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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 announced that the HELIOS-A Phase 3 study of vutrisiran met all 18-month secondary endpoints. Significant improvements were observed in neuropathy, quality of life, gait speed, nutritional status, and overall disability compared to external placebo data. Additionally, vutrisiran showed positive exploratory endpoint results, including improved NT-proBNP levels and technetium uptake, indicating reduced cardiac amyloid burden. The drug has an encouraging safety profile, with minimal discontinuations due to adverse events. Full results are expected to be presented at an upcoming medical conference.
Alnylam Pharmaceuticals (NASDAQ: ALNY) will announce its third-quarter financial results for 2021 on October 28, 2021, before U.S. markets open. The company will host a conference call at 8:30 am ET to discuss results and future expectations. Investors can access the live call by dialing 877-312-7507 (domestic) or +1-631-813-4828 (international), with a replay available later in the day. Alnylam develops innovative RNA interference (RNAi) therapeutics aimed at addressing severe diseases, with a commitment to delivering transformative medicines through its ongoing pipeline.
Alnylam Pharmaceuticals (NASDAQ: ALNY) has launched the Alnylam Challengers program, committing $1 million to Acumen America to enhance health equity initiatives. This partnership aims to assist 75 million individuals in the U.S. by 2024 by tackling health disparities due to poverty and discrimination. The program includes a Social Investment Portfolio, an Advisory Corps for expert volunteering, and cross-sector Convenings to address health equity issues. Alnylam emphasizes its corporate responsibility and investment in sustainable health solutions.
Alnylam Pharmaceuticals announced promising pre-clinical research at the 17th Annual Meeting of the Oligonucleotide Therapeutics Society, showcasing innovations in RNAi therapeutics. Their hexadecyl (C16) conjugate demonstrates over 90% target knockdown potential in the CNS with an annual dosing regimen. The presentation also highlighted the IKARIA platform, which facilitates effective siRNA delivery. Additionally, pre-clinical data on ALN-APP for early-onset Alzheimer's Disease showed promising results, with plans for a clinical trial application later this year.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present a company overview at the Chardan Virtual 5th Annual Genetic Medicines Conference on Tuesday, October 5, 2021, at 2:30 pm ET. The presentation will be accessible via a live audio webcast on the Investors section of Alnylam's website, with a replay available within 48 hours post-event. Alnylam is a leader in RNA interference therapeutics, developing innovative treatments for rare diseases and more, including commercial products like ONPATTRO®, GIVLAARI®, and OXLUMO®. The company aims to transform medicine through its robust RNAi platform.
Alnylam Pharmaceuticals has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for vutrisiran, aimed at treating hereditary transthyretin-mediated (hATTR) amyloidosis in adults. This investigational RNAi therapeutic shows promise, having met primary and secondary endpoints in the HELIOS-A Phase 3 study, including improvements in neuropathy and quality of life at 9 months. Vutrisiran has Orphan Drug Designation in the EU and U.S. and a New Drug Application is under review by the FDA, set for action on April 14, 2022.
Alnylam Pharmaceuticals announced encouraging results from its HELIOS-A Phase 3 study of vutrisiran, targeting hereditary transthyretin-mediated (ATTR) amyloidosis. The study demonstrated statistically significant improvements in patient health outcomes, including neuropathy impairment and quality of life, after 9 months. Notably, improvements were consistent regardless of prior TTR stabilizer use. The company aims to release 18-month results later this year, with the FDA accepting its New Drug Application for vutrisiran, and an EU submission planned ahead of schedule.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present company overviews at several virtual conferences in September 2021. Key presentation dates include the Morgan Stanley 19th Annual Global Healthcare Conference on September 9 at 3:30 PM ET, H.C. Wainwright's 23rd Annual Global Investment Conference on September 13 at 7:00 AM ET, BofA Global Healthcare Conference on September 16 at 10:05 AM ET, and Cantor Virtual Global Healthcare Conference on September 28 at 10:40 AM ET. Live webcasts will be available on the company's website, with replays accessible within 48 hours.
Alnylam Pharmaceuticals (Nasdaq: ALNY) achieved full enrollment in its HELIOS-B Phase 3 study for vutrisiran, an RNAi therapeutic aimed at treating cardiomyopathy in patients with ATTR amyloidosis. Over 600 patients were enrolled across 123 sites in 32 countries, ahead of schedule. The study will assess vutrisiran's efficacy against placebo, focusing on all-cause mortality and cardiovascular events over 30 months. Topline results are anticipated in early 2024, with potential interim analyses linked to the APOLLO-B study. This milestone reflects strong interest in innovative treatment options.
Alnylam Pharmaceuticals (ALNY) reported strong second-quarter results for 2021, with total net product revenues of $160.8 million, a 107% increase year-over-year. ONPATTRO, GIVLAARI, and OXLUMO all showed significant growth, with ONPATTRO revenues rising to $114 million, GIVLAARI to $31 million, and OXLUMO achieving $16 million. The company received marketing approvals for GIVLAARI in Japan and OXLUMO in Brazil. Alnylam also raised its revenue guidance for the year to between $640 million and $665 million. Despite a GAAP net loss of $189.6 million, positive developments in their clinical pipeline and collaborations with Regeneron and Novartis are promising.