(Albany, USA) DelveInsight's "Myelodysplastic Syndrome - Pipeline Insight, 2025" offers a detailed analysis of 150+ emerging therapies in development, targeting various MDS subtypes and risk categories.
The Myelodysplastic Syndrome 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.
The pipeline includes a rich mix of first-in-class agents, next-generation HMAs, immune checkpoint inhibitors, targeted therapies (e.g., IDH1/2, TP53, and FLT3 inhibitors), and novel cellular and gene-modifying strategies. Companies such as Novartis, Syros Pharmaceuticals, Curis, Ryvu Therapeutics, Chia Tai Tianqing Pharmaceutical Group, Amgen, and Sanofi are advancing innovative assets, including telomerase inhibitors, BET inhibitors, and oral formulations of established drugs, aiming to improve patient compliance and outcomes.
Interested in learning more about the current treatment landscape and the key drivers shaping the myelodysplastic syndrome pipeline? Click here: Myelodysplastic Syndrome Pipeline Insight
Key Takeaways from the Myelodysplastic Syndrome Pipeline Report
- DelveInsight's myelodysplastic syndrome pipeline analysis depicts a strong space with 120+ active players working to develop 150+ pipeline drugs for myelodysplastic syndrome treatment.
- The leading myelodysplastic syndrome companies include Novartis, Syros Pharmaceuticals, Curis, Ryvu Therapeutics, Chia Tai Tianqing Pharmaceutical Group, Amgen, Sanofi, Forma Therapeutics, Agios Pharmaceuticals, AbbVie, Daiichi Sankyo Company, Geron Corporation, Astex Pharmaceuticals, Jazz Pharmaceuticals, Maxinovel Pty. Ltd., Mabwell (Shanghai) Bioscience Co., Ltd., BeiGene, Ellipses Pharma, Treadwell Therapeutics, Bellicum Pharmaceuticals, and others are evaluating their lead assets to improve the myelodysplastic syndrome treatment landscape.
- Key myelodysplastic syndrome pipeline therapies in various stages of development include MBG453, Tamibarotene, CA-4948, RVU120, TQB2618, AMG 176, SAR443579, Etavopivat, AG-946, Venetoclax, Quizartinib, Imetelstat, Azacitidine, Vyxeos, MAX-40279-01, 6MW3211, BGB-11417, EP0042, CFI-400945, BPX-501, and others.
- In March 2025, Faron Pharmaceuticals Ltd., a biopharmaceutical company specializing in immune system manipulation for cancer treatment, announced that the FDA granted Orphan Drug Designation for bexmarilimab, its leading drug candidate for myelodysplastic syndromes (MDS).
- In January 2025, the FDA approved GRAFAPEX in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and pediatric patients aged 1 and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), as announced by Medexus, the drug's manufacturer.
- In January 2025, Medexus announced that the FDA approved GRAFAPEX, an alkylating agent, with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients aged one year and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
- In January 2025, R289, a potent and selective dual inhibitor of IRAK1 and IRAK4, received orphan drug designation from the FDA for the treatment of patients with myelodysplastic syndromes (MDS). An ongoing open-label phase Ib study is evaluating R289's safety, tolerability, pharmacokinetics, and preliminary activity, specifically in patients with LR-MDS who are relapsed or refractory to prior therapies.
- In January 2025, Rigel Pharmaceuticals, Inc. announced that the FDA has granted Orphan Drug designation to R289 for the treatment of myelodysplastic syndromes (MDS).
Request a sample and discover the recent breakthroughs happening in the myelodysplastic syndrome pipeline landscape at Myelodysplastic Syndrome Pipeline Drugs
Myelodysplastic Syndrome Overview
Myelodysplastic Syndrome (MDS) is a group of bone marrow disorders caused by poorly formed or dysfunctional blood cells. It results from clonal abnormalities in hematopoietic stem cells, leading to ineffective blood cell production and varying degrees of anemia, infection risk, or bleeding. MDS can progress to acute myeloid leukemia (AML), particularly in high-risk patients.
Most cases occur in people over 65 and may be linked to aging, environmental exposures (like benzene or radiation), or prior chemotherapy. MDS may be de novo or treatment-related, often associated with genetic mutations or chromosomal changes, such as 5q deletion or trisomy 8, which influence prognosis through scoring systems like IPSS-R.
While some patients remain stable and require only supportive care, others may need disease-modifying treatments, including hypomethylating agents or, in select cases, allogeneic stem cell transplant, the only potential cure. Treatment plans are highly individualized based on risk, age, and overall health.
Find out more about myelodysplastic syndrome medication at Myelodysplastic Syndrome Companies and Therapies
Myelodysplastic Syndrome Treatment Analysis: Drug Profile
Tamibarotene: Syros Pharmaceuticals
Tamibarotene (formerly SY-1425) is an oral, selective retinoic acid receptor alpha (RARα) agonist developed for patients with RARA overexpression, observed in about 50% of MDS and 30% of AML cases. Excess RARα can disrupt normal myeloid differentiation, contributing to hematologic malignancies. Tamibarotene restores this process by binding to RARα and reactivating gene expression. It is currently being evaluated in the Phase III SELECT-MDS-1 trial in newly diagnosed higher-risk MDS (HR-MDS) patients with RARA overexpression.
RVU120: Ryvu Therapeutics
RVU120 (SEL120) is a selective CDK8/CDK19 inhibitor showing strong antileukemic activity in preclinical studies. It has also demonstrated the potential to promote erythroid differentiation in CD34+ cells from Diamond-Blackfan anemia patients. RVU120 is currently in Phase II clinical trials for the treatment of myelodysplastic syndromes.
Emavusertib: Curis
Emavusertib (CA-4948) is an IRAK4 kinase inhibitor developed by Curis in collaboration with Aurigene. By targeting IRAK4-L, it disrupts signaling pathways critical to leukemic growth. This small-molecule therapy is being studied in Phase I/II trials for MDS, as well as other B-cell malignancies and inflammatory diseases.
Learn more about the novel and emerging myelodysplastic syndrome pipeline therapies at Myelodysplastic Syndrome Medication and Drug Manufacturers
Therapies under clinical evaluation range from low-risk MDS supportive approaches to aggressive disease-modifying treatments for higher-risk patients. Investigational combinations such as azacitidine with venetoclax, magrolimab (anti-CD47) with HMAs, and immune-based regimens represent the next wave of treatment evolution. DelveInsight's report segments candidates by stage (Preclinical to Phase III), mechanism of action, route of administration, and therapy class, while also offering insights into discontinued and dormant assets. It highlights biomarkers driving trial design, clinical endpoints shaping regulatory pathways, and competitive positioning across global regions. As the understanding of MDS biology deepens, the 2025 pipeline holds promise for more precise, tolerable, and effective therapies that can delay progression, reduce transfusion dependency, and offer better quality of life for patients with this challenging bone marrow disorder.
Myelodysplastic Syndrome Therapeutics Assessment
By Product Type
- Mono
- Combination
- Mono/Combination.
By Stage
- 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
By Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
By Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
Scope of the Myelodysplastic Syndrome Pipeline Report
- Coverage: Global
- Key Myelodysplastic Syndrome Companies: Novartis, Syros Pharmaceuticals, Curis, Ryvu Therapeutics, Chia Tai Tianqing Pharmaceutical Group, Amgen, Sanofi, Forma Therapeutics, Agios Pharmaceuticals, AbbVie, Daiichi Sankyo Company, Geron Corporation, Astex Pharmaceuticals, Jazz Pharmaceuticals, Maxinovel Pty. Ltd., Mabwell (Shanghai) Bioscience Co., Ltd., BeiGene, Ellipses Pharma, Treadwell Therapeutics, Bellicum Pharmaceuticals, and others.
- Key Myelodysplastic Syndrome Pipeline Therapies: MBG453, Tamibarotene, CA-4948, RVU120, TQB2618, AMG 176, SAR443579, Etavopivat, AG-946, Venetoclax, Quizartinib, Imetelstat, Azacitidine, Vyxeos, MAX-40279-01, 6MW3211, BGB-11417, EP0042, CFI-400945, BPX-501, and others.
To dive deep into rich insights for drugs used for myelodysplastic syndrome treatment, visit: Myelodysplastic Syndrome Clinical Trails and Recent Advancements
Table of Contents
1. Introduction
2. Executive Summary
3. Myelodysplastic Syndrome Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Myelodysplastic Syndrome Pipeline Therapeutics
6. Myelodysplastic Syndrome Pipeline: Late-Stage Products (Phase III)
7. Myelodysplastic Syndrome Pipeline: Mid-Stage Products (Phase II)
8. Myelodysplastic Syndrome Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix
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