Agios Initiates Phase 1 Study of AG-348, a First-in-class PKR Activator, for Pyruvate Kinase Deficiency
- First Agios Clinical Program in Inborn Errors of Metabolism -
- Third Agios Program to
“Today’s announcement marks the initiation of our first clinical program
in IEMs and our third as a company,” said
“Preclinical studies have demonstrated that AG-348 activates a broad
spectrum of PK-R mutant proteins, and corrects the metabolic defects
found in patient-derived blood samples,” said
About the Study
The Phase 1, single-center, randomized, double-blind, placebo-controlled clinical trial will assess the safety and tolerability of AG-348 through dose escalation in healthy adult men and women. Key objectives of the trial include characterizing the safety, pharmacokinetic and pharmacodynamic relationships of AG-348 and select metabolic biomarkers. Please refer to www.clinicaltrials.gov for additional clinical trial details.
About Pyruvate Kinase (PK) Deficiency
PK deficiency is a rare inherited disease that presents as hemolytic anemia due to the accelerated destruction of red blood cells (RBCs). PK deficiency is caused by mutations that affect the activity of the metabolic enzyme PK-R, the form of pyruvate kinase that is present in RBCs. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, chelation therapy to address iron overload and/or interventions for other treatment- and disease-related morbidities. Currently, there is no approved therapy to treat the underlying cause of PK deficiency.
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 pyruvate kinase R as a therapeutic
target; the potential benefits of Agios’ product candidate AG-348; its
plans and timelines for the clinical development of AG-348; and 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.
Source:
Agios Pharmaceuticals, Inc.
Media Contact:
Dan Budwick,
973-271-6085
dan@purecommunicationsinc.com
or
Investor
Contact:
Glenn Goddard
investors@agios.com
