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Despite missing a key endpoint, there's still likely to be value in mitapavit.
Management plans for a marketing application early in 2026.
Shares in Agios Pharmaceuticals (NASDAQ: AGIO) crashed by 50% by noontime today. The move comes as the market digests a mixed set of topline results from its Phase 3 trial (RISE UP) of its sickle cell disease drug, mitapavit.
The trial was designed with two primary endpoints, and it hit one but missed the other. Sickle cell disease is an inherited blood disorder in which a gene mutation causes red blood cells to produce an abnormal form of hemoglobin, known as hemoglobin S. When hemoglobin S releases oxygen, it causes red blood cells to clump, forming sickle cells, which typically die in a 10 to 20-day period compared to 120 days for normal red blood cells.
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Some of its symptoms include fatigue, itching and discomfort, and intense periods of pain referred to as sickle cell pain crises (SCPCs).
The mitapavit trial met its primary endpoint of generating a statistically significant response in average hemoglobin (40.6% of treated patients compared to 2.9% in the placebo group). However, it failed in generating a statistically significant reduction in SCPCs, with an annual rate of 2.62 in the treatment group and 3.05 in the placebo group.
Image source: Getty Images.
Clearly, the market was left unimpressed by the SCPC news, but there's obviously some value in mitapavit, with management lauding its "anti-hemolytic profile", not least as increased hemoglobin will combat fatigue. As such, management plans to engage with the Food and Drug Administration (FDA) regarding Mitapavit and plans to submit a marketing application in the first quarter of 2026. Time will tell if the market is overreacting here.
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