FDA Grants Fast Track Designation to Eprenetapopt for Treatment of TP53-Mutant AML


The FDA granted a Fast Track designation to eprenetapopt as treatment of patients with TP53-mutant acute myeloid leukemia.

The FDA has granted a Fast Track designation to eprenetapopt (formerly APR-548) as treatment of patients with TP53-mutant acute myeloid leukemia (AML), according to a press release from Aprea Therapeutics, Inc.

“We are pleased to have received Fast Track designation for eprenetapopt in the treatment of TP53-mutant AML, a cancer for which outcomes are poor and there are no current therapeutic options specifically for these patients,” said Eyal C. Attar, MD, chief medical officer of Aprea, in a statement. “Emerging data from our AML trials evaluating eprenetapopt with azacitidine, and with eprenetapopt, azacitidine and venetoclax, are promising and we continue to enroll patients to identify the best treatment regimen.”

The agent previously received a Breakthrough Therapy designation, Orphan Drug designation, and a Fast Track designation for the treatment of patients with myelodysplastic syndromes (MDS) who harbor a TP53mutation. Eprenetapopt has demonstrated anti-tumor activity in the pre-clinical setting for patients with MDS and AML, as well as other hematologic and solid tumor types.

Strong synergy has been observed with both traditional anti-cancer drugs, such as chemotherapy and newer mechanism-based anti-cancer therapies and immune-oncology checkpoint inhibitors. A prior phase 1/2 study demonstrated a favorable safety profile with both biological and confirmed clinical responses in patients with TP53-mutant hematologic malignancies and solid tumors.

A pivotal phase 3 study will evaluate the combination of eprenetapopt with azacitidine for the frontline treatment of patients with TP53-mutant MDS, which is currently ongoing and enrolling patients. A phase 1 clinical trial of eprenetapopt is expected to begin enrollment in the first quarter of 2021, which will evaluate this next-generation small molecule p53 reactivator as treatment of patients with TP53-mutant cancer.

“As these data mature in 2021, we look forward to continued interaction with FDA as we map out opportunities for an accelerated pathway to potential approval,” stated Attar.

The p53 tumor suppressor gene is the most common mutation in cancer and is known to occur in approximately 50% of tumors. They’re often associated with resistance to anti-cancer drugs and poor overall survival, which represents a major unmet medical need in the field. By restoring wild-type p53 conformation and function, eprenetapopt induced programmed cell death in human cancer cells.


Aprea Therapeutics receives FDA fast track designation for eprenetapopt in the treatment of tp53 mutant acute myeloid leukemia (AML). News Release. Aprea Therapeutics, Inc. November 30, 2020. Accessed November 30, 2020. https://bit.ly/39tqipN

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