ILLUMINATE-A phase III study of ALN GO1 meets primary endpoint in primary hyperoxaluria type 1.- Alnylam Pharma

Alnylam Pharmaceuticals announced that the ILLUMINATE-A Phase III study of ALN GO1 (lumasiran), an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1), met its primary efficacy endpoint and all tested secondary endpoints. Specifically, lumasiran met the primary efficacy endpoint of percent change from baseline, relative to placebo, in 24-hour urinary oxalate excretion averaged across months 3 to 6 (p less than 0.0001). The study also achieved statistically significant results for all six tested secondary endpoints (p less than or equal to 0.001).

Lumasiran also demonstrated an encouraging safety and tolerability profile. There were no serious or severe adverse events in the study, and results showed that lumasiran was generally well tolerated with an overall profile generally consistent with that observed in Phase I/II and open-label extension studies of lumasiran. Based on these results, the Company plans to submit a New Drug Application (NDA) and file a Marketing Authorisation Application (MAA) for lumasiran in early 2020.

Comment: Lumasiran has received U.S. and EU Orphan Drug Designations, Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), and a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). Full ILLUMINATE-A study results will be presented in an oral plenary session on Tuesday, March 31, 2020 at OxalEurope International Congress.