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 announced the upcoming presentation of full 18-month results from the HELIOS-A Phase 3 study of vutrisiran, aimed at treating hereditary transthyretin-mediated (hATTR) amyloidosis. This presentation will occur on January 21, 2022, at the Société Francophone du Nerf Périphérique Annual Meeting in Paris. Vutrisiran is under review by multiple regulatory agencies, including the FDA and EMA, with a PDUFA action date set for April 14, 2022. The study involved 164 patients and sought to evaluate the drug's efficacy and safety.
Soleo Health has announced FDA-approved access to OXLUMO™ (lumasiran) for treating primary hyperoxaluria type 1 (PH1) in patients as young as three months. This marks a significant advancement in specialty pharmacy services for an ultra-rare genetic disorder characterized by excessive oxalate production, leading to kidney issues. Soleo Health will administer OXLUMO injections in its Ambulatory Infusion Centers, supported by trained clinical teams. The initiative aligns with Soleo's commitment to innovative pharmacy services and patient-centered care.
Alnylam Pharmaceuticals reported preliminary global net product revenues of $662 million for 2021, marking an 83% growth compared to 2020. The company also maintained a strong balance sheet with approximately $2.4 billion in cash and investments. Key revenue contributions came from ONPATTRO, GIVLAARI, and OXLUMO, with ONPATTRO alone generating around $475 million for the full year. Alnylam anticipates significant improvement in its non-GAAP operating loss for 2021 and highlighted its successful launch of Leqvio in partnership with Novartis.
Alnylam Pharmaceuticals has entered a collaboration with Novartis to develop a siRNA-based therapy aimed at restoring functional liver cells for patients with end-stage liver disease (ESLD). This partnership seeks to create a non-invasive alternative to liver transplantation. A major challenge in ESLD is the limited availability of donor organs, impacting over one million deaths annually. The three-year agreement allows Alnylam to leverage Novartis' proprietary assays to identify therapeutic candidates, with further development transitioning to Novartis.
Alnylam Pharmaceuticals has appointed Akshay Vaishnaw, M.D., Ph.D., as President, enhancing its commitment to scientific leadership and sustainable innovation. Dr. Vaishnaw has been with the company since 2006, previously serving as President of R&D. His promotion reflects Alnylam's strategy to advance RNAi therapeutics, which are poised to transform treatment options for various diseases. CEO Yvonne Greenstreet emphasized the importance of this leadership change in driving long-term value through innovative medicines.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present a company overview at the 40th Annual J.P. Morgan Healthcare Conference on January 10, 2022, at 9:45 am ET. The presentation will include updates on unaudited fourth-quarter and full-year 2021 global net product revenues. A Q&A breakout session will follow at 10:05 am ET. The event will be available via live audio webcast on the company’s website, with a replay accessible within 48 hours. Alnylam is a leader in RNA interference therapeutics, focusing on rare genetic diseases and innovative treatment options.
Alnylam Pharmaceuticals announced the FDA's approval of Leqvio (inclisiran), the fourth RNAi therapeutic approved in the U.S., targeting LDL-C reduction in patients with atherosclerotic cardiovascular disease (ASCVD) and heterozygous familial hypercholesterolemia (HeFH). This therapy is the first RNAi treatment for high LDL-C, potentially benefiting up to 30 million Americans. The approval allows Alnylam to earn royalties on global sales by its partner Novartis, who holds commercialization rights.
Alnylam Pharmaceuticals has submitted a Clinical Trial Authorization (CTA) to the UK's MHRA to begin a Phase 1 study of ALN-APP, an RNAi therapeutic targeting amyloid precursor protein for Alzheimer's disease. The trial is set to start in early 2022, pending approval, with initial human data expected by year-end 2022. ALN-APP is notable as the first RNAi therapeutic for CNS diseases, developed in collaboration with Regeneron. This initiative underscores Alnylam's commitment to addressing significant unmet medical needs in neurodegenerative disorders.
Alnylam Pharmaceuticals has released its third annual Patient Access Philosophy Report, detailing progress towards improving access to RNAi therapeutics globally. The report highlights the company's commitment to ensuring affordability and accessibility for over 2,250 patients managing rare diseases. Notable achievements include expansion into 45 markets, high coverage rates for therapies, and numerous partnerships to enhance care accessibility. The report also underscores the impact of the ongoing pandemic on patient access initiatives.
Alnylam Pharmaceuticals has launched a global Phase 2 study to assess the safety and efficacy of lumasiran in treating patients with recurrent kidney stone disease linked to elevated urinary oxalate levels. This study is randomized, double-blind, and placebo-controlled, aiming to enroll 120 adults. Lumasiran, already FDA and EMA approved for primary hyperoxaluria type 1 (PH1), targets the HAO1 gene to reduce urinary oxalate. The primary endpoint focuses on the change in urinary oxalate after six months of treatment.