ALNY Down Despite Positive Data From Heart Disease Drug Study

Core Viewpoint - Alnylam Pharmaceuticals' shares fell 8.5% following the announcement of detailed data from the phase III HELIOS-B study for its investigational RNAi therapeutic, Amvuttra, aimed at treating transthyretin-mediated amyloidosis with cardiomyopathy [1][2]. 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 [1]. - Treatment with vutrisiran resulted in a 30% reduction in mortality in the monotherapy population during the double-blind period, but this reduction was deemed non-significant by management [1]. Group 2: Market Performance - Alnylam's shares have increased by 37.3% year-to-date, significantly outperforming the industry growth of 0.6% [4]. - The company reported sales of $425.4 million for Amvuttra in the first half of 2024, reflecting an 82% year-over-year increase [6]. Group 3: Regulatory and Future Prospects - Alnylam plans to submit a supplemental new drug application for vutrisiran to the FDA using a Priority Review Voucher later in 2024 [5]. - If approved for the new indication, vutrisiran is expected to become the new standard of care for treating ATTR amyloidosis with cardiomyopathy, potentially increasing sales and expanding the patient population [6].