Myelodysplastic Syndrome Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies

March 06 20:26 2025
Myelodysplastic Syndrome Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies

DelveInsight’s, “Myelodysplastic Syndrome Pipeline Insight” report provides comprehensive insights about 120+ companies and 125+ pipeline drugs in Myelodysplastic Syndrome pipeline landscape. It covers the Myelodysplastic Syndrome pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Myelodysplastic Syndrome therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Myelodysplastic Syndrome Pipeline Report

  • In March 2025, Taiho Oncology, Inc. initiated a Phase 1-2, multicenter, open-label study to evaluate the safety, pharmacodynamics (PD), pharmacokinetics (PK), and hematologic response of various ASTX727 LD doses and schedules in subjects with low-risk or Intermediate-1 Myelodysplastic Syndrome (MDS) based on the IPSS risk category. The study will be conducted in two phases.
  • In March 2025, Janssen Pharmaceuticals conducted a study to assess the efficacy of talacotuzumab (JNJ-56022473) or daratumumab in transfusion-dependent participants with low or intermediate-1 risk MDS whose disease has relapsed during treatment with or is refractory to Erythropoiesis-Stimulating Agents (ESAs), focusing on achieving transfusion independence (TI).
  • DelveInsight’s Myelodysplastic Syndrome pipeline report depicts a robust space with 120+ active players working to develop 150+ pipeline therapies for Myelodysplastic Syndrome treatment.
  • The leading Myelodysplastic Syndrome Companies such as 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.
  • Promising Myelodysplastic Syndrome Therapies such as Deferasirox, Bortezomib, Decitabine and cedazuridine, Luspatercept, Epoetin Alfa, and others.

Learn how leading Myelodysplastic Syndrome Companies are positioning themselves for success in the evolving pharmaceutical market—access the full report today!” @ Myelodysplastic Syndrome Clinical Trials Assessment

Myelodysplastic Syndrome Emerging Drugs

  • Tamibarotene: Syros Pharmaceuticals

Tamibarotene (formerly SY-1425) is an oral selective retinoic acid receptor alpha (RARα) agonist. The company is developing treatments for genomically defined subsets of patients whose disease is characterized by the overexpression of the RARA gene. Approximately 50% of MDS patients and 30% of AML patients have RARA overexpression. When RARα is expressed in excess of its tightly controlled natural ligand, cells in the bone marrow may not differentiate into healthy myeloid cells, which can lead to hematological malignancies. However, when oral tamibarotene is administered, tamibarotene binds to RARα, allowing for the restoration of gene expression and myeloid differentiation. The company is currently investigating tamibarotene in the Phase III SELECT-MDS-1 trial in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients with RARA overexpression.

  • CA-4948: Curis

CA-4948, which is being developed by Curis, is a small-molecule IRAK4 kinase inhibitor. Inhibition of IRAK4-L activity with emavusertib (CA-4948) blocks leukemic growth in non-clinical experiments. Because IRAK4 plays a central role in this pathway, it is considered an attractive target for the generation of therapeutics to treat these B-cell malignancies as well as certain inflammatory diseases. As part of the collaboration with Aurigene, in October 2015, Curis exclusively licensed a program of orally available, small molecule inhibitors of IRAK4 kinase, including emavusertib (CA-4948). Currently, the drug is in the Phase II stage of its development for the treatment of Myelodysplastic syndromes.

  • RVU120: Ryvu Therapeutics

RVU120 (SEL120) is a specific, selective inhibitor of CDK8 and its paralog, CDK19. Preclinical studies indicated the strong antileukemic potential of RVU120, which was often associated with the multilineage commitment of CD34+ AML cells. Moreover, RVU120 could improve proliferation and induce erythroid differentiation of CD34+ cells derived from Diamond-Blackfan anemia (DBA) patients. Currently, the drug is in the Phase II stage of its development for the treatment of Myelodysplastic syndromes.

  • MNV-201: Minovia Therapeutics

MNV-201 is an investigational cell therapy developed by Minovia Therapeutics, targeting low-risk Myelodysplastic Syndromes (MDS). It utilizes a novel approach known as Mitochondrial Augmentation Technology (MAT), which involves enriching a patient’s own hematopoietic stem and progenitor cells (HSPCs) with mitochondria derived from allogeneic placental sources. This therapy aims to address mitochondrial dysfunction, which is implicated in the pathophysiology of MDS. Currently, MNV-201 is undergoing a Phase I clinical trial (NCT06465160) that is actively recruiting participants. The study’s primary objective is to evaluate the safety and therapeutic effects of MNV-201 in patients diagnosed with low-risk MDS.

The Myelodysplastic Syndrome pipeline report provides insights into

  • The report provides detailed insights about companies that are developing therapies for the treatment of Myelodysplastic Syndrome with aggregate therapies developed by each company for the same.
  • It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Myelodysplastic Syndrome Treatment.
  • Myelodysplastic Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • Myelodysplastic Syndrome Drugs 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.
  • Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Myelodysplastic Syndrome market.

From early-stage research to late-phase Myelodysplastic Syndrome Clinical Trials, our analysis covers key Myelodysplastic Syndrome Companies, innovative treatment approaches, and the next wave of Emerging Drugs. Don’t miss this opportunity to stay informed—download now! @ Myelodysplastic Syndrome Treatment Drugs

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.

Myelodysplastic Syndrome 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

Myelodysplastic Syndrome Products have been categorized under various Molecule types such as

  • Recombinant fusion proteins
  • Small molecule
  • Monoclonal antibody
  • Peptide
  • Polymer
  • Gene therapy

Stay updated with the latest Myelodysplastic Syndrome Pipeline Insights—download our report for a deep dive into the next generation of therapeutics! @ Myelodysplastic Syndrome Market Drivers and Barriers, and Future Perspectives

Scope of the Myelodysplastic Syndrome Pipeline Report

  • Coverage- Global
  • 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.
  • Myelodysplastic Syndrome Therapies- Deferasirox, Bortezomib, Decitabine and cedazuridine, Luspatercept, Epoetin Alfa, and others.
  • Myelodysplastic Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
  • Myelodysplastic Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Dive deep into rich insights for new drugs for Myelodysplastic Syndrome Treatment, visit @ Myelodysplastic Syndrome Drugs

Table of Content

  1. Introduction
  2. Executive Summary
  3. Myelodysplastic Syndrome: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Myelodysplastic Syndrome– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. Tamibarotene: Syros Pharmaceuticals
  9. Mid Stage Products (Phase II)
  10. CA-4948: Curis
  11. Early Stage Products (Phase I)
  12. MNV-201: Minovia Therapeutics
  13. Preclinical and Discovery Stage Products
  14. Drug name: Company name
  15. Inactive Products
  16. Myelodysplastic Syndrome Key Companies
  17. Myelodysplastic Syndrome Key Products
  18. Myelodysplastic Syndrome- Unmet Needs
  19. Myelodysplastic Syndrome- Market Drivers and Barriers
  20. Myelodysplastic Syndrome- Future Perspectives and Conclusion
  21. Myelodysplastic Syndrome Analyst Views
  22. Myelodysplastic Syndrome Key Companies
  23. Appendix

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