Polycythemia Vera - Pipeline Insight, 2024

This “Polycythemia Vera - Pipeline Insight, 2024” 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 is a type of blood cancer. It causes your bone marrow to make too many red blood cells. These excess cells thicken your blood, slowing its flow, which may cause serious problems, such as blood clots. Polycythemia vera is rare. It usually develops slowly, and you might have it for years without knowing. Often the condition is found during a blood test done for another reason. Without treatment, polycythemia vera can be life-threatening. Polycythemia vera occurs when a mutation in a gene causes a problem with blood cell production. Normally, your body regulates the number of each of the three types of blood cells you have - red blood cells, white blood cells and platelets. But in polycythemia vera, your bone marrow makes too many of some of these blood cells.

Specific symptoms include itchiness, particularly after a warm bath or shower; numbness, tingling, burning, or weakness in hands, feet, arms, or legs; a feeling of fullness shortly after eating and bloating or pain in the left upper abdomen due to an enlarged spleen; unusual bleeding, such as a nosebleed or bleeding gums; painful swelling of one joint, often the big toe; and shortness of breath and difficulty breathing when lying down, Additionally, patients may experience more subtle symptoms such as headache, dizziness, fatigue, blurred vision, and joint pain.

Treatment options depend on the patient's risk for clotting complications and may include phlebotomy, low-dose aspirin, and medication to reduce the number of blood cells. High-risk patients may also be prescribed cytoreductive drugs such as hydroxyurea. Interferon-alfa and ruxolitinib are other medications that may be used to slow the production of new red blood cells. In addition to medical treatment, self-care tips such as exercise, staying hydrated, and avoiding low-oxygen environments can help manage the condition.

“Polycythemia Vera - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. 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 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

PTG-300: Protagonist TherapeuticsPTG-300 (Rusfertide) is a mimetic of the natural hormone hepcidin that is currently in a Phase III study in Polycythemia Vera (PV), a rare blood disorder, and a phase II study in hereditary hemochromatosis (HH), a blood disorder arising from absence or deficiency of hepcidin gene. This investigational drug works by lowering iron levels in the blood, which is essential for red blood cell production. Hepcidin is a master regulator of iron homeostasis and controls the absorption, storage, and distribution of iron in the body.

Sapablursen: Ionis PharmaceuticalsSapablursen, formerly known as IONIS-TMPRSS6-LRx, is an investigational ligand-conjugated antisense (LICA) medicine designed to target the TMPRSS6 gene to modulate the production of hepcidin, which is the key regulator of iron homeostasis. By modulating hepcidin expression, sapablursen has the potential to positively impact diseases characterized by iron deficiency, such as polycythemia vera (PV). Currently, the drug is in Phase II stage of its clinical trial for the treatment of Polycythemia vera.

PPMX-T003: Perseus ProteomicsPPMX-T003 is an antibody targeting transferrin receptor (TfR) that is related to iron intake into cells. TfR is highly expressed in cells that require much more iron than usual cells, such as cancer cells that proliferate at a significant pace, and erythroblasts, which are nucleated cells in bone marrow from which RBCs derive. When PPMX-T003 binds to TfR, it inhibits cell proliferation by inhibiting iron uptake into cancer cells. As PPMX-T003 is expected to have therapeutic effects for a wide variety of cancers including ANKL and acute myeloma leukemia (AML), its development has been the main target of the Company. Currently, the drug is in Phase I stage of its clinical trial for the treatment of polycythemia vera.

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

This 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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

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

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?

Key Players

• Silence Therapeutics
• Ionis Pharmaceuticals
• Perseus Proteomics
• Kartos Therapeutics
• Disc Medicine

Key Products

• SLN124
• Sapablursen
• PTG-300
• KRT-232
• DISC-3405

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