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ASH 2020 | KO-539: a KMT2A inhibitor with promising results in R/R AML

Eunice Wang, MD, Roswell Park Comprehensive Cancer Center, Buffalo, NY shares the results of a Phase I/IIa study (NCT04067336) of KO-539 in relapsed/refractory (R/R) acute myeloid leukemia (AML). KO-539 targets the menin-KMT2A complex, which is involved in HOX gene regulation and epigenetic dysregulation in AML. Perturbations of the KMT2A gene occur in 5-10% of AML, generating an aggressive cancer with poor prognosis. The Phase I study aims to assess safety and tolerability, and the patients enrolled to date have experienced no dose-limiting toxicities. Evidence of biological activity is present, and the Phase II expansion cohort will further assess this. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.


Dr Eunice Wang, MD, has participated in advisory boards for AbbVie, Astellas, BMS/Celgene, Genentech, Jazz, Kite Pharmaceuticals, Macrogenics, Novartis, Pfizer, PTC Therapeutics, Stemline and Takeda; has fulfilled speaker roles for Stemline, Pfizer and Dava Oncology; and has been a member of data monitoring committees for AbbVie and Rafael Pharmaceuticals.

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