Alnylam Pharmaceuticals develops and commercializes RNA interference (RNAi) therapeutics, with news centered on its approved medicines, TTR franchise, clinical pipeline and financial performance. Company updates commonly address AMVUTTRA/vutrisiran and ONPATTRO/patisiran for transthyretin-mediated amyloidosis, GIVLAARI/givosiran for acute hepatic porphyria, OXLUMO/lumasiran for primary hyperoxaluria type 1, and partner-commercialized RNAi medicines.
Recurring developments include quarterly product revenue reports, guidance, clinical and real-world data for ATTR-CM and other cardiovascular programs, pipeline progress across cardio-metabolic disease, neuroscience and hematology, disease-awareness collaborations, and investor conference presentations. Alnylam also reports on its Alnylam 2030 strategy and commercialization of RNAi medicines in global markets.
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.
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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.
Alnylam Pharmaceuticals announced positive interim Phase 1 study results for zilebesiran, a new treatment for hypertension. Single doses led to sustained reductions in serum angiotensinogen and blood pressure over six months. Patients receiving doses of 200 mg and above showed significant mean reductions in systolic blood pressure, with no serious adverse events reported. The drug was well tolerated, even during low-salt diets, indicating potential for extended dosing intervals. Further evaluation is planned in two Phase 2 studies, assessing its effectiveness as a monotherapy and in combination with standard treatments.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will host a virtual R&D Day on November 19, 2021, from 8:30 am to 1:15 pm ET, featuring presentations by senior leaders and guest speakers. The event will be accessible via the Company’s website, www.alnylam.com, with a replay available within 48 hours. Alnylam is a pioneering company in RNA interference (RNAi) therapeutics, focused on transforming the treatment of rare and chronic diseases.
Alnylam Pharmaceuticals has announced the start of the KARDIA-2 Phase 2 study, assessing zilebesiran, an investigational RNAi therapeutic for hypertension management. This global trial will enroll 800 adults whose blood pressure is inadequately controlled by existing medications. The primary endpoint focuses on changes in 24-hour mean systolic blood pressure after three months. The study aims to address the rising prevalence of hypertension, projected to increase by 20% by 2025, highlighting Alnylam's commitment to innovative therapies for unmet needs in hypertension.
Alnylam Pharmaceuticals announced positive results from the ILLUMINATE-C Phase 3 study of lumasiran for treating advanced primary hyperoxaluria type 1 (PH1). This RNAi therapeutic showed significant plasma oxalate reductions in patients with chronic kidney disease, achieving decreases of 33% and 42% in two patient cohorts. These results highlight its efficacy in reducing oxalate levels, essential for preventing complications like systemic oxalosis. Moreover, lumasiran had an acceptable safety profile, with mild injection site reactions being the most common adverse event.
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