The phase 3 PANTHER study, assessing pevonedistat, an investigational NEDD8-activating enzyme inhibitor, plus azacytidine for patients with certain hematologic malignancies, failed to meet its primary endpoint of improved event-free survival compared with azacytidine alone, according to a press release from the manufacturer.  . 

The trial assessed the treatment combination among patients with higher-risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and low-blast acute myeloid leukemia (AML)

Event-free survival served as the primary endpoint, and an event was defined as death or transformation to AML for patients with higher-risk MDS or CMML, whichever occurred first, and death in patients with AML. 

“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,” Chris Arendt, PhD, head of the oncology cell therapy and therapeutic area Unit atTakeda, said in the release. 

The complete data from the study is expected to be submitted for presentation at an upcoming conference. The release noted that the company will work with investigators to determine the best course of action for patients enrolled in the study.