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
For further information, refer to the detailed Myelodysplastic Syndrome Unmet Needs, Myelodysplastic Syndrome Market Drivers, and Myelodysplastic Syndrome Market Barriers, click here for Myelodysplastic Syndrome Ongoing Clinical Trial Analysis
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
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 (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, 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 (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
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