Agios Pharma reports mixed results in late-stage study for sickle cell disease drug

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(Reuters) -Agios Pharmaceuticals said on Wednesday its sickle cell drug met one of its two main goals in a late-stage study of patients aged 16 and older, but failed to show a statistically significant reduction in pain crises.

Shares of the Cambridge, Massachusetts-based drugmaker were down nearly 50% in premarket trading.

Sickle cell disease is a severe inherited blood ailment that affects the shape of red blood cells, which carry oxygen to all parts of the body.

Agios Pharma’s drug, mitapivat, works by activating an enzyme in red blood cells to boost energy production, helping them survive longer and improve hemoglobin levels.

In a late-stage study testing mitapivat, 40.6% of patients showed increased levels of hemoglobin compared with 2.9% patients on a placebo.

During the study, the drug improved hemoglobin levels and reduced markers of red blood cell breakdown – from week 24 through week 52.

However, it did not significantly reduce sickle cell pain crises, which was one of the main goals to assess treatment efficacy. A pain crisis is caused by sickled red blood cells blocking small blood vessels, reducing oxygen delivery to tissues and organs.

It also did not meet a secondary goal of fatigue score, the company said. Fatigue score measures how tired patients feel, affecting their daily life after the treatment.

The company added that three patients on mitapivat and two on placebo died during the trial, but none of the deaths were related to the treatment.

Mitapivat, under the brand name of Pyrukynd, is already approved by the U.S. Food and Drug Administration in 2022 to treat low red blood cell counts caused by the early breakdown of red blood cells in adults with pyruvate kinase deficiency (PKD).

The company plans to submit a marketing application for mitapivat in the U.S. for sickle cell disease after meeting with the FDA in the first quarter of 2026.

(Reporting by Siddhi Mahatole in Bengaluru; Editing by Krishna Chandra Eluri)