On September 1, 2021 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported that the Phase 3 PANTHER (Pevonedistat-3001) study did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS) (Press release, Takeda, SEP 1, 2021, View Source [SID1234587122]). 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.
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"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, PhD, Head, Oncology Cell Therapy and Therapeutic Area Unit, Takeda. "We would like to thank the patients, families, advocacy organizations and investigators that participated in this trial, without whom this meaningful research would not have been possible. Takeda remains committed to conducting important research and transforming the lives of patients with cancer."
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.
About Pevonedistat
Pevonedistat is a NEDD8-activating enzyme (NAE) inhibitor that leads to cancer cell death by disrupting protein homeostasis. The Phase 3 PANTHER study (Pevonedistat-3001) did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS) and the data results are currently being evaluated. The safety profile was consistent with previously reported data for this combination. Pevonedistat is an investigational drug for which safety and efficacy have not been established.
Takeda’s Commitment to Oncology
Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it’s with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.