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

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Paragraph-level year-over-year comparison of Keros 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.

+521 added−574 removedSource: 10-K (2026-03-04) vs 10-K (2025-02-26)

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

521 paragraphs added · 574 removed · 370 edited across 7 sections

Item 1. Business

Business — how the company describes what it does

113 edited+51 added−125 removed332 unchanged
Biggest changeConduct and Ethics At Keros, we are committed to fostering a culture of integrity and ensuring each of our employees is equipped with resources to help them do the right thing. We believe it is imperative that the board of directors and senior management strongly support a no-tolerance stance for workplace harassment, biases and unethical behavior.
Biggest changeWe believe it is imperative that the board of directors and senior management strongly support a no-tolerance stance for workplace harassment, biases and unethical behavior. All employees are required to abide by, review and confirm compliance to the Company’s Code of Business Conduct and Ethics and Insider Trading policies upon hire and on an annual basis thereafter.
We are a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including blood, bone, skeletal muscle, adipose and heart tissue.
We are a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including skeletal muscle, bone, adipose, heart tissue and blood.
Our strategy focuses on the role of members of the TGF-ß family of proteins in the development of a number of tissues, including skeletal muscle, bone, blood, adipose and heart tissue. Aged and damaged cells are routinely replaced by new cells in normally functioning organs.
Our strategy focuses on the role of members of the TGF-ß family of proteins in the development of a number of tissues, including skeletal muscle, bone, adipose, heart tissue and blood. Aged and damaged cells are routinely replaced by new cells in normally functioning organs.
Neuromuscular disorders can progress rapidly or slowly. Decline in muscle mass can also be associated with secondary osteoporosis and obesity. One example of a rapidly progressive condition is Duchenne muscular dystrophy, or DMD, which is the most common form of muscular dystrophy and results in muscle degeneration and premature death.
Neuromuscular disorders can progress rapidly or slowly. Decline in muscle mass can also be associated with secondary osteoporosis and obesity. Duchenne Muscular Dystrophy One example of a rapidly progressive condition is Duchenne muscular dystrophy, or DMD, which is the most common form of muscular dystrophy and results in muscle degeneration and premature death.
We are also subject to additional similar U.S. state and foreign law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to sales or marketing arrangements and claims involving healthcare 42 items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor, state laws which require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, state and local laws which require pharmaceutical companies to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures, state laws which require the reporting of information related to drug pricing, state and local laws requiring the registration of pharmaceutical sales representatives, and state and foreign laws governing the privacy and security of health information which, in some cases, differ from each other in significant ways, and may not have the same effect, thus complicating compliance efforts.
We are also subject to additional similar U.S. state and foreign law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor, state and foreign laws which require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the government, state and local laws which require pharmaceutical companies to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures, state laws which require the reporting of information related to drug pricing, state and local laws requiring the registration of pharmaceutical sales representatives, and state and foreign laws governing the privacy and security of health information which, in some cases, differ from each other in significant ways, and may not have the same effect, thus complicating compliance efforts.
The process required by the FDA before biopharmaceutical product candidates may be marketed in the United States generally involves the following: ▪ completion of extensive preclinical laboratory tests and animal studies performed in accordance with applicable regulations, including the FDA’s Good Laboratory Practice, or GLP, requirements; ▪ submission to the FDA of an Investigational New Drug, or IND, application which must become effective before human clinical trials may begin; ▪ approval by an independent institutional review board, or IRB, or ethics committee at each clinical site before the trial may be initiated; ▪ performance of adequate and well-controlled human clinical trials in accordance with applicable IND regulations, good clinical practice, or GCP, requirements and other clinical trial-related regulations to establish the safety and efficacy of the investigational drug product for each proposed indication and to establish the safety, purity and potency of the investigational biologic product candidate for each proposed indication; ▪ preparation of and submission to the FDA of an NDA for a small molecule product candidate or a BLA for a biologic after completion of all pivotal clinical trials; ▪ payment of user fees for FDA review of the NDA or BLA; 33 ▪ a determination by the FDA within 60 days of its receipt of the NDA or BLA to file the application for review; ▪ satisfactory completion of one or more FDA pre-approval inspections of the manufacturing facility or facilities at which the proposed product will be produced to assess compliance with current Good Manufacturing Practice, or cGMP, requirements and to assure that the facilities, methods and controls are adequate to preserve the product’s continued identity, strength, quality and purity; ▪ potential FDA audit of the preclinical study and/or clinical trial sites that generated the data in support of the NDA or BLA; ▪ satisfactory completion of an FDA Advisory Committee review, if applicable; ▪ FDA review and approval of an NDA or licensure of a BLA, including consideration of the views of any FDA Advisory Committee, prior to any commercial marketing or sale of the product for particular indications for use in the United States; and ▪ compliance with any post-approval requirements, including the potential requirement to conduct post-approval studies.
The process required by the FDA before biopharmaceutical product candidates may be marketed in the United States generally involves the following: ▪ completion of extensive preclinical laboratory tests and animal studies performed in accordance with applicable regulations, including the FDA’s Good Laboratory Practice, or GLP, requirements; ▪ submission to the FDA of an Investigational New Drug, or IND, application which must become effective before human clinical trials may begin; ▪ approval by an independent institutional review board, or IRB, or ethics committee at each clinical site before the trial may be initiated; 20 ▪ performance of adequate and well-controlled human clinical trials in accordance with applicable IND regulations, good clinical practice, or GCP, requirements and other clinical trial-related regulations to establish the safety and efficacy of the investigational drug product for each proposed indication and to establish the safety, purity and potency of the investigational biologic product candidate for each proposed indication; ▪ preparation of and submission to the FDA of an NDA for a small molecule product candidate or a BLA for a biologic after completion of all pivotal clinical trials; ▪ payment of user fees for FDA review of the NDA or BLA; ▪ a determination by the FDA within 60 days of its receipt of the NDA or BLA to file the application for review; ▪ satisfactory completion of one or more FDA pre-approval inspections of the manufacturing facility or facilities at which the proposed product will be produced to assess compliance with current Good Manufacturing Practice, or cGMP, requirements and to assure that the facilities, methods and controls are adequate to preserve the product’s continued identity, strength, quality and purity; ▪ potential FDA audit of the preclinical study and/or clinical trial sites that generated the data in support of the NDA or BLA; ▪ satisfactory completion of an FDA Advisory Committee review, if applicable; ▪ FDA review and approval of an NDA or licensure of a BLA, including consideration of the views of any FDA Advisory Committee, prior to any commercial marketing or sale of the product for particular indications for use in the United States; and ▪ compliance with any post-approval requirements, including the potential requirement to conduct post-approval studies.
The U.S. federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics and medical supplies that are reimbursable under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to the Centers for Medicare & Medicaid Services, or CMS, information related to certain payments and other transfers of value made in the prior year to physicians (defined to include doctors, dentists, optometrists, podiatrists, and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members.
The U.S. federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics and medical supplies that are reimbursable under Medicare, Medicaid or the Children’s Health Insurance Program, with specific 29 exceptions, to report annually to the Centers for Medicare & Medicaid Services, or CMS, information related to certain payments and other transfers of value made in the prior year to physicians (defined to include doctors, dentists, optometrists, podiatrists, and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members.
These studies are designed to test the safety, dosage tolerance, absorption, metabolism and distribution of the investigational product in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness. 34 ▪ Phase 2 clinical trials involve studies in a limited population of disease-affected patients to evaluate the preliminary efficacy, optimal dosages and dosing schedule and to identify possible adverse side effects and safety risks. ▪ Phase 3 clinical trials generally involve a large number of patients at multiple geographically dispersed clinical trial sites and are designed to further evaluate dosage, to provide statistically significant evidence of clinical efficacy and to further test for safety.
These studies are designed to test the safety, dosage tolerance, absorption, metabolism and distribution of the investigational product in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness. ▪ Phase 2 clinical trials involve studies in a limited population of disease-affected patients to evaluate the preliminary efficacy, optimal dosages and dosing schedule and to identify possible adverse side effects and safety risks. ▪ Phase 3 clinical trials generally involve a large number of patients at multiple geographically dispersed clinical trial sites and are designed to further evaluate dosage, to provide statistically significant evidence of clinical efficacy and to further test for safety.
If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan drug exclusivity, which means that the FDA may not approve any other applications, including a full NDA or BLA, to market the same product for the same indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity or if the FDA finds that the holder of the orphan drug exclusivity has not shown that it can assure the availability of sufficient quantities of the orphan drug 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 the disease for which it has such designation, the product is entitled to orphan drug exclusivity, which means that the FDA may not approve any other applications, including a full NDA or BLA, to market the same product for the same indication for seven years, except in 23 limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity or if the FDA finds that the holder of the orphan drug exclusivity has not shown that it can assure the availability of sufficient quantities of the orphan drug to meet the needs of patients with the disease or condition for which the drug was designated.
In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. The National 11 Organization for Rare Disorders estimates that approximately one in every 3,500 male births is affected by DMD worldwide.
In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. The National Organization for Rare Disorders estimates that approximately one in every 3,500 male births is affected by DMD worldwide.
These non-RS patients have differentiation and maturation defects occurring across the spectrum from early through terminal stages of hematopoiesis. 18 Limitations of Current Treatment Options for Cytopenias in Patients with MDS Patients with MDS-associated anemia are generally treated with red blood cell transfusions and erythropoiesis-stimulating agents, or ESAs, which are not approved for such treatment.
These non-RS patients have differentiation and maturation defects occurring across the spectrum from early through terminal stages of hematopoiesis. Limitations of Current Treatment Options for Cytopenias in Patients with MDS Patients with MDS-associated anemia are generally treated with red blood cell transfusions and erythropoiesis-stimulating agents, or ESAs, which are not approved for such treatment.
Myelofibrosis is characterized by ineffective hematopoiesis, an enlarged spleen, bone marrow fibrosis and shortened survival. Patients often experience multiple disease-associated and treatment-emergent cytopenias, including anemia and thrombocytopenia. 19 The ineffective hematopoiesis in myelofibrosis is driven by molecular abnormalities in the Janus kinase 2, or JAK2, -signal transducers and activators of transcription, or JAK-STAT, signaling pathway of transcriptional activators.
Myelofibrosis is characterized by ineffective hematopoiesis, an enlarged spleen, bone marrow fibrosis and shortened survival. Patients often experience multiple disease-associated and treatment-emergent cytopenias, including anemia and thrombocytopenia. The ineffective hematopoiesis in myelofibrosis is driven by molecular abnormalities in the Janus kinase 2, or JAK2, -signal transducers and activators of transcription, or JAK-STAT, signaling pathway of transcriptional activators.
We may terminate the agreement for any reason upon specified prior written notice to MGH. 31 Intellectual Property Overview We strive to protect the proprietary technology, inventions and improvements that we believe are commercially important to our business, including obtaining, maintaining, enforcing and defending our intellectual property rights, including patent rights, whether developed internally or licensed from third parties.
We may terminate the agreement for any reason upon specified prior written notice to MGH. Intellectual Property Overview We strive to protect the proprietary technology, inventions and improvements that we believe are commercially important to our business, including obtaining, maintaining, enforcing and defending our intellectual property rights, including patent rights, whether developed internally or licensed from third parties.
The CTR foresaw a three-year transition period that ended on January 31, 2025. Since this date, all new or ongoing trials are subject to the provisions of the CTR. In all cases, clinical trials must be conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.
The CTR foresaw a three-year transition period that ended on January 31, 2025. Since this date, all new or ongoing trials are subject to the provisions of the CTR. 25 In all cases, clinical trials must be conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.
Eligible products must target conditions for which there is an unmet medical need (there is no satisfactory method of diagnosis, prevention or 39 treatment in the EU or, if there is, the new medicinal product will bring a major therapeutic advantage) and they must demonstrate the potential to address the unmet medical need by introducing new methods of therapy or improving existing ones.
Eligible products must target conditions for which there is an unmet medical need (there is no satisfactory method of diagnosis, prevention or treatment in the EU or, if there is, the new medicinal product will bring a major therapeutic advantage) and they must demonstrate the potential to address the unmet medical need by introducing new methods of therapy or improving existing ones.
Symptoms of neuromuscular disease include increasing generalized weakness and fatigue, dysphagia, dyspnea on exertion and at rest, sleepiness, morning headache, difficulties with concentration and mood changes. Most neuromuscular diseases are characterized by progressive muscular impairment leading to loss of muscle function and can lead to loss of ambulation, being wheelchair-bound, swallowing difficulties, respiratory muscle weakness and death.
Symptoms of neuromuscular disease include increasing generalized weakness and fatigue, dysphagia, dyspnea on exertion and at rest, sleepiness, morning headache, difficulties with concentration and mood changes. Most neuromuscular diseases are characterized by progressive muscular impairment 2 leading to loss of muscle function and can lead to loss of ambulation, being wheelchair-bound, swallowing difficulties, respiratory muscle weakness and death.
While ESAs have been shown to alleviate anemia in a subset of patients with MDS, patients that have elevated endogenous erythropoietin levels are unlikely to respond. In two controlled Phase 3 clinical trials evaluating darbepoetin alfa (Aranesp) and epoetin alpha (Epogen/Procrit) for the treatment of MDS-associated anemia, 15% to 31% of patients responded, respectively.
While ESAs have been shown to alleviate anemia in a subset of patients with MDS, patients that have elevated endogenous erythropoietin levels are unlikely to respond. In two controlled Phase 3 clinical trials evaluating darbepoetin alfa (Aranesp) and epoetin alpha (Epogen/Procrit) for the treatment of MDS-associated anemia, 15% to 31% of 11 patients responded, respectively.
In August 29 2023, Bristol-Myers Squibb Company announced that the FDA approved Reblozyl for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk MDS who may require regular red blood cell transfusions.
In August 2023, Bristol-Myers Squibb Company announced that the FDA approved Reblozyl for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk MDS who may require regular red blood cell transfusions.
Medicines used in clinical trials, including ATMPs, must 38 be manufactured in accordance with the guidelines on cGMP and in a GMP licensed facility, which can be subject to GMP inspections. EU Review and Approval Process In the EU, medicinal products can only be commercialized after a related marketing authorization, or MA, has been granted.
Medicines used in clinical trials, including ATMPs, must be manufactured in accordance with the guidelines on cGMP and in a GMP licensed facility, which can be subject to GMP inspections. EU Review and Approval Process In the EU, medicinal products can only be commercialized after a related marketing authorization, or MA, has been granted.
Additionally, bone marrow analysis performed 24 hours post-dose demonstrated that administration of RKER-050 increased the megakaryocyte precursor population, and that these 21 cells had increased ploidy, compared to vehicle. These data suggest that RKER-050 promoted the maturation of early megakaryocyte populations and primed megakaryocytes for proplatelet production. • Reduced accumulation of progenitor cells.
Additionally, bone marrow analysis performed 24 hours post-dose demonstrated that administration of RKER-050 increased the megakaryocyte precursor population, and that these cells had increased ploidy, compared to vehicle. These data suggest that RKER-050 promoted the maturation of early megakaryocyte populations and primed megakaryocytes for proplatelet production. • Reduced accumulation of progenitor cells.
Any patents issuing from these applications will have expiration dates between November 9, 2037 and February 27, 2045, absent any patent term adjustments or extensions. Other We plan to seek United States and international patent protection for a variety of additional technologies.
Any patents issuing from these applications will have expiration dates between November 9, 2037 and February 27, 2045, absent any patent term adjustments or extensions. 19 Other We plan to seek United States and international patent protection for a variety of additional technologies.
Physicians may prescribe legally available products for uses that are not described in the product’s labeling and that differ from those tested by us and approved by the FDA. Such off-label uses are common across medical specialties. Physicians may believe that such off-label uses are the best treatment for many patients in varied circumstances.
Physicians may prescribe legally available products for uses that are not described in the product’s labeling and that differ from those tested by us and approved by the FDA. Such off-label uses are common across medical specialties. Physicians may believe that such off-label uses are the best treatment for many patients 24 in varied circumstances.
See the section titled “Business—Collaborations and License Agreement—2024 License Agreement with Takeda Pharmaceuticals U.S.A., Inc.” set forth in Part I, Item 1 of this Annual Report on Form 10-K for additional information regarding our license agreement with Takeda.
See the section titled “Business—Collaborations and License Agreement—2024 License Agreement with Takeda 14 Pharmaceuticals U.S.A., Inc.” set forth in Part I, Item 1 of this Annual Report on Form 10-K for additional information regarding our license agreement with Takeda.
In a third-party Phase 3 clinical trial of Jakafi and a third-party Phase 3 clinical trial of Inrebic, treatment led to significant reductions in spleen volume and improvement in total symptom scores. However, JAK inhibitors interfere with normal hematopoiesis and treatment with Jakafi and Inrebic also resulted in clinically significant anemia and thrombocytopenia in these Phase 3 trials.
In a third-party Phase 3 clinical trial of Jakafi and a third-party Phase 3 clinical trial of Inrebic, treatment led to significant reductions in spleen volume 12 and improvement in total symptom scores. However, JAK inhibitors interfere with normal hematopoiesis and treatment with Jakafi and Inrebic also resulted in clinically significant anemia and thrombocytopenia in these Phase 3 trials.
Such decision could include either issue a refusal to file letter or acceptance of the NDA or BLA for filing, indicating that it is sufficiently complete to permit substantive review. Once an NDA or BLA has been accepted for filing, the FDA begins an in-depth review of the NDA or BLA.
Such decision could include either issue a refusal to file letter or acceptance of the NDA or BLA for filing, indicating that it is sufficiently complete to permit substantive review. 22 Once an NDA or BLA has been accepted for filing, the FDA begins an in-depth review of the NDA or BLA.
These cells progress through a series of intermediate stages before becoming a mature cell with a specialized function. At any given time, pools of each progenitor cell are maintained and primed to rapidly respond to a 16 reduction of red blood cells, white blood cells and platelets.
These cells progress through a series of intermediate stages before becoming a mature cell with a specialized function. At any given time, pools of each progenitor cell are maintained and primed to rapidly respond to a reduction of red blood cells, white blood cells and platelets.
We believe that we are well positioned to advance our product candidates and pursue the commercial opportunities in diseases where muscle and bone loss result in a debilitating impact on survival and quality of life, if our product candidates are successfully developed and approved.
We believe that we are well positioned to advance our product candidates and pursue commercial opportunities in diseases where muscle and bone loss result in a debilitating impact on survival and quality of life, if our product candidates are successfully developed and approved.
Either party may terminate the Agreement in its entirety (i) if the other party materially breaches the Takeda Agreement and fails to cure such breach; or (ii) upon the bankruptcy of the other party. 30 2021 License Agreement with Hansoh (Shanghai) Healthtech Co., Ltd.
Either party may terminate the Takeda Agreement in its entirety (i) if the other party materially breaches the Takeda Agreement and fails to cure such breach; or (ii) upon the bankruptcy of the other party. 2021 License Agreement with Hansoh (Shanghai) Healthtech Co., Ltd.
Orphan drug exclusivity does not prevent the FDA from approving a different drug or biologic for the same disease or condition, or the 36 same drug or biologic for a different disease or condition. Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the application fee.
Orphan drug exclusivity does not prevent the FDA from approving a different drug or biologic for the same disease or condition, or the same drug or biologic for a different disease or condition. Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the application fee.
We made payments of $50,000 and $300,000 in 2020 and 2021, respectively, for the achievement of the clinical and regulatory milestones of (i) filing of an IND in the first country and (ii) the completion of a Phase 1 clinical trial, respectively.
We made payments of $50,000 and $300,000 in 2020 and 2021, respectively, for the achievement of the clinical and regulatory milestones of (i) filing of an IND in the first 18 country and (ii) the completion of a Phase 1 clinical trial, respectively.
In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost-containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products.
In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost-containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products. For example, the U.S.
In both standard and priority reviews, the FDA does not always meet its PDUFA goal dates, and the review process is 35 often significantly extended by FDA requests for additional information or clarification.
In both standard and priority reviews, the FDA does not always meet its PDUFA goal dates, and the review process is often significantly extended by FDA requests for additional information or clarification.
Data and Market Exclusivity in the EU The EU provides opportunities for data and market exclusivity related to MAs. Upon receiving an MA, innovative medicinal products are generally entitled to receive eight years of data exclusivity and 10 years of market exclusivity.
Data and Market Exclusivity in the EU 27 The EU provides opportunities for data and market exclusivity related to MAs. Upon receiving an MA, innovative medicinal products are generally entitled to receive eight years of data exclusivity and 10 years of market exclusivity.
Lean Mass (Left) and Grip Strength (Right) *P value ≤0.05; **P value 14 Bone Volume Fraction (BV/TV; Left) and Polar Mass Moment of Inertia (MMI; Right) **P value Treatment with RKER-065 increased satellite cells in skeletal muscle In young boys with DMD, muscle undergoes continuous rounds of degeneration and regeneration, but eventually the ability of the muscle to regenerate declines due to a decline in muscle progenitor cells known as satellite cells.
Lean Mass (Left) and Grip Strength (Right) *P value ≤0.05; **P value 7 Bone Volume Fraction (BV/TV; Left) and Polar Mass Moment of Inertia (MMI; Right) **P value Treatment with RKER-065 increased satellite cells in skeletal muscle In young boys with DMD, muscle undergoes continuous rounds of degeneration and regeneration, but eventually the ability of the muscle to regenerate declines due to a decline in muscle progenitor cells known as satellite cells.
The market exclusivity period prevents a successful generic or biosimilar applicant from commercializing its product in the EU until 10 years have elapsed from the 40 initial MA of the reference product in the EU.
The market exclusivity period prevents a successful generic or biosimilar applicant from commercializing its product in the EU until 10 years have elapsed from the initial MA of the reference product in the EU.
Some EU Member States may approve a specific price for a product, or they may instead adopt a system of direct or indirect controls on the profitability of the company placing the product on the market.
Some EU Member States may approve a specific price for a product, or they may instead adopt a system of direct or indirect controls on the profitability of the 30 company placing the product on the market.
The SEC maintains an internet site 45 that contains reports, proxy and information statements and other information. The address of the SEC’s website is www.sec.gov.
The SEC maintains an internet site that contains reports, proxy and information statements and other information. The address of the SEC’s website is www.sec.gov.
We seek to address the limitations of current therapeutic approaches to treating diseases whose manifestations are linked to dysfunction of TGF-ß signaling pathways by: ▪ leveraging our comprehensive insights into the TGF-ß signaling pathways to discover therapeutics to treat disorders that are linked to dysfunctional TGF-ß signaling; 1 ▪ expanding our library of proprietary molecules that are engineered to induce desired biological effects, such as increased muscle mass and strength, improved muscle quality and reduced intramuscular fat, improved bone mineral density and modulated blood cell production; ▪ engineering proprietary molecules to selectively target specific proteins in the TGF-ß signaling pathways to provide therapeutic benefit while potentially minimizing safety risks; ▪ developing product candidates for the treatment of diseases where targeting the TGF-ß signaling pathways has clinical validation or biological rationale to improve our probability of success in the clinic; and ▪ targeting the TGF-ß family of proteins, which are highly conserved throughout evolution, permitting the use of animal models to potentially predict with high confidence the therapeutic benefit in patients.
We seek to address the limitations of current therapeutic approaches to treating diseases whose manifestations are linked to dysfunction of TGF-ß signaling pathways by: • leveraging our comprehensive insights into the TGF-ß signaling pathways to discover therapeutics to treat disorders that are linked to dysfunctional TGF-ß signaling; • expanding our library of proprietary molecules that are engineered to induce desired biological effects, such as increased muscle mass and strength, increased muscle regeneration, improved muscle quality and reduced tissue fat, improved bone mineral density, reduced inflammation, reduced fibrosis and modulated blood cell production; • engineering proprietary molecules to selectively target specific proteins in the TGF-ß signaling pathways to provide therapeutic benefit while potentially minimizing safety risks; • developing product candidates for the treatment of diseases where targeting the TGF-ß signaling pathways has clinical validation or biological rationale to improve our probability of success in the clinic; and • targeting the TGF-ß family of proteins, which are highly conserved throughout evolution, permitting the use of animal models to potentially predict with high confidence the therapeutic benefit in patients.
Evoked Force Maximum Gastrocnemius ***P value Co-treatment with prednisolone and RKER-065 increased both muscle mass and strength and trabecular bone and strength MDX mice were treated with vehicle or 2-prednisolone, or co-treated with 10 mg/kg of prednisolone and RKER-065 twice weekly.
Evoked Force Maximum Gastrocnemius ***P value Co-treatment with prednisolone and RKER-065 increased both muscle mass and strength and trabecular bone and strength MDX mice were treated with vehicle (Veh) or 2-prednisolone (Pred), or co-treated with 10 mg/kg of prednisolone and RKER-065 twice weekly (Pred+065).
Satellite Cell Population ****P value Co-treatment with PMO therapy and RKER-065 improved efficiency of exon skipping 15 MDX mice were treated with vehicle, 25 mg/kg of PMO therapy, or co-treated with 10 mg/kg of RKER-065 twice weekly and 25 mg/kg of PMO therapy once weekly.
Satellite Cell Population ****P value Co-treatment with PMO therapy and RKER-065 improved efficiency of exon skipping 8 MDX mice were treated with vehicle, 25 mg/kg of PMO therapy, or co-treated with 10 mg/kg of RKER-065 twice weekly and 25 mg/kg of PMO therapy once weekly.
(which was acquired by Swedish Orphan Biovitrum AB in June 2023) received FDA accelerated approval of its product, pacritinib (Vonjo), for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 10 9 /L.
(which was acquired by Swedish Orphan Biovitrum AB in June 2023) received FDA accelerated approval of its product, pacritinib (Vonjo), for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.
To encourage our employees to think like owners and share in the Company’s success, all employees are granted stock options. All employees are eligible for health insurance, paid and unpaid leaves including paid parental leave, retirement plans with an employer contribution match and life and disability/accident coverage.
To encourage our employees to think like owners and share in the Company’s success, all employees are granted stock options and/or restricted stock units. All employees are eligible for health insurance, paid and unpaid leaves including paid parental leave, retirement plans with an employer contribution match and life and disability/accident coverage.
Decreased mobility along with the use of glucocorticoids are associated with increased risk of obesity and the associated negative health consequences, including type 2 diabetes and cardiovascular disease, in DMD patients.
Decreased mobility, along with the use of glucocorticoids, is associated with increased risk of obesity and the associated negative health consequences, including type 2 diabetes and cardiovascular disease, in DMD patients.
Our discovery approach has built on these initial observations to generate product candidates designed to target ActRII receptors without certain downsides observed in third-party preclinical studies and clinical trials of ActRIIA-Fc and ActRIIB-Fc.
Our discovery approach has built on these initial observations to generate product candidates designed to target ActRII receptors that address certain downsides observed in third-party preclinical studies and clinical trials of ActRIIA-Fc and ActRIIB-Fc.
These new cells are derived from stem cells that have the ability to differentiate into cells with specialized function when appropriate signals are provided to maintain the homeostatic state of the tissue. Members of the TGF-ß family of proteins, including activins and BMPs, provide the necessary signals for this process of self-renewal and repair.
These new cells are derived from stem cells that have the ability to differentiate into cells with specialized functions when appropriate signals are provided to maintain the homeostatic state of the tissue. Members of the TGF-ß family of proteins, including activins and bone morphogenic proteins (BMPs), provide the necessary signals for this process of self-renewal and repair.
In March 2024, Italfarmaco S.p.A. announced that the FDA approved Duvyzat (givinostat), a histone deacetylase inhibitor for the treatment of DMD in patients aged six years and older. In addition, several companies are developing gene therapies to treat DMD, including Pfizer Inc., Audentes Therapeutics, Inc. and Solid Biosciences Inc.
In March 2024, Italfarmaco S.p.A. announced that the FDA approved Duvyzat (givinostat), a histone deacetylase inhibitor for the treatment of DMD in patients aged six years and older. In addition, several companies are developing gene therapies to treat DMD, including REGENXBIO Inc. and Solid Biosciences Inc.
FibroGen Inc. and Astellas Pharma Inc. are developing product candidates for the treatment of anemia, and Merck, Bristol-Myers Squibb Company and Disc Medicine are developing product candidates targeting diseases associated with MDS and myelofibrosis, including chronic anemia.
(formerly FibroGen Inc.) and Astellas Pharma Inc. are developing product candidates for the treatment of anemia, and Merck & Co. Inc., or Merck, Bristol-Myers Squibb Company and Disc Medicine are developing product candidates targeting diseases associated with MDS and myelofibrosis, including chronic anemia.
Exploratory endpoints include assessments of the pharmacodynamic effect on bone, adipose, muscle, cardiac tissue and fibrosis. To aid in the assessment of adipose tissue, volunteers will be required to have a BMI ≥ 27 to ≤ 35 kg/m 2 to be enrolled in Part 2 of this trial.
Exploratory endpoints included assessments of the pharmacodynamic effect on bone, adipose, muscle, cardiac tissue and fibrosis. To aid in the assessment of adipose tissue, volunteers were required to have a BMI ≥ 27 to ≤ 35 kg/m 2 to be enrolled in Part 2 of this trial.
Of these full-time employees, 135 employees are engaged in research and development and 34 employees are engaged in management or general and administrative activities. All of our employees are based in the United States. None of our employees are subject to a collective bargaining agreement or represented by a trade or labor union.
Of these full-time employees, 59 employees are engaged in research and development and 19 employees are engaged in management or general and administrative activities. All of our employees are based in the United States. None of our employees are subject to a collective bargaining agreement or represented by a trade or labor union.
Our second product candidate, KER-065, is being developed for the treatment of neuromuscular diseases. Our most advanced product candidate, elritercept (KER-050), is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndromes, or MDS, and in patients with myelofibrosis.
Our most advanced product candidate, elritercept (KER-050), is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndromes, or MDS, and in patients with myelofibrosis.
Innovative products that target an unmet medical need and are expected to be of major public health interest may be eligible for a number of expedited development and review programs, such as the Priority Medicines, or PRIME, scheme, which provides incentives similar to the breakthrough therapy designation in the U.S.
Accelerated and Alternative Marketing Authorization Mechanisms 26 Innovative products that target an unmet medical need and are expected to be of major public health interest may be eligible for a number of expedited development and review programs, such as the Priority Medicines, or PRIME, scheme, which provides incentives similar to the breakthrough therapy designation in the U.S.
KER-065 is designed to bind to and inhibit TGF-ß ligands, including myostatin (GDF8) and activin A, which are negative regulators of muscle and bone mass and strength.
Rinvatercept is designed to bind to and inhibit TGF-ß ligands, including myostatin (GDF8) and activin A, which are negative regulators of muscle and bone mass and strength.
We own 15 pending U.S. patent applications and 27 pending ex-U.S. applications that contain claims or supporting disclosure directed to ligand traps comprised of a modified ligand-binding domain derived from ActRIIA and ActRIIB and use thereof to treat muscle disease, bone disease, anemia, fibrosis, pulmonary hypertension, metabolic disease, thrombocytopenia, and neutropenia, among others.
We own one issued U.S. patent, 11 pending U.S. patent applications, and 26 pending ex-U.S. applications that contain claims or supporting disclosure directed to ligand traps comprised of a modified ligand-binding domain derived from ActRIIA and ActRIIB and use thereof to treat muscle disease, bone disease, anemia, fibrosis, pulmonary hypertension, metabolic disease, thrombocytopenia, and neutropenia, among others.
The scope of the FCPA includes interactions with certain healthcare professionals in many countries. Equivalent laws have been adopted in other foreign countries that impose similar obligations. Employees and Human Capital Resources As of December 31, 2024, we had 169 full-time employees, including 52 who hold Ph.D. or M.D. degrees.
The scope of the FCPA includes interactions with certain healthcare professionals in many countries. Equivalent laws have been adopted in other foreign countries that impose similar obligations. Employees and Human Capital Resources As of December 31, 2025, we had 78 full-time employees, including 27 who hold Ph.D. or M.D. degrees.
With a focus on developing differentiated product candidates, we aim to target the TGF-ß pathways critical for the growth, repair and maintenance of a number of tissue and organ systems. The key elements of our strategy include: ▪ Rapidly advance the clinical development of cibotercept, KER-065 and elritercept.
With a focus on developing differentiated product candidates, we aim to target the TGF-ß pathways critical for the growth, repair and maintenance of a number of tissue and organ systems. The key elements of our strategy include: • Rapidly advance the clinical development of rinvatercept .
We compete in the segments of the biotechnology, pharmaceutical and other related industries that develop and market therapies in our target indications. There are many other companies, including large biotechnology and pharmaceutical companies, that have commercialized and/or are developing therapies for the same therapeutic areas that our product candidates target. For example, in March 2024, Merck & Co.
We compete in the segments of the biotechnology, pharmaceutical and other related industries that develop and market therapies in our target indications. There are many other companies, including large biotechnology and pharmaceutical companies, that have commercialized and/or are developing therapies for the same therapeutic areas that our product candidates target.
Any patents issuing from these applications will have expiration dates between March 19, 2041 and February 27, 2045, absent any patent term adjustments or extensions. Elritercept Elritercept is a modified ActRIIA ligand trap that is designed to bind to different TGF-ß ligands that signal through a TGF-ß signaling pathway.
Any patents issuing from these applications will have expiration dates between March 19, 2041 and May 8, 2046, absent any patent term adjustments or extensions. Elritercept Elritercept is a modified ActRIIA ligand trap that is designed to bind to different TGF-ß ligands that signal through a TGF-ß signaling pathway.
We are led by a highly experienced management team and scientific advisory board who have significant experience and expertise researching and developing therapeutics in the TGF-ß family of proteins. Our team has collectively worked on marketed therapeutics such as Reblozyl, Takhzyro and Winrevair, and led drug discovery and clinical development at companies including Acceleron Pharma Inc.
We are led by a highly experienced management team and scientific advisory board who have significant experience and expertise researching and developing therapeutics in targeting the TGF-ß family of proteins. Our team has collectively worked on marketed therapeutics such as Reblozyl and Winrevair, and led drug discovery at companies including Acceleron Pharma Inc. (which was acquired by Merck & Co.
Treatment with RKER-065 increased utrophin expression and muscle strength in a mouse model of DMD 13 In preclinical studies conducted in the MDX mouse model of DMD, twice weekly, intraperitoneal 10 mg/kg dosing of RKER-065 increased expression of utrophin in muscle fibers, potentially contributing to the observed increased strength.
Treatment with RKER-065 increased utrophin expression and muscle strength in a mouse model of DMD 6 In preclinical studies conducted in the X-linked muscular dystrophy, or MDX, mouse model of DMD, twice weekly, intraperitoneal 10 mg/kg dosing of RKER-065 increased expression of utrophin in muscle fibers, potentially contributing to the observed increased strength.
We own four issued U.S. patents, 11 issued ex-U.S. patents, 13 pending U.S. patent applications and 59 pending ex-U.S. applications that contain claims or supporting disclosure directed to ActRIIA ligand traps and use thereof to treat muscle disease, bone disease, metabolic disease, anemia, fibrosis, pulmonary hypertension, thrombocytopenia, and neutropenia, among others.
We own six issued U.S. patents, 21 issued ex-U.S. patents, 10 pending U.S. patent applications and 58 pending ex-U.S. applications that contain claims or supporting disclosure directed to ActRIIA ligand traps and use thereof to treat muscle disease, bone disease, metabolic disease, anemia, fibrosis, pulmonary hypertension, thrombocytopenia, and neutropenia, among others.
In addition, Hansoh will use commercially reasonable efforts to develop, obtain regulatory approval for, and commercialize licensed products in any region in the Hansoh Territory. Pursuant to the terms of the Hansoh Agreement, we received an upfront payment in 2022.
In addition, Hansoh will use commercially reasonable efforts to develop, obtain regulatory approval for, and commercialize licensed products in any region in the Hansoh Territory. Pursuant to the terms of the Hansoh Agreement, we received a net $18.0 million upfront payment in January 2022.
Our Pipeline The following table sets forth our product candidates and their current development stages: Our Strategy Our mission is to deliver significant clinical benefit to patients suffering from disorders that are linked to dysfunctional signaling of the TGF-ß family of proteins .
Inc. in November 2021) and Wyeth Pharmaceuticals Inc. 1 Our Pipeline The following table sets forth our product candidates and their current development stages: [NTD: Our Strategy Our mission is to deliver significant clinical benefit to patients suffering from disorders that are linked to dysfunctional signaling of the TGF-ß family of proteins .
These and other laws govern the use, handling and disposal of various biologic, chemical and radioactive substances used in, and wastes generated by, operations. If our operations result in contamination of the environment or expose individuals to hazardous substances, we could be liable for damages and governmental fines.
These and other laws govern the use, handling and disposal of various biologic, chemical and radioactive substances used in, and wastes generated by, operations. If our operations result in contamination of the environment or expose individuals to hazardous substances, we could be liable for damages and governmental fines. Equivalent laws have been adopted in other countries that impose similar obligations.
We own two issued U.S. patents, four issued ex-U.S. patents, 12 pending U.S. patent applications and 22 pending ex-U.S. applications that contain claims or supporting disclosure directed to GDNF fusion polypeptides, ALK2 antibodies, crystal forms of an ALK2 inhibitor, ActRII chimera ligand traps, and uses of small molecule ALK2 inhibitors.
We own nine issued U.S. patents, 18 issued ex-U.S. patents, 21 pending U.S. patent applications, and 46 pending ex-U.S. applications that contain claims or supporting disclosure directed to cibotercept, GDNF fusion polypeptides, ALK2 antibodies, crystal forms of an ALK2 inhibitor, ActRII chimera ligand traps, and uses of small molecule ALK2 inhibitors.
The FDA does, however, restrict manufacturer’s communications on the subject of off-label use of their products. 37 Biosimilars and Exclusivity The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010, or collectively the ACA, includes a subtitle called the Biologics Price Competition and Innovation Act of 2009, or BPCI Act, which created an abbreviated approval pathway for biological products shown to be similar to, or interchangeable with, an FDA-licensed reference biological product.
Biosimilars and Exclusivity The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010, or collectively the ACA, includes a subtitle called the Biologics Price Competition and Innovation Act of 2009, or BPCI Act, which created an abbreviated approval pathway for biological products shown to be similar to, or interchangeable with, an FDA-licensed reference biological product.
Specifically, in preclinical studies: • KER-065 showed high affinity for and potent inhibition of ligands involved in the regulation of muscle and bone homeostasis; • RKER-065 increased utrophin expression and muscle strength in a mouse model of DMD; • Co-treatment with prednisolone and RKER-065 increased both muscle mass and strength and trabecular bone and strength; • RKER-065 increased satellite cells in skeletal muscle; and • Co-treatment with phosphorodiamidate morpholino oligomer, or PMO, therapy and RKER-065 improved grip strength and the efficiency of exon skipping.
Specifically, in preclinical studies: • Rinvatercept showed high affinity for and potent inhibition of ligands involved in the regulation of muscle and bone homeostasis; • RKER-065, the research form of rinvatercept, increased utrophin expression and muscle strength in a mouse model of DMD; • Co-treatment with prednisolone and RKER-065 increased both muscle mass and strength and trabecular bone and strength; • Co-treatment with an HDAC inhibitor and RKER-065 increased muscle mass and strength and reduced fibrosis; • RKER-065 increased satellite cells in skeletal muscle; • Co-treatment with phosphorodiamidate morpholino oligomer, or PMO, therapy and RKER-065 improved grip strength and the efficiency of exon skipping; and • RKER-065 treatment reduced muscle wasting and preserved muscle function in a mouse model of ALS.
Intellectual property rights may not address all potential threats to our competitive advantage As of February 21, 2025, our patent portfolio consisted of 14 issued U.S. patents, 33 pending U.S. patent applications, 23 issued ex-U.S. patents and 107 pending ex-U.S. applications, with expected expiry dates not earlier than between March 13, 2029 and January 9, 2046.
Intellectual property rights may not address all potential threats to our competitive advantage As of February 27, 2026, our patent portfolio consisted of 23 issued U.S. patents, 27 pending U.S. patent applications, 45 issued ex-U.S. patents and 102 pending ex-U.S. applications, with expected expiry dates not earlier than between March 13, 2029 and October 29, 2046.
Of these, 18 issued patents and 104 patent applications relate to cibotercept, KER-065 and elritercept, and 19 issued patents and 36 patent applications relate to other technologies, in each case as described in more detail below. Each of our pending international patent applications has been filed under the Patent Cooperation Treaty and has not yet entered any national jurisdictions.
Of these, 28 issued patents and 83 patent applications relate to rinvatercept and elritercept, and 40 issued patents and 46 patent applications relate to other technologies, in each case as described in more detail below. Each of our pending international patent applications has been filed under the Patent Cooperation Treaty and has not yet entered any national jurisdictions.
These reductions went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2032, unless additional action is taken by Congress.
These reductions went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2032, unless additional action is taken by Congress In addition, Congress is considering additional health reform measures as part of the other reform measures.
Ongoing Phase 1 Clinical Trial in Healthy Volunteers We have initiated a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of KER-065 in healthy volunteers. The primary objectives of this trial are to assess safety, tolerability and pharmacokinetics of KER-065.
Completed Phase 1 Clinical Trial in Healthy Adult Volunteers We completed a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of rinvatercept in healthy adult male volunteers. The primary objectives of this trial were to assess safety, tolerability and pharmacokinetics of rinvatercept.
Elritercept: For the Treatment of Ineffective Hematopoiesis to Address Cytopenias We are developing elritercept, our lead product candidate, for the treatment of cytopenias that occur due to ineffective hematopoiesis, including anemia and thrombocytopenia, in patients with MDS and in patients with myelofibrosis.
Elritercept: For the Treatment of Ineffective Hematopoiesis to Address Cytopenias Elritercept, our most advanced product candidate, is being developed for the treatment of cytopenias that occur due to ineffective hematopoiesis, including anemia and thrombocytopenia, in patients with MDS and in patients with myelofibrosis.
Our deep knowledge and expertise of the TGF-ß family of proteins provides a streamlined approach to screen and develop novel product candidates for hematological, pulmonary and cardiovascular disorders. 28 Manufacturing We rely, and expect to continue to rely for the foreseeable future, on third-party contract manufacturing organizations, or CMOs, to produce our product candidates for preclinical and clinical testing, as well as for commercial manufacture if our product candidates receive marketing approval.
Our deep knowledge and expertise of the TGF-ß family of proteins provides a streamlined approach to screen and develop novel product candidates for a broad range of indications, including neuromuscular and neurodegenerative disorders, rare bone and fibrosis diseases and obesity/frailty indication. 15 Manufacturing We rely, and expect to continue to rely for the foreseeable future, on third-party contract manufacturing organizations, or CMOs, to produce our product candidates for preclinical and clinical testing, as well as for commercial manufacture if our product candidates receive marketing approval.
Additional Regulation In addition to the foregoing, state and federal laws regarding environmental protection and hazardous substances, including the Occupational Safety and Health Act, the Resource Conservation and Recovery Act and the Toxic Substances Control Act, affect our business.
We expect that additional state, federal, and foreign healthcare reform measures will be adopted in the future. Additional Regulation In addition to the foregoing, state and federal laws regarding environmental protection and hazardous substances, including the Occupational Safety and Health Act, the Resource Conservation and Recovery Act and the Toxic Substances Control Act, affect our business.
Equivalent laws have been adopted in other countries that impose similar obligations. 44 U.S. Foreign Corrupt Practices Act The U.S. Foreign Corrupt Practices Act, or FCPA, prohibits U.S. corporations and individuals from engaging in certain activities to obtain or retain business abroad or to influence a person working in an official capacity.
U.S. Foreign Corrupt Practices Act The U.S. Foreign Corrupt Practices Act, or FCPA, prohibits U.S. corporations and individuals from engaging in certain activities to obtain or retain business abroad or to influence a person working in an official capacity.
Through inhibition of these TGF-ß ligands, we believe that KER-065 has the potential to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength. We are developing KER-065 for the treatment of neuromuscular disorders, with an initial focus on Duchenne muscular dystrophy, or DMD.
Through inhibition of these TGF-ß ligands, we believe that rinvatercept has the potential to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength. We are developing rinvatercept for the treatment of Duchenne muscular dystrophy, or DMD and for the treatment of amyotrophic lateral sclerosis, or ALS.
In a third-party clinical trial of ActRIIA-Fc, increased bone mineral density and red blood cell production was reported in healthy post-menopausal women.
Specifically: ▪ Wild-type ActRIIA-Fc was associated with increased bone growth and red blood cell production in rodents and non-human primates. In a third-party clinical trial of ActRIIA-Fc, increased bone mineral density and red blood cell production was reported in healthy post-menopausal women.
We do not currently have arrangements in place for redundant supply. We could be unable to find alternative suppliers of acceptable quality, in the appropriate volumes and at an acceptable cost, if needed.
We obtain our supplies from these CMOs on a purchase order basis and do not have any long-term supply arrangements in place. We do not currently have arrangements in place for redundant supply. We could be unable to find alternative suppliers of acceptable quality, in the appropriate volumes and at an acceptable cost, if needed.
In the United States, there are 60,000 to 170,000 patients with MDS and 15,000 to 20,000 new cases of MDS reported each year. MDS predominantly affects older adults, with approximately 75% of patients aged 60 years or older at diagnosis. Median survival ranges from approximately nine years for very low-risk patients to less than a year for high-risk patients.
MDS predominantly affects older adults, with approximately 75% of patients aged 60 years or older at diagnosis. Median survival ranges from approximately nine years for very low-risk patients to less than a year for high-risk patients.
In many hematological disorders, there is abnormal proliferation and differentiation of the progenitor cells for red blood cells, platelets and neutrophils. This failure to produce fully mature cells is termed ineffective hematopoiesis, and may be due to single or multiple defects that can lead to a hyperproliferation or a shortage of progenitor cells.
This failure to produce fully mature cells is termed ineffective hematopoiesis, and may be due to single or multiple defects that can lead to hyperproliferation or a shortage of progenitor cells.
Pursuant to the terms of the Takeda Agreement, we received a $200.0 million upfront payment in February 2025. In addition to the upfront payment, we are entitled to receive up to an aggregate of (i) $370.0 million upon the achievement of specified development and commercial milestones and (ii) $740.0 million upon the achievement of specified sales milestones.
In addition to the upfront payment and milestone payment, we are entitled to receive up to an aggregate of (i) $80.0 million upon the achievement of specified development milestones, (ii) $280.0 million upon the achievement of specified commercial milestone and (iii) $740.0 million upon the achievement of specified sales milestones.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeHowever, if these patents were asserted against us and our defenses to such an action were unsuccessful, unless we obtain a license to these patents, which may not be available on commercially reasonable terms, or at all, we could be liable for damages and precluded from commercializing cibotercept or KER-065, as applicable, in certain indications, which could have a material adverse effect on our business, financial condition, cash flows or results of operations. 70 If a third-party claims that we infringe its intellectual property rights, we may face a number of issues, including, but not limited to: ▪ infringement and other intellectual property claims which, regardless of merit, may be expensive and time-consuming to litigate and may divert our management’s attention from our core business; ▪ substantial damages for infringement, which we may have to pay if a court decides that the product candidate or technology at issue infringes on or violates the third party’s rights, and, if the court finds that the infringement was willful, we could be ordered to pay treble damages and the patent owner’s attorneys’ fees; ▪ a court prohibiting us from developing, manufacturing, marketing or selling our product candidates, or from using our proprietary technologies, unless the third party licenses its product rights to us, which it is not required to do; ▪ if a license is available from a third party, we may have to pay substantial royalties, upfront fees and other amounts, and/or grant cross-licenses to intellectual property rights for our products, if any; and ▪ redesigning our product candidates or processes so they do not infringe, which may not be possible or may require substantial monetary expenditures and time.
Biggest changeIf a third-party claims that we infringe its intellectual property rights, we may face a number of issues, including, but not limited to: ▪ infringement and other intellectual property claims which, regardless of merit, may be expensive and time-consuming to litigate and may divert our management’s attention from our core business; ▪ substantial damages for infringement, which we may have to pay if a court decides that the product candidate or technology at issue infringes on or violates the third party’s rights, and, if the court finds that the infringement was willful, we could be ordered to pay treble damages and the patent owner’s attorneys’ fees; ▪ a court prohibiting us from developing, manufacturing, marketing or selling our product candidates, or from using our proprietary technologies, unless the third party licenses its product rights to us, which it is not required to do; ▪ if a license is available from a third party, we may have to pay substantial royalties, upfront fees and other amounts, and/or grant cross-licenses to intellectual property rights for our products, if any; and ▪ redesigning our product candidates or processes so they do not infringe, which may not be possible or may require substantial monetary expenditures and time.
For example, under GDPR, companies may face temporary or definitive bans on data processing and other corrective actions; fines of up to €20 million under the EU GDPR, 17.5 million pounds sterling under the UK GDPR, or in each case,4% of annual global revenue, whichever is greater; or private litigation related to processing of personal data brought by classes of data subjects or consumer protection organizations authorized at law to represent their interests.
For example, under the GDPR, companies may face temporary or definitive bans on data processing and other corrective actions; fines of up to €20 million under the EU GDPR, 17.5 million pounds sterling under the UK GDPR, or in each case,4% of annual global revenue, whichever is greater; or private litigation related to processing of personal data brought by classes of data subjects or consumer protection organizations authorized at law to represent their interests.
This could limit the amount of NOLs or R&D credit carryforwards that we can utilize annually to offset future taxable income or tax liabilities. Subsequent ownership changes and changes to the U.S. tax rules in respect of the utilization of NOLs and R&D credits carried forward may further affect the limitation in future years.
This could limit the amount of NOLs or R&D tax credit carryforwards that we can utilize annually to offset future taxable income or tax liabilities. Subsequent ownership changes and changes to the U.S. tax rules in respect of the utilization of NOLs and R&D tax credits carried forward may further affect the limitation in future years.
For example: ▪ others may be able to make or use compounds or cells that are similar to the biological compositions of our product candidates but that are not covered by the claims of our patents; ▪ the active biological ingredients in our current product candidates will eventually become commercially available in biosimilar drug products, and no patent protection may be available with regard to formulation or method of use; ▪ we or our licensors, as the case may be, may fail to meet our obligations to the U.S. government in regards to any in-licensed patents and patent applications funded by U.S. government grants, leading to the loss of patent rights; ▪ we or our licensors, as the case may be, might not have been the first to file patent applications for these inventions; ▪ others may independently develop similar or alternative technologies or duplicate any of our technologies; ▪ it is possible that our pending patent applications will not result in issued patents; ▪ it is possible that there are prior public disclosures that could invalidate our or our licensors’ patents, as the case may be, or parts of our or their patents; 68 ▪ it is possible that others may circumvent our owned or in-licensed patents; ▪ it is possible that there are unpublished applications or patent applications maintained in secrecy that may later issue with claims covering our products, if any, or technology similar to ours; ▪ the laws of foreign countries may not protect our or our licensors’, as the case may be, proprietary rights to the same extent as the laws of the United States; ▪ the claims of our owned or in-licensed issued patents or patent applications, if and when issued, may not cover our product candidates; ▪ our owned or in-licensed issued patents may not provide us with any competitive advantages, may be narrowed in scope, or be held invalid or unenforceable as a result of legal challenges by third parties; ▪ the inventors of our owned or in-licensed patents or patent applications may become involved with competitors, develop products or processes which design around our patents, or become hostile to us or the patents or patent applications on which they are named as inventors; ▪ it is possible that our owned or in-licensed patents or patent applications omit individual(s) that should be listed as inventor(s) or include individual(s) that should not be listed as inventor(s), which may cause these patents or patents issuing from these patent applications to be held invalid or unenforceable; ▪ we have engaged in scientific collaborations in the past, and will continue to do so in the future.
For example: ▪ others may be able to make or use compounds or cells that are similar to the biological compositions of our product candidates but that are not covered by the claims of our patents; ▪ the active biological ingredients in our current product candidates will eventually become commercially available in biosimilar drug products, and no patent protection may be available with regard to formulation or method of use; ▪ we or our licensors, as the case may be, may fail to meet our obligations to the U.S. government in regards to any in-licensed patents and patent applications funded by U.S. government grants, leading to the loss of patent rights; ▪ we or our licensors, as the case may be, might not have been the first to file patent applications for these inventions; ▪ others may independently develop similar or alternative technologies or duplicate any of our technologies; ▪ it is possible that our pending patent applications will not result in issued patents; ▪ it is possible that there are prior public disclosures that could invalidate our or our licensors’ patents, as the case may be, or parts of our or their patents; ▪ it is possible that others may circumvent our owned or in-licensed patents; ▪ it is possible that there are unpublished applications or patent applications maintained in secrecy that may later issue with claims covering our products, if any, or technology similar to ours; ▪ the laws of foreign countries may not protect our or our licensors’, as the case may be, proprietary rights to the same extent as the laws of the United States; ▪ the claims of our owned or in-licensed issued patents or patent applications, if and when issued, may not cover our product candidates; ▪ our owned or in-licensed issued patents may not provide us with any competitive advantages, may be narrowed in scope, or be held invalid or unenforceable as a result of legal challenges by third parties; ▪ the inventors of our owned or in-licensed patents or patent applications may become involved with competitors, develop products or processes which design around our patents, or become hostile to us or the patents or patent applications on which they are named as inventors; ▪ it is possible that our owned or in-licensed patents or patent applications omit individual(s) that should be listed as inventor(s) or include individual(s) that should not be listed as inventor(s), which may cause these patents or patents issuing from these patent applications to be held invalid or unenforceable; ▪ we have engaged in scientific collaborations in the past, and will continue to do so in the future.
Food and Drug Administration, or the FDA, or any comparable foreign regulatory authority agree with the design, endpoints or implementation; ▪ sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; ▪ receiving regulatory approvals or authorizations for conducting our planned clinical trials or future clinical trials; 49 ▪ commencement of and successful patient enrollment in, and completion of, additional clinical trials on a timely basis; ▪ our ability to demonstrate to the satisfaction of the FDA or any comparable foreign regulatory authority that the applicable product candidate is safe and effective as a treatment for our targeted indications or, in the case of an applicable product candidate which is regulated as a biological product, that the applicable product candidate is safe, pure, and potent for our targeted indications; ▪ our ability to demonstrate to the satisfaction of the FDA or any comparable foreign regulatory authority that the applicable product candidate’s risk-benefit ratio for its proposed indication is acceptable; ▪ timely receipt of marketing approvals for our product candidates from applicable regulatory authorities; ▪ the extent of any required post-marketing approval commitments to applicable regulatory authorities; ▪ establishing and scaling up, either alone or with third-party manufacturers, manufacturing capabilities of clinical supply for our clinical trials and commercial manufacturing, if any of our product candidates are approved; ▪ obtaining and maintaining patent and trade secret protection or regulatory exclusivity for our product candidates, both in the United States and internationally; ▪ successfully scaling a sales and marketing organization and launching commercial sales of our product candidates, if approved; ▪ acceptance of our product candidates’ benefits and uses, if approved, by patients, the medical community and third-party payors; ▪ maintaining a continued acceptable safety profile of our product candidates following approval; ▪ effectively competing with companies developing and commercializing other therapies in the indications which our product candidates target; ▪ obtaining and maintaining healthcare coverage and adequate reimbursement from third-party payors; and ▪ enforcing and defending intellectual property rights and claims.
Food and Drug Administration, or the FDA, or any comparable foreign regulatory authority agree with the design, endpoints or implementation; ▪ sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; ▪ receiving regulatory approvals or authorizations for conducting our planned clinical trials or future clinical trials; ▪ commencement of and successful patient enrollment in, and completion of, additional clinical trials on a timely basis; ▪ our ability to demonstrate to the satisfaction of the FDA or any comparable foreign regulatory authority that the applicable product candidate is safe and effective as a treatment for our targeted indications or, in the case of an applicable product candidate which is regulated as a biological product, that the applicable product candidate is safe, pure, and potent for our targeted indications; ▪ our ability to demonstrate to the satisfaction of the FDA or any comparable foreign regulatory authority that the applicable product candidate’s risk-benefit ratio for its proposed indication is acceptable; ▪ timely receipt of marketing approvals for our product candidates from applicable regulatory authorities; ▪ the extent of any required post-marketing approval commitments to applicable regulatory authorities; ▪ establishing and scaling up, either alone or with third-party manufacturers, manufacturing capabilities of clinical supply for our clinical trials and commercial manufacturing, if any of our product candidates are approved; ▪ obtaining and maintaining patent and trade secret protection or regulatory exclusivity for our product candidates, both in the United States and internationally; ▪ successfully scaling a sales and marketing organization and launching commercial sales of our product candidates, if approved; ▪ acceptance of our product candidates’ benefits and uses, if approved, by patients, the medical community and third-party payors; ▪ maintaining a continued acceptable safety profile of our product candidates following approval; ▪ effectively competing with companies developing and commercializing other therapies in the indications which our product candidates target; ▪ obtaining and maintaining healthcare coverage and adequate reimbursement from third-party payors; and ▪ enforcing and defending intellectual property rights and claims.
Our product candidates could fail to receive regulatory approval for many reasons, including the following: ▪ the FDA or comparable foreign regulatory authorities may disagree with the design or implementation of our clinical trials; ▪ we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a product candidate is safe and effective as a treatment for our targeted indications, or, in the case of a product candidate regulated as a biological product, that the product candidate is safe, pure and potent for its proposed indication; ▪ the population studied may not be sufficiently broad or representative to assure safety or efficacy in the population for which we seek approval; ▪ the results of clinical trials may not meet the level of statistical significance required by the FDA or comparable foreign regulatory authorities for approval; ▪ we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks; ▪ the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data from preclinical studies or clinical trials; ▪ the FDA or comparable foreign regulatory authorities may require additional preclinical studies or clinical trials beyond those that we currently anticipate; ▪ the data collected from clinical trials of our product candidates may not be sufficient to support the submission of a New Drug Application, or NDA, or a Biologics License Application, or BLA, as applicable, to the FDA or other submission or to obtain regulatory approval in the United States or elsewhere; ▪ the FDA or comparable foreign regulatory authorities may find deficiencies with or fail to approve the manufacturing processes or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and ▪ the approval policies or regulations of the FDA or any comparable foreign regulatory authorities or the laws they enforce may significantly change in a manner rendering our clinical data insufficient for approval.
Our product candidates could fail to receive regulatory approval for many reasons, including the following: ▪ the FDA or comparable foreign regulatory authorities may disagree with the design or implementation of our clinical trials; ▪ we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a product candidate is safe and effective as a treatment for our targeted indications, or, in the case of a product candidate regulated as a biological product, that the product candidate is safe, pure and potent for its proposed indication; ▪ the population studied may not be sufficiently broad or representative to assure safety or efficacy in the population for which we seek approval; ▪ the results of clinical trials may not meet the level of statistical significance required by the FDA or comparable foreign regulatory authorities for approval; ▪ we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks; ▪ the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data from preclinical studies or clinical trials; ▪ the FDA or comparable foreign regulatory authorities may require additional preclinical studies or clinical trials beyond those that we currently anticipate; ▪ the data collected from clinical trials of our product candidates may not be sufficient to support the submission of a New Drug Application, or NDA, or a Biologics License Application, or BLA, as applicable, to the FDA or other submission or to obtain regulatory approval in the United States or elsewhere; ▪ the FDA or comparable foreign regulatory authorities may find deficiencies with or fail to approve the manufacturing processes or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and 42 ▪ the approval policies or regulations of the FDA or any comparable foreign regulatory authorities or the laws they enforce may significantly change in a manner rendering our clinical data insufficient for approval.
Our net loss and other operating results will be affected by numerous factors, including: ▪ variations in the level of expense related to the ongoing development of our product candidates and preclinical and clinical development programs; ▪ results of preclinical studies and ongoing and future clinical trials, or the addition or termination of clinical trials or funding support by us, or current or future collaborators or licensing partners; ▪ our execution of any collaboration, licensing or similar arrangements, and the timing of payments we may make or receive under existing or future arrangements or the termination or modification of any such existing or future arrangements; ▪ any intellectual property infringement lawsuit or opposition, interference or cancellation proceeding in which we may become involved; ▪ additions and departures of key personnel; ▪ strategic decisions by us, such as acquisitions, divestitures, spin-offs, joint ventures, strategic investments or changes in business strategy; ▪ if any of our product candidates receives regulatory approval, the terms of such approval and market acceptance and demand for such product candidates; ▪ regulatory developments affecting our product candidates; and ▪ general economic conditions, including as a result of bank failures, as well as economic conditions specifically affecting the biopharmaceutical industry.
Our net loss and other operating results will be affected by numerous factors, including: ▪ variations in the level of expense related to the ongoing development of our product candidates and preclinical and clinical development programs; ▪ results of preclinical studies and ongoing and future clinical trials, or the addition or termination of clinical trials or funding support by us, or current or future collaborators or licensing partners; 77 ▪ our execution of any collaboration, licensing or similar arrangements, and the timing of payments we may make or receive under existing or future arrangements or the termination or modification of any such existing or future arrangements; ▪ any intellectual property infringement lawsuit or opposition, interference or cancellation proceeding in which we may become involved; ▪ additions and departures of key personnel; ▪ strategic decisions by us, such as acquisitions, divestitures, spin-offs, joint ventures, strategic investments or changes in business strategy; ▪ if any of our product candidates receives regulatory approval, the terms of such approval and market acceptance and demand for such product candidates; ▪ regulatory developments affecting our product candidates; and ▪ general economic conditions, including as a result of bank failures, as well as economic conditions specifically affecting the biopharmaceutical industry.
Among other things, these provisions: ▪ establish a classified board of directors such that not all members of the board are elected at one time; ▪ allow the authorized number of our directors to be changed only by resolution of our board of directors; ▪ limit the manner in which stockholders can remove directors from the board; ▪ establish advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and nominations to our board of directors; ▪ require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written consent; ▪ prohibit our stockholders from calling a special meeting of our stockholders; ▪ authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a stockholder rights plan, or so-called “poison pill,” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and ▪ require the approval of the holders of at least 66 2/3% of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our charter or bylaws.
Among other things, these provisions: ▪ establish a classified board of directors such that not all members of the board are elected at one time; ▪ allow the authorized number of our directors to be changed only by resolution of our board of directors; ▪ limit the manner in which stockholders can remove directors from the board; 81 ▪ establish advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and nominations to our board of directors; ▪ require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written consent; ▪ prohibit our stockholders from calling a special meeting of our stockholders; ▪ authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a stockholder rights plan, or so-called “poison pill,” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and ▪ require the approval of the holders of at least 66 2/3% of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our charter or bylaws.
Patient enrollment and trial completion is affected by many factors, including the: ▪ size and nature of the patient population and process for identifying patients; 53 ▪ proximity and availability of clinical trial sites for prospective patients; ▪ ability of patients to travel to clinical trial sites; ▪ eligibility and exclusion criteria for the trial; ▪ design of the clinical trial; ▪ safety profile, to date, of the product candidate under study; ▪ perceived risks and benefits of the product candidate under study; ▪ perceived risks and benefits of our approach; ▪ approval of competing product candidates currently under investigation for the treatment of similar diseases or conditions, or competing clinical trials for similar product candidates or targeting patient populations meeting our patient eligibility criteria; ▪ severity of the disease under investigation; ▪ degree of progression of the patient’s disease at the time of enrollment and throughout the clinical trial; ▪ ability to obtain and maintain patient consent; ▪ risk that enrolled patients will drop out before completion of the trial; ▪ patient referral practices of physicians; and ▪ ability to adequately monitor patients during and after treatment.
Patient enrollment and trial completion is affected by many factors, including the: ▪ size and nature of the patient population and process for identifying patients; ▪ proximity and availability of clinical trial sites for prospective patients; ▪ ability of patients to travel to clinical trial sites; ▪ eligibility and exclusion criteria for the trial; ▪ design of the clinical trial; ▪ safety profile, to date, of the product candidate under study; ▪ perceived risks and benefits of the product candidate under study; ▪ perceived risks and benefits of our approach; ▪ approval of competing product candidates currently under investigation for the treatment of similar diseases or conditions, or competing clinical trials for similar product candidates or targeting patient populations meeting our patient eligibility criteria; ▪ severity of the disease under investigation; ▪ degree of progression of the patient’s disease at the time of enrollment and throughout the clinical trial; ▪ ability to obtain and maintain patient consent; ▪ risk that enrolled patients will drop out before completion of the trial; ▪ patient referral practices of physicians; and ▪ ability to adequately monitor patients during and after treatment.
If our operations are found to be in violation of any of the laws described above or any other governmental laws and regulations that may apply to us, we may be subject to significant penalties, including civil, criminal and administrative penalties, damages, fines, exclusion from government-funded healthcare programs, such as Medicare and Medicaid or similar programs in other countries or jurisdictions, integrity oversight and reporting obligations to resolve allegations of non-compliance, disgorgement, imprisonment, contractual damages, reputational harm, diminished profits and the curtailment or restructuring of our operations.
If our operations are found to be in violation of any of the laws described above or any other governmental laws and regulations that may apply to us, we may be subject to significant penalties, including civil, criminal and administrative penalties, damages, fines, exclusion from government-funded healthcare 53 programs, such as Medicare and Medicaid or similar programs in other countries or jurisdictions, integrity oversight and reporting obligations to resolve allegations of non-compliance, disgorgement, imprisonment, contractual damages, reputational harm, diminished profits and the curtailment or restructuring of our operations.
Disruptions at the FDA, the SEC and other government agencies or comparable regulatory authorities caused by funding shortages or global health concerns could hinder their ability to hire and retain key leadership and other personnel, otherwise prevent new products and services from being developed, approved or commercialized in a 64 timely manner or at all, or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
Disruptions at the FDA, the SEC and other government agencies or comparable regulatory authorities caused by funding shortages or global health concerns could hinder their ability to hire and retain key leadership and other personnel, otherwise prevent new products and services from being developed, approved or commercialized in a timely manner or at all, or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
Later discovery of previously unknown problems with our product candidates, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in revisions to the approved labeling to add new safety information; imposition of 56 post-market studies or clinical trials to assess new safety risks; or imposition of distribution restrictions or other restrictions under a REMS program, or comparable foreign program.
Later discovery of previously unknown problems with our product candidates, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in revisions to the approved labeling to add new safety information; imposition of post-market studies or clinical trials to assess new safety risks; or imposition of distribution restrictions or other restrictions under a REMS program, or comparable foreign program.
Many of our competitors have substantially greater financial, technical and other 60 resources, such as larger research and development staff and experienced marketing and manufacturing organizations and well-established sales forces. Smaller or early-stage companies may also prove to be significant competitors, particularly as they develop novel approaches to treating disease indications that our product candidates are also focused on treating.
Many of our competitors have substantially greater financial, technical and other resources, such as larger research and development staff and experienced marketing and manufacturing organizations and well-established sales forces. Smaller or early-stage companies may also prove to be significant competitors, particularly as they develop novel approaches to treating disease indications that our product candidates are also focused on treating.
Such collaborators may develop adjacent or competing products to ours that are outside the scope of our patents; ▪ we may not develop additional proprietary technologies for which we can obtain patent protection; ▪ it is possible that product candidates or diagnostic tests we develop may be covered by third parties’ patents or other exclusive rights; or ▪ the patents of others may have an adverse effect on our business.
Such collaborators may develop adjacent or competing products to ours that are outside the scope of our patents; ▪ we may not develop additional proprietary technologies for which we can obtain patent protection; ▪ it is possible that product candidates or diagnostic tests we develop may be covered by third parties’ patents or other exclusive rights; or 56 ▪ the patents of others may have an adverse effect on our business.
The overall 10-year period may, occasionally, be extended for a further year to a maximum of 11 years if, during the first eight years of those 10 years, the marketing authorization holder obtains an authorization for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing therapies.
The overall 10-year period may, occasionally, be extended for a further year to a maximum of 11 years if, during the first eight years of those 10 years, the marketing authorization holder obtains an authorization for one or more new 45 therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing therapies.
Disruptions in the financial markets in general, geopolitical conflicts and economic instability, may make equity and debt financing more difficult to obtain, and may have a material adverse effect on our ability 48 to meet our fundraising needs. We cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to us, if at all.
Disruptions in the financial markets in general, geopolitical conflicts and economic instability, may make equity and debt financing more difficult to obtain, and may have a material adverse effect on our ability to meet our fundraising needs. We cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to us, if at all.
Reliance on third-party providers may expose us to more risk than if we were to manufacture our product candidates ourselves. We are dependent on our CMOs for the production of our product candidates in accordance with relevant regulations, such as cGMP, which includes, among other things, quality control, quality assurance and the maintenance of 77 records and documentation.
Reliance on third-party providers may expose us to more risk than if we were to manufacture our product candidates ourselves. We are dependent on our CMOs for the production of our product candidates in accordance with relevant regulations, such as cGMP, which includes, among other things, quality control, quality assurance and the maintenance of records and documentation.
If the preliminary and interim data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, our product candidates may be harmed, which could harm our business, operating results, prospects or financial condition. Preclinical development is uncertain.
If the preliminary and interim data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, our product candidates may be harmed, which could harm our business, operating results, prospects or financial condition. 41 Preclinical development is uncertain.
For example, physicians are often reluctant to switch 62 their patients and patients may be reluctant to switch from existing therapies even when new and potentially more effective or safer treatments enter the market. Efforts to educate the medical community and third-party payors on the benefits of our product candidates may require significant resources and may not be successful.
For example, physicians are often reluctant to switch their patients and patients may be reluctant to switch from existing therapies even when new and potentially more effective or safer treatments enter the market. Efforts to educate the medical community and third-party payors on the benefits of our product candidates may require significant resources and may not be successful.
The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property protection, particularly those relating to biopharmaceutical products, which could make it difficult for us to stop the infringement of our patents or marketing of competing products against third parties in violation of our proprietary rights generally.
The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property protection, particularly those relating to biopharmaceutical products, which could make it difficult for us to stop the infringement of our patents or marketing of competing products against third 62 parties in violation of our proprietary rights generally.
For example, the EU's General Data Protection Regulation, or EU GDPR, the United Kingdom’s GDPR (collectively referred to as the GDPR), Brazil’s General Data Protection Law (Lei Geral de Proteção de Dados Pessoais) (Law No. 13,709/2018), Turkey’s Personal Data Protection Law, South Korea’s Personal Information Protection Act, Taiwan’s Personal Data Protection Act, Peru’s Personal Data Protection Law, South Africa’s Protection of Personal Information Act, and China’s Personal Information Protection Law impose strict requirements for processing personal data.
For example, the EU's General Data Protection Regulation, or EU GDPR, the United Kingdom’s GDPR, or UK GDPR (collectively referred to as the GDPR), Brazil’s General Data Protection Law (Lei Geral de Proteção de Dados Pessoais) (Law No. 13,709/2018), Turkey’s Personal Data Protection Law, South Korea’s Personal Information Protection Act, Taiwan’s Personal Data Protection Act, Peru’s Personal Data Protection Law, South Africa’s Protection of Personal Information Act, and China’s Personal Information Protection Law impose strict requirements for processing personal data.
If a court were to find the exclusive-forum provision contained in our amended and restated certificate of incorporation to be inapplicable or unenforceable in an action, we may incur additional costs associated with resolving such action in other jurisdictions, which could harm our business. ITEM 1B. UNRESOLVED STAFF COMMENTS None.
If a court were to find the exclusive-forum provision contained in our amended and restated certificate of incorporation to be inapplicable or unenforceable in an action, we may incur additional costs associated with resolving such action in other jurisdictions, which could harm our business. ITEM 1B. UNRESOLVED STAFF COMMENTS None. 82
In the ordinary course of our business, we and the third parties with whom we work, collect, receive, store, process, generate, use, transfer, disclose, make accessible, protect, secure, dispose of, transmit and share, or collectively, process, proprietary, confidential and sensitive data, including personal data (such as health-related data), intellectual property and trade secrets, or collectively, sensitive information.
In the ordinary course of our business, we and the third parties with whom we work, collect, receive, store, process, generate, use, transfer, disclose, make accessible, protect, secure, dispose of, transmit and share (collectively, process), proprietary, confidential and sensitive data, including personal data (such as health-related data), intellectual property and trade secrets (collectively, sensitive information).
For example, some of our data processing practices may be challenged under wiretapping laws, if we obtain consumer information from third parties through various methods, including chatbot and session replay providers, or via third-party marketing pixels. These practices may be subject to increased challenges by class action plaintiffs.
For example, some of our data processing practices may be challenged under wiretapping laws, if we obtain consumer information from third parties through various methods, including cookies, chatbot and session replay providers, or via third-party marketing pixels. These practices may be subject to increased challenges by class action plaintiffs.
Such mechanisms include re-examination, post grant review, and equivalent proceedings in foreign jurisdictions (e.g., opposition proceedings). Such proceedings could result in revocation or amendment 73 to our patents in such a way that they no longer cover our product candidates. The outcome following legal assertions of invalidity and unenforceability is unpredictable.
Such mechanisms include re-examination, post grant review, and equivalent proceedings in foreign jurisdictions (e.g., opposition proceedings). Such proceedings could result in revocation or amendment to our patents in such a way that they no longer cover our product candidates. The outcome following legal assertions of invalidity and unenforceability is unpredictable.
These claims might be made by patients who use the product, healthcare providers, pharmaceutical companies, our collaborators or others selling such 58 products. Any claims against us, regardless of their merit, could be difficult and costly to defend and could materially adversely affect the market for our product candidates or any prospects for commercialization of our product candidates.
These claims might be made by patients who use the product, healthcare providers, pharmaceutical companies, our collaborators or others selling such products. Any claims against us, regardless of their merit, could be difficult and costly to defend and could materially adversely affect the market for our product candidates or any prospects for commercialization of our product candidates.
If we, our investigators or any of our CROs or contracted laboratories fail to comply with applicable GLPs and GCPs, the clinical data generated in our clinical 76 trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional preclinical studies or clinical trials before approving our marketing applications.
If we, our investigators or any of our CROs or contracted laboratories fail to comply with applicable GLPs and GCPs, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional preclinical studies or clinical trials before approving our marketing applications.
The collaborator may also consider alternative product candidates or technologies for similar indications that may be available to collaborate on and whether such a collaboration could be more attractive than the one with us. We may not be able to negotiate collaborations on a timely basis, on acceptable terms, or at all.
The collaborator may also consider alternative product candidates or technologies for similar indications that may be available to collaborate on and whether such a collaboration could be more attractive than the one with us. 67 We may not be able to negotiate collaborations on a timely basis, on acceptable terms, or at all.
We conduct certain research and development and clinical operations in Australia, New Zealand, Europe, the United Kingdom and other foreign countries and may also conduct certain future clinical trials outside of the United States. Additionally, while we have not taken any steps to enter into any non-U.S. markets, we may do so in the future.
We have conducted and conduct certain research and development and clinical operations in Australia, New Zealand, Europe, the United Kingdom and other foreign countries and may also conduct certain future clinical trials outside of the United States. Additionally, while we have not taken any steps to enter into any non-U.S. markets, we may do so in the future.
Our collaborations with Takeda and Hansoh and any future collaboration arrangements may not ultimately be successful, which could have a negative impact on our business, results of operations, financial condition and growth prospects. We do not maintain significant rights or control of future development and commercialization activities under our collaboration with Takeda.
Our collaborations with Takeda and Hansoh and any future collaboration arrangements may not ultimately be successful, which could have a negative impact on our business, results of operations, financial condition and growth prospects. We do not maintain significant rights or control of future development and commercialization 66 activities under our collaboration with Takeda.
Open-label clinical trials are also subject to various limitations that may exaggerate any therapeutic effect as patients in open-label clinical trials are aware when they are receiving treatment. Open-label clinical trials may be subject to a “patient bias” where patients perceive their symptoms to have improved merely due to their awareness of receiving an experimental treatment.
Open-label clinical trials are also subject to various limitations that may exaggerate any therapeutic effect as patients in open-label clinical trials are aware when they are receiving treatment. Open-label clinical trials may be subject to a “patient bias” 39 where patients perceive their symptoms to have improved merely due to their awareness of receiving an experimental treatment.
If any of our confidential or proprietary information, such as our trade secrets, were to be disclosed or misappropriated, or if any such information was independently developed by a competitor, our competitive position could be harmed. In addition, courts outside the United States are sometimes less willing to protect trade secrets.
If any of our confidential or proprietary information, such as our trade secrets, were to be disclosed or misappropriated, or if any such information was independently developed by a competitor, our competitive position could be harmed. 57 In addition, courts outside the United States are sometimes less willing to protect trade secrets.
In addition, the use of third-party service providers requires us to disclose our proprietary information to these parties, which could increase the risk that this information will be misappropriated. Our CROs have the right to terminate their agreements with us in the event of an uncured material breach.
In addition, the use of third-party service providers requires us to disclose our proprietary information to these parties, which could increase the risk that this information will be misappropriated. 64 Our CROs have the right to terminate their agreements with us in the event of an uncured material breach.
We and the third parties with whom we work are subject to a variety of evolving threats, including but not limited to social-engineering attacks (including through deep fakes, which are increasingly more difficult to identify as fake, and phishing attacks), malicious code (such as viruses and worms), malware (including as a result of advanced persistent threat intrusions), denial-of-service attacks, credential stuffing attacks, credential harvesting, personnel misconduct or error, ransomware attacks, supply-chain attacks, software bugs, server malfunctions, software or 81 hardware failures, loss of data or other information technology assets, adware, telecommunications failures, attacks enhanced or facilitated by AI, and other similar threats.
We and the third parties with whom we work are subject to a variety of evolving 69 threats, including but not limited to social-engineering attacks (including through deep fakes, which are increasingly more difficult to identify as fake, and phishing attacks), malicious code (such as viruses and worms), malware (including as a result of advanced persistent threat intrusions), denial-of-service attacks, credential stuffing attacks, credential harvesting, personnel misconduct or error, ransomware attacks, supply-chain attacks, software bugs, server malfunctions, software or hardware failures, loss of data or other information technology assets, adware, telecommunications failures, attacks enhanced or facilitated by AI, and other similar threats.
Disputes may also arise between us and our licensors regarding intellectual property subject to a license agreement, including: ▪ the scope of rights granted under the license agreement and other interpretation-related issues; whether and the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement; ▪ our right to sublicense patent and other rights to third parties under collaborative development relationships; ▪ our diligence obligations with respect to the use of the licensed technology in relation to our development and commercialization of our product candidates, and what activities satisfy those diligence obligations; and ▪ the inventorship or ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners.
Disputes may also arise between us and our licensors regarding intellectual property subject to a license agreement, including: ▪ the scope of rights granted under the license agreement and other interpretation-related issues; whether and the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement; ▪ our right to sublicense patents and other rights to third parties under collaborative development relationships; ▪ our diligence obligations with respect to the use of the licensed technology in relation to our development and commercialization of our product candidates, and what activities satisfy those diligence obligations; and ▪ the inventorship or ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners.
Our competitors may have filed, and may in the future file, patent applications covering our products, if approved, or technology similar to ours. Any such patent application may have priority over our owned and in-licensed patent applications or patents, which could require us to obtain rights to issued patents covering such technologies.
Our competitors may have filed, and may in the future file, patent applications covering our products, if approved, or technology similar to ours. Any such patent application may 60 have priority over our owned and in-licensed patent applications or patents, which could require us to obtain rights to issued patents covering such technologies.
Applicable data privacy and security obligations may require us, or we may voluntarily choose, to notify relevant stakeholders, including affected individuals, customers, regulators, and investors, of security incidents, or to take other actions, such as providing credit monitoring and identity theft protection services.
Furthermore, applicable data privacy and security obligations may require us, or we may voluntarily choose, to notify relevant stakeholders, including affected individuals, customers, regulators, and investors, of security incidents, or to take other actions, such as providing credit monitoring and identity theft protection services.
Further, the process of obtaining regulatory approval is expensive, often takes many years following the commencement of clinical trials and can vary substantially based upon the type, complexity and novelty of the product candidates involved, as well as the target indications, patient population and regulatory agency.
Further, the process of obtaining regulatory approval is expensive, often takes many years following the commencement of clinical trials and can vary substantially based upon the type, complexity and novelty of the 38 product candidates involved, as well as the target indications, patient population and regulatory agency.
In the event that any such launch is delayed or does not occur for any reason, we would have prematurely or unnecessarily incurred these commercialization expenses, and our investment would be lost if we cannot retain or reposition our sales and marketing personnel.
In the event that any such launch is delayed or does not occur for any reason, we would have prematurely or unnecessarily incurred these 49 commercialization expenses, and our investment would be lost if we cannot retain or reposition our sales and marketing personnel.
In the past, securities class action litigation has often been brought against public companies following declines in the market prices of their securities. This risk is especially relevant for biopharmaceutical companies, which have experienced significant stock price volatility in recent years.
In the past, securities class action litigation has often been brought against public companies following declines in the market prices of their securities. This risk is especially relevant for biopharmaceutical companies, which have experienced significant 78 stock price volatility in recent years.
The market price for our common stock may fluctuate significantly in response to numerous factors, many of which are beyond our control, including the factors listed below and other factors described in this “Risk Factors” section: ▪ results of preclinical studies and clinical trials of cibotercept, KER-065, elritercept and any other product candidate we may develop or those of our competitors; ▪ actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts; ▪ commencement or termination of collaboration, licensing or similar arrangements for our development programs; ▪ announcements by our competitors of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments; ▪ failure or discontinuation of any of our development programs; ▪ results of clinical trials of product candidates of our competitors; ▪ regulatory or legal developments in the United States and other countries; ▪ developments or disputes concerning patent applications, issued patents or other proprietary rights; ▪ the recruitment or departure of key personnel; ▪ the level of expenses related to the development of cibotercept, KER-065, elritercept and any other product candidate we may develop; ▪ variations in our financial results or those of companies that are perceived to be similar to us; ▪ announcements or expectations of additional financing efforts by us; ▪ sales of our common stock by us, our insiders or other stockholders; ▪ recommendations and changes in estimates or recommendations by securities analysts, if any, that cover our stock; ▪ changes in the structure of healthcare payment systems; ▪ market conditions in the pharmaceutical and biotechnology sectors; ▪ general economic, political, and market conditions and overall fluctuations in the financial markets in the United States and abroad, such as potential future disruptions in access to bank deposits or lending commitments due to bank failures; and ▪ investors’ general perception of us and our business.
The market price for our common stock may fluctuate significantly in response to numerous factors, many of which are beyond our control, including the factors listed below and other factors described in this “Risk Factors” section: ▪ results of preclinical studies and clinical trials of rinvatercept, elritercept and any other product candidate we may develop or those of our competitors; ▪ actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts; ▪ commencement or termination of collaboration, licensing or similar arrangements for our development programs; ▪ announcements by our competitors of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments; ▪ failure or discontinuation of any of our development programs; ▪ results of clinical trials of product candidates of our competitors; ▪ regulatory or legal developments in the United States and other countries; ▪ developments or disputes concerning patent applications, issued patents or other proprietary rights; ▪ the recruitment or departure of key personnel; ▪ the level of expenses related to the development of rinvatercept, elritercept and any other product candidate we may develop; ▪ variations in our financial results or those of companies that are perceived to be similar to us; ▪ announcements or expectations of additional financing efforts by us; ▪ sales of our common stock by us, our insiders or other stockholders; ▪ recommendations and changes in estimates or recommendations by securities analysts, if any, that cover our stock; ▪ changes in the structure of healthcare payment systems; ▪ market conditions in the pharmaceutical and biotechnology sectors; ▪ general economic, political, and market conditions and overall fluctuations in the financial markets in the United States and abroad, such as potential future disruptions in access to bank deposits or lending commitments due to bank failures; and ▪ investors’ general perception of us and our business.
Moreover, in some circumstances, we may 67 not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the patents, covering technology that we license from or license to third parties and are reliant on our licensors or licensees.
Moreover, in some circumstances, we may not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the patents, covering technology that we license from or license to third parties and are reliant on our licensors or licensees.
If we are unable to enter into new collaborations, or if these or our current collaborations are not successful, our business could be adversely affected. 46 • Public health crises could adversely impact our business, including the timing or results of our preclinical studies and clinical trials.
If we are unable to enter into new collaborations, or if these or our current collaborations are not successful, our business could be adversely affected. • Public health crises could adversely impact our business, including the timing or results of our preclinical studies and clinical trials.
In addition, if the FDA or a comparable foreign regulatory authority approves our product candidates, we will have to comply with requirements including submissions of safety and other post-marketing information and reports and registration.
In addition, if the FDA 43 or a comparable foreign regulatory authority approves our product candidates, we will have to comply with requirements including submissions of safety and other post-marketing information and reports and registration.
In September 2023, GSK plc announced that the FDA approved its product, Ojjaara, for the treatment of intermediate or high- 61 risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythaemia vera and post-essential thrombocythaemia), in adults with anemia.
In September 2023, GSK plc announced that the FDA approved its product, Ojjaara, for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythaemia vera and post-essential thrombocythaemia), in adults with anemia.
The timely completion of our clinical trials in accordance with their protocols depends, among other things, on our ability to recruit a sufficient number of eligible patients to participate and remain in the trial until its conclusion.
The timely completion of our clinical trials in accordance with their protocols depends, among other things, on our ability to recruit a 40 sufficient number of eligible patients to participate and remain in the trial until its conclusion.
We are relying on Hansoh to commercialize elritercept in the Hansoh Territory, and if Hansoh is not able to commercialize elritercept in those countries, or determines not to pursue development 78 or commercialization of elritercept in those countries, we will not receive any milestone or royalty payments under the agreement.
We are relying on Hansoh to commercialize elritercept in the Hansoh Territory, and if Hansoh is not able to commercialize elritercept in those countries, or determines not to pursue development or commercialization of elritercept in those countries, we will not receive any milestone or royalty payments under the agreement.
Even if we 55 believe the data collected from clinical trials of our product candidates are promising, such data may not be sufficient to support approval by the FDA or comparable foreign regulatory authorities.
Even if we believe the data collected from clinical trials of our product candidates are promising, such data may not be sufficient to support approval by the FDA or comparable foreign regulatory authorities.
As an example, the regulatory landscape related to clinical trials in the EU has evolved. The EU Clinical Trials Regulation, or CTR, which was adopted in April 2014 and repeals the EU Clinical Trials Directive, became applicable on January 31, 2022.
As an example, the regulatory landscape related to clinical trials in the EU has evolved. The EU Clinical Trials Regulation, or CTR, which was adopted in April 2014 and repeals the EU Clinical Trials Directive, or CTD, became applicable on January 31, 2022.
This type of litigation or proceeding could substantially increase our operating losses and reduce our resources available for development activities. We may not have sufficient financial or other resources to adequately conduct such litigation or proceedings.
This type of litigation or proceeding could substantially increase our operating losses and reduce our resources available for development activities. We may not have sufficient financial or other resources to adequately conduct such 59 litigation or proceedings.
Neither the principal stockholders nor the representatives of the principal stockholders on our board of directors, by the terms of our amended and restated certificate of incorporation, are required to offer us any transaction opportunity of which they become aware and could take any such opportunity for themselves or offer it their other affiliates, unless such opportunity is expressly offered to them solely in their capacity as members of our board of directors. 90 Sales of a substantial number of shares of our common stock in the public market could cause our stock price to fall.
Neither the principal stockholders nor the representatives of the principal stockholders on our board of directors, by the terms of our amended and restated certificate of incorporation, are required to offer us any transaction opportunity of which they become aware and could take any such opportunity for themselves or offer it their other affiliates, unless such opportunity is expressly offered to them solely in their capacity as members of our board of directors. 79 Sales of a substantial number of shares of our common stock in the public market could cause our stock price to fall.
If any of these claims succeed, we may be forced to pay damages on behalf of our customers, suppliers or distributors, or may be 71 required to obtain licenses for the product candidates or services they use.
If any of these claims succeed, we may be forced to pay damages on behalf of our customers, suppliers or distributors, or may be required to obtain licenses for the product candidates or services they use.
An adverse result in 72 any litigation or defense proceedings could put one or more of our patents at risk of being invalidated, held unenforceable, or interpreted narrowly and could put our patent applications at risk of not issuing.
An adverse result in any litigation or defense proceedings could put one or more of our patents at risk of being invalidated, held unenforceable, or interpreted narrowly and could put our patent applications at risk of not issuing.
In each case, these consequences may include: government enforcement actions (for example, investigations, fines, penalties, audits, and inspections); additional reporting requirements and/or oversight; 82 restrictions on processing sensitive information (including personal data); litigation (including class action claims); indemnification obligations; negative publicity; reputational harm; monetary fund diversions; diversion of management attention; interruptions in our operations (including availability of data); financial loss; and other similar harms.
In each case, these consequences may include: government enforcement actions (for example, investigations, fines, penalties, audits, and inspections); additional reporting requirements and/or oversight; restrictions on processing sensitive information (including personal data); litigation (including class action 70 claims); indemnification obligations; negative publicity; reputational harm; monetary fund diversions; diversion of management attention; interruptions in our operations (including availability of data); financial loss; and other similar harms.
Public Health Service Act, which prohibits, among other things, the introduction into interstate commerce of a biological product unless a biologics license is in effect for that product; 65 ▪ the U.S.
Public Health Service Act, which prohibits, among other things, the introduction into interstate commerce of a biological product unless a biologics license is in effect for that product; ▪ the U.S.
The foregoing items, as well as any other future changes in tax laws, could have a material adverse effect on our business, cash flow, financial condition or results of operations.
The foregoing items, as well as any other changes in tax laws, could have a material adverse effect on our business, cash flow, financial condition or results of operations.
These laws may constrain the business or financial arrangements and relationships through which we conduct our operations, including how we research, market, sell and distribute our product candidates, if approved.
These laws may constrain the business or financial arrangements and relationships through 52 which we conduct our operations, including how we research, market, sell and distribute our product candidates, if approved.
If we are successful in obtaining marketing approval from applicable regulatory authorities for cibotercept, KER-065, elritercept or any other product candidate, our ability to generate revenues from any such products will depend on our success in: ▪ launching commercial sales of such products, whether alone or in collaboration with others; ▪ receiving approved labels with claims that are necessary or desirable for successful marketing, and that do not contain safety or other limitations that would impede our ability to market such products; ▪ creating market demand for such products through marketing, sales and promotion activities; ▪ hiring, training, and deploying a sales force or contracting with third parties to commercialize such products in the United States; ▪ creating strategic collaborations with, or offering licenses to, third parties to promote and sell such products in foreign markets where we receive marketing approval; ▪ manufacturing such products in sufficient quantities and at acceptable quality and cost to meet commercial demand at launch and thereafter; ▪ establishing and maintaining agreements with wholesalers, distributors, and group purchasing organizations on commercially reasonable terms; ▪ maintaining patent and trade secret protection and regulatory exclusivity for such products; ▪ achieving market acceptance of such products by patients, the medical community, and third-party payors; ▪ achieving coverage and adequate reimbursement from third-party payors for such products; ▪ patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement from third-party payors; ▪ effectively competing with other therapies; and ▪ maintaining a continued acceptable safety profile of such products following launch.
If we are successful in obtaining marketing approval from applicable regulatory authorities for rinvatercept, elritercept or any other product candidate, our ability to generate revenues from any such products will depend on our success in: ▪ launching commercial sales of such products, whether alone or in collaboration with others; ▪ receiving approved labels with claims that are necessary or desirable for successful marketing, and that do not contain safety or other limitations that would impede our ability to market such products; 47 ▪ creating market demand for such products through marketing, sales and promotion activities; ▪ hiring, training, and deploying a sales force or contracting with third parties to commercialize such products in the United States; ▪ creating strategic collaborations with, or offering licenses to, third parties to promote and sell such products in foreign markets where we receive marketing approval; ▪ manufacturing such products in sufficient quantities and at acceptable quality and cost to meet commercial demand at launch and thereafter; ▪ establishing and maintaining agreements with wholesalers, distributors, and group purchasing organizations on commercially reasonable terms; ▪ maintaining patent and trade secret protection and regulatory exclusivity for such products; ▪ achieving market acceptance of such products by patients, the medical community, and third-party payors; ▪ achieving coverage and adequate reimbursement from third-party payors for such products; ▪ patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement from third-party payors; ▪ effectively competing with other therapies; and ▪ maintaining a continued acceptable safety profile of such products following launch.
Although we have completed our Phase 1 clinical trial of elritercept and our Phase 1 clinical trial of cibotercept, both in healthy volunteers, we may experience delays in our ongoing clinical trials or preclinical studies and we do not know whether planned clinical trials will begin on time, need to be redesigned, enroll patients on time, have sufficient drug supply for our product candidates on a timely basis or be completed on schedule, if at all.
Although we have completed our Phase 1 clinical trial of rinvatercept and our Phase 1 clinical trial of elritercept, both in healthy volunteers, we may experience delays in our ongoing clinical trials or preclinical studies and we do not know whether planned clinical trials will begin on time, need to be redesigned, enroll patients on time, have sufficient drug supply for our product candidates on a timely basis or be completed on schedule, if at all.
The regulation will permit EU Member States to use common HTA tools, methodologies, and procedures across the EU to identify promising technologies early, and continuing voluntary cooperation in other areas. Individual EU Member States will continue to be responsible for assessing non-clinical (e.g., economic, social, ethical) aspects of health technologies, and making decisions on pricing and reimbursement.
The regulation permits EU Member States to use common HTA tools, methodologies, and procedures across the EU to identify promising technologies early, and continuing voluntary cooperation in other areas. Individual EU Member States will continue to be responsible for assessing non-clinical (e.g., economic, social, ethical) aspects of health technologies, and making decisions on pricing and reimbursement.
In addition, the laws of some 74 foreign countries do not protect intellectual property rights to the same extent as do federal and state laws in the United States.
In addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as do federal and state laws in the United States.
We also may experience numerous unforeseen events during our clinical trials that could delay or prevent our ability to receive marketing approval or commercialize cibotercept, KER-065, elritercept or any future product candidates, including: ▪ delays in or failure to obtain regulatory authorizations to commence a trial; ▪ delays in reaching a consensus with regulatory agencies as to the design or implementation of our clinical trials; ▪ delays in or failure to reach agreement on acceptable terms with prospective contract research organizations, or CROs, and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; ▪ delays in or failure to obtain institutional review board, or IRB, or positive ethics committee opinions at each site; ▪ delays in or failure to recruit a sufficient number of suitable patients to participate in a trial; ▪ failure to have patients complete a trial or return for post-treatment follow-up, including disruptions in our ability to treat patients or conduct post-treatment follow-up due to public health crises; ▪ clinical sites deviating from trial protocol, missing data or dropping out of a trial; ▪ delays in adding new clinical trial sites; ▪ failure to manufacture sufficient quantities of our product candidates for use in clinical trials in a timely manner; ▪ occurrence of adverse events associated with the product candidate that are viewed to outweigh its potential benefits, or safety or tolerability concerns that could cause us or our collaborators, as applicable, to suspend or terminate a trial if we or our collaborators find that the participants are being exposed to unacceptable health risks; 50 ▪ failure to perform clinical trials in accordance with the FDA’s or any comparable foreign regulatory authority’s good clinical practices, or GCP, requirements, or regulatory guidelines in other countries; ▪ changes in regulatory requirements, policies and guidelines; ▪ failure of our third-party research contractors to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all; ▪ delays in establishing the appropriate dosage levels and frequency of dosing in clinical trials; ▪ the quality or stability of our product candidates falling below acceptable standards; and ▪ business interruptions resulting from geopolitical actions, including war, such as the current Russia-Ukraine war and the war in Israel, and terrorism or the perception that such hostilities may be imminent, another outbreak of a contagious disease, or natural disasters including earthquakes, typhoons, floods and fires.
We also may experience numerous 37 unforeseen events during our clinical trials that could delay or prevent our ability to receive marketing approval or commercialize rinvatercept, elritercept or any future product candidates, including: ▪ delays in or failure to obtain regulatory authorizations to commence a trial; ▪ delays in reaching a consensus with regulatory agencies as to the design or implementation of our clinical trials; ▪ delays in or failure to reach agreement on acceptable terms with prospective contract research organizations, or CROs, and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; ▪ delays in or failure to obtain institutional review board, or IRB, or positive ethics committee opinions at each site; ▪ delays in or failure to recruit a sufficient number of suitable patients to participate in a trial; ▪ failure to have patients complete a trial or return for post-treatment follow-up, including disruptions in our ability to treat patients or conduct post-treatment follow-up due to public health crises; ▪ clinical sites deviating from trial protocol, missing data or dropping out of a trial; ▪ delays in adding new clinical trial sites; ▪ failure to manufacture sufficient quantities of our product candidates for use in clinical trials in a timely manner; ▪ occurrence of adverse events associated with the product candidate that are viewed to outweigh its potential benefits, or safety or tolerability concerns that could cause us or our collaborators, as applicable, to suspend or terminate a trial if we or our collaborators find that the participants are being exposed to unacceptable health risks; ▪ failure to perform clinical trials in accordance with the FDA’s or any comparable foreign regulatory authority’s good clinical practices, or GCP, requirements, or regulatory guidelines in other countries; ▪ changes in regulatory requirements, policies and guidelines; ▪ failure of our third-party research contractors to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all; ▪ delays in establishing the appropriate dosage levels and frequency of dosing in clinical trials; ▪ the quality or stability of our product candidates falling below acceptable standards; and ▪ business interruptions resulting from geopolitical actions, including war, such as the current Russia-Ukraine war and the war in the Middle East, and terrorism or the perception that such hostilities may be imminent, another outbreak of a contagious disease, or natural disasters including earthquakes, typhoons, floods and fires.
The costs of these opposition or similar proceedings could be substantial, and may result in a loss of scope of some claims or 75 a loss of the entire patent.
The costs of these opposition or similar proceedings could be substantial, and may result in a loss of scope of some claims or a loss of the entire patent.
Many EU Member States also periodically review their reimbursement procedures for medicinal products, which could have an adverse impact on reimbursement status.
Many EU Member States also periodically review their reimbursement procedures for medicinal products, which could have 51 an adverse impact on reimbursement status.
In addition, it is uncertain if and to what extent various states will conform to the Tax Act, as amended by the CARES Act, or newly enacted federal tax legislation. Our ability to use our net operating loss, or NOL, carryforwards and certain tax credit carryforwards may be subject to limitation.
In addition, it is uncertain if and to what extent various states will conform to the Tax Act, as amended by the CARES Act, or newly enacted federal tax legislation such as the OBBBA. Our ability to use our net operating loss, or NOL, carryforwards and certain tax credit carryforwards may be subject to limitation.
We may experience delays in completing, or ultimately be unable to complete, the development and commercialization of cibotercept (KER-012), KER-065, elritercept (KER-050) or any future product candidates. • If we are unable to successfully commercialize any product candidate for which we receive regulatory approval, or experience significant delays in doing so, our business will be materially harmed. • We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively. • Our success depends in part on our ability to protect our intellectual property.
We may experience delays in completing, or ultimately be unable to complete, the development and commercialization of rinvatercept (KER-065), elritercept (KER-050) or any future product candidates. • If we are unable to successfully commercialize any product candidate for which we receive regulatory approval, or experience significant delays in doing so, our business will be materially harmed. • We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively. 33 • Our success depends in part on our ability to protect our intellectual property.
Our future capital requirements for cibotercept, KER-065, elritercept or our other preclinical programs will depend on many factors, including: ▪ the progress, timing and completion of preclinical studies and clinical trials for our current or any future product candidates, as well as the associated costs, including any unforeseen costs we may incur as a result of preclinical study or clinical trial delays due to public health crises or other causes; ▪ the timing and amount of milestone and royalty payments we are required to make or are eligible to receive under our license agreements with each of The General Hospital Corporation and Hansoh (Shanghai) Healthtech Co., Ltd., or Hansoh; ▪ the number of potential new product candidates we identify and decide to develop; ▪ the need for additional or expanded preclinical studies and clinical trials beyond those that we plan to conduct with respect to our current and future product candidates; ▪ the costs involved in growing our organization to the size needed to allow for the research, development and potential commercialization of our current or any future product candidates; ▪ the costs involved in filing patent applications, maintaining and enforcing patents or defending against infringement or other claims raised by third parties; ▪ the maintenance of our existing license and collaboration agreements and the entry into new license and collaboration agreements; ▪ the time and costs involved in obtaining regulatory approval for our product candidates and any delays we may encounter as a result of evolving regulatory requirements or adverse results with respect to any of our product candidates; ▪ the effect of competing technological and market developments; ▪ the cost of manufacturing cibotercept, KER-065, elritercept and future product candidates for clinical trials in preparation for marketing approval applications and in preparation for commercialization; ▪ the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products, if approved, on our own; ▪ the amount of revenues, if any, we may derive either directly or in the form of royalty payments from future sales of our product candidates, if approved; and ▪ market acceptance of any approved product candidates.
Our future capital requirements for rinvatercept, elritercept or our other preclinical programs will depend on many factors, including: ▪ the progress, timing and completion of preclinical studies and clinical trials for our current or any future product candidates, as well as the associated costs, including any unforeseen costs we may incur as a result of preclinical study or clinical trial delays due to public health crises or other causes; ▪ the timing and amount of milestone and royalty payments we are required to make or are eligible to receive under our license agreements with each of The General Hospital Corporation, Takeda and Hansoh; ▪ the number of potential new product candidates we identify and decide to develop; ▪ the need for additional or expanded preclinical studies and clinical trials beyond those that we plan to conduct with respect to our current and future product candidates; ▪ the costs involved in growing our organization to the size needed to allow for the research, development and potential commercialization of our current or any future product candidates; ▪ the costs involved in filing patent applications, maintaining and enforcing patents or defending against infringement or other claims raised by third parties; ▪ the maintenance of our existing license and collaboration agreements and the entry into new license and collaboration agreements; ▪ the time and costs involved in obtaining regulatory approval for our product candidates and any delays we may encounter as a result of evolving regulatory requirements or adverse results with respect to any of our product candidates; ▪ the effect of competing technological and market developments; 35 ▪ the cost of manufacturing rinvatercept and future product candidates for clinical trials in preparation for marketing approval applications and in preparation for commercialization; ▪ the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products, if approved, on our own; ▪ the amount of revenues, if any, we may derive either directly or in the form of royalty payments from future sales of our product candidates, if approved; and ▪ market acceptance of any approved product candidates.
In addition, under Sections 382 and 383 of the Code and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation’s ability to use its pre-change NOL carryforwards and research and development, or R&D, credits to offset its post-change income may be limited.
In addition, under Sections 382 and 383 of the Code and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation’s ability to use its pre-change NOL and research and development, or R&D, tax credit carryforwards to offset its post-change taxable income and tax may be limited.
CMS 66 decides whether and to what extent a new product will be covered and reimbursed under Medicare, and private third-party payors often follow CMS’s decisions regarding coverage and reimbursement to a substantial degree. However, one third-party payor’s determination to provide coverage for a product candidate does not assure that other payors will also provide coverage for the product candidate.
The CMS decide whether and to what extent a new product will be covered and reimbursed under Medicare, and private third-party payors often follow CMS’s decisions regarding coverage and reimbursement to a substantial degree. However, one third-party payor’s determination to provide coverage for a product candidate does not assure that other payors will also provide coverage for the product candidate.
Cibotercept, KER-065, elritercept and any future product candidates we develop will require additional preclinical and clinical development, management of clinical, preclinical and manufacturing activities, marketing approval in the United States and other jurisdictions for specific indications for use, demonstrating effectiveness to pricing and reimbursement authorities, obtaining sufficient manufacturing supply for both clinical development and commercial production, building of a commercial organization and substantial investment and significant marketing efforts before we generate any revenues from product sales.
Rinvatercept, elritercept and any future product candidates we develop will require additional preclinical and clinical development, management of clinical, preclinical and manufacturing activities, marketing approval in the United States and other jurisdictions for specific indications for use, demonstrating effectiveness to pricing and reimbursement authorities, obtaining sufficient manufacturing supply for both clinical development and commercial production, building of a commercial organization and substantial investment and significant marketing efforts before we generate any revenues from product sales.
These provisions could discourage potential acquisition proposals and could delay or prevent a change in control transaction. They could also have 92 the effect of discouraging others from making tender offers for our common stock, including transactions that may be in your best interests.
These provisions could discourage potential acquisition proposals and could delay or prevent a change in control transaction. They could also have the effect of discouraging others from making third-pary tender offers for our common stock, including transactions that may be in your best interests.
Future growth would impose significant added responsibilities on members of management, including: ▪ identifying, recruiting, integrating, maintaining and motivating additional employees; ▪ managing our development efforts effectively, including the clinical and FDA review process for cibotercept, KER-065, elritercept and any future product candidates, while complying with our contractual obligations to contractors and other third parties; and ▪ improving our operational, financial and management controls, reporting systems and procedures.
Future growth would impose significant added responsibilities on members of management, including: ▪ identifying, recruiting, integrating, maintaining and motivating additional employees; ▪ managing our development efforts effectively, including the clinical and FDA review process for rinvatercept, elritercept and any future product candidates, while complying with our contractual obligations to contractors and other third parties; and ▪ improving our operational, financial and management controls, reporting systems and procedures.
The costs to respond to a security incident and/or to mitigate any security vulnerabilities that may be identified could be significant, our efforts to address these problems may not be successful, and these problems could result in unexpected interruptions, delays, cessation of service, negative publicity, and other harm to our business and our competitive position.
The costs to respond to a security incident and/or to mitigate any security vulnerabilities that we identify could be significant, our efforts to address these problems may not be successful, and these problems could result in unexpected interruptions, delays, cessation of service, negative publicity, and other harm to our business and our competitive position.
For example, in November 2023, we announced the deprioritization of our small molecule product candidate, KER-047, a potent and selective inhibitor of activin receptor-like kinase-2, a TGF-ß superfamily receptor, including our decision to pause all development activities associated with this asset.
Additionally, in November 2023, we announced the deprioritization of our small molecule product candidate, KER-047, a potent and selective inhibitor of activin receptor-like kinase-2, a TGF-ß superfamily receptor, including our decision to pause all development activities associated with this asset.
Risks Related to Our Employee Matters, Managing Our Growth and Other Risks Relating to Our Operations We are highly dependent on our key personnel, including our Chief Executive Officer, Chief Operating Officer and Chief Medical Officer. If we are not successful in attracting, motivating and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.
Risks Related to Our Employee Matters, Managing Our Growth and Other Risks Relating to Our Operations We are highly dependent on our key personnel, including our Chief Executive Officer and Chief Scientific Officer. If we are not successful in attracting, motivating and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.
To obtain the requisite regulatory approvals to market and sell any of our product candidates, including cibotercept, KER-065, elritercept and any other future product candidates, we must demonstrate through extensive preclinical studies and clinical trials that our investigational drug products are safe and effective for use in each targeted indication, and in the case of our product candidates regulated as biological products, such as cibotercept, KER-065 and elritercept, that the product candidate is safe, pure and potent for use in its targeted indication.
To obtain the requisite regulatory approvals to market and sell any of our product candidates, including rinvatercept, elritercept and any other future product candidates, we must demonstrate through extensive preclinical studies and clinical trials that our investigational drug products are safe and effective for use in each targeted indication, and in the case of our product candidates regulated as biological products, such as rinvatercept and elritercept, that the product candidate is safe, pure and potent for use in its targeted indication.
Prior to obtaining approval to commercialize cibotercept, KER-065, elritercept and any future product candidates in the United States or abroad, we, our collaborators or our potential future collaborators must demonstrate with substantial evidence from adequate and well-controlled clinical trials, and to the satisfaction of the FDA or comparable foreign regulatory authorities, that such product candidates are safe and effective for their intended uses.
Prior to obtaining approval to commercialize rinvatercept, elritercept and any future product candidates in the United States or abroad, we, our collaborators or our potential future collaborators must demonstrate with substantial evidence from adequate and well-controlled clinical trials, and to the satisfaction of the FDA or comparable foreign regulatory authorities, that such product candidates are safe and effective for their intended uses.
Other states are considering and may adopt similar laws. Additionally, under various privacy laws and other obligations, we may be required to obtain certain consents to process personal data.
Other states have passed, are considering, and may adopt similar laws. Additionally, under various privacy laws and other obligations, we may be required to obtain certain consents to process personal data.
Other legislative changes have been proposed and adopted in the United States since the ACA was enacted. In August 2011, the Budget Control Act of 2011, among other things, led to aggregate reductions of Medicare payments to providers of 2% per fiscal year.
Other legislative changes have also been proposed and adopted in the United States since the ACA was enacted. For example, in August 2011, the Budget Control Act of 2011, among other things, led to aggregate reductions of Medicare payments to providers of 2% per fiscal year.
In addition, if we are unable to effectively manage our outsourced activities or if the quality or accuracy of the services provided by consultants is compromised for any reason, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain marketing approval of cibotercept, KER-065, elritercept and our other product candidates or otherwise advance our business.
In addition, if we are unable to effectively manage our outsourced activities or if the quality or accuracy of the services provided by consultants is compromised for any reason, our clinical trials may be extended, delayed or terminated, and we may not be able to obtain marketing approval of rinvatercept, elritercept and our other product candidates or otherwise advance our business.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeOur cybersecurity risk assessment and management processes are implemented and maintained by leaders from our IT, finance and legal teams, including our Executive Director who serves as the Head of IT and Cybersecurity, who has twenty years of experience in various roles involving information technology and cybersecurity.
Biggest changeThis includes existing and new cybersecurity risks, status on how management is addressing and/or mitigating those risks, cybersecurity and data privacy incidents (if any) and status on key information security initiatives. 83 Our cybersecurity risk assessment and management processes are implemented and maintained by leaders from our IT, finance and legal teams, including our Executive Director who serves as the Head of IT and Cybersecurity, who has twenty years of experience in various roles involving information technology and cybersecurity.
We implement and maintain various technical, physical, and organizational measures, processes, standards and policies designed to manage and mitigate material risks from cybersecurity threats to our Information Systems and Data, including, 93 for example: maintaining an incident response plan and policy, incident detection and response, maintaining a vulnerability management policy, maintaining disaster recovery and business continuity plans, conducting risk assessments, implementing security standards and certifications, maintaining network security controls, access controls, and physical security measures, asset management, systems monitoring, conducting proactive privacy and cybersecurity reviews of systems and applications, performing penetration testing using external third-party tools and techniques to test security controls, conducting employee training, monitoring emerging laws and regulations related to data protection and information security and implement appropriate changes and encrypting Information Systems and Data.
We implement and maintain various technical, physical, and organizational measures, processes, standards and policies designed to manage and mitigate material risks from cybersecurity threats to our Information Systems and Data, including, for example: maintaining an incident response plan and policy, incident detection and response, maintaining a vulnerability management policy, maintaining disaster recovery and business continuity plans, conducting risk assessments, implementing security standards and certifications, maintaining network security controls, access controls, and physical security measures, asset management, systems monitoring, conducting proactive privacy and cybersecurity reviews of systems and applications, performing penetration testing using external third-party tools and techniques to test security controls, conducting employee training, monitoring emerging laws and regulations related to data protection and information security and implement appropriate changes and encrypting Information Systems and Data.
Our Head of IT works with the Company’s incident response team to help the Company mitigate and remediate cybersecurity incidents of which they are notified. In addition, our incident response plan includes reporting to our audit committee for certain cybersecurity incidents. 94
Our Head of IT works with the Company’s incident response team to help the Company mitigate and remediate cybersecurity incidents of which they are notified. In addition, our incident response plan includes reporting to our audit committee for certain cybersecurity incidents.
Our cybersecurity incident response plan is designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Chief Financial Officer, Chief Operating Officer, General Counsel and Head of Human Resources. Incidents are evaluated to determine materiality as well as operational and business impact, and reviewed for privacy impact.
Our cybersecurity incident response plan is designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Chief Financial Officer and General Counsel. Incidents are evaluated to determine materiality as well as operational and business impact, and reviewed for privacy impact.
Removed
This includes existing and new cybersecurity risks, status on how management is addressing and/or mitigating those risks, cybersecurity and data privacy incidents (if any) and status on key information security initiatives.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeThe results of any future claims or proceedings cannot be predicted with certainty, and regardless of the outcome, litigation can have an adverse impact on us because of defense and litigation costs, diversion of management resources, and other factors. ITEM 4. MINE SAFETY DISCLOSURES Not applicable. 95 PART II
Biggest changeThe results of any future claims or proceedings cannot be predicted with certainty, and regardless of the outcome, litigation can have an adverse impact on us because of defense and litigation costs, diversion of management resources, and other factors. ITEM 4. MINE SAFETY DISCLOSURES Not applicable. 84 PART II

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeThe payment of dividends will be at the discretion of our board of directors and will depend on our results of operations, capital requirements, financial condition, prospects, contractual arrangements, any limitations on payment of dividends present in any future debt agreements and other factors that our board of directors may deem relevant.
Biggest changeThe payment of dividends will be at the discretion of our board of directors and will depend on our results of operations, capital requirements, financial condition, prospects, contractual arrangements, any limitations on payment of dividends present in any future debt agreements and other factors that our board of directors may deem relevant. Recent Sales of Unregistered Securities None.
Holders As of February 20, 2025, there were 16 stockholders of record of our common stock, one of which is Cede & Co., a nominee for Depository Trust Company, or DTC.
Holders As of February 27, 2026, there were 12 stockholders of record of our common stock, one of which is Cede & Co., a nominee for Depository Trust Company, or DTC.
Removed
Stock Performance Graph This performance graph below shall not be deemed "soliciting material" or to be "filed" with the SEC for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities under that Section, and shall not be deemed to be incorporated by reference into this Annual Report on Form 10-K or any other filing of Keros Therapeutics, Inc. under the Exchange Act or the Securities Act, whether made before or after the date hereof and irrespective of any general incorporation language in any such filing, except to the extent that we specifically incorporate this information by reference therein.
Added
Purchase of Equity Securities by the Issuer and Affiliated Purchasers Our stock repurchase activity for each of the three months in the quarter ended December 31, 2025 was: Period Total Number of Shares Purchased Average Price Paid per Share Total Number of Shares Purchased as Part of Publicly Announced Plans or Programs Approximate Dollar Value of Shares That May Yet Be Purchased Under the Plans or Programs October 1-31, 2025 10,176,595(1)(2) $ 17.75 10,176,595 $ — November 1-30, 2025 10,950,165(3) 17.75 10,950,165 — December 1-31, 2025 — — — — Total 21,126,760 21,126,760 (1) On October 15, 2025, we entered into stock purchase agreements with certain entities affiliated with ADAR1 Capital Management (collectively the “ADAR1 Parties”, and such stock purchase agreement, the “ADAR1 Repurchase Agreement”), and certain entities affiliated with Pontifax Venture Capital (collectively, the “Pontifax Parties”, and such stock purchase agreement, the “Pontifax Repurchase Agreement” and, together with the ADAR1 Repurchase Agreement, the “Repurchase Agreements”).
Removed
The graph set forth below compares the cumulative total stockholder return on our common stock between April 8, 2020 (the date our common stock commenced trading on the Nasdaq Global Market) and December 31, 2024, with the cumulative total return of (a) the Nasdaq Composite Index and (b) the Nasdaq Biotechnology Index, over the same period.
Added
Pursuant to the terms and conditions of the Repurchase Agreements, the ADAR1 Parties and the Pontifax Parties sold all of the shares of common stock beneficially owned by them, being an aggregate of 10,176,595 shares of common stock, to us at a per share purchase price of $17.75 per share, for an aggregate purchase price of $180.6 million.
Removed
This graph assumes the investment of $100 on April 8, 2020 in our common stock, the Nasdaq Composite Index, and the Nasdaq Biotechnology Index and assumes the reinvestment of dividends, if any.
Added
(2) On October 20, 2025, we announced that our board of directors authorized the repurchase of up to $194.4 million shares of our common stock at a cash purchase price of $17.75 per share, through a tender offer, which we refer to as the Tender Offer.
Removed
The graph assumes our closing sales price on April 8, 2020 of $20.08 per share as the initial value of our common stock and not the initial offering price to the public of $16.00 per share. The comparisons shown in the graph below are based upon historical data.
Added
We launched the Tender Offer on October 20, 2025 and it was completed on November 21, 2025. (3) On November 20, 2025, we disclosed that a total of 17,712,262 shares of our common stock were validly tendered and not validly withdrawn.
Removed
We caution that the stock price performance shown in the graph below is not necessarily indicative of, nor is it intended to forecast, the potential future performance of our common stock. 96 Recent Sales of Unregistered Securities None. Use of Proceeds None. Purchase of Equity Securities by the Issuer and Affiliated Purchasers None. ITEM 6. [Reserved]
Added
In accordance with the terms and conditions of the Tender Offer, we accepted for purchase a total of 10,950,165 shares of common stock, or the Tender Offer Shares, at a purchase price of $17.75 per share, for an aggregate purchase price of approximately $194.4 million, excluding fees and expenses of $9.6 million relating to the Repurchase Agreements and the Tender Offer.
Added
As a result of the Tender Offer, we had 19,543,706 shares of common stock outstanding immediately following the closing of the Tender Offer. 85 ITEM 6. [Reserved]

Item 7. Management's Discussion & Analysis

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

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Biggest changeWe have not recorded any income tax benefits for the losses incurred as it is more likely than not that these benefits will not be realized based on our history of losses and expected future losses. 101 Results of Operations Comparison for the years ended December 31, 2024, 2023, and 2022 The following table summarizes our results of operations for the years ended December 31, 2024, 2023, and 2022 (in thousands): YEAR ENDED DECEMBER 31, 2024 2023 2022 REVENUE: Service and other revenue $ 550 $ 151 $ — License revenue $ 3,000 $ — $ — Total revenue 3,550 151 — OPERATING EXPENSES: Research and development (173,629) (135,258) (87,265) General and administrative (40,754) (34,834) (27,525) Total operating expenses (214,383) (170,092) (114,790) LOSS FROM OPERATIONS (210,833) (169,941) (114,790) OTHER INCOME (EXPENSE), NET: Interest expense, net — — (1) Research and development incentive income 1,238 2,400 7,081 Dividend income 23,496 14,755 3,644 Other expense, net (954) (206) (613) Total other income (expense), net 23,780 16,949 10,111 Loss before income taxes (187,053) (152,992) (104,679) Income tax provision (300) — — Net loss $ (187,353) $ (152,992) $ (104,679) Revenue Our revenue for the year ended December 31, 2024 consisted of service and other revenue substantially related to the Tech Transfer Agreement and Supply Agreement and license revenue related to the Hansoh Agreement.
Biggest changeWe continue to maintain a full valuation allowance against our net deferred tax assets due to our history of losses and expected future losses. 90 Results of Operations Comparison for the years ended December 31, 2025 and 2024 The following table summarizes our results of operations for the years ended December 31, 2025 and 2024 (in thousands): YEAR ENDED DECEMBER 31, 2025 2024 REVENUE: Service and other revenue $ 38,706 $ 550 License revenue $ 205,355 $ 3,000 Total revenue 244,061 3,550 OPERATING EXPENSES: Research and development (129,643) (173,629) General and administrative (46,849) (40,754) Total operating expenses (176,492) (214,383) INCOME (LOSS) FROM OPERATIONS 67,569 (210,833) OTHER INCOME (EXPENSE), NET: Research and development incentive income — 1,238 Dividend income 24,867 23,496 Other expense, net (539) (954) Total other income, net 24,328 23,780 Income (loss) before income taxes 91,897 (187,053) Income tax provision (4,883) (300) Net income (loss) $ 87,014 $ (187,353) Revenue We recognized $205.4 million of license revenue related to the upfront payment and achievement of a development milestone under the Takeda Agreement and $38.5 million of service and other revenue related to the transition services agreement with Takeda, or the TSA, for the year ended December 31, 2025, compared to zero for the year ended December 31, 2024.
We are a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including blood, bone, skeletal muscle, adipose and heart tissue.
We are a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including skeletal muscle, bone, adipose, heart tissue and blood.
We do not expect to generate any revenue from product sales unless and until we successfully complete development and obtain regulatory approval for one or more of our product candidates, which we expect will take a number of years. Since our inception, we have funded our operations primarily through equity financings and through research collaborations or licensing of intellectual property.
We do not expect to generate any revenue from product sales unless and until we successfully complete development and obtain regulatory approval for one or more of our product candidates, which we expect will take a number of years. Since our inception, we have funded our operations primarily through equity financings, research collaborations or licensing of intellectual property.
Cash Provided by Financing Activities Net cash provided by financing activities was $391.8 million for the year ended December 31, 2024, which was primarily related to (i) net proceeds of $151.1 million received from our public offering of common stock in January 2024, after deducting underwriting discounts, commissions and offering expenses; (ii) net proceeds of $228.6 million received from sales of our common stock under the ATM Program, after deducting sales agent commissions and offering expenses; and (iii) proceeds of $12.1 million related to exercises of options to purchase common stock.
Net cash provided by financing activities was $391.8 million for the year ended December 31, 2024, which was primarily related to (i) net proceeds of $151.1 million received from our public offering of common stock in January 2024, after deducting underwriting discounts, commissions and offering expenses; (ii) net proceeds of $228.6 million received from sales of our common stock under the ATM Program, after deducting sales agent commissions and offering expenses; and (iii) proceeds of $12.1 million related to exercises of options to purchase common stock.
For stock options with performance conditions, stock-based compensation costs are recognized as expense using the accelerated attribution method when it is probable that the performance condition will be achieved. Our Black-Scholes option-pricing model requires the input of subjective assumptions, including the expected volatility of the price of our common stock. We lack company-specific historical and implied volatility information.
For stock options and RSUs with performance conditions, stock-based compensation costs are recognized as expense using the accelerated attribution method when it is probable that the performance condition will be achieved. Our Black-Scholes option-pricing model requires the input of subjective assumptions, including the expected volatility of the price of our common stock. We lack company-specific historical and implied volatility information.
If factors change and these assumptions either increase or decrease, our stock-based compensation expense could materially differ in the future. Stock-based compensation expense is classified in the accompanying statements of operations based on the function to which the related services are provided. We recognize stock-based compensation expense for the portion of awards that have vested.
If factors change and these assumptions either increase or decrease, our stock-based compensation expense could materially differ in the future. Stock-based compensation expense is classified in the accompanying statements of operations based on the function to which the related services are 96 provided. We recognize stock-based compensation expense for the portion of awards that have vested.
Although we do not expect our estimates to 107 be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in reporting amounts that are too high or too low in any particular period.
Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in reporting amounts that are too high or too low in any particular period.
To date, we have not generated any revenue from product sales 97 as none of our product candidates have been approved for commercialization. We have historically financed our operations primarily through the sale of convertible preferred stock and common stock and cash received from licensing agreements.
To date, we have not generated any revenue from product sales as none of our product candidates have been approved for commercialization. We have historically financed our operations primarily through the sale of convertible preferred stock and common stock and cash received from licensing agreements.
Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and 100 are reflected in our consolidated financial statements as prepaid or accrued research and development expenses.
Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our consolidated financial statements as prepaid or accrued research and development expenses.
Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses and expensed as the related goods are delivered or the services are performed. Research and development activities are central to our business model.
Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses and expensed as the related goods are delivered or the services are performed. 89 Research and development activities are central to our business model.
During the royalty term, neither party will directly or indirectly commercialize a competing product in the Hansoh Territory. Effective in June 2023, in connection with the Hansoh Agreement, we entered into a manufacturing technology transfer agreement, or the Tech Transfer Agreement, with Hansoh.
During the royalty term, neither party will directly or indirectly commercialize a competing product in the Hansoh Territory. 88 Effective in June 2023, in connection with the Hansoh Agreement, we entered into a manufacturing technology transfer agreement, or the Tech Transfer Agreement, with Hansoh.
The measurement date for awards is the date of grant. For stock options that vest based on service conditions, stock-based compensation costs are recognized as expense over the requisite service period, which is the vesting period, on a straight-line basis.
The measurement date for awards is the date of grant. For stock-based awards that vest based on service conditions, stock-based compensation costs are recognized as expense over the requisite service period, which is the vesting period, on a straight-line basis.
Inflationary factors, such as increases in the cost of materials and 98 supplies relating to our preclinical studies, clinical trials, interest rates and overhead costs may adversely affect our operating results.
Inflationary factors, such as increases in the cost of materials and supplies relating to our preclinical studies, clinical trials, interest rates and overhead costs may adversely affect our operating results.
In addition, public health crises, bank failures, geopolitical tensions and resulting global slowdown of economic activity continue to rapidly evolve and have already resulted in a significant disruption of global financial markets. If the disruption persists and deepens, we could experience an inability to access additional capital when and if needed.
In addition, implementation of tariffs, public health crises, bank failures, geopolitical tensions and resulting global slowdown of economic activity continue to rapidly evolve and have already resulted in a significant disruption of global financial markets. If the disruption persists and deepens, we could experience an inability to access additional capital when and if needed.
Our future funding requirements, both near and long-term, will depend on many factors, including: ▪ the progress, timing and completion of preclinical studies and clinical trials for our current or any future product candidates, as well as the associated costs, including any unforeseen costs we may incur as a result of preclinical study or clinical trial delays due to public health crises or other causes; ▪ the timing and amount of milestone and royalty payments we are required to make or are eligible to receive under our license agreements with each of The General Hospital Corporation and Hansoh; ▪ the number of potential new product candidates we identify and decide to develop; ▪ the need for additional or expanded preclinical studies and clinical trials beyond those that we plan to conduct with respect to our current and future product candidates; ▪ the costs involved in growing our organization to the size needed to allow for the research, development and potential commercialization of our current or any future product candidates; ▪ the costs involved in filing patent applications, maintaining and enforcing patents or defending against infringement or other claims raised by third parties; ▪ the maintenance of our existing license and collaboration agreements and the entry into new license and collaboration agreements; 104 ▪ the time and costs involved in obtaining regulatory approval for our product candidates and any delays we may encounter as a result of evolving regulatory requirements or adverse results with respect to any of our product candidates; ▪ the effect of competing technological and market developments; ▪ the costs of operating as a public company; ▪ the cost of manufacturing cibotercept, KER-065, elritercept and future product candidates for clinical trials in preparation for marketing approval and in preparation for commercialization; ▪ the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products, if approved, on our own; ▪ the amount of revenues, if any, we may derive either directly or in the form of royalty payments from future sales of our product candidates, if approved; and ▪ market acceptance of any approved product candidates.
Our future funding requirements, both near and long-term, will depend on many factors, including: ▪ the progress, timing and completion of preclinical studies and clinical trials for our current or any future product candidates, as well as the associated costs, including any unforeseen costs we may incur as a result of preclinical study or clinical trial delays due to public health crises or other causes; ▪ the timing and amount of milestone and royalty payments we are required to make or are eligible to receive under our license agreements with each of The General Hospital Corporation, Hansoh and Takeda; ▪ the number of potential new product candidates we identify and decide to develop; ▪ the need for additional or expanded preclinical studies and clinical trials beyond those that we plan to conduct with respect to our current and future product candidates; ▪ the costs involved in growing our organization to the size needed to allow for the research, development and potential commercialization of our current or any future product candidates; ▪ the costs involved in filing patent applications, maintaining and enforcing patents or defending against infringement or other claims raised by third parties; ▪ the maintenance of our existing license and collaboration agreements and the entry into new license and collaboration agreements; 93 ▪ the time and costs involved in obtaining regulatory approval for our product candidates and any delays we may encounter as a result of evolving regulatory requirements or adverse results with respect to any of our product candidates; ▪ the effect of competing technological and market developments; ▪ the costs of operating as a public company; ▪ the cost of manufacturing rinvatercept and future product candidates for clinical trials in preparation for marketing approval and in preparation for commercialization; ▪ the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products, if approved, on our own; ▪ the amount of revenues, if any, we may derive either directly or in the form of royalty payments from future sales of our product candidates, if approved; and ▪ market acceptance of any approved product candidates.
Although we do not believe that inflation or higher interest rates have had a material impact on our financial position or results of operations to date, we may experience increases in the near future (especially if inflation rates rise more quickly) on our operating costs, including our labor costs and research and development costs, due to supply chain constraints, consequences associated with public health crises and global geopolitical tensions, such as the ongoing war between Russia and Ukraine and the war in Israel, worsening global macroeconomic conditions, including as a result of bank failures, and employee availability and wage increases, which may result in additional stress on our working capital resources.
Although we do not believe that inflation, higher interest rates or tariffs have had a material impact on our financial position or results of operations to date, we may 87 experience increases in the near future (especially if inflation rates rise more quickly) on our operating costs, including our labor costs and research and development costs, due to supply chain constraints, consequences associated with public health crises and global geopolitical tensions, such as the ongoing war between Russia and Ukraine and the war in the Middle East, worsening global macroeconomic conditions, including as a result of bank failures, and employee availability and wage increases, which may result in additional stress on our working capital resources.
Rising interest rates also present a recent challenge impacting the U.S. economy and could make it more difficult for us to obtain traditional financing on acceptable terms, if at all, in the future.
Rising interest rates and implementation of tariffs also present a recent challenge impacting the U.S. economy and could make it more difficult for us to obtain traditional financing on acceptable terms, if at all, in the future.
Dividend Income Dividend income consists of income earned on our money market funds that are recorded as cash equivalents on our consolidated balance sheets. Other Income (Expense), Net Other income (expense), net primarily consists of unrealized and realized gains and losses on foreign currency.
Dividend Income Dividend income consists of income earned on our money market funds that are recorded as cash equivalents on our consolidated balance sheets. Other Expense, Net Other expense, net consists of unrealized and realized gains and losses on foreign currency and other taxes and fees.
Our second product candidate, KER-065, is being developed for the treatment of neuromuscular diseases. Our most advanced product candidate, elritercept (KER-050), is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndromes, or MDS, and in patients with myelofibrosis.
Our most advanced product candidate, elritercept (KER-050), is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndromes, or MDS, and in patients with myelofibrosis.
To date, there have not been any material adjustments to our prior estimates of accrued and prepaid research and development expenses. Stock-Based Compensation We account for all stock-based compensation awards granted to employees and non-employees as stock-based compensation expense at fair value. Our stock-based awards include stock options and performance-based stock options.
To date, there have not been any material adjustments to our prior estimates of accrued and prepaid research and development expenses. Stock-Based Compensation We account for all stock-based compensation awards granted to employees and non-employees as stock-based compensation expense at fair value. Our stock-based awards include stock options and restricted stock unit, or RSU, awards.
Although we continue to pursue these plans, there is no assurance that we will be successful in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all. As of December 31, 2024, we had cash and cash equivalents of $559.9 million.
Although we continue to pursue these plans, there is no assurance that we will be successful in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all. As of December 31, 2025, we had cash and cash equivalents of $287.4 million.
Under such agreements, the exact amounts owed by us in the event of termination will be based on the timing of the termination and the exact terms of the agreement. As of December 31, 2024, we have committed up to approximately $27.2 million under these agreements which are expected to be paid through 2029.
Under such agreements, the exact amounts owed by us in the event of termination will be based on the timing of the termination and the exact terms of the agreement. As of December 31, 2025, we have committed up to approximately $1.6 million under these agreements which are expected to be paid through 2029.
As of December 31, 2024, we have sold a total of 4,290,096 shares of our common stock pursuant to the ATM Offering for aggregate net proceeds of approximately $228.6 million after deducting sales agent commissions and estimated offering expenses.
As of December 31, 2025, we have sold a total of 4,290,096 shares of our common stock pursuant to the ATM Offering for aggregate net proceeds of approximately $228.6 million after deducting sales agent commissions and estimated offering expenses. As of December 31, 2025, we may not offer and sell any ATM shares.
The increase of $6.8 million is primarily related to an increase of $8.7 million of dividend income, partially offset by (i) a decrease of $1.2 million in R&D Incentive income in Australia; and (ii) an increase of $0.7 million in other expense, net.
The increase of $0.5 million was primarily related to (i) an increase of $1.4 million of dividend income and (ii) a decrease of $0.4 million in other expense, net, partially offset by a decrease of $1.2 million in R&D Incentive income in Australia.
We recognized $96.1 thousand and $150.8 thousand of service and other revenue for the years ended December 31, 2024 and 2023, respectively. Effective in February 2024, in connection with the Hansoh Agreement, we entered into a clinical product supply agreement with Hansoh, or the Supply Agreement.
We recognized $0.1 million and $0.1 million of service and other revenue for the years ended December 31, 2025 and 2024, respectively. Effective in February 2024, in connection with the Hansoh Agreement, we entered into a clinical product supply agreement with Hansoh, or the Supply Agreement.
We recognized $421.1 thousand of other revenue for the year ended December 31, 2024. 2024 License Agreement with Takeda Pharmaceuticals U.S.A., Inc. In December 2024, we entered into a license agreement with Takeda, which became effective on January 16, 2025.
We recognized $0.1 million and $0.4 million of other revenue for the years ended December 31, 2025 and 2024, respectively. 2024 License Agreement with Takeda Pharmaceuticals U.S.A., Inc. In December 2024, we entered into a license agreement with Takeda, which became effective on January 16, 2025.
Income Tax Provision The tax provision recorded for the year ended December 31, 2024 resulted from withholding tax related to taxable income generated from the Hansoh Agreement.
Income Tax Provision The tax provision recorded for the year ended December 31, 2025 resulted from income tax related to taxable income generated from the Takeda Agreement.
Net cash used in operating activities was $124.5 million for the year ended December 31, 2023, which was driven by a net loss of $153.0 million and $2.7 million net cash used by operating assets and liabilities and non-cash charges, partially offset by non-cash charges including $28.8 million of stock-based compensation expense, $1.6 million in lease expenses and $0.8 million in depreciation.
Net cash used in operating activities was $160.9 million for the year ended December 31, 2024, which was driven by a net loss of $187.4 million and $11.4 million net cash used by operating assets and liabilities, partially offset by non-cash charges, including $34.9 million of stock-based compensation expense, $1.8 million in lease expenses and $1.2 million in depreciation.
Additionally, the general consensus among economists suggests that we should expect a higher recession risk to continue over the next year, which, together with the foregoing, could result in further economic uncertainty and volatility in the capital markets in the near term, and could negatively affect our operations. Furthermore, such economic conditions have produced downward pressure on share prices.
Additionally, the general consensus among economists suggests that we should expect a higher recession risk to continue over the next year due in part to ongoing tariff and trade uncertainties, which, together with the foregoing, could result in further economic uncertainty and volatility in the capital markets in the near term, and could negatively affect our operations.
While our significant accounting policies are described in greater detail in Note 2 to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K, we believe that the following accounting policies are those most critical to the judgments and estimates used in the preparation of our consolidated financial statements.
While our significant accounting policies are described in greater detail in Note 2 to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K, we believe that the following accounting policies are those most critical to the judgments and estimates used in the preparation of our consolidated financial statements. 95 Revenue Recognition To date, our revenues have consisted solely of payments received related to research collaborations and licensing of intellectual property.
The increase of $38.4 million was primarily due to an increase in program-related costs, including (i) an $8.8 million increase of cibotercept-related expenses, which was driven by a $7.1 million increase in clinical spend associated with our Phase 2 clinical trial and a $1.7 million increase in manufacturing costs; (ii) a $10.1 million increase in KER-065-related expenses, primarily driven by a net increase of $5.8 million in manufacturing and preclinical activities and an increase of $4.3 million in clinical spend associated with our ongoing Phase 1 clinical trial; (iii) a net increase of $3.1 million of elritercept-related expenses, primarily driven by a $9.7 million increase in clinical spend associated with our ongoing Phase 2 clinical trials, one in 102 patients with MDS and one in patients with myelofibrosis, and the advancement of a Phase 3 clinical trial in patients with MDS, partially offset by a decrease of $6.6 million in manufacturing and preclinical activities; (iv) a $12.5 million increase in personnel costs, including an increase of $4.2 million of additional stock-based compensation costs, driven by the increase in headcount to support the advancement of our pipeline; (v) a $1.8 million increase in professional fees; and (v) a $2.4 million increase in facilities and supplies and other expenses due to the continued growth of our organization.
The decrease of $44.0 million was primarily due to a decrease in program-related costs, including (i) a $9.0 million decrease of rinvatercept-related expenses, which was driven by a net decrease of $7.8 million in manufacturing and preclinical activities and a $1.2 million decrease in clinical spend associated with our completed Phase 1 clinical trial; (ii) a net decrease of $6.3 million in elritercept-related expenses, primarily driven by a decrease of $8.4 million in clinical spend associated with our ongoing Phase 2 clinical trials, one in patients with MDS and 91 one in patients with myelofibrosis, and the advancement of the Phase 3 RENEW clinical trial, as clinical activities transitioned to Takeda during 2025, partially offset by an increase of $2.1 million in manufacturing activities; (iii) a $17.2 million decrease of cibotercept-related expenses, primarily driven by a $9.5 million decrease in clinical spend associated with our terminated Phase 2 clinical trial and a net decrease of $7.7 million in manufacturing and preclinical activities; (iv) a $5.2 million decrease in preclinical pipeline and development activities; and (v) a $8.3 million decrease in personnel costs, including a decrease of $5.0 million of stock-based compensation costs, driven by a reduction in headcount.
Pursuant to the terms of the Takeda Agreement, we received a $200.0 million upfront payment in February 2025. In addition to the upfront payment, we are entitled to receive up to an aggregate of (i) $370.0 million upon the achievement of specified development and commercial milestones and (ii) $740.0 million upon the achievement of specified sales milestones.
In addition to the upfront payment and milestone payment, we are entitled to receive up to an aggregate of (i) $80.0 million upon the achievement of specified development milestones, (ii) $280.0 million upon the achievement of specified commercial milestones and (iii) $740.0 million upon the achievement of specified sales milestones.
We will not generate any revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for one or more of our product candidates. If we obtain regulatory approval for any of our product candidates, we expect to incur significant expenses related to developing our internal commercialization capability to support product sales, marketing and distribution.
If we obtain regulatory approval for any of our product candidates, we expect to incur significant expenses related to developing our internal commercialization capability to support product sales, marketing and distribution.
The $2.7 million of cash used in operating assets and liabilities was primarily comprised of (i) a $9.8 million increase in prepaid expenses and other assets due to timing of expense recognition for our research and development costs and (ii) a $0.1 million increase in accounts receivable, which was partially offset by (a) a $6.9 million increase in accounts payable and accrued expenses to support the advancement of our programs and (b) a $0.4 million change in operating lease liabilities.
The $12.1 million of cash used by operating assets and liabilities was primarily comprised of (i) a $13.1 million decrease in accounts payable and accrued expenses primarily driven by the transition of activities to Takeda and reduction in other clinical spend; (ii) a $2.3 million increase in current income tax receivable; (iii) a $0.8 million increase in accounts receivable; and (iv) a $2.0 million change in operating lease liabilities; partially offset by a $6.1 million decrease in prepaid expenses and other assets due to timing of expense recognition for our research and development costs.
Income Tax Provision Income tax provision was $0.3 million for the year ended December 31, 2024, compared to zero for the years ended December 31, 2023, and 2022, respectively.
Income Tax Provision Income tax provision was $4.9 million for the year ended December 31, 2025, compared to $0.3 million for the year ended December 31, 2024.
The increase of $0.3 million in income tax provision is attributed to withholding taxes related to the taxable income generated in 2024 from the Hansoh Agreement. 103 Liquidity and Capital Resources Since our inception, we have incurred significant operating losses.
The increase of $4.6 million in income tax provision is attributed to taxable income generated from the Takeda Agreement. 92 Liquidity and Capital Resources Since our inception, we have incurred significant operating losses each fiscal year through December 31, 2024.
We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we advance the preclinical activities and clinical trials of our product candidates. Furthermore, we expect to incur additional costs associated with operating as a public company, including significant legal, accounting, investor relations, director and officer insurance premiums and other expenses.
Furthermore, we expect to incur costs associated with operating as a public company, including significant legal, accounting, investor relations, director and officer insurance premiums and other expenses.
By leveraging this understanding, we have discovered and are developing protein therapeutics that have the potential to provide meaningful and potentially disease-modifying benefit to patients. One of our product candidates, cibotercept (KER-012), is being developed for the treatment of pulmonary arterial hypertension, or PAH, and for the treatment of cardiovascular disorders.
By leveraging this understanding, we have discovered and are developing protein therapeutics that have the potential to provide meaningful and potentially disease-modifying benefit to patients. Our lead product candidate, rinvatercept (KER-065), is being developed for the treatment of Duchenne muscular dystrophy and for the treatment of amyotrophic lateral sclerosis.
We recognized $3.0 million as revenue and $0.3 million in withholding tax upon the achievement of a development milestone related to the Hansoh Agreement on our consolidated statement of operations for the year ended December 31, 2024, and a receivable, net of withholding tax, on our consolidated balance sheet as of December 31, 2024. 99 Hansoh’s obligation to pay royalties for a given licensed product in a given region in the Hansoh Territory will begin on the date of the first commercial sale for such licensed product in such region and continue until the latest of (i) ten years from the date of the first commercial sale for such licensed product in such region, (ii) the expiration of the last valid claim of certain licensed patents or joint patents, and (iii) expiration of regulatory exclusivity in such region.
Hansoh’s obligation to pay royalties for a given licensed product in a given region in the Hansoh Territory will begin on the date of the first commercial sale for such licensed product in such region and continue until the latest of (i) ten years from the date of the first commercial sale for such licensed product in such region, (ii) the expiration of the last valid claim of certain licensed patents or joint patents, and (iii) expiration of regulatory exclusivity in such region.
Cash Flows The following table summarizes our cash flows for each of the periods presented (in thousands): YEAR ENDED DECEMBER 31, 2024 2023 2022 Net cash used in operating activities $ (160,869) $ (124,508) $ (70,062) Net cash used in investing activities (1,931) (2,464) (1,241) Net cash provided by financing activities 391,821 178,956 120,309 Net increase in cash and cash equivalents, and restricted cash $ 229,021 $ 51,984 $ 49,006 Cash Used in Operating Activities Net cash used in operating activities was $160.9 million for the year ended December 31, 2024, which was driven by a net loss of $187.4 million and $11.4 million net cash used by operating assets and liabilities, partially offset by non-cash charges including $34.9 million of stock-based compensation expense, $1.8 million in lease expenses and $1.2 million in depreciation.
Cash Flows The following table summarizes our cash flows for each of the periods presented (in thousands): YEAR ENDED DECEMBER 31, 2025 2024 Net cash provided by (used in) operating activities $ 107,505 $ (160,869) Net cash used in investing activities (1,551) (1,931) Net cash provided by (used in) financing activities (378,470) 391,821 Net increase (decrease) in cash and cash equivalents, and restricted cash $ (272,516) $ 229,021 Cash Provided by (Used in) Operating Activities Net cash provided by operating activities was $107.5 million for the year ended December 31, 2025, which was driven by (i) a net income of $87.0 million and (ii) non-cash charges, including $28.7 million of stock-based compensation expense, $2.4 million in lease expenses and $1.5 million in depreciation, partially offset by $12.1 million net cash used by operating assets and liabilities.
To date, we have devoted the majority of our efforts into business planning, research and development of our product candidates, including conducting clinical trials and preclinical studies, raising capital and recruiting management and technical staff to support these operations. Our primary uses of cash are to fund operating expenses, primarily research and development expenditures.
As of December 31, 2025 and December 31, 2024, we had an accumulated deficit of $481.8 million and $568.8 million, respectively. To date, we have devoted the majority of our efforts into business planning, research and development of our product candidates, including conducting clinical trials and preclinical studies, raising capital and recruiting management and technical staff to support these operations.
Cash Used in Investing Activities Net cash used in investing activities was $1.9 million, $2.5 million, and $1.2 million for the years ended December 31, 2024, 2023, and 2022, respectively. The cash used in investing activities in each period was due to purchases of property and equipment.
Cash Used in Investing Activities Net cash used in investing activities was $1.6 million and $1.9 million for the years ended December 31, 2025 and 2024, respectively.
We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect.
Based on our current operating assumptions, we believe that our existing cash and cash equivalents will be sufficient to fund our projected liquidity requirements into the first half of 2028. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect.
The increase of $5.9 million was primarily due to (i) a $3.6 million increase in personnel expenses, which includes an increase of $1.9 million of additional stock-based compensation costs, to support our organizational growth and achievement of our corporate goals; (ii) a net $0.6 million increase in facilities, supplies and other expenses due to growth of our organization; and (iii) a $2.3 million increase in professional fees.
The increase of $6.1 million was primarily due to (i) a $5.9 million increase in professional fees and (ii) a net $0.9 million increase in facilities, supplies and other expenses.
Revenue Recognition To date, our revenues have consisted solely of payments received related to research collaborations and licensing of intellectual property. We apply the revenue recognition guidance in accordance with Financial Accounting Standards Board, Accounting Standards Codification, or ASC, Subtopic 606, Revenue from Contracts with Customers, or ASC 606.
We apply the revenue recognition guidance in accordance with Financial Accounting Standards Board, Accounting Standards Codification, or ASC, Subtopic 606, Revenue from Contracts with Customers, or ASC 606.
Based on our current operating assumptions, we expect that our existing cash and cash equivalents as of December 31, 2024, together with the $200 million upfront payment pursuant to the license agreement with Takeda Pharmaceuticals U.S.A., Inc., or Takeda, which we received in February 2025, will enable us to fund our operating expenses and capital expenditure requirements into 2029.
Based on our current operating assumptions, we expect that our existing cash and cash equivalents as of December 31, 2025 will enable us to fund our operating expenses and capital expenditure requirements into the first half of 2028.
Therefore, we estimate our expected stock volatility based on the historical volatility of a publicly traded set of peer companies and expect to continue to do so until such time as we have adequate historical data regarding the volatility of our own traded stock price. These estimates involve inherent uncertainties and the application of management’s judgment.
Therefore, we estimate our expected stock volatility based on the historical volatility of a group of publicly traded peer companies, weighted with our own historical volatility for the period during which our stock has been publicly traded. These estimates involve inherent uncertainties and the application of management’s judgment.
As of and during the year ended December 31, 2024, we have sold a total of 4,290,096 shares of our common stock pursuant to the ATM Program for aggregate net proceeds of approximately $228.6 million after deducting sales agent commissions and estimated offering expenses.
As of December 31, 2025, we have sold a total of 4,290,096 shares of our common stock pursuant to the ATM Program. As of December 31, 2025, we were not eligible to offer and sell any shares of our common stock under the ATM Program and did not sell any shares during the year ended December 31, 2025.
We anticipate that the filing of this Annual Report on Form 10-K will render us unable to use our currently effective New Shelf Registration Statement as we expect that, on the date of filing of this report, we will no longer meet the criteria of a well-known seasoned issuer.
As of the filing of our Annual Report on Form 10-K for the year ended December 31, 2024, we no longer qualified as a well-known seasoned issuer and therefore were not eligible to use the New Shelf Registration Statement as an automatic shelf registration statement.
Research and Development Expenses The following table summarizes our research and development expenses for the years ended December 31, 2024, 2023, and 2022 (in thousands): YEAR ENDED DECEMBER 31, $ CHANGE 2024 2023 2022 2024 vs 2023 2023 vs 2022 Cibotercept $ 29,777 $ 20,931 $ 12,433 $ 8,846 $ 8,498 KER-065 16,090 5,962 6,010 10,128 (48) Elritercept 44,319 41,249 24,492 3,070 16,757 Preclinical and development fees 12,008 12,420 9,221 (412) 3,199 Personnel expenses (including stock-based compensation) 55,946 43,408 27,683 12,538 15,725 Professional fees 5,083 3,298 3,844 1,785 (546) Facilities and supplies 7,832 6,135 2,546 1,697 3,589 Other expenses 2,574 1,855 1,036 719 819 $ 173,629 $ 135,258 $ 87,265 $ 38,371 $ 47,993 Research and development expenses were $173.6 million for the year ended December 31, 2024, compared to $135.3 million for the year ended December 31, 2023.
Research and Development Expenses The following table summarizes our research and development expenses for the years ended December 31, 2025 and 2024 (in thousands): YEAR ENDED DECEMBER 31, $ CHANGE 2025 2024 2025 vs 2024 Rinvatercept $ 7,081 $ 16,090 $ (9,009) Elritercept 38,030 44,319 (6,289) Cibotercept 12,562 29,777 (17,215) Preclinical and development fees 6,847 12,008 (5,161) Personnel expenses (including stock-based compensation) 47,647 55,946 (8,299) Professional fees 6,474 5,083 1,391 Facilities and supplies 8,947 7,832 1,115 Other expenses 2,055 2,574 (519) $ 129,643 $ 173,629 $ (43,986) Research and development expenses were $129.6 million for the year ended December 31, 2025, compared to $173.6 million for the year ended December 31, 2024.
These increases were partially offset by a $0.6 million decrease in director and officer insurance premiums. General and administrative expenses were $34.8 million for the year ended December 31, 2023, compared to $27.5 million for the year ended December 31, 2022.
These decreases were partially offset by (a) a $1.4 million increase in professional fees and (b) a net increase of $0.6 million in facilities and supplies and other expenses. General and Administrative Expenses General and administrative expenses were $46.8 million for the year ended December 31, 2025, compared to $40.8 million for the year ended December 31, 2024.
These increases were partially offset by a $0.9 million decrease in director and officer insurance premiums. Total Other Income (Expense), Net Total other income (expense), net was $23.8 million for the year ended December 31, 2024, compared to $16.9 million for the year ended December 31, 2023.
Total Other Income, Net Total other income, net was $24.3 million for the year ended December 31, 2025, compared to $23.8 million for the year ended December 31, 2024.
The aggregate net proceeds to us from the public offering were approximately $151.1 million, after deducting underwriting discounts and commissions and estimated offering expenses. We have incurred recurring operating losses since inception in 2015. Our ability to generate product revenue sufficient to achieve profitability will depend on the successful development and commercialization of one or more of our product candidates.
Our ability to generate product revenue sufficient to achieve profitability will depend on the successful development and commercialization of one or more of our product candidates.
Net cash provided by financing activities was $120.3 million for the year ended December 31, 2022, which was primarily related to (i) net proceeds of $119.5 million received from sales of our common stock under the ATM Sales Agreement, after deducting sales agent commissions and before deducting offering expenses; and (ii) proceeds of $0.8 million related to exercises of options to purchase common stock.
The cash used in investing activities in each period was due to purchases of property and equipment. 94 Cash Provided by (Used in) Financing Activities Net cash used in financing activities was $378.5 million for the year ended December 31, 2025, which was primarily related to $375.0 million used to repurchase shares of common stock via the ADAR1 Repurchase Agreement, the Pontifax Repurchase Agreement and the Tender Offer and $3.9 million in cash paid for direct expenses associated with the Repurchase Agreements and the Tender Offer, which was partially offset by (i) proceeds from short-swing profit settlement of $0.1 million, (ii) proceeds of $0.1 million related to issuance of common stock under the employee stock purchase plan; and (iii) proceeds of $0.2 million related to exercises of options to purchase common stock.
Our net losses were $187.4 million, $153.0 million, and $104.7 million for the years ended December 31, 2024, 2023, and 2022, respectively. As of December 31, 2024 and December 31, 2023, we had an accumulated deficit of $568.8 million and $381.4 million, respectively.
Our net income, which was primarily driven by revenue related to the Takeda Agreement, was $87.0 million for the year ended December 31, 2025 compared to a net loss of $187.4 million for the year ended December 31, 2024. As of December 31, 2025, we had an accumulated deficit of $481.8 million.
Our revenue for the year ended December 31, 2023 consisted of service and other revenue related to the Tech Transfer Agreement. We did not recognize any revenue for the year ended December 31, 2022.
In connection with the Hansoh Agreement, we recognized $0.2 million of service and other revenue for the year ended December 31, 2025, compared to $3.0 million of license revenue and $0.5 million of service and other revenue for the year ended December 31, 2024.
Removed
As of December 31, 2024, we may offer and sell ATM shares at an aggregate offering price of up to the remaining $117.7 million available under the ATM Offering.
Added
In December 2024, we entered into an exclusive license agreement with Takeda Pharmaceuticals U.S.A., Inc., or Takeda, which became effective on January 16, 2025, to further develop, manufacture and commercialize elritercept worldwide outside of mainland China, Hong Kong and Macau.
Removed
Our net loss was $187.4 million, $153.0 million, and $104.7 million for the years ended December 31, 2024, 2023, and 2022, respectively. As of December 31, 2024, we had an accumulated deficit of $568.8 million. We expect to continue to generate operating losses and negative operating cash flows for the foreseeable future in connection with our ongoing activities.
Added
As of the filing of our Annual Report on Form 10-K for the year ended December 31, 2024, we no longer qualified as a well-known seasoned issuer and therefore were not eligible to use the New Shelf Registration Statement as an automatic shelf registration statement, and no shares were sold during the year ended December 31, 2025.
Removed
These increases were partially offset by a $0.4 million decrease in preclinical pipeline and development activities. Research and development expenses were $135.3 million for the year ended December 31, 2023, compared to $87.3 million for the year ended December 31, 2022.
Added
The aggregate net proceeds to us from the public offering were approximately $151.1 million, after deducting underwriting discounts and commissions and estimated offering expenses. 86 October 2025 Share Repurchases On October 15, 2025, we entered into the Repurchase Agreements with the ADAR1 Parties and the Pontifax Parties.
Removed
The increase of $48.0 million was primarily due to an increase in program-related costs, including (i) an $8.5 million increase of cibotercept-related expenses, which was driven by a $6.8 million increase in activities to support the clinical advancement of the program and a $1.7 million increase in manufacturing costs and preclinical activities; (ii) a net increase of $16.8 million of elritercept-related expenses, primarily driven by (a) a $5.5 million increase in clinical and preclinical program activities due to the progression of our two Phase 2 clinical trials of elritercept, one in patients with MDS and one in patients with myelofibrosis, and the initial expenses associated with our planned advancement of elritercept into a Phase 3 clinical trial in patients with MDS and (b) an increase of $11.3 million in manufacturing activities; (iii) a $3.2 million increase in preclinical pipeline and development activities; (iv) a $15.7 million increase in personnel costs, including an increase of $5.9 million of additional stock-based compensation costs, driven by the increase in headcount to support the advancement of our pipeline; and (v) a $4.4 million increase in facilities and supplies and other expenses due to the continued growth of our organization.
Added
Pursuant to the terms and conditions of the Repurchase Agreements, the ADAR1 Parties and the Pontifax Parties sold all of the shares of our common stock beneficially owned by them, being an aggregate of 10,176,595 shares of common stock, to us at a per share purchase price of $17.75 per share, for an aggregate purchase price of $180.6 million.
Removed
These increases were partially offset by a $0.5 million decrease in professional fees.
Added
In addition, concurrently with the execution of the Pontifax Repurchase Agreement, each of Tomer Kariv and Ran Nussbaum resigned from our board of directors and all committees thereof.
Removed
We are no longer separately disclosing KER-047-related expenses in 2024 due to our decision to deprioritize the KER-047 program in 2023, and have updated prior period research and development expense tables to include KER-047-related expenses in preclinical expenses in order to provide a meaningful comparison of the year-over-year expenses.
Added
Pursuant to the Repurchase Agreements, each of the ADAR1 Parties and the Pontifax Parties agreed to customary standstill restrictions and voting commitments, which will remain in effect until immediately following the final certification of the voting results for our 2028 annual stockholder meeting.
Removed
We expect research and development expenses to fluctuate from quarter to quarter depending on the timing of clinical trial activities, clinical manufacturing and other development activities. General and Administrative Expenses General and administrative expenses were $40.8 million for the year ended December 31, 2024, compared to $34.8 million for the year ended December 31, 2023.
Added
We and each of the ADAR1 Parties and the Pontifax Parties also agreed to customary mutual non-disparagement obligations to remain in effect during the same period.
Removed
The increase of $7.3 million was primarily due to (i) a $6.0 million increase in personnel expenses, which includes an increase of $4.2 million of additional stock-based compensation costs, to support our organizational growth and achievement of our corporate goals; (ii) a $1.5 million increase in facilities, supplies and other office expenses due to growth of our organization; and (iii) a $0.7 million increase in professional fees.
Added
November 2025 Issuer Tender Offer On October 20, 2025, we announced that our board of directors authorized the Tender Offer, which we launched on October 20, 2025 and completed on November 21, 2025 On November 20, 2025, we disclosed that a total of 17,712,262 shares of our common stock were validly tendered and not validly withdrawn.
Removed
Total other income (expense), net was $16.9 million for the year ended December 31, 2023, compared to $10.1 million for the year ended December 31, 2022. The increase of $6.8 million is primarily related to an increase of $11.1 million of dividend income, partially offset by a decrease of $4.7 million in R&D Incentive income in Australia.
Added
In accordance with the terms and conditions of the Tender Offer, we accepted for purchase a total of 10,950,165 Tender Offer Shares at a purchase price of $17.75 per share, for an aggregate purchase price of approximately $194.4 million.
Removed
As of December 31, 2024, we were eligible to offer and sell, from time to time, shares of our common stock for an aggregate offering amount of up to the remaining $117.7 million available under the ATM Program.
Added
As a result of the Tender Offer, we had 19,543,706 shares of common stock outstanding immediately following the closing of the Tender Offer. Since our inception, we have incurred recurring operating losses each fiscal year through December 31, 2024.
Removed
Accordingly, we will need to file a post-effective amendment to the New Shelf Registration Statement to convert it to a non-automatic shelf registration statement that we are eligible to use, or to file a new shelf registration statement on Form S-3.
Added
We expect to continue to generate operating losses and negative operating cash flows for the foreseeable future in connection with our ongoing activities. We will not generate any revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for one or more of our product candidates.
Removed
Such post-effective amendment or shelf registration statement on Form S-3 is subject to review by the SEC and must be declared effective by the SEC, which could delay our ability to raise debt or equity capital under the registration statement and may adversely affect our ability to access financing and the capital markets in a timely fashion.
Added
Furthermore, such economic conditions have produced downward pressure on share prices.

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Item 7A. Quantitative and Qualitative Disclosures About Market Risk

Market Risk — interest-rate, FX, commodity exposure

0 edited+1 added−5 removed1 unchanged
Removed
ITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK We are exposed to market risks in the ordinary course of our business. Market risk represents the risk of loss that may impact our financial position due to adverse changes in financial market prices and rates. Our market risk exposure is primarily the result of interest rate sensitivities.
Added
ITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK We are a "”smaller reporting company,” as defined in Item 10(f)(1) of Regulation S-K. As a result, pursuant to Item 305(e) of Regulation S-K, we are not required to provide the information required by this Item 7A. 97 ITEM 8.
Removed
Interest Rate Sensitivity As of December 31, 2024 and December 31, 2023, we had cash and cash equivalents of $559.9 million and $331.1 million, respectively. Our exposure to interest rate sensitivity is impacted by changes in the underlying U.S. bank interest rates.
Removed
Our surplus cash has been invested in money market fund accounts as well as interest-bearing savings accounts from time to time. We have not entered into investments for trading or speculative purposes.
Removed
Due to the conservative nature of our investment portfolio, which is predicated on capital preservation of investments with short-term maturities, we do not believe an immediate one percentage point change in interest rates would have a material effect on the fair market value of our portfolio, and therefore we do not expect our operating results or cash flows to be significantly affected by changes in market interest rates.
Removed
As of December 31, 2024 and 2023, we had no debt outstanding that is subject to interest rate variability. Therefore, we are not subject to interest rate risk related to debt. 108 ITEM 8.

Other KROS 10-K year-over-year comparisons