Adult R/R AML pts with a central lab–confirmed
As of 18 Jan 2021, 30 pts were treated, with 17 remaining on treatment. In the prespecified 9 PK-evaluable pts, the Cmax (4730 ng/mL) was reached 3.98 h after single-dose administration and AUC0-24 was 62100 ng*h/mL. Systemic exposure parameters demonstrated moderate to high variability. The CR+CRh rate was 30.0% (9/30; 95% CI: 14.7–49.4%), with all 9 pts achieving CR. Median duration of CR+CRh was not reached (range: 0.03–10.09mos) and median time to CR+CRh was 2.79 mos (range: 1.0–6.5). Objective response rate was 36.7% (11/30; 95% CI: 19.9–56.1%). One (3.3%) pt received hematopoietic stem cell transplantation after achieving CR. Transfusion independence was achieved in 7/18 pts (38.9%) and maintained in 8/12 pts (66.7%). All pts reported treatment-emergent adverse events (TEAEs). Grade ≥3 TEAEs occurred in 26 (86.7%) pts, most commonly platelet count decreased (36.7%), neutrophil count decreased (33.3%) and anemia (33.3%). Two fatal TEAEs occurred in 2 (6.7%) pts, with neither related to ivosidenib. Serious AEs were reported in 18 (60.0%) pts.
Ivosidenib was well tolerated and induced durable remissions with clinical benefits in Chinese pts with mIDH1 R/R AML, potentially fulfilling an unmet medical need for this rare pt population in China.
Y. Zhang, R. Chen, Z. Shen, X. Yu, K. Liu: Financial interests, Personal, Full or part-time Employment: CStone Pharmaceuticals. J. Yang: Financial Interests, Personal, Officer: CStone Pharmaceuticals; Financial Interests, Personal, Stocks/Shares: CStone Pharmaceuticals. Financial interests, Personal, Full or part-time Employment: CStone Pharmaceuticals. All other authors have declared no conflicts of interest.