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 FDA approval of OXLUMO™ (lumasiran) for treating primary hyperoxaluria type 1 (PH1). This injectable therapy is the first to target urinary oxalate levels in both pediatric and adult patients. In the pivotal ILLUMINATE-A study, OXLUMO achieved a 65% reduction in urinary oxalate versus 12% with placebo, demonstrating significant efficacy. The drug also showed a favorable safety profile, with injection site reactions being the most common side effect. OXLUMO is set for distribution by year-end and has received multiple designations, including Breakthrough Therapy.
Alnylam Pharmaceuticals (ALNY) has received marketing authorization from the European Commission for OXLUMO™ (lumasiran), an RNAi therapeutic aimed at treating primary hyperoxaluria type 1 (PH1), an ultra-rare disease. This approval fills a significant gap as there were previously no approved treatment options for PH1 in Europe. Clinical studies, ILLUMINATE-A and ILLUMINATE-B, showed lumasiran significantly reduced urinary oxalate levels. Alnylam intends to work with health authorities across Europe to ensure patient access while minimizing budget uncertainty for health services.
Alnylam Pharmaceuticals (Nasdaq: ALNY) reported positive interim data from its Phase 1 study of ALN-AGT, an RNAi therapeutic targeting angiotensinogen for hypertension treatment. Presented at the AHA Scientific Sessions 2020, data from 60 patients revealed significant AGT knockdown of up to 97.6% and considerable systolic and diastolic blood pressure reductions at 200 mg doses. The treatment demonstrated a favorable safety profile, with mild, transient adverse events observed. These results suggest a potential for innovative hypertension management with infrequent dosing, addressing the unmet need for improved patient adherence.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announces virtual presentations at two upcoming conferences. The 29th Annual Credit Suisse Virtual Healthcare Conference is scheduled for November 9, 2020, at 12:30 pm ET, followed by the Stifel 2020 Virtual Healthcare Conference on November 16, 2020, at 2:00 pm ET. Investors can access a live audio webcast of each presentation on Alnylam's website, with replays available within 48 hours post-event. Alnylam is a leader in RNA interference therapeutics, focused on innovative medicines for rare genetic and other severe diseases.
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Alnylam Pharmaceuticals (Nasdaq: ALNY) will announce its third-quarter financial results for the period ending September 30, 2020, on November 5, 2020, before the U.S. markets open. The management team will host a conference call at 8:30 am ET on the same day to discuss the results and future expectations. Interested parties can access the call through a dedicated phone line or via a live audio webcast on the company's website. Alnylam focuses on RNA interference therapeutics aimed at treating various severe diseases with a robust product pipeline.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced positive 6-month results from the ILLUMINATE-B Phase 3 study of lumasiran, an RNAi therapeutic for primary hyperoxaluria type 1 (PH1). The study showed a 72% mean reduction in urinary oxalate levels among children under six. Lumasiran's safety profile was assessed as acceptable, with no severe adverse events reported. Lumasiran is also under FDA review, with a decision expected by December 3, 2020. If approved, it will be marketed as OXLUMO in the EU, following a Positive Opinion from the EMA's CHMP.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced a positive opinion from the European Medicines Agency's CHMP for lumasiran, an RNAi therapeutic targeting the HAO1 mRNA for treating primary hyperoxaluria type 1 (PH1). PH1 is a rare disease affecting 3.5 to 4 individuals per million, characterized by excessive oxalate production leading to severe kidney damage. If approved by the European Commission, lumasiran will be marketed as OXLUMO™. Clinical trials indicate significant reductions in urinary and plasma oxalate levels, demonstrating a promising safety profile.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announced positive topline results from the ILLUMINATE-B Phase 3 study of lumasiran, an investigational RNAi therapeutic for primary hyperoxaluria type 1 (PH1). This trial is significant as it is the first assessing the safety and efficacy of lumasiran in children under six, including infants. The study enrolled 18 young patients and reported a clinically meaningful reduction in urinary oxalate levels. Lumasiran has received various FDA and EMA designations and a New Drug Application is pending with the FDA, set for review by December 3, 2020.