Alnylam reports positive topline results from ILLUMINATE-B phase III study of lumasiran for the treatment of primary hyperoxaluria type 1 in children under the age of six.

Alnylam Pharmaceuticals, Inc. announced positive topline results from the ILLUMINATE-B pediatric Phase III 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 III 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. ILLUMINATE-B Topline Study Results: ILLUMINATE-B (NCT03905694) is a single arm, open-label, multicenter Phase III trial that enrolled 18 patients with PH1 under the age of six (range: 3-72 months), with an estimated glomerular filtration rate (eGFR) of greater than 45 mL/min/1.73 m or normal serum creatinine if less than 12 months old, at nine study sites, in five countries around the world. Lumasiran was administered according to a weight-based dosing regimen. The primary efficacy endpoint of the study was the percent change from baseline to Month 6 in spot urinary oxalate:creatinine ratio averaged across Months 3 to 6. At six months, relative to baseline, lumasiran demonstrated a clinically meaningful reduction in spot urinary oxalate:creatinine ratio. Reduction of urinary oxalate relative to baseline was consistent across all three body weight categories (less than 10 kg; 10 kg to less than 20 kg, and 20 kg or higher). Lumasiran demonstrated positive results across secondary endpoints, including additional measures of urinary and plasma oxalate. There were no serious or severe adverse events related to study drug, and the overall safety and tolerability profile of lumasiran was consistent with that observed in the ILLUMINATE-A study. Full ILLUMINATE-B study results will be presented on October 22, 2020 at the ASN virtual congress. The Company is also conducting ILLUMINATE-C – a global single-arm Phase III study of lumasiran in PH1 patients of all ages with advanced renal disease, including patients on dialysis, with results expected in 2021.