Agios Announces Phase 3 ACTIVATE-T Trial of Mitapivat Achieved Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused

– 37 Percent of Patients Treated with Mitapivat Achieved ≥33% Reduction in Transfusion Burden Compared to Individual Historical Transfusion Burden Standardized to 24 Weeks (1-Sided p=0.0002) –

– 22 Percent of Patients Treated with Mitapivat Were Transfusion-Free During the 24-Week Fixed Dose Period –

– Safety Profile Consistent with Previously Reported Data –

– Company Expects to File for Regulatory Approval for Mitapivat for the Treatment of Adults with Pyruvate Kinase (PK) Deficiency in the U.S. in Q2 2021 and in the EU in Mid-2021 –

CAMBRIDGE, Mass., Jan. 26, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced that the global, open-label Phase 3 ACTIVATE-T trial of mitapivat in regularly transfused adults with PK deficiency demonstrated a statistically significant and clinically meaningful reduction in transfusion burden. All 27 patients enrolled in the study were treated with mitapivat. In the 24-week fixed dose period, 37 percent (n = 10) achieved a ≥33% reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks (1-sided p=0.0002). In addition, 22 percent (n = 6) were transfusion-free during the 24-week fixed dose period. Mitapivat is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.

Agios recently reported that its global, randomized, double-blind, placebo-controlled Phase 3 ACTIVATE trial of mitapivat in adults with PK deficiency who do not receive regular transfusions met its primary endpoint, with 40 percent of patients randomized to mitapivat achieving a hemoglobin response, defined as a ≥1.5 g/dL sustained increase in hemoglobin concentration from baseline, compared to 0 patients randomized to placebo (2-sided p<0.0001). Agios anticipates filing for regulatory approval based on data from ACTIVATE and ACTIVATE-T in the U.S. in Q2 2021 and in the EU in mid-2021, with a potential 2022 commercial launch in both geographies.

“Based on results from both the ACTIVATE and ACTIVATE-T Phase 3 trials, we believe mitapivat has the potential to make a meaningful difference for people living with pyruvate kinase deficiency, a debilitating, lifelong hemolytic anemia characterized by serious complications regardless of patients’ transfusion status. The ACTIVATE-T study represents our first study of mitapivat in regularly transfused patients, and when taken together with the ACTIVATE results, demonstrates mitapivat’s potential clinical benefit for patients regardless of transfusion burden,” said Chris Bowden, M.D., chief medical officer at Agios. “We look forward to working with regulatory authorities in both the U.S. and EU to rapidly bring mitapivat to patients as the first potentially disease-modifying therapy for this community that currently has limited treatment options.”