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Polycythemia Vera Pipeline and Drug Development in 2025: 10+ Therapies and 8+ Companies Reshaping the Treatment Landscape, estimates DelveInsight

01-08-2026 03:36 AM CET | Health & Medicine

Press release from: DelveInsight Business Research

Polycythemia Vera Pipeline

Polycythemia Vera Pipeline

DelveInsight's "Polycythemia Vera Pipeline Insight 2025" report delivers an in-depth overview of the Polycythemia Vera pipeline landscape, covering more than 8 companies and 10+ pipeline candidates. The report analyzes both clinical-stage and preclinical assets, offering detailed drug profiles across various stages of development. It also evaluates Polycythemia Vera therapies based on product classification, development stage, route of administration, and molecular category, while additionally spotlighting inactive or discontinued pipeline assets.

Explore the most recent therapeutic advancements and evolving treatment strategies in the Polycythemia Vera pipeline. Gain access to DelveInsight's exhaustive analysis today! @ Polycythemia Vera Pipeline Outlook - https://www.delveinsight.com/report-store/polycythemia-vera-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Key Highlights from the Polycythemia Vera Pipeline Report
• December 08, 2025 - Novartis Pharmaceuticals initiated a study aimed at validating predictive markers associated with hydroxyurea (HU) treatment failure, including hemoglobin (HGB) parameters.
• December 06, 2025 - Protagonist Therapeutics, Inc. (NASDAQ: PTGX), together with Takeda (TSE:4502 / NYSE: TAK), announced the presentation of new 52-week data from the pivotal Phase III VERIFY study evaluating rusfertide in patients with Polycythemia Vera. The data were scheduled for presentation at the 67th ASH Annual Meeting & Exposition. The oral presentation highlights long-term efficacy and safety findings, reinforcing rusfertide's therapeutic potential. Notably, 61.9% of patients receiving continuous rusfertide treatment remained free from phlebotomy eligibility from baseline through Week 52, demonstrating sustained clinical benefit and a stable safety profile.
• December 04, 2025 - Disc Medicine, Inc. disclosed the initiation of a Phase II clinical study evaluating up to two dose levels of DISC-3405. The trial is designed to assess safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of DISC-3405 in individuals diagnosed with Polycythemia Vera.
• DelveInsight's Polycythemia Vera Pipeline report highlights a dynamic and competitive landscape, with over 8 active developers advancing more than 10 pipeline therapies for the treatment of Polycythemia Vera.
• Key players operating in the Polycythemia Vera space include Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals, among others.
• Notable Polycythemia Vera pipeline candidates include Rusfertide, Givinostat, Hydroxyurea, Bomedemstat, PTG-300, SLN124, Ruxolitinib, P1101 (Ropeginterferon alfa-2b-njft), AOP2014, and additional emerging therapies.

Uncover insights into ongoing clinical trials, novel drug candidates, and leading market participants with DelveInsight @ Polycythemia Vera Treatment Drugs - https://www.delveinsight.com/sample-request/polycythemia-vera-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Polycythemia Vera Emerging Drug Profiles
Rusfertide: Protagonist Therapeutics
Rusfertide (PTG-300) is a subcutaneously administered therapeutic designed to replicate the activity of the endogenous hormone hepcidin, offering enhanced potency, stability, and solubility. Hepcidin plays a crucial role in iron regulation and normal erythropoiesis. Its biological target, ferroportin, is a transmembrane protein responsible for exporting intracellular iron from macrophages, hepatocytes, and intestinal enterocytes into systemic circulation. By binding to ferroportin, hepcidin restricts iron release into the bloodstream, thereby redistributing iron stores. In Polycythemia Vera, dysregulated iron metabolism contributes to ineffective erythropoiesis and oxidative stress. As a hepcidin mimetic, PTG-300 is designed to rebalance iron distribution, mitigate iron-driven oxidative damage in the bone marrow, and support healthy red blood cell production. Additionally, limiting systemic iron release may protect vital organs such as the liver and heart from iron overload. Protagonist Therapeutics and Takeda have entered into a global licensing and collaboration agreement for rusfertide's development and commercialization. The therapy is currently in Phase III clinical development for Polycythemia Vera.

Givinostat: Italfarmaco
Givinostat, developed by Italfarmaco, is a histone deacetylase (HDAC) inhibitor under investigation for the treatment of Polycythemia Vera, a myeloproliferative disorder characterized by excessive blood cell production driven largely by JAK2 mutations, particularly JAK2 V617F. The drug is presently undergoing Phase III clinical trials for Polycythemia Vera.

Sapablursen: Ionis Pharmaceuticals
Sapablursen (also known as ISIS 702843 or IONIS-TMPRSS6-LRx) is an experimental RNA-targeted therapy developed by Ionis Pharmaceuticals for Polycythemia Vera. The drug inhibits TMPRSS6, a regulator of hepcidin expression, thereby increasing hepcidin levels and improving iron homeostasis. This mechanism is intended to reduce PV-related symptoms and minimize reliance on repeated phlebotomy. Sapablursen is currently in Phase II clinical development.

PPMX-T003: Perseus Proteomics
PPMX-T003 is an innovative human monoclonal antibody developed by Perseus Proteomics that targets transferrin receptor 1 (TfR1). The therapy is presently in the Phase I stage of clinical evaluation for Polycythemia Vera.

Insights Covered in the Polycythemia Vera Pipeline Report
• Comprehensive profiles of companies developing Polycythemia Vera therapies, including the total number of candidates per organization.
• Classification of therapeutic candidates across early-, mid-, and late-stage development.
• Overview of targeted therapy development efforts, including both active and inactive (discontinued or dormant) projects.
• Detailed segmentation of pipeline drugs based on development stage, route of administration, target receptor, monotherapy or combination approach, mechanism of action, and molecular class.
• In-depth evaluation of strategic collaborations, licensing agreements, and funding activities supporting future growth in the Polycythemia Vera market.

Discover innovative therapies and advancing clinical programs in the Polycythemia Vera pipeline. Access DelveInsight's comprehensive report today! @ New Polycythemia Vera Drugs - https://www.delveinsight.com/sample-request/polycythemia-vera-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Polycythemia Vera Key Companies
Major organizations involved in Polycythemia Vera drug development include Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals, and others.
The Polycythemia Vera Pipeline report also assesses therapeutic candidates by route of administration, including:
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Pipeline products are further categorized by molecule type, such as:
• Recombinant fusion proteins
• Small molecules
• Monoclonal antibodies
• Peptides
• Polymers
• Gene therapies

Stay informed on emerging drugs, pipeline progress, and leading industry players through DelveInsight's expert insights @ Polycythemia Vera Market Drivers and Barriers - https://www.delveinsight.com/sample-request/polycythemia-vera-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Scope of the Polycythemia Vera Pipeline Report
• Geographical Coverage: Global
• Key Companies: Protagonist Therapeutics, Italfarmaco, Ionis Pharmaceuticals, Perseus Proteomics, Agios Pharmaceuticals, and others
• Pipeline Therapies: Rusfertide, Givinostat, Hydroxyurea, Bomedemstat, PTG-300, SLN124, Ruxolitinib, P1101 (Ropeginterferon alfa-2b-njft), AOP2014, and more
• Therapeutic Assessment by Product Type: Monotherapy, Combination Therapy, Mono/Combination
• Therapeutic Assessment by Development Stage: Discovery, Preclinical, Phase I, Phase II, Phase III

Download DelveInsight's detailed pipeline intelligence today! @ Polycythemia Vera Companies, Key Products and Unmet Needs - https://www.delveinsight.com/sample-request/polycythemia-vera-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Table of Contents
• Introduction
• Executive Summary
• Polycythemia Vera Overview
• Pipeline Therapeutics
• Therapeutic Assessment
• Polycythemia Vera - DelveInsight's Analytical Perspective
• Late-Stage Products (Phase III)
o Rusfertide: Protagonist Therapeutics
o Detailed drug profiles
• Mid-Stage Products (Phase II)
o Sapablursen: Ionis Pharmaceuticals
o Detailed drug profiles
• Early-Stage Products (Phase I)
o PPMX-T003: Perseus Proteomics
o Detailed drug profiles
• Preclinical and Discovery-Stage Products
o Drug Name: Company Name
o Detailed drug profiles
• Inactive Products
• Polycythemia Vera Key Companies
• Polycythemia Vera Key Products
• Polycythemia Vera Unmet Needs
• Polycythemia Vera Market Drivers and Barriers
• Polycythemia Vera Future Outlook and Conclusions
• Analyst Perspectives
• Appendix

Contact Us:
Ankit Nigam
Manager Marketing
info@delveinsight.com
+14699457679

About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.

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