Agios Pharmaceuticals Enrolls First Patient in Phase 1 Study of AG-221 in Advanced Hematologic Malignancies with an IDH2 Mutation
-- First-in-class Clinical Program Targets Cancer Metabolism in Genetically Defined Patient Population --
“IDH2 represents one of the most promising targets in cancer biology
today,” said
“Cancer drug development is advancing at a remarkable pace, and we think
that patients will benefit as our therapies evolve to target specific
genetic abnormalities,” said
AG-221 is part of Agios' global strategic collaboration with
About the Study
The Phase 1 multicenter, open-label, dose escalation clinical trial of AG-221 is designed to assess the safety and tolerability of AG-221 as a single agent administered orally twice daily in a 28-day cycle. The study is expected to only enroll subjects who have an IDH2-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 the oncometabolite 2-hydroxyglutarate, or 2-HG) and preliminary anti-tumor activity of AG-221. Disease-specific expansion cohorts will be enrolled at the maximally tolerated or biologically relevant dose. Please refer to www.clinicaltrials.gov for additional clinical trial details.
About IDH Mutations
The IDH protein is a critical metabolic enzyme in the citric acid cycle, also known as the tricarboxylic acid (TCA), or Krebs cycle. Agios' scientists first established that the mutated forms of IDH produce a metabolite, 2-HG, which may contribute to the formation and malignant progression of various forms of cancer. Agios and its collaborators recently demonstrated that IDH1 and IDH2 mutations initiate and drive cancer growth by blocking differentiation, also referred to as 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 these mutations.
The connection between cancer and metabolism has been the central focus
of scientists at Agios, who were the first to identify the neo-activity
of IDH1 mutations to produce the oncometabolite 2-HG in research
published in Nature in 2009. These insights revealed the
potential of IDH1 and IDH2 mutations as novel therapeutic targets in
cancer. The IDH1 gene mutation was initially discovered in brain cancers
in 2008 by researchers at
About
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 and IDH2 mutants as therapeutic
targets; the potential benefits of Agios’ product candidates targeting
IDH1 and IDH2 mutations, including AG-221; its plans and timelines for
the clinical development of AG-221; 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.
Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Source:
Agios Pharmaceuticals, Inc.
Media:
Dan Budwick,
973-271-6085
dan@purecommunicationsinc.com
or
Investors:
Glenn
Goddard, 617-649-8600
investors@agios.com