This “Polycythemia vera - Pipeline Insight, 2020” report provides comprehensive insights about 10+ 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, or PV, is a rare blood disease in which body makes too many red blood cells. The extra red blood cells make the blood thicker than normal. As a result, blood clots can form more easily. These clots can block blood flow through arteries and veins, which can cause a heart attack or stroke. Thicker blood also doesn't flow as quickly to body as normal blood. Slowed blood flow prevents organs from getting enough oxygen, which can cause serious problems, such as angina.

Symptoms

The signs and symptoms of PV include:
Headaches, dizziness, and weakness
Shortness of breath and problems breathing while lying down
Feelings of pressure or fullness on the left side of the abdomen due to an enlarged spleen (an organ in the abdomen)
Double or blurred vision and blind spots
Itching all over (especially after a warm bath), reddened face, and a burning feeling on your skin (especially your hands and feet)
Bleeding from your gums and heavy bleeding from small cuts
Unexplained weight loss
Fatigue (tiredness)
Excessive sweating
Very painful swelling in a single joint, usually the big toe (called gouty arthritis)
Diagnosis

Polycythemia vera (PV) may not cause signs or symptoms for years. The disease often is found during routine blood tests done for other reasons. If the results of the blood tests aren't normal, the doctor may want to do more tests. The doctor will diagnose PV based on signs and symptoms, age and overall health, medical history, a physical exam, and test results of the patients. During the physical exam, the doctor will look for signs of PV. He or she will check for an enlarged spleen, red skin on your face, and bleeding from gums. Once it is confirmed that the patient have polycythemia, the next step is to find out whether they have primary polycythemia (polycythemia vera) or secondary polycythemia.

The medical history and physical exam may confirm which type of polycythemia you have. If not, the patient may have tests that check the level of the hormone erythropoietin (EPO) in blood.

People who have PV have very low levels of EPO. People who have secondary polycythemia usually have normal or high levels of EPO.

Treatment

The goals of treating PV are to control symptoms and reduce the risk of complications, especially heart attack and stroke. To do this, PV treatments reduce the number of red blood cells and the level of hemoglobin (an iron-rich protein) in the blood. This brings the thickness of your blood closer to normal. Blood with normal thickness flows better through the blood vessels. This reduces the chance that blood clots will form and cause a heart attack or stroke. Blood with normal thickness also ensures that your body gets enough oxygen. This can help reduce some of the signs and symptoms of PV, such as headaches, vision problems, and itching.

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

PTG-300: Protagonist Therapeutics

PTG-300 is a mimetic of the natural hormone hepcidin that is currently in a phase II 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. Hepcidin is a master regulator of iron homeostasis and controls the absorption, storage, and distribution of iron in the body.

Bomedemstat: Imago BioSciences

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of platelets and cytokines that drive essential thrombocythemia pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent, and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. It is currently in phase II stage of development.

Givinostat: Italfarmaco

Givinostat is being developed by Italfarmaco for the treatment of polycythemia vera. Proof of evidence has been obtained with givinostat earlier in JAK2V617F mutated cells and then in two clinical trials in patients with PV. Treatment resulted effective in inducing hematological response, symptomatic improvement and reduction of enlarged spleen, with good tolerability.

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.

Major Players in Polycythemia vera

There are approx. 10+ 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 II include Protagonist Therapeutics and others.

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 products (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
Infusion
Intradermal
Intramuscular
Intranasal
Intravenous
Oral
Parenteral
Subcutaneous
Topical.
Molecule Type
Products have been categorized under various Molecule types such as
Gene therapies
Small molecule
Vaccines
Polymers
Peptides
Monoclonal antibodies
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 III, 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.

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.
In June 2020, Protagonist Therapeutics announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTG-300 for the treatment of polycythemia vera.
In August 2019, Italfarmaco plans a phase III trial of Givinostat in Polycythaemia vera (PO).
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
Protagonist Therapeutics
Imago BioSciences
Italfarmaco
Sanofi
Roche
Kartos Therapeutics, Inc.
AOP Orphan Pharmaceuticals AG
Key Products
PTG-300
Bomedemstat
Givinostat
Fedratinib
Idasanutlin
KRT-232
Pegylated-Proline-interferon alpha-2b