Benzinga | Jul 1, 2021 08:08AM EDT

Rafael Pharmaceuticals Announces Preplanned Interim Futility Analysis Of Pivotal Phase 3 Trial Of CPI-613 For Relapsed Or Refractory Acute Myeloid Leukemia; Says Independent Data Monitoring Committee Recommended Co. Continue Trial As Is

Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a leader in the growing field of cancer metabolism-based therapeutics, announced positive outcome from its preplanned interim futility analysis of its pivotal Phase 3 trial (ARMADA 2000) of CPI-613(r) (devimistat) for relapsed or refractory acute myeloid leukemia (AML) as determined by an independent Data Monitoring Committee (IDMC). The IDMC met on Friday, Jun. 25 and based on the 142 within the intention to treat population, the trial was determined to be non-futile. The IDMC recommended to continue the trial as is.

"The findings from this interim futility analysis further solidify the potential that devimistat holds in safely and effectively treating AML and other hard-to-treat cancers," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "As we continue to advance devimistat through clinical trials, we remain hopeful for the cancer community."

ARMADA 2000 is a Phase 3, multicenter, open-label, randomized, pivotal trial that is evaluating the efficacy and safety of its lead compound devimistat in combination with high dose cytarabine and mitoxantrone in older patients with relapsed or refractory AML. The futility analysis compared two cohorts -- one that received devimistat in combination with high dose cytarabine and mitoxantrone and the other that received only high dose cytarabine and mitoxantrone therapy and control subgroups: combination of mitoxantrone, etoposide and cytarabine, and combination of fludarabine, cytarabine and filgrastim. This analysis demonstrated that the response rate achieved the predefined threshold for continuation of the study.