Dr. Sallman on APR-246 Plus Azacitidine in TP53-Mutant MDS and AML

David Sallman, MD, assistant member, Moffitt Cancer Center, discusses the combination of APR-246 and azacitidine as a treatment for patients with TP53-mutant myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML).

In a phase Ib/II study, investigators evaluated the safety, preliminary activity, and minimal residual disease (MRD) status of APR-246 in combination with azacitidine in patients with TP53-mutant MDS or AML. APR-246 is a novel agent that is classified as a p53 reactivator, according to Sallman. This agent is able to bind the mutant protein and restores a more wild-type confirmation. Sallman says that the patients focused on in this study have had poor molecular outcomes to treatments thus far, emphasizing the need for more novel therapies.

In this intial report, 9 of 12 patients were evaluable. This combination was well tolerated, and responses were achieved in all patients. Sallman reports that there was a 100% overall response rate, with 8 of 9 patients achieving a complete remission.