Polycythemia Vera Pipeline: 10+ Therapies in Development by Pharma Giants Poised to Transform the Polycythemia Vera Treatment Landscape | DelveInsight

The Polycythemia Vera (PV) treatment landscape is undergoing significant evolution, driven by leading biopharmaceutical companies such as Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, and Perseus Proteomics. With JAK inhibitors like ruxolitinib already established, the focus has now shifted to next-generation therapeutics targeting disease-modifying mechanisms, cytokine signaling pathways, and novel erythropoiesis regulation. These therapies aim to reduce thrombotic risk, alleviate symptom burden, and delay progression to myelofibrosis.

DelveInsight's "Polycythemia Vera - Pipeline Insight, 2025" offers a detailed review of the clinical development landscape, covering investigational drugs from preclinical to late-stage clinical trials. The report examines the therapeutic potential of each candidate, development status, mechanisms of action, and future market prospects.

This pipeline analysis provides an in-depth 360° view of polycythemia vera drug development trends based on molecule type, route of administration, and stage of development. It highlights unmet needs, strategic collaborations, licensing agreements, and potential market disruptors poised to reshape the PV space.

Key Takeaways from the Polycythemia Vera Pipeline Report
• DelveInsight's polycythemia vera pipeline analysis depicts a strong space with 8+ active players working to develop 10+ pipeline drugs for polycythemia vera treatment.
• The leading polycythemia vera companies include Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals, and others are evaluating their lead assets to improve the polycythemia vera treatment landscape.
• Key polycythemia vera pipeline therapies in various stages of development include SLN124, Sapablursen, PTG-300, KRT-232, DISC-3405, and others.
• In March 2025, Takeda and its partner Protagonist Therapeutics announced a Phase III success for rusfertide, which met the primary endpoint in a study involving patients with polycythemia vera (PV). The companies plan to use the results from the VERIFY trial to support marketing applications for the hepcidin mimetic peptide therapeutic.
• In December 2024, Vanda Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor, for the treatment of polycythemia vera (PV), a rare hematologic malignancy that leads to the overproduction of red blood cells by the bone marrow.

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Polycythemia Vera Overview
Polycythemia vera (PV) is a type of myeloproliferative neoplasm marked by the excessive production of red blood cells, often accompanied by elevated white blood cell and platelet counts. This overproduction stems from abnormal clonal hematopoietic stem cells, which are hypersensitive to growth factors. A key complication of PV is increased blood viscosity, which raises the risk of thrombotic events.

The underlying pathophysiology of PV is closely linked to the JAK2V617F mutation, present in over 90% of PV cases. This mutation causes constant activation of cytokine signaling pathways, promoting unregulated blood cell production and panmyelosis. It also contributes to the suppression of normal stem cell function within the bone marrow. The mutation is also observed in a significant portion of other related disorders, such as primary myelofibrosis and essential thrombocythemia.

Although PV has no known cure, current treatment strategies aim to minimize complications and control symptoms. First-line management includes phlebotomy to reduce red cell mass and blood thickness. Pharmacological options like hydroxyurea are used to suppress bone marrow activity, while low-dose aspirin is employed to reduce clotting risks. For high-risk or treatment-resistant cases, JAK2 inhibitors like ruxolitinib are recommended. Ruxolitinib sales have seen significant growth due to its effectiveness in treating PV, especially in patients who are resistant to other therapies.

In addition to medical therapy, lifestyle interventions play a vital role in PV management. Patients are encouraged to stay well-hydrated, exercise regularly, avoid smoking, and manage cardiovascular risk factors to help mitigate the disease's complications.

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Polycythemia Vera Treatment Analysis: Drug Profile
Rusfertide (PTG-300): Protagonist Therapeutics
Rusfertide (PTG-300) is an injectable agent designed to mimic the effects of the natural hormone hepcidin, offering enhanced potency, solubility, and stability. Hepcidin is essential for regulating iron balance and is crucial for proper red blood cell development. It exerts its effects by binding to the transmembrane protein ferroportin, which regulates the export of iron from macrophages, liver hepatocytes, and duodenal enterocytes into the bloodstream. By inhibiting ferroportin, hepcidin reduces iron release into circulation, ensuring proper iron distribution. PTG-300, as a hepcidin mimetic, aims to redistribute iron to macrophages, decrease iron-induced oxidative stress in the bone marrow, and support effective red blood cell production. Additionally, by limiting iron release into the bloodstream, PTG-300 may help prevent organ damage due to excessive iron, particularly in the liver and heart. Protagonist Therapeutics and Takeda have partnered under a global licensing and collaboration agreement for the development and commercialization of rusfertide. The drug is currently in Phase III trials for treating Polycythemia Vera.

Givinostat: Italfarmaco
Givinostat, developed by Italfarmaco, is a histone deacetylase (HDAC) inhibitor under investigation for the treatment of Polycythemia Vera (PV), a myeloproliferative disorder associated with abnormal blood cell production driven by JAK2 mutations, particularly the JAK2 V617F mutation. The drug is currently in Phase III clinical trials for PV treatment.

Sapablursen: Ionis Pharmaceuticals
Sapablursen, also known as ISIS 702843 or IONIS-TMPRSS6-LRx, is an investigational RNA-targeted therapy developed by Ionis Pharmaceuticals for Polycythemia Vera (PV). The drug works by inhibiting the TMPRSS6 protein, which regulates hepcidin, a hormone crucial for iron homeostasis. By increasing hepcidin levels, sapablursen aims to reduce the symptoms of PV and decrease the need for frequent phlebotomy treatments. Sapablursen is currently in Phase II clinical development for PV.

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Polycythemia Vera 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 Polycythemia Vera Pipeline Report
• Coverage: Global
• Key Polycythemia Vera Companies: Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals, and others.
• Key Polycythemia Vera Pipeline Therapies: SLN124, Sapablursen, PTG-300, KRT-232, DISC-3405, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Polycythemia Vera Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Polycythemia Vera Pipeline Therapeutics
6. Polycythemia Vera Pipeline: Late-Stage Products (Phase III)
7. Polycythemia Vera Pipeline: Mid-Stage Products (Phase II)
8. Polycythemia Vera 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

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This release was published on openPR.