Agios Says First Patient Dosed With AG-881 In Phase 1 Study - Quick Facts

(RTTNews) - Agios Pharmaceuticals Inc. (AGIO) announced dose administration for the first patient in a Phase 1, open-label, dose-escalation and expansion study of single agent AG-881, a small molecule that has shown in preclinical studies to fully penetrate the blood-brain barrier and inhibit isocitrate dehydrogenase-1 (IDH1) and IDH2 mutations in cancer models.

The purpose of the Phase 1 multi-center, open-label study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-881 in advanced solid tumors. AG-881 will be administered continuously as a single agent dosed orally in a 28-day cycle.

The first portion of the study includes a dose-escalation phase in which cohorts of patients will receive ascending oral doses of AG-881 to determine the maximum tolerated dose or MTD and/or the recommended Phase 2 dose based on safety and tolerability.

The second portion of the study is a dose expansion phase where patients will receive AG-881 to further evaluate the safety, tolerability and clinical activity of the recommended Phase 2 dose.

A second dose-escalating and expansion trial, for patients with advanced IDH1 or IDH2 mutant-positive hematologic malignancies whose cancer has progressed on a prior IDH inhibitor therapy, is expected to begin shortly.