Polycythemia Vera Pipeline Outlook 2025: Clinical Trial Studies, EMA, PDMA, FDA Approvals, MOA, ROA, NDA, IND, And Companies

DelveInsight's, “Polycythemia Vera Pipeline Insight 2025” report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Polycythemia Vera pipeline landscape. It covers the Polycythemia Vera pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Polycythemia Vera therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Polycythemia Vera Pipeline Report

• On 28 August 2025, Celgene announced a Phase 3 study designed to evaluate the efficacy and safety of fedratinib compared to best available therapy (BAT) in patients 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) who had previously been treated with ruxolitinib. The primary objective of the study is to assess the proportion of patients achieving at least a 35% reduction in spleen volume in both the fedratinib and BAT treatment arms.
• On 24 August 2025, Syntara announced an open-label Phase 1/2a study to evaluate the safety and tolerability of PXS-5505 in patients with primary myelofibrosis, post-polycythemia vera (PV) myelofibrosis, or post-essential thrombocythemia (ET) myelofibrosis. The dose-escalation phase will follow a 3+3 design, beginning with a starting dose of 100 mg twice daily for a treatment duration of 4 weeks. Patients will be eligible to participate across multiple dose levels.
• DelveInsight's Polycythemia Vera Pipeline report depicts a robust space with 8+ active players working to develop 10+ pipeline therapies for Polycythemia Vera treatment.
• The leading Polycythemia Vera Companies such as Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals and others.
• Promising Polycythemia Vera Pipeline Therapies such as Rusfertide, Givinostat, Hydroxyurea, Bomedemstat, PTG-300, SLN124, Ruxolitinib, P1101 (Ropeginterferon alfa-2b-njft), AOP2014 and others.

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Polycythemia Vera Emerging Drugs Profile

• Rusfertide: Protagonist Therapeutics

Rusfertide (PTG-300) is an injectable compound that mimics the effect of the natural hormone hepcidin but with greater potency, solubility, and stability. Hepcidin is a key hormone in regulating iron equilibrium and is critical to the proper development of red blood cells. The molecular target of the hormone hepcidin is the cellular trans-membrane protein ferroportin, which functions as an export channel for intracellular iron in macrophages, liver hepatocytes, and duodenal enterocytes. By binding to the extracellular domain of ferroportin, hepcidin redistributes iron by reducing the export of iron from inside the enterocytes and macrophages to the systemic circulation. Excessive quantities of iron relative to the lower levels of beta-globin chains in the bone marrow induce ineffective erythropoiesis resulting in anemia. As a hepcidin mimetic, PTG-300 may redistribute iron to the macrophages, reduce iron-induced oxidative stress in the bone marrow, and allow sufficient production of red blood cells. Also, by limiting the release of iron into the blood, PTG-300 may inhibit the damage caused by excessive iron absorption by vital organs such as the liver and heart. Takeda and Protagonist Therapeutics have signed a worldwide license and collaboration agreement for the development and commercialization of rusfertide. Currently, the drug is in the Phase III stage of its development for the treatment of Polycythemia Vera.

• Givinostat: Italfarmaco

Givinostat, developed by Italfarmaco, is a histone deacetylase (HDAC) inhibitor currently being evaluated for the treatment of Polycythemia Vera (PV), a myeloproliferative neoplasm characterized by excessive production of blood cells due to mutations in the JAK2 gene, particularly the JAK2 V617F mutation. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Polycythemia Vera.

• Sapablursen: Ionis Pharmaceuticals

Sapablursen, also known as ISIS 702843 or IONIS-TMPRSS6-LRx, is an investigational RNA-targeted medicine developed by Ionis Pharmaceuticals for the treatment of polycythemia vera. It functions by inhibiting the TMPRSS6 protein, which regulates hepcidin, a key hormone involved in iron homeostasis. By increasing hepcidin levels, sapablursen aims to alleviate symptoms associated with PV and reduce the need for frequent phlebotomy treatments. Currently, the drug is in the Phase II stage of development to treat Polycythemia Vera.

• PPMX-T003: Perseus Proteomics

PPMX-T003 is a novel human monoclonal antibody developed by Perseus Proteomics, specifically targeting transferrin receptor 1 (TfR1). Currently, the drug is in the Phase I stage of development to treat Polycythemia Vera.

The Polycythemia Vera Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Polycythemia Vera with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Polycythemia Vera Treatment.
• Polycythemia Vera Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Polycythemia Vera Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Polycythemia Vera market

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Polycythemia Vera Companies

Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals and others.

Polycythemia Vera Pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Polycythemia Vera Products have been categorized under various Molecule types such as

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

Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Polycythemia Vera Market Drivers and Barriers

Scope of the Polycythemia Vera Pipeline Report

• Coverage- Global
• Polycythemia Vera Companies- Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals and others.
• Polycythemia Vera Pipeline Therapies- Rusfertide, Givinostat, Hydroxyurea, Bomedemstat, PTG-300, SLN124, Ruxolitinib, P1101 (Ropeginterferon alfa-2b-njft), AOP2014 and others.
• Polycythemia Vera Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Polycythemia Vera Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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