Agios shares tumble on mixed results for sickle cell drug trial

By Siddhi Mahatole

(Reuters) -Agios Pharmaceuticals said on Wednesday its sickle cell disease 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, sending the company’s shares down 50% in morning trade.

Although the drug mitapivat improved hemoglobin levels, it failed to reduce sickle cell pain crises, the debilitating pain episodes that are a hallmark of sickle cell disease, missing one of the study’s two main goals.

In sickle cell disease, normally round, flexible red blood cells become rigid and sickle-shaped causing them to get stuck in small blood vessels and trigger severe pain crises.

In the trial, 40.6% of patients given mitapivat achieved a meaningful rise in hemoglobin, compared with 2.9% of those on placebo.

The treatment also missed a secondary goal of fatigue score, which gauges how tired patients feel in daily life after the treatment.

“We think investors wanted to see both of these endpoints hit, and as such we view the results as falling short of expectations,” said J.P. Morgan analyst Tessa Romero, adding that the goal of reducing severe pain “was unanimously viewed as more challenging to meet, which kept us more measured.”

Three patients on mitapivat and two on placebo died during the trial, but none of the deaths were linked to treatment, the company said.

Mitapivat, sold as Pyrukynd, is already approved in the U.S. to treat adults with pyruvate kinase deficiency.

Agios said it plans to pursue U.S. approval for mitapivat in 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 and Tasim Zahid)