Agios Pharmaceuticals, Inc. presented detailed results from the 52-week double-blind period of the global RISE UP Phase 3 trial of mitapivat in patients with sickle cell disease at the 31st European Hematology Association (EHA) Congress in Stockholm, Sweden. The data demonstrated a statistically significant improvement in hemoglobin response and a clinically meaningful reduction in transfusion burden compared with placebo. These findings reinforce the potential of mitapivat, an oral pyruvate kinase (PK) activator, to address the critical need for new treatments in this patient population.

The RISE UP trial met its primary endpoint of hemoglobin response, with 40.6% of patients in the mitapivat arm achieving a ≥1.0 g/dL increase from baseline compared with 2.9% in the placebo arm. New analyses revealed that patients receiving mitapivat experienced a 41.1% relative reduction in the proportion of patients requiring blood transfusions and a 55.9% relative reduction in average red blood cell units transfused per patient. The safety profile was consistent with previous trials, with no treatment-related deaths reported.

Transfusion Burden and Hemoglobin Response

New analyses from the RISE UP trial highlighted the impact of mitapivat on transfusion burden. Patients in the mitapivat arm showed a significant decrease in the need for supportive care measures.

The trial also included a post-hoc analysis of hemoglobin responders, defined as patients achieving a ≥1.0 g/dL increase in average hemoglobin from Week 24 through Week 52. Among responders in the mitapivat arm, the mean change from baseline in hemoglobin concentration was 1.6 g/dL.

Clinical Benefits for Responders

Hemoglobin responders in the mitapivat arm experienced clinically meaningful reductions in pain crises and related healthcare utilization compared with non-responders. These patients reported improvements across several patient-reported outcomes, including measures of pain, sleep, and physical function.

Improvements in patient-reported fatigue scores exceeded the predefined threshold for clinical meaning. Responders also showed favorable results in PROMIS Pain Intensity, ASCQ-Me Pain Impact, PROMIS Physical Functioning, ASCQ-Me Sleep Impact, and EQ-5D VAS scores.

Safety Profile and Regulatory Status

Mitapivat was well-tolerated during the trial, with a safety profile consistent with previous studies in sickle cell disease. The incidence of treatment-emergent adverse events was similar between the mitapivat and placebo arms. In May 2026, Agios announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of mitapivat in sickle cell disease.

JP Morgan analyst Tessa Romero has maintained a Neutral rating on Agios Pharmaceuticals (NASDAQ: AGIO) while lowering the price target to $31 from $36. The revised target indicates a change in the firm's valuation expectations for the stock.

Rating and Price Target Details

The brokerage's decision to retain the Neutral stance suggests that the risk-reward profile for Agios Pharmaceuticals remains balanced at current levels. The reduction in the price target to $31 signals a more conservative view on the stock's near-term upside potential.

The adjustment comes as market participants assess the company's operational progress and pipeline developments. Agios Pharmaceuticals continues to focus on its core therapeutic areas, though the specific drivers for the target cut were not detailed in the immediate release.