Dicerna Posts Mixed Nedosiran Data In Rare Kidney Disease Trial

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  • Dicerna Pharmaceuticals Inc DRNA has announced top-line results from its PHYOX4 study of nedosiran for primary hyperoxaluria type 3 (PH3). 
  • Related: Why Is Dicerna Stock Getting Slammed Today?
  • 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. 
  • Price Action: DRNA shares are up 1.73% at $21.15 during the market session on the last check Tuesday.
Posted In: BiotechNewsHealth CareSmall CapMoversTrading IdeasGeneralBriefsKidney Diseases
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