Myelofibrosis - Pipeline Insight, 2025: Novel Disease-Modifying Therapies Target the Fibrotic and Cytokine Drivers of Disease Progression | DelveInsight

Myelofibrosis, a rare and chronic myeloproliferative neoplasm, is characterized by bone marrow fibrosis, abnormal blood cell production, and splenomegaly. Historically treated with symptom-focused therapies, the treatment landscape for myelofibrosis is now undergoing a significant transformation as disease-modifying approaches gain traction.

DelveInsight's "Myelofibrosis - Pipeline Insight, 2025" highlights over 40+ active pipeline candidates targeting various pathogenic mechanisms, including JAK-STAT signaling, fibrosis pathways, anemia management, and the malignant stem cell clone. Prominent therapeutic classes in development include JAK inhibitors, BET inhibitors, telomerase inhibitors, anti-fibrotic agents, and novel immunomodulators.

Key biopharma innovators such as iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, and others are advancing differentiated therapies-many in combination with approved JAK inhibitors to enhance efficacy and manage cytopenias.

The report delivers comprehensive insights into late-stage trials, combination regimens, anemia-targeting strategies, and emerging biomarkers. With improved understanding of the fibrotic niche and clonal evolution, next-gen therapies aim not only to control symptoms but to alter the course of disease.

This shift signals a paradigm change in myelofibrosis care-from palliative symptom relief toward potential disease modification and prolonged survival for patients.

Interested in learning more about the current treatment landscape and the key drivers shaping the myelofibrosis pipeline? Click here: https://www.delveinsight.com/report-store/myelofibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Myelofibrosis Pipeline Report
• DelveInsight's myelofibrosis pipeline analysis depicts a strong space with 35+ active players working to develop 40+ pipeline drugs for myelofibrosis treatment.
• The leading myelofibrosis companies include Geron Corporation, Merck, Telios Pharma, Ryvu Therapeutics, Taiga Biotechnologies, Morphic Therapeutics, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, and others are evaluating their lead assets to improve the myelofibrosis treatment landscape.
• Key myelofibrosis pipeline therapies in various stages of development include Imetelstat, Bomedemstat, TL-895, RVU120, TBX-2400, MORF-088, IOA-244, PXS-5505, NS-018, Tasquinimod, Parsaclisib, TP-3654, and others.
• In June 2025, Sumitomo Pharma America received FDA Fast Track designation for nuvisertib (TP-3654), an oral PIM1 kinase inhibitor for intermediate/high-risk myelofibrosis, based on positive Phase 1/2 data.
• In June 2025, Syntara Limited was granted FDA Fast Track designation for SNT-5505, aimed at myelofibrosis patients with inadequate response to JAK inhibitors.
• In May 2025, the FDA approved belzutifan for patients aged 12 and older with von Hippel-Lindau disease-associated tumors, including myelofibrosis lesions.

Request a sample and discover the recent breakthroughs happening in the myelofibrosis pipeline landscape at https://www.delveinsight.com/report-store/myelofibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Myelofibrosis Overview
Myelofibrosis (MF) is a rare type of chronic myeloproliferative neoplasm that affects the bone marrow, leading to excessive scar tissue (fibrosis), impaired blood cell production, and enlargement of the spleen (splenomegaly). It can occur de novo (primary myelofibrosis) or as a progression from other blood cancers like polycythemia vera (PV) or essential thrombocythemia (ET).

The condition is primarily driven by mutations in JAK2, CALR, or MPL genes, which lead to dysregulated JAK-STAT signaling and abnormal hematopoiesis. Symptoms commonly include fatigue, anemia, night sweats, bone pain, and early satiety due to an enlarged spleen.

Myelofibrosis can be asymptomatic in early stages, but often progresses to severe cytopenias or acute myeloid leukemia (AML) in advanced disease. Diagnosis is based on bone marrow biopsy, genetic testing, and clinical presentation.

Current treatment options aim to relieve symptoms and slow disease progression. These include JAK inhibitors (like ruxolitinib and fedratinib), blood transfusions, and stem cell transplantation for eligible patients. However, curative options remain limited, making myelofibrosis a major focus for ongoing clinical research.

Find out more about myelofibrosis medication at https://www.delveinsight.com/report-store/myelofibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Myelofibrosis Treatment Analysis: Drug Profile
Imetelstat: Geron Corporation
Imetelstat sodium is an investigational small oligonucleotide comprised of a proprietary nucleic acid backbone and a lipid moiety. The unique structure enhances resistance to degradation and improves binding affinity to its target. The lipid component increases cell permeability, resulting in higher potency and improved pharmacokinetics. Imetelstat works by binding with high affinity to the RNA component of telomerase, directly inhibiting its enzymatic activity. It shows prolonged retention in bone marrow, liver, and spleen. Currently, Imetelstat is in Phase III clinical trials for the treatment of myelofibrosis.

Bomedemstat: Merck
Bomedemstat is an oral, irreversible inhibitor of lysine-specific demethylase 1 (LSD1), a key regulator of hematopoietic stem cell proliferation and differentiation. By targeting LSD1, the drug aims to reduce abnormal myeloid cell growth and fibrosis in the bone marrow. Bomedemstat is currently being evaluated in Phase III trials for patients with myelofibrosis.

RVU120: Ryvu Therapeutics SA
RVU120 is a first-in-class, selective dual CDK8/19 kinase inhibitor developed by Ryvu Therapeutics. It modulates transcriptional regulation in hematologic malignancies, and preclinical studies suggest strong activity in patients with DNMT3A and NPM1 mutations. RVU120 is currently in Phase II development for the treatment of myelofibrosis.

DISC-0974: Disc Medicine
DISC-0974 is an investigational monoclonal antibody targeting hemojuvelin (HJV) to suppress hepcidin production and enhance iron availability, addressing anemia of inflammation seen in myelofibrosis. Originally in-licensed from AbbVie, DISC-0974 is currently in Phase I/II trials for the treatment of anemia associated with myelofibrosis.

Learn more about the novel and emerging myelofibrosis pipeline therapies at https://www.delveinsight.com/report-store/myelofibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Myelofibrosis Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

By Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Scope of the Myelofibrosis Pipeline Report
• Coverage: Global
• Key Myelofibrosis Companies: Geron Corporation, Merck, Telios Pharma, Ryvu Therapeutics, Taiga Biotechnologies, Morphic Therapeutics, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, and others.
• Key Myelofibrosis Pipeline Therapies: Imetelstat, Bomedemstat, TL-895, RVU120, TBX-2400, MORF-088, IOA-244, PXS-5505, NS-018, Tasquinimod, Parsaclisib, TP-3654, and others.

Dive deep into rich insights for drugs used for myelofibrosis treatment, visit: https://www.delveinsight.com/report-store/myelofibrosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Myelofibrosis Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Myelofibrosis Pipeline Therapeutics
6. Myelofibrosis Pipeline: Late-Stage Products (Phase III)
7. Myelofibrosis Pipeline: Mid-Stage Products (Phase II)
8. Myelofibrosis Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

About DelveInsight
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This release was published on openPR.