FDA grant orphan drug designation for eprenetapopt (APR-246) for the treatment of acute myeloid leukaemia (AML)

Pre-clinical anti-tumor activity has been observed with APR-246 in a wide variety of solid and haematological cancers, including MDS, AML and ovarian cancer among others. Enrolment in Phase 1 clinical trial of APR-548 is anticipated to begin early in the second quarter of 2021.

SPS commentary:

Eprenetapopt (APR-246) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein thereby inducing programmed cell death in human cancer cells.

Biospace Inc.