Keros Therapeutics, Inc.

LEXINGTON, MA, USA I March 31, 2025 I Keros Therapeutics, Inc. (“Keros”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (“TGF-ß”) family of proteins, today announced initial topline results from the Phase 1 clinical trial of KER-065 in healthy volunteers. Topline results from this ongoing trial are through the multiple ascending dose (“MAD”) treatment period (Day 85). “We are pleased to report topline results that met the key objectives of the Phase 1 clinical trial and provided important insights to inform the development of KER-065 for patients with DMD,” said Jasbir S. Seehra, Ph.D., Chair and Chief Executive Officer. “Considering the limitations of currently available therapies, the need for additional treatments in DMD remains high, and KER-065 has the potential to address multiple aspects of the disease, including across important tissues and underlying genetic deficiencies. Our strong financial foundation enables us to continue advancing our promising pipeline of novel therapeutics, and we look forward to engaging with regulators towards the aim of moving KER-065 to a Phase 2 clinical trial in the first quarter of 2026.” Key findings of this trial as of a February 6, 2025 data cut-off date include the following: “We observed evidence of activin inhibition based on multiple biomarkers and body composition data. These data, coupled with preclinical and mechanistic insights on the pivotal role of the activin pathway in neuromuscular pathobiology, demonstrate the exciting therapeutic potential of KER-065 in DMD and other neuromuscular disorders,” said Yung H. Chyung, M.D., Chief Medical Officer. Keros plans on engaging with regulatory authorities, starting in the third quarter of 2025. Subject to the outcome of these regulatory interactions, Keros expects to initiate a Phase 2 clinical trial of KER-065 in patients with DMD in the first quarter of 2026. Conference Call and Webcast Information Keros will host a conference call and webcast today, March 31, 2025, at 8:00 a.m. Eastern time. The conference call will be webcast live at: https://event.webcasts.com/starthere.jsp?ei=1713593&tp_key=5723864d86 . The live teleconference may be accessed by dialing (877) 407-0309 (domestic) or (201) 389-0853 (international). An archived version of the call will be available in the investors section of the Keros website at http://ir.kerostx.com for 90 days following the conclusion of the call. About the KER-065 Phase 1 Clinical Trial The KER-065 Phase 1 clinical trial is a randomized, double-blind, placebo-controlled, two-part dose escalation (single ascending dose and MAD) trial in healthy volunteers. The primary objectives of this trial were to assess safety, tolerability and pharmacokinetics of KER-065. Exploratory endpoints include assessments of the pharmacodynamic effect on bone, adipose, muscle, cardiac tissue and fibrosis. About KER-065 KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB that is fused to the portion of the human antibody known as the Fc domain. KER-065 is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin A, two ligands that signal through activin receptors, 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 diseases, with an initial focus on DMD. About Duchenne Muscular Dystrophy (DMD) DMD is the most common form of muscular dystrophy and results in muscle degeneration and premature death. DMD results from the lack of functional dystrophin protein that helps promote myofiber stability, caused by a gene mutation. The lack of dystrophin, an important structural component of muscle cells, causes muscle cells to have increased susceptibility to damage and to progressively die. Additionally, the absence of dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and the replacement of muscle with fibrotic and fatty tissue. The replacement of muscle fibers with fatty and fibrotic tissue leads to progressive loss of muscle strength and function leading to immobility and respiratory and cardiac complications. 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. About Keros Therapeutics, Inc. Keros is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the TGF-ß family of proteins. Keros is 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. By leveraging this understanding, Keros has discovered and is developing protein therapeutics that have the potential to provide meaningful and potentially disease-modifying benefit to patients. One of Keros’ product candidates, cibotercept (KER-012), is being developed for the treatment of pulmonary arterial hypertension and for the treatment of cardiovascular disorders. Keros’ second product candidate, KER-065, is being developed for the treatment of neuromuscular diseases. Keros’ 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 syndrome and in patients with myelofibrosis. SOURCE: Keros Therapeutics

Developed by Sanofi, CABLIVI is the first FDA-approved nanobody therapy for this condition. Increasing awareness, improved diagnostic rates, and rising demand for targeted therapies in hematology drive the market growth. With its niche indication and high treatment costs, CABLIVI holds a strong position in the rare disease therapeutics segment. LAS VEGAS, March 19, 2025 /PRNewswire/ -- DelveInsight's " CABLIVI Market Size, Forecast, and Market Insight Report" highlights the details around CABLIVI, a von Willebrand factor (vWF)-directed antibody fragment. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CABLIVI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Ablynx NV's CABLIVI (caplacizumab-yhdp) Overview CABLIVI is an antibody fragment that targets von Willebrand factor (vWF) and is used to treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. It works by binding to the A1-domain of vWF, preventing its interaction with platelets, which helps reduce vWF-mediated platelet adhesion and consumption. CABLIVI is approved in both the US and EU for treating aTTP. In 2024, CABLIVI sales reached €249 million, reflecting a 9.7% increase, driven by a rise in the number of patients identified for suitable treatment in the US, as well as new launches in Europe and other regions. The recommended CABLIVI dosing schedule is as follows: Day 1: Administer an 11 mg intravenous bolus injection at least 15 minutes before plasma exchange, followed by an 11 mg subcutaneous injection after the exchange on the same day. During Plasma Exchange Therapy: Administer 11 mg subcutaneously once daily after each plasma exchange session. After Plasma Exchange Therapy: Continue with 11 mg subcutaneous injections once daily for 30 days after the final plasma exchange. If signs of persistent underlying disease, such as low ADAMTS13 activity, are still present, treatment can be extended for up to 28 additional days. CABLIVI treatment should be discontinued if the patient experiences more than two recurrences of aTTP while on the medication. Learn more about CABLIVI projected market size for thrombocytopenia @ CABLIVI Market Potential Thrombocytopenia is a medical condition where the platelet count in the blood drops below the normal level (<150,000/µL). Common causes include heparin-induced thrombocytopenia, chemotherapy-induced thrombocytopenia, thrombocytopenia linked to chronic liver disease, immune thrombocytopenia (ITP), and thrombotic thrombocytopenic purpura (TTP). Among the 7MM, the United States reported the highest number of thrombocytopenia cases, accounting for about 40% of the total cases in 2023, according to DelveInsight. Treatment for thrombocytopenia varies based on its cause and severity, with the primary goal of preventing bleeding-related complications and improving patient outcomes. Approved therapies for thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Additionally, danaparoid, argatroban, and lepirudin are approved for treating thrombosis in patients with heparin-induced thrombocytopenia. For severe thrombocytopenia related to chronic liver disease, treatment options include platelet transfusion, splenic artery embolization, splenectomy, and transjugular intrahepatic portosystemic stent shunt (TIPSS) placement. The US holds approximately 60% of the thrombocytopenia market share, surpassing the combined market size of the EU4, the UK, and Japan. Market growth is expected to be driven by an increasing patient population, the introduction of new therapies, and deeper market penetration within the 7MM. Discover more about the thrombocytopenia market in detail @ Thrombocytopenia Market Report Emerging Competitors of CABLIVI The potential therapies that can mark a significant change in the thrombocytopenia treatment landscape and give tough competition to CABLIVI include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), PF-06835375 (Pfizer), Povetacicept (Vertex), Cevidoplenib (Genosco/Oscotec), and Efgartigimod (ARGX-113) (Argenx), among others. In December 2024, Sanofi shared encouraging Phase III results at ASH for rilzabrutinib, its investigational oral BTK inhibitor, in patients with ITP. In June 2024, Takeda reported late-breaking Phase IIb data on Mezagitamab, highlighting its potential to revolutionize the treatment of Primary ITP. In Novartis' Q2 2024 update, the company forecasted key trial results: VAYHIT1 (NCT05653349) for first-line therapy, expected in 2026, and VAYHIT2 (NCT05653219) for second-line therapy, anticipated in 2025. Novartis also aims to file for ianalumabin approval in both first- and second-line ITP treatment by 2027 or later. To know more about the number of competing drugs in development, visit @ CABLIVI Market Positioning Compared to Other Drugs Key Milestones of CABLIVI In June 2022, at the 27th Annual European HematologyAssociation (EHA) Congress, Sanofi presented long-term safety and efficacy results from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study of patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial. In February 2019, the FDA approved CABLIVI (caplacizumab-yhdp) injection as the first therapy specifically indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder. It is to be used in combination with plasma exchange and immunosuppressive therapy. In September 2018, the European Commission approved CABLIVI (caplacizumab) for marketing, authorizing its use in treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. CABLIVI is the first therapy specifically designed for aTTP treatment. In July 2017, Ablynx announced a research collaboration and global exclusive licensing agreement with Sanofi, aiming to develop and commercialize Nanobody-based therapies for treating various immune-mediated inflammatory diseases. Discover how CABLIVI is shaping the thrombocytopenia treatment landscape @ CABLIVI FDA Approval CABLIVI Market Dynamics The primary driver of CABLIVI's market growth is the increasing recognition and diagnosis of aTTP, coupled with improved access to specialized treatments. Growing awareness among healthcare providers and advancements in diagnostic capabilities have facilitated early and accurate diagnosis, thereby boosting the demand for targeted therapies like CABLIVI. The drug's ability to prevent microthrombi formation, shorten the duration of plasma exchange, and reduce the risk of relapses has positioned it as a preferred option in the treatment landscape for aTTP. Moreover, Sanofi's robust commercial infrastructure and strategic marketing efforts have further contributed to market penetration and adoption. Despite its therapeutic benefits, CABLIVI faces challenges related to its high cost and reimbursement complexities. The specialized nature of aTTP treatment, combined with the requirement for hospital-based administration and close monitoring, adds to the overall cost burden on healthcare systems and patients. Additionally, competition from alternative therapies, including plasma exchange and immunosuppressive treatments, may limit its market expansion. Regulatory hurdles and the need for long-term safety data could also impact the drug's market trajectory. The CABLIVI market is expected to grow steadily, driven by ongoing clinical research and potential label expansions into related thrombotic disorders. Increased investments in rare disease treatments and orphan drug incentives are likely to support market growth. Moreover, the rise in strategic collaborations and partnerships to improve patient access and streamline reimbursement processes could enhance market uptake. However, balancing pricing pressures with broader patient access will remain a key factor in sustaining long-term market performance. Dive deeper to get more insight into CABLIVI's strengths & weaknesses relative to competitors @ CABLIVI Market Drug Report Table of Contents Related Reports Acquired Thrombotic Thrombocytopenic Purpura Pipeline Acquired Thrombotic Thrombocytopenic Purpura Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acquired thrombotic thrombocytopenic purpura companies, including Sanofi, TargED Biopharmaceuticals, Takeda, Genentech, Alexion Pharmaceuticals, among others. Thrombocytopenia Market Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key thrombocytopenia companies including Sanofi, Principia Biopharma, Baxalta, Takeda, Argenx, Millennium Pharmaceuticals, Biotest, GC Pharma, Genosco (Subsidiary of Oscotec), Rigel Pharmaceuticals, Kissei Pharmaceutical, Shionogi & Co., Ltd, Amgen, Novartis, Zenyaku Kogyo, among others. Thrombocytopenia Pipeline Thrombocytopenia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key thrombocytopenia companies, including Takeda, Argenx, Keros Therapeutics, Principia Biopharma, Momenta Pharmaceuticals, Veralox Therapeutics, Novartis Pharmaceuticals, Pfizer, HUTCHMED, Principia Biopharma, Genosco, UCB, among others. Immune Thrombocytopenia Market Immune Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key immune thrombocytopenia companies including Rigel Pharmaceuticals, Kissei Pharmaceutical, Sobi (Dova Pharmaceuticals), Amgen, Argenx, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Takeda\'s oncology leaders are focused on building off their own \"institutional knowledge.\"\n Takeda’s revamped R&D strategy means the drugmaker’s oncology leaders are prioritizing work in tumors across thoracic, gastrointestinal (GI) and hematology, while pushing the Japanese company’s cell therapy work focus to the back burner.“We\'re not doing this in a prescriptive way that would never consider anything outside of that,” Teresa Bitetti, president of Takeda’s global oncology business unit, told Fierce Biotech. “But we are looking at areas that we\'ve defined because of the white space, in terms of the unmet need, because of where the science is moving and where our strength lies.”The oncology approach meshes with the drugmaker’s new overall strategy, P.K. Morrow, M.D., head of Takeda’s oncology therapeutic area unit, told Fierce in a joint interview.In January, Takeda\'s R&D head Andy Plump, M.D., Ph.D., explained that the pharma had narrowed its focus down from 10 or 12 modalities to just four: small molecules, biologics, antibody-drug conjugates (ADCs) and allogeneic cell therapies.  Of those core priorities, Takeda’s oncology leaders are taking a deep interest in ADCs, small molecules and complex biologics, Morrow said. So, everything but cell therapy.“It\'s unlikely we\'re going to do any cell therapy deals in the near future,” she said about the fourth modality.“Anytime we\'re dealing with gene therapy or cell therapy, there\'s the potential risk for a durable negative effect,” said Morrow, who joined Takeda last year after serving as Crispr Therapeutics’ chief medical officer. “It doesn\'t happen most of the time, but there\'s always the potential, right?”To protect the patient, cell and gene therapies often require extended follow-up, Morrow explained, noting that the component is an important consideration in drug development.   The shift isn’t shocking—Bitetti told Fierce that Takeda wasn’t “actively pursuing” autologous cell therapies at last year’s American Society of Clinical Oncology (ASCO) conference.The sentiment shadows a broader industry trend as cell and gene companies fight to remain relevant. Most recently, Pfizer discontinued hemophilia product Beqvez, emptying its gene therapy portfolio, while bluebird bio sold for just $29 million after having at one time carried a value of $10 billion.Back at Takeda, the team still touts GDX012, also called TAK-012-1501, an allogeneic gamma delta T cell therapy the company gained from buying GammaDelta Therapeutics in 2021. The asset is currently in phase 1/2 testing for adults with acute myeloid leukemia (AML).The team will “continue for now” to pursue early-stage work in cell therapy, Bitetti said.After a wide-ranging $900 million restructure in 2024, Takeda now touts its “most robust late-stage pipeline” in the pharma’s history, consisting of six late-stage programs across 14 studies, Plump told Fierce at the beginning of the year.Developing a more mature pipeline meant that Takeda had to raise its standards, Plump said. Lifting the bar resulted in several programs being left behind, including a midstage CD19 CAR-NK cell therapy in B-cell malignancies. The asset is still being studied in a phase 1 autoimmune disease program.Momentum amid streamlining?  “We did do some streamlining, but I sort of feel like we\'ve come out of the back end with a lot of energy [and] focus,” Bitetti said. “I don\'t feel like we\'ve lost momentum with it.”The restructure included implementing a more rigorous approach when considering benefit and safety thresholds that determine what programs should progress, according to Morrow.    One of those programs is rusfertide, an injectable hepcidin mimetic designed to treat a rare chronic blood cancer called polycythemia vera (PV). Last year, Takeda paid out $300 million to license the asset from Protagonist Therapeutics.Just this month, the partners reported a phase 3 win, finding that rusfertide reduced blood procedures among patients with PV.Takeda’s plans to file for approval with regulatory authorities are currently “on track,” with a submission expected toward the end of this year, according to Bitetti.If rusfertide passes muster with regulators, the hepcidin mimetic peptide could go on to generate $1 billion to $2 billion in peak sales. The candidate is one of six pipeline drugs that Takeda is hoping will collectively bring in between $10 billion and $20 billion, CEO Christophe Weber said during the company’s J.P. Morgan presentation in January.Takeda isn\'t counting out China When asked about geopolitical changes, such as the BIOSECURE Act and President Donald Trump’s “America First Investment Policy,” Bitetti said Takeda isn’t counting out science from China.“One can\'t afford to ignore the innovation that\'s available today coming out of China,” she said. “It\'s not just me-toos that are coming out of China—there\'s some really interesting science.”Bitetti also cited the country’s rapid pace and ability to get the proof of concept very quickly.While she noted that companies still need to be “judicious” given the changing dynamics, she said one should “proceed at your own risk to ignore what\'s happening in China.”Meanwhile, Morrow pointed to the Japanese drugmaker’s successful partnership with Hong Kong-based Hutchmed, in which Takeda paid $400 million upfront for commercial rights outside of China to a colorectal cancer drug called fruquintinib. The oral vascular endothelial growth factor receptors (VEGFR) 1/2/3 tyrosine kinase inhibitor received FDA approval at the end of 2023 and is now marketed as Fruzaqla. Last summer, Takeda also inked a deal worth up to $1.3 billion biobucks that provides the opportunity to license Ascentage Pharma’s olverembatinib, which is being developed for chronic myeloid leukemia (CML), along with other hematological cancers. The Chinese biotech\'s third-generation BCR-ABL tyrosine kinase inhibitor is currently approved in China for certain patients with CML.As for future deals with biotechs, the leaders are looking for validated modalities in disease areas Takeda believes it can make the greatest impact in.“That\'s what you\'ll see in particular in the deals that we\'ve done in the very recent past,” Morrow said, pointing to a December deal with Keros Therapeutics that included $200 million upfront to secure ex-China rights to a molecule that could compete with Bristol Myers Squibb’s Reblozyl in blood cancer indications.The deal covers Keros’ activin inhibitor elritercept that’s in development to treat anemia tied to blood cancers such as myelodysplastic syndromes and myelofibrosis. Keros sees elritercept as a molecule that can act on all stages of platelet and red blood cell production, leading it to identify opportunities across a broad patient population.Building on strengths Takeda’s pipeline decisions also take the company’s strengths into account, Bitetti said.“We have an enormous strength on the [hematology] side,” she explained. “We have very deep relationships that go back a very long time from the early days of Velcade.”The “institutional knowledge” regarding myeloid cancers and commercializing drugs in the area is something Takeda wants to continue to build on.This is exemplified by Takeda’s purchase of oncology biotech Millennium way back in 2008, according to Morrow.The “Millennium legacy” is vital to the oncology unit because it helped Takeda better understand how to develop therapies that could become backbones to other regimens and helped draw in key talent that has expertise in precision medicine. When asked how diversity, equity and inclusion play a role in the department’s hiring practices, Bitetti said DEI considerations continue to be important.“Our position as a company hasn’t changed on diversity, in terms of doing what we think is right for the business and ensuring that we have the right breadth of perspective, not only in our clinical trials, but also how we manage the business,” Bitetti said.