Key clinical point : Eprenetapopt in combination with azacitidine yielded high rates of clinical response and complete remission (CR) and was well tolerated in patients with TP53-mutant myelodysplastic syndromes (MDS).
Major finding: Overall response rate in patients with MDS was 73% (95% confidence, [CI], 56%-85%) with 50% (95% CI, 34%-66%) of patients achieving CR. The median time to first response and CR was 1.9 and 3.1 months, respectively. Adverse events were similar to those reported for azacitidine or eprenetapopt monotherapies.
Study details : Findings come from a phase 1b/2 open-label, dose-escalation/expansion study that assessed safety, recommended dose, and efficacy of eprenetapopt and azacitidine combination in 55 patients with TP53-mutant MDS (n=40) or acute myeloid leukemia with 20%-30% marrow blasts.
Disclosures: The study was supported by the MDS Foundation Young Investigator Grant, the Early Career Award of the Dresner Foundation (DAS), and the Edward P. Evans MDS Clinical Research Consortium. The study was supported in part by the Flow Cytometry and Molecular Genomics Core Facilities at the H. Lee Moffitt Cancer Center & Research Institute. Some of the authors reported relationships with various pharmaceutical companies while some others declared no conflicts of interest.
Source: Sallman DA et al. J Clin Oncol. 2021 Jan 15. doi: 10.1200/JCO.20.02341.