Dicerna Posts Mixed Nedosiran Data In Rare Kidney Disease Trial

Dicerna Pharmaceuticals Inc DRNA has announced top-line results from its PHYOX4 study of nedosiran for primary hyperoxaluria type 3 (PH3).
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Nedosiran demonstrated safety and tolerability results consistent with previously reported studies.
Patients administered nedosiran also showed a trend in urinary oxalate (Uox) reduction; however, these reductions did not meet prespecified secondary efficacy endpoint criteria.
Dicerna plans to submit an FDA marketing application for nedosiran to treat PH1 in Q4 of 2021.
No subjects in either group achieved the prespecified secondary efficacy endpoint, a greater than 30% decrease from baseline in 24-hour Uox excretion on at least two consecutive visits over the three-month observation period.
However, all patients administered a single dose of nedosiran demonstrated Uox reductions relative to baseline at one or more time points during the three months.
Primary hyperoxaluria (PH) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys.
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