The combination of ASTX727, a fixed-dose formulation of decitabine and cedazuridine, plus venetoclax was safe and demonstrated efficacy among patients with acute myeloid leukemia (AML), particularly among the advanced elderly and patients unfit for chemotherapy, according to the results of a phase 2 study presented at the 2022 ASCO Annual Meeting.
The standard of care for patients with AML is hypomethylating agents combined with venetoclax for patients who are ineligible for intensive chemotherapy. The aim of this study was to determine if a complete oral regimen including ASTX72 was feasible and safe in this population.
The phase 2 trial treated 28 patients with AML, including 15 as their first-line therapy, with ASTX727 plus venetoclax. Patients were eligible if they had relapsed or refractory disease, or previously untreated aged 75 or older or had comorbidities that precluded the use of intensive chemotherapy.
At baseline, the median age was 75 for the entire study population, which included a median age of 81 in the frontline cohort and 72 in the relapsed/refractory cohort. Patients with relapsed/refractory disease had received a median of 2 prior lines of therapy. Both cohorts received a median of 2 cycles of ASTX727 plus venetoclax.
The ORR was 61% and 45% in the frontline and relapsed/refractory cohorts, respectively. The median OS was not yet reached in the frontline cohort and was 7.2 months in the relapsed/refractory cohort during a median follow-up of 5 months.
Treatment-related grade 3 or higher adverse events were primarily associated with myelosuppression, including neutropenic infection and elevated livre enzymes.
The authors concluded that “total oral therapy of ASTX727 plus venetoclax is safe and feasible in the advanced elderly population, and demonstrates significant efficacy in patients unfit for chemotherapy.”
Disclosures: This study was supported by Taiho.
Ravandi F, Abuasab T, Valero YA, et al. Phase 2 study of ASTX727 (cedazuridine/decitabine) plus venetoclax (ven) in patients with relapsed/refractory acute myeloid leukemia (AML) or previously untreated, elderly patients (pts) unfit for chemotherapy. Presented at ASCO 2022; June 3-7, 2022. Abstract 7037.