Alnylam Pharmaceuticals, Inc. announced positive topline results from the ILLUMINATE-B pediatric Phase 3 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). ILLUMINATE-B is now the seventh Phase 3 study of an RNAi therapeutic that has yielded positive results, and the first-ever study evaluating the safety and efficacy of this new class of medicines in children under the age of six, including infants.
“We are pleased to report these positive topline results that we believe hold promise for many families impacted by PH1. The safety and efficacy of lumasiran are consistent with that reported for the ILLUMINATE-A study in patients six and older, demonstrating that lumasiran can significantly reduce the hepatic production of oxalate across all ages, which we believe can thereby address the underlying pathophysiology of PH1,” said Pritesh J. Gandhi, PharmD., Vice President and General Manager, Lumasiran Program at Alnylam. “The current standard of care for young children and infants diagnosed with PH1 is burdensome, including the frequent need for gastrostomy tube placement to enable hyperhydration, and, for those who have progressed to advanced disease, the risks associated with performing dialysis and, ultimately, organ transplantation. Thus, we believe, a meaningful reduction in urinary oxalate levels has the potential to favorably impact disease progression and management in very young patients. We look forward to reporting complete data from the ILLUMINATE-B study at the ASN virtual congress later this fall.”
“The ILLUMINATE-B results signal hope for the many families with children whose lives are deeply impacted by PH1. This is especially encouraging given that children as young as a few months old could benefit from the therapeutic approach that lumasiran offers, curbing production of oxalate at its source,” said Kim Hollander, Executive Director of the Oxalosis and Hyperoxaluria Foundation. “We are grateful to Alnylam for their continued commitment to the PH1 community and for designing and successfully conducting a study that addresses a particularly vulnerable group of patients – young children and babies.”