Karyopharm repots interim data from open-label Phase 1/2 study of eltanexor

Karyopharm Therapeutics announced interim data from the Phase 2 portion of the open-label Phase 1/2 study of single-agent eltanexor in patients with higher R/R myelodysplastic neoplasms. The data, featured in a poster presentation at the 17th International Congress on Myelodysplastic Syndromes, showed that eltanexor has promising single-agent efficacy with a generally manageable safety profile. As of the February 8, 2023 data cut-off date, 30 patients had been treated with 10mg eltanexor, oral, on Days 1-5 of each week. Eltanexor demonstrated a 27% overall response rate, or ORR, in the intent-to-treat, or ITT, population and a 31% ORR in the efficacy evaluable population. Median overall survival (mOS) was 8.7 months in both populations. Transfusion independence rate for red blood cells and/or platelets was 29%. Eltanexor was generally well-tolerated and manageable. The most common adverse events were asthenia, 47%; diarrhea, 43%; and nausea, 33%, the majority of which were Grade 1-2. The most common Grade greater than or equal to3 treatment-emergent AEs were neutropenia, thrombocytopenia, and asthenia.