Alnylam's lumasiran shows treatment benefit in rare kidney disorder

• Following up on its announcement in late September, Alnylam Pharmaceuticals (ALNY -0.1%) reports detailed positive results from its Phase 3 clinical trial, ILLUMINATE-B, evaluating RNAi therapeutic lumasiran in adults and children with primary hyperoxaluria type 1 (PH1), a disorder caused by excessive production of oxalate which leads to the formation of calcium oxalate crystals in the kidneys and urinary tract. The data were presented at the American Society of Nephrology Kidney Week conference.
• Treatment with lumasiran led to a 72% average reduction in spot urinary oxalate:creatinine ratio (a metric for renal stone disease) from baseline at month 6, successfully meeting the primary endpoint. Nephrocalcinosis (the deposition of calcium oxalate and calcium phosphate in the kidneys) improved in 44% (n=8/18) of treated patients.
• 12-month data from another Phase 3, ILLUMINATE-A, showed sustained reductions in 24-hour urinary oxalate excretion from month 6.
• Results from an open-label Phase 2 extension study also showed durable efficacy and long-term safety. Ongoing treatment with lumasiran resulted in a 74% mean maximal reduction in urinary oxalate from baseline. 94% (n=17/18) of patients achieved normal or near-normal levels of urinary oxalate while mean eGFR levels (metric for kidney function) remained stable over time.
• Lumasiran, branded as Oxlumo, is designed to reduce oxalate in the body by reducing the levels of an enzyme called glycolate oxidase which depletes the substrate necessary for oxalate production.
• The company's U.S. marketing application is currently under FDA review with an action date of December 3. Last week, an advisory group in Europe issued a positive opinion backing approval. The nod from the European Commission should happen in December.
• https://seekingalpha.com/news/3624882-alnylams-lumasiran-shows-treatment-benefit-in-rare-kidney-disorder