NEW YORK – Boston-based Aprea Therapeutics said on Thursday that it will expand patient enrollment in a Phase I clinical trial evaluating its lead drug candidate, eprenetapopt, as a frontline treatment for acute myeloid leukemia patients with TP53 mutations.
Roughly 20 percent of AML patients have TP53 mutations. The expansion cohort, which follows an initial safety assessment, will evaluate eprenetapopt in combination with the chemotherapy azacitidine, with and without the BCL-2 inhibitor venetoclax (Genentech/AbbVie's Venclexta). Aprea anticipates treating roughly 30 patients with the three-drug combination and 30 patients with the two-drug combination.
In the lead-in safety portion of the study, the company found that both regimens were tolerated and that none of the enrolled patients experienced dose-limiting toxicities. The expanded trial will continue to evaluate the treatments' safety along with its efficacy.
Eprenetapopt is a small molecule designed to reactivate p53 in patients with TP53-mutant hematologic and solid malignancies, including myelodysplastic syndromes and AML. The drug's potential to restore wild-type p53 function, in turn inducing apoptosis in cancer cells, has been demonstrated in preclinical studies, and there is early evidence of confirmed clinical responses.
While eprenetapopt's clinical development for AML is in the early phases, its development in MDS is further along. The agent is currently being evaluated for TP53-mutant MDS together with azacitidine in a multicenter, randomized Phase III trial, and the company expects to report topline results by year end.Â