Receive our newsletter – data, insights and analysis delivered to you
  1. News
September 2, 2021

Takeda’s pevonedistat fails to meet primary goal in Phase III leukaemia trial

The safety profile of pevonedistat plus azacitidine in the PANTHER trial was in line with already established data. 

Takeda Pharmaceutical has reported that its investigational drug, pevonedistat, in combination with azacitidine failed to meet the primary goal of the Phase III PANTHER (Pevonedistat-3001) clinical trial in patients with rare leukaemias.

A NEDD8-activating enzyme (NAE) inhibitor, pevonedistat can interrupt protein homeostasis and subsequently lead to cancer cell death.

The trial analysed if pevonedistat combination as first-line therapy could boost event-free survival (EFS) in patients with higher-risk myelodysplastic syndromes (HR-MDS), chronic myelomonocytic leukaemia (CMML) and low-blast acute myeloid leukaemia (AML) compared with azacitidine alone.

Death or change to AML in higher-risk MDS or CMML patients, whichever happens first, and death in AML patients, were defined as an event in the trial.

Data showed that the PANTHER trial did not meet the pre-defined statistical significance for EFS.

Furthermore, the safety profile of the combination therapy was in line with already reported results.

Content from our partners
Why this global life sciences COO believes relocation to Charleston, SC, was key to achieving next-level success
Patient-centric pharma logistics: How CRYOPDP delivers hope worldwide
Why Asia-Pacific is the next frontier for decentralized clinical trials

Takeda Pharmaceutical oncology cell therapy and therapeutic area unit head Chris Arendt said: “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 III study will provide information to help guide research and development for potential treatment options for these underserved patient populations.”

The company notified the investigators of the results and will work with them to decide on the suitable action for patients who are part of the trial.

In July 2020, the US Food and Drug Administration granted breakthrough therapy status to pevonedistat to treat HR-MDS patients.

The combination therapy was demonstrated to be a very active, favourable treatment approach in the Phase II Pevonedistat-2001 trial.

It had also indicated benefit in the HR-MDS subgroup across various clinically meaningful endpoints such as overall survival, EFS, complete remission and transfusion independence.

Takeda reported positive data from the Phase III trial assessing recombinant von Willebrand factor prophylaxis for von Willebrand disease in July 2021.

Related Companies

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy