alliances.
If we elect to fund drug development or research programs on our own, we will have to increase our expenditure and will need to obtain additional funding, which may be unavailable or available only on unfavorable terms. To the extent we are able to enter into strategic transactions, we will be exposed to risks related to those collaborations and alliances.
The policies of the FDA and of other regulatory authorities may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidates. We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad.
The policies of the FDA and of other regulatory authorities may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidate. We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad.
If we are unable to compete successfully in our marketplace, it will harm our business. There are existing products in the marketplace that compete with our products. Companies may develop new products that compete with our products. Certain of these competitors and potential competitors have longer operating histories, substantially greater product development capabilities and financial, scientific, marketing and sales resources.
If we are unable to compete successfully in our marketplace, it will harm our business. There are existing products in the marketplace that compete with our products. Companies may develop new products that compete with our products. Certain competitors and potential competitors have longer operating histories, substantially greater product development capabilities and financial, scientific, marketing and sales resources.
Because we have to prioritize our development candidates as a result of budget constraints, we may not be able to fully realize the value of our product candidates in a timely manner, if at all. We are exposed to risks related to foreign currency exchange rates. Some of our costs and expenses are denominated in foreign currencies.
Because we have to prioritize our development candidate as a result of budget constraints, we may not be able to fully realize the value of our product candidate in a timely manner, if at all. We are exposed to risks related to foreign currency exchange rates. Some of our costs and expenses are denominated in foreign currencies.
Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in regulatory actions, such as the issuance of FDA Form 483 notices of observations, warning letters or sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of product candidates or drugs, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect supplies of our products.
Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in 50 Table of Contents regulatory actions, such as the issuance of FDA Form 483 notices of observations, warning letters or sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of product candidates or drugs, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect supplies of our products.
In addition, even if we were to obtain regulatory approval in one or more jurisdictions, regulatory authorities may approve any of our drug candidates for fewer or more limited indications than we request, may not approve prices we may propose to charge for our products, may grant approval contingent on the performance of costly post-marketing 31 Table of Contents clinical trials (referred to as “conditional” or “accelerated” approval depending on the jurisdiction), or may approve a drug candidate with a label that does not include the labeling claims necessary or desirable for the successful commercialization of that drug candidate.
In addition, even if we were to obtain regulatory approval in one or more jurisdictions, regulatory authorities may approve any of our drug candidates for fewer or more limited indications than we request, may not approve prices we may propose to charge for our products, may grant approval contingent on the performance of costly post-marketing clinical trials (referred to as “conditional” or “accelerated” approval depending on the jurisdiction), or may approve a drug candidate with a label that does not include the labeling claims necessary or desirable for the successful commercialization of that drug candidate.
This could reduce the ultimate demand for our product candidates, if approved, or put pressure on our product pricing, which could negatively affect our business, results of operations, financial condition and prospects. In the European Union, similar political, economic and regulatory developments may affect our ability to profitably commercialize our products.
This could reduce the ultimate demand for our product candidate, if approved, or put pressure on our product pricing, which could negatively affect our business, results of operations, financial condition and prospects. In the European Union, similar political, economic and regulatory developments may affect our ability to profitably commercialize our products.
Depending upon the timing, duration and specifics of FDA marketing approval of our product candidates, if any, one or more of our United States patents may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, referred to as the Hatch-Waxman Act.
Depending upon the timing, duration and specifics of FDA marketing approval of our product candidate, if any, one or more of our United States patents may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, referred to as the Hatch-Waxman Act.
We face four primary risks relative to protecting this critical information: loss of access; unauthorized disclosure; unauthorized modification; and inadequate monitoring of our controls over the first three risks. We utilize information technology, or IT, systems and networks to process, transmit and store electronic information in connection with our business activities.
We face four primary risks related to protecting this critical information: loss of access; unauthorized disclosure; unauthorized modification; and inadequate monitoring of our controls over the first three risks. We utilize information technology, or IT, systems and networks to process, transmit and store electronic information in connection with our business activities.
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act (HITECH), establishes a set of U.S. national privacy and security standards for the protection of individually identifiable health information, including protected health information, or PHI, by health plans, certain healthcare clearinghouses and healthcare providers that submit certain covered transactions electronically, or covered entities, and their “business associates,” which are persons or entities that perform certain services for, or on behalf of, a covered entity that involve creating, receiving, maintaining or transmitting PHI.
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act (HITECH), establishes a set of U.S. national privacy and security standards for the protection of individually identifiable health information, including protected health information, or PHI, by health plans, certain healthcare clearinghouses and healthcare providers that submit certain covered transactions electronically, or covered entities, and their “business 41 Table of Contents associates,” which are persons or entities that perform certain services for, or on behalf of, a covered entity that involve creating, receiving, maintaining or transmitting PHI.
The time and expense of the approval process, as well as the unpredictability of future clinical trial results and other contributing factors, may result in our failure to obtain regulatory approval to market any of our product candidates in one or more jurisdictions, which would significantly harm our business, results of operations and prospects.
The time and expense of the approval process, as well as the unpredictability of future clinical trial results and other contributing factors, may result in our failure to obtain regulatory approval to market any of our product candidate in one or more jurisdictions, which would significantly harm our business, results of operations and prospects.
Obtaining marketing approval requires the submission of extensive nonclinical and clinical data and supporting information to regulatory authorities for each therapeutic indication to establish the product candidate’s safety and effiveness. Securing marketing approval also requires the submission of information about the product manufacturing process, and in many cases the inspection of manufacturing, processing and packaging facilities by the regulatory authorities.
Obtaining marketing approval requires the submission of extensive nonclinical and clinical data and supporting information to regulatory authorities for each therapeutic indication to establish the product candidate’s safety and effectiveness. Securing marketing approval also requires the submission of information about the product manufacturing process, and in many cases the inspection of manufacturing, processing and packaging facilities by the regulatory authorities.
In the event that our clinical trials produce undesirable side effects, our trials could be suspended or terminated and the FDA or comparable foreign regulatory authorities could order us to cease further development of or deny approval of our product candidates for any or all targeted indications.
In the event that our clinical trials produce undesirable side effects, our trials could be suspended or terminated and the FDA or comparable foreign regulatory authorities could order us to cease further development of or deny approval of our product candidate for any or all targeted indications.
As a public company, we are subject to Section 404 of the Sarbanes Oxley Act relating to internal control over financial reporting. We have completed a formal process to evaluate our internal controls for purposes of Section 404, and we concluded that as of December 31, 2023, our internal control over financial reporting was effective.
As a public company, we are subject to Section 404 of the Sarbanes Oxley Act relating to internal control over financial reporting. We have completed a formal process to evaluate our internal controls for purposes of Section 404, and we concluded that as of December 31, 2024, our internal control over financial reporting was effective.
You could, therefore, experience a decline in the value of your investment as a result of short sales of our common stock. We are exposed to risks related to the marketable securities we may purchase. We may invest cash not required to meet short-term obligations in short term marketable securities.
You could, therefore, experience a decline in the value of your investment as a result of short sales of our common stock. We are exposed to risks related to the marketable securities we may purchase. We may invest cash that is not required to meet short-term obligations in short term marketable securities.
Some of these provisions: ● authorize the issuance of preferred stock that can be created and issued by the Board of Directors without prior stockholder approval, commonly referred to as “blank check” preferred stock, with rights senior to those of our common stock; ● provide for the Board of Directors to be divided into three classes; and ● require that stockholder actions must be effected at a duly called stockholder meeting and prohibit stockholder action by written consent.
Some of these provisions: ● authorize the issuance of preferred stock that can be created and issued by the Board of Directors without prior stockholder approval, commonly referred to as “blank check” preferred stock, with rights senior to those of our 58 Table of Contents common stock; ● provide for the Board of Directors to be divided into three classes; and ● require that stockholder actions must be effected at a duly called stockholder meeting and prohibit stockholder action by written consent.
Moreover, because our product candidates represent a departure from more commonly used methods for cancer treatment, potential patients and their doctors may be inclined to use conventional therapies rather than enroll patients in any future clinical trial.
Moreover, because our product candidate represent a departure from more commonly used methods for cancer treatment, potential patients and their doctors may be inclined to use conventional therapies rather than enroll patients in any future clinical trial.
Factors giving rise to this volatility may include: ● disclosure of actual or potential clinical results with respect to product candidates we are developing; ● regulatory developments in both the United States and abroad; ● developments concerning proprietary rights, including patents and litigation matters; ● public concern about the safety or efficacy of our product candidates or technology, or related technology, or new technologies generally; ● concern about the safety or efficacy of our product candidates or technology, or related technology, or new technologies generally; ● public announcements by our competitors or others; and ● general market conditions and comments by securities analysts and investors. 57 Table of Contents Fluctuations in our operating losses could adversely affect the price of our common stock.
Factors giving rise to this volatility may include: ● disclosure of actual or potential clinical results with respect to the product candidate we are developing; ● regulatory developments in both the United States and abroad; ● developments concerning proprietary rights, including patents and litigation matters; ● public concern about the safety or efficacy of our product candidate or technology, or related technology, or new technologies generally; ● concern about the safety or efficacy of our product candidate or technology, or related technology, or new technologies generally; ● public announcements by our competitors or others; and ● general market conditions and comments by securities analysts and investors. 56 Table of Contents Fluctuations in our operating losses could adversely affect the price of our common stock.
To the extent that any disruption or security breach were to result in a loss of or damage to our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur liability and the further development of our product candidates could be delayed.
To the extent that any disruption or security breach were to result in a loss of or damage to our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur liability and the further development of our product candidate could be delayed.
Notwithstanding the Reverse Stock Split and our compliance with the Nasdaq Capital market requirements, we cannot be sure that our share price will continue to comply with the requirements for continued listing of our common stock on the Nasdaq Capital Market in the future, or that we will continue to comply with the other continued listing requirements.
Notwithstanding the Reverse Stock Split and our compliance with the Equity Rule, we cannot be sure that our share price will continue to comply with the requirements for continued listing of our common stock on the Nasdaq Capital Market in the future, or that we will continue to comply with the other continued listing requirements.
In addition, our independent certified public accounting firm has not provided an opinion on the effectiveness of our internal controls over financial reporting for the year ended December 31, 2023 because we are a smaller reporting company.
In addition, our independent certified public accounting firm has not provided an opinion on the effectiveness of our internal controls over financial reporting for the year ended December 31, 2024 because we are a smaller reporting company.
In addition, if we make manufacturing or formulation changes to our product candidates, we may need to conduct additional nonclinical studies and/or clinical trials, or the results obtained from such new formulation may not be consistent with previous results obtained.
In addition, if we make manufacturing or formulation changes to our product candidate, we may need to conduct additional nonclinical studies and/or clinical trials, or the results obtained from such new formulation may not be consistent with previous results obtained.
The probability of success of these newly announced policies, many of which have been subjected to legal challenge in the federal court system, and their potential impact on the U.S. prescription drug marketplace is unknown. There are likely to be continued political and legal challenges associated with implementing these reforms as they are currently envisioned.
The probability of success of these newly announced policies, many of which have been subjected to legal challenge in the federal court system, and their potential 37 Table of Contents impact on the U.S. prescription drug marketplace is unknown. There are likely to be continued political and legal challenges associated with implementing these reforms as they are currently envisioned.
We intend to expand our insurance coverage for products to include the sale of commercial products if we obtain marketing approval for our product candidates in development, but we may be unable to obtain commercially reasonable product liability insurance for any products approved for marketing.
We intend to expand our insurance coverage for products to include the sale of commercial products if we obtain marketing approval for our product candidate in development, but we may be unable to obtain commercially reasonable product liability insurance for any products approved for marketing.
To meet our long-term financing requirements, we may raise funds through public or private equity offerings, debt financings or strategic alliances. Raising additional funds by issuing equity or convertible debt securities may cause our stockholders to experience substantial dilution in their ownership interests and new investors may have rights superior to the rights of our other stockholders.
To meet our long-term financing requirements, we may raise funds through public or private equity offerings, debt financings or strategic alliances. 43 Table of Contents Raising additional funds by issuing equity or convertible debt securities may cause our stockholders to experience substantial dilution in their ownership interests and new investors may have rights superior to the rights of our other stockholders.
The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant liability.
In addition, the FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant liability.
If our shares of Common Stock lose their status on the Nasdaq Capital Market, we believe that our shares of Common Stock would likely be eligible to be quoted on the inter-dealer electronic quotation and trading system operated by Pink OTC Markets Inc., commonly referred to as the Pink Sheets and now known as the OTCQB market.
If our shares of common stock lose their status on the Nasdaq Capital Market, we believe that our shares of common stock would likely be eligible to be quoted on the inter-dealer electronic quotation and trading system operated by Pink OTC Markets Inc., 46 Table of Contents commonly referred to as the Pink Sheets and now known as the OTCQB market.
In such case, we may also not have the resources to conduct new clinical trials and/or we may determine that further clinical development of any such drug candidate is not justified and may discontinue any such programs.
In such cases, we may also not have the resources to conduct new clinical trials and/or we may determine that further clinical development of any such drug candidate is not justified and may discontinue any such programs.
Any regulatory approvals that we receive for our product candidates may be subject to limitations on the approved indicated uses for which the product may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase 4 clinical trials, and surveillance to monitor the safety and efficacy of the product candidate.
Any regulatory approvals that we receive for plogo may be subject to limitations on the approved indicated uses for which the product may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase 4 clinical trials, and surveillance to monitor the safety and efficacy of the product candidate.
Section 505(b)(2) provides an alternate path to FDA approval for new or improved formulations or new uses of previously approved products; for example, a follow-on applicant may be seeking approval to market a previously approved drug for new indications or for a new patient population that would require new clinical data to 35 Table of Contents demonstrate safety or effectiveness.
Section 505(b)(2) provides an alternate path to FDA approval for new or improved formulations or new uses of previously approved products; for example, a follow-on applicant may be seeking approval to market a previously approved drug for new indications or for a new patient population that would require new clinical data to demonstrate safety or effectiveness.
As the third-party manufacturers are the sole supplier of the products, any delays may impact our sales. In all the countries where we may sell our products, governmental regulations exist to define standards for manufacturing, packaging, labeling and storing. All of our suppliers of raw materials and contract manufacturers must comply with these regulations.
As the third-party manufacturers are the sole supplier of the products, any delays may impact our sales. 35 Table of Contents In all the countries where we may sell our products, governmental regulations exist to define standards for manufacturing, packaging, labeling and storing. All of our suppliers of raw materials and contract manufacturers must comply with these regulations.
In addition, sales, marketing and business arrangements 46 Table of Contents in the health care industry are subject to extensive laws and regulations intended to prevent fraud, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements.
In addition, sales, marketing and business arrangements in the health care industry are subject to extensive laws and regulations intended to prevent fraud, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements.
If we are unable to obtain or maintain third-party manufacturing for product candidates, or to do so on commercially reasonable terms, we may not be able to develop and commercialize our product candidates successfully.
If we are unable to obtain or maintain third-party manufacturing for product candidate, or to do so on commercially reasonable terms, we may not be able to develop and commercialize our product candidate successfully.
In addition, our CMOs are subject to inspection and approval by regulatory authorities before we can commence the manufacture and sale of any of our product candidates, and thereafter are subject to ongoing inspection from time to time. Our CMOs may not be able to comply with applicable cGMP regulations or similar regulatory requirements outside of the United States.
In addition, our CMOs are subject to inspection and approval by regulatory authorities before we can commence the manufacture and sale of any of plogo and thereafter are subject to ongoing inspection from time to time. Our CMOs may not be able to comply with applicable cGMP regulations or similar regulatory requirements outside of the United States.
Several pharmaceutical and biotechnology companies have CDK inhibitors, PLK1 inhibitors or other products on the market or in clinical trials which may be competitive to our drugs in both hematological and oncology indications. Our competitors, either alone or together with collaborators, may have substantially greater financial resources and research and development staff.
Several pharmaceutical and biotechnology companies have PLK1 inhibitors or other products on the market or in clinical trials which may be competitive to our drug in both hematological and oncology indications. Our competitors, either alone or together with collaborators, may have substantially greater financial resources and research and development staff.
We expect to incur continued losses for several years as we continue our research and development of our drug candidates, seek regulatory approvals and commercialize any approved drugs. If our drug candidates are unsuccessful in clinical trials or we are unable to obtain regulatory approvals, or if our drugs are unsuccessful in the market, we will not be profitable.
We expect to incur continued losses for several years as we continue our research and development of our drug candidates, seek regulatory approvals and commercialize any approved drugs. If our remaining drug candidate is unsuccessful in clinical trials or we are unable to obtain regulatory approvals, or if our drugs are unsuccessful in the market, we will not be profitable.
Obtaining and maintaining marketing approval of our current and future product candidates in one jurisdiction does not guarantee that we will be able to obtain or maintain marketing approval in any other jurisdiction, while a failure or delay in obtaining marketing approval in one jurisdiction may have a negative effect on the marketing approval process in others.
Obtaining and maintaining marketing approval of our remaining product candidate, and any future product candidates, in one jurisdiction does not guarantee that we will be able to obtain or maintain marketing approval in any other jurisdiction, while a failure or delay in obtaining marketing approval in one jurisdiction may have a negative effect on the marketing approval process in others.
Our or a third-party’s failure to execute on our manufacturing requirements could adversely affect our business in a number of ways, including: ● an inability to initiate or continue clinical trials of product candidates under development, which may impact our potential economic benefits; ● delay in submitting regulatory applications, or receiving regulatory approvals, for product candidates; ● loss of the cooperation of a collaborator; ● subjecting our product candidates to additional inspections by regulatory authorities; ● requirements to cease distribution or to recall batches of our product candidates; and ● in the event of approval to market and commercialize a product candidate, an inability to meet commercial demands for our products.
Our or a third-party’s failure to execute our manufacturing requirements could adversely affect our business in a number of ways, including: ● an inability to initiate or continue clinical trials of plogo under development, which may impact our potential economic benefits; ● delay in submitting regulatory applications, or receiving regulatory approvals, for plogo or any future product candidate; ● loss of the cooperation of a collaborator; ● subjecting plogo or any future of our product candidate to additional inspections by regulatory authorities; ● requirements to cease distribution or to recall batches of plogo or any future of our product candidate; and ● in the event of approval to market and commercialize plogo or any future product candidate, an inability to meet commercial demands for our products.
To the extent equity valuations, including the trading price of our common stock, are depressed as a result 45 Table of Contents of economic disruptions or other uncertainties, for example due to rising inflationary pressures, ongoing military conflicts or other factors, the potential magnitude of this dilution will increase.
To the extent equity valuations, including the trading price of our common stock, are depressed as a result of economic disruptions or other uncertainties, for example due to rising inflationary pressures, ongoing military conflicts or other factors, the potential magnitude of this dilution will increase.
However, trade secrets are difficult to protect. Our employees, consultants, contractors, outside scientific collaborators and other advisors may unintentionally or willfully disclose our confidential information to competitors, and confidentiality agreements may not provide an adequate remedy in the event of unauthorized disclosure of confidential 53 Table of Contents information.
However, trade secrets are difficult to protect. Our employees, consultants, contractors, outside scientific collaborators and other advisors may unintentionally or willfully disclose our confidential information to competitors, and confidentiality agreements may not provide an adequate remedy in the event of unauthorized disclosure of confidential information.
Any inability to successfully complete clinical development and obtain regulatory approval for our product candidates could result in additional costs to us or impair our ability to generate revenue.
Any inability to successfully complete clinical development and obtain regulatory approval for our product candidate could result in additional costs to us or impair our ability to generate revenue.
If we fail to obtain additional funding, we may be unable to complete the development and commercialization of our lead drug candidates, fadraciclib and plogosertib, or continue to fund our research and development programs.
If we fail to obtain additional funding, we may be unable to complete the development and commercialization of our lead drug candidates or continue to fund our research and development programs.
Our commercial revenues from our product candidates currently in development, if any, will be derived from sales of drugs that will not become marketable for several years, if at all.
Our commercial revenues from our product candidate currently in development, if any, will be derived from sales of drugs that will not become marketable for several years, if at all.
These factors would increase our reliance on such manufacturer or require us to obtain a license from such manufacturer in order to have another third party manufacture our product candidates.
These factors would increase our reliance on such manufacturer or require us to obtain a license from such manufacturer in order to have another third party manufacture our product candidate.
Failure to achieve and maintain a diverse workforce and leadership team, 37 Table of Contents compensate our employees competitively and fairly, maintain a safe and inclusive environment or promote the well-being of our employees could affect our reputation and also result in lower performance and an inability to retain valuable employees.
Failure to achieve and maintain a diverse workforce and leadership team, compensate our employees competitively and fairly, maintain a safe and inclusive environment or promote the well-being of our employees could affect our reputation and also result in lower performance and an inability to retain valuable employees.
If we fail to obtain and maintain patent protection and trade secret protection of our product candidates, proprietary technologies and their uses, we could lose our competitive advantage and competition we face would increase, reducing our potential revenues and adversely affecting our ability to attain or maintain profitability.
If we fail to obtain and maintain patent protection and trade secret protection of Plogo or any of our future product candidates, proprietary technologies and their uses, we could lose our competitive advantage and competition we face would increase, reducing our potential revenues and adversely affecting our ability to attain or maintain profitability.
We will not distribute any cash to the holders of the securities to pay these potential tax liabilities. If we automatically convert the convertible preferred stock, there is a substantial risk of fluctuation in the price of our common stock from the date we elect to automatically convert to the conversion date.
We will not distribute any cash to the holders of the securities to pay these potential tax liabilities. 60 Table of Contents If we automatically convert the convertible preferred stock, there is a substantial risk of fluctuation in the price of our common stock from the date we elect to automatically convert to the conversion date.
These threats pose a risk to the security of our systems and networks and the confidentiality, availability and integrity of our 49 Table of Contents data. There can be no assurance that we will be successful in preventing cybersecurity incidents or successfully mitigating their effects.
These threats pose a risk to the security of our systems and networks and the confidentiality, availability and integrity of our data. There can be no assurance that we will be successful in preventing cybersecurity incidents or successfully mitigating their effects.
If any of our relationships with these third-party laboratories, CROs or clinical investigators 50 Table of Contents terminate, we may not be able to enter into arrangements with alternative laboratories, CROs or investigators or to do so in a timely manner or on commercially reasonable terms.
If any of our relationships with these third-party laboratories, CROs or clinical investigators terminate, we may not be able to enter into arrangements with alternative laboratories, CROs or investigators or to do so in a timely manner or on commercially reasonable terms.
The regulatory framework for collecting, using, safeguarding, sharing, transfering and other processing of information worldwide is rapidly evolving and is likely to remain uncertain for the foreseeable future.
The regulatory framework for collecting, using, safeguarding, sharing, transferring and other processing of information worldwide is rapidly evolving and is likely to remain uncertain for the foreseeable future.
The delays associated with the verification of a new manufacturer could negatively affect our ability to develop product candidates in a timely manner or within budget.
The delays associated with the verification of a new manufacturer could negatively affect our ability to develop product candidate in a timely manner or within budget.
Even if we successfully complete the clinical trials for one or more of our product candidates, the product candidates may fail for other reasons, including, without limitation, the possibilities that the product candidates will: ● fail to receive the regulatory approvals required to market them as drugs; ● be subject to proprietary rights held by others requiring the negotiation of a license agreement prior to marketing; 34 Table of Contents ● be difficult or expensive to manufacture on a commercial scale; ● have adverse side effects that make their use less desirable; or ● fail to compete effectively with product candidates or other treatments commercialized by our competitors.
Even if we successfully complete the clinical trials for our product candidate, the product candidate may fail for other reasons, including, without limitation, the possibilities that the product candidate will: ● fail to receive the regulatory approvals required to market them as drugs; ● be subject to proprietary rights held by others requiring the negotiation of a license agreement prior to marketing; ● be difficult or expensive to manufacture on a commercial scale; ● have adverse side effects that make their use less desirable; or ● fail to compete effectively with product candidates or other treatments commercialized by our competitors.
The GDPR imposed a broad data protection framework that expanded the scope of EU and UK data protection law, including to non-EU and non-UK entities meeting the jurisdictional requirements that process, or control the processing of, personal 42 Table of Contents data relating to individuals located in the EU or UK, including clinical trial data.
The GDPR imposed a broad data protection framework that expanded the scope of EU and UK data protection law, including to non-EU and non-UK entities meeting the jurisdictional requirements that process, or control the processing of personal data relating to individuals located in the EU or UK, including clinical trial data.
Certain of our competitors and potential competitors have broader product offerings and extensive customer bases, allowing them to adopt aggressive pricing policies that would enable them to gain market share. Competitive pressures could result in price reductions, reduced margins and loss of market share.
Certain competitors and potential competitors have broader product offerings and extensive customer bases, allowing them to adopt aggressive pricing policies that would enable them to gain market share. Competitive pressure could result in price reductions, reduced margins and loss of market share.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeOur response to an incident involves the coordination of activities to detect, respond to and recover from cybersecurity incidents, which include processes to triage, assess severity for, escalate, contain, investigate and remediate the incident, as well as to comply with potentially applicable legal obligations and mitigate damage to our business and reputation.
Biggest changeOur response to an incident involves the coordination of activities to detect, respond to and recover from cybersecurity incidents, which include processes to triage, assess severity for, escalate, contain, investigate and remediate the incident, as well as to comply with potentially applicable legal obligations and mitigate damage to our business and reputation. 63 Table of Contents As part of the above processes, we regularly engage with consultants, auditors and other third parties, including having a third-party independent qualified and accredited advisor review our cybersecurity program to help identify areas for continued focus, improvement and compliance.
To provide for the availability of critical data and systems, maintain regulatory compliance, manage our material risks from cybersecurity threats, and protect against and respond to cybersecurity incidents, we undertake the following activities: ● monitor emerging data protection laws and implement changes to our processes that are designed to comply with such laws; ● through our policies, practices and contracts (as applicable), require employees, as well as third parties that provide services on our behalf, to treat confidential information and data with care; ● employ technical safeguards that are designed to protect our information systems from cybersecurity threats, including firewalls, intrusion prevention and detection systems, anti-malware functionality and access controls, which are evaluated and improved through vulnerability assessments and cybersecurity threat intelligence; ● provide regular training for our employees regarding cybersecurity threats as a means to equip them with effective tools to address cybersecurity threats, and to communicate our evolving information security policies, standards, processes and practices; ● leverage the National Cyber Security Centre incident handling framework to help us identify, protect, detect, respond and recover when there is an actual or potential cybersecurity incident; and 64 Table of Contents ● carry information security risk insurance that provides protection against the potential losses arising from a cybersecurity incident.
To provide for the availability of critical data and systems, maintain regulatory compliance, manage our material risks from cybersecurity threats, and protect against and respond to cybersecurity incidents, we undertake the following activities: ● monitor emerging data protection laws and implement changes to our processes that are designed to comply with such laws; ● through our policies, practices and contracts (as applicable), require employees, as well as third parties that provide services on our behalf, to treat confidential information and data with care; ● employ technical safeguards that are designed to protect our information systems from cybersecurity threats, including firewalls, intrusion prevention and detection systems, anti-malware functionality and access controls, which are evaluated and improved through vulnerability assessments and cybersecurity threat intelligence; ● provide regular training for our employees regarding cybersecurity threats as a means to equip them with effective tools to address cybersecurity threats, and to communicate our evolving information security policies, standards, processes and practices; ● leverage the National Cyber Security Centre incident handling framework to help us identify, protect, detect, respond and recover when there is an actual or potential cybersecurity incident; and ● carry information security risk insurance that provides protection against the potential losses arising from a cybersecurity incident.
Our audit committee also receive prompt and timely information regarding any cybersecurity incident that meets establishing reporting thresholds, as well as ongoing updates regarding any such incident until it has been addressed. To date, we have not experience any cyber security incident.
Our audit committee also receive prompt and timely information regarding any cybersecurity incident that meets establishing reporting thresholds, as well as ongoing updates regarding any such incident until it has been addressed. To date, we have not experienced any cyber security incident.
Our Chief Financial Officer has been responsible for our IT functions for 20 years, and has managed the IT function of companies generally for the last 35 years. Our IT manager has managed our IT functions for over 20 years.
Our Chief Financial Officer has been responsible for our IT functions for 20 years and has managed the IT function of companies generally for the last 35 years.
The management team members are informed about and monitor the prevention, mitigation, detection, and remediation of cybersecurity incidents through their management of, and participation in, the cybersecurity risk 65 Table of Contents management and strategy processes described above, including the operation of our incident response plan.
The management team members are informed about and monitor the prevention, mitigation, detection, and remediation of cybersecurity incidents through their management of, and participation in, the cybersecurity risk management and strategy processes described above, including the operation of our incident response plan.
As discussed above, the management team members report to the audit committee of our board of directors about cybersecurity threat risks, among other cybersecurity related matters, periodically.
As discussed above, the management team members report to the audit committee of our board of directors about cybersecurity threat risks, among other cybersecurity related matters, periodically. 64 Table of Contents
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As part of the above processes, we regularly engage with consultants, auditors and other third parties, including having a third-party independent qualified and accredited advisor review our cybersecurity program to help identify areas for continued focus, improvement and compliance.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeItem 3. Legal Proceedings From time to time, we may be involved in routine litigation incidental to the conduct of our business. As of December 31, 2023, we were not a party to any material legal proceedings. Item 4. Mine Safety Disclosures Not applicable. PART II
Biggest changeItem 3. Legal Proceedings From time to time, we may be involved in routine litigation incidental to the conduct of our business. As of December 31, 2024, we were not a party to any material legal proceedings. Item 4. Mine Safety Disclosures Not applicable. PART II

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeOn March 15, 2024, we had approximately 11 registered holders of record of our 1,318,257 shares of common stock outstanding. On March 15, 2024, the closing sale price of our common stock as reported by Nasdaq was $2.43 per share.
Biggest changeOn March 24, 2025, we had approximately 14 registered holders of record of our 207,336,389 shares of common stock outstanding. On March 24, 2025, the closing sale price of our common stock as reported by Nasdaq was $0.32 per share.
Holders of Common Stock We effected a 15:1 reverse stock split of the our common stock on December 18, 2023 (the “Reverse Stock Split”). All share and per share information has been retroactively adjusted to give effect to the Reverse Stock Split for all periods presented, unless otherwise indicated.
Holders of Common Stock We effected a 15:1 reverse stock split of our common stock on December 18, 2023 (the “Reverse Stock Split”). All share and per share information has been retroactively adjusted to give effect to the Reverse Stock Split for all periods presented, unless otherwise indicated.
Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information Our common stock is traded on The Nasdaq Capital Market, or Nasdaq, under the symbol “CYCC”. Our preferred stock currently trades on Nasdaq under the symbol “CYCCP”.
Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information Our common stock is traded on The Nasdaq Capital Market, or Nasdaq, under the symbol “CYCC”. Our 6% convertible exchangeable preferred stock currently trades on Nasdaq under the symbol “CYCCP”.

Item 7. Management's Discussion & Analysis

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

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Biggest changeResearch and development expenses primarily include: ● Clinical trial and regulatory-related costs; ● Payroll and personnel-related expenses, including consultants and contract research organizations; ● Preclinical studies and materials; ● Technology license costs; ● Stock-based compensation; and ● Rent and facility expenses for our office. ​ 70 Table of Contents The following table provides information with respect to our research and development expenditures for the years ended December 31, 2023 and 2022 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ ​ December 31, ​ Difference ​ ​ 2023 2022 $ % Transcriptional Regulation (fadraciclib) ​ $ 13,358 ​ $ 14,043 ​ $ (685) ​ (5) Epigenetic/anti-mitotic (plogosertib) ​ ​ 4,987 ​ ​ 5,546 ​ ​ (559) ​ (10) Other research and development expenses ​ ​ 810 ​ ​ 685 ​ ​ 125 ​ 18 Total research and development expenses ​ $ 19,155 ​ $ 20,274 ​ $ (1,119) ​ (6) ​ Research and development expenses represented 74% and 73% of our operating expenses for the years ended December 31, 2023 and 2022, respectively.
Biggest changeThe following table provides information with respect to our research and development expenditures for the years ended December 31, 2024 and 2023 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ December 31, ​ Difference ​ 2024 2023 $ % Transcriptional Regulation (fadraciclib) $ 4,970 ​ $ 13,358 ​ $ (8,388) ​ (63) Anti-mitotic (plogo) ​ 1,566 ​ ​ 4,987 ​ ​ (3,421) ​ (69) Other research and development expenses ​ 119 ​ ​ 810 ​ ​ (691) ​ (85) Total research and development expenses $ 6,655 ​ $ 19,155 ​ $ (12,500) ​ (65) ​ Research and development expenses represented 55% and 74% of our operating expenses for the years ended December 31, 2024 and 2023, respectively.
Our future funding requirements will depend on many factors, including but not limited to: ● the rate of progress and cost of our clinical trials, preclinical studies and other discovery and research and development activities; ● the costs associated with establishing manufacturing and commercialization capabilities; ● the costs of acquiring or investing in businesses, product candidates and technologies; ● the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; ● the costs and timing of seeking and obtaining FDA and EMA approvals; ● the effect of competing technological and market developments; and ● the economic and other terms and timing of any collaboration, licensing or other arrangements into which we may enter.
Our future funding requirements will depend on many factors, including but not limited to: ● the rate of progress and cost of our clinical trials, preclinical studies and other discovery and research and development activities; ● the costs associated with establishing manufacturing and commercialization capabilities; ● the costs of acquiring or investing in businesses, Plogo or any of our future product candidates and technologies; ● the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; ● the costs and timing of seeking and obtaining FDA and EMA approvals; ● the effect of competing technological and market developments; and ● the economic and other terms and timing of any collaboration, licensing or other arrangements into which we may enter.
Certain factors that could cause results to differ materially from those projected or implied in the forward-looking statements are set forth in this Annual Report on Form 10-K for the year ended December 31, 2023 under the caption “Item 1A — Risk factors”. We encourage you to read those descriptions carefully.
Certain factors that could cause results to differ materially from those projected or implied in the forward-looking statements are set forth in this Annual Report on Form 10-K for the year ended December 31, 2024 under the caption “Item 1A — Risk factors”. We encourage you to read those descriptions carefully.
While we have plans in place to 68 Table of Contents mitigate this risk, which primarily consist of raising additional capital through a combination of public or private equity or debt financings or by entering into partnership agreements for further development of our drug candidates, there is no guarantee that we will be successful in these mitigation efforts.
While we have plans in place to mitigate this risk, which primarily consist of raising additional capital through a combination of public or private equity or debt financings or by entering into partnership agreements for further development of our drug candidates, there is no guarantee that we will be successful in these mitigation efforts.
The decrease in other income primarily relates to royalties receivable under a December 2005 Asset Purchase Agreement, or APA, whereby Xcyte Therapies, Inc., or Xcyte (a business acquired by us in March 2006) sold through the APA and other related agreements certain assets and intellectual property which are not related to our product development plans to ThermoFisher Scientific Company, or TSC.
Other income, net relates to royalties receivable under a December 2005 Asset Purchase Agreement, or APA, whereby Xcyte Therapies, Inc., or Xcyte (a business acquired by us in March 2006) sold through the APA and other related agreements certain assets and intellectual property which are not related to our product development plans to ThermoFisher Scientific Company, or TSC.
Although we are not reliant on institutional credit finance and therefore not subject to debt covenant compliance requirements or potential withdrawal of credit by banks, we are reliant on the availability of funds and activity in equity markets. We do not know whether additional funding will be available on acceptable terms, or at all.
Although we are not reliant on institutional credit finance and therefore not subject to debt covenant compliance requirements or potential withdrawal of credit by banks, we are reliant on the availability of funds and activity in equity markets. We do not know whether additional funding will be available on acceptable terms, or at 68 Table of Contents all.
Current business and capital market risks could have a detrimental effect on the availability of sources of 67 Table of Contents funding and our ability to access them in the future, which may delay or impede our progress of advancing our drugs currently in the clinical pipeline to approval by the FDA or EMA for commercialization.
Current business and capital market risks could have a detrimental effect on the availability of sources of funding and our ability to access them in the future, which may delay or impede our progress of advancing our drugs currently in the clinical pipeline to approval by the FDA or EMA for commercialization.
Prior to becoming aware of the expiration, but following the expiration, we sold an aggregate of 132,473 shares of our common stock at market prices for aggregate proceeds of approximately $2,721,187. The sale of these shares were subject to potential rescission rights by certain stockholders.
Prior to becoming aware of the expiration, but following the expiration, we sold an aggregate of 132,473 shares of our common stock at market prices for aggregate proceeds of approximately $2,721,187. The sale of these shares were subject to potential 70 Table of Contents rescission rights by certain stockholders.
We accrue for unbilled laboratory and manufacturing activities performed by third parties based on estimates of their progress towards completing the requested tasks. As of December 31, 2023, we accrued $3.7 million research and development costs, that we have estimated to have been incurred as of year-end but had not been invoiced.
We accrue for unbilled laboratory and manufacturing activities performed by third parties based on estimates of their progress towards completing the requested tasks. As of December 31, 2024, we accrued $1.3 million research and development costs, that we have estimated to have been incurred as of year-end but had not been invoiced.
This represents approximately 18% of our total research and development expense for the year ended December 31, 2022. When recording these accruals, we must make judgments about the progress of our various clinical activities.
This represents approximately 19% of our total research and development expense for the year ended December 31, 2023. When recording these accruals, we must make judgments about the progress of our various clinical activities.
The fair value of restricted stock and restricted stock units is determined based on the number of shares granted and the quoted price of our common stock on the date of grant.
The fair value of restricted stock and restricted stock units is determined based on the number of 76 Table of Contents shares granted and the quoted price of our common stock on the date of grant.
Changes in any of these variables could result in significant adjustments to the costs recognized for share-based payments. 75 Table of Contents
Changes in any of these variables could result in significant adjustments to the costs recognized for share-based payments.
Accordingly, we presented $50,000 and $1.3 million as other income received from TSC during the years ended December 31, 2023 and 2022 respectively. We have no knowledge of TSC’s activities and cannot predict when we may receive income under the APA, if any.
Accordingly, we presented $52,000 and $50,000 as other income received from TSC during the years ended December 31, 2024 and 2023 respectively. We have no knowledge of TSC’s activities and cannot predict when we may receive income under the APA, if any.
The epigenetic/anti-mitotic program is evaluating plogosertib, a PLK1 inhibitor, in advanced cancers. We currently retain all marketing rights worldwide to the compounds associated with our drug programs. Revenue We have not generated any revenues from product sales to date. Our product candidates will require significant additional research and development efforts, including extensive preclinical and clinical testing.
The epigenetic/anti-mitotic program is evaluating Plogo, a PLK1 inhibitor, in advanced cancers. We currently retain all marketing rights worldwide to our product candidate Plogo. Revenue We have not generated any revenues from product sales to date. Our product candidates will require significant additional research and development efforts, including extensive preclinical and clinical testing.
This represents approximately 19% of our total research and development expense for the year ended December 31, 2023. As of December 31, 2022, we accrued $3.6 million research and development costs, that we have estimated to have been incurred as of year-end but had not been invoiced.
This represents approximately 20% of our total research and development expense for the year ended December 31, 2024. As of December 31, 2023, we accrued $3.7 million research and development costs, that we have estimated to have been incurred as of year-end but had not been invoiced.
Results of Operations Years Ended December 31, 2023 and 2022 Results of Continuing Operations Revenues The following table summarizes the revenues for years ended December 31, 2023 and 2022 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ Difference ​ 2023 2022 ​ $ % Clinical trial supply ​ 420 ​ — ​ 420 ​ 100 Total Revenue ​ $ 420 ​ $ — ​ $ 420 ​ 100 ​ We recognize recognized $420,000 of revenue for the year ended December 31, 2023.
Results of Operations Years Ended December 31, 2024 and 2023 Results of Continuing Operations Revenues The following table summarizes the revenues for years ended December 31, 2024 and 2023 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ Difference ​ 2024 2023 ​ $ % Clinical trial supply ​ 43 ​ 420 ​ (377) ​ (90) Total Revenue ​ $ 43 ​ $ 420 ​ $ (377) ​ (90) ​ We recognize recognized $43,000 of revenue for the year ended December 31, 2024.
Contractual Obligations The following table summarizes our long-term contractual obligations as of December 31, 2023 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Payments Due by Period ​ ​ ​ Less than ​ ​ ​ ​ More than ​ ​ Total ​ 1 year ​ 1 – 3 years ​ 3 – 5 years ​ 5 years Operating Lease Obligations (1) ​ $ 105 ​ $ 67 ​ $ 38 ​ $ — ​ $ — (1) Operating lease obligations relate primarily to leasing office space at our Berkeley Heights, New Jersey location.
Contractual Obligations The following table summarizes our long-term contractual obligations as of December 31, 2024 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Payments Due by Period ​ ​ ​ Less than ​ ​ ​ ​ More than ​ ​ Total ​ 1 year ​ 1 – 3 years ​ 3 – 5 years ​ 5 years Operating Lease Obligations (1) ​ $ 5 ​ $ 5 ​ $ — ​ $ — ​ $ — ​ (1) Operating lease obligations relates to leasing office space at our Berkeley Heights, New Jersey location.
We continue to work to raise additional capital however as of the date of the Consolidated Financial Statements accompanying this Annual Report on Form 10-K, there is no guarantee that we will be able to raise additional funds to extend operations past April 2024.
We continue to work to raise additional capital however as of the date of the Consolidated Financial Statements accompanying this Annual Report on Form 10-K, there is no guarantee that we will be able to raise additional funds to extend operations beyond the second quarter of 2025.
Funding Requirements and Going Concern As of December 31, 2023, we had cash and cash equivalents of $3.4 million We have incurred losses since our inception and as of December 31, 2023, we had an accumulated deficit of $428.3 million.We expect to continue to incur substantial operating losses in the future.
Funding Requirements and Going Concern As of December 31, 2024, we had cash and cash equivalents of $3.1 million We have incurred losses since our inception and as of December 31, 2024, we had an accumulated deficit of $439.5 million. We expect to continue to incur substantial operating losses in the future.
Favorable unrealized foreign exchange movements related to intercompany loans resulted in a gain of $12.9 million for the year ended December 31, 2023 compared to a loss of $21.2 million for the year ended December 31, 2022.
Unfavorable unrealized foreign exchange movements related to intercompany loans resulted in a loss of $2.9 million for the year ended December 31, 2024 compared to a gain of $12.6 million for the year ended December 31, 2023.
This decrease was primarily due to a decrease in clinical trial costs of $0.2 million associated with the progression of clinical trials for the evaluation of plogosertib in Phase 1/2 studies, and a decrease in manufacturing and other non-clinical expenditure of $0.4 million.
This decrease was primarily due to a decrease in clinical trial costs of $1.6 million associated with the progression of clinical trials for the evaluation of Plogo in Phase 1/2 studies, a decrease in manufacturing costs of $0.5 million, employment costs of $0.6 million and other non-clinical expenditure of $0.7 million.
This revenue relates to recovery of clinical manufacturing costs associated with an investigator sponsored study managed by Cedars-Sinai Medical Center. There were no revenues recognized for the comparative periods in 2022. We do not expect to report revenue for the foreseeable future. Research and development We expense all research and development costs as they are incurred.
This revenue relates to recovery of clinical manufacturing costs associated with an investigator sponsored study managed by Cedars-Sinai Medical Center. We recognized $420,000 of revenue for the comparative period in 2023. We do not expect to report revenue for the foreseeable future. Research and development We expense all research and development costs as they are incurred.
Pursuant to the Securities Purchase Agreement, we agreed to sell in a registered direct offering (“Registered Direct Offering”) 168,500 shares (“Shares”) of our common stock, $0.001 par value per share ( “Common Stock”), and pre-funded warrants (“Pre-Funded Warrants”) to purchase up to 219,700 shares of Common Stock.
Pursuant to the Securities Purchase Agreement, we agreed to sell in a registered direct offering (“Registered Direct Offering”) 168,500 shares (“Shares”) of our common stock and pre-funded warrants (“Pre-Funded Warrants”) to purchase up to 219,700 shares of common stock.
All product candidates that we advance to clinical testing will require regulatory approval prior to commercial use and will require significant costs for commercialization. We have recognized revenue of $0.4 million for the year ended December 31, 2023 related to the recovery of clinical manufacturing costs associated with an investigator sponsored study managed by Cedars Sinai Medical Center.
Plogo and any future product candidates that we advance to clinical testing will require regulatory approval prior to commercial use and will require significant costs for commercialization. We have recognized revenue of $43,000 for the year ended December 31, 2024 related to the recovery of clinical manufacturing costs associated with an investigator sponsored study managed by Cedars Sinai Medical Center.
Based on our current operating plan, we anticipate that our cash and cash equivalents of $3.4 million as of December 31, 2023 will allow us to meet our liquidity requirements into April of 2024. As of March 21, 2024 our cash balance on hand was approximately $3.4 million.
Based on our current operating plan, we anticipate that our cash and cash equivalents of $3.1 million as of December 31, 2024, will allow us to meet our liquidity requirements into the second quarter of 2025. As of March 24, 2025, our cash balance on hand was approximately $3.5 million.
This decrease was primarily due to a decrease in clinical trial costs of $2.2 million associated with the temporary halt in the Phase 1/2 study in hematological malignancies and the completion of a bioequivalence study during the prior year, offset by an increase in manufacturing and other non-clinical expenditure of $1.6 million.
This decrease was primarily due to a decrease in clinical trial costs of $2.4 million associated with the temporary halt in the Phase 1/2 study in hematological malignancies, the completion of a bioequivalence and tox studies during the prior year of $2.1 million, reduction in manufacturing costs of $3.5 million and other non-clinical expenditure of $0.4 million.
Financing activities Net cash provided by financing activities was $0.8 million for the year ended December 31, 2023 as a direct result of receiving approximately: - $1.0 million in net proceeds from the issuance of common stock and pre-funded warrants pursuant to the Registered Direct Offering, - offset by dividend payments of approximately $0.2 million to the holders of our 6% Convertible Exchangeable Preferred Stock.
Financing activities Net cash provided by financing activities was $7.8 million for the year ended December 31, 2024 as a direct result of receiving approximately: - $6.2 million, net of expenses, from the issuance of common stock and warrants under a Securities Purchase Agreement with an institutional investor, - $1.6 million in net proceeds from a warrant exercise and reload agreement ​ Net cash provided by financing activities was $0.8 million for the year ended December 31, 2023 as a direct result of receiving approximately: - $1.0 million in net proceeds from the issuance of common stock and pre-funded warrants pursuant to the Registered Direct Offering, - offset by dividend payments of approximately $0.2 million to the holders of our 6% Convertible Exchangeable Preferred Stock in 2023 that were not paid in 2024.
Research and development expenses decreased by $1.1 million from $20.3 million for the year ended December 31, 2022 to $19.2 million for the year ended December 31, 2023. Expenditure for the transcriptional regulation program decreased by $0.7 million for the year ending December 31, 2023 relative to the respective comparative period.
Research and development expenses decreased by $12.5 million from $19.2 million for the year ended December 31, 2023 to $6.7 million for the year ended December 31, 2024. Expenditure for the transcriptional regulation program decreased by $8.4 million for the year ending December 31, 2024 relative to the respective comparative period.
Research and development expenses relating to plogosertib decreased by $0.6 million for the year ending December 31, 2023 relative to the respective comparative period.
Research and development expenses relating to Plogo decreased by $3.4 million for the year ending December 31, 2024 relative to the respective comparative period.
The following table summarizes the total general and administrative expenses for the years ended December 31, 2023 and 2022 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ ​ December 31, ​ Difference ​ 2023 2022 $ % Total general and administrative expenses ​ $ 6,718 ​ $ 7,382 ​ $ (664) ​ (9) ​ Total general and administrative expenses represented 26% and 27% of our operating expenses for the years ended December 31, 2023 and 2022, respectively.
The following table summarizes the total general and administrative expenses for the years ended December 31, 2024 and 2023 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ December 31, ​ Difference ​ 2024 2023 $ % Total general and administrative expenses $ 5,392 ​ $ 6,718 ​ $ (1,326) ​ (20) ​ Total general and administrative expenses represented 45% and 26% of our operating expenses for the years ended December 31, 2024 and 2023, respectively.
A cash receipt of approximately $4.8 million in research and development tax credit was received during the year ended December 31, 2023. Investing activities Net cash used in investing activities decreased by $1,000 for the year ended December 31, 2023 due to a slight decrease in capital expenditures on information technology (“IT”) during the respective comparative period.
A cash receipt of approximately $3.7 million in research and development tax credit was received during the year ended December 31, 2024. Investing activities There was no net cash used in investing activities for the year ended December 31, 2024 and $6,000 in capital expenditures on information technology (“IT”) during the respective comparative period.
Foreign exchange gains (losses) Foreign exchange gains decreased by $0.6 million to a loss of $0.4 million for the year ended December 31, 2023 compared to a gain of approximately $0.2 million for the year ended December 31, 2022. We have intercompany loans in place between our parent company based in New Jersey and our subsidiary based in Scotland.
Foreign exchange losses Foreign exchange losses increased by $360,000 to a loss of $54,000 for the year ended December 31, 2024 compared to a loss of $414,000 for the year ended December 31, 2023. We have intercompany loans in place between our parent company based in New Jersey and our subsidiary based in Scotland.
The level of tax credits recoverable is linked directly to qualifying research and development expenditure incurred in any one year and the availability of trading losses.
The $0.8 million tax benefit in 2024 relates to a deferred claim based on 2023 qualifying research and development expenditure. The level of tax credits recoverable is linked directly to qualifying research and development expenditure incurred in any one year and the availability of trading losses.
The decrease in cash used by operating activities was primarily the result of a change in working capital of $6.3 million, offset by an increase in net loss of $1.3 million. The $6.3 million change in working capital was primarily due to decreased balances in clinical trial deposits and receivables for research and development tax credits.
The decrease in cash used by operating activities was primarily the result of a decrease in net loss of $11.3 million, offset by a change in working capital of $2.3 million and stock based compensation of $0.9 million. The $2.3 million change in working capital was primarily due to receivables for research and development tax credits.
During the third quarter of 2023, upon expiration of the rescission rights and with no claims or demands to exercise such rights, we reclassified all 207,807 shares back to permanent equity.
During the third quarter of 2023, upon expiration of the rescission rights and with no claims or demands to exercise such rights, we reclassified all 207,807 shares back to permanent equity. In all periods presented, the shares subject to the rescission rights were treated as issued and outstanding for purposes of earnings per share and general financial reporting.
The future We anticipate that overall research and development expenses for the year ended December 31, 2024 will decrease compared to the year ended December 31, 2023 as we focus on our Phase 1/2 programs in advanced solid tumors and lymphomas.
The future We anticipate that overall research and development expenses for the year ended December 31, 2025 will decrease significantly compared to the year ended December 31, 2024 as we focus on our Plogo clinical program.
Liquidity and Capital Resources The following is a summary of our key liquidity measures as of December 31, 2023 and 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, ​ 2023 2022 Cash and cash equivalents ​ $ 3,378 ​ $ 18,345 Working capital: ​ ​ ​ ​ ​ ​ Current assets ​ $ 7,444 ​ $ 24,411 Current liabilities ​ (8,161) ​ (7,511) Total working capital ​ $ (717) ​ $ 16,900 ​ Cash Flows Cash provided by (used in) operating, investing and financing activities for the years ended December 31, 2023 and 2022 is summarized as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ 2023 2022 Net cash used in operating activities ​ $ (16,112) ​ $ (20,827) Net cash used in investing activities ​ (6) ​ (7) Net cash provided by financing activities ​ 848 ​ 2,998 ​ 73 Table of Contents Operating activities Net cash used in operating activities decreased by $4.7 million, from $20.8 million for the year ended December 31, 2022 to $16.1 million for the year ended December 31, 2023.
The future We do not expect to continue to be eligible to receive United Kingdom research and development tax credits for the year ending December 31, 2025 Liquidity and Capital Resources The following is a summary of our key liquidity measures as of December 31, 2024 and 2023 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, ​ 2024 2023 Cash and cash equivalents ​ $ 3,137 ​ $ 3,378 Working capital: ​ ​ ​ ​ ​ ​ Current assets ​ $ 3,674 ​ $ 7,444 Current liabilities ​ (6,268) ​ (8,161) Total working capital deficit ​ $ (2,594) ​ $ (717) ​ Cash Flows Cash provided by (used in) operating, investing and financing activities for the years ended December 31, 2024 and 2023 is summarized as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ 2024 2023 Net cash used in operating activities ​ $ (7,990) ​ $ (16,112) Net cash used in investing activities ​ — ​ (6) Net cash provided by financing activities ​ 7,822 ​ 848 ​ 74 Table of Contents Operating activities Net cash used in operating activities decreased by $8.1 million, from $16.1 million for the year ended December 31, 2023 to $8.0 million for the year ended December 31, 2024.
Off-Balance Sheet Arrangements Since our inception, we have not had any off-balance sheet arrangements or relationships with unconsolidated entities or financial partnerships, such as entities often referred to as structured finance or variable interest entities, which are typically established for the purpose of facilitating off-balance sheet arrangements or other contractually narrow or limited purposes.
Off-Balance Sheet Arrangements Since our inception, we have not had any off-balance sheet arrangements or relationships with unconsolidated entities or financial partnerships, such as entities often referred to as structured finance or variable interest entities, which are typically established for the purpose of facilitating off-balance sheet arrangements or other contractually narrow or limited purposes. 75 Table of Contents Recently Issued Accounting Pronouncements Please see Note 2 to the consolidated financial statements for a discussion of the potential effects that recently issued, but not yet effective, accounting standards will have on our financial statements when adopted in a future period.
We did not have any revenue for the year ended December 31, 2022 and do not expect to report revenue for the foreseeable future.
We recognized $0.4 million of revenue for the year ended December 31, 2023. We do not expect to report revenue for the foreseeable future.
Recently Issued Accounting Pronouncements Please see Note 2 to the consolidated financial statements for a discussion of the potential effects that recently issued, but not yet effective, accounting standards will have on our financial statements when adopted in a future period. 74 Table of Contents Critical Accounting Policies and Estimates Our discussion and analysis of our financial condition and results of operations is based on our financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States.
Critical Accounting Policies and Estimates Our discussion and analysis of our financial condition and results of operations is based on our financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States.
All share and per share information has been retroactively adjusted to give effect to the Reverse Stock Split for all periods presented, unless otherwise indicated. Overview We are a clinical-stage biopharmaceutical company developing innovative cancer medicines based on cell cycle, transcriptional regulation, epigenetics and mitosis control biology.
All share and per share information has been retroactively adjusted to give effect to the Reverse Stock Split for all periods presented, unless otherwise indicated.
Credit is taken for research and development tax credits, which are claimed from the United Kingdom’s taxation and customs authority (HMRC), in respect of qualifying research and development costs incurred. 72 Table of Contents The following table summarizes total income tax benefit from such credits for the years ended December 31, 2023 and 2022 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ ​ December 31, ​ Difference ​ ​ 2023 2022 $ % Total income tax benefit ​ $ 2,996 ​ $ 4,717 ​ $ (1,721) ​ (36) ​ The income tax benefit decreased significantly by approximately $1.7 million, from $4.7 million for the year ended December 31, 2022 to $3.0 million for the year ended December 31, 2023, due to legislative changes that took effect in April 2023.
The following table summarizes total income tax benefit from such credits for the years ended December 31, 2024 and 2023 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ December 31, ​ Difference ​ 2024 2023 $ % Total income tax benefit $ 782 ​ $ 2,996 ​ $ (2,214) ​ (74) ​ The income tax benefit decreased significantly by approximately $2.2 million, from $3.0 million for the year ended December 31, 2023 to $0.8 million for the year ended December 31, 2024, due to the ineligibility to recover qualifying research and developments expenditure incurred during 2024.
Our general and administrative expenditures decreased by $0.7 million from $7.4 million for the year ended December 31, 2022 to $6.7 million for the year ended December 31, 2023. This decrease was primarily due to a non-recurring $0.4 million cost associated with the Controlled Equity Offering Sales Agreement with Cantor Fitzgerald & Co.
Our general and administrative expenditures decreased by $1.3 million from $6.7 million for the year ended December 31, 2023 to $5.4 million for the year ended December 31, 2024.
The future We expect general and administrative expenditures for the year ended December 31, 2024 to remain relatively flat compared to the year ended December 31, 2023. 71 Table of Contents Other income (expense), net The following table summarizes the other income (expense) for years ended December 31, 2023 and 2022 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ ​ December 31, ​ Difference ​ ​ 2023 2022 $ % Foreign exchange gains (losses) ​ $ (414) ​ $ 233 ​ $ (647) ​ (278) Interest income ​ 266 ​ 210 ​ 56 ​ 27 Other income (expense), net ​ 50 ​ 1,298 ​ (1,248) ​ (96) Total other income (expense), net ​ $ (98) ​ ​ 1,741 ​ $ (1,839) ​ (106) ​ Total other income, net, decreased by approximately $1.8 million from an income of approximately $1.7 million for the year ended December 31, 2022 to an expense of approximately $0.1 million for the year ended December 31, 2023.
Other expense, net The following table summarizes the other income (expense) for years ended December 31, 2024 and 2023 (in thousands except percentages): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended ​ December 31, ​ Difference ​ 2024 2023 $ % Foreign exchange losses $ (54) ​ $ (414) ​ $ 360 ​ (87) Interest income 12 ​ 266 ​ (254) ​ (95) Other income, net 52 ​ 50 ​ 2 ​ 4 Total other income (expense), net $ 10 ​ ​ (98) ​ $ 108 ​ (110) ​ Total other expense, net, increased by $108,000 from an expense of $98,000 for the year ended December 31, 2023 to an income of $10,000 for the year ended December 31, 2024.
Agreements to Sell Securities On December 21, 2023, we entered into a securities purchase agreement (the “Securities Purchase Agreement”) with certain institutional investors (the “Purchasers”).
In connection with the Private Placement, the Company entered into a registration rights agreement (the “Registration Rights Agreement”), dated as of April 30, 2024, with the Purchaser, pursuant to which the Company agreed to prepare and file a registration statement with the Securities and Exchange Commission (the “SEC”) registering the resale of the securities issued in the Private Placement. ​ On December 21, 2023, we entered into a securities purchase agreement (the “Securities Purchase Agreement”) with certain institutional investors (the “Purchasers”).
We reported revenue of $0.4 million for the year ended December 31, 2023 and no revenues for the year ended December 31, 2022. We do not expect to report revenue for the foreseeable future. During 2023, our primary focus has been on our transcriptional regulation program, which is evaluating fadraciclib, a CDK2/9 inhibitor, in solid tumors and hematological malignancies .
The information contained on, or that can be accessed through, our website is not part of, and is not incorporated by reference into, this Annual Report. 67 Table of Contents During 2024, our primary focus has been on our transcriptional regulation program, which evaluated fadraciclib, a CDK2/9 inhibitor, in solid tumors and hematological malignancies .
General and administrative General and administrative expenses include costs for administrative personnel, legal and other professional expenses and general corporate expenses.
There will be no expenditure related to fadraciclib as the program is being marketed for sale by the joint liquidator of the Subsidiary. ​ General and administrative General and administrative expenses include costs for administrative personnel, legal and other professional expenses and general corporate expenses.
Removed
In all periods presented, the shares subject to the rescission rights were treated as issued and outstanding for purposes of earnings per share and general financial reporting. 69 Table of Contents On August 15, 2022, due to expiry of the Registration Statement, the Sales Agreement was mutually terminated.
Added
Recent developments In December 2024 the Company announced that it was in the process of exploring and reviewing strategic alternatives on an expedited basis in order to preserve the Company’s cash, including a potential transaction with investor David Lazar of Activist Investing, LLC, or “Lazar”.
Removed
A total of 218,738 shares, for gross proceeds of approximately $7.6 million, had been sold pursuant to the Sales Agreement, including 168,576 shares during the year ended December 31, 2022 for gross proceeds of approximately $3.4 million.
Added
The Company’s Board of Directors (the “Board”) reviewed a range of appropriate strategies to realize value from its assets. The Board directed management to reduce operating costs, which included the potential liquidation of the Company’s wholly owned United Kingdom subsidiary Cyclacel Limited, or Subsidiary, while such alternatives were being explored.
Removed
Dividend on Preferred Stock On January 12, 2024, the Board of Directors declared a quarterly cash dividend in the amount of $0.15 per share on our 6% Convertible Exchangeable Preferred Stock.
Added
On January 2, 2025 the Company entered into a securities purchase agreement with investor Lazar, pursuant to which he agreed to purchase from the Company 1,000,000 shares of Series C Convertible Preferred Stock and 2,100,000 shares of Series D Convertible Preferred Stock of Cyclacel at a purchase price of $1.00 per share for aggregate gross proceeds of $3.1 million, subject to the terms and conditions of the Agreement.
Removed
The cash dividend of approximately $50,000 was paid on February 1, 2024 to the holders of record of the 6% Convertible Exchangeable Preferred Stock as of the close of business on January 22, 2024.
Added
The proceeds of the transaction will be used to settle outstanding liabilities of the Company and other general corporate and operating purposes.
Removed
(the “Sales Agreement”) in the comparative prior period.
Added
On January 2, 2025 the Company entered into a securities purchase agreement with investor Lazar, pursuant to which he agreed to purchase from the Company 1,000,000 shares of Series C Convertible Preferred Stock and 2,100,000 shares of Series D Convertible Preferred Stock of Cyclacel at a purchase price of $1.00 per share for aggregate gross proceeds of $3.1 million, subject to the terms and conditions of the Agreement.
Removed
As the funding advanced through intercompany loans is that of a long-term investment in nature, unrealized foreign exchange gains and losses on such funding will be recognized in other comprehensive income (loss) until repayment of the intercompany loan becomes foreseeable. Income tax benefit We record research and development tax credits within income taxes.
Added
The proceeds of the transaction will be used to settle outstanding liabilities of the Company and other general corporate and operating purposes. On January 2, 2025, the Company entered into settlement agreements with the Resigning Directors effective as of the signing of the Purchase Agreement.
Removed
The future We expect to continue to be eligible to receive United Kingdom research and development tax credits for the year ending December 31, 2024 and will continue to elect to receive payment of the tax credit.
Added
Pursuant to the terms of the Director Settlement Agreements, each Resigning Director resigned his or her position as a member of the Board of Directors, and any positions held on committees of the Board of Directors.
Removed
The legislative changes which took effect in April 2023 have been partially reversed in early 2024 resulting in additional income tax benefit of $0.8 million in relation to expenditure incurred during the year ended December 31, 2023 and receivable during the second quarter of 2024.
Added
Each Resigning Director has received his or her accrued Board fees in full consideration of the release of claims against the Company and other promises and covenants set forth in the Director Settlement Agreements. On January 2, 2025, the Company entered into a settlement agreement with Mr. Spiro Rombotis (the “Rombotis Settlement Agreement”).
Removed
Beyond 2024, there is no guarantee that we will be eligible to receive this tax credit or if eligible, any amount that may be receivable due to proposed changes by HMRC to the eligibility criteria would be correctly estimated.
Added
Pursuant to the terms of the Rombotis Settlement Agreement, Mr.
Removed
We expect research and development tax credits for the year ending December 31, 2024 to be lower compared to the year ended December 31, 2023.
Added
Rombotis resigned his position as President and Chief Executive Officer of the Company effective as of the signing of the Purchase Agreement, and agreed to provide transition services to the Company in his capacity as a member of the Board of Directors through 66 Table of Contents the filing of the Company’s Annual Report on Form 10-K for the year ended 2024.
Removed
Net cash provided by financing activities was $3.0 million for the year ended December 31, 2022 as a direct result of receiving approximately: - $3.2 million in net proceeds from the issuance of common stock under a Controlled Equity Offering Sales Agreement with Cantor Fitzgerald & Co., - offset by dividend payments of approximately $0.2 million to the holders of our 6% Convertible Exchangeable Preferred Stock.
Added
On January 2, 2025, the Company also entered into a settlement agreement with Paul McBarron (together with Mr.
Removed
The lease for our Berkeley Heights location, which was entered into in April 2022, expires in July 2025. The table does not include any amouns relating to a short term lease for offices in Dundee, Scotland.
Added
Rombotis, the “Resigning Officers”) effective immediately following the Initial Closing, as such term is defined in the Purchase Agreement (the “McBarron Settlement Agreement” and together with the Rombotis Settlement Agreement, the “Executive Officer Settlement Agreements” and together with the Director Settlement Agreements, the “Settlement Agreements”). Pursuant to the terms of the McBarron Settlement Agreement, Mr.
Added
McBarron agreed to provide transition services to the Company in his capacity as a member of the Board of Directors through the filing of the Company’s Annual Report on Form 10-K for the year ended 2024. Pursuant to the Executive Officer Settlement Agreements, and subject to the Purchase Agreement, the Company will pay to Mr. Rombotis and Mr.
Added
McBarron payments of $279,415.50 and $165,164.50, respectively, as soon as practicable, and three months later a further one-time payment of $279,415.50 and $165,164.50 either in cash or through the issuance of common stock, respectively, in full consideration of the release of claims against the Company and other promises and covenants set forth in the Executive Officer Settlement Agreements (the “Settlement Payments”) and the Purchase Agreement.
Added
Pursuant to the terms of the Settlement Agreements, the Company will provide continuing indemnification to the Resigning Directors and Resigning Officers in a manner consistent with that which was in place as of the effective date of the Settlement Agreements, and will cause to be maintained in effect the Company’s existing director and officer liability insurance pursuant to the Company’s tail insurance coverage and will not modify its governing documents to modify the Resigning Directors’ and Resigning Officers’ rights under such policy, as further set forth in the Settlement Agreements.
Added
The Settlement Agreements contain a mutual non-disparagement clause. On January 31, 2025, the creditors voluntary liquidation of Cyclacel Limited was announced in the London Gazette, one of the official public records of the government of the United Kingdom.
Added
As part of the Company’s efforts to reduce operating costs it has determined to focus on the development of the Plogo clinical program only and therefore fadraciclib, the Subsidiary’s other drug development program, is being marketed for sale by the joint liquidator through Hilco Appraisals Limited, a firm of professional valuation agents and will no longer be part of the assets of the Company as of January 2025.
Added
On March 10, 2025, the Company entered into an Agreement for the Sale and Purchase of certain assets related to plogosertib (“Plogo”) with Cyclacel Limited and the joint liquidator. ​ On February 26, 2025, the Company entered into settlement agreements with Dr. Barker. Pursuant to the terms of the settlement agreement, Dr.
Added
Barker resigned his position as a member of the Board of Directors, and any positions held on committees of the Board of Directors. In addition, Dr. Barker will receive his accrued Board fees in full consideration of the release of claims against the Company and other promises and covenants set forth in the settlement agreement.
Added
Pursuant to the terms of the settlement agreement, the Company will provide continuing indemnification to Dr.
Added
Barker in a manner consistent with that which was in place as of the effective date of the settlement agreement, and will cause to be maintained in effect the Company’s existing director and officer liability insurance pursuant to the Company’s tail insurance coverage and will not modify its governing documents to modify Dr.

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