FDA orders clinical hold on Aprea's p53 reactivator as struggling biotech pledges to address safety concerns
Little Aprea Therapeutics just tripped yet again on its rocky path to develop a drug that can reactivate the mutant tumor suppressor protein p53.
The Boston-based biotech said the FDA has placed a partial clinical hold on its trials of eprenetapopt, where the drug is combined with the chemo azacitidine as an experimental treatment for multiple myeloid malignancies.
While Aprea didn’t specify the reason for the hold or what exact questions the agency is asking, CEO Christian Schade hinted in a statement it has to do with toxicity concerns.
“Patient safety is our highest priority,” he said. “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.”
Aprea reported disappointing results at the end of last year, as adding eprenetapopt to azacitidine failed to outperform chemo alone in terms of spurring responses among patients with TP53 mutant myelodysplastic syndromes.
Shares $APRE in the company, which fell off a cliff following the readout, dropped another 9.77% pre-market to $4.30.
Across the MDS, AML and post-transplant maintenance trials, around 20 patients are currently receiving the regimen as part of the myeloid malignancy programs. While Aprea can’t enroll additional patients to these studies, investigators can continue to give the drugs to patients who are benefiting from treatment.
It added that the hold doesn’t affect other ongoing trials in lymphoid malignancies and solid tumors involving eprenetapopt, its lead candidate. Aprea also has a preclinical, next-gen p53 reactivator dubbed APR-548.