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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) reported strong financial results for Q4 and full year 2020, showcasing significant growth in product revenues amid regulatory approvals. ONPATTRO achieved $306 million in annual revenue, marking over 80% growth from 2019, while GIVLAARI brought in $55 million in its launch year. The company expects combined 2021 product revenues between $610 million and $660 million, indicating over 75% growth from 2020. Key developments included positive Phase 3 results for vutrisiran and the launch of OXLUMO. Alnylam anticipates continued expansion and product launches in 2021.
Alnylam Pharmaceuticals (Nasdaq: ALNY) is set to announce financial results for Q4 and the full year ending December 31, 2020, on February 11, 2021. The conference call will occur at 8:30 am ET, discussing performance and future expectations. Investors can access the live audio webcast through their website. Alnylam, founded in 2002, focuses on RNA interference therapeutics, including products like ONPATTRO, GIVLAARI, and OXLUMO. The company’s innovative pipeline aims to address rare and common diseases, underpinned by a robust financial outlook and strategic growth initiatives.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced its 5-year strategy named Alnylam P5x25, aiming to be among the top 5 biotech firms by market capitalization. Key goals include reaching over 0.5 million patients on its RNAi therapeutics, marketing at least 6 products, and achieving ≥40% revenue CAGR through 2025. Preliminary Q4 2020 revenues for ONPATTRO and GIVLAARI were approximately $90 million and $22 million, respectively, marking significant growth and strong demand for its therapies.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced positive results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic for hATTR amyloidosis. The study met primary and secondary endpoints, showing significant improvements in neurologic impairment and quality of life at 9 months, with a favorable safety profile. The company plans to submit a New Drug Application (NDA) to the FDA in early 2021 and expects additional regulatory filings globally. Vutrisiran's promising results position it as a potential treatment for this serious condition.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present at the 39th Annual J.P. Morgan Healthcare Conference on January 11, 2021, at 8:20 am ET, discussing unaudited fourth quarter and full year 2020 revenues. A Q&A breakout session will follow at 8:40 am ET. Investors can access a live audio webcast on the company's website, with a replay available within 48 hours. Alnylam is a leader in RNA interference therapeutics, working to develop innovative treatments for rare diseases.
Alnylam Pharmaceuticals (Nasdaq: ALNY) appointed Tolga Tanguler as Chief Commercial Officer, effective January 5, 2021. Tanguler brings over 20 years of experience from Pfizer and Alexion, where he significantly drove product launches and revenue growth. Alongside Tanguler, Alnylam expanded its leadership team with Kasha Witkos, Salil Patel, and Agnieszka Gallagher, enhancing its commercial and medical expertise. This strategic move aims to support global product launches and advance Alnylam's robust pipeline while striving for profitability.
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.