Business
Agios Reports Business Highlights and First Quarter 2024 Financial Results
– Topline Data Readout from the Phase 3 ENERGIZE-T Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia now expected in Q2 2024

About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– Topline Data Readout from the Phase 3 ENERGIZE-T Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia now expected in Q2 2024 – – Announced Positive Results from the Phase 3 ENERGIZE Study of Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia in January – – Phase 3 Readouts from Mitapivat RISE UP Study in Sickle Cell Disease, ACTIVATE-KIDS and ACTIVATE KIDS-T in Pediatric PK Deficiency, All Expected by End of 2025 – – PYRUKYND® (Mitapivat) Net Revenue of $8.2 Million in Q1; Cash, Cash Equivalents and Marketable Securities of $714.3 Million as of March 31, 2024 – CAMBRIDGE, Mass., May 02, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today reported business highlights and financial results for the first quarter ended March 31, 2024. “We were delighted to report positive data from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia and look forward to announcing topline data from the Phase 3 ENERGIZE-T study in transfusion-dependent thalassemia in the second quarter of this year,” said Brian Goff, chief executive officer at Agios. “Mitapivat has the potential to become the first therapy approved for all thalassemia subtypes, and our commercial organization is actively preparing for a potential launch next year. Beyond thalassemia, we look forward to the RISE UP Phase 3 readout in sickle cell disease next year, with potential for approval in 2026, as we progress toward our vision of becoming a leading rare disease company with a potential multi-billion-dollar franchise in PK activation.” First Quarter 2024 and Recent Highlights PYRUKYND® Revenues: Generated $8.2 million in net revenue for the first quarter of 2024, a 15 percent sequential increase from the fourth quarter of 2023, primarily driven by increased patient demand. A total of 188 unique patients have completed prescription enrollment forms, representing an increase of 6 percent over the fourth quarter of 2023. A total of 120 patients are on PYRUKYND® therapy, a 10 percent increase from the fourth quarter of 2023.Thalassemia: Announced positive results from the Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent alpha- or beta-thalassemia.Earlier-Stag...