Agios Announces Enrollment of First Patient in Phase 1 Study of AG-120 in Advanced Hematologic Malignancies with an IDH1 Mutation
Second Agios Clinical Program in the New Field of Cancer Metabolism
“Today’s announcement marks the second cancer metabolism clinical
program that we’ve initiated in less than six months, both of which
utilize a precision medicine approach that could potentially set up a
rapid clinical development pathway,” said
Groundbreaking research by Agios' scientists (published in the
“We expect that this study will generate important insights about the
utility of AG-120 among this genetically defined patient population, for
whom there are very few treatment options available,” said
AG-120 is a part of Agios’ global strategic collaboration with
About the Study
The Phase 1, multicenter, open-label, dose-escalation clinical trial of AG-120 is designed to assess the safety and tolerability of AG-120 as a single agent. The study is expected to only enroll subjects with an IDH1-mutant hematologic malignancy, including acute myelogenous leukemia (AML) and myelodysplastic syndrome. Key objectives in the study include determining maximum tolerated dose, pharmacokinetics, pharmacodynamics (including inhibition of 2HG) and preliminary anti-tumor activity. Disease-specific expansion cohorts will be enrolled at the maximally tolerated or biologically relevant dose. Please refer to www.clinialtrials.gov for additional clinical trial details.
About IDH Mutations and Cancer
The connection between cancer and metabolism has been the central focus for scientists at Agios, who were the first to identify the neo-activity of IDH1 mutations to produce 2HG in research published in Nature in 2009. These insights revealed the potential of IDH1 and IDH2 mutations as novel therapeutic targets in cancer. Mutations in both IDH1 and IDH2 have been linked to numerous hematologic and solid tumor malignancies.
Agios and its collaborators recently demonstrated that IDH1 and IDH2 mutations initiate and drive cancer growth by blocking differentiation, or maturation, of primitive cells. Agios believes that inhibition of these mutated proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry them.
Agios is also conducting a Phase 1 study of AG-221, evaluating its safety, pharmacokinetics, pharmacodynamics and clinical activity in patients with advanced hematologic malignancies that harbor an IDH2 mutation.
Agios Pharmaceuticals is focused on discovering and developing novel drugs to treat cancer and inborn errors of metabolism (IEMs), which are rare genetic metabolic 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 multiple first-in-class lead product candidates in cancer metabolism and IEMs in clinical and preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit our website at www.agios.com.
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’ expectations
and beliefs about: the potential of IDH1/IDH2 as therapeutic targets;
the potential benefits of Agios’ product candidates AG-120 and AG-221;
its plans and timelines for the clinical development of AG-120 and
AG-221; and the benefit of its strategic plans and focus. The words
“anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,”
“predict,” “project,” “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.