Myelodysplastic Syndrome Drug Development Pipeline Analysis 2025

DelveInsight's comprehensive "Myelodysplastic Syndrome Pipeline Insights 2025" analysis delivers thorough insights about over 120 organizations and more than 150 investigational medications within the Myelodysplastic Syndrome development landscape. The report encompasses drug candidate profiles across both clinical and preclinical phases, offering therapeutic evaluations organized by product category, developmental phase, administration method, and molecular classification. Additionally, it spotlights dormant pipeline candidates in this therapeutic area.

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Major Highlights from the Myelodysplastic Syndrome Pipeline Analysis

December 15, 2025 – FibroGen, Inc. (NASDAQ: FGEN) disclosed that the U.S. Food and Drug Administration's Office of Orphan Products Development has awarded Orphan Drug Designation to roxadustat for managing myelodysplastic syndromes (MDS).

December 15, 2025 – Institut de Recherches Internationales Servier launched a clinical investigation enrolling patients diagnosed with myelodysplastic syndromes (MDS) harboring an isocitrate dehydrogenase 1 (IDH1) mutation who have not previously received hypomethylating agent therapy. Participants will be randomized to receive either ivosidenib (IVO) monotherapy or azacitidine (AZA) monotherapy. IVO will be administered daily across each 28-day treatment cycle, while AZA will be given during the first seven days of each cycle. Study visits are scheduled weekly during Cycle 1 and on Day 1 of each subsequent cycle.

December 08, 2025 – Groupe Francophone des Myelodysplasies performed a Phase I/II multicenter investigation evaluating the combination of luspatercept in patients diagnosed with lower-risk MDS without ring sideroblasts (LR-MDS without RS) who have failed or are ineligible for erythropoiesis-stimulating agents (ESAs).

December 05, 2025 – Bristol Myers Squibb launched a Phase III clinical investigation comparing the efficacy and safety of luspatercept versus epoetin alfa for managing anemia in ESA-naïve, non–transfusion-dependent adults diagnosed with IPSS-R very low-, low-, or intermediate-risk MDS.

DelveInsight's Myelodysplastic Syndrome development pipeline analysis reveals a dynamic field with over 120 active organizations advancing more than 150 investigational therapies for Myelodysplastic Syndrome management.

Premier Myelodysplastic Syndrome pharmaceutical organizations include 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, among others.

Notable Myelodysplastic Syndrome investigational agents include Deferasirox, Bortezomib, Decitabine and cedazuridine, Luspatercept, Epoetin Alfa, among others.

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Myelodysplastic Syndrome Disease Background

Myelodysplastic syndrome (MDS) represents a heterogeneous group of hematologic neoplasms classically described as a clonal disorder of hematopoietic stem cells resulting in dysplasia and ineffective hematopoiesis in the bone marrow. Some patients diagnosed with MDS may experience transformation into acute myeloid leukemia (AML). MDS is usually diagnosed in older patients beyond the age of 65. Clinical manifestations encompass a decrease in the number of red blood cells (RBC), platelets, and white blood cells (WBC). The disease course is variable.

Emerging Myelodysplastic Syndrome Drug Candidates Profile

Tamibarotene: Syros Pharmaceuticals

Tamibarotene (formerly SY-1425) represents 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 demonstrate 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 result in 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.

RVU120: Ryvu Therapeutics

RVU120 (SEL120) represents 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 enhance proliferation and induce erythroid differentiation of CD34+ cells derived from Diamond-Blackfan anemia (DBA) patients. Currently, this drug is in Phase II stage of its development for managing Myelodysplastic syndromes.

Emavusertib: Curis

Emavusertib, which is under development by Curis, represents 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 function in this pathway, it is considered an attractive target for the generation of therapeutics to manage 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, this drug is in Phase I/II stage of its development for managing Myelodysplastic syndromes.

MNV-201: Minovia Therapeutics

MNV-201 represents 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 investigation'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 Analysis Delivers Insights into

The analysis provides comprehensive insights about organizations developing therapeutic interventions for Myelodysplastic Syndrome management with aggregate therapies developed by individual companies for this indication.

It evaluates various therapeutic candidates organized into early-stage, mid-stage, and late-stage development for Myelodysplastic Syndrome Treatment.

Myelodysplastic Syndrome pharmaceutical organizations are engaged in targeted therapeutic development with corresponding active and inactive (dormant or discontinued) programs.

Myelodysplastic Syndrome investigational drugs categorized by developmental phase, administration route, target receptor, monotherapy or combination approach, various mechanisms of action, and molecular classification.

Comprehensive analysis of partnerships (company-company collaborations and company-academic partnerships), licensing agreements, and financial arrangements for future progression of the Myelodysplastic Syndrome marketplace.

From early-stage research to late-phase Myelodysplastic Syndrome Clinical Investigations, our evaluation covers key organizations, innovative treatment approaches, and the next wave of Emerging Drugs—Access now! @ Myelodysplastic Syndrome Treatment Drugs

Myelodysplastic Syndrome Pharmaceutical Organizations

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, among others.

The Myelodysplastic Syndrome pipeline analysis delivers therapeutic evaluation of pipeline medications by Administration Route. Agents have been classified under various administration routes including:

• Oral

• Intravenous

• Subcutaneous

• Parenteral

• Topical

Myelodysplastic Syndrome Products have been organized under various Molecular classifications including:

• Recombinant fusion proteins

• Small molecule

• Monoclonal antibody

• Peptide

• Polymer

• Gene therapy

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Coverage of the Myelodysplastic Syndrome Pipeline Analysis

Coverage: Global

Myelodysplastic Syndrome Pharmaceutical Organizations: 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, among others.

Myelodysplastic Syndrome Therapeutic Candidates: Deferasirox, Bortezomib, Decitabine and cedazuridine, Luspatercept, Epoetin Alfa, among others.

Myelodysplastic Syndrome Therapeutic Evaluation by Product Category: Mono, Combination, Mono/Combination

Myelodysplastic Syndrome Therapeutic Evaluation by Clinical Phases: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Report Structure

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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