Agios Shares Plunge 50% After Mixed Phase 3 RISE UP Results For Mitapivat

(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO) fell 50.27%, closing at $22.62, down $22.87, following the release of topline results from its Phase 3 RISE UP trial of mitapivat in sickle cell disease.

The study met its primary endpoint: 40.6% of patients on mitapivat achieved a hemoglobin response versus 2.9% on placebo. The drug also produced statistically significant improvements in hemoglobin concentration and indirect bilirubin levels. However, it failed to achieve statistical significance for the annual rate of sickle-cell pain crises or fatigue, both key endpoints.

The company plans to meet with the U.S. Food and Drug Administration for a pre-sNDA discussion in Q1 2026 and intends to submit a marketing application thereafter. Tied to its prior thalassemia approval of PYRUKYND, the company emphasized efforts to manage costs and maintain focus on its rare-disease pipeline.

On the day of the announcement, AGIO experienced unusually heavy trading volume as investors absorbed the mixed trial findings. The stock's 52-week range is approximately $12.34 - $48.75.