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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) commenced its virtual R&D Day, highlighting its progress in commercial and R&D sectors. The company aims to file one new NDA in 2021 and report results from two Phase 3 studies, aiming for a self-sustainable financial profile for continued growth. With four marketed products and 12 clinical programs, Alnylam underlines its commitment to RNAi therapeutics, including updates on ONPATTRO, GIVLAARI, and its pipeline's impact on diseases like hATTR amyloidosis and hypertension.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will host a virtual R&D Day on December 15-16, 2020, from 9:00 am to 12:15 pm ET each day. The event aims to showcase presentations from Alnylam senior leaders and guest speakers, including renowned experts in cardiomyopathy and amyloidosis. Replays will be available within 48 hours on their website. As a leader in RNAi therapeutics, Alnylam is committed to developing innovative medicines for various severe diseases and operates under the 'Alnylam 2020' strategy, focusing on a robust pipeline and commercial-stage products.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has published its second annual Patient Access Philosophy Report and its inaugural Rare Disease Trend Report. The company highlights its achievement of over 98% coverage for its treatments ONPATTRO and GIVLAARI across U.S. insurers, ensuring minimal out-of-pocket costs for patients. Alnylam has implemented nearly 30 value-based agreements with payers, and the Rare Disease Trend Report reveals payer concerns regarding rising costs and the need for innovative payment models.
PANTHERx Rare Pharmacy has been selected by Alnylam Pharmaceuticals as a limited distribution pharmacy partner for OXLUMO™ (lumasiran), aimed at treating primary hyperoxaluria type 1 (PH1). Approved by the FDA, OXLUMO significantly reduces urinary oxalate levels, showing a mean reduction of 53.5% in the pivotal trial compared to placebo. This represents a breakthrough for PH1 patients, providing a pharmacologic treatment option previously unavailable. The partnership aims to enhance patient access to this innovative therapy.
On December 3, 2020, Orsini Specialty Pharmacy announced its selection by Alnylam Pharmaceuticals as a limited distribution partner for OXLUMO™ (lumasiran), the first approved treatment for primary hyperoxaluria type 1 (PH1). This ultra-rare genetic disease affects 1-3 individuals per million in the U.S. and Europe. OXLUMO is an RNAi therapeutic designed to lower urinary oxalate levels. It showcases Alnylam's commitment to innovative medicines and technology, enhancing the treatment landscape for rare diseases.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced the completion of patient enrollment in the ILLUMINATE-C Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1), for treating advanced primary hyperoxaluria type 1 (PH1). The trial enrolled 21 patients across 13 sites in 10 countries. This milestone is significant for evaluating lumasiran's efficacy in severe PH1 cases, particularly those on dialysis. Lumasiran is already approved in the U.S. and EU under the brand OXLUMO™.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present an overview at the 43rd Nasdaq Virtual Investor Conference on December 4, 2020, at 11:00 am ET. Investors can access the live audio webcast on the company’s website, with a replay available within 48 hours. Alnylam is a pioneer in RNA interference (RNAi) therapeutics, developing innovative medicines for severe diseases. Their commercial products include ONPATTRO®, GIVLAARI®, and OXLUMO™, along with a robust pipeline of investigational medicines aimed at addressing unmet patient needs.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has unveiled a new framework for value-based agreements (VBAs) to ensure access to OXLUMO™ (lumasiran) for patients with primary hyperoxaluria type 1 (PH1). Approved by the FDA, OXLUMO is the first targeted therapy for PH1, aimed at reducing urinary oxalate levels. Alnylam has engaged with key payers, including Express Scripts and Highmark, to establish VBAs that enhance budget predictability and align pricing with value. Approximately 1,000-1,700 individuals with PH1 in the U.S. are potential candidates for treatment, emphasizing the need for innovative access solutions.
Alnylam Pharmaceuticals announced FDA approval of OXLUMO™ (lumasiran) for treating primary hyperoxaluria type 1 (PH1). This injectable therapy is the first to target urinary oxalate levels in both pediatric and adult patients. In the pivotal ILLUMINATE-A study, OXLUMO achieved a 65% reduction in urinary oxalate versus 12% with placebo, demonstrating significant efficacy. The drug also showed a favorable safety profile, with injection site reactions being the most common side effect. OXLUMO is set for distribution by year-end and has received multiple designations, including Breakthrough Therapy.
Alnylam Pharmaceuticals (ALNY) has received marketing authorization from the European Commission for OXLUMO™ (lumasiran), an RNAi therapeutic aimed at treating primary hyperoxaluria type 1 (PH1), an ultra-rare disease. This approval fills a significant gap as there were previously no approved treatment options for PH1 in Europe. Clinical studies, ILLUMINATE-A and ILLUMINATE-B, showed lumasiran significantly reduced urinary oxalate levels. Alnylam intends to work with health authorities across Europe to ensure patient access while minimizing budget uncertainty for health services.