The following article features coverage from the Tandem Meetings 2022. Click here to read more of Cancer Therapy Advisor’s conference coverage.

Among patients with TP53-mutated acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS), adding eprenetapopt to maintenance azacitidine after hematopoietic stem cell transplant (HCT) appears both safe and effective, according to research presented at the Tandem Meetings 2022.

Eprenetapopt is a small molecule that stabilizes p53 proteins, and research suggests that it may work in tandem with azacitidine to promote cancer cell death.

For this phase 2 study, researchers evaluated the safety and efficacy of adding eprenetapopt to maintenance azacitidine among patients with TP53-mutant AML or MDS who undergo HCT.

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Overall, 33 patients were enrolled and received maintenance therapy per this protocol. Nineteen patients had MDS, and 14 had AML. The median patient age was 65 years, 64% of patients were men, 97% had TP53 mutations, and 82% had complex cytogenetics.

The median follow-up for relapse-free survival (RFS) was 413 days. At this point, the median RFS was 368 days (95% CI: 233-not evaluable), and the 1-year RFS rate was 59.9%.

The median overall survival was 586 days (95% CI: 369-not evaluable), and the 1-year overall survival rate was 79%. As of June 2021, 18% remained on study treatment; 9 had completed 12 cycles.

Grade 3 or worse treatment-related adverse events occurring in at least 10% of patients included cytopenias and hypertension. A total of 13 patients developed acute or chronic graft vs host disease.

Asmita Mishra, MD, of the H. Lee Moffitt Cancer Center and Research Institute in Tampa, Florida, who presented the study’s findings, noted that outcomes were “substantially improved over historical data.”

Disclosures: The study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 

Read more of Cancer Therapy Advisor’s coverage of Tandem Meetings 2022 by visiting the conference page.


Mishra A, Tamari R, Byrne M, et al. Phase II trial of eprenetapopt (APR-246) in combination with azacitidine (AZA) as maintenance therapy for TP53 mutated acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) following allogeneic hematopoietic cell transplantation (HCT). Tandem Meetings 2022; April 23-26, 2022. Abstract 39.

This article originally appeared on Hematology Advisor