Profile: Agios Pharmaceuticals Inc (AGIO.OQ)
21 Jul 2017
Agios Pharmaceuticals, Inc., incorporated on August 7, 2007, is a clinical-stage biopharmaceutical company. The Company's therapeutic areas of focus are cancer and rare genetic metabolic disorders, which are a group of over 600 rare genetic diseases caused by mutations, or defects, of single metabolic genes. The Company's cancer product candidates are enasidenib and ivosidenib (AG-120), which target mutated isocitrate dehydrogenase 2 (IDH2) and isocitrate dehydrogenase 1 (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 rare genetic metabolic disorder (RGD), programs, AG-348, targets pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency.
Enasidenib is a therapeutic candidate for the treatment of patients with cancers that harbor IDH2 mutations, including those with acute myeloid leukemia (AML). Enasidenib is being evaluated in a Phase I/II multicenter, open-label, clinical trial to assess the safety, clinical activity, and tolerability of enasidenib in patients with advanced hematologic malignancies with an IDH2 mutation. AG-120 is a therapeutic candidate for the treatment of patients with cancers that harbor IDH1 mutations. Enasidenib is being evaluated in IDHENTIFY, an international Phase III, multi-center, open-label, randomized clinical trial designed to compare the efficacy and safety of enasidenib versus conventional care regimens in patients 60 years or older with IDH2 mutant-positive AML that is refractory to or relapsed after second- or third-line therapy. Ivosidenib (AG-120) is an orally available, selective, potent inhibitor of the mutated IDH1 protein, making it a therapeutic candidate for the treatment of patients with cancers that harbor IDH1 mutations. Ivosidenib (AG-120) is being evaluated in a Phase I multicenter, open-label, dose-escalation and expansion clinical trial, as a single agent in patients with advanced hematologic malignancies and advanced solid tumors with an IDH1 mutation.
Ivosidenib (AG-120) is being evaluated in ClarIDHy, a Phase III, multicenter, randomized, double-blind, placebo-controlled clinical trial of ivosidenib (AG-120) in previously-treated patients with nonresectable or metastatic cholangiocarcinoma with an IDH1 mutation. AG-881 is an orally available, selective, brain-penetrant, pan-IDH mutant inhibitor. The Company is conducting two Phase I multi-center, open-label clinical trials of AG-881, one in patients with advanced IDH1 or IDH2 mutant-positive solid tumors, including glioma, and the other in patients with advanced IDH1 or IDH2 mutant-positive hematologic malignancies whose cancer has progressed on a prior IDH inhibitor therapy. AG-348 is an orally available small molecule and a potent activator of the wild-type (normal) and mutated PKR enzyme, which has resulted in restoration of adenosine triphosphate (ATP), levels and a decrease in 2,3-diphosphoglycerate (2,3-DPG), levels in blood sampled from patients with PK deficiency and treated ex-vivo with AG-348.
The Company competes with AstraZeneca Plc, Bayer AG, Calithera Biosciences, Cornerstone Pharmaceuticals, Inc., Daiichi Sankyo Company, Ltd., Eli Lilly and Company, Forma Therapeutics Holdings, LLC, GlaxoSmithKline plc, Merck & Co., Novartis International AG, Pfizer, Inc., Roche Holdings, Inc., Bristol-Myers Squibb Company, Alexion Pharmaceuticals, Inc., BioMarin Pharmaceutical, Inc., Sanofi, Shire Biochem, Inc., Jazz Pharmaceuticals plc, Seattle Genetics, Inc. and Abbvie Inc.
Agios Pharmaceuticals Inc
88 Sidney St
CAMBRIDGE MA 02139-4137
Company Web Links
- BRIEF-Agios appoints David Scadden to the board of directors
- BRIEF-Thermo Fisher Scientific signs development agreement with Agios Pharmaceuticals
- BRIEF-Agios Pharmaceuticals to present updated clinical data from PKR activator AG-348
- BRIEF-Agios Pharmaceuticals Inc Q1 net loss was $66.2 million
- BRIEF-Agios announces closing of over-allotment option in public offering