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BioWorld - Saturday, December 6, 2025
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Home » Regeneron's cemdisiran hits phase III endpoints in myasthenia gravis
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Regeneron's cemdisiran hits phase III endpoints in myasthenia gravis

Aug. 26, 2025
By Karen Carey
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Amid the increasingly competitive myasthenia gravis drug development space, siRNA candidate cemdisiran met phase III endpoints, with the monotherapy showing numerically higher results than a combination product. Regeneron Pharmaceuticals Inc., which has a worldwide license to cemdisiran from Alnylam Pharmaceuticals Inc., plans to file for U.S. approval in generalized myasthenia gravis, a rare and chronic autoimmune disease leading to life-threatening muscle weakness, in the first quarter of 2026.
BioWorld Clinical Immune Musculoskeletal RNAi U.S.

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