Dicerna Pharmaceuticals (DRNA +3.3%) announces positive preliminary data from a Phase 3 clinical trial, PHYOX3, evaluating once-monthly RNAi therapeutic nedosiran in patients with the three known types of primary hyperoxaluria (PH1, PH2 and PH3), a rare inherited disorder caused by excessive production of oxalate that leads to the formation of calcium oxalate crystals in the kidneys and urinary tract. The results were presented at the American Society of Nephrology Kidney Week conference.
100% (n=13/13) of treated patients (10 with PH1 and three with PH2) who completed the PHYOX1 Phase 1 study and reached day 180 in PHYOX3 achieved normal or near-normal urinary oxalate at one or more timepoints. 12 of the 13 achieved normal urinary oxalate at one or more visits. 62% (n=8/13) showed normalized urinary oxalate on at least three consecutive visits (7 of 10 with PH1 and 1 of 3 with PH2). The mean maximum reduction from baseline in urinary oxalate was 70.9% by day 180. 16 patients who completed PNYOX1 entered PHYOX3.
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