Agios Announces FDA Orphan Drug Designation Granted to Tebapivat (AG-946) for Treatment of Myelodysplastic Syndromes (MDS)
September 11 2024 - 7:00AM
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular
metabolism and PK activation pioneering therapies for rare
diseases, today announced that the U.S. Food and Drug
Administration (FDA) has granted orphan drug designation to the
company’s novel pyruvate kinase (PK) activator tebapivat (AG-946)
for the treatment of myelodysplastic syndromes (MDS).
“Receiving orphan drug designation for tebapivat in MDS
underscores the importance of bringing new oral treatment options
to patients suffering from this rare disease,” said Sarah Gheuens,
M.D., Ph.D., chief medical officer and head R&D at Agios. “We
aim to deliver the first oral therapy that addresses anemia due to
ineffective erythropoiesis in lower-risk MDS, which affects
approximately 75,000-80,000 patients in the U.S. and EU5 and
accounts for approximately 70% of MDS cases.”
Agios completed a Phase 2a study of tebapivat in lower-risk MDS
late last year and is currently initiating a Phase 2b study of
tebapivat in lower-risk MDS.
The FDA’s Office of Orphan Drug Products grants orphan drug
designation to support the development of medicines for rare
disorders that affect fewer than 200,000 people in the U.S. Under
the Orphan Drug Act, orphan drug designation qualifies a company
for incentives, including tax credits, exemptions from certain FDA
fees for clinical trials, and the potential for seven years of
market exclusivity following drug approval.
Mitapivat, the company’s lead PK activator, was previously
granted FDA orphan drug designation for the treatment of PK
deficiency, thalassemia, and sickle cell disease.
Tebapivat is not approved for use by any regulatory
authority.
About AgiosAgios is the pioneering leader in PK
activation and is dedicated to developing and delivering
transformative therapies for patients living with rare diseases. In
the U.S., Agios markets a first-in-class pyruvate kinase (PK)
activator for adults with PK deficiency, the first
disease-modifying therapy for this rare, lifelong, debilitating
hemolytic anemia. Building on the company's deep scientific
expertise in classical hematology and leadership in the field of
cellular metabolism and rare hematologic diseases, Agios is
advancing a robust clinical pipeline of investigational medicines
with programs in alpha- and beta-thalassemia, sickle cell disease,
pediatric PK deficiency, MDS-associated anemia and phenylketonuria
(PKU). In addition to its clinical pipeline, Agios is advancing a
preclinical TMPRSS6 siRNA as a potential treatment for polycythemia
vera. For more information, please visit the company’s 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 PYRUKYND® (mitapivat), tebapivat (AG-946),
TMPRSS6 siRNA and AG-181, Agios’ PAH stabilizer; Agios’ plans,
strategies and expectations for its preclinical, clinical and
commercial advancement of its drug development, including
PYRUKYND®, tebapivat and AG-181; Agios’ use of proceeds from the
transaction with Royalty Pharma; potential U.S. net sales of
vorasidenib and potential future royalty payments; Agios’ strategic
vision and goals, including its key milestones for 2024; and the
potential benefits of Agios’ strategic plans and focus. The words
“anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,”
“potential,” “possible,” “strategy,” “will,” “vision,” 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, without limitation: risks and
uncertainties related to the impact of pandemics or other public
health emergencies 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; 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, the EMA or 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 establish and maintain key collaborations; uncertainty
regarding any milestone or royalty payments related to the sale of
its oncology business or its in-licensing of TMPRSS6 siRNA, and the
uncertainty of the timing of any such payments; uncertainty of the
results and effectiveness of the use of Agios’ cash and cash
equivalents; and general economic and market 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:
Investor ContactChris Taylor, VP, Investor
Relations and Corporate CommunicationsAgios
PharmaceuticalsIR@agios.com
Media Contact Dan Budwick1AB
dan@1abmedia.com
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