Agios Pharmaceuticals Announces Orphan Drug Designation of AG-348 for Treatment of Pyruvate Kinase Deficiency
- PK deficiency is a severe and rare genetic disease with currently no approved or disease modifying treatments -
"We are pleased to achieve another milestone in the clinical program for AG-348, the first medicine in development designed to treat the underlying cause of PK deficiency," said
About AG-348 Clinical Development Plans and Upcoming Milestones
Based on findings presented at ASH, Agios expects to initiate a Phase 2 study of AG-348 in patients with PK deficiency in the first half of 2015. The company plans to provide final results from the Phase 1 multiple ascending dose study at a medical conference in mid-2015. A natural history study of PK deficiency is also ongoing and Agios expects to report initial data from this study at a medical conference in mid-2015.
About Pyruvate Kinase (PK) Deficiency, a Rare, Inherited Hemolytic Anemia
Pyruvate kinase (PK) deficiency, a rare, inherited hemolytic anemia affecting children and adults. It 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. When this enzyme (or protein) is deficient, people with PK deficiency have fewer healthy 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. Agios retains worldwide development and commercialization rights for AG-348.
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' drug candidate 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 Senior Director, Investor Relations and Public Relations firstname.lastname@example.org