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Alnylam Announces Publication of ILLUMINATE-A Phase 3 Study Results for Lumasiran in The New England Journal of Medicine
- ILLUMINATE-A Phase 3 Study Evaluated the Efficacy and Safety of Lumasiran in Adult and Pediatric Patients with Primary Hyperoxaluria Type 1 (PH1) - -
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n- ILLUMINATE-A Phase 3 Study Evaluated the Efficacy and Safety of Lumasiran in Adult and Pediatric Patients with Primary Hyperoxaluria Type 1 (PH1) -\n\n- Lumasiran Demonstrated a Clinically Significant Reduction in Urinary Oxalate, a Primary Determinant of Progression to Renal Failure in PH1, Compared to Placebo -\n\n- Manuscript Marks the Tenth Publication of Clinical Trial Results for Alnylam Programs in The New England Journal of Medicine, Highlighting the Impact of RNAi Therapeutics as a New Class of Medicines -\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were published online in The New England Journal of Medicine (NEJM). In November 2020, OXLUMO™ (lumasiran) was approved by the U.S. Food and Drug Administration for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients, and received marketing authorization from the European Commission for the treatment of PH1 in all age groups. OXLUMO is the first-ever treatment approved for PH1 and the first RNAi therapeutic evaluated in both children and adults. The full manuscript titled “Phase 3 Trial of Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1,” will appear in the April 1, 2021 issue of NEJM.\n\nThe data reported in the ILLUMINATE-A Phase 3 study publication demonstrated that RNAi-mediated targeting of liver GO by lumasiran led to substantial and sustained reductions in urinary oxalate—the toxic metabolite responsible for the debilitating and life-threatening clinical manifestations of PH1. Relative to placebo, treatment with lumasiran resulted in a clinically significant (53.5 percent) reduction in 24-hour urinary oxalate excretion from baseline to month 6 – the primary endpoint of the study.\n\nImprovements were also observed in a number of secondary endpoints, including the proportion of patients achieving normala or near-normalb levels of urinary oxalate, with 84 percent of lumasiran-treated patients meeting this endpoint compared with no patients (0 percent) on placebo. Patients treated with lum...