Essential Thrombocythemia - Pipeline Insight, 2024

This “Essential thrombocythemia - Pipeline Insight, 2024,” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Essential thrombocythemia 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

Essential thrombocythemia Understanding

Essential thrombocythemia: Overview

Essential thrombocythemia, also known as ET, is a rare disease. Essential thrombocytosis (primary thrombocythemia) is a nonreactive, chronic myeloproliferative disorder in which sustained megakaryocyte proliferation leads to an increase in the number of circulating platelets. Most commonly diagnosed in women over the age of 50, ET is associated with a proliferation of platelet precursors in the bone marrow and complications frequently include blood clotting and/or bleeding. Less common consequences in the later stages of ET include a transformation to myelofibrosis (marrow scarring) or acute leukemia. The causes of ET are still unknown. About half of ET patients have a mutation called JAK2V617F within their blood-forming cells, which leads to many characteristic features of the disease. ET is often detected on blood tests done for other reasons, before there are any symptoms. Additional tests may include: Complete blood count, blood smear, genetic testing, and bone marrow test. Treatments for ET can vary depending on a patient's symptoms. Some patients may not need active treatment but should still be monitored by a physician expert. Treatment includes: Low-dose aspirin, Platelet-lowering medications, and Plateletpheresis.

Essential thrombocythemia - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Essential thrombocythemia pipeline landscape is provided which includes the disease overview and Essential thrombocythemia treatment guidelines. The assessment part of the report embraces, in depth Essential thrombocythemia 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, Essential thrombocythemia 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 Essential thrombocythemia R&D. The therapies under development are focused on novel approaches to treat/improve Essential thrombocythemia.

Essential thrombocythemia Emerging Drugs Chapters

This segment of the Essential thrombocythemia 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.

Essential thrombocythemia Emerging Drugs

Ropeginterferon alfa-2b: PharmaEssentiaRopeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). Its pharmacokinetic properties offer a new level of tolerability. Ropeginterferon alfa-2b is designed to be self-administered subcutaneously with a pen once every two weeks, or monthly during long-term maintenance. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons. PharmaEssentia Corporation is developing a pegylated (PEG) IFN-a product, P1101, for the treatment of ET.

The Phase III multi-site, open-label, randomized, active-controlled study will compare the efficacy, safety, and tolerability and pharmacokinetics of P1101 as second line therapy for ET as compared with anagrelide, a commonly used oral platelet-reduction therapy.

IMG-7289: Imago BiosciencesIMG-7289 (Bomedemstat) is an orally available small molecule, discovered and developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, bomedemstat demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other therapeutic agents. The drug is being evaluated in Phase II clinical trial to treat patients with Essential thrombocythaemia, Myelofibrosis, and Polycythaemia Vera. The Drug has been granted orphan and fast track designation from US FDA.

Essential thrombocythemia: Therapeutic Assessment

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

Major Players in Essential thrombocythemia

There are approx. 6+ key companies which are developing the therapies for Essential thrombocythemia. The companies which have their Essential thrombocythemia drug candidates in the most advanced stage, i.e. Phase III include, PharmaEssentia.

Phases

This report covers around 6+ 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

Essential thrombocythemia 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
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Essential thrombocythemia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Essential thrombocythemia 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 Essential thrombocythemia drugs.

Essential thrombocythemia Report Insights

• Essential thrombocythemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Essential thrombocythemia 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 Essential thrombocythemia drugs?
• How many Essential thrombocythemia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Essential thrombocythemia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Essential thrombocythemia 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 Essential thrombocythemia and their status?
• What are the key designations that have been granted to the emerging drugs?