Alnylam PharmaceuticalsNasdaqGS:ALNY Stock Report

Alnylam Pharmaceuticals, Inc. to Present Full 18-Month Results from the HELIOS-A Phase 3 Study of Vutrisiran at the Société Francophone du Nerf Périphérique Annual Meeting

Alnylam Pharmaceuticals, Inc. announced that the Company will present full 18-month results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis, at the Société Francophone du Nerf Périphérique (SFNP) Annual Meeting. The meeting will be held January 21-22 in Paris, France, and convenes healthcare professionals focused on the peripheral nervous system. The Company previously announced positive topline 18-month results from the HELIOS-A study in October 2021. David Adams M.D., Ph.D., Department of Neurology, Coordinator of the National Reference Center for Familial Amyloid Polyneuropathy (FAP) and Rare Neuropathies, Bicêtre Hospital, Greater Paris University Hospitals, AP-HP, will present “HELIOS-A: Study of Vutrisiran in Patients with hATTR Amyloidosis” on January 21, 2022. Vutrisiran is under review by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults. In the U.S., vutrisiran has received an action date under the Prescription Drug User Fee Act (PDUFA) of April 14, 2022. The Company received Orphan Drug Designation in Japan for transthyretin type familial amyloidosis with polyneuropathy.

Forecast to breakeven in 2024

The 19 analysts covering Alnylam Pharmaceuticals expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$434.1m in 2024. Average annual earnings growth of 62% is required to achieve expected profit on schedule.

President of Research & Development notifies of intention to sell stock

Akshay Vaishnaw intends to sell 3k shares in the next 90 days after lodging an Intent To Sell Form on the 23rd of December. If the sale is conducted around the recent share price of US$189, it would amount to US$545k. Since March 2021, Akshay's direct individual holding has decreased from 18.34k shares to 11.73k. Company insiders have collectively sold US$34m more than they bought, via options and on-market transactions in the last 12 months.

Alnylam Comments on FDA Approval of Leqvio, the First Sirna (Rnai Therapeutic) Approved to Reduce LDL-C

Alnylam Pharmaceuticals, Inc. Initiates Phase 2 Study of Lumasiran in Patients with Recurrent Kidney Stone Disease

Alnylam Pharmaceuticals, Inc. announced that the company has initiated a global Phase 2 study to evaluate the safety and efficacy of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in patients with recurrent kidney stone disease and elevated urinary oxalate levels. Lumasiran is approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients and by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups, in each region under the brand name OXLUMO. The Phase 2 trial is a randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacodynamics, and pharmacokinetics of lumasiran administered subcutaneously in patients with recurrent calcium oxalate kidney stone disease and elevated urinary oxalate levels. This global, multicenter trial is expected to enroll 120 adults with a documented diagnosis of recurrent calcium oxalate kidney stone disease, based on two or more stone events within five years prior to screening, and whose 24-hour urinary oxalate levels are greater than the upper limit of normal. Study participants will be randomized 1:1:1 to receive either placebo or lumasiran at 567 mg or 284 mg on Day 1, Month 3, and Month 9 of a 15-Month double-blind treatment period that includes a six-month primary analysis period followed by a nine-month treatment extension period. The primary endpoint of the Phase 2 study is the percent change in 24-hour urinary oxalate after six months of treatment. Key secondary endpoints include the percentage of patients who achieve a 20% or greater reduction in 24-hour urinary oxalate and percent change in urinary calcium oxalate supersaturation after six months of treatment.