AGIO Stock Hits 52-Week Low on Mixed Sickle Cell Disease Study Results

Core Insights - Agios Pharmaceuticals' shares dropped approximately 51% following mixed results from the phase III RISE UP study of Pyrukynd in sickle cell disease patients [1][6] - The study met its primary endpoint of improving hemoglobin levels but failed to reduce the annualized rate of sickle cell pain crises [1][7] Efficacy Results - Nearly 41% of patients treated with Pyrukynd achieved a hemoglobin response compared to about 3% in the placebo group [2] - Pyrukynd-treated patients showed a numerical reduction in sickle cell pain crises (2.62 vs. 3.05 for placebo) but did not achieve statistical significance [2][3] - Statistically significant improvements were observed in average hemoglobin concentration and indirect bilirubin levels, but no improvement in patient-reported fatigue was noted [3] Regulatory Plans - Despite mixed results, Agios plans to proceed with a regulatory filing for Pyrukynd, focusing on a subgroup of hemoglobin responders who showed clearer clinical benefits [4] - The company aims to submit this filing after discussions with the FDA in the first quarter of 2026 [4] Current Drug Status - Pyrukynd is already approved in the U.S. and Europe for treating hemolytic anemia in adults with pyruvate kinase deficiency [5] - A regulatory filing for label expansion to include non-transfusion-dependent and transfusion-dependent thalassemia patients is under FDA review, with a decision expected by December 7, 2025 [5] Stock Performance - Following the study results, Agios shares reached a 52-week low of $22.24, reflecting investor disappointment [6] - Year-to-date, Agios shares have declined by 32%, contrasting with a 17% growth in the industry [6] Competitive Landscape - Shares of Fulcrum Therapeutics rose over 18% after Agios' announcement, as Fulcrum is developing a different investigational drug for sickle cell disease [9]