Alnylam (ALNY) Soars as Heart Disease Drug Study Meets Goals

Core Viewpoint - Alnylam Pharmaceuticals achieved a new 52-week high following positive results from the phase III HELIOS-B study of Amvuttra (vutrisiran), leading to a 34.5% increase in stock price [1]. Group 1: Study Results - The HELIOS-B study met its primary endpoint, showing a statistically significant reduction in all-cause mortality and recurrent cardiovascular events in both the overall and monotherapy populations [2]. - Statistically significant improvements were observed across all secondary endpoints, including key measures of disease progression at Month 30 [2]. - Vutrisiran reduced all-cause mortality in both populations up to Month 42, based on a pre-specified intent-to-treat analysis [2]. Group 2: Patient Population and Safety - The study enrolled 655 adult patients with ATTR amyloidosis and cardiomyopathy, who received either vutrisiran or placebo [3]. - Vutrisiran demonstrated a favorable safety and tolerability profile, with adverse event rates similar to the placebo group [4]. Group 3: Market Potential and Financial Performance - Alnylam plans to submit global regulatory applications starting in late 2024, aiming for vutrisiran to become the new standard of care for ATTR amyloidosis with cardiomyopathy [4]. - Amvuttra generated $195 million in sales in Q1, reflecting a 92% increase, driven by new patient uptake and switches from Onpattro [5].