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ALNY >Alnylam Reports Positive Initial Results from Ongoing Phase 1/2 Study of ALN-GO1, an Investigational RNAi Therapeutic for the Treatment of Primary Hyperoxaluria Type 1

http://seekingalpha.com/pr/16614854-alnylam-reports-positive-initial-results-ongoing-phase-1-2-study-aln-go1-investigational-rnai

Initial results include all available data as of the data transfer dates on August 17, 2016 (for safety) and September 2, 2016 (for pharmacodynamic activity). Subjects in Part A (N=32) were enrolled in four single ascending dose cohorts (N=8 per group, randomized 3:1 drug:placebo), with subjects receiving ALN-GO1 at doses ranging from 0.3 to 6.0 mg/kg. ALN-GO1 administration resulted in dose-dependent and statistically significant (nominal two-sided p values less than 0.05) increases from baseline in plasma and urinary glycolate as compared to placebo, with up to an 8-fold increase in plasma glycolate in the highest dose cohort. Based on extrapolation from pre-clinical studies, the observed 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.

Single doses of ALN-GO1 were shown to be generally well tolerated in healthy adult volunteers. There were no SAEs reported. Adverse events (AEs) were reported in 88% (N=21) of ALN-GO1 treated subjects and 63% (N=5) of placebo treated subjects. Common AEs occurring in greater than 10% of ALN-GO1 treated subjects included nasopharyngitis (N=6), headache (N=5), and transient injection site pain (N=4). All AEs were mild to moderate with the exception of one subject in the lowest dose cohort who had transient, asymptomatic CPK elevation which was unrelated to study drug.