Shots:

• The P-III (HELIOS-A) study evaluated the efficacy & safety of vutrisiran (25mg, SC, q3mos.) vs patisiran (0.3 mg/kg, IV, q3w) in a ratio (3:1) in 164 patients with hATTR amyloidosis with polyneuropathy across 57 sites in 22 countries
• The trial met all 2EPs i.e., improvement in clinical EPs & non-inferiority in serum TTR reduction over patisiran, improvement in mNIS+7 (48% vs 4%), gait speed, nutritional status, QoL (57% vs 10%) & overall disability over PBO
• The therapy also showed an improvement in exploratory cardiac EPs @18mos., including biomarker NT-proBNP, echocardiographic parameters & cardiac uptake of technetium on scintigraphy imaging along with promising safety profile

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