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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) revealed plans for management to present at two upcoming virtual conferences. The events are the BofA Health Care Conference on May 11, 2021, at 2:45 pm ET and the UBS Global Healthcare Virtual Conference on May 25, 2021, at 3:00 pm ET. Investors can access the live audio webcast on the company's website, with a replay available within 48 hours post-event. Alnylam is at the forefront of RNA interference therapeutics, focusing on innovative medicines for rare genetic and other diseases with a diverse pipeline of investigational products.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced promising early results from the 12-month analysis of the ILLUMINATE-A Phase 3 study of OXLUMO (lumasiran), targeting primary hyperoxaluria type 1 (PH1). The data, presented at the ASPN/PAS virtual meeting, revealed significant reductions in urinary oxalate levels among patients, with notable improvements in nephrocalcinosis observed in 46% of those treated for 12 months. OXLUMO's safety profile remains acceptable, with injection site reactions being the most common adverse effect. Alnylam anticipates reporting further data later this year.
Alnylam Pharmaceuticals reported strong first-quarter 2021 results, with total net product revenues of $135.8 million, marking an 89% increase year-over-year. ONPATTRO drove growth with revenues of $102 million, up 13% quarter-over-quarter. OXLUMO's initial launch generated $9 million in revenues. The company filed an NDA for vutrisiran, with positive HELIOS-A Phase 3 results and accelerated enrollment in HELIOS-B expected to complete by late 2021. Alnylam aims to transition into a top five biotech by 2025 with its P5x25 strategy while maintaining a strong cash position of $1.71 billion.
Alnylam Pharmaceuticals announced successful outcomes from the HELIOS-A Phase 3 study of vutrisiran, targeting transthyretin-mediated amyloidosis. The investigational RNAi therapeutic met primary and secondary endpoints at nine months, showing a significant 83% mean reduction in serum TTR levels and improvements in the Neuropathy Impairment Score and quality of life measures. A New Drug Application has been submitted to the FDA for vutrisiran's approval. The treatment demonstrated a favorable safety profile, with no drug-related discontinuations or deaths reported.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will release its first-quarter 2021 financial results on April 29, 2021, before market opens. The company's management will hold a conference call at 8:30 am ET to discuss these results and future expectations. Interested parties can access the call via phone or through a live audio webcast on Alnylam's investor website. Alnylam aims to leverage its RNA interference (RNAi) platform to address various diseases, showcasing a strong pipeline and ongoing commitment to innovation in the biotech sector.
Alnylam Pharmaceuticals (Nasdaq: ALNY) reported positive interim results from its Phase 1 study of ALN-AGT, a novel RNAi therapeutic for hypertension. The study showed significant dose-dependent reductions in serum angiotensinogen (AGT) and blood pressure, with a maximum 17 mm Hg reduction in 24-hour systolic blood pressure at the 800 mg dose. The treatment was well tolerated, with most adverse events being mild. The company plans to initiate Phase 2 KARDIA studies in mid-2021, suggesting ALN-AGT may transform hypertension management.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present an overview at the 20th Annual Needham Virtual Healthcare Conference on April 13, 2021, at 1:30 pm ET. A live audio webcast will be accessible via the Investors section of the Company's website, with a replay available within 48 hours post-event. Alnylam is a leader in RNA interference (RNAi) therapeutics, aiming to transform treatments for rare genetic, cardio-metabolic, hepatic infectious, and CNS diseases. Their portfolio includes commercially available products like ONPATTRO, GIVLAARI, OXLUMO, and Leqvio.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced pivotal results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic for primary hyperoxaluria type 1 (PH1). Published in NEJM, the study showed a significant 53.5% reduction in urinary oxalate levels compared to placebo at six months. Notably, 84% of patients on lumasiran reached normal or near-normal oxalate levels. The treatment had a favorable safety profile with no severe adverse events reported. OXLUMO is the first approved therapy for PH1, addressing a critical unmet medical need for patients.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present a company overview at the Stifel 3rd Annual CNS Day on April 1, 2021, at 11:00 am ET. A live audio webcast will be accessible via the Investors section of Alnylam's website, with a replay available within 48 hours. Alnylam is a leader in RNA interference therapeutics, focusing on innovative medicines for rare genetic and CNS diseases. They have launched products like ONPATTRO and are advancing multiple investigational medicines under the Alnylam P5x25 strategy.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced the upcoming presentation of full 9-month results from the HELIOS-A Phase 3 study of vutrisiran at the AAN Virtual Annual Meeting, scheduled for April 17-22. Vutrisiran is an investigational RNAi therapeutic for hereditary ATTR amyloidosis. This milestone follows the positive topline results previously reported in January. The company aims to submit a U.S. regulatory filing for vutrisiran in early 2021. The presentation will also feature updates from ongoing studies on ONPATTRO and GIVLAARI.