Press release
Alnylam Initiates Phase 2 Study of Lumasiran in Patients with Recurrent Kidney Stone Disease
– Phase 2 Study will Evaluate the Safety and Efficacy of Lumasiran in Patients with Recurrent Kidney Stone Disease and Elevated Urinary Oxalate Levels –
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Phase 2 Study will Evaluate the Safety and Efficacy of Lumasiran in Patients with Recurrent Kidney Stone Disease and Elevated Urinary Oxalate Levels –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the company has initiated a global Phase 2 study to evaluate the safety and efficacy of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in patients with recurrent kidney stone disease and elevated urinary oxalate levels. Lumasiran is approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients and by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups, in each region under the brand name OXLUMO®.\n\n“Recurrent kidney stone disease is a prevalent disease, associated with significant clinical burden, including pain, infection, hospitalizations, and an increased risk of developing chronic kidney disease and kidney failure. The majority of kidney stones in adults are formed from calcium oxalate crystals and are accompanied by elevated urinary oxalate,” said Jeroen Valkenburg, General Manager, Lumasiran program at Alnylam. “To that end, we believe the reductions in urinary oxalate due to silencing of hepatic HAO1 we see with lumasiran in patients with primary hyperoxaluria type 1 may potentially apply to other diseases characterized by oxalate overproduction, such as recurrent kidney stone disease, where despite standard of care, stones continue to recur. We look forward to testing this hypothesis in this Phase 2 study.”\n\nAbout the Phase 2 Study in Patients with Recurrent Kidney Stone Disease\n\nThe Phase 2 trial is a randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacodynamics, and pharmacokinetics of lumasiran administered subcutaneously in patients with recurrent calcium oxalate kidney stone disease and elevated urinary oxalate levels. This global, multicenter trial is expected to enroll 120 adults with a documented diagnosis of recurrent calcium oxalate kidney stone disease, based on two or more stone events within five years prior to screening, and whose 24-hour urinary oxalate levels ...