Myelofibrosis - Pipeline Insight, 2025

This “Myelofibrosis - Pipeline Insight, 2025” report provides comprehensive insights about 35+ companies and 40+ pipeline drugs in 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.

Myelofibrosis: Understanding

Myelofibrosis: Overview

Myelofibrosis is a rare type of chronic leukemia that disrupts the normal production of blood cells in the bone marrow, leading to extensive scarring (fibrosis). This condition results in severe anemia, weakness, and fatigue as the bone marrow's ability to generate red blood cells diminishes. Other symptoms include an enlarged spleen (splenomegaly), night sweats, fever, and bone pain. As the disease progresses, it can cause complications like bleeding and an increased risk of infections due to the impaired function of white blood cells. Myelofibrosis is part of a group of diseases known as myeloproliferative neoplasms, where there is an overproduction of one or more types of blood cells.

The exact cause of myelofibrosis remains unclear, but it often involves mutations in the JAK2, CALR, or MPL genes. These mutations lead to abnormal signaling pathways that promote excessive cell proliferation and fibrosis. Diagnosis typically involves blood tests, bone marrow biopsy, and genetic testing to identify mutations. Treatment aims to manage symptoms and improve quality of life, including medications like JAK inhibitors, blood transfusions, and in some cases, stem cell transplantation. Prognosis varies widely among individuals, depending on the severity of symptoms, age, and overall health, with some patients living many years after diagnosis while others experience more rapid disease progression.

Diagnosis requires assessing complete cell blood counts, bone marrow morphology, deep genetic evaluations, and disease history. Driver molecular events consist of JAK2V617F, CALR, and MPL mutations, whereas about 8% to 10% of MF are 'triple-negative.' Additional myeloid-gene variants are described in roughly 80% of patients. Currently available clinical-based and integrated clinical/molecular-based scoring systems predict the survival of patients with MF and are applied for conventional treatment decision-making, indication to stem cell transplant (SCT) and allocation in clinical trials. Standard treatment consists of anemia-oriented therapies, hydroxyurea, and JAK inhibitors such as ruxolitinib, fedratinib, and pacritinib. Overall, spleen volume reduction of 35% or greater at week 24 can be achieved by 42% of ruxolitinib-, 47% of fedratinib-, 19% of pacritinib-, and 27% of momelotinib-treated patients.

'Myelofibrosis- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Myelofibrosis pipeline landscape is provided which includes the disease overview and Myelofibrosis treatment guidelines. The assessment part of the report embraces, in depth Myelofibrosis 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, Myelofibrosis 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 Myelofibrosis R&D. The therapies under development are focused on novel approaches to treat/improve Myelofibrosis.

Myelofibrosis Emerging Drugs Chapters

This segment of the Myelofibrosis 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.

Myelofibrosis Emerging Drugs

Imetelstat: Geron Corporation

Imetelstat sodium (imetelstat) is a small oligonucleotide composed of a nucleic acid and a lipid moiety. The proprietary nucleic acid backbone provides resistance to degradation, thus conferring improved stability in plasma and tissues, as well as significantly improved binding affinity to its target. The lipid group enhances cell permeability, which results in increased potency and improved pharmacokinetic and pharmacodynamic properties. The compound has a long residence time in bone marrow, spleen, and liver. Imetelstat binds with high affinity to the template region of the RNA component of telomerase, resulting in direct, competitive inhibition of telomerase enzymatic activity rather than eliciting its effect through an antisense inhibition of protein translation. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Myelofibrosis.

Bomedemstat: Merck

Bomedemstat is an irreversible inhibitor of lysine-specific demethylase 1 (LSD1), an enzyme critical for regulating the proliferation of hematopoietic stem cells and the maturation of progenitors. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Myelofibrosis.

TL-895: Telios Pharma, Inc.

It is an orally bioavailable inhibitor of tyrosine kinase, with potential anti-inflammatory and antineoplastic activities. Upon oral administration, tyrosine kinase inhibitor TL-895 targets, binds to, and inhibits tyrosine kinase. This may result in the inhibition of tumor angiogenesis and cell proliferation, and the inhibition of immune-mediated inflammatory processes. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Myelofibrosis.

RVU120: Ryvu Therapeutics SA

RVU120 is a selective, first-in-class dual CDK8/19 kinase inhibitor developed by Ryvu Therapeutics. RVU120's mechanism of action (MoA) involves targeting CDK8/19 kinases. Specifically, translational data confirm the proposed MoA in a molecularly-defined subset of patients with DNMT3A and NPM1 mutations. Currently, the drug is in the Phase II stage of development to treat Myelofibrosis.

TBX-2400: Taiga Biotechnologies, Inc.

TBX-2400 is an allogeneic stem cell therapy developed by Taiga Biotechnologies that aims to improve the rate of engraftment and reconstitution of the immune system for patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Currently, the drug is in the Phase I stage of development to treat Myelofibrosis.

Myelofibrosis: Therapeutic Assessment

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

Major Players in Myelofibrosis

• There are approx. 35+ key companies which are developing the therapies for Myelofibrosis. The companies which have their Myelofibrosis drug candidates in the most advanced stage, i.e. Phase III include, Merck and Geron Corporation.

Phases

The report covers around 40+ 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

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

• 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.

Myelofibrosis: Pipeline Development Activities

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

Myelofibrosis Report Insights

• Myelofibrosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Geron Corporation
• Merck
• Telios Pharma, Inc.

Ryvu Therapeutics SA

• Taiga Biotechnologies, Inc.

Morphic Therapeutic

• iOnctura
• Pharmaxis
• Nippon Shinyaku
• Active Biotech
• Incyte Corporation
• Sumitomo Pharma America, Inc.

Key Products

• Imetelstat
• Bomedemstat
• TL-895
• RVU120
• TBX-2400
• MORF-088
• IOA-244
• PXS-5505
• NS-018
• Tasquinimod
• Parsaclisib
• TP-3654

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