New molecular entity lumasiran shows positive results in clinical trial for treatment of rare kidney stone disease – primary hyperoxaluria type 1 (PH1)

Lumasiran, anRNA interference therapy that targets hydroxyacid oxidase 1, met primary endpoint with a 53.5% mean reduction in urinary oxalate (UO) vs. placebo. There was a 65.4% mean reduction in UO relative to baseline. US FDA granted priority review in May.


Biospace Inc.