Phase 3 trial of pevonedistat for blood cancers misses primary endpoint
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The addition of pevonedistat to azacitidine for first-line treatment of certain patients with blood cancers failed to extend EFS, according to topline data released by the agent’s manufacturer.
Pevonedistat (TAK-924, Takeda), a small-molecule inhibitor of NEDD8-activating enzyme, works by disrupting proteasomal degradation of select proteins.
As Healio previously reported, a phase 2 trial showed the addition of pevonedistat to azacitidine prolonged EFS among patients with higher-risk myelodysplastic syndrome (MDS).
The phase 3 PANTHER trial included patients with higher-risk MDS, chronic myelomonocytic leukemia and low-blast acute myeloid leukemia.
Patients received first-line azacitidine with or without pevonedistat.
EFS served as the primary endpoint. Researchers defined events as death or transformation to AML among participants with higher-risk MDS or chronic myelomonocytic leukemia, and death among participants with AML.
Results showed no statistically significant EFS benefit with pevonedistat.
“[Although] 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,” Chris Arendt, PhD, head of the oncology therapeutic area unit at Takeda, said in a company-issued press release.
The safety profile of pevonedistat-azacitidine appeared consistent with previously reported data of the combination.
Complete results from the PANTHER trial will be submitted for presentation at a medical meeting.
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