Published on 31 May 2024 on Zacks via Yahoo Finance
Ultragenyx (RARE) DTX401 Meets Goals in Metabolic Disorder Study
Ultragenyx Pharmaceutical RARE announced positive top-line results from the late-stage study of its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia (GSDIa) patients aged eight years and older. GSDIa is an inherited metabolic disorder that affects approximately 6,000 patients worldwide.
Per the data readout, the phase III GlucoGene study (NCT05139316) achieved its primary endpoint demonstrating a statistically significant and clinically meaningful reduction in daily cornstarch intake upon treatment with DTX401 compared with placebo at week 48.