07 Jun, 2020 ILLUMINATE-A Phase 3 Results for Lumasiran Presented at ERA-EDTA
Positive results from the ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic in development for the treatment of Primary Hyperoxaluria Type 1 (PH1), were presented by study investigators at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress, held June 6-9, 2020 as a virtual event.
The ILLUMINATE-A trial enrolled 39 patients across 16 study sites in eight countries. Patients were randomized 2:1 to receive lumasiran or placebo, with lumasiran administered subcutaneously monthly for three months followed by quarterly maintenance doses at 3 mg/kg.
- Lumasiran met the primary efficacy endpoint of 24-hour urinary oxalate reduction from Month 3 to Month 6 (averaged across timepoints), with lumasiran treatment (N=26) resulting in a 65.4% mean reduction in urinary oxalate relative to baseline, with a mean treatment difference of 53.5% relative to placebo (N=13) (p=1.7 x 10-14).
- Lumasiran also met all tested secondary endpoints, including a 62.5% mean reduction in 24-hour urinary oxalate:creatinine ratio from baseline (51.8% mean treatment difference relative to placebo; p=5.0 x 10-10), as well as the proportion of patients receiving lumasiran with 24-hour urinary oxalate levels at or below 1.5 x ULN (21/25 or 84%; p=8.3 x 10-7) or at or below ULN (13/25 or 52%; p=0.001).
- Lumasiran demonstrated encouraging safety and tolerability. There were no deaths and no severe or serious adverse events (AEs) reported. The most common AE (> 20%) was injection site reactions occurring in 35% of patients. All AEs were mild to moderate in severity.
We believe these results demonstrate the potential of lumasiran to substantially reduce oxalate overproduction – the cause of progressive kidney failure in PH1.