DRNA - Dicerna concludes enrollment in mid-stage nedosiran trial in rare metabolic disorder

Dicerna Pharmaceuticals ([[DRNA]] +1.2%) has completed enrollment in PHYOX2 Phase 2 trial evaluating its lead investigational therapy, nedosiran, as a once-monthly treatment of all three known types of primary hyperoxaluria, a rare disorder that mainly affects the kidneys and results from buildup of a substance called oxalate.The company expects the last patient visit in the trial to occur in the first half of 2021 and to report top-line results from the study mid-year, with a goal of submitting marketing application in Q3 of 2021.The PHYOX2 trial is designed to evaluate the efficacy and safety of nedosiran in participants aged six years and older who have PH1 or PH2 types of hyperoxaluria.The primary endpoint of the study is the percent change from baseline in 24-hour urinary oxalate excretion between Days 90 and 180.

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Dicerna concludes enrollment in mid-stage nedosiran trial in rare metabolic disorder