Polycythemia Vera Pipeline Insight 2025

DelveInsight’s “Polycythemia Vera Pipeline Insight 2025” report offers detailed information on over 8 companies and more than 10 pipeline drugs in the Polycythemia Vera treatment landscape. It features Polycythemia Vera pipeline drug profiles for clinical and nonclinical stage products. It also evaluates Polycythemia Vera therapeutics by product type, development stage, administration route, and molecule type. Additionally, it emphasizes inactive pipeline products in this area.

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Key Insights from the Polycythemia Vera Pipeline Report

On December 08, 2025, Novartis Pharmaceuticals carried out a study to identify predictive indicators for hydroxyurea (HU) ineffectiveness (hemoglobin (HGB)).
On December 06, 2025, Protagonist Therapeutics, Inc. (“Protagonist”) (NASDAQ:PTGX), partnering with Takeda (TSE:4502/NYSE:TAK), revealed that updated 52-week data from the key Phase 3 VERIFY trial assessing rusfertide in polycythemia vera (PV) will be presented at the 67th ASH Annual Meeting & Exposition. The oral session will showcase effectiveness and safety results that bolster rusfertide’s potential for prolonged therapy. In the 52-week data, 61.9% of participants on ongoing rusfertide treatment avoided phlebotomy eligibility from start to Week 52, indicating sustained clinical advantages with a steady safety record.
On December 04, 2025, Disc Medicine Inc. disclosed a phase 2 trial testing up to 2 dosage levels of DISC-3405 to evaluate the safety, tolerability, effectiveness, pharmacokinetics, and pharmacodynamics of DISC-3405 in individuals with polycythemia vera (PV).
DelveInsight’s Polycythemia Vera Pipeline report portrays a dynamic field with over 8 active contributors advancing more than 10 pipeline therapies for Polycythemia Vera care.
Leading Polycythemia Vera Companies include Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals, and others.
Promising Polycythemia Vera Pipeline Therapies include 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 agent that replicates the action of the natural hormone hepcidin with enhanced strength, solubility, and durability. Hepcidin plays a vital role in iron balance and is essential for proper red blood cell formation. The hormone targets the cell membrane protein ferroportin, acting as an export pathway for iron in macrophages, liver cells, and intestinal cells. By attaching to ferroportin's external region, hepcidin shifts iron distribution by decreasing its release from enterocytes and macrophages into the bloodstream. Surplus iron compared to reduced beta-globin levels in bone marrow leads to unproductive erythropoiesis causing anemia. As a hepcidin mimic, PTG-300 could redirect iron to macrophages, lessen iron-related oxidative damage in bone marrow, and support adequate red blood cell generation. Moreover, by restricting iron entry into the blood, PTG-300 might prevent harm from excess iron uptake by organs like the liver and heart. Takeda and Protagonist Therapeutics have entered a global licensing and partnership deal for rusfertide's development and marketing. The drug is currently in Phase III for Polycythemia Vera treatment.

Givinostat: Italfarmaco
Givinostat, created by Italfarmaco, is a histone deacetylase (HDAC) inhibitor under assessment for Polycythemia Vera (PV), a myeloproliferative disorder marked by overproduction of blood cells due to JAK2 gene mutations, especially JAK2 V617F. The drug is in Phase III trials for Polycythemia Vera.

Sapablursen: Ionis Pharmaceuticals
Sapablursen, also referred to as ISIS 702843 or IONIS-TMPRSS6-LRx, is an experimental RNA-based therapy from Ionis Pharmaceuticals for polycythemia vera. It works by blocking the TMPRSS6 protein, which controls hepcidin, a crucial hormone for iron regulation. By elevating hepcidin levels, sapablursen seeks to relieve PV symptoms and lessen the frequency of phlebotomy. The drug is in Phase II development for Polycythemia Vera.

PPMX-T003: Perseus Proteomics
PPMX-T003 is an innovative human monoclonal antibody from Perseus Proteomics, aimed at transferrin receptor 1 (TfR1). The drug is in Phase I trials for Polycythemia Vera.

The Polycythemia Vera Pipeline Report Provides Insights into

• The report delivers in-depth details on organizations creating therapies for Polycythemia Vera, including the total therapies per company.

• It examines diverse therapeutic candidates divided into early, mid, and late development phases for Polycythemia Vera management.

• Polycythemia Vera Companies are engaged in focused therapeutics development, with active and inactive (dormant or terminated) initiatives.

• Polycythemia Vera Drugs in progress, classified by development phase, delivery method, target receptor, monotherapy or combo therapy, unique action mechanisms, and molecular structure.

• Comprehensive review of partnerships (corporate-corporate and corporate-academic), licensing deals, and funding for advancing 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 assesses pipeline drugs by Route of Administration. Products are grouped under various ROAs such as

Oral
Intravenous
Subcutaneous
Parenteral
Topical

Polycythemia Vera Products are classified by Molecule types such as

Recombinant fusion proteins
Small molecule
Monoclonal antibody
Peptide
Polymer
Gene therapy

Find out about novel medications, pipeline advancements, and major companies through DelveInsight's professional 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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Table of Contents

1. Introduction

2. Executive Summary

3. Polycythemia Vera: Overview

4. Pipeline Therapeutics

5. Therapeutic Assessment

6. Polycythemia Vera– DelveInsight’s Analytical Perspective

7. Late Stage Products (Phase III)

8. Rusfertide: Protagonist Therapeutics

9. Drug profiles in the detailed report…..

10. Mid Stage Products (Phase II)

11. Sapablursen: Ionis Pharmaceuticals

12. Drug profiles in the detailed report…..

13. Early Stage Products (Phase I)

14. PPMX-T003: Perseus Proteomics

15. Drug profiles in the detailed report…..

16. Preclinical and Discovery Stage Products

17. Drug name: Company name

18. Drug profiles in the detailed report…..

19. Inactive Products

20. Polycythemia Vera Key Companies

21. Polycythemia Vera Key Products

22. Polycythemia Vera- Unmet Needs

23. Polycythemia Vera- Market Drivers and Barriers

24. Polycythemia Vera- Future Perspectives and Conclusion

25. Polycythemia Vera Analyst Views

26. Polycythemia Vera Key Companies

27. Appendix

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