Phase III trial of pracinostat discontinued following results from interim analysis

On July 2, 2020, it was announced that the phase III, randomized study (NCT03151408) of pracinostat in combination with azacitidine in patients with AML who are unfit to receive standard intensive chemotherapy, due to age (≥ 75 years) or comorbidities, was discontinued following results from the interim analysis. The decision was made by the Independent Data Monitoring Committee and was based on a lack of efficacy in terms of overall survival (compared to azacitidine alone) and not on safety concerns.^1,2

The study was a double-blind, placebo-controlled, multicenter trial, due to recruit 500 participants randomized into two treatment arms: (1) to receive pracinostat plus azacitidine or (2) to receive azacitidine plus placebo. Pracinostat was administered orally at 60 mg once daily, 3 times a week for 3 weeks, followed by 1 week of rest in each 28-day cycle. Azacitidine was administered subcutaneously or intravenously at 75 mg/m² daily, for 7 days of each 28-day cycle. The primary outcome measure was efficacy, based on overall survival from randomization until death from any cause, assessed up to 60 months.²

Pracinostat is an oral histone deacetylase inhibitor that received orphan drug designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) based on the phase II study (NCT01912274). It is also being investigated in phase II studies in patients with high or very high-risk myelodysplastic syndromes. Pending further evaluation, patients currently enrolled in these studies will continue treatment.