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What changed in Inhibikase Therapeutics, Inc.'s 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of Inhibikase Therapeutics, Inc.'s 2024 and 2025 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 2025 report.

+581 added−586 removedSource: 10-K (2026-03-26) vs 10-K (2025-03-27)

Top changes in Inhibikase Therapeutics, Inc.'s 2025 10-K

581 paragraphs added · 586 removed · 384 edited across 8 sections

Item 1. Business

Business — how the company describes what it does

86 edited+66 added−52 removed164 unchanged
Biggest changeSecurities and Exchange Commission (the “SEC”). • In May 2024, we completed a registered direct offering and raised gross proceeds of $4,000,000 through the sale of our Common Stock and Common Stock equivalents and warrants. • In October 2024, we completed a private placement and raised gross proceeds of $110,000,000 through the sale of our Common Stock, Common Stock equivalents and warrants. • In January 2025, we reported results from the 201 Trial and decided to pause further development of risvodetinib as we focus our resources on advancing lead program IKT-001. • In February 2025, we entered into an Agreement and Plan of Merger and Reorganization (“Merger Agreement”) and acquired CorHepta Pharmaceuticals, Inc., a Delaware corporation (“CorHepta”), to expand our product pipeline and to strengthen our scientific leadership.
Biggest changeIn 2020, we completed an Initial Public Offering and listed the Company's shares of common stock ( “Common Stock”) on Nasdaq under the symbol “IKT.” Key recent operational and financing milestones are: • In January 2025, we reported results from the Phase 2 201 trial (“201 Trial”) and decided to pause further development of risvodetinib in favor of focusing our resources on advancing IKT-001. • In February 2025, we entered into an Agreement and Plan of Merger and Reorganization (“Merger Agreement”) and acquired CorHepta Pharmaceuticals, Inc., a Delaware corporation (“CorHepta”), to expand our product pipeline and to strengthen our scientific leadership.
Material Agreements Sphaera Pharma Pte. Ltd. On March 2, 2012, we entered into a collaborative research and development agreement (“Sphaera Agreement”), with Sphaera Pharma Pte. Ltd. (“Sphaera”), to collaborate on the development of the prodrug technology to be applied to protein kinase inhibitors for oncology and non-oncology indications.
Material Agreements Collaborative Research and Development Agreement with Sphaera Pharma Pte. Ltd. On March 2, 2012, we entered into a collaborative research and development agreement (“Sphaera Agreement”), with Sphaera Pharma Pte. Ltd. (“Sphaera”), to collaborate on the development of prodrug technology to be applied to protein kinase inhibitors for oncology and non-oncology indications.
The parties agreed that no further FDA Approval milestone payment(s) shall be due to Pivot in the event that we receive additional FDA Approval(s). The prosecution of patents related to the Company Compounds, which includes the prodrug technology, is the responsibility the Company.
The parties agreed that no further FDA Approval milestone payment(s) shall be due to Pivot in the event that we receive additional FDA Approval(s). The prosecution of patents related to the Company Compounds, which includes the prodrug technology, is the responsibility of the Company.
Surrogate endpoints can often be measured more easily or more rapidly than clinical endpoints. An intermediate clinical endpoint is an measurement of a therapeutic effect that is considered reasonably likely to predict the clinical benefit of a drug, such as an effect on IMM.
Surrogate endpoints can often be measured more easily or more rapidly than clinical endpoints. An intermediate clinical endpoint is a measurement of a therapeutic effect that is considered reasonably likely to predict the clinical benefit of a drug, such as an effect on IMM.
Individually identifiable health information is considered sensitive data that merits stronger safeguards; • federal government price reporting laws, which require us to calculate and report complex pricing metrics in an accurate and timely manner to government programs; • the federal Physician Payments Sunshine Act requirements, under the Patient Protection and Affordable Care Act (“ACA”), which require manufacturers of certain drugs and biologics to track and report to Centers for Medicare & Medicaid Services (“CMS”), payments and other transfers of value they make to U.S. physicians and teaching hospitals as well as physician ownership and investment interests in the manufacturer; and • analogous state and foreign laws and regulations, such as state and foreign anti-kickback, false claims, consumer protection and unfair competition laws which may apply to pharmaceutical business practices, including but not limited to, research, distribution, sales, and marketing arrangements as well as submitting claims involving healthcare items or services reimbursed by any third-party payor, including commercial insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government that otherwise restricts payments that may be made to healthcare providers and other potential referral sources; state laws that require drug manufacturers to file reports with states regarding pricing and marketing information, such as the tracking and reporting of gifts, compensations and other remuneration and items of value provided to healthcare professionals and entities; and state and local laws requiring the registration of pharmaceutical sales representatives.
Individually identifiable health information is considered sensitive data that merits stronger safeguards; • federal government price reporting laws, which require us to calculate and report complex pricing metrics in an accurate and timely manner to government programs; • the federal Physician Payments Sunshine Act requirements, under the Patient Protection and Affordable Care Act (“ACA”), which require manufacturers of certain drugs and biologics to track and report to Centers for Medicare & Medicaid Services (“CMS”), payments and other transfers of value they make to U.S. physicians, other licensed healthcare professionals, and teaching hospitals as well as physician ownership and investment interests in the manufacturer; and • analogous state and foreign laws and regulations, such as state and foreign anti-kickback, false claims, consumer protection and unfair competition laws which may apply to pharmaceutical business practices, including but not limited to, research, distribution, sales, and marketing arrangements as well as submitting claims involving healthcare items or services reimbursed by any third-party payor, including commercial insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government that otherwise restricts payments that may be made to healthcare providers and other potential referral sources; state laws that require drug manufacturers to file reports with states regarding pricing and marketing information, such as the tracking and reporting of gifts, compensations and other remuneration and items of value provided to healthcare professionals and entities; and state and local laws requiring the registration of pharmaceutical sales representatives.
The centralized procedure is optional for products containing a new active substance not yet authorized in the EU, or for products that constitute a significant therapeutic, scientific or technical innovation or which are in the interest of public health in the EU; and • National MAs, which are issued by the competent authorities of the Member States of the EU and only cover their respective territory, are available for products not falling within the mandatory scope of the centralized procedure.
The centralized procedure is optional for products containing a new active substance not yet authorized in the EU, or for products that constitute a significant therapeutic, scientific or technical innovation or which are in the interest of public health in the EU; and 15 • National MAs, which are issued by the competent authorities of the Member States of the EU and only cover their respective territory, are available for products not falling within the mandatory scope of the centralized procedure.
For more information regarding the risks related to our intellectual property, see “Risk Factors - Risks Related to Our Intellectual Property.” The patent positions of pharmaceutical companies like ours are generally uncertain and involve complex legal, scientific and factual questions. Our commercial success will also depend in part on not infringing upon the proprietary rights of third parties.
For more information regarding the risks related to our intellectual property, see “Risk Factors — Risks Related to Our Intellectual Property.” 8 The patent positions of pharmaceutical companies like ours are generally uncertain and involve complex legal, scientific and factual questions. Our commercial success will also depend in part on not infringing upon the proprietary rights of third parties.
Accordingly, manufacturers must continue to expend time, money, and effort in the area of production and quality control to maintain cGMP compliance. Once an approval of a drug or medical device 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.
Accordingly, manufacturers must continue to expend time, money, and effort in the area of production and quality control to maintain cGMP compliance. 13 Once an approval of a drug or medical device 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.
The FDCA also provides three years of marketing exclusivity for an NDA, 505(b)(2) NDA or supplement to an existing NDA if new clinical investigations, other than bioavailability studies, that were conducted or sponsored by the applicant are deemed by 17 the FDA to be essential to the approval of the application, for example, new indications, dosages or strengths of an existing drug.
The FDCA also provides three years of marketing exclusivity for an NDA, 505(b)(2) NDA or supplement to an existing NDA if new clinical investigations, other than bioavailability studies, that were conducted or sponsored by the applicant are deemed by the FDA to be essential to the approval of the application, for example, new indications, dosages or strengths of an existing drug.
The FDA reviews an NDA to determine, among other things, whether the drug is safe and effective and whether the facility in which it is manufactured, processed, packaged or held meets standards designed to assure the product’s continued safety, quality and purity. The FDA may refer an application for a novel drug to an advisory committee.
The FDA reviews an NDA to determine, among other things, whether the drug is safe and effective and whether the facility in which it is manufactured, processed, packaged or held meets standards designed to assure the product’s continued safety, quality and purity. 10 The FDA may refer an application for a novel drug to an advisory committee.
Other Agreements Consulting Agreements Our non-employee Directors, non-employee management and non-employee technical staff have signed multi-year consulting agreements that provide for protections of intellectual property, trade secrets and ensure consistent commitment to Company research and development activities. These agreements provide a scope of work, reimbursement for incurred costs of travel and equity compensation.
Consulting Agreements Our non-employee Directors, non-employee management and non-employee technical staff have signed multi-year consulting agreements that provide for protections of intellectual property, trade secrets and ensure consistent commitment to Company research and development activities. These agreements provide a scope of work, reimbursement for incurred costs of travel and equity compensation.
For more information, see “Risk Factors - Risks Related to Our Intellectual Property.” 8 Government Regulation In the United States, the FDA regulates drugs under the Federal Food, Drug, and Cosmetic Act, (“FDCA”), and its implementing regulations.
For more information, see “Risk Factors - Risks Related to Our Intellectual Property.” Government Regulation In the United States, the FDA regulates drugs under the Federal Food, Drug, and Cosmetic Act, (“FDCA”), and its implementing regulations.
The FTC expects a company’s data security measures to be reasonable and appropriate in light of the sensitivity and volume of consumer information it holds, the size and complexity of its business and the cost of available tools to improve security and reduce vulnerabilities.
The FTC expects a company’s data security measures to be reasonable and appropriate in light of the sensitivity and volume of consumer information it holds, the size and complexity of its business and the cost of available tools to improve security 14 and reduce vulnerabilities.
Human clinical trials are typically conducted in three sequential phases, which may overlap or be combined: • Phase 1: The drug is initially introduced into healthy human subjects or patients with the target disease or condition and tested for safety, dosage tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an early indication of its effectiveness. 9 • Phase 2: The drug is administered to a limited patient population to identify possible adverse effects and safety risks, to preliminary evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance and optimal dosage. • Phase 3: The drug is administered to an expanded patient population, generally at geographically dispersed clinical trial sites, in well-controlled clinical trials to generate enough data to statistically evaluate the efficacy and safety of the product for approval, to establish the overall risk-benefit profile of the product, and to provide adequate information for the labeling of the product.
Human clinical trials are typically conducted in three sequential phases, which may overlap or be combined: • Phase 1: The drug is initially introduced into healthy human subjects or patients with the target disease or condition and tested for safety, dosage tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an early indication of its effectiveness. • Phase 2: The drug is administered to a limited patient population to identify possible adverse effects and safety risks, to preliminarily evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance and optimal dosage. • Phase 3: The drug is administered to an expanded patient population, generally at geographically dispersed clinical trial sites, in well-controlled clinical trials to generate enough data to statistically evaluate the efficacy and safety of the product for approval, to establish the overall risk-benefit profile of the product, and to provide adequate information for the labeling of the product.
During this market exclusivity period, the EMA or the EU Member State competent authorities, cannot accept another application for a marketing authorization, or grant a marketing authorization, for a similar medicinal product for the same indication as the authorized orphan product.
During this market exclusivity period, the EMA and the EU Member State competent authorities, cannot accept another application for a marketing authorization, or grant a marketing authorization, for a similar medicinal product for the same indication as the authorized orphan product.
The period of market exclusivity is extended by two years for orphan medicines that have also complied with an agreed pediatric investigational plan. 15 This period may, however, 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 drug designation, for example because the product is sufficiently profitable not to justify market exclusivity.
The period of market exclusivity is extended by two years for orphan medicines that have also complied with an agreed pediatric investigational plan. 16 This period may, however, 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 drug designation, for example because the product is sufficiently profitable not to justify market exclusivity.
On January 19, 2024, we met with the Food and Drug Administration (“FDA”) Hematological Malignancy Review Team (“Review Team”) in a Pre-New Drug Application (“pre-NDA”), meeting to discuss our bioequivalence studies of IKT-001 and its path to approval. All questions were addressed and summarized in official meeting minutes issued by the FDA on February 12, 2024.
On January 19, 2024, we met with the Food and Drug Administration (“FDA”) Hematological Malignancy Review Team (“Review Team”) in a Pre-New Drug Application (“pre-NDA”) to discuss our bioequivalence studies of IKT-001 and the potential path to approval. All questions were addressed and summarized in official meeting minutes issued by the FDA on February 12, 2024.
The study was designed to evaluate the 96-hour single-dose pharmacokinetics of imatinib delivered as IKT-001 and determine the dose relationship between IKT-001 and imatinib mesylate.
The study was designed to evaluate the 96-hour single-dose pharmacokinetics of imatinib delivered as IKT-001 and determine the dose relationship between IKT-001 and imatinib.
In sum, other than the three U.S. federal registrations noted above and the registrations in the ex-US territories listed above, we have not secured trademark protection for any of our trademarks or trade names in any of our other geographic markets, and failure to secure those registrations could adversely affect our business.
In sum, other than the two U.S. federal registrations noted above and the registrations in the ex-US territories listed above, we have not secured trademark protection for any of our trademarks or trade names in any of our other geographic markets, and failure to secure those registrations could adversely affect our business.
Santa Barbara. History of Business Operations and Key Events We commenced operations in September 2008 as a Georgia limited liability company with in-licensed intellectual property relating protein kinase inhibitors to the control of bacterial and viral infectious diseases.
History of Business Operations and Key Events We commenced operations in September 2008 as a Georgia limited liability company with in-licensed intellectual property relating to protein kinase inhibitors to the control of bacterial and viral infectious diseases.
Budget Control Act of 2011, among other things, included aggregate reductions of Medicare payments to providers of 2% per fiscal year that remain in effect through 2031. 16 • The U.S.
Budget Control Act of 2011, among other things, included aggregate reductions of Medicare payments to providers of 2% per fiscal year that remain in effect through 2031. 17 • The U.S.
On October 5, 2012, we and Sphaera amended the Sphaera Agreement to reflect joint patent applications in the U.S. and India by us and Sphaera for a series of novel compounds.
On October 5, 2012, we and Sphaera amended the Sphaera Agreement to reflect joint patent applications (the “Joint Application”) in the U.S. and India by us and Sphaera for a series of novel compounds.
Failure to conduct required post-approval studies, or confirm a clinical benefit during post-marketing studies, would allow the FDA to withdraw the drug from the market on an expedited basis. All promotional materials for drug candidates approved under accelerated regulations are subject to prior review by the FDA.
Failure to conduct required post-approval studies, or confirm a clinical benefit during post-marketing studies, would allow the FDA to withdraw the drug from the market on an expedited basis. All promotional materials for drug candidates approved under accelerated regulations are subject to prior review by the FDA, unless otherwise informed by the FDA.
Patents issuing from the applications in this portfolio, if granted, will expire between 2033 and 2045, not taking into account any potential patent-term adjustments or extensions that may be available in the future. Two families of patents and applications covers compositions of matter for IKT-001 and related chemical compounds, as well as methods of using those compositions.
Patents issuing from the applications in this portfolio, if granted, will expire between 2033 and 2045, not taking into account any potential patent-term adjustments or extensions that may be available in the future. Three families of patents and applications claim compositions of matter for IKT-001 and related chemical compounds, as well as methods of using those compositions.
This family does not include any pending patent applications in the U.S. Outside the U.S., this family includes issued patents in Europe, Japan, and Australia, and a pending patent application in Canada.
This family does not include any pending patent applications in the U.S. Outside the U.S., this family includes issued patents in Europe, Japan, Canada, and Australia.
We may develop one or all of our products and commercialize them ourselves, or we may license or form partnerships with other companies for commercialization of our products in the future. 6 Competition The pharmaceutical industries, including in the PAH disease field, are characterized by rapidly advancing technologies, strong competition and an emphasis on intellectual property.
We may develop our products and commercialize them ourselves, if approved, or we may license or form partnerships with other companies for commercialization of our products in the future. 6 Competition The pharmaceutical industries, including in the PAH disease field, are characterized by rapidly advancing technologies, strong competition and an emphasis on intellectual property.
The information on our website is not incorporated by reference into this Annual Report on Form 10-K and should not be considered to be a part of this Annual Report on Form 10-K. Our website address is included in this Annual Report on Form 10-K as an inactive technical reference only.
The information on our website is not incorporated by reference into this Annual Report and should not be considered to be a part of this Annual Report. Our website address is included in this Annual Report as an inactive technical reference only.
Companies currently marketing products for PAH include large companies with significant financial resources, that include but are not limited to GlaxoSmithKline, Johnson and Johnson, United Therapeutics, Gilead Sciences, and Merck and Co.
Companies currently marketing products for PAH include large companies with significant financial resources, that include but are not limited to GlaxoSmithKline, Johnson and Johnson, United Therapeutics, Bayer Pharmaceuticals, Liquidia, Gilead Sciences, and Merck and Co.
Primary research to validate our strategy with pharmacy benefit managers and payors suggests a commercial path exists as there remains high unmet need in the PAH market. IKT-001, if approved, will compete for market share from other therapies in PAH and could be one of only a few therapies approved that is disease modifying.
Primary research to validate our strategy with PAH prescribers suggests a commercial path exists as there remains high unmet need in the PAH market. IKT-001, if approved, will compete for market share from other therapies in PAH and could be one of only a few therapies approved that is disease modifying.
On September 30, 2024, we and Pivot agreed to amend the agreement and for us to pay $500,000 upon signing as well as payment of the following milestones by us. A one-time payment of $4.4 million upon FDA Approval (as described in the Sphaera Agreement) and a single low digit royalty on net sales of an FDA approved drug.
On September 30, 2024, we and Pivot agreed to amend the agreement and for us to pay $500,000 upon signing as well as a one-time payment of $4.4 million upon FDA Approval (as described in the Sphaera Agreement), with a low single digit royalty due on net sales of an FDA approved drug.
At the meeting, the FDA confirmed that IKT-001 would be viewed as a New Molecular Entity (“NME”) and that the appropriate path for approval remained to be the 505(b)(2) statute. This opens up the possibility of IKT-001 being granted NME status and market exclusivity on approval.
At the meeting, the FDA confirmed that IKT-001 would be viewed as a New Molecular Entity (“NME”) and that the appropriate path for approval remained the 505(b)(2) pathway. This opened up the possibility of IKT-001 being granted NME status and market exclusivity on approval.
The EU CTR requires, among other things, that the prior authorization of an ethics committee and the submission and approval of a clinical trial authorization application be obtained in each applicable EU Member State before commencing a clinical trial in that EU Member State.
The EU CTR requires, among other things, that the prior authorization of an ethics committee and the approval of a clinical trial authorization application by the competent authority be obtained in each applicable EU Member State before commencing a clinical trial in that EU Member State.
Companies currently developing products in PAH are both large companies with significant financial resources as wells as smaller companies and include but are not limited to Merck and Co., Apollo Therapeutics, Novartis Pharmaceuticals, Pfizer, Gossamer Bio, and Insmed, Inc. Intellectual Property The proprietary nature of, and protection for, our product candidates, processes, and know-how are important to our business.
Companies currently developing products in PAH are both large companies with significant financial resources as well as smaller companies and include but are not limited to Merck and Co., GlaxoSmithKline, Regeneron, Liquidia, Novartis Pharmaceuticals, Pfizer, Gossamer Bio, and Insmed, Inc. Intellectual Property The proprietary nature of, and protection for, our product candidates, processes, and know-how are important to our business.
Based on this study it was determined that bioequivalence was established with a 300 mg dose of IKT-001 to a dose of 230 mg of imatinib mesylate while a 500 mg dose of IKT-001 was established as bioequivalent to a dose of 383 mg of imatinib mesylate.
Based on this study it was determined that bioequivalence was established with a 300 mg dose of IKT-001 bioequivalent to a dose of 230 mg of imatinib, while a 400 mg dose of IKT-001 was established as bioequivalent to a dose of 306 mg of imatinib and a 500 mg dose of IKT-001 was established as bioequivalent to a dose of 383 mg of imatinib.
An approval letter authorizes commercial marketing of the drug with specific prescribing information for specific indications. 10 Even if the FDA approves a product, it may limit the approved indications for use of the product, require that contraindications, warnings or precautions be included in the product labeling, require that post-approval studies, including Phase 4 clinical trials, be conducted to further assess a drug’s safety after approval, require testing and surveillance programs to monitor the product after commercialization, or impose other conditions, including distribution and use restrictions or other risk management mechanisms under a REMS, which can materially affect the potential market and profitability of the product.
Even if the FDA approves a product, it may limit the approved indications for use of the product, require that contraindications, warnings or precautions be included in the product labeling, require that post-approval studies, including Phase 4 clinical trials, be conducted to further assess a drug’s safety after approval, require testing and surveillance programs to monitor the product after commercialization, or impose other conditions, including distribution and use restrictions or other risk management mechanisms under a REMS, which can materially affect the potential market and profitability of the product.
Even with submission of this additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteria for approval. If and when those conditions have been met to the FDA’s satisfaction, the FDA will typically issue an approval letter.
Even with submission of this additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteria for approval. If and when those conditions have been met to the FDA’s satisfaction, the FDA will typically issue an approval letter. An approval letter authorizes commercial marketing of the drug with specific prescribing information for specific indications.
Regulation Outside the United States To the extent that any of our product candidates, once approved, are sold in a foreign country, we may be subject to similar foreign laws and regulations, which may include, for instance, applicable post-marketing requirements, including safety surveillance, anti-fraud and abuse laws and implementation of corporate compliance programs and reporting of payments or other transfers of value to healthcare professionals. 14 When conducting clinical trials in the EU, we must adhere to the provisions of the EU Clinical Trials Regulation (EU) No 536/2014 (“EU CTR”).
Regulation Outside the United States To the extent that any of our product candidates, once approved, are sold in a foreign country, we may be subject to similar foreign laws and regulations, which may include, for instance, applicable post-marketing requirements, including safety surveillance, anti-fraud and abuse laws and implementation of corporate compliance programs and reporting of payments or other transfers of value to healthcare professionals.
Even if a product qualifies for one or more of these programs, the FDA may later decide that the product no longer meets the conditions for qualification or decide that the time period for FDA review or approval will not be shortened.
Even if a product qualifies for one or more of these programs, the FDA may later decide that the product no longer meets the conditions for qualification or decide that the time period for FDA review or approval will not be shortened. We may explore some of these opportunities for our product candidates as appropriate.
The requirements governing drug pricing and reimbursement vary widely from country to country. For example, the EU provides options for its Member States to restrict the range of medicinal products for which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use.
For example, the EU provides options for its Member States to restrict the range of medicinal products for which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use.
Moreover, under the provisions of the Food and Drug Administration Safety and Innovation Act (“FDASIA”), passed in July 2012, a sponsor can request designation of a product candidate as a “breakthrough therapy.” A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
As a condition of approval, the FDA may require a sponsor of a drug receiving accelerated approval to perform post-marketing studies to verify and describe the predicted effect on IMM or other clinical endpoint, and the drug may be subject to accelerated withdrawal procedures. 11 Moreover, under the provisions of the Food and Drug Administration Safety and Innovation Act (“FDASIA”), passed in July 2012, a sponsor can request designation of a product candidate as a “breakthrough therapy.” A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
We may explore some of these opportunities for our product candidates as appropriate. 11 Accelerated Approval Pathway The FDA may grant accelerated approval to a drug for a serious or life-threatening condition that provides meaningful therapeutic advantage to patients over existing treatments based upon a determination that the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.
Accelerated Approval Pathway The FDA may grant accelerated approval to a drug for a serious or life-threatening condition that provides meaningful therapeutic advantage to patients over existing treatments based upon a determination that the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.
Individual states have also been increasingly active in passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
Although it is voluntary, the GENEROUS Model could also impact the drug pricing landscape for manufacturers. 18 Individual states have also been increasingly active in passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
Further, under the IRA, orphan drugs are exempted from the Medicare drug price negotiation program, but only if they have one orphan designation and for which the only approved indication is for that disease or condition. If a product receives multiple orphan designations or has multiple approved indications, it may not qualify for the orphan drug exemption.
Further, under the IRA, orphan drugs are exempted from the Medicare drug price negotiation program, but only if they have at least one orphan designation and the only approved indications can be for rare diseases. If a product receives multiple orphan designations or has multiple approved indications, it may not qualify for the orphan drug exemption.
Clinical Trials Clinical trials involve the administration of the investigational new drug to human subjects under the supervision of qualified investigators in accordance with cGCP requirements, which include the requirement that all research subjects provide their informed consent in writing for their participation in any clinical trial.
As a result, submission of an IND may not result in the FDA allowing clinical trials to initiate. 9 Clinical Trials Clinical trials involve the administration of the investigational new drug to human subjects under the supervision of qualified investigators in accordance with cGCP requirements, which include the requirement that all research subjects provide their informed consent in writing for their participation in any clinical trial.
However, members of our board of directors and executive team have commercial experience and we have conducted a commercial opportunity assessment for our lead product IKT-001 for PAH in the U.S. market.
Therefore, we have no sales, marketing or commercial product distribution capabilities and have no experience as a company in marketing drugs. However, members of our board of directors and executive team have commercial experience and we have conducted a commercial opportunity assessment for our lead product IKT-001 for PAH in the U.S. market.
For example, the EU CTR implements a coordinated procedure for authorization of clinical trials (through a centralized EU portal known as the Clinical Trials Information System) that is similar to the mutual recognition procedure for marketing authorization of medicinal products, and includes obligations on sponsors to publish clinical trial results.
For example, the EU CTR implements a coordinated procedure for authorization of clinical trials (through the submission of a single application through a centralized EU portal known as the Clinical Trials Information System), and includes obligations on sponsors to publish clinical trial results.
PAH is a rare disease of the pulmonary microvasculature found in 15 to 50 persons per million within the United States and Europe. The global PAH market size was valued at $7.66 billion in 2023 and is estimated to grow at a compound annual growth rate of 5.4% between 2024 to 2030.
PAH PAH is a rare disease of the pulmonary microvasculature found in 15 to 50 persons per million within the United States and Europe. The estimated global PAH market size was valued at approximately $8.3 billion in 2025 and is estimated to grow at a compound annual growth rate of 3.3% through 2034.
We have had discussions with the FDA regarding Orphan Drug Designation (“ODD”) for delivery of imatinib by IKT-001 for PAH and plan to apply for ODD once the required pre-clinical studies are complete.
We have had discussions with the FDA regarding Orphan Drug Designation (“ODD”) for delivery of imatinib by IKT-001 for PAH and are in the process of applying for ODD having successfully completed the required pre-clinical studies.
The European Commission introduced legislative proposals in April 2023 that, if implemented, will replace the current regulatory framework in the EU for all medicines (including those for rare diseases and for children).
The European Commission introduced legislative proposals in April 2023 that, if implemented, will replace the current regulatory framework in the EU for all medicines (including those for rare diseases and for children). In April 2024 the European Parliament adopted its position on the legislative proposals and, in June 2025, the Council of the European Union adopted its position.
Employees and Human Capital Resources As of the date of this Annual Report, we had fifteen full-time employees and one part-time employee. None of our employees are represented by a labor union or covered under a collective bargaining agreement. Our human capital resources objectives include, as applicable, identifying, recruiting, retaining, incentivizing and integrating our existing and additional employees.
None of our employees are represented by a labor union or covered under a collective bargaining agreement. Our human capital resources objectives include, as applicable, identifying, recruiting, retaining, incentivizing and integrating our existing and additional employees.
For more information regarding the risks related to our intellectual property, see “Risk Factors - Risks Related to Our Intellectual Property.” As of March 12, our patent portfolio included: (i) nine issued patents and three pending patent applications in the United States (ii) eleven issued foreign patents and four pending foreign patent applications, and (iii) a PCT application.
For more information regarding the risks related to our intellectual property, see “Risk Factors — Risks Related to Our Intellectual Property.” As of March 16, 2026, our IKT-001 patent portfolio included: (i) two issued patents in the United States (ii) six issued foreign patents, (iii) a pending application in the United States, (iv) a PCT application, and (v) a provisional patent application.
Orphan product designation does not convey any advantage in or shorten the duration of the regulatory review and approval process. 12 If a product with orphan status receives the first FDA approval for the disease or condition for which it has such designation or for a select indication or use within the rare disease or condition for which it was designated, the product generally will be receiving orphan product exclusivity.
If a product with orphan status receives the first FDA approval for the disease or condition for which it has such designation or for a select indication or use within the rare disease or condition for which it was designated, the product generally will be receiving orphan product exclusivity.
In addition to new legislation, FDA regulations, guidance, and policies are often revised or reinterpreted by the agency in ways that may significantly affect the manner in which pharmaceutical products are regulated and marketed. 13 U.S.
In addition to new legislation, FDA regulations, guidance, and policies are often revised or reinterpreted by the agency in ways that may significantly affect the manner in which pharmaceutical products are regulated and marketed. U.S. Healthcare Fraud and Abuse Laws and Compliance Requirements We are subject to various federal and state laws targeting fraud and abuse in the healthcare industry.
We currently have commercialization rights to all of our development programs and patent protection in the United States until 2033 for IKT-001 with upcoming patent application filings potentially extending patent protection for certain methods of treatment using IKT-001 until 2045.
We currently have global commercialization rights to IKT-001 and patent protection in the United States until 2039 for IKT-001 including potential patent term extension with upcoming patent application filings potentially extending patent protection for certain methods of treatment using IKT-001 until 2044.
These doses are adequate to cover the target systemically and were similar to the doses of imatinib mesylate used in the Phase 3 IMPRES trial in PAH.
We believe these doses are adequate to provide sustained target inhibition systemically and were similar to the doses of imatinib used in the Phase 3 IMPRES study in PAH.
In addition, we may be subject to patient privacy regulation by both the federal government and the states in which we conduct our business.
These laws may impact, among other things, our proposed sales and marketing programs. In addition, we may be subject to patient privacy regulation by both the federal government and the states in which we conduct our business.
We are planning to conduct clinical studies with the goal of eventually gaining FDA approval for the marketing of IKT-001 in the future. To achieve this commercial goal, we will require implementation of an appropriate commercial strategy for prescribers, pharmacy benefit managers and payors.
We have initiated a Phase 3 clinical study with the goal of eventually gaining FDA approval for the marketing of IKT-001 in the future. To achieve this goal, we will require implementation of an appropriate commercial strategy for prescribers, patients and payors.
As previously mentioned, imatinib inhibits Abelson Tyrosine Kinase and type III receptor tyrosine kinases and through these pathways inhibits Platelet-derived growth factor receptor which is involved in cell proliferation and angiogenesis as well as Stem cell factor receptor which targets mast cells and other hematopoietic progenitors. Through these targets imatinib may inhibit vascular smooth muscle cell proliferation and fibrosis.
As previously mentioned, imatinib inhibits type III receptor tyrosine kinases and through these pathways inhibits platelet-derived growth factor receptors which are involved in cell proliferation and migration as well as c-Kit which modulates mast cell and hematopoietic stem cell activity. Through these targets, imatinib may inhibit vascular cell proliferation, migration and fibrosis.
A company must request orphan product designation before submitting an NDA. If the request is granted, the FDA will disclose the identity of the therapeutic agent and its potential use.
A company must request orphan product designation before submitting an NDA. If the request is granted, the FDA will disclose the identity of the therapeutic agent and its potential use. Orphan product designation does not convey any advantage in or shorten the duration of the regulatory review and approval process.
Pursuant to the Merger Agreement, we paid consideration for CorHepta of $15.0 million, subject to a customary purchase price adjustment mechanism. We paid the purchase price pursuant to the issuance of an aggregate of 4,979,101 shares of Common Stock to the former stockholders of CorHepta.
Pursuant to the Merger Agreement, we paid consideration for CorHepta of $15.0 million, subject to a customary purchase price adjustment mechanism.
In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical trial can begin. As a result, submission of an IND may not result in the FDA allowing clinical trials to initiate.
In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical trial can begin.
In 2013, the outcome of a Phase 3 trial (IMPRES) evaluating imatinib mesylate as a treatment for PAH was reported, demonstrating that imatinib may improve key parameters associated with PAH.
In 2013, the outcome of a Phase 3 study, known as IMPRES, evaluating imatinib as a treatment for PAH was published, demonstrating that imatinib may improve key parameters associated with PAH. In this study, imatinib improved exercise capacity and hemodynamics in patients with advanced PAH.
The implementation of the IRA is currently subject to ongoing litigation challenging the constitutionality of the IRA’s Medicare drug price negotiation program. The effect of IRA on our business and the healthcare industry in general is not yet known.
The implementation of the IRA is currently subject to ongoing litigation challenging the constitutionality of the IRA’s Medicare drug price negotiation program.
We have applied to federally register our primary trademarks in our primary market, the United States. Three of the four trademark applications that we filed for (INHIBIKASE, IKT (and Design) and RAMP) have issued to registration, and the fourth application (a second application for INHIBIKASE) is allowed and is currently awaiting registration by the United States Patent and Trademark Office.
Four of the six trademark applications that we filed for (INHIBIKASE (two applications) and IKT (and Design)) have issued to registration, and the fourth and fifth applications (a third application for INHIBIKASE and a second application for IKT (and Design)) have been published and are currently awaiting registration by the United States Patent and Trademark Office.
Even if favorable coverage and reimbursement status is attained for one or more products for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future. In addition, in many foreign countries, the proposed pricing for a drug must be approved before it may be lawfully marketed.
Coverage policies and third-party reimbursement rates may change at any time. Even if favorable coverage and reimbursement status is attained for one or more products for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future.
There can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will allow favorable reimbursement and pricing arrangements for any of our products. Historically, products launched in the European Union do not follow price structures of the United States and generally prices tend to be significantly lower.
There can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will allow favorable reimbursement and pricing arrangements for any of our products.
Item 1. Bu siness. Company Overview We are a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary and other diseases including those that arise from aberrant signaling through the Abelson Tyrosine Kinase, and type III receptor tyrosine kinases including platelet derived growth factor receptors and proto-oncogene c-KIT ("c-KIT").
Item 1. Bu siness. Company Overview We are a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary diseases, namely, Pulmonary Arterial Hypertension (“PAH”), in which aberrant signaling through type III receptor tyrosine kinases, including platelet derived growth factor receptors and a stem cell factor receptor, known as “c-Kit”, has been implicated.
Under the terms of our agreement with Sphaera, described above under “ Material Agreements - Sphaera Pharma Pte. Ltd.” we have the exclusive right to commercialize certain compounds disclosed in these applications, including IKT-001, for cancer treatments. The other family includes a pending United States provisional application that covers methods of using IKT-001 to treat PAH.
Under the terms of our agreement with Sphaera, described above under “ Material Agreements - Sphaera Pharma Pte. Ltd.” we have the exclusive right (including as to Sphaera) to commercialize certain compounds disclosed in these applications for which an IND has been filed, including IKT-001.
The United States government, state legislatures and foreign governments have shown significant interest in implementing cost containment programs to limit the growth of government-paid health care costs, including price-controls, restrictions on reimbursement and requirements for substitution of generic products for branded prescription drugs.
As a result, the coverage determination process is often a time-consuming and costly process that will require us to provide scientific and clinical support for the use of our products to each payor separately, with no assurance that coverage and adequate reimbursement will be obtained. 19 The United States government, state legislatures and foreign governments have shown significant interest in implementing cost containment programs to limit the growth of government-paid health care costs, including price-controls, restrictions on reimbursement and requirements for substitution of generic products for branded prescription drugs.
Most of the treatments that constitute the standard of care (e.g. ERAs, PDE5s, prostacyclins) primarily act as vasodilators. In 2024, sotatercept was approved for the treatment of PAH on top of SOC. Sotatercept is recombinant fusion protein that acts as a trap for transforming growth factor-beta superfamily ligands, including activin A and bone morphogenetic protein 9.
Most of the current treatments that constitute the standard of care (e.g., ERAs, PDE5s, prostacyclins) primarily act as vasodilators. In 2024, sotatercept was approved for the treatment of PAH on top of standard-of-care.
Data and Marketing Exclusivity In the EU, new products authorized for marketing, or reference products, qualify for eight years of data exclusivity and an additional two years of market exclusivity upon marketing authorization.
The GDPR also imposes strict rules on the transfer of personal data outside of the EEA to countries that do not ensure an adequate level of protection. Data and Marketing Exclusivity In the EU, new products authorized for marketing, or reference products, qualify for eight years of data exclusivity and an additional two years of market exclusivity upon marketing authorization.
We face substantial competition from many different sources, including large and specialty pharmaceutical companies, academic research institutions, governmental agencies and public and private research institutions. We believe that the key competitive factors affecting the success of any of our product candidates will include efficacy, safety profile, method of administration, cost, level of promotional activity and intellectual property protection.
We believe that the key competitive factors affecting the success of any of our product candidates will include efficacy, safety profile, method of administration, cost, access and reimbursement, disease re-modeling capability, level of promotional activity and intellectual property protection.
An emphasis on cost containment measures in the United States has increased, and we expect will continue to increase, the pressure on pharmaceutical pricing. Coverage policies and third-party reimbursement rates may change at any time.
As noted above, the marketability of any products for which we receive regulatory approval for commercial sale may suffer if the government and third-party payors fail to provide adequate coverage and reimbursement. An emphasis on cost containment measures in the United States has increased, and we expect will continue to increase, the pressure on pharmaceutical pricing.
This pathway may provide an alternate pathway for disease modification in PAH. 2 The first reports of the use of imatinib in PAH were published in 2005 and 2006. A phase 2, RCT was subsequently conducted showing clinical benefit of imatinib in PAH.
The first reported uses of imatinib in PAH were published in 2005 and 2006. A Phase 2, randomized controlled trial, was subsequently conducted and published in 2010 showing clinical benefit of imatinib in PAH.
Additionally, if either party files an IND for a Company Compound that has been abandoned by the other party for an oncology indication in humans, the non-filing party is prohibited from developing such Company Compound. In 2023, Sphaera liquidated and transferred its interests to Pivot Holding LLC, a U.S. entity (“Pivot”) .
IKT-001 is a Company Compound, and we have informed Sphaera that an IND has been filed for IKT-001. In 2023, Sphaera liquidated and transferred its interests to Pivot Holding LLC, a U.S. entity (“Pivot”) .
In addition, as 340B drug prices are determined based on AMP and Medicaid rebate data, the revisions to the Medicaid rebate formula and AMP definition described above could cause the required 340B discount to increase. 18 As noted above, the marketability of any products for which we receive regulatory approval for commercial sale may suffer if the government and third-party payors fail to provide adequate coverage and reimbursement.
In addition, as 340B drug prices are determined based on AMP and Medicaid rebate data, the revisions to the Medicaid rebate formula and AMP definition described above could cause the required 340B discount to increase.
Outside the U.S., these families include issued patents in Japan, Australia, and Canada, pending patent applications in Japan, Canada, and Europe. These families are solely owned by us. In addition to patent protection, we also rely on trade secrets, know-how, other proprietary information and continuing technological innovation to develop and maintain our competitive position.
We thus expect that these future patents, if granted, will protect our planned commercialization of IKT-001 to treat PAH independently from and after the expiration of our jointly owned U.S. Patents. In addition to patent protection, we also rely on trade secrets, know-how, other proprietary information and continuing technological innovation to develop and maintain our competitive position.
We have completed non-human primate safety studies and a bioequivalence clinical trial in healthy volunteers to determine the doses of IKT-001 that are equivalent to imatinib mesylate and the results are being utilized to set the doses in a Phase 2b trial to determine if IKT-001 could be a disease-modifying treatment for PAH.
PAH is a progressive, life-threatening disease characterized by pulmonary vascular remodeling and elevated pulmonary vascular resistance that affects approximately 50,000 Americans. We have completed a non-human primate safety study and a bioequivalence clinical study in healthy volunteers to determine the doses of IKT-001 that are equivalent to imatinib.
As we considered revisiting the use of imatinib in PAH, we recognized that changes in standard-of-care for these patients may have alleviated much of the safety risk previously observed for imatinib in PAH patients. This analysis prompted us to file a pre-IND (“PIND”) meeting request to discuss the application of IKT-001 as a potential disease-modifying treatment for PAH.
As we considered developing IKT-001 in PAH, we also recognized that the learnings of over 20-years of oncology clinical experience with imatinib, together with changes in the standard of care for these patients, may have alleviated much of the safety risk previously observed for imatinib in PAH patients.
We rely on third-party contract manufacturers (“CMOs”), to manufacture and supply our preclinical and clinical materials to be used during the development of our product candidates.
We rely on third-party contract development and manufacturing organizations ( “ CDMOs ” ), to manufacture and supply our preclinical and clinical materials to be used during development of our product candidates. Formulation and finishing services are provided through contracts on an as-needed basis, including current Good Manufacturing Practice (“cGMP”) manufacturing of active pharmaceutical ingredients.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeWe have never generated any revenue from product sales, we may never generate any revenue from product sales, and we may fail to generate further revenue from grants or contracts or to be profitable; • Adverse developments affecting financial institutions, companies in the financial services industry or the financial services industry generally, including those we do business with, could adversely affect our operations and liquidity; • We have incurred significant net losses since our inception and anticipate that we will continue to incur net losses for the foreseeable future; • If we fail to obtain additional financing, we may be unable to complete the development of and, if approved, commercialization of our product candidates; • Due to the significant resources required for the development of our programs, and depending on our ability to access capital, we must prioritize development of certain product candidates; • Our business is highly dependent on the success of our initial product candidates targeting neurodegenerative, cardiopulmonary and oncological diseases; • Our focus on IKT-001 as a treatment for PAH may not prove successful; • We currently contract with various research institutions to perform the research and development activities needed to develop our products, and if we ever choose to or need to find alternative research institutions, we may not be able to do so at all or, if we are able to do so, it may be costly and may cause significant delays in the development and commercialization of our products; • Positive results from early preclinical or clinical studies of our product candidates are not necessarily predictive of the results of later preclinical studies and any current and future clinical trials of our product candidates; • We have limited experience with conducting clinical trials for novel drug substances and no history of commercializing pharmaceutical products, which may make it difficult to evaluate the prospects for our future viability; • Our clinical trials may reveal significant adverse events, toxicities or other side effects not seen in our preclinical studies and may result in a safety profile that could inhibit regulatory approval or market acceptance of any of our product candidates; • We have concentrated much of our research and development efforts on the treatment of neurodegenerative diseases, a field that has seen limited success in drug development; • We may encounter substantial delays in our current and planned clinical trials, or may not be able to conduct or complete our clinical trials on the timelines we expect, if at all; • Our current and planned clinical trials may fail to demonstrate substantial evidence of the safety and efficacy of our product candidates, which would prevent, delay or limit the scope of regulatory approval and commercialization; • Clinical development is a lengthy and expensive process with an uncertain outcome, and failure can occur at any stage of clinical development; • The manufacture of our product candidates is complex and difficulties may be encountered in production; 20 • If, in the future, we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell and market any product candidates we may develop, we may not be successful in commercializing those product candidates if and when they are approved; • Even if any product candidates we develop receive marketing approval, they may fail to achieve the degree of market acceptance by physicians, patients, healthcare payors, and others in the medical community necessary for commercial success; • Even if we are able to commercialize any product candidates, such products may become subject to unfavorable pricing regulations, third-party reimbursement practices, or healthcare reform initiatives, which would harm our business; • The regulatory approval processes of the FDA, EMA and comparable foreign regulatory authorities are lengthy, time consuming, and inherently unpredictable.
Biggest changeWe have never generated any revenue from product sales and, we may never gain approval for our product candidates or, even if our product candidates are approved, generate any revenue from product sales or be profitable; • Adverse developments affecting financial institutions, companies in the financial services industry or the financial services industry generally, including those we do business with, could adversely affect our operations and liquidity; • We have incurred significant net losses since our inception and anticipate that we will continue to incur net losses for the foreseeable future; • If we fail to obtain additional financing on a timely basis or on acceptable terms, we may be unable to complete the development of and, if approved, commercialization of our product candidates; • Our business is highly dependent on the success of our initial product candidate, IKT-001, targeting pulmonary arterial hypertension (“PAH”); • Our focus on IKT-001 as a treatment for PAH may not prove successful; • Positive results from early preclinical or clinical studies of our product candidates including, without limitation, the Phase 3 IMPRES study, are not necessarily predictive of the results of later preclinical studies and any current and future clinical trials of our product candidates; • We have never completed a registrational clinical trial as a company and may be unable to successfully do so with IKT-001, which may make it difficult to evaluate the prospects for our future viability; • Our clinical trials may reveal significant adverse events, toxicities or other side effects not seen in our preclinical or earlier clinical studies and may result in a safety profile that could inhibit regulatory approval or market acceptance of any of our product candidates; • We may encounter substantial delays in our current and planned clinical trials, or may not be able to conduct or complete our clinical trials on the timelines we expect, if at all; • Our current and planned clinical trials may fail to demonstrate substantial evidence of the safety and efficacy of our product candidates, or may require additional clinical studies to be undertaken, which would prevent, delay or limit the scope of regulatory approval and commercialization; • Clinical development is a lengthy and expensive process with an uncertain outcome, and failure can occur at any stage of clinical development; • We have limited manufacturing experience and the manufacture of our product candidates is complex, reliant on external expertise and capabilities, and difficulties or delays may be encountered in production; • If, in the future, we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell and market any product candidates we may develop, we may not be successful in commercializing those product candidates if and when they are approved; • Even if any product candidates we develop receive marketing approval, they may fail to achieve the degree of market acceptance by physicians, patients, healthcare payors, and others in the medical community necessary for commercial success; • Even if we are able to commercialize any product candidates, such products may become subject to unfavorable pricing regulations, third-party reimbursement practices, or healthcare reform initiatives, which would harm our business; • The regulatory approval processes of the United States Food and Drug Administration (“FDA”), European Medicines Agency (“EMA”) and comparable foreign regulatory authorities are lengthy, costly, time consuming, and inherently unpredictable.
If patients participating in our current and planned clinical trials suffer drug-related adverse actions during the course of such clinical trials, if we or the FDA believe that patients are being exposed to unacceptable health risks, or no meaningful benefits are observed from our product candidates, such clinical trials may have to be suspended or terminated.
If patients participating in our current and planned clinical trials suffer drug-related adverse actions during the course of such clinical trials, and we or the FDA believe that patients are being exposed to unacceptable health risks, or no meaningful benefits are observed from our product candidates, such clinical trials may have to be suspended or terminated.
If a product that has orphan drug designation subsequently receives the first FDA approval for a particular active ingredient for the disease for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other NDA or BLA applications to market the same drug or biologic for the same indication for seven years, except in limited circumstances such as a showing of clinical superiority to the product with orphan exclusivity or if the FDA finds that the holder of the orphan exclusivity has not shown that it can assure the availability of sufficient quantities of the orphan product to meet the needs of patients with the disease or condition for which the drug was designated.
If a product that has orphan drug designation subsequently receives the first FDA approval for a particular active ingredient for the indication for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other NDA or BLA applications to market the same drug or biologic for the same indication for seven years, except in limited circumstances such as a showing of clinical superiority to the product with orphan exclusivity or if the FDA finds that the holder of the orphan exclusivity has not shown that it can assure the availability of sufficient quantities of the orphan product to meet the needs of patients with the disease or condition for which the drug was designated.
In addition, each of our license agreements, and we expect our future agreements, will impose various development, diligence, commercialization, and other obligations on us. Certain of our license agreements also require us to meet development timelines, or to exercise commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses.
In addition, each of our license agreements, and we expect our future agreements will impose various development, diligence, commercialization, and/or other obligations on us. Certain of our license agreements also require us to meet development timelines, or to exercise commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses.
A person or entity does not need to have actual knowledge of these statutes or specific intent to violate them in order to have committed a violation; • HIPAA, which created federal criminal statutes that prohibit knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private) and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false statements in connection with the delivery of, or payment for, healthcare benefits, items or services relating to healthcare matters; 45 • HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (“HITECH”), and their respective implementing regulations, which impose requirements on certain covered healthcare providers, health plans, and healthcare clearinghouses as well as their respective business associates that perform services for them that involve the use, or disclosure of, individually identifiable health information, relating to the privacy, security and transmission of individually identifiable health information without appropriate authorization; • federal government price reporting laws, which require us to calculate and report complex pricing metrics in an accurate and timely manner to government programs; • the federal Physician Payments Sunshine Act, created under the ACA, and its implementing regulations, which require manufacturers of drugs, devices, biologicals and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program to report annually to the U.S.
A person or entity does not need to have actual knowledge of these statutes or specific intent to violate them in order to have committed a violation; • HIPAA, which created federal criminal statutes that prohibit knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private) and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false statements in connection with the delivery of, or payment for, healthcare benefits, items or services relating to healthcare matters; • HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (“HITECH”), and their respective implementing regulations, which impose requirements on certain covered healthcare providers, health plans, and healthcare clearinghouses as well as their respective business associates that perform services for them that involve the use, or disclosure of, individually identifiable health information, relating to the privacy, security and transmission of individually identifiable health information without appropriate authorization; • federal government price reporting laws, which require us to calculate and report complex pricing metrics in an accurate and timely manner to government programs; • the federal Physician Payments Sunshine Act, created under the ACA, and its implementing regulations, which require manufacturers of drugs, devices, biologicals and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program to report annually to the U.S.
Applications for our product candidates could fail to receive regulatory approval for many reasons, including, but not limited to the following: • the FDA, EMA or comparable foreign regulatory authorities may disagree with the design, implementation or results of our preclinical or clinical trials; 38 • the FDA, EMA or comparable foreign regulatory authorities may determine that our product candidates are not safe and effective, only moderately effective or have undesirable or unintended side effects, toxicities or other characteristics that preclude our obtaining marketing approval or prevent or limit commercial use; • the population studied in the clinical program may not be sufficiently broad or representative to assure efficacy and safety in the full population for which we seek approval; • we may be unable to demonstrate to the FDA or comparable foreign regulatory authorities that a product candidate’s risk-benefit ratio when compared to the standard of care is acceptable; • the data collected from preclinical or clinical trials of our product candidates may not be sufficient to support the submission of an NDA, or other submission or to obtain regulatory approval in the United States or elsewhere; • we may be unable to demonstrate to the FDA, EMA or comparable foreign regulatory authorities that a product candidate’s risk-benefit ratio for its proposed indication is acceptable; • the FDA, EMA or comparable foreign regulatory authorities may fail to approve the manufacturing processes, test procedures and specifications, or facilities of third-party manufacturers with which we contract for preclinical, clinical and commercial supplies; and • the approval policies or regulations of the FDA, EMA or comparable foreign regulatory authorities may significantly change in a manner rendering our preclinical or clinical data insufficient for approval.
Applications for our product candidates could fail to receive regulatory approval for many reasons, including, but not limited to the following: • the FDA, EMA or comparable foreign regulatory authorities may disagree with the design, implementation or results of our preclinical or clinical trials; • the FDA, EMA or comparable foreign regulatory authorities may determine that our product candidates are not safe and effective, only moderately effective or have undesirable or unintended side effects, toxicities or other characteristics that preclude our obtaining marketing approval or prevent or limit commercial use; • the population studied in the clinical program may not be sufficiently broad or representative to assure efficacy and safety in the full population for which we seek approval; • we may be unable to demonstrate to the FDA or comparable foreign regulatory authorities that a product candidate’s risk-benefit ratio when compared to the standard of care is acceptable; • the data collected from preclinical or clinical trials of our product candidates may not be sufficient to support the submission of an NDA, or other submission or to obtain regulatory approval in the United States or elsewhere; • we may be unable to demonstrate to the FDA, EMA or comparable foreign regulatory authorities that a product candidate’s risk-benefit ratio for its proposed indication is acceptable; • the FDA, EMA or comparable foreign regulatory authorities may fail to approve the manufacturing processes, test procedures and specifications, or facilities of third-party manufacturers with which we contract for preclinical, clinical and commercial supplies; and • the approval policies or regulations of the FDA, EMA or comparable foreign regulatory authorities may significantly change in a manner rendering our preclinical or clinical data insufficient for approval.
Such collaborations pose the following risks to us: • collaborators generally have significant discretion in determining the efforts and resources that they will apply to these collaborations; • collaborators may not properly obtain, maintain, enforce, or defend intellectual property or proprietary rights relating to our product candidates or research programs or may use our proprietary information in such a way as to expose us to potential litigation or other intellectual property related proceedings, including proceedings challenging the scope, ownership, validity and enforceability of our intellectual property; • we may need the cooperation of our collaborators to enforce or defend any intellectual property we contribute to or that arises out of our collaborations, which may not be provided to us; • disputes may arise between the collaborators and us that result in the delay or termination of the research, development, or commercialization of our product candidates or research programs or that result in costly litigation or arbitration that diverts management attention and resources; • collaborators may decide to not pursue development and commercialization of any product candidates we develop or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborator’s strategic focus or available funding or external factors such as an acquisition that diverts resources or creates competing priorities; • collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials, or require a new formulation of a product candidate for clinical testing; 48 • collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our product candidates or research programs if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; • collaborators with marketing and distribution rights to one or more product candidates may not commit sufficient resources to the marketing and distribution of such product candidates; • we may lose certain valuable rights under circumstances identified in our collaborations; collaborators may undergo a change of control and the new owners may decide to take the collaboration in a direction which is not in our best interest; • collaborators may become bankrupt, which may significantly delay our research or development programs, or may cause us to lose access to valuable technology, know-how or intellectual property of the collaborator relating to our products, product candidates or research programs; • key personnel at our collaborators may leave, which could negatively impact our ability to productively work with our collaborators; collaborations may require us to incur short- and long-term expenditures or issue securities that dilute our stockholders or disrupt our management and business; collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates; and • collaboration agreements may not lead to development or commercialization of product candidates in the most efficient manner or at all.
Such collaborations pose the following risks to us: • collaborators generally have significant discretion in determining the efforts and resources that they will apply to these collaborations; • collaborators may not properly obtain, maintain, enforce, or defend intellectual property or proprietary rights relating to our product candidates or research programs or may use our proprietary information in such a way as to expose us to potential litigation or other intellectual property related proceedings, including proceedings challenging the scope, ownership, validity and enforceability of our intellectual property; • we may need the cooperation of our collaborators to enforce or defend any intellectual property we contribute to or that arises out of our collaborations, which may not be provided to us; • disputes may arise between the collaborators and us that result in the delay or termination of the research, development, or commercialization of our product candidates or research programs or that result in costly litigation or arbitration that diverts management attention and resources; • collaborators may decide to not pursue development and commercialization of any product candidates we develop or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborator’s strategic focus or available funding or external factors such as an acquisition that diverts resources or creates competing priorities; • collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials, or require a new formulation of a product candidate for clinical testing; • collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our product candidates or research programs if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; • collaborators with marketing and distribution rights to one or more product candidates may not commit sufficient resources to the marketing and distribution of such product candidates; • we may lose certain valuable rights under circumstances identified in our collaborations; collaborators may undergo a change of control and the new owners may decide to take the collaboration in a direction which is not in our best interest; • collaborators may become bankrupt, which may significantly delay our research or development programs, or may cause us to lose access to valuable technology, know-how or intellectual property of the collaborator relating to our products, product candidates or research programs; • key personnel at our collaborators may leave, which could negatively impact our ability to productively work with our collaborators; collaborations may require us to incur short- and long-term expenditures or issue securities that dilute our stockholders or disrupt our management and business; collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates; and 49 • collaboration agreements may not lead to development or commercialization of product candidates in the most efficient manner or at all.
If we, our product candidates or the manufacturing facilities for our product candidates fail to comply with applicable regulatory requirements, a regulatory agency or enforcement authority may, among other things: • issue warning letters that would result in adverse publicity; • impose civil or criminal penalties; • suspend or withdraw regulatory approvals; • suspend any of our ongoing clinical trials; • refuse to approve pending applications or supplements to approved applications submitted by us; • impose restrictions on our operations, including closing our contract manufacturers’ facilities; • mandate modifications to promotional materials or require us to provide corrective information to healthcare practitioners; • refuse to allow us to enter into government contracts; • seize or detain products, refuse to permit the import or export of products; or • require a product recall.
If we, our product candidates or the manufacturing facilities for our product candidates fail to comply with applicable regulatory requirements, a regulatory agency or enforcement authority may, among other things: • issue warning letters that would result in adverse publicity; • impose civil or criminal penalties; • suspend or withdraw regulatory approvals; • suspend any of our ongoing clinical trials; • refuse to approve pending applications or supplements to approved applications submitted by us; 42 • impose restrictions on our operations, including closing our contract manufacturers’ facilities; • mandate modifications to promotional materials or require us to provide corrective information to healthcare practitioners; • refuse to allow us to enter into government contracts; • seize or detain products, refuse to permit the import or export of products; or • require a product recall.
Any acquisition or strategic partnership may entail numerous risks, including: • increased operating expenses and cash requirements; • the potential issuance of our equity securities which would result in dilution to our stockholders; • assimilation of operations, intellectual property, products and product candidates of an acquired company, including difficulties associated with integrating new personnel; • the diversion of our management’s attention from our existing product programs and initiatives in pursuing such an acquisition or strategic partnership; • retention of key employees, the loss of key personnel, and uncertainties in our ability to maintain key business relationships; • risks and uncertainties associated with the other party to such a transaction, including the prospects of that party and their existing products or product candidates and regulatory approvals; and • our inability to generate revenue from acquired intellectual property, technology and/or products sufficient to meet our objectives or even to offset the associated transaction and maintenance costs.
Any acquisition or strategic partnership may entail numerous risks, including: • increased operating expenses and cash requirements; • the potential issuance of our equity securities which would result in dilution to our stockholders; 68 • assimilation of operations, intellectual property, products and product candidates of an acquired company, including difficulties associated with integrating new personnel; • the diversion of our management’s attention from our existing product programs and initiatives in pursuing such an acquisition or strategic partnership; • retention of key employees, the loss of key personnel, and uncertainties in our ability to maintain key business relationships; • risks and uncertainties associated with the other party to such a transaction, including the prospects of that party and their existing products or product candidates and regulatory approvals; and • our inability to generate revenue from acquired intellectual property, technology and/or products sufficient to meet our objectives or even to offset the associated transaction and maintenance costs.
Department of Health and Human Services under the Open Payments Program, information related to payments or other transfers of value made to physicians and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members, as well as other state and foreign laws regulating marketing activities; • federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers; and • analogous state and foreign laws and regulations, such as state and foreign anti-kickback, false claims, consumer protection and unfair competition laws which may apply to pharmaceutical business practices, including, but not limited to, research, distribution, sales and marketing arrangements as well as submitting claims involving healthcare items or services reimbursed by any third-party payer, including commercial insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government that otherwise restricts payments that may be made to healthcare providers and other potential referral sources; state laws that require drug manufacturers to file reports with states regarding pricing and marketing information, such as the tracking and reporting of gifts, compensations and other remuneration and items of value provided to healthcare professionals and entities; and state and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and may not have the same effect, thus complicating compliance efforts.
Department of Health and Human Services under the Open Payments Program, information related to payments or other transfers of value made to physicians, and other types of licensed healthcare professionals, and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members, as well as other state and foreign laws regulating marketing activities; • federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers; and • analogous state and foreign laws and regulations, such as state and foreign anti-kickback, false claims, consumer protection and unfair competition laws which may apply to pharmaceutical business practices, including, but not limited to, research, distribution, sales and marketing arrangements as well as submitting claims involving healthcare items or services reimbursed by any third-party payer, including commercial insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government that otherwise restricts payments that may be made to healthcare providers and other potential referral sources; state laws that require drug manufacturers to file reports with states regarding pricing and marketing information, such as the tracking and reporting of gifts, compensations and other remuneration and items of value provided to healthcare professionals and entities; and state and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and may not have the same effect, thus complicating compliance efforts.
Even if we are able to establish agreements with third-party manufacturers, reliance on third party manufacturers entails additional risks, including: • the possible breach of the manufacturing agreement by the third-party; 49 • the possible termination or non-renewal of the agreement by the third-party at a time that is costly or inconvenient for us; • reliance on the third-party for regulatory compliance, quality assurance, safety, and pharmacovigilance and related reporting; and • the inability to produce required volume in a timely manner and to quality standards.
Even if we are able to establish agreements with third-party manufacturers, reliance on third party manufacturers entails additional risks, including: • the possible breach of the manufacturing agreement by the third-party; • the possible termination or non-renewal of the agreement by the third-party at a time that is costly or inconvenient for us; • reliance on the third-party for regulatory compliance, quality assurance, safety, and pharmacovigilance and related reporting; and • the inability to produce required volume in a timely manner and to quality standards.
To the extent that we continue to qualify as a “smaller reporting company” as such term is defined in Rule 12b-2 under the Exchange Act, after we cease to qualify as an emerging growth company, certain of the exemptions available to us as an “emerging growth company” may continue to be available to us as a “smaller reporting company,” including exemption from compliance with the auditor attestation requirements pursuant to SOX and reduced disclosure about our executive compensation arrangements.
To the extent that we continue to qualify as a “smaller reporting company” as such term is defined in Rule 12b-2 under the Exchange Act, after we cease to qualify as an emerging growth company, certain of the exemptions available to us as an “emerging growth company” continue to be available to us as a “smaller reporting company,” including exemption from compliance with the auditor attestation requirements pursuant to SOX and reduced disclosure about our executive compensation arrangements.
If CROs 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 preclinical or clinical data they obtain is compromised due to the failure to adhere to our preclinical or future clinical protocols, regulatory requirements or for other reasons, our preclinical and any future clinical trials may be extended, 47 delayed or terminated and we may not be able to obtain regulatory approval for or successfully commercialize our product candidates.
If CROs 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 preclinical or clinical data they obtain is compromised due to the failure to adhere to our preclinical or future clinical protocols, regulatory requirements or for other reasons, our preclinical and any future 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.
Even if the side effects do not preclude the drug from obtaining or maintaining marketing approval, undesirable side effects may inhibit market acceptance of the approved product due to its tolerability versus other therapies or could require a REMS plan, which could include a medication guide outlining the risks of such side effects for distribution to patients, a 28 communication plan for healthcare providers, and/or other elements to assure safe use.
Even if the side effects do not preclude the drug from obtaining or maintaining marketing approval, undesirable side effects may inhibit market acceptance of the approved product due to its tolerability versus other therapies or could require a REMS plan, which could include a medication guide outlining the risks of such side effects for distribution to patients, a communication plan for healthcare providers, and/or other elements to assure safe use.
In addition, if we undertake such a transaction, we may issue dilutive securities, assume or incur debt obligations, incur large one-time expenses and acquire intangible assets that could result in significant future amortization expense. 67 Our business activities may be subject to the Foreign Corrupt Practices Act (“FCPA”), and similar anti-bribery and anti-corruption laws.
In addition, if we undertake such a transaction, we may issue dilutive securities, assume or incur debt obligations, incur large one-time expenses and acquire intangible assets that could result in significant future amortization expense. Our business activities may be subject to the Foreign Corrupt Practices Act (“FCPA”), and similar anti-bribery and anti-corruption laws.
Despite our 56 efforts, any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive, and time-consuming, and the outcome is unpredictable.
Despite our efforts, any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive, and time-consuming, and the outcome is unpredictable.
In addition, the manufacturing process for any products that we may develop is subject to the FDA, EMA and foreign regulatory authority approval processes and continuous oversight, and we will need to contract with manufacturers who can meet all applicable FDA, EMA and foreign regulatory authority requirements, including complying with current good manufacturing processes, or on an ongoing basis.
In addition, the manufacturing process for any products that we may develop is subject to the FDA, EMA and foreign regulatory authority approval processes and continuous oversight, and we will need to contract with manufacturers who can meet all applicable FDA, EMA and foreign regulatory authority requirements, including complying with current good manufacturing practices, or on an ongoing basis.
The holder of an approved NDA, BLA, or MAA must submit new or supplemental applications and obtain approval for certain changes to the approved product, product labeling, or manufacturing process. We could also be asked to conduct post-marketing clinical trials to verify the safety and efficacy of our products in general or in specific patient subsets.
The holder of an approved NDA or MAA must submit new or supplemental applications and obtain approval for certain changes to the approved product, product labeling, or manufacturing process. We could also be asked to conduct post-marketing clinical trials to verify the safety and efficacy of our products in general or in specific patient subsets.
Events that may prevent successful or timely initiation or completion of clinical trials include: • inability to generate sufficient preclinical, toxicology, or other in vivo or in vitro data to support the initiation or continuation of clinical trials; • delays in confirming target engagement, patient selection or other relevant biomarkers to be utilized in preclinical and clinical product candidate development; • delays in reaching a consensus with regulatory agencies on study design; • delays in reaching agreement on acceptable terms with prospective CROs, and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and clinical trial sites; • delays in identifying, recruiting and training suitable clinical investigators; • delays in obtaining required IRB approval at each clinical trial site; • imposition of a temporary or permanent clinical hold by regulatory agencies for a number of reasons, including, but not limited to, after review of an IND or amendment, CTA or amendment, or equivalent application or amendment; as a result of a new safety finding that presents unreasonable risk to clinical trial participants; a negative finding from an inspection of our clinical trial operations or study sites; developments in trials conducted by competitors that raise FDA or EMA concerns about risk to patients broadly; or if the FDA or EMA finds that the investigational protocol or plan is clearly deficient to meet its stated objectives; • delays or difficulties resulting from future epidemics or pandemics; • delays in identifying, recruiting and enrolling suitable patients to participate in our clinical trials, and delays caused by patients withdrawing from clinical trials or failing to return for post-treatment follow-up; • difficulty collaborating with patient groups and investigators; • failure by our CROs, other third parties, or us to adhere to clinical trial requirements; • failure to perform in accordance with the FDA’s or any other regulatory authority’s current good clinical practices, or cGCPs, requirements, or applicable EMA or other regulatory guidelines in other countries; • occurrence of adverse events associated with a product candidate that are viewed to outweigh its potential benefits; • changes in regulatory requirements and guidance that require amending or submitting new clinical protocols; • changes in the standard of care on which a clinical development plan was based, which may require new or additional trials; • the cost of clinical trials of our product candidates being greater than we anticipate; 30 • clinical trials of our product candidates producing negative or inconclusive results, which may result in our deciding, or regulators requiring us, to conduct additional clinical trials or abandon product development programs; and • delays in manufacturing, testing, releasing, validating, or importing/exporting sufficient stable quantities of our product candidates for use in clinical trials or the inability to do any of the foregoing.
Events that may prevent successful or timely initiation or completion of clinical trials include: • inability to generate sufficient preclinical, toxicology, or other in vivo or in vitro data to support the initiation or continuation of clinical trials; • delays in confirming target engagement, patient selection or other relevant biomarkers to be utilized in preclinical and clinical product candidate development; • delays in reaching a consensus with regulatory agencies on study design; • delays in reaching agreement on acceptable terms with prospective CROs, and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and clinical trial sites; • delays in identifying, recruiting and training suitable clinical investigators; • delays in obtaining required IRB approval at each clinical trial site; • imposition of a temporary or permanent clinical hold by regulatory agencies for a number of reasons, including, but not limited to, after review of an IND or amendment, CTA or amendment, or equivalent application or amendment; as a result of a new safety finding that presents unreasonable risk to clinical trial participants; a negative finding from an inspection of our clinical trial operations or study sites; developments in trials conducted by competitors that raise FDA or other regulatory authority concerns about risk to patients broadly; or if the FDA or another regulatory authority finds that the investigational protocol or plan is clearly deficient to meet its stated objectives; • delays or difficulties resulting from future epidemics or pandemics; • delays in identifying, recruiting and enrolling suitable patients to participate in our clinical trials, and delays caused by patients withdrawing from clinical trials or failing to return for post-treatment follow-up; • difficulty collaborating with patient groups and investigators; • failure by our CROs, other third parties, or us to adhere to clinical trial requirements; • failure to perform in accordance with the FDA’s or any other regulatory authority’s current good clinical practices, or cGCPs, requirements, or applicable EMA or other regulatory guidelines in other countries; • occurrence of adverse events associated with a product candidate that are viewed to outweigh its potential benefits; • developments with current or future treatment options or changes in the standard of care for patients with PAH; • changes in regulatory requirements and guidance that require amending or submitting new clinical protocols; • changes in the standard of care on which a clinical development plan was based, which may require new or additional trials; • the cost of clinical trials of our product candidates being greater than we anticipate; • clinical trials of our product candidates producing negative or inconclusive results, which may result in our deciding, or regulators requiring us, to conduct additional clinical trials or abandon product development programs; and • delays in manufacturing, testing, releasing, validating, or importing/exporting sufficient stable quantities of our product candidates for use in clinical trials or the inability to do any of the foregoing.
Given the highly uncertain nature of drug development, we may never initiate or complete clinical trials for any of our product candidates, obtain marketing approval for any product candidates, manufacture a commercial scale product or arrange for a third-party to do so on our behalf, or conduct sales and marketing activities necessary for successful product commercialization.
Given the highly uncertain nature of drug development, we may never initiate or complete additional clinical trials for any of our product candidates, obtain marketing approval for any product candidates, manufacture a commercial scale product or arrange for a third-party to do so on our behalf, or conduct sales and marketing activities necessary for successful product commercialization.
As a result, even if one of our product candidates receives orphan exclusivity, the FDA can still approve other drugs that have a different active ingredient for use in treating the same indication or 42 disease. Furthermore, the FDA can waive orphan exclusivity if we are unable to manufacture sufficient supply of our product.
As a result, even if one of our product candidates receives orphan exclusivity, the FDA can still approve other drugs that have a different active ingredient for use in treating the same indication or disease. Furthermore, the FDA can waive orphan exclusivity if we are unable to manufacture sufficient supply of our product.
Our disclosure controls and procedures are designed to reasonably assure that information required to be disclosed by us in reports we file or submit under the Exchange Act are accumulated and communicated to management, and recorded, processed, summarized and reported within the time periods specified in 66 the rules and forms of the SEC.
Our disclosure controls and procedures are designed to reasonably assure that information required to be disclosed by us in reports we file or submit under the Exchange Act are accumulated and communicated to management, and recorded, processed, summarized and reported within the time periods specified in the rules and forms of the SEC.
Risks Related to Our Business, Financial Condition and Capital Requirements We are a clinical-stage drug development company with limited resources, a limited operating history and have no products approved for commercial sale, which may make it difficult to evaluate our current business and predict our future success and viability.
Risks Related to Our Business, Financial Condition and Capital Requirements We are a clinical-stage drug development company with limited resources, no revenue, a limited operating history and have no products approved for commercial sale, which may make it difficult to evaluate our current business and predict our future success and viability.
Supreme Court’s July 2024 decision to overturn prior established case law giving deference to regulatory agencies’ interpretations of ambiguous statutory language has introduced uncertainty regarding the extent to which FDA’s regulations, policies and decisions may become subject to increasing legal challenges, delays, and/or changes.
Supreme Court’s July 2024 decision to overturn prior established case law giving deference to regulatory agencies’ interpretations of ambiguous statutory language has introduced uncertainty regarding the extent to which FDA’s regulations, 38 policies and decisions may become subject to increasing legal challenges, delays, and/or changes.
We may also rely on collaborators or partners to conduct the required activities to support an application for regulatory approval, and to seek approval, for one or more of our product candidates. We cannot be sure that collaborators or partners will conduct these activities or do so within the time frame we desire.
We may also rely on collaborators or partners to conduct the required activities to support an application for regulatory approval, and to seek approval, for one or more of our product candidates. We cannot be sure that collaborators or partners will conduct these activities or do so within the cost and time frame we desire.
Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees. Any of the foregoing could have a material adverse effect on our business, prospects, financial condition and results of operations.
Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to 56 management and other employees. Any of the foregoing could have a material adverse effect on our business, prospects, financial condition and results of operations.
Among other things, our charter documents: • establish that our board of directors is divided into three classes, Class I, Class II and Class III, with each class serving staggered three-year terms; • provide that vacancies on our board of directors may be filled only by a majority of directors then in office, even though less than a quorum; 65 • provide that our directors may only be removed for cause; • eliminate cumulative voting; • authorize our board of directors to issues shares of preferred stock and determine the price and other terms of those shares, including preferences and voting rights, without stockholder approval; • provide our board of directors with the exclusive right to elect a director to fill a vacancy or newly created directorship; • permit stockholders to only take actions at a duly called annual or special meeting and not by written consent; • prohibit stockholders from calling a special meeting of stockholders; • require that stockholders give advance notice to nominate directors or submit proposals for consideration at stockholder meetings; • authorize our board of directors, by a majority vote, to amend the bylaws; and • require the affirmative vote of at least 66 2/3% or more of the outstanding shares of common stock to amend many of the provisions described above.
Among other things, our charter documents: • establish that our board of directors is divided into three classes, Class I, Class II and Class III, with each class serving staggered three-year terms; 66 • provide that vacancies on our board of directors may be filled only by a majority of directors then in office, even though less than a quorum; • provide that our directors may only be removed for cause; • eliminate cumulative voting; • authorize our board of directors to issue shares of preferred stock and determine the price and other terms of those shares, including preferences and voting rights, without stockholder approval; • provide our board of directors with the exclusive right to elect a director to fill a vacancy or newly created directorship; • permit stockholders to only take actions at a duly called annual or special meeting and not by written consent; • prohibit stockholders from calling a special meeting of stockholders; • require that stockholders give advance notice to nominate directors or submit proposals for consideration at stockholder meetings; • authorize our board of directors, by a majority vote, to amend the bylaws; and • require the affirmative vote of at least 66 2/3% or more of the outstanding shares of common stock to amend many of the provisions described above.
Changes in either the patent laws or their interpretation in the United States and other countries may diminish our ability to protect our inventions, obtain, maintain, and enforce our intellectual property rights and, more generally, could affect the value of our intellectual property or narrow the scope of our owned and licensed patents.
Changes in either the patent laws or their interpretation in the United States and other countries may diminish our ability to protect our inventions, obtain, maintain, and enforce our intellectual property rights and, more generally, could affect the value of our 52 intellectual property or narrow the scope of our owned and licensed patents.
Likewise, we rely on our third-party research institution 61 collaborators for research and development of our product candidates and other third parties for the manufacture of our product candidates and to conduct clinical trials, and similar events relating to their computer systems could also have a material adverse effect on our business.
Likewise, we rely on our third-party research institution collaborators for research and development of our product candidates and other third parties for the manufacture of our product candidates and to conduct clinical trials, and similar events relating to their computer systems could also have a material adverse effect on our business.
If original marketing approval was obtained via the accelerated approval pathway, we could be required to conduct a successful post-marketing 41 clinical trial to confirm clinical benefit for our products. An unsuccessful post-marketing study or failure to complete such a study could result in the withdrawal of marketing approval.
If original marketing approval was obtained via the accelerated approval pathway, we could be required to conduct a successful post-marketing clinical trial to confirm clinical benefit for our products. An unsuccessful post-marketing study or failure to complete such a study could result in the withdrawal of marketing approval.
Similarly, even if we are able to complete our planned preclinical studies or any future clinical trials of our product candidates according to our current development timeline, the positive results from such clinical or preclinical studies and current and future clinical trials of our product candidates may not be replicated in subsequent preclinical studies or clinical trial results.
Similarly, even if we are able to complete our current clinical studies or any future clinical trials of our product candidates according to our current development timeline, the positive results from such clinical or preclinical studies and current and future clinical trials of our product candidates, if any, may not be replicated in subsequent preclinical studies or clinical trial results.
Even if our planned clinical trials were to be successfully completed, we cannot guarantee that the FDA or foreign regulatory authorities will interpret the results as we do, and more trials could be required before we submit our product candidates for approval.
Even if our planned clinical trials were to be successfully completed, we cannot guarantee that the FDA or foreign regulatory authorities will interpret the results as we do, and more preclinical or clinical trials could be required before we submit our product candidates for approval.
Compliance with applicable environmental laws and regulations may be expensive, and current or future environmental laws and regulations may impair our research, product development and manufacturing efforts. In addition, we cannot entirely eliminate the risk 46 of accidental injury or contamination from these materials or wastes.
Compliance with applicable environmental laws and regulations may be expensive, and current or future environmental laws and regulations may impair our research, product development and manufacturing efforts. In addition, we cannot entirely eliminate the risk of accidental injury or contamination from these materials or wastes.
If we are unable to obtain a necessary license to a third-party patent on commercially reasonable terms, we may be unable to commercialize our product candidates or other technologies, or such commercialization efforts may be significantly delayed, which could in turn significantly harm our business.
If we are unable to obtain a necessary license to a third-party patent on commercially reasonable terms, we may be unable to commercialize our product 58 candidates or other technologies, or such commercialization efforts may be significantly delayed, which could in turn significantly harm our business.
The occurrence of any of the events or developments described below could harm our business, financial condition, 19 results of operations and growth prospects. In such an event, the market price of our securities could decline, and you may lose all or part of your investment.
The occurrence of any of the events or developments described below could harm our business, financial condition, results of operations and growth prospects. In such an event, the market price of our securities could decline, and you may lose all or part of your investment.
We expect that healthcare reform measures that have and 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 our product and product candidates, if approved, and could 44 seriously harm our future revenues.
We expect that healthcare reform measures that have and 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 our product and product candidates, if approved, and could seriously harm our future revenues.
Our employees, independent contractors, consultants, commercial partners and vendors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements. We are exposed to the risk of fraud, misconduct or other illegal activity by our employees, independent contractors, consultants, commercial partners and vendors.
Our employees, independent contractors, consultants, commercial partners and vendors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements. We are exposed to the risk of fraud, negligence, misconduct or other illegal activity by our employees, independent contractors, consultants, commercial partners and vendors.
Each of our product candidates will require significant clinical development, management of preclinical, clinical, and manufacturing activities, regulatory approval, adequate manufacturing supply, a commercial organization, and significant marketing efforts before we generate any revenue from product sales, if at all.
Each of our product candidates will 27 require significant clinical development, management of preclinical, clinical, and manufacturing activities, regulatory approval, adequate manufacturing supply, a commercial organization, and significant marketing efforts before we generate any revenue from product sales, if at all.
Our clinical trials may reveal significant adverse events, toxicities or other side effects not seen in our preclinical studies and may result in a safety profile that could inhibit regulatory approval or market acceptance of any of our product candidates.
Our clinical trials may reveal significant adverse events, toxicities or other side effects not seen in our preclinical or earlier clinical studies and may result in a safety profile that could inhibit regulatory approval or market acceptance of any of our product candidates.
As a result, the commercial prospects for our product candidates would be harmed, our costs could increase substantially and our ability to generate revenue could be delayed. Switching or adding additional CROs involves additional cost and requires management time and focus.
As a result, the commercial prospects for our product candidates would be harmed, our costs could increase substantially and our ability to generate revenue could be delayed. Switching or adding additional CROs involves delays, additional cost and requires management time and focus.
A patent term extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent may be extended and only those claims covering the approved drug, a method for using it, or a 55 method for manufacturing it may be extended.
A patent term extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent may be extended and only those claims covering the approved drug, a method for using it, or a method for manufacturing it may be extended.
Moreover, even if these trials begin, issues may arise that could suspend or terminate such clinical trials. A failure of one or more clinical trials can occur at any stage of testing, and our future clinical trials may not be successful.
Moreover, even if these trials begin, issues may arise that could suspend or terminate such clinical trials. A failure of one or more clinical trials can occur at any stage of testing, and our current or future clinical trials may not be successful.
These products may compete with our products, and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing. Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.
These products may compete with our products, and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing. 54 Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.
Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or 58 proceedings adequately.
Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or proceedings adequately.
Regulatory authorities have substantial discretion in the approval process and may refuse to accept an application, may disagree with our regulatory strategy or proposed pathway for approval or may decide that our data are insufficient for approval and require additional preclinical, clinical or other studies; • We expect to depend in whole or in part on collaborations with third parties for the research, development and commercialization of any product candidates we may develop; • We contract with third parties for the manufacture of materials for our research programs, preclinical studies and current clinical trials and expect to continue to do so for any future clinical trials and for commercialization of any product candidates that we may develop; • We depend on a small number of third-party suppliers for key raw materials used in the manufacturing processes for our product candidates, and the loss of these third-party suppliers or their inability to supply us with adequate raw materials could harm our business; and • If we are unable to obtain and maintain patent protection for any product candidates we develop, our competitors could develop and commercialize products or technology similar or identical to ours, and our ability to successfully commercialize any product candidates we may develop, and our technology may be adversely affected.
Regulatory authorities have substantial discretion in the approval process and may refuse to accept an application, may disagree with our regulatory strategy or proposed pathway for approval or may decide that our data are insufficient for approval and require additional preclinical, clinical or other studies; • We may depend on collaborations with third parties for the research, development and commercialization of any product candidates we may develop; • We contract with third parties for the manufacture of materials for our research programs, preclinical studies and current clinical trials and expect to continue to do so for any future clinical trials and for commercialization of any product candidates that we may develop; 22 • We depend on a small number of third-party suppliers for key raw materials used in the manufacturing processes for our product candidates, and the loss of these third-party suppliers or their inability to supply us with adequate raw materials could harm our business; and • If we are unable to obtain and maintain patent protection for any product candidates we develop, our competitors could develop and commercialize products or technology similar or identical to ours, and our ability to successfully commercialize any product candidates we may develop, and our technology may be adversely affected.
The PRIME scheme is intended to encourage development of products in areas of unmet medical need and provides accelerated assessment of products representing substantial innovation, where the marketing authorization application will be made through the centralized procedure in the EU.
The PRIME scheme is intended to encourage development of products in areas of unmet medical need and accelerated assessment of products representing substantial innovation, where the marketing authorization application will be made through the centralized procedure in the EU.
Under certain environmental laws, we could be held responsible for costs relating to any contamination at our current or past facilities and at third-party facilities. We also could incur significant costs associated with civil or criminal fines and penalties.
Under certain environmental laws, we could be held responsible for costs relating to any contamination at our past facilities and at third-party facilities. We also could incur significant costs associated with civil or criminal fines and penalties.
There can be no assurance that any such patent applications will issue as granted patents, and even if they do issue, such 51 patent claims may be insufficient to prevent third parties, such as our competitors, from utilizing our technology.
There can be no assurance that any such patent applications will issue as granted patents, and even if they do issue, such patent claims may be insufficient to prevent third parties, such as our competitors, from utilizing our technology.
Our commercial opportunities could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient, or are less expensive than any products that we may develop.
Our commercial opportunities could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient, or are less expensive than any product that we may develop.
Accordingly, a third-party may attempt to use the USPTO procedures to invalidate our patent claims that would not have been invalidated if first challenged by the third-party as a defendant in a district court action.
Accordingly, a third-party may attempt to use the USPTO procedures to invalidate our patent claims that would not have been invalidated if first challenged by the third-party as a defendant in a district court 55 action.
For example, even if the FDA or European Commission grants marketing approval of a product candidate, comparable regulatory authorities in foreign jurisdictions must also approve the manufacturing, marketing and promotion of the product candidate for those countries.
For example, even if the FDA or European Commission grants marketing approval of a product candidate, comparable regulatory authorities in foreign jurisdictions must also 41 approve the manufacturing, marketing and promotion of the product candidate for those countries.
Any of these delays or additional requirements could cause our product candidates to not be approved, or if approved, significantly impact the timing and commercialization of our product candidates and significantly increase our overall costs of drug development.
Any of these delays or additional requirements could cause our product candidates to be delayed, not be approved, or if approved, significantly impact the timing and commercialization of our product candidates and significantly increase our overall costs of drug development.
Assuming that other 54 requirements for patentability are met, prior to March 2013, in the United States, the first to invent the claimed invention was entitled to the patent, while outside the United States, the first to file a patent application was entitled to the patent.
Assuming that other requirements for patentability are met, prior to March 2013, in the United States, the first to invent the claimed invention was entitled to the patent, while outside the United States, the first to file a patent application was entitled to the patent.
Commencing December 31, 2025, we will no longer qualify as an “emerging growth company” as defined in the JOBS Act, and the reduced disclosure requirements applicable to emerging growth companies will no longer apply to us.
Commencing December 31, 2025, we no longer qualify as an “emerging growth company” as defined in the JOBS Act, and the reduced disclosure requirements applicable to emerging growth companies no longer apply to us.
Further, bad actors around the world use increasingly sophisticated methods, including the use of artificial intelligence, to engage in illegal activities involving the theft and misuse of personal information, confidential information and intellectual property.
Bad actors around the world use increasingly sophisticated methods, including the use of artificial intelligence, to engage in illegal activities involving the theft and misuse of personal information, confidential information and intellectual property.
Accordingly, we and others with whom we work must continue to expend time, money, and effort in all areas of regulatory compliance, including manufacturing, production and quality control.
Accordingly, we and others with whom we work must continue to expend significant time, money, and effort in all areas of regulatory compliance, including manufacturing, production and quality control.
Such proceedings also may 52 result in substantial cost and require significant time from our scientists and management, even if the eventual outcome is favorable to us.
Such proceedings also may result in substantial cost and require significant time from our scientists and management, even if the eventual outcome is favorable to us.
Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud. We are subject to the periodic reporting requirements of the Exchange Act.
Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud. 67 We are subject to the periodic reporting requirements of the Exchange Act.
Such laws include, without limitation, federal and state anti-kickback, fraud and abuse, false claims, data privacy and security and physician and other healthcare provider payment transparency laws and regulations.
Such laws include, without limitation, federal and state anti-kickback, fraud and abuse, false claims, data privacy and security, and physician 45 and other healthcare provider payment transparency laws and regulations.
We will request milestone-based meetings with the Review Team to ensure the Company and the Review Team remain aligned as we complete the necessary preclinical, clinical, manufacturing and quality control requirements for potential 39 approval, but there is no guarantee that Review Team interactions will ultimately lead to the approval of IKT-001 or any other product candidates we may develop.
We expect to request milestone-based meetings with the Review Team to ensure the Company and the Review Team remain aligned as we complete the necessary preclinical, clinical, manufacturing and quality control requirements for potential approval, but there is no guarantee that Review Team interactions will ultimately lead to the approval of IKT-001 or any other product candidates we may develop.
We will become subject to certain disclosure requirements that are applicable to other public companies that were not applicable to us as an emerging growth company, for example, compliance with any requirement that may be adopted by the Public Company Accounting Oversight Board regarding mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the audit and the consolidated financial statements and compliance with the requirements of holding a nonbinding advisory vote on executive compensation and stockholder approval of any golden parachute payments not previously approved.
We are also subject to certain disclosure requirements that are applicable to other public companies that were not applicable to us as an emerging growth company, for example, compliance with any requirement that may be adopted by the Public Company Accounting Oversight Board regarding mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the audit and the consolidated financial statements and compliance with the requirements of holding a nonbinding advisory vote on executive compensation and stockholder approval of any golden parachute payments not previously approved.
Our exclusive rights to certain of these patents and patent applications are dependent, in part, on operating agreements between the joint owners of such patents and patent applications.
Our exclusive rights to certain of these patents and patent applications are dependent, in part, on agreements between the joint owners of such patents and patent applications.
Our limited operating history as a company makes any assessment of our future success and viability subject to significant uncertainty. We will encounter risks and difficulties frequently experienced by early-stage pharmaceutical companies in rapidly evolving fields, and we have not yet demonstrated an ability to successfully overcome such risks and difficulties.
Our limited operating history as a company makes any assessment of our future success and viability subject to significant uncertainty. We will encounter risks and difficulties frequently experienced by clinical stage pharmaceutical companies in rapidly evolving fields, and we have not yet demonstrated an ability to successfully overcome such risks and difficulties.
The development and commercialization of new drug products is highly competitive. Moreover, the neurodegenerative field is characterized by strong and increasing competition, and a strong emphasis on intellectual property. Our competitors may be able to develop other compounds, drugs, cellular or gene therapies that are able to achieve similar or better results.
The development and commercialization of new drug products is highly competitive. Moreover, the cardiopulmonary field is characterized by strong and increasing competition, and a strong emphasis on intellectual property. Our competitors may be able to develop other compounds, drugs, cellular or gene therapies that are able to achieve similar or better results.
Many benefits accrue to sponsors of product candidates with PRIME 43 designation, including but not limited to, early and proactive regulatory dialogue with the EMA, frequent discussions on clinical trial designs and other development program elements, and accelerated marketing authorization application assessment once a dossier has been submitted.
Many benefits accrue to sponsors of product candidates with PRIME designation, including but not limited to, early and proactive regulatory dialogue with the EMA, frequent discussions on clinical trial designs and other development program elements, and the possibility of accelerated marketing authorization application assessment once a dossier has been submitted.
The results of our planned clinical trials in one set of patients or disease indications may not be predictive of those obtained in another.
The results of our planned clinical trials in one set of 31 patients or disease indications may not be predictive of those obtained in another.
The Review Team acknowledged that the 505(b)(2) pathway appears to be the appropriate pathway for approval of IKT-001 and indicated that, pending formal review of our clinical data, clinical studies completed to date indicate that 600 mg and 800 mg IKT-001 provides similar exposures to 400 mg and 600 mg imatinib mesylate, respectively, subject to review of the NDA upon filing.
DHMI acknowledged that the 505(b)(2) pathway appears to be the appropriate pathway for approval of IKT-001 and indicated that, pending formal review of our clinical data, clinical studies completed to date indicate that 600 mg and 800 mg IKT-001 provides similar exposures to 400 mg and 600 mg imatinib mesylate, respectively, subject to review of the NDA upon filing.
Future growth will impose significant added responsibilities on members of management, including: • identifying, recruiting, integrating, retaining, and motivating additional employees and consultants; • identifying and leasing suitable corporate, development and/or research facilities; • managing our internal development efforts effectively, including the clinical and FDA review process for our current and future product candidates, while complying with our contractual obligations to contractors and other third parties; expanding our operational, financial and management controls, reporting systems, and procedures; and • managing increasing operational and managerial complexity.
Future growth will impose significant added responsibilities on members of management, including: • identifying, recruiting, integrating, retaining, and motivating additional employees and consultants; • potentially identifying and leasing suitable corporate, development and/or research facilities; • managing our development efforts effectively, including the clinical and FDA review process for our current and future product candidates, while complying with our contractual and other legal obligations to contractors and other third parties; expanding our operational, financial and management controls, reporting systems, and procedures; and • managing increasing operational and managerial complexity.
Even if we receive regulatory approval to market any of our product candidates, whether for the treatment of neurodegenerative diseases or other diseases, we cannot assure you that any such product candidate will be successfully commercialized, widely accepted in the marketplace or more effective than other commercially available alternatives.
Even if we receive regulatory approval to market any of our product candidates, whether for the treatment of cardiopulmonary diseases or other diseases, we cannot assure you that any such product candidate will be successfully commercialized, widely accepted in the marketplace or be more effective than other commercially available alternatives.
In sum, other than the three U.S. federal registrations noted above and the registrations in the ex-US territories listed above, we have not secured trademark protection for any of our trademarks or trade names in any of our other geographic markets, and failure to secure those registrations could adversely affect our business.
In sum, other than the two U.S. federal registrations noted above and the registrations in the ex-US territories listed above, we have not secured trademark protection for any of our trademarks or trade names in any of our other geographic markets, and failure to secure those registrations could adversely affect our business.
We may experience difficulties in patient enrollment in our planned clinical trials for a variety of reasons, including: • the size and nature of the patient population; • the patient eligibility criteria defined in the protocol, and/or certain criteria related to stage of disease progression, which may limit the patient populations eligible for our clinical trials; • the size of the study population required for analysis of a trial’s primary endpoints; • the proximity of patients to a trial site; • the occurrence of future epidemics or pandemics; • the design of a trial; • our ability to recruit clinical trial investigators with the appropriate competencies and experience; • competing clinical trials for similar therapies or targeting patient populations meeting our patient eligibility criteria; • clinicians’ and patients’ perceptions as to the potential advantages and side effects of the product candidate being studied in relation to other available therapies and product candidates; • our ability to obtain and maintain patient consents; • pandemics or similar public health crises; and • the risk that patients enrolled in clinical trials will not complete such trials, for any reason.
We may experience difficulties in patient enrollment in our planned clinical trials for a variety of reasons, including: • the size and nature of the patient population; • the patient eligibility criteria defined in the protocol, and/or certain criteria related to stage of disease progression, which may limit the patient populations eligible for our clinical trials; • the size of the study population required for analysis of a trial’s primary endpoints; • the proximity of patients to a trial site; • the occurrence of future epidemics or pandemics or similar public health crises; • the design of a trial; • our ability to recruit clinical trial investigators with the appropriate competencies and experience; • competing clinical trials for similar therapies or targeting patient populations meeting our patient eligibility criteria; • changes in regulatory requirements in countries in which we conduct our clinical trials; • clinicians’ and patients’ perceptions as to the potential advantages and side effects of the product candidate being studied in relation to other available therapies and product candidates; • our ability to obtain and maintain patient consents; and • the risk that patients enrolled in clinical trials will not complete such trials, for any reason.
Violations of these laws and regulations could result in fines, criminals actions against us, our officers, or our employees, the closing down of our facilities, requirements to obtain export licenses, cessation of business activities in sanctioned countries, implementation of compliance programs, and prohibitions on the conduct of our business.
Violations of these laws and regulations could result in fines, criminal actions against us, our officers, or our employees, the closing down of our facilities, requirements to obtain export licenses, cessation of business activities in sanctioned countries, implementation of compliance programs, and prohibitions on the conduct of our business.
We do not have a sales or marketing infrastructure, nor have we sold, marketed, or distributed pharmaceutical products. To achieve commercial success for any approved product for which we retain sales and marketing responsibilities, we must either develop a sales and marketing organization or outsource these functions to third parties.
We do not have a sales or marketing infrastructure, nor have we sold, marketed, or distributed pharmaceutical products as a company. To achieve commercial success for any approved product for which we retain sales and marketing responsibilities, we must either develop a sales and marketing organization or outsource these functions to third parties.
Clinical trial delays could also shorten any periods during which our product shave patent protection and may allow our competitors to bring products to market before we do, which could impair our ability to successfully commercialize our product candidates and may harm our business and results of operations.
Clinical trial delays could also shorten any periods during which our products have patent protection and may allow our competitors to bring products to market before we do, which could impair our ability to successfully commercialize our product candidates and may harm our business and results of operations.
Any material decline in available funding or our ability to access our cash and cash equivalents or our ability to access bank financing could adversely impact our ability to meet our operating expenses and result in breaches of our contractual obligations which could have material adverse impacts on our operations and liquidity.
Any material decline in available funding or our ability to access our cash and cash equivalents or our ability to access bank financing could adversely impact our ability to meet our operating expenses and result in breaches of our contractual obligations which could have material adverse impacts on our operations and liquidity or otherwise adversely impact our viability.
Any such required financing may not be available in amounts or on terms acceptable to us, and the failure to procure such required financing could have a material and adverse effect on our business, financial condition and results of operations.
If available, any such required financing may not be available timely or in amounts or on terms acceptable to us, and the failure to procure such required financing could have a material and adverse effect on our business, financial condition and results of operations.
Notice of the certification must be given to the innovator, too, and if within 45 days of receiving notice the innovators use to protect its patents, approval of the ANDA is stayed for 30 months, or as lengthened or shortened by the court.
Notice of the certification must be given to the innovator, too, and if within 45 days of receiving notice the innovators sue to protect its patents, approval of the ANDA is stayed for 30 months, or as lengthened or shortened by the court.
We may encounter substantial delays in our current and planned clinical trials, or may not be able to conduct or complete our clinical trials on the timelines we expect, if at all. Our current and planned clinical trials are expensive, time consuming, and subject to uncertainty.
We may encounter substantial delays in our current and planned clinical trials, or may not be able to conduct or complete our clinical trials on the timelines we expect, if at all. Our current and planned clinical trials are expensive, time consuming, complex to manage and subject to uncertainty.
If the FDA does not approve the NDA under the Section 505(b)(2) regulatory pathway as anticipated, we may need to conduct additional clinical trials, provide additional data and information, and meet additional standards for regulatory approval.
If the FDA does not approve the NDA under the Section 505(b)(2) regulatory pathway as anticipated, we may need to conduct additional preclinical and/or clinical trials, provide additional data and information, and meet additional standards for regulatory approval.
We will continue to be a “smaller reporting company” until we have $250 million or more in public float (based on our Common Stock) measured as of the last business day of our most recently completed second fiscal quarter or, in the event we have no public float (based 64 on our Common Stock) or a public float (based on our Common Stock) that is less than $700 million, annual revenues of $100 million or more during the most recently completed fiscal year.
We will continue to be a “smaller reporting company” until we have $250 million or more in public float (based on our outstanding common 65 stock) measured as of the last business day of our most recently completed second fiscal quarter or, in the event we have no public float or a public float that is less than $700 million, annual revenues of $100 million or more during the most recently completed fiscal year.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeRisk Factors-Risks Related to Our Operations" in this Annual Report. Board Oversight of Cybersecurity Risks Our Board is responsible for the oversight of our risk management program and regularly reviews information regarding our most significant strategic, operational, financial, legal and compliance risks, including cybersecurity risks.
Biggest changeBoard is responsible for the oversight of our risk management program and regularly reviews information regarding our most significant strategic, operational, financial, legal and compliance risks, including cybersecurity risks. The Board reviews mitigation plans through discussions with management, which includes regular Board reports and findings from management’s monthly discussions with our outside IT group .
Our digital and technology organization outside the Company continually addresses cybersecurity risk in alignment with our business objectives and operational needs. Our cybersecurity program is focused on the following areas: • Governance: We leverage multiple cybersecurity frameworks (e.g., ISO 27001 and NIST CSF) and regulatory requirements to inform our externally managed information technology (“IT”) infrastructure.
Our digital and technology organization outside the Company continually addresses cybersecurity risk in alignment with our business objectives and operational needs. Our cybersecurity program is focused on the following areas: • Governance: We leverage multiple cybersecurity frameworks, primarily NIST CSF 2.0 and regulatory requirements to inform our externally managed information technology (“IT”) infrastructure.
We also disseminate security awareness information periodically throughout the year. • Third-Party Suppliers and Service Providers : We manage our IT infrastructure with an external vendor who maintains and evaluates risk exposure in real-time and conducts monthly risk assessments and adjustments to preclude cyberattacks.
All employees can be required to complete yearly cybersecurity awareness training based on internal testing. • Third-Party Suppliers and Service Providers : We manage our IT infrastructure with an external vendor who maintains and evaluates risk exposure in real-time and conducts monthly risk assessments and adjustments to preclude cyberattacks.
Instead, we deploy technical and procedural measures using a distributed model so that no one location or machine could cripple the company’s IT infrastructure.
Instead, we deploy technical and procedural measures using a distributed model so that no one location or machine could cripple the company’s IT infrastructure. Protection measures include, but are not limited to, network intrusion detection and prevention, vulnerability assessments and monthly risk assessments and management, threat intelligence, anti-malware and access controls.
Management's Role in Assessing and Managing Cybersecurity Risks 69 Our CEO in conjunction with our external IT team are responsible for setting the strategy and communicating cybersecurity risks. With the Company’s distributed model of data storage, risks have been limited and the Company has not experienced a cybersecurity breach.
Our Board of Directors also reviews and approves our cybersecurity policies, strategies, and budgets on an annual basis. Management's Role in Assessing and Managing Cybersecurity Risks The Company's CEO in conjunction with our external IT team are responsible for setting the strategy and communicating cybersecurity risks.
The Company’s security measures and monthly system audits to identify potential vulnerabilities and remediate deficiencies in real time.
With the Company’s distributed model of data storage, risks have been limited and the Company has not experienced a cybersecurity breach. The Company’s security measures and monthly system audits are designed to identify potential vulnerabilities and remediate deficiencies in real time.
Policies for IT use and management for each employee are part of the employee onboarding process and those policies are refreshed periodically as threats emerge that could be relevant to our IT infrastructure. • Technical Safeguards: The Company does not use a centralized server where all information is stored and therefore where all information is placed at risk.
The Company maintains an active IT security program that performs annual risk assessments, technical reviews, evaluation and implementation of IT controls, collects results and evidence of compliance and develops IT policies. • Technical Safeguards: The Company does not use a centralized server where all information is stored and therefore where all information is placed at risk.
Protection measures include network firewalls, network intrusion detection and prevention, penetration testing, vulnerability assessments and monthly risk assessments and management, threat intelligence, anti-malware and access controls, plus data loss prevention and monitoring. • Security Awareness / Training: All employees are required to adhere to our Standards of Business Conduct, which identifies an employee's responsibility for information security.
The Company's security team, along with our 24x7 security operations center (SOC) provides breach detection, monitoring, and remediation of identified risks. • Security Awareness / Training: All employees are required to adhere to our Standards of Business Conduct, which identifies an employee's responsibility for information security. We also disseminate security awareness information periodically throughout the year.
Removed
The Board reviews mitigation plans through discussions with management, which includes regular Board reports and findings from management’s monthly discussions with our outside IT group . Our Board of Directors also reviews and approves our cybersecurity policies, strategies, and budgets on an annual basis.
Added
The Company has implemented a Managed Detection and Response (MDR) program that provides a single pane of glass visibility to our security activities through a unified dashboard monitored by our security team. The Company collects threat intelligence data from endpoints and network data flows and send logs for analysis and storage to our cloud-based log collector.
Added
Risk Factors-Risks Related to Our Operations" in this Annual Report. Board Oversight of Cybersecurity Risks 70 Our management is responsible for assessing and managing cybersecurity risks as part of its overall risk management process. The Board of Directors maintains oversight of our risk management framework and considers cybersecurity risks as part of its oversight of operational and information technology matters.

Item 2. Properties

Properties — owned and leased real estate

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Biggest changeItem 2. Pro perties. Our corporate headquarters is located in Atlanta, Georgia, where we lease a single corporate office. Additionally, in August 2022 we opened an office, consisting of approximately 4,200 square feet, in Lexington, Massachusetts which we use as office and conference spaces for our Massachusetts based team.
Biggest changeItem 2. Pro perties. Our corporate headquarters is located in Wilmington, Delaware. Additionally, in August 2022 we opened an office, consisting of approximately 4,200 square feet, in Lexington, Massachusetts which we used as office and conference spaces for our Massachusetts based team.
Removed
We have an option to extend the lease term for an additional three years thereafter. It is anticipated that these facilities will be sufficient to meet our needs for the foreseeable future.
Added
The lease ran through September 2025 and we did not renew or continue occupancy of this premises upon lease expiration.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeMine Saf ety Disclosures. Not applicable. 70 PAR T II
Biggest changeMine Saf ety Disclosures. Not applicable. 71 PAR T II

Item 4. Mine Safety Disclosures

Mine Safety Disclosures — required of mining issuers

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Biggest changeItem 4. Mine Safety Disclosures 70 PART II Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities 71 Item 6. [Reserved] 71 Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations 72 Item 7A. Quantitative and Qualitative Disclosures About Market Risk 80 Item 8.
Biggest changeItem 4. Mine Safety Disclosures 71 PART II Item 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities 72 Item 6. [Reserved] 72 Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations 73 Item 7A. Quantitative and Qualitative Disclosures About Market Risk 81 Item 8.

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeNot reflected in the number of stockholders of record are persons who beneficially own shares of common stock held in nominee or street name. Dividends We have never previously declared or paid any cash dividends on our capital stock.
Biggest changeHolders of Record Equiniti Trust Company, LLC is the transfer agent for our common stock. As of March 23, 2026, there were 27 holders of record of our common stock. Not reflected in the number of stockholders of record are persons who beneficially own shares of common stock held in nominee or street name.
Any future determination to pay cash dividends will be at the sole discretion of our Board of Directors and will depend upon the financial condition of the Company, its operating results, capital requirements, general business conditions and any other factors that our Board of Directors deems relevant.
Any future determination to pay cash dividends will be at the sole discretion of our Board of Directors and will depend upon our financial condition, our operating results, capital requirements, general business conditions and any other factors that our Board of Directors deems relevant.
We currently intend to retain earnings and profits, if any, to support our business strategy and do not intend to pay any cash dividends within the foreseeable future.
Dividends We have never previously declared or paid any cash dividends on our capital stock and have incurred losses continuously since our inception. We currently intend to retain earnings and profits, if any, to support our business strategy and do not intend to pay any cash dividends within the foreseeable future.
Removed
Holders of Record Equiniti Trust Company, LLC (formerly known as American Stock Transfer & Trust Company, LLC) is the transfer agent for our common stock. As of March 21, 2025, there were 29 holders of record of our common stock.

Item 7. Management's Discussion & Analysis

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

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Biggest changeCash Flows The following table sets forth a summary of the primary sources and uses of cash for each of the years presented below: Year ended December 31, 2024 2023 Net cash used in operating activities $ (19,148,067 ) $ (18,085,043 ) Net cash (used in) provided by investing activities (37,004,201 ) 11,656,666 Net cash provided by financing activities 103,477,668 8,405,003 Net increase in cash and cash equivalents $ 47,325,400 $ 1,976,626 Net Cash Flows Used in Operating Activities Net cash flows used in operating activities for the year ended December 31, 2024 totaled $19,148,067, and consisted primarily of a net loss of $27.5 million adjusted for non-cash stock compensation of $8.1 million, a decrease in prepaid expenses and other assets of $0.6 million, an increase in accounts payable of $0.3 million, an increase in accrued expenses and other current liabilities of $0.4 million and an increase in prepaid research and development of $0.1 million. 77 Net cash flows used in operating activities for the year ended December 31, 2023 totaled $18,085,043, and consisted primarily of a net loss of $19.0 million adjusted for non-cash stock compensation of $0.5 million, depreciation and lease expense of $0.2 million, increase in prepaid expenses and other assets of $0.1 million, decrease in accounts payable of $0.5 million, decrease in prepaid research and development of $0.9 million and a decrease in accrued expenses and other current liabilities of $0.1 million.
Biggest changeCash Flows The following table sets forth a summary of the primary sources and uses of cash for each of the years presented below: Year ended December 31, 2025 2024 Net cash used in operating activities $ (27,786,464 ) $ (19,148,067 ) Net cash provided by (used in) investing activities 2,053,130 (37,004,201 ) Net cash provided by financing activities 108,462,963 103,477,668 Net increase in cash and cash equivalents $ 82,729,629 $ 47,325,400 Net Cash Flows Used in Operating Activities Net cash flows used in operating activities for the year ended December 31, 2025 totaled $27.8 million, and consisted primarily of a net loss of $48.3 million adjusted for non-cash stock compensation of $15.3 million, a write-off of in-process research and development of $7.4 million associated with the CorHepta transaction, a decrease in the fair value of contingent consideration of $1.4 million associated with the CorHepta transaction, non-cash accretion on marketable securities of $0.9 million, an increase prepaid research and development of $1.9 million mainly associated with our PAH program, a decrease in prepaid expenses and other current assets of $0.5 million and an increase in accrued expenses and other current liabilities of $1.4 million.
Outside professional services consist of legal, accounting and audit services and other consulting fees. Allocated expenses consist of rent expenses related to our offices in Lexington, Massachusetts and Atlanta, Georgia not otherwise included in research and development expenses.
Outside professional services consist of legal, accounting and audit services and other consulting fees. Allocated expenses consist of rent expenses related to our former offices in Lexington, Massachusetts and Atlanta, Georgia not otherwise included in research and development expenses.
Stock-Based Compensation We have granted stock-based awards, consisting of non-qualified stock options, to our employees, certain non-employee consultants and members of our board of directors, both past and present.
Stock-Based Compensation We have granted stock-based awards, consisting of non-qualified stock options and incentive stock options, to our employees, and non-qualified stock options to certain non-employee consultants and members of our board of directors, both past and present.
We believe that our existing cash, cash equivalents and marketable securities as of December 31, 2024 will enable us to fund our operating requirements for at least the next twelve months following the date of this Annual Report.
We believe that our existing cash, cash equivalents and marketable securities as of December 31, 2025, will enable us to fund our operating requirements for at least the next twelve months following the date of this Annual Report.
All external costs are tracked by therapeutic indication. We do not track personnel or other operating expenses incurred for our research and development programs on a program-specific basis. These expenses primarily relate to salaries and benefits and stock-based compensation and office consumables.
All external costs are tracked by therapeutic indication. We do not track other operating expenses incurred for our research and development programs on a program-specific basis. These expenses primarily relate to stock-based compensation and office consumables.
The timing and amount of our operating expenditures will depend largely on: • the timing and progress of preclinical and clinical development activities; • the number and scope of preclinical and clinical programs we decide to pursue; • the progress of the development efforts of third parties with whom we have entered into license and collaboration agreements; • our ability to maintain our current research and development programs and to establish new research and development, license or collaboration arrangements; • our ability and success in securing manufacturing relationships with third parties or, in the future, in establishing and operating a manufacturing facility; • the costs involved in prosecuting, defending and enforcing patent claims and other intellectual property claims; • the cost and timing of regulatory approvals; • our efforts to enhance operational, financial and information management systems and hire additional personnel, including personnel to support development of our product candidates; • the costs and ongoing investments to in-license and/or acquire additional technologies; and • possible delays or interruptions to preclinical studies, clinical trials, our receipt of services from our third-party service providers on whom we rely, or our supply chain due to epidemics or pandemics.
The timing and amount of our operating expenditures will depend largely on: • the timing and progress of preclinical and clinical development activities; 76 • the number and scope of preclinical and clinical programs we decide to pursue; • the progress of the development efforts of third parties with whom we have entered into license and collaboration agreements; • our ability to maintain our current research and development programs and to establish new research and development, license or collaboration arrangements; • our ability and success in securing manufacturing relationships with third parties or, in the future, in establishing and operating a manufacturing facility; • any costs, including upfront of or licensing costs, associated with new programs such as any in-licensed new compounds or expanded indications of IKT-001; • the costs involved in prosecuting, defending and enforcing patent claims and other intellectual property claims; • the cost and timing of regulatory approvals; • our efforts to enhance operational, financial and information management systems and hire additional personnel, including personnel to support development of our product candidates; • the costs and ongoing investments to in-license and/or acquire additional technologies; and • possible delays or interruptions to preclinical studies, clinical trials, our receipt of services from our third-party service providers on whom we rely, or our supply chain due to epidemics or pandemics.
We are also increasing our administrative headcount as a public company and as we advance our product candidates through clinical development, which will also likely require us to increase our selling, general and administrative expenses.
We also are increasing our headcount as we advance our product candidates through clinical development, which will also require us to increase our selling, general and administrative expenses.
Research and development expenses represent costs incurred by us for the discovery and development of our product candidates and the development of our RAMP™ drug discovery program and prodrug technologies and include: employee-related expenses, such as salaries, benefits, travel and non-cash stock-based compensation expense; external research and development expenses incurred under arrangements with third parties, such as CROs, preclinical testing organizations, clinical testing organizations, CMOs, academic and non-profit institutions and consultants; costs to acquire technologies to be used in research and development that have not reached technological feasibility and have no alternative future use; license fees; and other expenses, which include direct and allocated expenses for laboratory, facilities and other costs. 78 As part of the process of preparing consolidated financial statements, we are required to estimate and accrue expenses.
Research and development expenses represent costs incurred by us for the discovery and development of our product candidates and prodrug technologies and include: employee-related expenses, such as salaries, benefits, travel and non-cash stock-based compensation expense; external research and development expenses incurred under arrangements with third parties, such as CROs, preclinical testing organizations, clinical testing organizations, CMOs, academic and non-profit institutions and consultants; costs to acquire technologies to be used in research and development that have not reached technological feasibility and have no alternative future use; license fees; and other expenses, which include direct and allocated expenses for laboratory, facilities and other costs.
This is due to the numerous risks and uncertainties associated with drug development, including the uncertainty of: • our ability to add and retain key research and development personnel and other key employees; • our ability to successfully file IND and NDA applications with the FDA; • our ability to conduct and commence trials; • our ability to establish an appropriate safety profile with IND-enabling toxicology studies; • our ability to successfully develop, obtain regulatory approval for, and then successfully commercialize, our product candidates; • our successful enrollment in and completion of our current and future clinical trials; • the costs associated with the development of any additional product candidates we identify in-house or acquire through collaborations; • our ability to discover, develop and utilize biomarkers to demonstrate target engagement, pathway engagement and the impact on disease progression of our molecules; • our ability to establish agreements with third party manufacturers for clinical supply for any future clinical trials and commercial manufacturing, if our product candidates are approved; • the terms and timing of any collaboration, license or other arrangement, including the terms and timing of any milestone payments thereunder; • our ability to obtain and maintain patent, trade secret and other intellectual property protection and regulatory exclusivity for our product candidates if and when approved; • our receipt of marketing approvals from applicable regulatory authorities; 74 • the impact of the outbreak of the COVID-19 pandemic or other future pandemics; • our ability to commercialize products, if and when approved, whether alone or in collaboration with others; and • the continued acceptable safety profiles of the product candidates following approval.
This is due to the numerous risks and uncertainties associated with drug development, including the uncertainty of: • our ability to add and retain key research and development personnel and other key employees; 73 • our ability to successfully file IND and NDA applications with the FDA; • our ability to commence and conduct trials; • our ability to establish an appropriate safety or tolerability profile with IND-enabling toxicology studies; • our ability to successfully develop, obtain regulatory approval for, and then successfully commercialize, our product candidates; • our successful enrollment in and completion of our current and future clinical trials; • our ability to produce sufficient clinical product in a timely or cost effective manner to support our clinical trials; • the ability of our products to adequately exhibit product features (safety, efficacy, convenience) that are attractive to physicians and patients relative to offerings of our competitors; • the costs associated with the development of any additional product candidates we identify in-house or acquire through collaborations; • our ability to discover, develop and utilize biomarkers to demonstrate target engagement, pathway engagement and the impact on disease progression of our molecules; • our ability to establish agreements with third party manufacturers for clinical supply for any future clinical trials and commercial manufacturing, if our product candidates are approved; • the terms and timing of any collaboration, license or other arrangement, including the terms and timing of any milestone payments thereunder; • our ability to obtain and maintain patent, trade secret and other intellectual property protection and regulatory exclusivity for our product candidates if and when approved; • our receipt of marketing approvals from applicable regulatory authorities; • our ability to commercialize products, if and when approved, whether alone or in collaboration with others; and • the continued acceptable safety profiles of the product candidates following approval.
Cash Provided by Financing Activities Net cash provided by financing activities for the year ended December 31, 2024 totaled $103,477,668, which consisted of $3.8 million of net proceeds from issuance of common stock and pre-funded warrants in connection with our May 2024 Offering and our ATM Offering and $99.6 million of net proceeds from issuance of common stock and pre-funded warrants in connection with our October 2024 Offering.
Net cash provided by financing activities for the year ended December 31, 2024 totaled $103.5 million, which consisted of $3.8 million of net proceeds from issuance of common stock and pre-funded warrants in connection with our registered direct offering in May 2024 and our at-the-market offering and $99.6 million of net proceeds from the private placement of common stock and pre-funded warrants in October 2024.
Contractual Obligations and Commitments On April 18, 2022, the Company entered into an operating lease agreement through September 30, 2025 for its office space in Lexington, Massachusetts. The Lexington lease contains escalating payments during the lease period.
Contractual Obligations and Commitments In April 2022, we entered into an operating lease agreement through September 30, 2025 for our office space in Lexington, Massachusetts. The Lexington lease contained escalating payments during the lease period.
Unless otherwise discussed below, the Company does not believe that the adoption of recently issued standards has had or may have a material impact on the Company's consolidated financial statements or disclosures 79
Recent Accounting Pronouncements Unless otherwise discussed below, we do not believe that the adoption of recently issued standards has had or may have a material impact on our consolidated financial statements or disclosures. 80
The Company has incurred recurring losses and at December 31, 2024 had an accumulated deficit of $94,420,611. Future Funding Requirements To date, we have not generated any revenue from the sale of commercial products.
We have incurred recurring losses since our inception and at December 31, 2025 had an accumulated deficit of $142.7 million. Future Funding Requirements To date, we have not generated any revenue from the sale of commercial products.
Research and development expenses accounted for 60% and 67% of our operating expenses for the years ended December 31, 2024 and 2023, respectively. We record research and development expenses as incurred.
Components of Operating Results Operating Expenses Research and Development Research and development activities account for a significant portion of our operating expenses. Research and development expenses accounted for 57% and 60% of our operating expenses for the years ended December 31, 2025 and 2024, respectively. We record research and development expenses as incurred.
Additionally, any future collaborations we enter into with third parties may provide capital in the near term but limit our potential cash flow and revenue in the future.
Additionally, any future collaborations we enter into with third parties may provide capital in the near term but limit our potential cash flow and revenue in the future. Any of the foregoing could have a material adverse effect on our business, financial condition and results of operations.
Please also see the section titled “Special Note Regarding Forward-Looking Statements.” Overview We are a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary and other diseases including those that arise from aberrant signaling through the Abelson Tyrosine Kinase, and type III receptor tyrosine kinases including platelet derived growth factor receptors and c-KIT.
Please also see the section titled “Special Note Regarding Forward-Looking Statements.” Overview We are a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary diseases, namely, Pulmonary Arterial Hypertension (“PAH”), in which aberrant signaling through type III receptor tyrosine kinases, including platelet derived growth factor receptors and a stem cell factor receptor, known as “c-Kit”, has been implicated.
Liquidity and Capital Resources Sources of Liquidity From our inception up until our December 2020 Initial Public Offering, we funded our operations primarily through private, state and federal contracts and grants. At December 31, 2024, the Company had cash, cash equivalents, and marketable securities of $97,543,528.
The increase was driven by interest earned on our increased balances of cash, cash equivalents and marketable securities. 75 Liquidity and Capital Resources Sources of Liquidity From our inception up until our December 2020 initial public offering, we funded our operations primarily through private, state and federal contracts and grants.
A portion of our research and development expenses are external costs, which we track on a program-specific basis. We record the estimated expenses of research and development activities conducted by third party service providers as they are incurred and provided within research and development expense in the statements of operations.
We record the estimated expenses of research and development activities conducted by third party service providers as they are incurred and provided within research and development expense in the statements of operations. These services include the conduct of preclinical studies and consulting services. These costs are a significant component of our research and development expenses.
Upon execution of this lease agreement, the Company prepaid one month of rent, applied to the first month's rent, and a security deposit, which will be held in escrow and credited at the termination of the lease. Our total lease obligation is $114,966, consisting of minimum annual rental obligations of $114,966 for fiscal year 2025.
Upon execution of this lease agreement, we prepaid one month of rent, which applied to the first month's rent, and a security deposit, which is held in escrow and will be credited after the termination of the lease with the refund expected in the first half of 2026. Our total lease obligation at December 31, 2025 is $0.
We expect to incur additional expenses as a result of operating as a public company, including expenses related to compliance with the rules and regulations of the Securities and Exchange Commission (“SEC”) and those of any national securities exchange on which our securities are traded, additional insurance expenses, investor relations activities and other administrative and professional services.
As a public company, we incur expenses related to compliance with the rules and regulations of the SEC and those of Nasdaq, additional insurance expenses, investor relations activities and other administrative and professional services.
However, we may be unable to raise additional working capital, or if we are able to raise additional working capital we may be unable to do so on commercially favorable terms.
We expect to fund our operations through public equity or private equity or debt financings, as well as other sources. However, we may be unable to raise additional working capital, or if we are able to raise additional working capital, we may be unable to do so on commercially favorable terms.
Cash (Used in) Provided by Investing Activities Net cash flows used in investing activities for the year ended December 31, 2024, totaled $37,004,201, of which $60.5 million was used for the purchase of marketable securities investments and $23.5 million was provided by maturity of marketable securities.
Cash Provided by (Used in) Investing Activities Net cash flows provided by investing activities for the year ended December 31, 2025, totaled $2.1 million, of which $41.6 million was provided by maturity of marketable securities, $39.1 million was used for the purchase of marketable securities and $0.4 million related to acquired in-process research and development associated with the CorHepta acquisition discussed above. 77 Net cash flows provided by investing activities for the year ended December 31, 2024, totaled $37.0 million, of which $60.5 million was used for the purchase of marketable securities investments and $23.5 million was provided by maturity of marketable securities.
We are subject to all of the risks typically related to the development of new product candidates, and we may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect our business. Moreover, following the completion of the December 2020 initial public offering, we incurred additional costs associated with operating as a public company.
We are subject to all of the risks typically related to the development of new product candidates, and we may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect our business. We anticipate that we will need substantial additional funding in connection with our continuing operations.
Net cash provided by financing activities for the year ended December 31, 2023 totaled $8,405,003, which consisted of $8.5 million net from the issuance of common stock and pre-funded warrants and $0.1 million of deferred offering costs.
Cash Provided by Financing Activities Net cash provided by financing activities for the year ended December 31, 2025 totaled $108.5 million, which consisted of $107.6 million of net proceeds from issuance of common stock and pre-funded warrants in connection with our underwritten public offering in November 2025 and $0.8 million of net proceeds from the issuance of common stock related to the exercise of stock options.
We do not allocate personnel-related discretionary bonus or stock-based compensation costs, laboratory and related expenses, depreciation or other indirect costs that are deployed across multiple projects under development and, as such, the costs are separately classified as other research and development expenses in the table below: Year ended December 31, 2024 2023 Change PD $ 9,333,359 $ 9,036,750 $ 296,609 PAH 1,672,869 — 1,672,869 Other research and development expenses 6,204,320 4,581,598 1,622,722 Total research and development expenses $ 17,210,548 $ 13,618,348 $ 3,592,200 Selling, General and Administrative Selling, general and administrative expenses include personnel related expenses, such as salaries, benefits, travel and non-cash stock-based compensation expense, expenses for outside professional services and allocated expenses.
We do not allocate stock-based compensation costs, depreciation or other indirect costs that are deployed across multiple projects under development and, as such, the costs are separately classified as other research and development expenses in the table below: Year ended December 31, 2025 2024 Change Parkinson's disease $ 647,242 $ 9,333,359 $ (8,686,117 ) PAH (1) 22,453,185 1,672,869 20,780,316 Other research and development expenses 6,692,719 6,204,320 488,399 Total research and development expenses $ 29,793,146 $ 17,210,548 $ 12,582,598 (1) This amount includes a one-time (non-cash) charge of $7.4 million for the acquired In-Process Research and Development ( “IPR&D”) related to the CorHepta acquisition during the year ended December 31, 2025. 74 Selling, General and Administrative Selling, general and administrative expenses include personnel related expenses, such as salaries, benefits, travel and non-cash stock-based compensation expense, expenses for outside professional services and allocated expenses.
Critical Accounting Policies and Significant Judgments and Estimates This discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States (“GAAP”).
In March 2026, we signed a change order with the CRO related to our transition to a Phase 3 study in the amount of $48.2 million, increasing the total contracted amount under the arrangement to $73.7 million, of which $7.5 million is subject to achievement of certain performance milestones by the CRO. 78 Critical Accounting Policies and Significant Judgments and Estimates This discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States (“GAAP”).
The $4.6 million increase was primarily driven by an increase of $3.1 million in stock-based compensation, a $2.0 million increase in legal, consulting and compliance related fees partially offset by a $0.3 million decrease in Directors and Officers (“D&O”) insurance, a $0.5 million decrease in advertising and promotions and a net increase of $0.3 million in all other selling, general and administrative expenses.
The $12.2 million increase was primarily driven by an increase of $6.4 million in stock-based compensation, a $4.2 million increase in personnel-related costs, including severance costs of approximately $1.0 million for our former Chief Executive Officer and Chief Financial Officer, $0.8 million increase in legal, compliance and support service fees, a $0.2 million increase in insurance, primarily in Directors and Officers (“D&O”) insurance, and a $0.6 million increase in miscellaneous other expenses.
We have completed non-human primate safety studies and a bioequivalence clinical trial in healthy volunteers to determine the doses of IKT-001 that are equivalent to imatinib mesylate and the results are being utilized to set the doses in a Phase 2b trial to determine if IKT-001 could be a disease-modifying treatment for PAH.
PAH is a progressive, life-threatening disease characterized by pulmonary vascular remodeling and elevated pulmonary vascular resistance that affects approximately 50,000 Americans. We have completed a non-human primate safety study and a bioequivalence clinical study in healthy volunteers to determine the doses of IKT-001 that are equivalent to imatinib.
Results of Operations Comparison of the Years Ended December 31, 2024 and 2023 The following table sets forth the significant components of our results of operations: Year ended December 31, Change 2024 2023 ($) (%) Grant revenue $ — $ 260,501 $ (260,501 ) (100.0 ) Research and development (17,210,548 ) (13,618,348 ) (3,592,200 ) 26.4 Selling, general and administrative (11,378,520 ) (6,731,945 ) (4,646,575 ) 69.0 Loss from operations (28,589,068 ) (20,089,792 ) (8,499,276 ) (42.3 ) Interest income 1,069,182 1,060,909 8,273 0.8 Net loss $ (27,519,886 ) $ (19,028,883 ) $ (8,491,003 ) (44.6 ) 75 Grant Revenue Grant revenue for the year ended December 31, 2024 decreased by $260,501 or 100.0% to $0 from $260,501 in the prior year.
Results of Operations Comparison of the Years Ended December 31, 2025 and 2024 The following table sets forth the significant components of our results of operations: For the year ended December 31, Change 2025 2024 ($) (%) Research and development $ (29,793,146 ) $ (17,210,548 ) $ (12,582,598 ) 73.1 Selling, general and administrative (23,555,079 ) (11,378,520 ) (12,176,559 ) 107.0 Change in fair value contingent consideration 1,373,942 — 1,373,942 100.0 Loss from operations (51,974,283 ) (28,589,068 ) (23,385,215 ) 81.8 Interest income 3,715,094 1,069,182 2,645,912 247.5 Net loss $ (48,259,189 ) $ (27,519,886 ) $ (20,739,303 ) 75.4 Research and Development Research and development expenses increased by $12,582,598 or 73.1% to $29,793,146 from $17,210,548 in the prior year.
The $3.6 million increase was due to an increase of $4.5 million in stock-based compensation, an increase of $1.7 million in PAH expenses, a $0.3 million increase in risvodetinib (IkT-148009) expenses partially offset by a net decrease of $2.9 million in all other research and development activities.
The $12.6 million increase was primarily due to an increase of $11.4 million in the PAH program and other research and development expenses, together with a $9.9 million research and development expense related to the CorHepta transaction comprising a one-time (non-cash) expense charge for acquired IPR&D related to the CorHepta acquisition cost of $7.4 million and $2.5 million of stock-based compensation expense.
Any of the foregoing could have a material adverse effect on our business, financial condition and results of operations. 76 Since our inception, we have incurred significant losses and negative cash flows from operations. We have an accumulated deficit of $94,420,611 at December 31, 2024.
Since our inception, we have incurred significant losses and negative cash flows from operations. We have an accumulated deficit of $142.7 million at December 31, 2025. We expect to incur substantial additional losses in the future as we conduct and expand our research and development activities.
Interest Income Interest income increased by $8,273 or 0.8% to $1,069,182 from $1,060,909 in the prior comparable period. The increase was driven by interest earned on our cash, cash equivalents and marketable securities.
Interest Income Interest income increased by $2,645,912 or 247.5% to $3,715,094 from $1,069,182 in the prior comparable period.
Selling, General and Administrative Selling, general and administrative expenses increased by $4,646,575 or 69.0% to $11,378,520 from $6,731,945 in the prior year.
These increases were offset by a decrease of $8.7 million in the discontinued (outlicensed) risvodetinib (IkT-148009) program. Selling, General and Administrative Selling, general and administrative expenses increased by $12,176,559 or 107% to $23,555,079 from $11,378,520 in the prior year.
Removed
The Company’s multi-therapeutic pipeline is developing IKT-001, a prodrug of imatinib mesylate, for Pulmonary Arterial Hypertension (“PAH”).
Added
Our lead product candidate is IKT-001, a prodrug of imatinib mesylate (“imatinib”), for PAH which is an orphan indication.
Removed
We have also developed risvodetinib (also known as IkT-148009), a selective inhibitor of the non-receptor Abelson Tyrosine Kinases that targets the treatment of Parkinson’s disease inside and outside the brain. In 2021, we commenced clinical development of risvodetinib.
Added
Imatinib was first approved in the United States in 2001 for various cancers and blood disorders and, following more than 20 years of clinical use, has a well-characterized safety profile with the first reported use of imatinib in PAH occurring in 2005.
Removed
In 2023, we initiated the Phase 2 201 trial (“201 Trial”) for risvodetinib (IkT-148009) as a treatment for Parkinson’s disease and completed that trial on October 6, 2024. In January 2025, we reported results from the 201 Trial and decided to pause further development of risvodetinib as we focus our resources on advancing lead program IKT-001 in PAH.
Added
Our Phase 3 clinical study, named IMPROVE-PAH ( I KT-001 for M easuring P ulmonary Vascular R esistance and O utcome V ariables in a Phase 3 E valuation of PAH ), has been initiated with the activation of a small number of sites and the recent commencement of patient pre-screening activities at those sites.
Removed
We will consider our strategic options for the risvodetinib program. IKT-001 and PAH IKT-001 emerged from the Company’s medicinal chemistry program that aimed to develop improvements to drugs that inhibit Abelson Tyrosine Kinase and type III receptor tyrosine kinases.
Added
Change in Fair Value Contingent Consideration Change in fair value contingent consideration increased by $1,373,942 or 100% from $0 in the prior comparable period. The increase is due to the change in fair value of the contingent consideration related to the CorHepta transaction from the acquisition date of February 21, 2025 to December 31, 2025.
Removed
IKT-001, a prodrug of imatinib mesylate, was designed to improve areas of the molecule that might play a role in the gastrointestinal (“GI”) side effects commonly observed with oral imatinib mesylate, the current standard of care. A three-part dose finding/dose equivalence study in 66 healthy volunteers (known as ‘the 501 trial’) was completed with IKT-001 in 2023.
Added
In October 2024, we raised approximately $99.6 million in net proceeds from a private placement and in November 2025, we raised approximately $107.6 million in net proceeds from our underwritten public offering.
Removed
The study was designed to evaluate the 96-hour single-dose pharmacokinetics of imatinib delivered as IKT-001 and determine the dose relationship between IKT-001 and imatinib mesylate.
Added
On June 20, 2025, we entered into an Open Market Sale Agreement SM (the “Sales Agreement”) with Jefferies LLC, as sales agent ("Jefferies"), pursuant to which we may, from time to time, issue and sell shares of our common stock through or to Jefferies.
Removed
Based on this study it was determined that bioequivalence was established with a 300 mg dose of IKT-001 to a dose of 230 mg of imatinib mesylate while a 500 mg dose of IKT-001 was established as bioequivalent to a dose of 383 mg of imatinib mesylate.
Added
Under the terms of the Sales Agreement, Jefferies may sell the shares of our common stock at market prices by any method that is deemed to be an "at the market offering" as defined in Rule 415 under the Securities Act of 1933, as amended.
Removed
These doses are adequate to cover the target systemically and were similar to the doses of imatinib mesylate used in the Phase 3 IMPRES trial in PAH.
Added
As of December 31, 2025, no shares of our common stock had been sold under the Sales Agreement. In February 2026, we sold 1,904,762 shares of common stock pursuant to the Sales Agreement for an aggregate gross sales price of $3.0 million. At December 31, 2025, we had cash, cash equivalents, and marketable securities of $178.8 million.
Removed
On January 19, 2024, we met with the Food and Drug Administration (“FDA”) Hematological Malignancy Review Team (“Review Team”) in a Pre-New Drug Application (“pre-NDA”), meeting to discuss our bioequivalence studies of IKT-001 and its path to approval. All questions were addressed and summarized in official meeting minutes the issued by the FDA on February 12, 2024.
Added
Net cash flows used in operating activities for the year ended December 31, 2024 totaled $19.1 million, and consisted primarily of a net loss of $27.5 million adjusted for non-cash stock compensation of $8.1 million, a decrease in prepaid expenses and other assets of $0.6 million, an increase in accounts payable of $0.3 million, an increase in accrued expenses and other current liabilities of $0.4 million and an increase in prepaid research and development of $0.1 million.
Removed
During the meeting, we inquired whether additional clinical studies would be needed to seek approval and discussed manufacturing and quality control requirements for approval. The Review Team acknowledged that the 505(b)(2) pathway appeared to be the appropriate pathway for approval of IKT-001.
Added
In July 2025, we entered into a clinical trial supply agreement in the amount of approximately $6.5 million with a clinical trial supply organization whereby the clinical trial supply organization will provide services for our Phase 2b clinical study in PAH, known as IMPROVE-PAH. In November 2025, we began transitioning the PAH Phase 2b study to a Phase 3 study.
Removed
The Review Team also discussed the possible difference between IKT-001 and imatinib mesylate absorption in the gut and recommended that we evaluate whether IKT-001 and imatinib mesylate behave differently with respect to certain gut transporters that regulate absorption.
Added
The estimated total remaining contract costs as of December 31, 2025 is approximately $6.3 million. The estimated period of performance for the committed work with the clinical trial supply organization is through the first quarter of 2028.
Removed
This evaluation was completed and determined that IKT-001 and imatinib mesylate have similar behavior toward the transporters P-glycoprotein (“PGP”) and the Breast Cancer Resistance Protein (“BCRP”). Finally, a number of recommendations were discussed to prevent the potential mix-up between IKT-001 and imatinib mesylate either at the pharmacy or by patients for two drugs delivering the same active ingredient.
Added
In August 2025, we entered into an arrangement with a contract research organization (“CRO”) to support our Phase 2b clinical study in PAH, known as IMPROVE-PAH. As of December 31, 2025, the total contracted amount under this arrangement is $25.5 million, of which $2.6 million is subject to achievement of certain performance milestones by the CRO.
Removed
The Company discussed alternate dosage forms for IKT-001 relative to imatinib mesylate as the primary mitigation strategy and will provide a justification of the dosage forms chosen and why they are unlikely to cause medication errors if/when the Company submits a New Drug Application (“NDA”) for approval of IKT-001 in these cancer indications.
Added
In November 2025, we transitioned the PAH Phase 2b study to a Phase 3 study and began evaluating the arrangement together with the CRO. The estimated total remaining contract costs as of December 31, 2025 is approximately $18.4 million, excluding potential milestone payments. The estimated period of performance for the committed work with the CRO is through 2028.
Removed
PAH is a rare disease of the pulmonary microvasculature found in 15 to 50 persons per million within the United States and Europe. The global PAH market size was valued at $7.66 billion in 2023 and is estimated to grow at a compound annual growth rate of 5.4% between 2024 to 2030.
Added
We made an upfront payment of $1.9 million to the CRO, of which $1.0 million will be held as a retainer until the end of the study and applied against final invoicing and $0.9 million will be applied to passthrough costs as incurred.
Removed
Most of the treatments that constitute the standard of care (e.g. ERAs, PDE5s, prostacyclins) primarily act as vasodilators. In 2024, sotatercept was approved for the treatment of PAH on top of SOC. Sotatercept is recombinant fusion protein 72 that acts as a trap for transforming growth factor-beta superfamily ligands, including activin A and bone morphogenetic protein 9.
Added
The amount and timing of any such payments related to the $2.6 million performance milestones are contingent upon the vendor meeting specific contractual criteria. As of December 31, 2025, the achievement of these milestones is not considered probable, and the potential payments cannot be reasonably estimated. Accordingly, no liability has been recorded in the accompanying consolidated financial statements.
Removed
These ligands may play a role in the development and progression of PAH by promoting cell proliferation and fibrosis. The success of sotatercept has created renewed enthusiasm around the anti-proliferative pathways in PAH.
Added
We will continue to evaluate this arrangement each reporting period and will recognize a liability when achievement of the milestones become probable, and the amount can be reasonably estimated.
Removed
As previously mentioned, imatinib inhibits Abelson Tyrosine Kinase and type III receptor tyrosine kinases and through these pathways inhibits Platelet-derived growth factor receptor which is involved in cell proliferation and angiogenesis as well as Stem cell factor receptor which targets mast cells and other hematopoietic progenitors. Through these targets imatinib may inhibit vascular smooth muscle cell proliferation and fibrosis.
Added
As part of the process of preparing consolidated financial statements, we are required to estimate and accrue expenses. A portion of our research and development expenses are external costs, which we track on a program-specific basis.
Removed
This pathway may provide an alternate pathway for disease modification in PAH. The first reports of the use of imatinib in PAH were published in 2005 and 2006. A phase 2, RCT was subsequently conducted showing clinical benefit of imatinib in PAH.
Added
The intrinsic value of all in-the-money outstanding options as of December 31, 2025 was approximately $7.7 million, based on the closing price of our common stock of $2.05 per share at December 31, 2025, and $5.7 million of the intrinsic value of options was exercisable. 79 Contingent Consideration Liabilities We evaluate acquisitions of assets and other similar transactions to assess whether or not the transaction should be accounted for as a business combination or asset acquisition by first applying a screen test to determine if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets.
Removed
In 2013, the outcome of a Phase 3 trial (IMPRES) evaluating imatinib mesylate as a treatment for PAH was reported, demonstrating that imatinib may improve key parameters associated with PAH.
Added
If the screen is met, the transaction is accounted for as an asset acquisition. Significant judgment is required in the application of the screen test to determine whether an acquisition is a business combination or an acquisition of assets.
Removed
In this study imatinib improved exercise capacity and hemodynamics in patients with advanced PAH but approval was precluded because of the bleeding risk associated with concomitant anti-coagulant therapy and the high discontinuation rate in the imatinib group.
Added
On February 21, 2025, we entered into an Agreement and Plan of Merger and Reorganization (“Merger Agreement”) with Project IKT Merger Sub, Inc., a Delaware corporation and our wholly-owned subsidiary and CorHepta Pharmaceuticals, Inc. (“CorHepta”).
Removed
As we considered revisiting the use of imatinib in PAH, we recognized that changes in standard-of-care for these patients may have alleviated much of the safety risk previously observed for imatinib in PAH patients. This analysis prompted us to file a pre-IND (“PIND”) meeting request to discuss the application of IKT-001 as a potential disease-modifying treatment for PAH.
Added
We determined that the transaction represented an asset acquisition as defined by ASC 805 as substantially all of the value was attributed to a single intangible asset, in-process research and development (“IPR&D”). The fair value was determined based on our share price at closing.
Removed
To evaluate this further, members of the Company met with the FDA Division of Cardiology and Nephrology in a PIND meeting to discuss our plan to utilize IKT-001 in a Phase 2b efficacy, safety and tolerability study in PAH.
Added
We agreed to issue 4,979,101 shares of our common stock to the shareholders of CorHepta, of which (i) 829,849 shares were fully vested on the acquisition date, (ii) 2,489,030 shares represented contingent consideration which were subject to the achievement of certain milestones by February 21, 2026, and (iii) 1,660,222 shares represented post-merger compensation expense, subject to both service- and performance-based vesting conditions.
Removed
At the meeting, the FDA confirmed that IKT-001 would be viewed as a New Molecular Entity (“NME”) and that the appropriate path for approval remained to be the 505(b)(2) statute. This opens up the possibility of IKT-001 being granted NME status and market exclusivity on approval.
Added
The performance milestone was not satisfied as of February 21, 2026 and therefore these shares were forfeited as of that date. As of the acquisition date, the achievement of one of the contingent consideration milestones was deemed probable, and the fair value of the related shares was included in the purchase price of the acquisition.
Removed
The FDA requested at the PIND meeting that we conduct a comparative cell-culture based study of the human Ether-a-go-go-related Gene (“hERG”) ion channel, a standard cardiovascular safety test performed for any NME for which a new Investigative New Drug Application (“IND”) is to be opened. Neither IKT-001 nor imatinib mesylate were found to be inhibitors of hERG.

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Other IKT 10-K year-over-year comparisons