Press Release

July 26, 2021
1 min read

Eprenetapopt-azacitidine regimen ‘incredibly exciting’ for TP53-mutant MDS, AML


Press Release

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Eprenetapopt with azacitidine exhibited encouraging activity as posttransplant maintenance therapy for patients with TP53-mutant myelodysplastic syndrome and acute myeloid leukemia, topline data released by the agent’s manufacturer showed.

The p53 tumor suppressor gene is mutated in approximately half of human tumors. The mutations often are associated with treatment resistance and poor survival.

Infographic showing RFS and OS outcomes at 1 year
Data derived from Aprea Therapeutics press release.

Eprenetapopt (APR-246, Aprea Therapeutics) is a small molecule that reactivates mutant and inactivated p53 protein by restoring p53 conformation and function, thereby inducing programmed cell death in cancer cells.

A phase 2 trial evaluated eprenetapopt with azacitidine for posttransplant maintenance therapy for 33 patients with TP53-mutant myelodysplastic syndrome (MDS) and AML.

Researchers reported a 58% RFS rate and 79% OS rate at 1 year. Results showed median RFS of 12.1 months and median OS of 19.3 months.

These outcomes compared favorably with prior trials that evaluated posttransplant outcomes in TP53-mutant MDS and AML, results of which showed 1-year RFS of about 30% and median OS of about 5.8 months.

The combination of eprenetapopt and azacitidine also appeared well-tolerated, according to an Aprea Therapeutics-issued press release.

Company officials intend to discuss the trial data with the FDA later this year and present the findings at a medical conference.

“The posttransplant RFS and OS data with eprenetapopt and azacitidine maintenance therapy in these [patients with] very difficult-to-treat TP53 mutant MDS and AML ... are incredibly exciting,” principal investigator Asmita Mishra, MD, assistant member of the department of blood and marrow transplant at Moffitt Cancer Center, said in the release. “Although transplant is currently the only potentially curative treatment for patients with TP53-mutant MDS and AML, the risk [for] relapse with current standard of care remains unacceptably high and the median OS [after] transplant is very limited at 8 months or less. Posttransplant maintenance therapy with eprenetapopt and azacitidine could, if approved, represent a new treatment paradigm that meaningfully improves outcomes for these patients with limited treatment options.”