Myelofibrosis Clinical Trials Outlook: Competitive Intelligence, Regulatory Progress, Emerging Therapies, Mechanisms of Action, and Administration Pathways – DelveInsight

Myelofibrosis Clinical Trials

DelveInsight’s latest report, “Myelofibrosis Pipeline Insight” delivers an extensive analysis of the evolving myelofibrosis therapeutic landscape, profiling more than 35 companies and 40+ pipeline candidates. The report provides detailed coverage of investigational assets spanning both clinical and preclinical development stages. It evaluates therapies across multiple parameters, including product category, development phase, route of administration, and molecular class, while also identifying discontinued and inactive pipeline programs within this space.

The Myelofibrosis Pipeline Insight offers a thorough clinical and commercial evaluation of drug candidates from early discovery through marketed stages. The report presents in-depth drug profiles covering mechanisms of action, ongoing and completed clinical studies, regulatory filings and approvals (where applicable), and development activities such as technology platforms, strategic collaborations, mergers and acquisitions, funding initiatives, special regulatory designations, and other critical product-related developments.

Explore the latest advancements shaping myelofibrosis research and development. Learn more about the pipeline here: Myelofibrosis Clinical Trials

Key Highlights from the Myelofibrosis Pipeline Report

• February 2026, Prelude Therapeutics announced a Phase 1 study of PRT12396 is an open-label, multi-center, safety and efficacy study in patients with high-risk polycythemia vera (PV) and intermediate and high-risk myelofibrosis (MF). The primary endpoints of the study include safety, efficacy and PK profile
• January 2026, Cellenkos® Inc., a clinical-stage biotechnology company developing allogeneic, off-the-shelf, regulatory T cell (Treg) therapies for autoimmune and inflammatory diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its investigational product, CK0804, for treatment of myelofibrosis, a rare blood cancer with an annual incidence of 1-3 new cases per 100,000 people per year and an estimated U.S. prevalence of approximately of 25,000 patients.
• September 2025: Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage oncology-focused biopharmaceutical company, announced the completion of patient enrollment in its Phase III SENTRY trial assessing selinexor in combination with ruxolitinib for JAK inhibitor–naïve myelofibrosis patients.
• July 2025: Incyte reported that Phase I clinical data evaluating its investigational therapy as monotherapy and in combination with ruxolitinib in myelofibrosis patients are expected in the latter half of 2025.
• June 2025: QIAGEN and Incyte revealed a global collaboration aimed at developing a novel diagnostic panel to support Incyte’s broad investigational portfolio for myeloproliferative neoplasms (MPNs), including the monoclonal antibody candidate INCA033989.
• January 2025 – Keros Therapeutics: KER-050, an investigational therapeutic protein, is being developed to enhance red blood cell and platelet production by inhibiting signaling from specific members of the TGF-β protein family. The therapy is intended for the treatment of cytopenias—such as anemia and thrombocytopenia—in patients with Myelodysplastic Syndromes (MDS) and Myelofibrosis (MF).
• January 2025 – Geron Corporation: A clinical study was initiated to assess overall survival in intermediate-2 and high-risk myelofibrosis patients treated with imetelstat compared with best available therapy following relapse or refractoriness to JAK inhibitor treatment.
• January 2025 – Karyopharm Therapeutics: A global, multicenter, two-part study was launched to evaluate the safety and efficacy of selinexor in combination with ruxolitinib in JAK inhibitor–naïve myelofibrosis patients. The trial includes an open-label Phase I component (now completed) to determine safety and recommended dosing, followed by a randomized, double-blind Phase III evaluation.
• January 2025 – Ryvu Therapeutics: A clinical trial was initiated to examine the efficacy, safety, pharmacokinetics, and pharmacodynamics of RVU120 in adult patients with intermediate- or high-risk primary or secondary myelofibrosis, administered either as monotherapy or in combination with ruxolitinib.
• January 2025 – AbbVie: The TRANSFORM-2 trial, a randomized, open-label Phase III study, was launched to compare navitoclax plus ruxolitinib versus best available therapy in patients with relapsed or refractory myelofibrosis.
• January 2025 – Celgene: A Phase III study was initiated to evaluate the efficacy and safety of luspatercept versus placebo in patients with MPN-associated myelofibrosis and anemia who are receiving concomitant JAK2 inhibitor therapy and require red blood cell transfusions.
• January 2025 – Stemline Therapeutics: A multicenter, multi-arm clinical trial assessed the safety and efficacy of tagraxofusp, a CD123-targeted therapy, in patients with chronic myelomonocytic leukemia (CMML) or myelofibrosis, including both treatment-naïve and previously treated cohorts with poor prognostic features.
• DelveInsight’s analysis highlights a dynamic and competitive myelofibrosis pipeline, with 35+ active companies advancing over 40 therapeutic candidates.
• Key companies operating in the myelofibrosis space include Incyte, Keros Therapeutics, Karyopharm Therapeutics, Celgene, PharmaEssentia, Novartis, SecuraBio, Pharmaxis, AstraZeneca, Cellenkos, Jacobio Pharmaceuticals, Geron Corporation, AbbVie, Sierra Oncology, Galecto Biotech, Kartos Therapeutics, Menarini Group, Telios Pharma, MorphoSys, Roche, and others.
• The myelofibrosis pipeline features notable therapies such as BESREMi, INCB057643, XPOVIO, RYTELO, REBLOZYL, Navtemadlin, Pelabresib, Bomedemstat, TL-895, RVU120, TP-3654, SNT-5505, DISC-0974, ELZONRIS, among several others.

Stay up to date with the latest innovations transforming myelofibrosis care. Access pipeline updates here: Myelofibrosis Pipeline Outlook

Myelofibrosis Pipeline Therapy Assessment

Imetelstat – Geron Corporation

Imetelstat sodium is a lipid-conjugated oligonucleotide engineered for enhanced stability, target binding affinity, and cellular uptake. Its modified nucleic acid backbone improves resistance to enzymatic degradation, while the lipid moiety enhances permeability and pharmacological activity. The drug exhibits prolonged retention in bone marrow, spleen, and liver tissues. Imetelstat directly inhibits telomerase by binding to the RNA template region, resulting in competitive suppression of enzymatic activity rather than antisense-mediated protein inhibition. The therapy is currently being evaluated in Phase III clinical trials for myelofibrosis.

Bomedemstat – Merck

Bomedemstat is a potent, irreversible inhibitor of lysine-specific demethylase 1 (LSD1), a key regulator of hematopoietic stem cell proliferation and progenitor differentiation. The agent is presently in Phase III development for the treatment of myelofibrosis.

TL-895 – Telios Pharma

TL-895 is an orally administered tyrosine kinase inhibitor with anti-inflammatory and anti-cancer properties. By inhibiting tyrosine kinase signaling, the therapy may suppress tumor angiogenesis, cellular proliferation, and immune-driven inflammatory responses. TL-895 is currently in Phase II clinical development for myelofibrosis.

RVU120 – Ryvu Therapeutics

RVU120 is a first-in-class, selective dual CDK8/19 kinase inhibitor. Its mechanism of action involves modulation of transcriptional regulation pathways, with translational data supporting efficacy in molecularly defined patient subsets harboring DNMT3A and NPM1 mutations. The candidate is currently in Phase II clinical trials for myelofibrosis.

TBX-2400 – Taiga Biotechnologies

TBX-2400 is an allogeneic stem cell therapy designed to enhance immune system reconstitution and engraftment following allogeneic hematopoietic stem cell transplantation. The therapy is in early Phase I development for the treatment of myelofibrosis.

Learn more about emerging opportunities in myelofibrosis drug development: Myelofibrosis Drugs and Therapies

Key Companies in the Myelofibrosis Landscape

Major companies active in myelofibrosis research include Incyte Corporation, Keros Therapeutics, Karyopharm Therapeutics, Celgene, PharmaEssentia, Novartis, SecuraBio, Pharmaxis, AstraZeneca, Cellenkos, Jacobio Pharmaceuticals, Geron Corporation, AbbVie, Sierra Oncology, Galecto Biotech, Kartos Therapeutics, Menarini Group, Telios Pharma, MorphoSys, Roche, and others.

Pipeline Analysis by Route of Administration

Myelofibrosis therapies are classified based on administration routes, including:

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Pipeline Classification by Molecule Type

The report categorizes pipeline assets across several molecular formats, such as:

• Recombinant fusion proteins
• Small molecules
• Monoclonal antibodies
• Peptides
• Polymers
• Gene therapies

Discover how DelveInsight is shaping the future of myelofibrosis treatment innovation: Myelofibrosis Companies and FDA Approvals

Scope of the Myelofibrosis Pipeline Report

• Geographic Coverage: Global
• Key Companies Covered: Incyte, Keros Therapeutics, Karyopharm Therapeutics, Celgene, PharmaEssentia, Novartis, AstraZeneca, Geron, AbbVie, Kartos Therapeutics, Menarini Group, MorphoSys, Roche, and others
• Key Therapies Analyzed: BESREMi, INCB057643, XPOVIO, RYTELO, REBLOZYL, Navtemadlin, Pelabresib, Bomedemstat, TL-895, RVU120, TP-3654, SNT-5505, DISC-0974, ELZONRIS, among others

• Therapeutic Assessment by Product Type: Monotherapy, Combination Therapy, Mono/Combination
• Therapeutic Assessment by Development Stage: Discovery, Preclinical, Phase I, Phase II, Phase III

For an in-depth view of ongoing research, emerging drugs, and future development strategies, explore the full Myelofibrosis Pipeline report here: Myelofibrosis Emerging Drugs Analysis

About DelveInsight

DelveInsight is a leading market research and business consulting firm focused exclusively on the life sciences industry. With expertise spanning market assessments, pipeline evaluations, competitive intelligence, and strategic consulting, DelveInsight empowers pharmaceutical companies, biotech innovators, and investors with actionable insights to make informed business decisions.

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