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) achieved full enrollment in its HELIOS-B Phase 3 study for vutrisiran, an RNAi therapeutic aimed at treating cardiomyopathy in patients with ATTR amyloidosis. Over 600 patients were enrolled across 123 sites in 32 countries, ahead of schedule. The study will assess vutrisiran's efficacy against placebo, focusing on all-cause mortality and cardiovascular events over 30 months. Topline results are anticipated in early 2024, with potential interim analyses linked to the APOLLO-B study. This milestone reflects strong interest in innovative treatment options.
Alnylam Pharmaceuticals (ALNY) reported strong second-quarter results for 2021, with total net product revenues of $160.8 million, a 107% increase year-over-year. ONPATTRO, GIVLAARI, and OXLUMO all showed significant growth, with ONPATTRO revenues rising to $114 million, GIVLAARI to $31 million, and OXLUMO achieving $16 million. The company received marketing approvals for GIVLAARI in Japan and OXLUMO in Brazil. Alnylam also raised its revenue guidance for the year to between $640 million and $665 million. Despite a GAAP net loss of $189.6 million, positive developments in their clinical pipeline and collaborations with Regeneron and Novartis are promising.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has entered into a collaboration with PeptiDream to develop peptide-siRNA conjugates for RNAi therapeutics targeting various tissues outside the liver. Alnylam will select receptors for PeptiDream's platform, which will optimize peptides for targeted delivery. This partnership aims to leverage PeptiDream's expertise in peptide ligands to expand treatment opportunities for RNAi therapeutics. The agreement includes an upfront payment and potential milestone payments totaling up to $2.2 billion, along with royalties on future product sales.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced positive topline results from the ILLUMINATE-C Phase 3 study of lumasiran for advanced primary hyperoxaluria type 1 (PH1). The trial showed significant decreases in plasma oxalate levels after six months in both hemodialysis-dependent and independent patients. The safety profile was encouraging, with mild injection site reactions as the most common adverse event. Alnylam plans to submit a Supplemental New Drug Application to the FDA and EMA based on these results, further solidifying lumasiran's therapeutic potential in treating PH1.
Alnylam Pharmaceuticals (Nasdaq: ALNY) plans to report its second quarter financial results for the period ending June 30, 2021, on August 3, 2021, prior to market opening. A conference call will be held at 8:30 am ET on the same day to discuss the results and future expectations. Alnylam is recognized for its pioneering work in RNA interference therapeutics, which have the potential to treat various rare and severe diseases. The company is advancing its research and commercial efforts with innovative products and a robust pipeline.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has initiated the KARDIA-1 Phase 2 study to evaluate the safety and efficacy of zilebesiran, an RNAi therapeutic for treating hypertension. The trial will assess blood pressure changes in approximately 375 participants over 12 months. Key endpoints include systolic blood pressure reduction after three months. This study follows promising Phase 1 results presented earlier this year. The global trial will be conducted in the U.S. and Europe and aims to address the high unmet need for effective hypertension management.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced that the FDA has accepted its New Drug Application for vutrisiran, an investigational RNAi therapeutic targeting hereditary transthyretin-mediated (hATTR) amyloidosis. The FDA has set an action date of April 14, 2022, and will not hold an advisory committee meeting for this review. Vutrisiran has received Orphan Drug and Fast-Track designations. The HELIOS-A Phase 3 study evaluates vutrisiran's efficacy, with promising results expected to provide a new treatment option for hATTR patients.
Alnylam Pharmaceuticals (Nasdaq: ALNY) is set to host its 8th annual series of RNAi Roundtable webinars featuring presentations from its scientists and medical thought leaders. The webinars will review progress in its pipeline programs and the advancements in RNAi therapeutics. Events are scheduled from June 30 to October 1, highlighting topics like ALN-AGT for hypertension and Givosiran for acute hepatic porphyria. Each event will be live-streamed, with a replay available on the company’s website within three hours post-event.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced positive findings from a Phase 3b study on patisiran, showing a 91% median reduction in serum TTR levels in hereditary ATTR amyloidosis patients post-liver transplant. The study, involving 23 patients, demonstrated improvements in neuropathy and quality of life over 12 months. Additionally, results from the HELIOS-A Phase 3 study of vutrisiran indicated consistent improvements across multiple patient subgroups, highlighting its potential as a new treatment. These findings may lead to important label changes for ONPATTRO in various markets.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has completed patient enrollment in the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic targeting ATTR amyloidosis. Over 300 patients participated across 90 sites in more than 20 countries. The study aims to evaluate the efficacy and safety of patisiran for treating cardiomyopathy in ATTR amyloidosis patients. Notably, the primary outcome measures will include changes in the 6-minute walk test compared to placebo. Topline results are anticipated in mid-2022, marking crucial progress for both the treatment and the company's pipeline.