Ultragenyx reports topline results from phase 3 study of DTX401 gene therapy for glycogen storage disease type Ia

Ultragenyx Pharmaceutical Inc., dedicated to addressing rare genetic diseases, reported positive findings from its phase 3 GlucoGene study (NCT05139316) evaluating DTX401, an investigational gene therapy for glycogen storage disease type Ia (GSDIa) in patients aged eight and older.

Results showed a significant reduction in daily cornstarch intake with DTX401 compared to placebo at Week 48. Patients on DTX401 achieved a mean reduction of 41.3% compared to 10.3% in the placebo group (p<0.0001). Secondary endpoints, including fewer cornstarch doses per day and improved glucose control, were also met.

The study confirmed DTX401's safety profile, with manageable hepatic effects. Long-term data demonstrated sustained reductions in cornstarch intake over five years.

GSDIa, a severe genetic disorder, affects glucose regulation and can lead to life-threatening complications. DTX401 offers promise as a potential treatment for this condition, affecting an estimated 6,000 patients worldwide.