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Home » Agios planning sNDA on mixed sickle cell data for mitapivat
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Agios planning sNDA on mixed sickle cell data for mitapivat

Nov. 19, 2025
By Jennifer Boggs
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“Our goal is that by the end of this call, you’ll share our conviction in the totality of the data.” So said Agios Pharmaceuticals Inc. CEO Brian Goff as the company reported top-line data from its 52-week Rise Up trial testing mitapivat in sickle cell disease showing the oral pyruvate kinase activator hit statistical significance on one of two primary endpoints and two of three key secondary endpoints.
BioWorld Clinical Hematologic Small molecule U.S.

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