Agios Q4 2019 Earnings Report

Agios expects the following key milestones in 2020: Hematologic Malignancies Deliver full-year U.S. revenue for TIBSOVO® of $105-115 million Receive European Medicines Agency CHMP opinion for TIBSOVO® in relapsed or refractory acute myeloid leukemia (AML) with an IDH1 mutation by year-end Complete enrollment of the Phase 3 AGILE trial of TIBSOVO® in combination with azacitidine in adult patients with previously untreated IDH1 mutant AML by year-end Complete enrollment of the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO® Phase 1 study of IDH1 mutant advanced hematologic malignancies by year-end Solid Tumors File supplemental new drug application (sNDA) for TIBSOVO® in previously treated IDH1 mutant cholangiocarcinoma by year-end Rare Genetic Diseases Announce topline data for ACTIVATE and ACTIVATE-T pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end Submit updated data from the Phase 2 study of mitapivat in thalassemia for presentation at the European Hematology Association (EHA) Congress in June and finalize pivotal development strategy by year-end Achieve proof-of-concept for mitapivat in sickle cell disease by mid-2020 Receive investigational new drug (IND) clearance for AG-946, a next generation PKR activator, and initiate a first-in-human study in healthy volunteers in the first half of 2020 Research Achieve at least one new development candidate by year-end