Initial Results from Ongoing Phase 1/2 Study with ALN-GO1, in Development for the Treatment of Primary Hyperoxaluria Type 1

We presented initial clinical data from ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1), at the 17th Congress of the International Pediatric Nephrology Association (IPNA), held September 20 – 24, 2016 in Iguaçu, Brazil.

ALN-GO1 administration in healthy volunteers enrolled in Part A of the ongoing Phase 1/2 study (N=32) resulted in dose-dependent and statistically significant increases in plasma and urinary glycolate as compared to placebo.  Up to an 8-fold increase in plasma glycolate was observed in the highest dose cohort as of the data transfer date of September 2, 2016.  Based on extrapolation from pre-clinical studies, this level of glycolate increase would correlate with an estimated greater than 80% silencing of the HAO1 mRNA, the transcript of the GO enzyme.  The effects of ALN-GO1 were highly durable, with levels sustained through 85 days at the highest dose, supportive of a once-monthly and possibly once-quarterly subcutaneous dose regimen.

ALN-GO1 was found to be generally well tolerated in healthy adult volunteers as of the safety data transfer on August 17, 2016.  There were no SAEs reported.  All AEs were mild to moderate with the exception of one subject in the lowest dose cohort who had a transient, asymptomatic CPK elevation deemed unrelated to study drug.

These clinical data provide preliminary human proof of concept for ALN-GO1, and we are now planning to transition to Part B of the Phase 1/2 trial, where we aim to achieve lowering of urinary oxalate in PH1 patients.  PH1 is an ultra-rare orphan disease that primarily afflicts children, with enormous unmet medical need and no approved therapeutics on the market.  We look forward to the continued advancement of ALN-GO1 as we work toward providing this patient community with a potentially transformative treatment option.