Shots:

• The P-III HELIOS-A study assessing vutrisiran (25mg- SC- once every 3mos.) vs patisiran (0.3 mg/kg- IV- q3w) in 164 patients in a ratio (3:1) with hATTR amyloidosis with polyneuropathy
• The study met its 1EPs & 2EPs i.e. change from baseline in the mNIS+7 @9mos. & changes in QoL assessed by Norfolk QoL-DN and gait speed assessed by the timed 10-MWT respectively. Additionally- therapy showed improvements in the 9mos. exploratory cardiac endpoint of NT-proBNP
• The company plans to submit NDA to the US FDA in H1’21- following the regulatory filing in Brazil and Japan. Moreover- Alnylam intends to submit MAA in the EU on obtaining the results of 18mos. analysis- anticipated in late 2021

 ­ Ref: Businesswire | Image: Alnylam