Alnylam Announces 2020 Product and Pipeline Goals and Provides Updates at R&D Day

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, is hosting an R&D Day in New York City today. During the event, the Company plans to discuss its commercial and R&D progress, including its product and pipeline goals for 2020, focusing on the potential for global approval and/or commercialization of four RNAi therapeutic products and the advancement of six late-stage investigational programs.

Alnylam also announces today that it has initiated HELIOS-B, a global Phase 3 placebo-controlled mortality and cardiovascular hospitalization trial with investigational vutrisiran in patients with ATTR amyloidosis with cardiomyopathy. In addition, the Company is reporting initial positive clinical data with ALN-AAT02, ALN-HBV02 (VIR-2218), and ALN-AGT, representing the first human proof of concept for Alnylam’s ESC+ GalNAc conjugate delivery technology – having the potential to enable greater target selectivity and a wider therapeutic index – and expansion of the Alnylam pipeline in highly prevalent chronic disease opportunities. The Company is also reporting progress in its efforts with CNS and ocular delivery of RNAi therapeutics, including an update on the status of its collaborative work with Regeneron, and announces a new investigational CNS program, ALN-HTT, in development for the treatment of Huntington’s disease. Finally, Alnylam is presenting its general framework for progressing toward a self-sustainable financial profile for future growth and value creation.

“At Alnylam, we believe that we’re at an exciting stage of growth, with a large number of commercial and development-stage opportunities with potentially transformative impact for patients across both rare and common diseases. Over the course of 2020 we expect to deliver on a Company profile with four medicines on the market, assuming additional regulatory approvals, while also advancing a large number of late- and earlier-stage programs, all while focusing on our pathway toward a self-sustainable financial profile. Our plans include continued global commercialization of ONPATTRO as a market-leading program, with ongoing clinical studies aimed at broadening our ATTR amyloidosis franchise to potentially address the full spectrum of disease, including the now initiated HELIOS-B study with investigational vutrisiran that we’re announcing today ,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “With the FDA approval just this week for GIVLAARI and through our efforts and those of our partners, we now believe that we’re poised to exceed our Alnylam 2020 goals of building a multi-product, global, commercial-stage biopharma company with a deep and sustainable clinical pipeline for future growth and a robust, organic research engine for sustainable innovation and great potential for patient impact.”