A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW)

This study (KER-050-D301) is evaluating the efficacy and safety of elritercept (KER-050) versus placebo in adult participants with transfusion-dependent anemia with very low, low, or intermediate risk MDS, or more recently defined as myelodysplastic neoplasms, with or without ring sideroblasts. The study is divided into the Screening Period, Double-blind Treatment Period, Safety Follow-Up Period and Long-term Follow-up Period. Approximately 255 participants will be enrolled, randomized 2:1 to receive either elritercept or placebo.

Start Date06 May 2025

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

[+1]

CTR20242732

/ RecruitingPhase 2

HS-20106在IPSS-R极低危、低危或中危骨髓增生异常综合征（MDS）贫血受试者中的有效性、安全性和药代动力学的II期临床研究

[Translation] A Phase II clinical study of the efficacy, safety and pharmacokinetics of HS-20106 in subjects with anemia in myelodysplastic syndrome (MDS) at very low, low or intermediate risk on IPSS-R

评估HS-20106皮下注射（SC）给药治疗在IPSS-R极低危、低危或中危MDS导致贫血的受试者中的有效性。

[Translation]

To evaluate the efficacy of HS-20106 administered subcutaneously (SC) in subjects with anemia due to MDS who were at very low, low, or intermediate IPSS-R risk.

Start Date14 Nov 2024

Sponsor / Collaborator

Shanghai Hansoh Biomedical Co. Ltd.

[+2]

NCT05037760

/ RecruitingPhase 2

A Phase 2 Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of KER-050 as Monotherapy or in Combination With Ruxolitinib in Participants With Myelofibrosis

This is a Phase 2, multicenter, open-label study to evaluate the safety and efficacy of KER-050 as monotherapy or in combination with ruxolitinib in participants with Myelofibrosis.

Start Date16 Dec 2021

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

100 Clinical Results associated with Elritercept

100 Translational Medicine associated with Elritercept

100 Patents (Medical) associated with Elritercept

Literatures (Medical) associated with Elritercept

01 Dec 2025·Current Hematologic Malignancy Reports

Emerging Therapeutic Approaches for Anemia in Myelofibrosis

Review

Author: Harrington, Patrick ; Palumbo, Giuseppe A ; Chifotides, Helen T ; Duminuco, Andrea ; Torre, Elena ; Bose, Prithviraj ; Vetro, Calogero

PURPOSE OF REVIEW:

In this review, we highlight conventional agents and novel emerging therapeutic strategies to treat anemia in MF.

RECENT FINDINGS:

Anemia is a common and challenging feature of myelofibrosis (MF). The pathobiology of anemia is multifactorial, including progressive bone marrow fibrosis, decreased erythropoiesis due to high hepcidin levels leading to iron sequestration in the reticuloendothelial system, hypersplenism, erythropoiesis inhibition by myelosuppressive JAK inhibitors (ruxolitinib, fedratinib), and others. MF-associated anemia has a negative impact on survival. Conventional agents to manage anemia include erythropoiesis-stimulating agents, danazol, corticosteroids, and immunomodulatory agents, but responses are infrequent and lack durability. Notable advancements have emerged in developing novel treatments for anemia in MF, including the regulatory approval of momelotinib (ACVR1/JAK1/2 inhibitor) in 2023 and development of novel promising agents targeting hemojuvelin and activins. Momelotinib and pacritinib (ACVR1/JAK2 inhibitor) are the preferred JAK inhibitors for patients with cytopenias (anemia, thrombocytopenia). Luspatercept and elritercept are activin receptor ligand traps, promoting erythroid maturation and late-stage erythropoiesis. Currently, luspatercept is being evaluated in a phase 3 trial (INDEPENDENCE™) for anemia in MF patients who are on a JAK2 inhibitor and require transfusions, and in a phase 2 trial (ODYSSEY) in combination with momelotinib in MF patients who are transfusion dependent, whether or not on a JAK inhibitor. Interim results of the RESTORE trial demonstrated that elritercept significantly decreased transfusions in MF patients. DISC-0974 is a first-in-class anti-hemojuvelin (positive hepcidin regulator) monoclonal antibody that decreased hepcidin expression, increased serum iron, and enhanced erythropoiesis in anemic patients with MF in a phase 1b/2 study. Burgeoning studies of novel anemia-targeted agents and combinations are significantly improving the quality of life and outcomes of patients with MF. The recent approval of momelotinib to treat MF with anemia and the emerging novel anemia-directed strategies in early and advanced clinical development have ushered in a new era in the treatment of MF-related anemia.

01 Jun 2025·AMERICAN JOURNAL OF HEMATOLOGY

Emerging Pathogenetic Mechanisms and New Drugs for Anemia in Myelofibrosis and Myelodysplastic Syndromes

Review

Author: Tefferi, Ayalew ; Gangat, Naseema

ABSTRACT:

Anemia in myeloid neoplasms is multifaceted, with heterogeneous pathogenetic mechanisms that include ineffective erythropoiesis, hepcidin‐induced iron‐restricted erythropoiesis, and abnormal inflammatory cytokine production. Current management of anemia is challenged by limited approved drugs that specifically treat anemia in myelofibrosis (MF) and myelodysplastic syndrome (MDS). Newer therapies target the transforming growth factor beta (TGF‐β)‐bone morphogenic protein/sons of mothers against decapentaplegic (BMP‐SMAD) signaling pathway, which plays a significant role in ineffective erythropoiesis (SMAD 2/3) and abnormal hepcidin production (SMAD 1/5/8). These include TGF‐β ligand traps (luspatercept, elritercept), activin A receptor type 1 (ACVR1)/activin receptor‐like kinase 2 (ALK2) inhibitors (momelotinib, zilurgisertib), and anti‐hemojuvelin antibody‐based therapies (DISC‐0974). Luspatercept and momelotinib are approved for anemia related to lower‐risk MDS and MF, respectively, and represent an important addition to the treatment armamentarium, along with imetelstat, a telomerase inhibitor, recently ratified for anemia in lower‐risk MDS. A promising strategy to overcome the limitations of existing anemia‐directed therapies includes the use of drug combinations with complementary mechanisms (luspatercept + erythropoiesis stimulating agents, luspatercept + momelotinib, DISC‐0974 + momelotinib), and harnessing the erythropoietic potential of sodium‐glucose co‐transporter‐2 inhibitors (SGLT‐2I). Future research should address the complex pathophysiology of anemia, standardize definitions for anemia with gender‐specified cutoffs, implement uniform erythroid response criteria, and consider early therapeutic intervention in clinical trials for anemia‐directed therapies.

01 Mar 2025·HEMATOLOGICAL ONCOLOGY

Managing Myelofibrosis: Matching Advances in Treatments With Clinical Unmet Needs

Review

Author: Chan, Tze Wei ; Than, Hein ; Billa, Gauri ; Huang, Feng‐Ju ; Lee, Lai Heng

ABSTRACT:

Myelofibrosis (MF) is characterized by anemia, constitutional symptoms, hepatosplenomegaly and bone marrow fibrosis, and is associated with poor survival. The janus kinase inhibitor (JAKi) ruxolitinib has been the mainstay of treatment for over a decade. Despite demonstrated symptomatic and quality of life improvement, unmet clinical needs persist. A literature review identified promising novel targeted treatment options in MF using pre‐set selection criteria (available Phase 2 or 3 data, minimum enrollment of 50 patients, trial end date within the last 5 years). Available data for novel and approved therapies were extracted, tabulated, and analyzed for clinical relevancy. From an initial shortlist of 48, 16 retained molecules were selected for inclusion. Other JAKi (pacritinib, momelotinib, jaktinib) address treatment‐related cytopenia, expanding the therapeutic utility of this class of agents to patients with baseline anemia or thrombocytopenia. Novel candidates exploit multiple molecular pathways, and offer the potential to improve the management of MF‐associated cytopenia (imetelstat, pelabresib, navitoclax, selinexor, luspatercept, sotatercept, elritercept, LCL161, bomedemstat) and recover bone marrow fibrosis (imetelstat, pelabresib, navitoclax and bomedemstat). It remains to be seen if these newer agents can induce any remission in MF and enable patients to come off therapy, but the future is beginning to look much brighter.

News (Medical) associated with Elritercept

08 Sep 2025

·www.globenewswire.com

Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting

LEXINGTON, Mass., Sept. 08, 2025 (GLOBE NEWSWIRE) -- 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 that it presented additional clinical data from its Phase 1 clinical trial of KER-065 in healthy male volunteers at the American Society of Bone and Mineral Research 2025 Annual Meeting on Saturday, September 6, 2025. “We are pleased to present additional data that highlights the broad therapeutic potential of KER-065, including its robust bone anabolic activity,” said Jasbir S. Seehra, President and Chief Executive Officer. “These data continue to demonstrate the potential of KER-065 in Duchenne muscular dystrophy, where bone loss occurs as a result of the progressive muscle weakness and chronic use of corticosteroids.” Clinical Presentation Phase 1 Trial in Healthy Participants of KER-065, a Modified Activin Receptor Ligand Trap, Supports Development in Duchenne Muscular Dystrophy and Bone Disorders This Phase 1 clinical trial was a randomized, double-blind, placebo-controlled, two-part dose escalation (single and multiple ascending dose) trial in healthy male 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. Initial topline data from this trial was reported in March 2025. As of the final data cut-off date of April 29, 2025, treatment with KER-065 was generally well-tolerated at all dose levels tested. No dose-limiting toxicities or serious adverse events were reported. One grade 4 treatment-emergent adverse event of elevated creatine kinase levels was observed, but deemed unrelated to treatment. The majority of the adverse events that were observed were mild to moderate in severity and resolved. Additional results from this trial, as of the data cut-off date, demonstrated that treatment with KER-065 resulted in: Changes in bone biomarkers of increased bone formation and reduced bone resorption that were consistent with tissue level changes, as demonstrated by observed increases in bone mineral density (“BMD”)Whole body BMD improvements at Day 85 that were sustained through Day 141 (three months after the last dose in the multiple ascending dose cohorts), suggesting a balance between osteoblast and osteoclast activityIncreased lumbar spine BMD following three doses that were sustained through Day 141 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. Keros’ lead product candidate, KER-065, is being developed for the treatment of neuromuscular diseases, with an initial focus on DMD. Keros’ most advanced product candidate, elritercept, is being developed for the treatment of cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndrome and in patients with myelofibrosis. Cautionary Note Regarding Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Words such as “anticipates,” “believes,” “continue,” “expects,” “enable,” “intention,” “potential” and “will” or similar expressions are intended to identify forward-looking statements. Examples of these forward-looking statements include statements concerning: Keros’ expectations regarding its strategy, progress and timing of its clinical trials for KER-065; and the broad therapeutic potential of KER-065. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Keros’ limited operating history and historical losses; Keros’ ability to raise additional funding to complete the development and any commercialization of its product candidates; Keros’ dependence on the success of its product candidates, KER-065 and elritercept; that Keros may be delayed in initiating, enrolling or completing any clinical trials; competition from third parties that are developing products for similar uses; Keros’ ability to obtain, maintain and protect its intellectual property; and Keros’ dependence on third parties in connection with manufacturing, clinical trials and preclinical studies. These and other risks are described more fully in Keros’ filings with the Securities and Exchange Commission (the “SEC”), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q, filed with the SEC on August 6, 2025, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Keros undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Contacts Investor Contact:Justin Frantzjfrantz@kerostx.com617-221-6042 Media Contact:Mahmoud Siddig / Adam Pollack / Viveca TressJoele Frank, Wilkinson Brimmer Katcher(212) 355-4449

Clinical ResultPhase 1

03 Sep 2025

·www.prnewswire.com

Myelofibrosis Market is Going to Grow at a CAGR of 9% in the Next Ten Years (2025-2034) | DelveInsight

The launch of emerging therapies like BESREMi (PharmaEssentia and AOP Orphan Pharmaceuticals), INCB057643 (Incyte), XPOVIO (Karyopharm Therapeutics), RYTELO (Geron), REBLOZYL (Bristol Myers Squibb), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), and others is going to shift the myelofibrosis market. LAS VEGAS, Sept. 3, 2025 /PRNewswire/ -- DelveInsight's Myelofibrosis Market Insights report includes a comprehensive understanding of current treatment practices, emerging myelofibrosis drugs, market share of individual therapies, and current and forecasted myelofibrosis market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan). Myelofibrosis Market Summary The total myelofibrosis treatment market size in the leading markets (the US, EU4, UK, and Japan) was USD 2.2 billion in 2024. The United States accounts for the largest market size of myelofibrosis, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. Based on DelveInsight's assessment in 2024, the 7MM had approximately 56K patient pool (prevalent cases) of myelofibrosis. Key myelofibrosis companies, including PharmaEssentia, AOP Orphan Pharmaceuticals, Incyte, Karyopharm Therapeutics, Geron, Bristol Myers Squibb, Kartos Therapeutics, Novartis, Merck, Telios Pharma, Ryvu Therapeutics, Sumitomo Pharma, Syntara, Disc Medicine, Menarini Group, and others, are actively working on innovative myelofibrosis drugs. Some of the key myelofibrosis therapies in clinical trials include BESREMi (ropeginterferon alfa-2b-njft/P-1101), INCB057643, XPOVIO (NEXPOVIO/selinexor/KPT-330), RYTELO (imetelstat), REBLOZYL (luspatercept/ACE-536), Navtemadlin (KRT-232), Pelabresib (DAK539), Bomedemstat (IMG-7289/MK-3543), TL-895, RVU120 ( SEL-120), TP-3654 (nuvisertib), SNT-5505 (PXS-5505), DISC-0974, ELZONRIS (tagraxofusp/SL-401), and others. These novel myelofibrosis therapies are anticipated to enter the myelofibrosis market in the forecast period and are expected to change the market. By 2034, among all the therapies, the highest revenue is expected to be generated by OJJAARA/OMJJARA. Discover which myelofibrosis therapies are expected to grab the largest market share @ Myelofibrosis Market Report Key Factors Driving the Growth of the Myelofibrosis Market Novel Therapies Drive Myelofibrosis Treatment Advancements Currently, four JAK inhibitors have been approved by the US FDA for the treatment of myelofibrosis, including JAKAFI (ruxolitinib), INREBIC (fedratinib), VONJO (pacritinib), and OJJAARA (momelotinib). No drug therapy can cure myelofibrosis. As myelofibrosis primarily affects older adults, stem cell transplantation is not a treatment option for most myelofibrosis patients. The launch of another JAK inhibitor, BESREMi, will further change the dynamics of the myelofibrosis market. Rich clinical pipeline and emerging non-JAK approaches driving the myelofibrosis landscape Beyond JAK inhibitors, active clinical development includes BET inhibitors (Incyte's INCB057643; Novartis' Pelabresib), XPO1 inhibitor (Karyopharm Therapeutics' XPOVIO), Telomerase inhibitor (Geron's RYTELO), MDM2 protein inhibitor (Kartos Therapeutics' Navtemadlin), Tyrosine kinase inhibitors (Telios Pharma's TL-895), PIM1 kinase inhibitor (Sumitomo Pharma's TP-3654), LOX inhibitor (Syntara's SNT-5505), and others, raising market potential by promising new label expansions, second-line options, and higher-value therapies should any prove to be disease-modifying. An aging population & increasing incidence with better survival Myelofibrosis primarily affects older adults; population aging increases the absolute number of patients. As treatments improve symptoms and (in some cases) survival, prevalent patient pools grow, supporting longer-term market demand. In the US in 2024, the 70+ years of age group had the highest number of cases, accounting for approximately 60% of the total prevalent cases, while the ≤39 years of age group accounted for just ~2%. Diagnostic & genomic testing improvements are driving myelofibrosis patient pool, leading to the growth of myelofibrosis market Wider use of molecular profiling (JAK2, CALR, MPL, and others) enables earlier and more accurate MF diagnosis and better patient stratification for targeted therapies. This both expands the diagnosed population and helps match patients to appropriate, often higher-value, treatments. Myelofibrosis Market Analysis JAK inhibitors have become the cornerstone of treatment for patients with myelofibrosis, offering significant benefits such as spleen reduction, symptom relief, and improved quality of life, which can also extend survival in those with advanced disease. All approved JAK inhibitors primarily target JAK2, particularly the wild-type form, but they differ in their activity against other JAK family members. For instance, JAKAFI myelofibrosis inhibits both JAK1 and JAK2; INREBIC selectively inhibits JAK2 while sparing JAK1 and also affects FLT3 and other targets; VONJO myelofibrosis inhibits JAK2 while sparing JAK1 but additionally impacts FLT3, IRAK, and ACVR1; and OJJAARA, approved through a different pathway, targets JAK1/JAK2 and ACVR1, mainly for myelofibrosis patients with anemia. These varying mechanisms lead to distinct patient outcomes. JAKAFI myelofibrosis continues to see strong demand and is expected to grow further, maintaining its position as the standard of care in myelofibrosis. Myelofibrosis will remain the largest segment of JAKAFI's patient population until polycythemia vera cases eventually increase. However, market growth may be limited due to patent expirations of key therapies, with JAKAFI patents set to expire in 2027 for Novartis and 2028 for Incyte, presenting potential opportunities for competitors. In response, Incyte is exploring combination trials with novel drugs to extend JAKAFI's therapeutic lifespan. Learn more about the treatment options for myelofibrosis @ Myelofibrosis Therapy The myelofibrosis treatment landscape offers significant opportunities for innovation, focusing on several key areas: Treating Lower-Risk Patients: Developing safe and effective therapies for patients with lower-risk myelofibrosis, allowing for earlier and more favorable interventions. Enhancing First-Line Treatments: Improving existing first-line therapies for intermediate- to high-risk patients through novel drugs or combination strategies. Managing Cytopenia: Developing targeted therapies to address cytopenia, a significant unmet need in patient care. Myelofibrosis Competitive Landscape The myelofibrosis clinical trial pipeline includes several drugs in mid- and late-stage development that are expected to enter the market during the forecast period. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including XPOVIO (Karyopharm Therapeutics), Elitracet (Takeda/Keros Therapeutics), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), INCB057643 (Incyte), Bomedemstat (Merck), and others, all of which are used in various lines of treatment. The expected launch of these therapies is expected to have a further positive impact on the market. INCB57643 is an orally administered small molecule. Bromodomain and extra-terminal (BET) proteins act as epigenetic readers that control the expression of key oncoproteins implicated in the development of myelofibrosis and other hematologic malignancies, including B-lymphoma-2, nuclear factor kappa, and c-Myc. In a prior Phase I/II clinical trial, the oral BET inhibitor INCB057643, tested both as a monotherapy and in combination with ruxolitinib, demonstrated favorable tolerability and promising clinical activity in patients with advanced cancers. In its Q2 2025 financial report, the company announced that the combination of INCB057643 with ruxolitinib and INCB57643 (a JAK1/JAK2 and BET inhibitor) is currently being evaluated in a Phase II trial for myelofibrosis. RYTELO (imetelstat) is an investigational telomerase inhibitor that targets telomerase, selectively eliminating malignant stem and progenitor cells in the bone marrow, which drive diseases like myelodysplastic syndromes (MDS) and myelofibrosis. By blocking the proliferation of these malignant cells, imetelstat supports the recovery of healthy bone marrow and blood cell production and has shown disease-modifying effects and clinical benefits in Phase III trials for myelofibrosis. This mechanism sets imetelstat apart from other approved or investigational therapies for these blood cancers. In January 2025, Geron reported achieving 75% enrollment in the Phase III IMpactMF trial, which is comparing imetelstat to Best Available Therapy (BAT) in intermediate-2 or high-risk myelofibrosis patients who have relapsed or are refractory to JAK inhibitor treatment. Elritercept is a late-stage investigational activin inhibitor aimed at treating anemia associated with hematologic malignancies, including MDS and myelofibrosis. It is currently undergoing Phase II evaluation in patients with myelofibrosis. In December 2024, Keros Therapeutics presented updated data from this ongoing Phase II trial at ASH 2024. Additionally, in December 2024, Takeda announced an exclusive licensing agreement with Keros to further develop, manufacture, and commercialize elritercept globally, excluding mainland China, Hong Kong, and Macau. The anticipated launch of these emerging myelofibrosis therapies are poised to transform the myelofibrosis market landscape in the coming years. As these cutting-edge myelofibrosis therapies continue to mature and gain regulatory approval, they are expected to reshape the myelofibrosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new myelofibrosis treatment, visit @ Myelofibrosis Treatment Market Recent Developments in the Myelofibrosis Market In July 2025, Incyte announced that the Phase I data in patients with myelofibrosis as monotherapy and in combination with ruxolitinib are anticipated in the second half of 2025. In June 2025, QIAGEN and Incyte announced a new global collaboration to develop a novel diagnostic panel to support Incyte's extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), including Incyte's monoclonal antibody INCA033989. In January 2025, Karyopharm Therapeutics stated that it expects to report top-line results from the Phase III SENTRY trial in the second half of 2025, which could represent a potentially transformative opportunity to establish a new treatment paradigm in myelofibrosis. What is Myelofibrosis? Myelofibrosis is a rare blood cancer marked by the accumulation of scar tissue, or 'fibrosis', in the bone marrow. This excess scar tissue prevents the bone marrow from producing enough healthy blood cells. It belongs to a group of blood cancers called 'myeloproliferative neoplasms (MPNs)', in which the blood cells produced by the bone marrow grow and function abnormally. When myelofibrosis arises independently, without being caused by another bone marrow disorder, it is referred to as primary myelofibrosis. In other cases, it can develop from another MPN, such as polycythemia vera or essential thrombocythemia. When this occurs, it is called secondary myelofibrosis, sometimes specifically termed post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Myelofibrosis Epidemiology Segmentation The myelofibrosis epidemiology section provides insights into the historical and current myelofibrosis patient pool and forecasted trends for the leading markets (the US, EU4, UK, and Japan). It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The myelofibrosis market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets (the US, EU4, UK, and Japan) segmented into: Total Prevalent Cases of Myelofibrosis Type-specific Cases of Myelofibrosis Myelofibrosis Cases Based on Risk Stratification Age-specific Prevalent Cases of Myelofibrosis Myelofibrosis Cases Based on Molecular Alterations Download the report to understand which factors are driving myelofibrosis epidemiology trends @ Myelofibrosis Treatment Drugs Scope of the Myelofibrosis Market Report Myelofibrosis Therapeutic Assessment: Myelofibrosis current marketed and emerging therapies Myelofibrosis Market Dynamics: Conjoint Analysis of Emerging Myelofibrosis Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Myelofibrosis Market Unmet Needs, KOL's views, Analyst's views, Myelofibrosis Market Access and Reimbursement Discover more about myelofibrosis drugs in development @ Myelofibrosis Clinical Trials Table of Contents Related Reports JAK Inhibitors Market JAK Inhibitors Market Size, Target Population, Competitive Landscape, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key JAK inhibitors companies, including Pfizer, AbbVie, Galapagos, Sierra Oncology, Theravance Biopharma, Dizal Pharmaceutical, Aclaris Therapeutics, Celon Pharma, Incyte Corporation, Gilead Sciences, Reistone Biopharma, Jiangsu Hengrui Medicine Co., MaxiNovel Pharmaceuticals, among others. Myelofibrosis Clinical Trial Analysis Myelofibrosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myelofibrosis companies, including Geron Corporation, Merck, Ryvu Therapeutics SA, Morphic Therapeutic, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, Inc., Cellenkos, Disc Medicine, among others. Polycythemia Vera Market Polycythemia Vera Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key polycythemia vera companies, including Incyte, Novartis, PharmaEssentia, AOP Orphan Pharmaceuticals, Protagonist Therapeutics, Merck (Imago BioSciences), Italfarmaco, Ionis Pharmaceutical, Silence Therapeutics, Perseus Proteomics, AbbVie, Johnson & Johnson Innovative Medicine, Mabwell (Shanghai) Bioscience, Disc Medicine, GluBio Therapeutics, among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., 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 . Connect with us on LinkedIn | Facebook | Twitter 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

Phase 2License out/inPhase 1Drug ApprovalClinical Result

22 Aug 2025

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Keros Announces U.S. FDA Orphan Drug Designation Granted to KER-065 for the Treatment of Duchenne Muscular Dystrophy

Aug. 20, 2025 -- 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 the U.S. Food and Drug Administration (“FDA”) granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (“DMD”). “Receiving Orphan Drug designation for KER-065 highlights the significant unmet medical need for patients with DMD,” said Jasbir S. Seehra, President and Chief Executive Officer. “This designation serves as a significant milestone for Keros as we advance KER-065 into a Phase 2 clinical trial in patients with DMD.” The FDA grants Orphan Drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. This designation provides certain potential benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. 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. 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. 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. Keros’ lead product candidate, KER-065, is being developed for the treatment of neuromuscular diseases, with an initial focus on DMD. Keros’ most advanced product candidate, elritercept, is being developed for the treatment of cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndrome and in patients with myelofibrosis. The content above comes from the network. if any infringement, please contact us to modify.

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Indication	Highest Phase	Country/Location	Organization	Date
Myelodysplastic Syndromes	Phase 3	United States	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Brazil	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Bulgaria	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Germany	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Hungary	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Israel	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Italy	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Lithuania	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Poland	Takeda Pharmaceutical Co., Ltd.	06 May 2025
Myelodysplastic Syndromes	Phase 3	Spain	Takeda Pharmaceutical Co., Ltd.	06 May 2025

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04419649 (PF635, EHA2025)	Phase 2	Myelodysplastic Syndromes non-RS	15	Elritercept	bfyzwnywae(dcnydbylax) = rbphgocvrd ltbknamjkl (wuuzjdaayw, 190 - 1632) View more	Positive	14 May 2025
NCT05037760 (RESTORE, EHA2025)	Phase 2	Myelofibrosis	73	Elritercept monotherapy	diiorxliim(xpqfjqmjzd) = An asymptomatic Hgb increase in one participant required dose reduction per protocol and was deemed a dose-limiting toxicity potdzylosl (matrpszdmv ) View more	Positive	14 May 2025
NCT05037760 (RESTORE, EHA2025)	Phase 2	Myelofibrosis	73	Elritercept + Ruxolitinib combination therapy		Positive	14 May 2025
NCT04419649 (Phase 2 trial, EHA2025)	Phase 2	Myelodysplastic Syndromes	95	Elritercept 3.75-5 mg/kg	wpeoohziqn(trkrfguodi) = Observed differences in FACT-An total score for TI≥24 wks responders vs non-responders were driven by greater improvements for responders in the Anemia subscale (AnS), especially the FACIT-Fatigue component. Improvements were observed across FACIT-Fatigue items including those related to both fatigue experience and impact. Moreover, FACIT-Fatigue scores improved over time in TI≥24 wks responders as did the proportion of participants with improvements meeting or exceeding the MCID bwhpgpdkfd (tlvihjcvwp )	Positive	14 May 2025
PB2774 (EHA2025)	Phase 1/2	Myelodysplastic Syndromes	770	Rigosertib	tvigmesodk(rgyikzpwye) = dnsdfbmdqg eyzsjspvip (daiingeztw, 0.012 - 0.039) View more	Positive	14 May 2025
NCT05037760 (RESTORE, ASH2024) Manual	Phase 2	Myelofibrosis	54	Elritercept monotherapyElritercept monotherapy	jrdommzlvd(pfqrqtzber) = hpuykmpidf nqpsnuwglj (ostzjcluzx ) View more	Positive	09 Dec 2024
NCT05037760 (RESTORE, ASH2024) Manual	Phase 2	Myelofibrosis	54	Elritercept + Ruxolitinib	jrdommzlvd(pfqrqtzber) = altguvzisl nqpsnuwglj (ostzjcluzx ) View more	Positive	09 Dec 2024
NCT04419649 (ASH2024) Manual	Phase 2	myelodysplastic anemia	15	Elritercept (Non-Transfusion Dependent)	xzddpajhbn(seicqjznvg) = yuoqluvmgr ztvdhaqluq (tmrjpuzvxh ) View more	Positive	09 Dec 2024
ASH2024	Not Applicable	Myelodysplastic Syndromes	-	Elritercept 3.75-5 mg/kg	olnnnmkdqc(udgidlzjfz) = svjsqqmpke ikmsprrnbm (yxddniieji ) View more	-	07 Dec 2024
NCT04419649 (Biospace) Manual	Phase 2	Myelodysplastic Syndromes	87	KER-050	ovemvkqebu(umkanhcsox) = ympiktpihl zapcfmkyic (lvuzvwzcmy ) View more	Positive	17 Jun 2024
NCT05037760 (RESTORE, Biospace) Manual	Phase 2	Myelofibrosis	54	elritercept with or without ruxolitinib	fmzgbvzvof(fyztjuplyp) = ktrhsvbtuq duthlzrnoq (uoebcsrxhf ) View more	Positive	17 Jun 2024
NCT05037760 (RESTORE, Biospace) Manual	Phase 2	Myelofibrosis	54	elritercept with or without ruxolitinib (3.0 mg/kg of elritercept or higher in combination with ruxolitinib)	aifaslnmsh(tohzfhaipc) = lzbfnxqvks wbvozucgyw (onfkynrjpy ) View more	Positive	17 Jun 2024

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