FDA grants fast track designation to SNDX-5613 for acute leukemia
The FDA granted fast track designation to SNDX-5613 for the treatment of adults and children with relapsed or refractory acute myeloid leukemia or acute lymphoblastic leukemia with certain genetic markers.
SNDX-5613 (Syndax) — a small-molecule inhibitor of the menin-mixed leukemia lineage (MLL) binding interaction — is designed to treat MLL-rearranged ALL or AML, and AML with a nucleophosmin (NPM1) mutation.
The agent showed robust, dose-dependent inhibition of tumor growth in preclinical models of MLL-rearranged ALL and AML, according to a company-issued press release. It also is being evaluated in the phase 1/phase 2 open-label AUGMENT-101 trial for the treatment of relapsed/refractory ALL and AML.
“Genetically defined acute leukemias represent an underserved area marked by particularly poor prognosis and limited therapeutic options,” Briggs W. Morrison, MD, CEO of Syndax, said in the release. “As we move toward initiating our pivotal study, receipt of fast track designation from the FDA underscores SNDX-5613’s potential to meaningfully improve outcomes for patients with MLL-rearranged and NPM1-mutant acute leukemias.”