Aprea faces another disappointment this week in the clinic. The FDA held up a yellow card, and the Boston-based biotech has placed a partial clinical hold on its myeloid malignancy program.
Aprea Therapeutics faces another disappointment this week in the clinic. The Food and Drug Administration (FDA) held up a yellow card, and the Boston-based biotech has placed a partial clinical hold on its myeloid malignancy program.
Aprea’s lead candidate, eprenetapopt, is a small molecule that reactivates the p53 ‘cancer guardian’ protein. Approximately half of the human tumors experience a mutation of p53 that results in treatment-resistant cancers and poor survival.
The drug is currently being tested in combination with chemo drug azacitidine in 20 patients in Aprea’s myeloid malignancy programs, including MDS, AML and post-transplant maintenance trials. The hold doesn’t cancel the test for those benefiting from treatment but does prevent new patients from being added for now.
While Aprea wasn’t overly forthcoming with details on the hold, the CEO stated that “safety is our highest priority” alluding to the hold potentially being called due to toxicity. The company said it will work with the FDA, analyzing data and answering questions to resolve the delay as soon as possible.
“Based on the totality of the data we have for eprenetapopt, we believe that it continues to be a promising therapeutic option for cancer patients. We are working closely with the FDA to review the data specific to eprenetapopt with azacitidine in our myeloid malignancy trials and will provide an update when we have additional information,” said CEO Christian S. Schade.
Eprenetapopt has been granted Orphan Drug and Fast Track designations from the FDA for both MDS and AML. Aprea announced positive results from its Phase II of Eprenetapopt + Azacitidine for Post-Transplant Maintenance Therapy in TP53 Mutant MDS and AML. At one year post-transplant, 58% of the 33 patients enrolled had relapse-free survival, while the overall survival was 79%.
However, the drug failed to meet its primary endpoint in Phase III data shared in December. The complete response rate for MDS patients was 33.3% in eprenetapopt + azacitidine arm, versus 22.4% in the AZA alone arm. The results were not considered statistically significant.
The drug is also being tested in lymphoid malignancies and solid tumors. The hold isn’t affecting those designations right now.
Aprea’s second pipeline candidate is a next-gen p53 reactivator that is still in the preclinical stage for hematologic malignancies.
