Ultragenyx’s DTX401 shows positive action in glycogen storage disease

Ultragenyx Pharmaceutical (NASDAQ:RARE) is up 4% premarket, albeit on only 100 shares, following its announcement of preliminary data from a Phase 1/2 trial evaluating gene therapy DTX401 in patients with glycogen storage disease type Ia, an inherited disorder characterized by the build up of sugar (glycogen) in cells leading to impaired organ function.

Three patients in the lowest dose cohort all experienced improved glucose control and increased time to hypoglycemia (low blood sugar) during fasting while two experienced clinically meaningful improvements in time to hypoglycemia during a controlled fasting challenge.

No infusion-related adverse events and no serious treatment-related adverse events have been reported. All adverse events to date have been mild or moderate.

Enrollment is the second cohort will begin this month with interim data expected mid-year.

https://seekingalpha.com/news/3420658-ultragenyxs-dtx401-shows-positive-action-glycogen-storage-disease-shares-4-percent-premarket