Olutasidenib Achieves Favorable Reponses in mIDH1 AML in Five-Year Analysis

By Melissa Badamo - Last Updated: August 30, 2024

Olutasidenib continues to produce rapid and durable responses in patients with relapsed or refractory acute myeloid leukemia (AML) with IDH1 mutation (mIDH1), according to a recent study.

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Jorge Cortes, MD, of the Georgia Cancer Center, and colleagues reported final five-year results from a registrational, phase II, open-label, multicenter trial published in the Journal of Clinical Oncology.

A total of 153 patients received olutasidenib at 150mg twice daily. The primary endpoint was complete remission (CR) plus CR with partial hematologic recovery (CRh).

Of the 147 efficacy-evaluable patients, 32% (95% CI, 24.5-40.2) achieved CR and 35% (95% CI, 27-43; P<.0001) achieved CRh. The median time to CR/CRh was 1.9 months (0.9-5.6), while the median duration of CR plus CRh was 25.3 months (95% CI, 13.5-not reached).

The overall response rate (ORR) was 48% (95% CI, 40-56.7), with a median duration of 15.5 months (95% CI, 7.4-26.2). The median overall survival (OS) was 11.6 months (95% CI, 8.9-15.5).

In the 12 patients who did not respond to prior venetoclax treatment, the CR plus CRh rate was 33%, and the median duration of CR plus CRh was not reached. The median OS was 16.2 months (95% CI, 2.6-not reached).

Of those who were transfusion-dependent at baseline, 39% reached transfusion independence from red blood cells and 41% from platelets. Following treatment, 11% of patients continued to hematopoietic stem cell transplantation (HSCT).

The safety profile of olutasidenib was comparable with previous data, the authors noted. The most frequent adverse events included febrile neutropenia (22%), constipation (27%), diarrhea (21%), nausea (39%), fatigue (23%), pyrexia (24%), hypokalemia (22%), a decrease in red blood cell count (26%), and an increase in white blood cell count (25%).

Fourteen percent of patients experienced differentiation syndrome, but no new instances were reported in the current five-year analysis.

“This analysis provides an additional two years of data beyond the results that led to [US Food and Drug Administration] approval of olutasidenib,” Dr. Cortes and colleagues concluded. “This first report of the five-year data further demonstrates the rapid and durable responses observed with olutasidenib in heavily pretreated patients with mIDH1 AML, including those [relapsed or refractory] to prior venetoclax.”

Reference

Cortes JE, Jonas BA, Watts JM, et al. Olutasidenib for mutated IDH1 acute myeloid leukemia: Final five-year results from the phase 2 pivotal cohort. JCO. 2024;42(16):6528-6528. doi:10.1200/JCO.2024.42.16_suppl.6528

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