Agios initiates AG-120 Phase 1 study to evaluate advanced solid tumors with IDH1 mutation

Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of cancer metabolism and inborn errors of metabolism, today announced dose administration for the first patient in a Phase 1 study of AG-120 in patients with advanced solid tumors with an isocitrate dehydrogenase-1 (IDH1) mutation. AG-120 is an orally available, selective, potent inhibitor of the mutated IDH1 protein and a highly targeted, first-in-class therapeutic candidate.

This is the second Phase 1 study of AG-120. Agios recently announced that the first patient received treatment in a Phase 1 study of AG-120 in patients with hematologic malignancies.

"We are pleased to have rapidly initiated two studies evaluating AG-120 in a broad range of solid tumors and hematologic cancers with IDH1 mutations," said David Schenkein, M.D., chief executive officer of Agios. "Both of these studies employ a precision medicine approach that will evaluate AG-120 among diagnostically identified patient populations, with a goal of generating valuable early data about this cancer metabolism development program. We believe the approach of targeting IDH mutations in cancer holds great promise for patients."

"Because this study will only enroll patients whose cancers have an IDH1 mutation, it is expected to provide us with important information about how AG-120 works among the patients who are most likely to respond to it," said Howard Burris, M.D., Sarah Cannon Research Institute, an investigator for the study. "We are hopeful that inhibiting these mutated metabolic enzymes will have a significant clinical benefit for patients with solid tumors that carry these mutations."

AG-120 is a part of Agios' global strategic collaboration with Celgene Corporation, a leading biotechnology company. Established in 2010, the goal of the collaboration is to discover, develop and deliver novel, disease-altering oncology therapies, based on Agios' cancer metabolism research platform. Agios recently announced that it has elected the option to take exclusive rights on U.S. development and commercialization for AG-120, subject to and in accordance with the terms of the collaboration agreement with Celgene, with Celgene retaining its option to ex-U.S. rights. The parties are also collaborating on the development of AG-221, an oral, selective, potent inhibitor of the mutated IDH2 protein.