Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia
June 08 2020 - 4:01PM
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field
of cellular metabolism to treat cancer and rare genetic diseases,
today announced that the U.S. Food and Drug Administration (FDA)
has granted orphan drug designation to the company’s first-in-class
pyruvate kinase-R (PKR) activator mitapivat for the treatment of
patients with thalassemia. Mitapivat is an investigational, oral,
small molecule allosteric activator of wild-type and a variety of
mutated PKR enzymes.
“Receiving orphan drug designation is an important milestone as
we continue to advance mitapivat for patients with thalassemia, a
serious hemolytic anemia with limited treatment options,” said
Chris Bowden, M.D., chief medical officer at Agios. “We look
forward to presenting updated data from our Phase 2 study of
mitapivat in both alpha- and beta-thalassemia patients at the
virtual European Hematology Association Annual Congress later this
week.”
The FDA’s Office of Orphan Drug Products grants orphan status to
support the 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 certain
benefits, including market exclusivity upon regulatory approval if
received, exemption of FDA application fees and tax credits for
qualified clinical trials.
Mitapivat was previously granted orphan drug designation by the
FDA and the European Medicines Agency for pyruvate kinase (PK)
deficiency, a rare, debilitating, hemolytic anemia.
Mitapivat Clinical Development Agios is
conducting a Phase 2 study evaluating the efficacy, safety,
pharmacokinetics and pharmacodynamics of treatment with mitapivat
in adults with non-transfusion-dependent β- and α-thalassemia
(NTDT). The trial is fully enrolled, and the primary endpoint is
hemoglobin response. Preliminary Phase 2 data establishing
proof-of-concept for mitapivat in thalassemia were disclosed at the
end of 2019, and updated data from this trial will be presented at
the 25th European Hematology Association (EHA) Annual Congress,
which is being held virtually on June 11-14, 2020.
In addition, Agios has two ongoing global, pivotal trials in
adults with PK deficiency that are fully enrolled.
- ACTIVATE: A placebo-controlled trial with a 1:1 randomization
evaluating patients who do not receive regular transfusions. The
primary endpoint of the trial is the proportion of patients who
achieve a sustained hemoglobin increase of ≥1.5 g/dL.
- ACTIVATE-T: A single arm trial of regularly transfused patients
with a primary endpoint of reduction in transfusion burden over six
months compared to individual historical transfusion burden over
prior 12 months.
Mitapivat is also being studied in sickle cell disease under
a Cooperative Research and Development Agreement (CRADA)
with the U.S. National Institutes of Health.
Mitapivat is not approved for use by any regulatory
authority.
About AgiosAgios is focused on discovering and
developing novel investigational medicines to treat malignant
hematology, solid tumors and rare genetic diseases through
scientific leadership in the field of cellular metabolism. In
addition to an active research and discovery pipeline across these
three therapeutic areas, Agios has two approved oncology precision
medicines and multiple first-in-class investigational therapies in
clinical and/or preclinical development. For more information,
please visit the company's website at www.agios.com.
Cautionary Note Regarding Forward-Looking
StatementsThis 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 mitapivat; Agios’ plans
regarding future data presentations; and the benefit of Agios’
strategic plans and focus. The words "expects," "anticipates,"
"believes," "intends," "estimates," "plans," "will," "outlook,"
"goal", "potential" 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, a positive opinion on Agios’ application for orphan drug
designation for mitapivat is not a guarantee of approval.
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: risks and uncertainties related to the impact of the
COVID-19 pandemic to Agios’ business, operations, strategy, goals
and anticipated milestones, including its ongoing and planned
research activities, ability to conduct ongoing and planned
clinical trials, clinical supply of current or future drug
candidates, commercial supply of current or future approved
products, and launching, marketing and selling current or future
approved products; the results of Agios’ 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 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 and conduct
its current and future 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; and general economic,
market and global health conditions. These and other risks are
described in greater detail under the caption "Risk Factors"
included in Agios’ public filings with the Securities and Exchange
Commission. 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,
except as required by law.
Contacts
Investors:Holly Manning, 617-844-6630Director,
Investor RelationsHolly.Manning@agios.com
Media:Jessica Rennekamp, 857-209-3286Associate
Director, Corporate CommunicationsJessica.Rennekamp@agios.com
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