Agios Announces First Patient Dosed with AG-881 in Phase 1 Study in Patients with Advanced Solid Tumors with an IDH Mutation
– Third IDH Mutant Inhibitor to Enter Clinical Trials –
– Brain Penetration and Pan-IDHm Inhibition Broaden Development Options for IDH Mutant-Positive Cancers, Including Gliomas –
– Second Phase 1 Study in Hematologic Malignancies on Track to Begin Dosing in Coming Weeks –
"The initiation of this study represents a significant milestone for Agios, as it marks the third program from our portfolio of IDH inhibitors to enter the clinic in less than two years," said
"We are eager to explore the profile of AG-881 as we continue to investigate the role of IDH inhibitors for the treatment of patients with IDH mutant-positive tumors," said
About the AG-881 Phase 1 Study in Advanced Solid Tumors, including Gliomas, with an IDH1 or IDH2 Mutation
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 (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. Please refer to www.clinicaltrials.gov for additional clinical trial information.
Upcoming Milestones for AG-881
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.
About IDH Mutations and Cancer
IDH1 and IDH2 are two metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies, including acute myeloid leukemia (AML) and gliomas. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH increases production of an oncometabolite 2-hydroxyglutarate (2HG) that alters the cells' epigenetic programming, thereby promoting cancer. 2HG has been found to be elevated in several tumor types. Agios believes that inhibition of the mutated IDH proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry them.
Summary of Agios and Celgene Collaboration on IDH Mutant Inhibitors
Each of these investigational medicines carries different financial terms and rights under the collaboration:
Celgenehas worldwide development and commercialization rights for AG-221. Agios is eligible for up to $120 millionin milestone payments and royalties on any net sales.
AG-120: Agios retains U.S. development and commercialization rights, while
Celgenehas development and commercialization rights outside the U.S. Agios is eligible to receive royalties on any net sales outside the U.S. and up to $120 millionin milestone payments. Celgeneis eligible to receive royalties on any net sales in the U.S.
AG-881: Joint worldwide development and 50/50 profit share collaboration. Agios is eligible to receive regulatory milestone payments up to
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of Agios' product candidates targeting IDH mutations, including AG-881; its plans and timelines for the clinical development of AG-881; and the benefit of its strategic plans and focus. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "potential," "possible," "hope," "could," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios' current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios' product candidates will successfully continue. There can be no guarantee that any positive developments in Agios' business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including: Agios' results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S.
Renee Leck, Senior Manager, Investor Relations and Public Relations Renee.Leck@agios.com 617-649-8299