Agios Pharmaceuticals Initiates Multiple Ascending Dose Trial in Healthy Volunteers of AG-348 for the Potential Treatment of PK Deficiency, a Rare, Hemolytic Anemia
Initial Safety and Pharmacokinetic Findings From Single Ascending Dose Trial Support Early Advancement
“AG-348 represents a completely new approach to treating patients with
PK deficiency, a serious form of inherited hemolytic anemia with no
approved therapy that targets the metabolic defect responsible for the
underlying disease,” said
Preclinical studies have demonstrated that AG-348 activates a broad spectrum of PKR mutant proteins, and corrects the metabolic defects found in patient-derived blood samples. AG-348 is currently the only drug candidate being evaluated for the potential of correcting metabolic defects found in patients with PK deficiency.
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 multiple ascending doses 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, a Rare, Inherited Hemolytic Anemia
Pyruvate kinase (PK) deficiency, a rare, inherited hemolytic anemia affecting children and adults, is caused by mutations that affect the activity of the metabolic enzyme pyruvate kinase-R (PKR), the form of pyruvate kinase that is present in red blood cells. 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. AG-348 is a first-in-class orally available, potent, selective small molecule activator of PKR, which, when mutated, leads to PK deficiency. AG-348 was discovered in the laboratory of Agios, and the company retains worldwide development and commercialization rights.
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,” “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.
Agios Pharmaceuticals, Inc.
Lora Pike, 617-649-8608