DelveInsight’s “Relapsed or Refractory Myelodysplastic Syndrome Pipeline Insights 2025” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in the Relapsed or Refractory Myelodysplastic Syndrome pipeline landscape. It covers the Relapsed or Refractory Myelodysplastic Syndrome pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Relapsed or Refractory Myelodysplastic Syndrome pipeline 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 Relapsed or Refractory Myelodysplastic Syndrome Pipeline Report

• In August 2025, ARCE Therapeutics Inc. announced a phase I/2, interventional, open-label, multicenter study to assess the safety and efficacy of ARD103 in patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome.
• DelveInsight’s Relapsed or Refractory Myelodysplastic Syndrome pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for Relapsed or Refractory Myelodysplastic Syndrome treatment.
• The leading Relapsed or Refractory Myelodysplastic Syndrome Companies such as Cantex Pharmaceuticals, Novartis, Newave Pharmaceuticals, BioTheryX, Agios Pharmaceuticals, and others.
• Promising Relapsed or Refractory Myelodysplastic Syndrome Pipeline Therapies such as CX-01, Azacitidine, SyB C-1101, NC525, LP-108, CC-91633, Talacotuzumab, Daratumumab, CC-90009, BTX-A51, and others.

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Relapsed or Refractory Myelodysplastic Syndrome Emerging Drugs Profile

• CX-01: Cantex Pharmaceuticals

CX-01 is a new chemical entity derived from unfractionated heparin with very low anticoagulant activity. CX-01 targets key protein pathways important for AML blast cell migration to the bone marrow and retention of these cells in the marrow where they are protected from chemotherapy. CX-01 also binds with proteins involved in chemotherapy resistance and the delay in platelet recovery after chemotherapy. Together, these activities are understood to sensitize AML blasts to chemotherapy and improve clinical responses. These mechanisms of action support the potential for development in myelodysplastic syndrome, multiple myeloma, and lymphomas. Currently in phase 2 of clinical trials for the treatment of Myelodysplastic Syndrome.

• LP-108: Newave Pharmaceuticals

Mechanism of Action: Proto-oncogene protein c-bcl-2 inhibitors. The drug is in phase 1 of clinical trials for the treatment of Relapsed or Refractory Myelodysplastic Syndrome

• BTX-A51: BioTheryX

BTX-A51 appears to block a specific leukemic stem cell target (CK1-alpha) as well as super enhancer targets (CDK7/CDK9) preventing transcription of key oncogenic genes. BTX-A51 has demonstrated remarkable preclinical animal efficacy implying the eradication of AML stem cells and the potential for use in multiple malignancies. Currently in phase 1 of clinical trials for the treatment of Myelodysplastic Syndrome.

The Relapsed or Refractory Myelodysplastic Syndrome Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Relapsed or Refractory 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 Relapsed or Refractory Myelodysplastic Syndrome Treatment.
• Relapsed or Refractory Myelodysplastic Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Relapsed or Refractory 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 Relapsed or Refractory Myelodysplastic Syndrome market

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Relapsed or Refractory Myelodysplastic Syndrome Companies

Cantex Pharmaceuticals, Novartis, Newave Pharmaceuticals, BioTheryX, Agios Pharmaceuticals, and others.

Relapsed or Refractory 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

• Subcutaneous
• Intravenous
• Oral
• Intramuscular
• Parenteral
• Molecule Type

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

• Small molecules
• Polymer
• Peptide
• Gene Therapy
• Monoclonal antibodies
• Product Type

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Scope of the Relapsed or Refractory Myelodysplastic Syndrome Pipeline Report

• Coverage- Global
• Relapsed or Refractory Myelodysplastic Syndrome Companies- Cantex Pharmaceuticals, Novartis, Newave Pharmaceuticals, BioTheryX, Agios Pharmaceuticals, and others.
• Relapsed or Refractory Myelodysplastic Syndrome Pipeline Therapies- CX-01, Azacitidine, SyB C-1101, NC525, LP-108, CC-91633, Talacotuzumab, Daratumumab, CC-90009, BTX-A51, and others.
• Relapsed or Refractory Myelodysplastic Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Relapsed or Refractory Myelodysplastic Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

1. Introduction
2. Executive Summary
3. Relapsed or Refractory Myelodysplastic Syndrome: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Relapsed or Refractory Myelodysplastic Syndrome – DelveInsight’s Analytical Perspective
7. In-depth Commercial Assessment
8. Relapsed or Refractory Myelodysplastic Syndrome Collaboration Deals
9. Late Stage Products (Phase III)
10. AG-120: Agios Pharmaceuticals
11. Mid Stage Products (Phase II)
12. CX-01: Cantex Pharmaceuticals
13. Early-Stage Products (Phase I)
14. BTX A51: BioTheryX
15. Inactive Products
16. Relapsed or Refractory Myelodysplastic Syndrome Key Companies
17. Relapsed or Refractory Myelodysplastic Syndrome Key Products
18. Relapsed or Refractory Myelodysplastic Syndrome- Unmet Needs
19. Relapsed or Refractory Myelodysplastic Syndrome- Market Drivers and Barriers
20. Relapsed or Refractory Myelodysplastic Syndrome- Future Perspectives and Conclusion
21. Relapsed or Refractory Myelodysplastic Syndrome Analyst Views
22. Relapsed or Refractory Myelodysplastic Syndrome Key Companies
23. Appendix

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