Welcome to our dedicated page for Alnylam Pharmaceuticals news (Ticker: ALNY), a resource for investors and traders seeking the latest updates and insights on Alnylam Pharmaceuticals stock.
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 that its investigational drug vutrisiran met all secondary endpoints of the HELIOS-A Phase 3 study at 18 months. The study demonstrated statistically significant improvements in neuropathy impairment, quality of life, gait speed, nutritional status, and overall disability compared to placebo. Additionally, vutrisiran showed potential for cardiac benefits, including reduced technetium uptake, suggesting possible amyloid regression. The drug is under regulatory review globally, with a potential FDA action date of April 14, 2022.
Ceptur Therapeutics, a biotechnology company focused on U1 Adaptor technology, has successfully completed a $75M Series A financing round. The round was co-led by venBio Partners and Qiming Venture Partners USA, with participation from various investors including Bristol Myers Squibb and Janus Henderson Investors. The funds will be used to advance their genetic medicine pipeline. Ceptur's U1 Adaptors aim to regulate gene expression at the pre-mRNA level, offering innovative therapeutic applications for genetic diseases.
Alnylam Pharmaceuticals has partnered with n-Lorem Foundation to support nano-rare disease patients through innovative therapies. The collaboration aims to utilize Alnylam's RNA interference technology alongside n-Lorem's experimental antisense oligonucleotide medicines. This partnership is designed to identify treatment opportunities for rare diseases affecting 1 to 30 patients globally, addressing significant healthcare challenges.
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.