Alnylam Pharmaceuticals, Inc. - ALNY STOCK NEWS

Alnylam Pharmaceuticals announced the FDA's approval of Leqvio (inclisiran), the fourth RNAi therapeutic approved in the U.S., targeting LDL-C reduction in patients with atherosclerotic cardiovascular disease (ASCVD) and heterozygous familial hypercholesterolemia (HeFH). This therapy is the first RNAi treatment for high LDL-C, potentially benefiting up to 30 million Americans. The approval allows Alnylam to earn royalties on global sales by its partner Novartis, who holds commercialization rights.

Alnylam Pharmaceuticals has submitted a Clinical Trial Authorization (CTA) to the UK's MHRA to begin a Phase 1 study of ALN-APP, an RNAi therapeutic targeting amyloid precursor protein for Alzheimer's disease. The trial is set to start in early 2022, pending approval, with initial human data expected by year-end 2022. ALN-APP is notable as the first RNAi therapeutic for CNS diseases, developed in collaboration with Regeneron. This initiative underscores Alnylam's commitment to addressing significant unmet medical needs in neurodegenerative disorders.

Alnylam Pharmaceuticals has released its third annual Patient Access Philosophy Report, detailing progress towards improving access to RNAi therapeutics globally. The report highlights the company's commitment to ensuring affordability and accessibility for over 2,250 patients managing rare diseases. Notable achievements include expansion into 45 markets, high coverage rates for therapies, and numerous partnerships to enhance care accessibility. The report also underscores the impact of the ongoing pandemic on patient access initiatives.

Alnylam Pharmaceuticals has launched a global Phase 2 study to assess the safety and efficacy of lumasiran in treating patients with recurrent kidney stone disease linked to elevated urinary oxalate levels. This study is randomized, double-blind, and placebo-controlled, aiming to enroll 120 adults. Lumasiran, already FDA and EMA approved for primary hyperoxaluria type 1 (PH1), targets the HAO1 gene to reduce urinary oxalate. The primary endpoint focuses on the change in urinary oxalate after six months of treatment.

Alnylam Pharmaceuticals has announced the submission of a clinical trial authorization (CTA) to the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for its investigational RNAi therapeutic, ALN-XDH, targeting gout treatment. The Phase 1/2 study is expected to begin in early 2022, with initial human data anticipated in late 2022. ALN-XDH aims to address unmet needs in gout management by providing potent urate-lowering effects with infrequent dosing, potentially improving patient adherence and outcomes.

Alnylam Pharmaceuticals has submitted a supplemental New Drug Application (sNDA) to the FDA and a Type II Filing Variation to the EMA for lumasiran, aimed at reducing plasma oxalate in patients with advanced primary hyperoxaluria type 1 (PH1). This follows positive results from the ILLUMINATE-C Phase 3 study, which demonstrated substantial reductions in plasma oxalate levels in PH1 patients, including those on hemodialysis. Injection site reactions were the most common adverse events, with a low incidence of severe reactions reported.

Alnylam Pharmaceuticals (Nasdaq: ALNY) will present company overviews at two virtual conferences: the Evercore ISI 4th Annual HealthCONx Conference on November 30, 2021, at 9:40 am ET, and the Piper Sandler 33rd Annual Virtual Healthcare Conference on December 2, 2021, at 10:00 am ET. Live webcasts will be available on the company's website, with replays posted within 48 hours. Alnylam is a leader in RNAi therapeutics, focusing on innovative treatments for rare and common diseases. Its product lineup includes ONPATTRO®, GIVLAARI®, and OXLUMO®.

Alnylam Pharmaceuticals (NASDAQ: ALNY) is launching a new investigational RNAi therapeutic, vutrisiran, expected to enter Phase 3 development for Stargardt Disease in late 2022. The company is also adding new preclinical programs, GEMINI-CVR and Gene X, with promising potential in cardiovascular disease. Alnylam highlighted significant advancements in extrahepatic delivery of RNAi therapeutics, including new CNS and ocular programs. The company's R&D Day webcast is scheduled for 8:30 a.m. ET today, November 19.