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Disease

Myelofibrosis

Late-stage therapeutic development
19
Clinical trials
12
Associated genes
5
Related proteins
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

A Phase I Open Label Peptide Based Vaccine in Patients With Myeloproliferative Neoplasm Harboring CALR Mutations

Clinical trial2026-09-28Status changed to Active, not recruiting · ClinicalTrials.gov

CHMP positive opinion: Ruxolitinib Zentiva (EMA)

Regulatory2026-09-18EMA

An Open-Label Clinical Trial of RVU120 as Monotherapy and in Combination With Ruxolitinib in Patients With Intermediate or High-Risk, Primary or Secondary Myelofibrosis (POTAMI-61)

Clinical trial2026-09-16Status changed to Terminated · ClinicalTrials.gov

CHMP positive opinion: Ruxolitinib Viatris (EMA)

Regulatory2026-07-20EMA

Haploidentical Allogeneic Peripheral Blood Transplantation: Clinical Trial and Laboratory Correlates Examining Checkpoint Immune Regulators' Expression

Clinical trial2026-07-17Status changed to Completed · ClinicalTrials.gov

A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK Inhibitor Therapy and Who Require Red Blood Cell Transfusions

Clinical trial2026-07-16Results posted · ClinicalTrials.gov

Phase I Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelofibrosis and Chronic Myelomonocytic Leukemia

Clinical trial2026-07-16Status changed to Active, not recruiting · ClinicalTrials.gov

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 4 clinical trials expected to report results, the earliest in Q4 2027.
  • 2 industry developments reported.
Upcoming Milestones
Major developments
Upcoming trial readoutHigh impact
A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive
Results expected Q2 20282026-10-09
Upcoming trial readoutHigh impact
A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy
Results expected Q4 20292026-10-01
CHMP positive opinion (EU decision pending)Important
CHMP positive opinion — Ruxolitinib
Treatment of myelofibrosis (MF) in adults, treatment of polycythaemia vera (PV) in adults and treatment of Graft versus host disease (GvHD) in adults and children2026-09-18
Clinical Milestones8View
Results posted 2
Results expected 4
Recently completed 1
Other updates 1
Industry & Market2View
Regulatory Updates2View
Activity timeline12

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Ruxolitinibapproved

Generic approval — Myelofibrosis (MF)  Ruxolitinib Viatris is indicated for the treatment of disease-… (2026)

Momelotinib dihydrochloride monohydrateapproved

Original application approval — Omjjara is indicated for the treatment of disease-related splenomegaly or symptoms in adu… (2024)

Clinical trials

14 sponsors · 2 new · 1 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
15
Late-stage
6
Completed
Late-stage studies

A Multicenter, Open-Label, Extension Study Evaluating the Safety and Efficacy of Bomedemstat for the Treatment of Participants Enrolled in a Prior Bomedemstat Clinical Study

Phase 3 · Recruiting · Merck Sharp & Dohme LLC

A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy

Phase 3 · Recruiting · Takeda

A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive

Phase 3 · Recruiting · Novartis Pharmaceuticals

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus (HOPE-PMF): The Core Study and Its Extension Study

Phase 3 · Active, not recruiting · PharmaEssentia

A Phase 3, Randomized, Double-blind, Add-on Study Evaluating the Safety and Efficacy of Navtemadlin Plus Ruxolitinib vs Placebo Plus Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib

Phase 3 · Recruiting · Kartos Therapeutics, Inc.

A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK Inhibitor Therapy and Who Require Red Blood Cell Transfusions

Phase 3 · Active, not recruiting · Celgene
Recruiting

A Multicenter, Open-Label, Extension Study Evaluating the Safety and Efficacy of Bomedemstat for the Treatment of Participants Enrolled in a Prior Bomedemstat Clinical Study

Phase 3 · Recruiting · Merck Sharp & Dohme LLC

A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy

Phase 3 · Recruiting · Takeda

A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive

Phase 3 · Recruiting · Novartis Pharmaceuticals

A Phase 3, Randomized, Double-blind, Add-on Study Evaluating the Safety and Efficacy of Navtemadlin Plus Ruxolitinib vs Placebo Plus Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib

Phase 3 · Recruiting · Kartos Therapeutics, Inc.
Recently completed

Evaluation of Ruxolitinib and Azacytidine Combination as a Therapy for Patients With Myelofibrosis and Myelodysplastic Syndrome/ Myeloproliferative Neoplasm

Phase 2 · Completed · M.D. Anderson Cancer Center

A Randomized, Double-blind, Double-simulated, Parallel-controlled, Multicenter Phase III Study Evaluating the Efficacy and Safety of Jaktinib Versus Hydroxycarbamide in Patients With Intermediate-2 or High-risk Myelofibrosis

Phase 3 · Completed · Suzhou Zelgen Biopharmaceuticals Co.,Ltd

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study Of Navitoclax In Combination With Ruxolitinib Versus Ruxolitinib In Subjects With Myelofibrosis (TRANSFORM-1)

Phase 3 · Completed · AbbVie

A Phase 3, Multicenter, Open-label, Randomized Study to Evaluate the Efficacy and Safety of Fedratinib Compared to Best Available Therapy (BAT) in Subjects With DIPSS (Dynamic International Prognostic Scoring System)-Intermediate or High-risk Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (Post-PV MF), or Post-essential Thrombocythemia Myelofibrosis (Post-ET MF) and Previously Treated With Ruxolitinib

Phase 3 · Completed · Celgene

Eltrombopag for the Management of Thrombocytopenia Associated With Tyrosine Kinase Therapy in Patients With Chronic Myeloid Leukemia (CML) and Myelofibrosis (MF)

Phase 2/3 · Completed · M.D. Anderson Cancer Center

A Randomized, Double-blind, Phase 3 Study to Evaluate the Activity of Momelotinib (MMB) Versus Danazol (DAN) in Symptomatic, Anemic Subjects With Primary Myelofibrosis (PMF), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis Who Were Previously Treated With JAK Inhibitor Therapy

Phase 3 · Completed · Sierra Oncology LLC - a GSK company

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

Product approvals and authorisations
2026emaGeneric approval — Ruxolitinib· Myelofibrosis (MF)  Ruxolitinib Viatris is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.  Polycythaemia vera (PV)  Ruxolitinib Viatris is indicated for the treatment of adult patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea.  Graft versus host disease (GvHD)   Acute GvHD Ruxolitinib Viatris is indicated for the treatment of adults and paediatric patients aged 28 days and older with acute graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1). Chronic GvHD Ruxolitinib Viatris is indicated for the treatment of adults and paediatric patients aged 6 months and older with chronic graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1). source ↗
2024emaOriginal application approval — Momelotinib dihydrochloride monohydrate· Omjjara is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. source ↗
2012emaOriginal application approval — Ruxolitinib· Myelofibrosis (MF)Jakavi is indicated for the treatment of disease related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.Polycythaemia vera (PV)Jakavi is indicated for the treatment of adult patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea.Graft versus host disease (GvHD)Acute GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 28 days and older with acute graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1).Chronic GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 6 months and older with chronic graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1). Myelofibrosis (MF)Jakavi is indicated for the treatment of disease related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.Polycythaemia vera (PV)Jakavi is indicated for the treatment of adult patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea.Graft versus host disease (GvHD)Acute GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 28 days and older with acute graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1).Chronic GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 6 months and older with chronic graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1). source ↗
Other regulatory activity
2026emaCHMP positive opinion — Ruxolitinib· Treatment of myelofibrosis (MF) in adults, treatment of polycythaemia vera (PV) in adults and treatment of Graft versus host disease (GvHD) in adults and children source ↗
2026emaCHMP positive opinion — Ruxolitinib· Treatment of myelofibrosis (MF), polycythaemia vera (PV) and Graft versus host disease (GvHD). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

JAK20.74FLT30.62JAK30.61JAK10.59ACVR10.56IRAK10.49U2AF10.37ZRSR20.37BCL2L10.37BRD20.36FBXW120.06MID20.05

Disease biology

5 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Tyrosine-protein kinase JAK2via JAK20.74Receptor-type tyrosine-protein kinase FLT3via FLT30.62Tyrosine-protein kinase JAK3via JAK30.61Tyrosine-protein kinase JAK1via JAK10.59Bcl-2-like protein 1via BCL2L10.37
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.