Alnylam Pharmaceuticals develops and commercializes RNA interference (RNAi) therapeutics, with news centered on its approved medicines, TTR franchise, clinical pipeline and financial performance. Company updates commonly address AMVUTTRA/vutrisiran and ONPATTRO/patisiran for transthyretin-mediated amyloidosis, GIVLAARI/givosiran for acute hepatic porphyria, OXLUMO/lumasiran for primary hyperoxaluria type 1, and partner-commercialized RNAi medicines.
Recurring developments include quarterly product revenue reports, guidance, clinical and real-world data for ATTR-CM and other cardiovascular programs, pipeline progress across cardio-metabolic disease, neuroscience and hematology, disease-awareness collaborations, and investor conference presentations. Alnylam also reports on its Alnylam 2030 strategy and commercialization of RNAi medicines in global markets.
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.
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.