Agios Outlines 2026 Strategic Priorities and Key Milestones to Accelerate Rare Disease Portfolio Growth

Core Insights - Agios Pharmaceuticals has outlined its strategic priorities and key milestones for 2026, focusing on the launch of AQVESME™ and expansion into additional indications for its PK activation franchise [1][3] Company Performance - In 2025, Agios achieved significant progress, highlighted by the U.S. approval of AQVESME™, the first medicine approved for treating anemia in adults with alpha- or beta-thalassemia [2][9] - The company aims to become a sustainable and diversified rare disease company, leveraging its existing commercial presence [2][3] 2026 Strategic Priorities - The company plans a high-impact U.S. launch of AQVESME in thalassemia and seeks to expand its PK activation franchise into sickle cell disease and lower-risk myelodysplastic syndromes [3][8] - Agios is committed to disciplined capital allocation and operational efficiency to support long-term sustainability [3] Anticipated Milestones - AQVESME's U.S. commercial launch is set for late January 2026, following FDA approval in December 2025 [9] - A pre-sNDA meeting with the FDA for mitapivat in sickle cell disease is anticipated in Q1 2026, with a regulatory submission to follow [8][9] - Topline results from the Phase 2b trial of tebapivat in lower-risk myelodysplastic syndromes are expected in the first half of 2026 [6][8] - Enrollment in the Phase 2 sickle cell disease trial of tebapivat was initiated in 2025, with topline results expected in the second half of 2026 [9] Pipeline Development - Agios is advancing its early- and mid-stage pipeline across multiple high-value indications, including a Phase 1 trial of AG-236 for polycythemia vera and a Phase 1b trial of AG-181 for phenylketonuria [10][11]