Alnylam Shares Detailed Patisiran Data From Rare Disease Study

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  • Alnylam Pharmaceuticals Inc ALNY announced positive results from the APOLLO-B Phase 3 study of patisiran for transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. 
  • The company previously announced topline results from the APOLLO-B study in August 2022.
  • The 12-month study achieved its primary endpoint, with patisiran demonstrating a statistically significant and clinically meaningful benefit on functional capacity, as measured by the 6-Minute Walk Test (6-MWT), compared to placebo, with a median difference of 14.7 meters favoring patisiran. 
  • The study also included additional secondary composite outcomes endpoints. A non-significant result was found on the composite endpoint of all-cause mortality, frequency of cardiovascular events, and change from baseline in 6-MWT over 12 months compared to placebo. 
  • Patisiran also demonstrated an encouraging safety and tolerability profile in patients with ATTR amyloidosis with cardiomyopathy.
  • Patisiran achieved a rapid and sustained reduction in serum TTR levels, with a mean percent reduction from baseline in serum TTR reduction of 87% at Month 12.
  • On the safety front, Patisiran demonstrated an encouraging safety and tolerability profile, including no cardiac safety concerns relative to the placebo.
  • Most adverse events (AEs) were mild or moderate in severity. The most common treatment-emergent AEs were infusion-related reactions and muscle spasms.
  • Five deaths were observed in patisiran-treated patients and eight in the placebo group.
  • Price Action: ALNY shares are up 2.80% at $214.72 on the last check Thursday.
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