Takeda's pevonedistat misses Phase III goal

Japan’s largest drugmaker Takeda Pharmaceutical (TYO: 4502) today revealed that the Phase III PANTHER (Pevonedistat-3001) study did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS), with the news edging its share down 1% to 3,689 yen by close of trading.

The trial evaluated whether the combination of pevonedistat plus azacitidine as first-line treatment for patients with higher-risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myeloid leukemia (AML) improved EFS versus azacitidine alone. An event in the trial is defined as death or transformation to AML in participants with higher-risk MDS or CMML, whichever occurs first, and death in participants with AML.

Pevonedistat was granted Breakthrough Therapy designation by the US Food and Drug Administration in for its investigational drug pevonedistat for the treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS), a rare form of bone marrow-related cancer, in July last year.