Myelofibrosis Pipeline Expands with Over 40 Candidates from 35+ Companies

The myelofibrosis therapeutic landscape is experiencing significant expansion, with more than 35 companies developing over 40 pipeline candidates across clinical and preclinical stages. A comprehensive pipeline analysis reveals a diverse range of investigational assets targeting this rare blood cancer, which has an annual incidence of 1-3 new cases per 100,000 people and an estimated U.S. prevalence of approximately 25,000 patients.

Recent developments highlight substantial clinical trial activity. In February 2026, Prelude Therapeutics announced a Phase 1 study of PRT12396, an open-label, multi-center safety and efficacy study in patients with high-risk polycythemia vera and intermediate and high-risk myelofibrosis. The primary endpoints include safety, efficacy, and pharmacokinetic profile.

In January 2026, Cellenkos Inc. received U.S. Food and Drug Administration Orphan Drug Designation for its investigational product CK0804 for myelofibrosis treatment. The company is developing allogeneic, off-the-shelf, regulatory T cell therapies for autoimmune and inflammatory diseases.

Several key clinical trial milestones were reached in late 2025 and early 2026. Karyopharm Therapeutics Inc. completed patient enrollment in its Phase III SENTRY trial assessing selinexor in combination with ruxolitinib for JAK inhibitor–naïve myelofibrosis patients in September 2025. The company also launched a global, multicenter, two-part study to evaluate the safety and efficacy of selinexor in combination with ruxolitinib in JAK inhibitor–naïve myelofibrosis patients, including an open-label Phase I component to determine safety and recommended dosing, followed by a randomized, double-blind Phase III evaluation.

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. The company also collaborated with QIAGEN in June 2025 to develop a novel diagnostic panel to support Incyte's broad investigational portfolio for myeloproliferative neoplasms, including the monoclonal antibody candidate INCA033989.

Multiple companies initiated clinical studies in January 2025. Geron Corporation began a clinical study 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. Ryvu Therapeutics initiated a clinical trial 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.

AbbVie launched the TRANSFORM-2 trial, a randomized, open-label Phase III study comparing navitoclax plus ruxolitinib versus best available therapy in patients with relapsed or refractory myelofibrosis. Celgene initiated a Phase III study 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.

Keros Therapeutics is developing KER-050, an investigational therapeutic protein intended 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 and Myelofibrosis.

Stemline Therapeutics conducted a multicenter, multi-arm clinical trial assessing the safety and efficacy of tagraxofusp, a CD123-targeted therapy, in patients with chronic myelomonocytic leukemia or myelofibrosis. The pipeline evaluation covers 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.