Takeda said that Phase III PANTHER, which studied the effectiveness of pevonedistat for different types of leukemia, did not produce significant results in terms of event-free survival.
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Japanese biopharmaceutical firm Takeda Pharmaceutical announced Wednesday that its drug candidate for leukemia failed to meet its primary endpoint in phase III.
In a statement, the company said that the Phase III PANTHER ((Pevonedistat-3001) study, which looked at the effectiveness of pevonedistat when combined with azacitidine as a first-line treatment for different types of leukemia, did not produce significant results in terms of event-free survival (EFS).
The trial observed the combination for treating chronic myelomonocytic leukemia (CMML), myelodysplastic syndromes (MDS) and low-blast acute myeloid leukemia (AML). The goal was to determine whether EFS would be significantly improved with the drug combination versus the azacitidine alone. The researchers defined an “event” as either a transformation to AML or death in patients with higher-risk MDS or CMML, or death in patients who already had AML.
CMML is a rare type of cancer that occurs in four out of one million people in the U.S. per year, accounting for roughly 1,100 cases annually. It starts in the blood-forming cells in the bone marrow and then invades the blood. It can affect any cell in any part of the body. Approximately nine out of 10 CMML cases occur in people aged 60 years old and over, more often in men than in women.
Statistics for MDS are yet to be established in the U.S., though researchers estimate the number of cases to be at about 10,000 per year. MDS occurs when the blood-forming cells in the bone marrow become abnormal. This type of cancer is usually observed after the age of 50, and the risk increases with after that. It is most common in people who are at least 70 years old.
AML accounts for around 1% of all cancer cases in the U.S., affecting 20,240 people of all ages. It is the second most common type of leukemia found in children and adults, with 31% of cases occurring in the latter.
Pevonedistat is a NEDD8-activating enzyme (NAE) inhibitor that results in the death of cancer cells by halting protein homeostasis. Its safety and efficacy have yet to be established. In the Phase III PANTHER trial, data showed that pevonedistat could not achieve EFS goals, although its safety profile had remained consistent with previous studies for the same combination.
“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,” said Takeda Oncology Cell Therapy and Therapeutic Area Unit head Chris Arendt, Ph.D.
Full data results from the Phase III PANTHER study will be shared at Takeda’s upcoming medical congress. The company is also currently speaking to the study participants’ families to ascertain the trial’s potential impact on the patients. The company is coordinating with the research investigators to determine the best possible treatment plan for each patient following the trial.
