Alnylam Pharmaceuticals, Inc, the leading RNAi therapeutics company, announced today positive results from the 6-month primary analysis of the ILLUMINATE-B Phase 3 pediatric study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of adults and children with primary hyperoxaluria type 1 (PH1). Results were presented from ILLUMINATE-B, as well as new 12-month results from the ILLUMINATE-A pivotal Phase 3 study and the ongoing Phase 2 open-label extension (OLE) study, at the American Society of Nephrology (ASN) Kidney Week 2020 held as a virtual event on October 22-25.
Lumasiran is under review by the Food and Drug Administration (FDA) and received a Positive Opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on October 16, 2020. If approved, lumasiran will be marketed as OXLUMOTM.
“We are delighted to present these positive data from ILLUMINATE-B that reinforce previously reported clinical study findings for lumasiran and underscore its potential to be an important treatment option for patients of all ages with PH1, a devastating and potentially fatal disease with no approved pharmaceutical treatment options,” said Pritesh J. Gandhi, PharmD., Vice President and General Manager, Lumasiran Program at Alnylam. “Based on longer term follow-up from the ILLUMINATE-A and Phase 2 open-label extension studies, investigators presented data showing enduring reductions of urinary oxalate – the disease-causing metabolite in PH1. Moreover, we believe that newly presented results of exploratory endpoints provide preliminary evidence that reductions in urinary oxalate may lead to reduced rates of renal stone events and improve nephrocalcinosis in some patients.”
“Pathologic overproduction of oxalate by the liver is the root cause of morbidity and mortality associated with PH1. There is strong evidence in the literature to suggest that levels of urinary oxalate correlate with clinical outcomes in patients with this ultra-rare disease. With that in mind, I am pleased to see the reduction in urinary oxalate levels in response to lumasiran in all three studies presented at this year’s meeting. More broadly, I am encouraged by the promise that these findings hold for my patients living with this condition,” said Jeffrey M. Saland, M.D., Professor and Chief, Pediatric Nephrology and Hypertension, Jack and Lucy Clark Department of Pediatrics, Mount Sinai Kravis Children’s Hospital, New York City, and Investigator on the ILLUMINATE-A trial. “With the sustained reductions in urinary oxalate during long-term treatment and the exploratory renal stone and nephrocalcinosis data presented, I am hopeful about the potential of lumasiran to have a positive impact on the severe clinical manifestations that individuals with PH1 suffer.”
Read the full press release here.