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Alnylam Presents Positive Phase 3 Results from ILLUMINATE-A Study of Lumasiran, an Investigational RNAi Therapeutic for Treatment of Primary Hyperoxaluria Type 1
- Lumasiran Achieved Primary Endpoint with 53.5 Percent Mean Reduction in Urinary Oxalate Relative to Placebo and Showed a 65.4 Percent Reduction Relative to
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Lumasiran Achieved Primary Endpoint with 53.5 Percent Mean Reduction in Urinary Oxalate Relative to Placebo and Showed a 65.4 Percent Reduction Relative to Baseline -\n\n\n- 84 Percent of Patients on Lumasiran Achieved Normal or Near-Normal Levels of Urinary Oxalate and More than Half of Patients Reached Normalization, Compared with Zero Percent in the Placebo Group -\n\n\n- Lumasiran Demonstrated an Encouraging Safety and Tolerability Profile -\n\n\n- Alnylam to Host Conference Call Today at 8:30 am ET -\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive Phase 3 results from the ILLUMINATE-A study of lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1). The clinical data were presented at a late-breaking session at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress being held as a virtual event on June 6-9.\n\n\nLumasiran achieved the ILLUMINATE-A primary endpoint with a 53.5 percent mean reduction in urinary oxalate relative to placebo (p=1.7x10-14) and showed a 65.4 percent mean reduction in urinary oxalate relative to baseline. All tested study secondary endpoints were met, including the proportion of patients achieving near-normalization (84 percent) or normalization (52 percent) of urinary oxalate, compared with zero percent in the placebo group. Lumasiran administration was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events (AEs) and with mild injection site reactions (ISRs) as the most common drug-related AE.\n\n\nPH1 is an ultra-rare orphan disease caused by excessive oxalate production, and elevated urinary oxalate levels are associated with progression to end-stage kidney disease and other systemic complications.1,2 \n\n\nBased on the ILLUMINATE-A results, Alnylam filed a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA). The FDA has granted a Priority Review for the NDA and has set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA). In addition, the Marketing Authorisation Application (MAA) for lumas...