Phase 3 IDHENTIFY Study Fails to Meet Primary End Point of Overall Survival

The phase 3 IDHENTIFY study evaluating enasidenib (Idhifa) plus best supportive care versus conventional care regimens did not meet the primary end point of overall survival (OS) in patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation, according to Bristol Myers Squibb, the developer of the agent.

Notably, the safety profile of enasidenib was consistent with previously reported findings.

“While we are disappointed by the outcome of the IDHENTIFY study, we remain confident in [enasidenib’s] established role as a treatment option for patients with relapsed or refractory AML with an IDH2 mutation and are grateful to all those who participated in the study,” Noah Berkowitz, MD, PhD, senior vice president of Global Clinical Development and Hematology at Bristol Myers Squibb, said in a press release. “AML is one of the most difficult-to-treat blood cancers, and we’re committed to furthering our research and improving on the standards of care for patients living with this aggressive disease.”

The international, multicenter, open-label, randomized, phase 3 IDHENTIFY study compared the efficacy and safety of enasidenib versus conventional care regimens, including continuous 28-day cycles of best supportive care only, azacitidine (Vidaza) subcutaneously plus best supportive care, low-dose cytarabine subcutaneously plus best supportive care, or intermediate-dose cytarabine intravenously plus best supportive care, in patients aged 60 years or older with AML refractory to or relapsed after second- or third-line AML therapy and positive for an IDH2 mutation. The primary end point of the study was OS, and key secondary end points included overall response rate, event-free survival, duration of response, and time to response.

Moving forward, Bristol Myers Squibb indicated it will complete a full evaluation of the IDHENTIFY data and work with investigators to present results from the study at a future medical meeting.

Bristol Myers Squibb received full approval in the US for enasidenib in August 2017 for the treatment of adult patients with relapsed or refractory AML with an IDH2 mutation as detected by an FDA-approved test. Enasidenib is the first and only FDA-approved therapy for patients with relapsed or refractory AML and positive for an IDH2 mutation, which represents up to 19% of patients with AML. Additionally, enasidenib is also approved in Australia and Canada.

Reference:

Bristol Myers Squibb Provides Update on Phase 3 IDHENTIFY Trial in Patients with Relapsed or Refractory Acute Myeloid Leukemia [news release]. Princeton, NJ. Published August 25, 2020. Accessed August 25, 2020. https://news.bms.com/press-release/corporatefinancial-news/bristol-myers-squibb-provides-update-phase-3-idhentify-trial-p