Eytan Stein, MD of @sloan_kettering discusses the first patient dosed in phase 1/2 AUGMENT-101 trial of SNDX-5613 for the treatment with relapsed/refractory acute leukemias.
Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that the first patient has been dosed in AUGMENT-101, a Phase 1/2 clinical trial evaluating SNDX-5613, Syndax's potent, highly selective oral Menin inhibitor, in patients with relapsed/refractory (R/R) acute leukemias.
"Dosing of the first patient in AUGMENT-101 represents an important milestone, both for the SNDX-5613 program and, importantly, for patients with genetically-defined acute leukemias, many of whom often do not achieve durable benefit from currently available treatment regimens," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Preclinical data strongly support our belief that SNDX-5613 has the potential to address a significant unmet need as a treatment for patients with MLL-r and NPM1 mutant leukemias."
AUGMENT-101 is a Phase 1/2 open-label trial designed to evaluate the efficacy, safety, tolerability and pharmacokinetics of orally administered SNDX-5613. The Phase 1 dose escalation portion of AUGMENT-101 will enroll adults with R/R acute leukemias and establish a recommended Phase 2 dose. The Phase 2 portion will evaluate efficacy, as defined by Complete Response rate (per International Working Group response criteria), across three expansion cohorts: MLL-rearranged (MLL-r) acute lymphoblastic leukemia (ALL), MLL-r AML and NPM1 mutant AML. The Company expects to report initial clinical data from the trial in 2020.
Additional information about the AUGMENT-101 trial is available via Clinicaltrials.gov (NCT 04065399).
SNDX-5613 is a potent, selective, small molecule inhibitor of the Menin-MLL binding interaction that is being developed for the treatment of MLL-rearranged (MLL-r) acute leukemias, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). MLL rearrangements occur in approximately 80% of acute leukemia cases in infants and up to 10% of all leukemias. In preclinical models of MLL-r acute leukemias, SNDX-5613 demonstrated robust, dose-dependent inhibition of tumor growth, resulting in a marked survival benefit. Menin-MLL interaction inhibitors have also demonstrated robust treatment benefit in multiple preclinical models of NPM1 mutant AML, which represents the most frequent genetic abnormality in adult AML. SNDX-5613 is currently being evaluated in the Company's AUGMENT-101 Phase 1/2 open-label clinical trial for the treatment of relapsed/refractory acute leukemias.