polycythemia vera pipeline insight

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 pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Geography Covered

• Global coverage

 

Polycythemia Vera: Understanding

Polycythemia Vera: Overview

Polycythemia vera (PV) is a myeloproliferative neoplastic disorder involving uncontrolled red blood cell production resulting in elevated red blood cell (RBC) mass. There is often a concurrent stimulation of myeloid and megakaryocytic lineages, leading to increased white blood cell and platelet production. The current understanding of pathophysiology involves increased sensitivity to growth factors due to an abnormal hematopoietic cell clone. Disease complications are associated with increased blood viscosity, including thrombosis. 

The bone marrow of patients with polycythemia vera (PV) contains normal stem cells and contains abnormal clonal stem cells that suppress normal stem cell growth and maturation. The cause of panmyelosis is unregulated neoplastic proliferation. JAK2 kinase mutation likely leads to the signaling derangements resulting in PV. A valine to phenylalanine substitution at position 617 of the JAK2 gene, or JAK2V617F, leads to constitutively active cytokine receptors. This mutation is observed in over 90% of patients with PV and 50% to 60% of primary myelofibrosis, and 50% of essential thrombocythemia. This process leads to increased production of red blood cells and platelets with associated complications of thrombosis and bleeding.

While there is no cure for PV, treatment focuses on reducing the risk of complications and managing symptoms. This includes phlebotomy, which involves the regular removal of blood to decrease red blood cell count and blood viscosity. Medications are also commonly used, such as hydroxyurea to suppress bone marrow activity, low-dose aspirin to reduce the risk of blood clots, and JAK2 inhibitors like ruxolitinib for patients with more severe symptoms or those who do not respond to other treatments. Lifestyle modifications are equally important and include maintaining hydration, engaging in regular exercise, and avoiding tobacco while managing cardiovascular risk factors. 

 

""Polycythemia Vera- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Polycythemia Vera pipeline landscape is provided which includes the disease overview and Polycythemia Vera treatment guidelines. The assessment part of the report embraces, in depth Polycythemia Vera commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Polycythemia Vera collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Polycythemia Vera R&D. The therapies under development are focused on novel approaches to treat/improve Polycythemia Vera.

 

Polycythemia Vera Emerging Drugs Chapters

This segment of the Polycythemia Vera report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

Polycythemia Vera Emerging Drugs

• 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.

Further product details are provided in the report……..

 

Polycythemia Vera: Therapeutic Assessment

This segment of the report provides insights about the different Polycythemia Vera drugs segregated based on following parameters that define the scope of the report, such as:

 

Major  Players in Polycythemia Vera

There are approx. 8+ key companies which are developing the therapies for Polycythemia Vera. The companies which have their Polycythemia Vera drug candidates in the most advanced stage, i.e. Phase III include, Protagonist Therapeutics.

 

Phases

DelveInsight’s report covers around 10+ products under different phases of clinical development like

• 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

 

Route of Administration

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

 

Molecule Type

Products have been categorized under various Molecule types such as

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

 

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Polycythemia Vera: Pipeline Development Activities 

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Polycythemia Vera therapeutic drugs key players involved in developing key drugs. 

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Polycythemia Vera drugs.

 

Polycythemia Vera Report Insights

• Polycythemia Vera Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

 

Polycythemia Vera Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

 

Explore Polycythemia Vera market insights, epidemiology, and forecasts up to 2034.

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Polycythemia Vera drugs?
• How many Polycythemia Vera drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Polycythemia Vera?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Polycythemia Vera therapeutics? 
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies? 
• What are the clinical studies going on for Polycythemia Vera and their status?
• What are the key designations that have been granted to the emerging drugs?