Ivosidenib + IC in ND IDH1mut AML: Updated phase I results

Updated response and safety results from a phase I trial (NCT02632708), investigating ivosidenib + intensive chemotherapy (IC) in 60 adult patients with newly diagnosed (ND) acute myeloid leukemia with IDH1 mutation (IDH1^mut AML), were presented by Eytan Stein at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, December 6–9, 2025, Orlando, US. Patients received ivosidenib + induction and consolidation chemotherapy followed by single-agent ivosidenib maintenance. The primary objective was to determine the safety and tolerability of ivosidenib + standard induction and consolidation chemotherapy.

Key data: The overall rate of complete remission + complete remission with partial hematological recovery (CR + CRh) was 76.7% (confidence interval [CI], 64.0–86.6), with a median duration of CR/CRh of 25.1 months (CI, 8.3–41.6), and a median time to CR + CRh of 34 days (CI, 22–246). The median time to neutrophil count recovery during induction and consolidation therapy was 28 and 32 days, respectively, with time to platelet count recovery of 28 and 26 days, respectively. The 3-year overall survival (OS) rate was 67%, with median OS not reached (CI, 39.4 months–not estimable [NE]). Rates of treatment-emergent adverse events (TEAEs) were higher in induction and consolidation than in maintenance, with Grade ≥3 TEAEs reported in 96.7%, 97.1%, and 31.6% of patients, respectively.

Key learning: Addition of ivosidenib to intensive induction and consolidation chemotherapy followed by single-agent ivosidenib maintenance demonstrated long-term responses with an acceptable safety profile in patients with ND IDH1^mut AML, supporting its potential incorporation into standard IC regimens in this patient population.