Andrzej Rostek Ultragenyx Pharmaceutical ( NASDAQ: RARE ) said a Phase 3 study of its gene therapy candidate DTX401 met its primary endpoint in the treatment of patients with glycogen storage disease type Ia, or GSDIa. The biotech company said the endpoint was to show the treatment resulted in a clinically meaningful and statistically significant reduction in daily cornstarch intake compared with placebo at week 48. The study also met its secondary endpoints of reduction of the number of cornstarch doses per day and maintenance of glucose control at week 48, Ultragenyx said .
The company said the results will be discussed with regulators to support the filing of a market application in 2025. More on Ultragenyx Pharmaceutical Ultragenyx Pharmaceutical, Inc. (RARE) Q1 2024 Earnings Call Transcript Ultragenyx Pharmaceutical Earnings: No Visible Path To Financial Success Yet Ultragenyx Pharmaceutical GAAP EPS of -$2.
03 misses by $0.28, revenue of $108.8M misses by $7.
64M Ultragenyx Pharmaceutical Q1 2024 Earnings Preview Seeking Alpha’s Quant Rating on Ultragenyx Pharmaceutical.
