Bristol Myers Squibb's Idhifa Flunks Acute Myeloid Leukemia Trial

Failure

Bristol Myers Squibb announced that the Phase III IDHENTIFY trial of Idhifa (enasidenib) plus best supportive care (BSC) compared to conventional care, did not meet the primary endpoint of overall survival (OS) in relapsed or refractory acute myeloid leukemia (r/r AML) with an isocitrate dehydrogenase-2 (IDH2) mutation.

Specifically, the trial evaluated Idhifa versus conventional care regimens, which include best support care (BSC) only, azacytidine plus BSC, low-dose cytarabine plus BSC or intermediate-dose cytarabine plus BSC. The patients were 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 endpoint was overall survival. Key secondary endpoints included overall response rate, event-free survival, duration of response and time to response.

Idhifa is approved by the U.S. Food and Drug Administration (FDA) for adults with r/r AML with IDH2 mutation detected by an FDA-approved test. It is also approved in Australia and Canada. It was approved for that indication in August 2017. The IDH2 mutation is seen in about 19% of AML patients.

“While we are disappointed by the outcome of the IDHENTIFY study, we remain confident in Idhifa’s established role as a treatment option for patients with relapsed or refractor AML with an IDH2 mutation and are grateful to all those who participated in the study,” said Noah Berkowitz, senior vice president, Global Clinical Development, Hematology, for Bristol Myers Squibb. “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.”

Yesterday, Bristol Myers Squibb announced plans to acquire Montreal-based Forbius. Forbius has a portfolio of highly selective and potent inhibitors of TGF-beta 1 and 3, which are key mediators of immunosuppression and fibrosis. Before the close, Forbius will transfer its non-TGF-beta assets to a newly formed private company, which will be held by the company’s existing shareholders.

Bristol Myers Squibb picks up the TGF-beta program, including its lead assets, AVID200. The initial focus with AVID200 will be in oncology, although it may consider developing it in other disease areas, such as fibrosis.

They expect the transaction to be completed in the fourth quarter of 2020. No financial details were disclosed, other than that Forbius will receive an upfront payment and success-based milestone payments.

“With this acquisition, we extend our leading position in oncology by including new pathways that complement our expansive oncology pipeline with the potential to serve more patients with cancer, including those who may not respond to immunotherapy,” said Rupert Vessey, executive vice president and president, Research & Early Development, Bristol Myers Squib. “As a science driven company, this transaction shows our continued commitment to source innovation internally and externally to develop new treatments for patients with significant unmet medical needs.”

Ilia A. Tikhomirov, president and chief executive officer of Forbius, added, “Our portfolio of highly selective TGF-beta inhibitors has shown potential across a broad range of therapeutic areas. We are proud that Bristol Myers Squibb recognizes this potential given their global leadership in oncology and unique position to translate innovative science into meaningful treatments for patients with cancer across the globe.”

Back to news