Aprea Phase 1b/2 Myelodysplastic Syndromes (MDS) Study at ASH

-95% ORR (by IWG) in 20 evaluable patients
– 70% complete remission (CR) rate in 20 evaluable patients
– No dose-limiting toxicities to date

Aprea Therapeutics presented results at the 2018 ASH Annual Meeting from its Phase Ib/II clinical study in MDS. The ongoing study is evaluating the safety and efficacy of APR-246 in combination with azacitidine for the treatment of TP53 mutated MDS. The study is sponsored by the Moffitt Cancer Center with financial support from the MDS Foundation and the Aplastic Anemia and MDS International Foundation as administrator for the Evans MDS Clinical Research Consortium.

The overall response rate in 20 evaluable patients was 95%, with 14 (70%) patients achieving a complete remission (CR) at data cutoff. Relative to baseline, p53 immunohistochemistry positivity, mutant TP53 variant allele frequency (VAF) and TP53 minimal residual disease (MRD) were significantly decreased at time of disease assessment. No dose-limiting toxicities have been experienced to date and no exacerbation of the expected AZA-related safety profile has been observed.

“The expanding data set from this study is very encouraging,” said David Sallman, M.D., lead principal investigator of the clinical study from the Moffitt Cancer Center.  “As of this latest data cutoff, responses have been achieved in nearly all patients, including a 70% complete remission rate, and accompanied by deep molecular remission in the majority of patients as assessed by serial TP53 analysis. In addition, the overall safety experience indicates that the combination regimen of APR-246 and azacitidine is both safe and well-tolerated. Comparison of the current data set to historical AZA clinical experience suggests that combination of APR-246 with AZA may offer these patients a better potential treatment option than AZA alone.”

“The continued positive data from this clinical study has created the potential for a new treatment paradigm for patients with few therapeutic options,” said Christian S. Schade, President and Chief Executive Officer of Aprea.  “As a result of this exciting progress, Aprea expects to soon begin enrolling a randomized, controlled Phase III clinical study of APR-246 in combination with AZA for the treatment of TP53 mutated MDS.”

https://www.biospace.com/article/releases/aprea-therapeutics-presents-results-from-phase-ib-ii-clinical-study-of-apr-246-and-azacitidine-aza-in-patients-with-tp53-mutant-myelodysplastic-syndromes-mds-at-the-2018-american-society-of-hematology-ash-annual-meeting-in-san-diego/