Full patient enrollment achieved in ILLUMINATE-A Phase III study of lumasiran to treat primary hyperoxaluria type 1

Alnylam Pharmaceuticals, Inc. announced that it has achieved full patient enrollment in its ILLUMINATE-A Phase III study of lumasiran , an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of adults and children with primary hyperoxaluria type 1 (PH1). The study enrolled patients across 16 sites in eight countries. Alnylam is on track to report topline results from ILLUMINATE-A expected in late 2019 and, if positive, to submit filings for global regulatory approvals starting in early 2020. The Company also announced complete positive results from its Phase 1/II clinical study and reiterated positive results from its ongoing Phase II open-label extension (OLE) study of lumasiran. Results will be presented at the 2019 Oxalosis & Hyperoxaluria (OHF) International Workshop being held in Boston, on June 21-22.

About the ILLUMINATE-A Phase III Stud : ILLUMINATE-A is a six month randomized, double-blind, placebo-controlled, global, multicenter Phase III study to evaluate the efficacy and safety of lumasiran in approximately 30 patients with a documented diagnosis of PH1, followed by a 54 month extension period where all patients will receive lumasiran. Patients are randomized 2:1 to receive three monthly doses of lumasiran or placebo at 3 mg/kg followed by quarterly maintenance doses. The primary endpoint is the percent change in 24-hour urinary oxalate excretion from months 3 to 6 relative to baseline in the patients treated with lumasiran as compared to placebo. Key secondary and exploratory endpoints will evaluate additional measures of urinary oxalate, plasma oxalate, estimated glomerular filtration rate (eGFR), safety and tolerability, and quality of life. At month 6, the patients receiving placebo will cross over to receive lumasiran for long-term follow up. For more information on ILLUMINATE-A (NCT03681184).