CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 10, 2015--
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of
cancer metabolism and rare genetic disorders of metabolism, today
announced that the U.S. Food and Drug Administration (FDA) has granted
the company orphan drug designation for AG-120 for treatment of patients
with acute myelogenous leukemia (AML). AG-120 is an oral, first-in-class
IDH1 mutant inhibitor being evaluated in a Phase 1 clinical trial in
patients with advanced hematologic malignancies that carry an IDH1
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 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-120 is an important milestone
as we continue to move this program to late-stage development,” said
Chris Bowden, M.D. chief medical officer of Agios. “We are pleased with
the progress we are making in the clinic and look forward to presenting
new data from our ongoing Phase 1 study of AG-120 at the Congress of the
European Hematology Association later this week. We believe that AG-120,
which is on track to initiate multiple expansion cohorts in the next
month, has the potential to play a significant role in shifting the
treatment paradigm for IDH1-mutant positive hematologic cancers from the
conventional chemotherapy approach.”
AML, a cancer of blood and bone marrow characterized by rapid disease
progression, is the most common acute leukemia in adults.
Undifferentiated blast cells proliferate in the bone marrow rather than
mature into normal blood cells. 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/refractory AML. The five-year
survival rate for AML is approximately 20 to 25 percent. IDH1 mutations
are present in about 6 to 10 percent of AML cases.
About Agios Pharmaceuticals, Inc.
Agios Pharmaceuticals is focused on discovering and developing novel
investigational medicines to treat cancer and rare genetic disorders of
metabolism 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
investigational medicines 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 the company’s website at 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 candidate AG-120; the potential benefits of
orphan drug status; 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. 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, 2015, 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.
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Source: Agios Pharmaceuticals , Inc.
Agios Pharmaceuticals, Inc.
Renee Leck, 617-649-8299
Senior
Manager, Investor Relations and Public Relations
Renee.Leck@agios.com