Analyst Note| Karen Andersen, CFA |
Alnylam Pharmaceuticals announced positive top-line results from the Helios-A phase 3 study of vutrisiran for treatment of transthyretin-mediated (ATTR) amyloidosis with polyneuropathy and indicated its intent to submit a new drug application with the U.S. Food and Drug Administration in the first half of 2021. Although full nine-month results and top-line 18-month results will not be read out until later this year, we think the firm is in a good position for filing, with an eventual launch in 2022. We're maintaining our fair value estimate of $150 per share, as our model already factors in additional polyneuropathy-related vutrisiran sales with our Onpattro assumptions. We are also maintaining our no-moat rating for this developing company.