Agios Pharmaceuticals announces anticipated 2024 milestones

“We were pleased to announce positive topline data from the Phase 3 study of our lead PK activator, mitapivat, in non-transfusion-dependent alpha- or beta-thalassemia last week, a segment of the population with no currently approved therapeutic options in the U.S. We look forward to data readouts from four additional Phase 3 studies across our industry-leading PK activator franchise by the end of 2025,” said Brian Goff, chief executive officer at Agios. “This robust series of near-term catalysts positions Agios for potential launches of a first- and best-in-class therapy in thalassemia in 2025 and in sickle cell disease in 2026, and we look forward to maximizing the commercial opportunities ahead of us. Supported by our strong cash position, Agios is poised for significant progress in the next 12-24 months, and we look forward to the opportunity to deliver a novel oral treatment option for two additional hematologic diseases with high unmet need.” Anticipated 2024 Milestones Thalassemia: Following the announcement of topline data from the Phase 3 ENERGIZE study last week, Agios plans to report topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia mid-year and submit a New Drug Application for mitapivat in thalassemia year-end; Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat year-end; Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat mid-2024. Report topline data from Phase 3 ACTIVATE kids-T study year-end; Lower-risk Myelodysplastic Syndromes: Dose first patient in Phase 2b study of AG-946 mid-year; Earlier-stage Pipeline: Dose the first patient in the Phase 1 study of PAH stabilizer for the treatment of PKU H1 2024; Four Additional Phase 3 Readouts and Two Potential New Indication Approvals Expected by End of 2026: 2024: Data readout from Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia announced January 3, 2024; Data readout from Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia mid-year; Data readout from Phase 3 ACTIVATE kids-T study of mitapivat in pediatric PK deficiency year-end; 2025: Data readout from Phase 3 portion of the RISE UP study of mitapivat in sickle cell disease; Data readout from Phase 3 ACTIVATE kids study of mitapivat in pediatric PK deficiency; Potential FDA approval for mitapivat in thalassemia; 2026: Potential FDA approval for mitapivat in sickle cell disease Potential FDA approval for mitapivat in pediatric PK deficiency.