
A phase II trial assessed E7820's efficacy in patients with recurrent or refractory splicing factor-mutant AML, MDS, or CMML. Patients received 100 mg doses of E7820 daily in 28-day cycles until specific endpoints. The study was conducted in collaboration with Eisai Pharmaceuticals and approved by Institutional Review Boards. The primary objective was to evaluate the overall response rate (ORR) within 6 cycles. However, the study was terminated early due to limited efficacy. RBM39 degradation was observed, and the safety profile of E7820 was consistent with prior studies, suggesting potential use in combination therapies.
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