DRNA - Dicerna completes dosing in nedosiran rare genetic disorder trial

Dicerna Pharmaceuticals (DRNA) has completed dosing in the company’s PHYOX4 single-dose safety and tolerability study of its investigational candidate, nedosiran, for the treatment of primary hyperoxaluria ((PH)) type 3 ((PH3)).Primary hyperoxaluria ((PH)) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys.The PHYOX4 trial is a randomized, placebo-controlled, double-blind, multicenter study designed to evaluate the safety and tolerability of a single subcutaneous dose of nedosiran in six patients with PH3 who have had at least one kidney stone event in the last 12 months.Study participants who respond to treatment with nedosiran and complete the trial are also eligible to enroll in the company’s PHYOX3 open-label extension study evaluating nedosiran’s long-term safety and efficacy in participants with PH1, PH2 or PH3.The company expects the results from ongoing PHYOX3 study to support the nedosiran New Drug Application ((NDA)) submission, which is planned for the fourth quarter of 2021.Shares

For further details see:

Dicerna completes dosing in nedosiran rare genetic disorder trial