DelveInsight’s, “Myelodysplastic Syndrome Pipeline Insight 2023” report provides comprehensive insights about 120+ companies and 150+ pipeline drugs in the Myelodysplastic Syndrome pipeline landscape. It covers the Myelodysplastic Syndrome pipeline drug profiles, including Myelodysplastic Syndrome clinical trials 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
- DelveInsight’s Myelodysplastic Syndrome Pipeline report depicts a robust space with 120+ active players working to develop 150+ pipeline therapies for Myelodysplastic Syndrome.
- 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
- Promising Myelodysplastic Syndrome Pipeline Therapies include Luspatercept, Lenalidomide 5 mg, Tamibarotene + Azacitidine, Group 1: Epoetin alfa, Lemzoparlimab+Azacitidine, and others
- The Myelodysplastic Syndrome companies and academics are working to assess challenges and seek opportunities that could influence Myelodysplastic Syndrome R&D. The Myelodysplastic Syndrome therapies under development are focused on novel approaches to treat/improve Myelodysplastic Syndrome.
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Myelodysplastic Syndrome Overview
Myelodysplastic syndrome (MDS) is a heterogeneous group of hematologic neoplasms classically described as a clonal disorder of hematopoietic stem cells leading to dysplasia and ineffective hematopoiesis in the bone marrow. Some patients with MDS may have a transformation into acute myeloid leukemia (AML). MDS is usually diagnosed in older patients over the age of 65. Clinical manifestations include a decrease in the number of red blood cells (RBC), platelets, and white blood cells (WBC). The disease course is variable.
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Myelodysplastic Syndrome Emerging Drugs
- MBG453: Novartis
Sabatolimab (MBG453) is a humanized monoclonal antibody (hIgG4, S228P) directed against the human T-cell immunoglobulin domain and mucin domain-3 (TIM-3). TIM-3 is a novel target expressed on multiple immune cell types and leukemic cells and blasts but not on the normal stem cells that induce blood formation. Currently, the drug is in the Phase III stage of its development for the treatment of Myelodysplastic syndromes.
- Tamibarotene: Syros Pharmaceuticals
Tamibarotene (formerly SY-1425) is an oral selective retinoic acid receptor alpha (RARa) 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 RARa 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 RARa, 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. A first-in-human Phase Ib clinical trial with RVU120 in patients with AML or high-risk (HR)-MDS is currently ongoing. 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.
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Myelodysplastic Syndrome Pipeline Therapeutics Assessment
There are approx. 120+ key companies which are developing the therapies for Myelodysplastic Syndrome. The companies which have their Myelodysplastic Syndrome drug candidates in the most advanced stage, i.e. Phase III include, Novartis.
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 Pipeline Therapies- Luspatercept, Lenalidomide 5 mg, Tamibarotene + Azacitidine, Group 1: Epoetin alfa, Lemzoparlimab+Azacitidine, and others.
- Myelodysplastic Syndrome Pipeline Segmentation: Product Type, Molecule Type, Route of Administration
Got Queries? Find out the related information on Myelodysplastic Syndrome Mergers and acquisitions, Myelodysplastic Syndrome Licensing Activities @ Myelodysplastic Syndrome Emerging Drugs, and Recent Trends
Table of Content
- Introduction
- Executive Summary
- Myelodysplastic Syndrome: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Myelodysplastic Syndrome– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- MBG453: Novartis
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- CA-4948: Curis
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- RVU120: Ryvu Therapeutics
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug name: Company name
- Drug profiles in the detailed report…..
- Inactive Products
- Myelodysplastic Syndrome Key Companies
- Myelodysplastic Syndrome Key Products
- Myelodysplastic Syndrome- Unmet Needs
- Myelodysplastic Syndrome- Market Drivers and Barriers
- Myelodysplastic Syndrome- Future Perspectives and Conclusion
- Myelodysplastic Syndrome Analyst Views
- Myelodysplastic Syndrome Key Companies
- Appendix
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