AGIO Stock Cracks After Sickle-Cell Disease Drug Delivers Mixed Results In Late-Stage Trial

• The company said that the late-stage study of Mitapivat in patients with sickle cell disease met the primary endpoint of hemoglobin response. 
• The drug also yielded positive outcomes on change from baseline in hemoglobin concentration and indirect bilirubin levels compared with placebo. 
• While Mitapivat also showed a reduction in the primary endpoint of annualized rate of sickle cell pain crises compared to placebo, this trend did not achieve statistical significance, the company noted.

Shares of Agios Pharmaceuticals, Inc. (AGIO) slumped 48% on Wednesday after the company reported mixed results from its drug trial in sickle cell disease.

Sickle cell disease is an inherited blood disorder where red blood cells become rigid, sickle-shaped, and unable to flow easily through blood vessels, causing pain and organ damage. The condition leads to complications like painful episodes or crises, anemia, infections, stroke, and damage to organs.

What Did The Study Show?

The company said that the late-stage study of Mitapivat in patients aged 16 years and older with sickle cell disease met the primary endpoint of hemoglobin response, with the drug demonstrating a statistically significant improvement compared to placebo. Hemoglobin response is defined as an increase from baseline in average hemoglobin by more than or equal to 1.0 g/dL.

The drug also showed positive effects on changes from baseline in hemoglobin concentration and indirect bilirubin levels compared with placebo.

While Mitapivat also showed a reduction in the primary endpoint of annualized rate of sickle cell pain crises (SCPCs) or acute pain needing medical contact compared to placebo, this trend did not achieve statistical significance, the company noted. Further, there were no improvements from baseline on fatigue scores among patients, the company noted.

The firm now expects to share the comprehensive results from the clinical program with the FDA in the first quarter of 2026 and seek potential U.S. regulatory approval.

How Did Stocktwits Users React?

On Stocktwits, retail sentiment around AGIO stock jumped from ‘bearish’ to ‘extremely bullish’ territory over the past 24 hours, while message volume increased from ‘high’ to ‘extremely high’ levels.

AGIO stock is down by 28% this year and by 57% over the past 12 months.

Also See: KZIA Stock Soars After Stage IV Cancer Patient Shows Initial Complete Remission On Its Drug Combination

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