Rare Daily Staff

Takeda Pharmaceutical said that its experimental therapy Pevonedistat, a fist-in-class therapy for rare blood cancers, failed to meet its primary endpoint of event-free survival in a phase 3 study.

An event in the trial is defined as death or transformation to acute myeloid leukemia (AML) in participants with higher-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML), whichever occurs first, and death in participants with AML.

Pevonedistat is a NEDD8-activating enzyme inhibitor that leads to cancer cell death by disrupting protein homeostasis. The safety profile was consistent with previously reported data for this combination. The trial evaluated whether the combination of pevonedistat plus azacitidine as first-line treatment for patients with higher-risk MDS, CMML, and low-blast AML improved EFS versus azacitidine alone.

“While we are disappointed with this outcome, we are continuing to gain a greater understanding of the full data set and hope that findings from this phase 3 study will provide information to help guide research and development for potential treatment options for these underserved patient populations,” said Chris Arendt, head of Oncology Cell Therapy and Therapeutic Area Unit for Takeda.

Full data results will be submitted for presentation at an upcoming medical congress. Investigators have been informed of the outcome so they can discuss the potential impact with study participants. Takeda will work with investigators who will determine the most appropriate action for each individual patient enrolled in the study.

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