CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 16, 2014--
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of
cancer metabolism and inborn errors of metabolism (IEMs), today
announced that the U.S. Food and Drug Administration (FDA) has granted
the company orphan drug designation for AG-221 for treatment of patients
with acute myelogenous leukemia (AML). AG-221 is an oral, first-in-class
IDH2 mutant inhibitor being evaluated in a Phase 1 clinical trial in
patients with advanced hematologic malignancies that carry an IDH2
mutation.
The FDA’s Office of Orphan Drug Products grants orphan status to support
development of medicines for underserved patient populations, or rare
disorders, that affect fewer than 200,000 people in the U.S. Orphan drug
designation provides to Agios and its collaborator Celgene certain
benefits, including market exclusivity upon regulatory approval if
received, exemption of FDA application fees and tax credits for
qualified clinical trials.
“Receiving orphan drug designation for AG-221 is another important
milestone to emerge from our innovative cancer metabolism program,” said
Chris Bowden, M.D. chief medical officer of Agios. “We are pleased with
the progress we are making in the clinic and believe that AG-221, which
is expected to enter multiple expansion cohorts in the second half of
this year, has the potential to play a significant role in shifting the
treatment paradigm for IDH2 mutant positive hematologic cancers from the
conventional chemotherapy approach.”
AML is a cancer of blood and bone marrow characterized by rapid disease
progression, and is the most common acute leukemia affecting adults. AML
incidence significantly increases with age, and according to the
American Cancer Society, the median age is 66. Less than 10 percent of
U.S. patients are eligible for bone marrow transplant, and the vast
majority of patients do not respond to chemotherapy and progress to
relapsed or refractory AML. The five-year survival rate for AML is
approximately 20 to 25 percent. AML prevalence is estimated to be
approximately 35,000 to 40,000 patients in the U.S., with approximately
15% of patients estimated to carry an IDH2 mutation.
About Agios Pharmaceuticals, Inc.
Agios Pharmaceuticals is focused on discovering and developing novel
drugs to treat cancer and inborn errors of metabolism, or 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/or 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 the potential
benefits of Agios’ product candidates AG-221 and AG-120; and the benefit
of its strategic plans and focus. The words “anticipate,” “believe,”
“estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,”
“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. FDA and other
regulatory authorities, investigational review boards at clinical trial
sites and publication review bodies; Agios’ ability to obtain and
maintain requisite regulatory approvals and to enroll patients in its
planned clinical trials; unplanned cash requirements and expenditures;
competitive factors; Agios’ ability to obtain, maintain and enforce
patent and other intellectual property protection for any product
candidates it is developing; Agios’ ability to maintain key
collaborations, such as its agreement with Celgene; and general economic
and market conditions. These and other risks are described in greater
detail under the caption “Risk Factors” included in Agios’ Quarterly
Report on Form 10-Q for the quarter ended March 31, 2014, and other
filings that Agios may make with the Securities and Exchange Commission
in the future. 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.
Agios Pharmaceuticals, Inc.
Lora Pike, 617-649-8608
Senior
Director, Investor Relations and Public Relations
lora.pike@agios.com
