Agios Pharmaceuticals, Inc., (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that it has submitted a New Drug Application (NDA) to the U.S.Food and Drug Administration (FDA) for ivosidenib (AG-120), an investigational oral treatment for patients with relapsed or refractory acute myeloid leukemia (R/R AML) and an isocitrate dehydrogenase-1 (IDH1) mutation. Agios has requested priority review for the application, which, if granted, could result in a six-month review process.
"People with AML who have relapsed or refractory disease have limited treatment options available to them, and it is our hope that we can change that," said Chris Bowden, M.D., chief medical officer at Agios. "Earlier this month at the ASH Annual Meeting, we presented compelling single agent ivosidenib data demonstrating durable responses in high-risk relapsed or refractory AML patients with an IDH1 mutation. These data highlight the potential for ivosidenib to be a first-in-class therapy for patients with R/R AML and an IDH1 mutation."
The NDA is supported by data from the ongoing Phase 1 dose-escalation and expansion study of ivosidenib in patients with advanced hematologic malignancies and an IDH1 mutation. Ivosidenib is wholly owned by Agios. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.
AG-270 IND Clearance
In addition, Agios announced that the FDA has completed their 30-day safety review of the investigational new drug (IND) application for AG-270, the development candidate targeting MTAP-deleted tumors, and has granted IND clearance to proceed with the proposed Phase 1 dose-escalation trial in multiple tumor types carrying an MTAP deletion. Agios expects to initiate the Phase 1 trial in the first quarter of 2018.
About Agios
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has an approved oncology precision medicine and multiple first-in-class investigational therapies in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics.
About the MTAP Pathway Program
The program focused on MTAP deleted cancers is part of a 2016 global co-development and co-commercialization agreement with Celgene focused on metabolic immuno-oncology. Celgene has the option to participate in a worldwide 50/50 cost and profit share with Agios, under which Agios is eligible for up to $169 million in clinical and regulatory milestone payments for the program. As described in a 2016 Cell Reports publication, Agios discovered a novel pathway in MTAP-deleted tumors which, when inhibited, results in robust anti-tumor activity in animal models. This pathway can be modulated by small molecule inhibitors, as demonstrated in a preclinical data presentation at the Keystone Tumor Metabolism meeting.
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 Agios’ plans, strategies and expectations for its and its collaborator’s preclinical, clinical and commercial advancement of its drug development programs including ivosidenib and AG-270; the potential benefits of its product candidates; its plans for regulatory submissions; and the potential benefit of its strategic plans and focus. The words "anticipate," "expect," "intend," "potential," "milestone," "goal," "will," "on track," "upcoming," 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 or its collaborator, Celgene, is ...
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