Positive Phase III results from ILLUMINATE-A study of lumasiran

Positive Phase III results from the ILLUMINATE-A 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), were presented by US RNAi specialist Alnylam Pharmaceuticals (Nasdaq: ALNY) at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress on Sunday.

Lumasiran achieved the ILLUMINATE-A primary endpoint with a 53.5% mean reduction in urinary oxalate relative to placebo (p=1.7x10) and showed a 65.4% mean reduction in urinary oxalate relative to baseline. All tested study secondary endpoints were met, including the proportion of patients achieving near-normalization (84%) or normalization (52%) of urinary oxalate, compared with zero percent in the placebo group. Lumasiran administration was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events (AEs) and with mild injection site reactions (ISRs) as the most common drug-related AE.

Sixth positive Phase III study for RNAi therapeutics