United States

Profile: Agios Pharmaceuticals Inc (AGIO.OQ)

AGIO.OQ on NASDAQ Stock Exchange Global Select Market

17 Jan 2017
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Agios Pharmaceuticals, Inc., incorporated on August 7, 2007, is a biopharmaceutical company. The Company is engaged in the discovery and development of orally available small molecule medicines for the treatment of cancer and rare genetic disorders (RGDs), which are a subset of orphan genetic metabolic diseases. The Company's cancer product candidates are AG-221 and AG-120, which targets mutated isocitrate dehydrogenase 2 and 1, or IDH2 and IDH1, respectively, and AG-881, which targets both mutated IDH1 and mutated IDH2. These mutations are found in a range of hematological malignancies and solid tumors. The lead product candidate in its RGD programs, AG-348, which targets pyruvate kinase-R for the treatment of pyruvate kinase deficiency.


AG-221 is an orally available, selective, potent inhibitor of the mutated IDH2 protein indicated for the treatment of patients with cancers that harbor IDH2 mutations, including those with acute myeloid leukemia (AML). The United States Food and Drug Administration (FDA) granted the Company orphan drug designation for AG-221 for treatment of patients with AML. The FDA also granted Fast Track designation to AG-221 for treatment of patients with AML that harbor an IDH2 mutation. The Company is conducting Phase I dose escalation trials on AG-221 for evaluating both hematological and solid tumor cancers with IDH2 mutations. The clinical and preclinical data shows reduction of plasma 2HG levels, as well as evidence of cellular differentiation and normalization of cell counts in the bone marrow and blood.


AG-120 is an orally available, selective, potent inhibitor of the mutated isocitrate dehydrogenase (IDH1) protein for the treatment of patients with cancers that harbor IDH1 mutations. The Company is conducting over two Phase I studies for AG-120, one in patients with advanced hematologic malignancies and the second in patients with advanced solid tumors.


The Company's AG-881 is an orally available, selective, brain-penetrant, pan-IDH mutant inhibitor. AG-881 has completed investigational new drug (IND)-enabling studies.


AG-348 is an orally available small molecule and a potent activator of the wild-type (normal) and mutated protein kinase R (PKR) enzyme, which has resulted in restoration of adenosine triphosphate (ATP) levels and a decrease in 2,3-DPG levels in blood sampled from patients with PK deficiency in nonclinical studies. The wild-type PKR activity of AG-348 allowed the study of enzyme activation in healthy volunteers.


The Company's AG-519 is an orally available small molecule and its second product candidate that is a potent activator of the PKR enzyme. The Company has initiated a placebo-controlled phase I clinical trials of AG-519.

The Company competes with AstraZeneca, Calithera Biosciences, Cornerstone Pharmaceuticals, Inc., Eli Lilly and Company, Forma Therapeutics Holdings, LLC, GlaxoSmithKline plc, Merck & Co., Novartis International AG, Pfizer, Inc., Roche Holdings, Inc., Alexion Pharmaceuticals, Inc., BioMarin Pharmaceutical, Inc. and Shire Biochem, Inc.

Company Address

Agios Pharmaceuticals Inc

38 Sidney St Fl 2
CAMBRIDGE   MA   02139-4169
P: +1617.2725275
F: +1302.6555049

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