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Alnylam's Patisiran-LNP Pregnancy Surveillance Study: Key Insights for Investors
ByAinvest
Wednesday, Aug 27, 2025 11:56 pm ET1min read
Alnylam Pharmaceuticals is conducting a study to assess pregnancy outcomes in women exposed to patisiran-LNP, a treatment for hATTR amyloidosis. The study aims to gather data on pregnancy complications and fetal/neonatal outcomes. The study began in July 2021 and is currently recruiting participants. The results could impact Alnylam Pharmaceuticals' stock performance and market confidence in the treatment of hATTR amyloidosis.
Regeneron Pharmaceuticals and Alnylam Pharmaceuticals have reported encouraging results from a late-stage study on cemdisiran, an RNA medicine developed to treat generalized myasthenia gravis. The drug demonstrated robust efficacy in controlling symptoms of the chronic autoimmune disease in adults [1].The study, dubbed NIMBLE, tested cemdisiran alone and in combination with Regeneron's antibody drug Veopoz. While both treatments significantly reduced complement factor activity, cemdisiran monotherapy showed numerically greater treatment effects at 24 weeks compared to the combination. The drug's efficacy was comparable to currently approved C5-inhibiting drugs [1].
Regeneron plans to submit cemdisiran for U.S. approval in myasthenia gravis by the first quarter of next year. The company is also testing the drug in paroxysmal nocturnal hemoglobinuria and geographic atrophy related to age-related macular degeneration [1].
The study results have sparked interest in the biopharmaceutical sector, with shares in both Regeneron and Alnylam rising by about 1% on Tuesday. However, the combination of cemdisiran and Veopoz may not be as effective as the drug alone, which could impact future market strategies for the companies [1].
Meanwhile, Alnylam is conducting a study to assess pregnancy outcomes in women exposed to patisiran-LNP, a treatment for hATTR amyloidosis. The study, which began in July 2021, aims to gather data on pregnancy complications and fetal/neonatal outcomes. The results could impact Alnylam's stock performance and market confidence in the treatment of hATTR amyloidosis [2].
References:
[1] https://www.biopharmadive.com/news/regeneron-myasthenia-gravis-cemdisiran-alnylam-study-results/758607/
[2] https://www.biopharmadive.com/news/alnylam-study-patisiran-lnp-pregnancy-outcomes/758608/
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