Exhibit 99.2
![LOGO](https://capedge.com/proxy/8-K/0001193125-20-167148/g935991img1.jpg)
AgiosFirst-in Class PKR Activator Mitapivat Demonstrates Sustained Hemoglobin Responses inNon-transfusion-dependentα- and ß-Thalassemia in Phase 2 Study
– Treatment with Mitapivat Induced Hemoglobin (Hb) Increase of³1.0 g/dL in 12 of 13 (92%) Evaluable Patients, Including 4 of 4 (100%)α-Thalassemia Patients, During Weeks4-12 –
– 7 of 8 (88%) Evaluable Patients Achieved Sustained Hb Response During Weeks12-24 –
– Thalassemia Pivotal Development Plan Expected to be Finalized byYear-End 2020 and Initiated in 2021 –
– Company to Host Investor Webcast Today at 7:30 a.m. ET –
CAMBRIDGE, Mass., June 12, 2020— Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today reported interim data from its ongoing Phase 2 study evaluating single agent mitapivat innon-transfusion-dependent α- and ß-thalassemia. Data from the study were featured in an oral presentation at the 25th European Hematology Association Annual Congress, which is being held virtually. Mitapivat is an investigational,first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvatekinase-R (PKR) enzymes.
“These data are exciting and further validate the potential of PKR activation as an entirely new mechanism for treating thalassemia, including α-thalassemia, for which there have been few medical advancements,” said Kevin Kuo, M.D., hematologist at University Health Network, University of Toronto, and an investigator in the study. “Findings from the study indicate that activation of wild-type PKR by mitapivat, an oral treatment option, improved hemoglobin and associated markers of hemolysis and erythropoiesis in patients with α- and ß-thalassemia. In addition, the safety profile was consistent with previously published data for mitapivat.”
“We are pleased to share the impressive interim results from our clinical study of mitapivat inα-and ß-thalassemia, as the data validatepre-clinical work conducted in our laboratories and with academic collaborators and demonstrate the potential for PKR activators in hemoglobinopathies such as thalasemmia and sickle cell disease,” said Chris Bowden, chief medical officer at Agios. “Our focus now is to advance the development of mitapivat for these patients as quickly and efficiently as possible. By the end of the year, we expect to finalize a robust pivotal development plan that spans bothα-and ß-thalassemia, as well as transfusion dependent and non-tranfusion dependent patients, with a goal of initiating a pivotal program in 2021.”
Mitapivat Phase 2Proof-of-concept Study
The ongoing, open-label Phase 2 study is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of mitapivat treatment in adults withnon-transfusion-dependent α- and ß-thalassemia who have a baseline hemoglobin (Hb) concentration of£10 g/dL. The trial is fully enrolled with 20 patients, and includes a24-week core period followed by a2-year extension