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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) is at the forefront of RNA interference (RNAi) therapeutics, pioneering a transformative class of medicines aimed at treating and potentially curing a wide range of diseases. Founded in 2002 and based in Cambridge, MA, Alnylam has translated Nobel Prize-winning science into five commercial products: ONPATTRO (patisiran), AMVUTTRA (vutrisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), and Leqvio (inclisiran), developed and commercialized in partnership with Novartis. These breakthrough therapeutics address conditions such as hATTR amyloidosis, acute hepatic porphyria, primary hyperoxaluria type 1, and hypercholesterolemia.
Alnylam is dedicated to expanding its pipeline with several investigational medicines in late-stage development, focusing on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and CNS/ocular diseases. The company’s commitment to sustainable innovation is reflected in its robust product pipeline and strategic collaborations with industry leaders like Roche and Regeneron.
Financially, Alnylam reported robust growth in 2023 with $1.24 billion in global net product revenues, a reflection of its strong commercial execution across its therapeutic products. The company maintains a strong cash position, bolstered by upfront fees from research partnerships and milestone achievements.
Alnylam is also dedicated to corporate responsibility, with initiatives aimed at reducing greenhouse gas emissions, promoting diversity and inclusion in clinical trials, and expanding global health equity through its Alnylam Challengers program. The company’s environmental impact data is third-party verified, underscoring its commitment to transparency and sustainability.
Looking ahead, Alnylam aims to sustain its leadership in RNAi therapeutics with the anticipated release of topline results from the HELIOS-B Phase 3 study and the continued development of promising candidates like zilebesiran for hypertension and mivelsiran for Alzheimer’s disease. For more details on Alnylam’s people, science, and pipeline, visit www.alnylam.com.
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.
Alnylam Pharmaceuticals reported a significant Q3 2021 revenue of $167 million for its products ONPATTRO, GIVLAARI, and OXLUMO. The company highlighted promising 18-month results from the HELIOS-A Phase 3 study of vutrisiran in patients with hATTR amyloidosis. Global net product revenues for ONPATTRO rose 6% to $120 million, while GIVLAARI grew 4% to $32 million. However, OXLUMO saw a 9% decline in revenue. Alnylam reaffirmed its 2021 guidance of $640-$665 million in revenues and announced a leadership transition from CEO John Maraganore to Yvonne Greenstreet.
Alnylam Pharmaceuticals announces a leadership transition with CEO John Maraganore passing the reins to Yvonne Greenstreet by year-end 2021. Maraganore, a founding figure since 2002, has led the development of RNAi therapeutics, contributing to the approval of four medicines in 25 countries. Greenstreet, currently President and COO, brings significant experience and will step into her new role while Maraganore continues as a consultant until March 2022. The Board expressed strong confidence in Greenstreet's leadership to advance Alnylam’s P5x25 strategy.