DelveInsight’s “Myelofibrosis – Pipeline Insight, 2024,” report provides comprehensive insights about 35+ companies and 40+ pipeline drugs in the Myelofibrosis 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.
Some of the key players such as Geron (Imetelstat), AbbVie (Navitoclax), Sierra Oncology (Momelotinib), Incyte Corporation (Parsaclisib), Celgene/BMS (Luspatercept-aamt), MorphoSys (Pelabresib), Imago Biosciences (Bomedemstat), Pharmaxis (PXS-5505), and others are evaluating their lead candidates in different stages of clinical development.
According to DelveInsight, the market for therapeutics in Myelofibrosis is poised for significant growth in the coming years. This growth is driven by increasing cases of Myelofibrosis, extensive research and development efforts by pharmaceutical companies, and the anticipated introduction of new pipeline therapies. Emerging treatments are exploring diverse mechanisms beyond JAK inhibitors, such as imetelstat (a telomerase inhibitor), navitoclax (an inhibitor of BCL-XL/BCL-2), GB2064 (inhibitors of LOXL2 protein), navtemadlin (an inhibitor of MDM2 protein), pelabresib (a BET inhibitor), among others. These therapies aim to address the needs of patients who are refractory or have relapsed after JAK inhibitor treatment, offering alternative therapeutic options.
Myelofibrosis Overview
Myelofibrosis is a rare form of chronic leukemia characterized by abnormal blood cell production in the bone marrow, leading to extensive fibrosis (scarring). This disrupts the marrow’s ability to produce red blood cells, causing severe anemia, weakness, and fatigue. Additional symptoms include enlarged spleen (splenomegaly), night sweats, fever, and bone pain. As the disease progresses, complications such as bleeding and increased susceptibility to infections arise due to impaired white blood cell function. Myelofibrosis belongs to a group of disorders called myeloproliferative neoplasms, characterized by excessive production of blood cells.
The exact cause of myelofibrosis remains unclear, but it often involves mutations in genes like JAK2, CALR, or MPL. These mutations disrupt normal cell signaling pathways, leading to abnormal cell growth and fibrosis. Diagnosis typically involves blood tests, bone marrow biopsy, and genetic testing to identify these mutations. Treatment focuses on symptom management and improving quality of life, including medications such as JAK inhibitors, blood transfusions, and potentially stem cell transplantation in certain cases. Prognosis varies widely, depending on factors such as symptom severity, age, and overall health, with some patients living for many years after diagnosis while others experience faster disease progression.
Diagnostic techniques require complete blood cell counts, bone marrow analysis, extensive genetic evaluations, and a comprehensive patient history. Molecular drivers like JAK2V617F, CALR, and MPL mutations are prevalent, with approximately 8% to 10% of cases being “triple-negative.” Additional genetic variants are observed in about 80% of patients. Current clinical and molecular scoring systems help predict survival and guide treatment decisions, including eligibility for stem cell transplantation and participation in clinical trials. Standard treatments focus on managing anemia, utilizing drugs like hydroxyurea and JAK inhibitors such as ruxolitinib, fedratinib, and pacritinib. Approximately 42% of ruxolitinib-treated patients achieve a spleen volume reduction of 35% or greater by week 24, along with similar outcomes observed with other JAK inhibitors.
The Myelofibrosis Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.
Myelofibrosis Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the emerging therapies for the treatment of Myelofibrosis and the aggregate therapies developed by major pharma companies.
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It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of clinical development.
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It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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The report evaluates the drugs that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Myelofibrosis market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
Analysis of Emerging Therapies by Phases
The report covers the emerging products under different phases of clinical development like –
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Late-stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I)
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
Route of Administration
Myelofibrosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
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Oral
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Parenteral
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Intravitreal
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Subretinal
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Topical
Molecule Type
The report covers the pipeline products that have been categorized under various Molecule types, such as
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Monoclonal Antibody
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Peptides
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Polymer
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Small molecule
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Gene therapy
Learn How the Ongoing Clinical & Commercial Activities will Affect the Myelofibrosis Therapeutic Segment @ https://www.delveinsight.com/sample-request/myelofibrosis-pipeline-insight
Myelofibrosis Therapeutics Landscape
Imetelstat: Geron Corporation
Imetelstat sodium (imetelstat) is a small oligonucleotide consisting of a nucleic acid backbone and a lipid moiety. The proprietary nucleic acid structure provides enhanced stability in plasma and tissues by resisting degradation, while the lipid component improves cell permeability, thereby increasing potency and enhancing pharmacokinetic and pharmacodynamic properties. Imetelstat exhibits prolonged residence time in bone marrow, spleen, and liver. It binds with high affinity to the RNA component of telomerase’s template region, competitively inhibiting telomerase enzymatic activity directly, rather than through antisense inhibition of protein translation. Currently, the drug is undergoing Phase III clinical trials for Myelofibrosis treatment.
Bomedemstat: Merck
Bomedemstat is an irreversible inhibitor of lysine-specific demethylase 1 (LSD1), a crucial enzyme in regulating hematopoietic stem cell proliferation and progenitor maturation. It is currently in Phase III clinical trials for Myelofibrosis treatment.
TL-895: Telios Pharma, Inc.
TL-895 is an orally bioavailable tyrosine kinase inhibitor with potential anti-inflammatory and antineoplastic activities. Upon oral administration, TL-895 targets and inhibits tyrosine kinase, potentially inhibiting tumor angiogenesis, cell proliferation, and immune-mediated inflammatory processes. The drug is currently in Phase II clinical trials for Myelofibrosis.
RVU120: Ryvu Therapeutics SA
RVU120 is a selective, first-in-class dual inhibitor of CDK8/19 kinases developed by Ryvu Therapeutics. It targets CDK8/19 kinases, with translational data confirming its proposed mechanism of action in a molecularly-defined subset of patients with DNMT3A and NPM1 mutations. RVU120 is currently in Phase II development for Myelofibrosis treatment.
TBX-2400: Taiga Biotechnologies, Inc.
TBX-2400 is an allogeneic stem cell therapy aimed at improving engraftment and immune system reconstitution in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). The drug is currently in Phase I clinical trials for Myelofibrosis treatment.
Leading Companies in the Myelofibrosis Market Include:
AbbVie, Actuate Therapeutics, AstraZeneca, Bristol Myers Squibb, Celgene Corporation, Cellenkos, Galecto Biotech, Geron Corporation, Imago BioSciences, Incyte Corporation, iOnctura, Jacobio Pharmaceuticals, Kartos Therapeutics, Karyopharm Therapeutics, Keros Therapeutics, Menarini Group, MorphoSys, Nippon Shinyaku, Novartis Oncology, NS Pharma, Ohm oncology, PharmaEssentia, Pharmaxis, Roche, Secura Bio, Sierra Oncology, Sumitomo Pharma Oncology, Telios Pharma, and others.
Myelofibrosis Emerging & Marketed Therapies and Key Companies:
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Imetelstat: Geron Corporation
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IOA-244: iOnctura
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KER-050: Keros Therapeutics
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Momelotinib: Sierra Oncology
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Navitoclax (ABT-263): AbbVie
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NS-018: Nippon Shinyaku
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Parsaclisib (INCB050465): Incyte
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PXS-5505: Pharmaxis
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REBLOZYL (Luspatercept/ACE-536): Celgene/Bristol Myers Squibb
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TP-3654: Sumitomo Pharma Oncology
Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @ https://www.delveinsight.com/sample-request/myelofibrosis-pipeline-insight
Table of Contents
1. Report Introduction
2. Executive Summary
3. Myelofibrosis Current Treatment Patterns
4. Myelofibrosis – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Myelofibrosis Late Stage Products (Phase-III)
7. Myelofibrosis Mid-Stage Products (Phase-II)
8. Myelofibrosis Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Myelofibrosis Discontinued Products
13. Myelofibrosis Product Profiles
14. Key Companies in the Myelofibrosis Market
15. Key Products in the Myelofibrosis Therapeutics Segment
16. Dormant and Discontinued Products
17. Myelofibrosis Unmet Needs
18. Myelofibrosis Future Perspectives
19. Myelofibrosis Analyst Review
20. Appendix
21. Report Methodology
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