Therapy Detail
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| Therapy Name | RMC-4550 + Venetoclax |
| Synonyms | |
| Therapy Description | |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
|---|---|---|---|---|
| RMC-4550 | RMC4550|RMC 4550 | SHP2 Inhibitor 20 | RMC-4550 is a small molecule allosteric inhibitor of Shp2 that blocks Ras-Raf-Mek-Erk pathway signaling, leading to tumor growth inhibition (PMID: 30104724). | |
| Venetoclax | Venclexta | ABT-199|RG7601|GDC-0199|ABT119|Venclyxto | BCL2 inhibitor 27 | Venclexta (venetoclax) is a BH3-mimetic that binds to and inhibits BCL2, resulting in increased tumor cell apoptosis (PMID: 26589495, PMID: 25048785). Venclexta (venetoclax) is FDA approved for use in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), and in combination with chemotherapy in patients 75 years old or older with newly-diagnosed acute myeloid leukemia (FDA.gov). |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| FLT3 exon 14 ins | acute myeloid leukemia | sensitive | RMC-4550 + Venetoclax | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, the combination of RMC-4550 and Venclexta (venetoclax) synergistically inhibited viability and induced apoptosis in acute myeloid leukemia cell lines harboring FLT3 ITD in culture, reduced tumor burden and improved survival in a cell line xenograft model, and reduced leukemia burden in a patient-derived xenograft (PDX) model (PMID: 37992684). | 37992684 |
| KIT N822K | acute myeloid leukemia | sensitive | RMC-4550 + Venetoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of RMC-4550 and Venclexta (venetoclax) synergistically inhibited viability and induced apoptosis in an acute myeloid leukemia cell line harboring KIT N822K in culture, and reduced tumor burden and improved survival in a cell line xenograft model (PMID: 37992684). | 37992684 |
| FLT3 exon 14 ins NRAS G12C | acute myeloid leukemia | sensitive | RMC-4550 + Venetoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of RMC-4550 and Venclexta (venetoclax) synergistically inhibited viability and induced apoptosis in an acute myeloid leukemia cell line harboring FLT3 ITD and NRAS G12C in culture, and reduced tumor burden and improved survival in a cell line xenograft model (PMID: 37992684). | 37992684 |
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|
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