As relapsed or refractory myelodysplastic syndrome (r/r MDS) continues to grow globally and contributes to comorbidities such as diabetes, cardiovascular disease, and certain cancers, there is an increasing need for safer and more effective treatments. According to DelveInsight, the r/r MDS pipeline includes more than five pharmaceutical and biotech companies developing over five therapeutic candidates, spanning various stages of clinical and preclinical development. This highlights the robust innovation and dedication to tackling this critical public health challenge.
DelveInsight’s report, “Relapsed or Refractory Myelodysplastic Syndrome Pipeline Insight 2025,” offers a comprehensive and strategic review of the R&D landscape. It examines clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives, serving as an essential resource for researchers, healthcare investors, and decision-makers seeking insights into the evolving r/r MDS therapeutics market and the breakthroughs shaping its future.
Explore the Cutting-Edge Landscape of Relapsed or Refractory Myelodysplastic Syndrome Drug Development
Key Takeaways from the Relapsed or Refractory Myelodysplastic Syndrome Pipeline Report
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DelveInsight’s report on the relapsed or refractory myelodysplastic syndrome (r/r MDS) pipeline highlights a dynamic landscape with over five active companies developing more than five therapeutic candidates for r/r MDS treatment.
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On October 24, 2023, the FDA approved ivosidenib (Tibsovo) for adult patients with r/r MDS carrying an IDH1 mutation. This approval was based on the AG120-C-001 trial, an open-label, single-arm, multicenter study with 18 adult participants, all receiving oral ivosidenib daily in 28-day cycles. The study reported complete responses in all patients.
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Leading companies such as Cantex Pharmaceuticals, Novartis, Newave Pharmaceuticals, BioTheryX, Agios Pharmaceuticals, and others are actively developing new therapies to enhance the r/r MDS treatment landscape. Promising pipeline candidates include CX-01, LP-108, BTX-A51, among others, which are at various stages of development.
Relapsed or Refractory Myelodysplastic Syndrome Overview:
Myelodysplastic syndromes (MDS) are disorders marked by impaired blood cell production, resulting in cytopenias and genetic instability, which can eventually progress to acute myeloid leukemia (AML). Patients with MDS often experience significant clinical symptoms and face a higher risk of mortality due to both cytopenias and AML transformation. The main goals of MDS treatment are to relieve symptoms, slow disease progression, and improve survival and quality of life. Some individuals may remain symptom-free, with MDS discovered incidentally during routine blood tests. Treatment approaches for MDS include supportive care, pharmacologic therapies, and hematopoietic stem cell transplantation.
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Relapsed or Refractory Myelodysplastic Syndrome Pipeline Analysis
The Relapsed or Refractory Myelodysplastic Syndrome pipeline insights report 2025, provides insights into:
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Provides comprehensive insights into key companies developing therapies in the Relapsed or Refractory Myelodysplastic Syndrome Market.
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Categorizes Relapsed or Refractory Myelodysplastic Syndrome therapeutic companies by development stage: early, mid, and late-stage.
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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.
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Reviews emerging Relapsed or Refractory Myelodysplastic Syndrome drugs under development based on:
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Stage of development
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Relapsed or Refractory Myelodysplastic Syndrome Route of administration
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Target receptor
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Monotherapy vs. combination therapy
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Relapsed or Refractory Myelodysplastic Syndrome Mechanism of action
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Molecular type
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Offers detailed analysis of:
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Company-to-company and company-academia collaborations
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Relapsed or Refractory Myelodysplastic Syndrome Licensing agreements
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Funding and investment activities supporting future Relapsed or Refractory Myelodysplastic Syndrome market advancement.
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Unlock key insights into emerging Relapsed or Refractory Myelodysplastic Syndrome therapies and market strategies here: https://www.delveinsight.com/report-store/relapsed-or-refractory-myelodysplastic-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr
Relapsed or Refractory Myelodysplastic Syndrome Emerging Drugs
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CX-01: Cantex Pharmaceuticals
CX-01 is a novel agent derived from unfractionated heparin with minimal anticoagulant activity. It interferes with critical protein pathways that enable AML cells to migrate to and persist in the bone marrow, where they are protected from chemotherapy. CX-01 also targets proteins associated with chemotherapy resistance and delayed platelet recovery, increasing AML cells’ sensitivity to treatment and potentially improving patient outcomes. These mechanisms make CX-01 a promising therapy for myelodysplastic syndrome, multiple myeloma, and lymphomas. It is currently being tested in Phase 2 clinical trials for Myelodysplastic Syndrome.
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LP-108: Newave Pharmaceuticals
This drug acts as an inhibitor of the proto-oncogene protein c-bcl-2 and is currently being evaluated in Phase 1 clinical trials for the treatment of relapsed or refractory Myelodysplastic Syndrome.
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BTX-A51: BioTheryX
BTX-A51 is thought to target CK1-alpha in leukemic stem cells, as well as super-enhancer-associated proteins CDK7 and CDK9, thereby inhibiting the transcription of key genes that drive cancer growth. Preclinical studies in animal models have demonstrated promising outcomes, indicating that BTX-A51 may effectively eradicate AML stem cells and could have potential applications in multiple cancer types. The drug is currently being evaluated in Phase 1 clinical trials for the treatment of Myelodysplastic Syndrome.
Relapsed or Refractory Myelodysplastic Syndrome Pipeline Therapeutic Assessment
Relapsed or Refractory Myelodysplastic Syndrome Assessment by Product Type
• Mono
• Combination
• Mono/Combination
Relapsed or Refractory Myelodysplastic Syndrome 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
Relapsed or Refractory Myelodysplastic Syndrome Assessment by Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical
Relapsed or Refractory Myelodysplastic Syndrome Assessment by Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
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Table of Contents
1. Report Introduction
2. Executive Summary
3. Relapsed or Refractory Myelodysplastic Syndrome Current Treatment Patterns
4. Relapsed or Refractory Myelodysplastic Syndrome - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Relapsed or Refractory Myelodysplastic Syndrome Late-Stage Products (Phase-III)
7. Relapsed or Refractory Myelodysplastic Syndrome Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Relapsed or Refractory Myelodysplastic Syndrome Discontinued Products
13. Relapsed or Refractory Myelodysplastic Syndrome Product Profiles
14. Relapsed or Refractory Myelodysplastic Syndrome Key Companies
15. Relapsed or Refractory Myelodysplastic Syndrome Key Products
16. Dormant and Discontinued Products
17. Relapsed or Refractory Myelodysplastic Syndrome Unmet Needs
18. Relapsed or Refractory Myelodysplastic Syndrome Future Perspectives
19. Relapsed or Refractory Myelodysplastic Syndrome Analyst Review
20. Appendix
21. Report Methodology
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