Alnylam' Reveals Detailed 18-Month Data From Vutrisiran Trial In Inherited Disease

Alnylam Pharmaceuticals Inc ALNY has announced detailed data from HELIOS-A Phase 3 study of vutrisiran at 18 months in patients with transthyretin-mediated (ATTR) amyloidosis.

• Transthyretin amyloidosis is characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. 
• The details follow topline data announced in October 2021, showing that the Phase 3 trial met all secondary endpoints measured at 18 months. 
• Vutrisiran treatment (N=122) resulted in a 0.46 point mean decrease (improvement) in the modified Neuropathy Impairment Score compared to a 28.09 point mean increase (worsening) in the placebo group (N=77).
• Vutrisiran showed a 1.2 point mean decrease (improvement) in quality of life score versus a 19.8 point mean increase (worsening) in the placebo arm.
• Vutrisiran treatment resulted in 0.024 meters/second mean decrease in 10-MWT compared to a 0.264 in the placebo group.
• Vutrisiran exhibited a 25.0 point mean increase (improvement) in nutritional status vs. a 115.7 point mean decrease in patients on placebo.
• The data exhibited statistically significant improvement in clinical endpoints compared to placebo and non-inferiority in serum TTR reduction compared to the within-study Onpattro (patisiran) arm.
• Vutrisiran is under review by the FDA (action date April 14, 2022), the European Medicines Agency, Brazilian & Japanese regulatory authorities.
• Price Action: ALNY shares are up 0.16% at $138.53 during the premarket session on the last check Friday.