other material
confidence high
sentiment positive
materiality 0.85
Ultragenyx Phase 3 DTX401 gene therapy meets primary endpoint in GSDIa
Ultragenyx Pharmaceutical Inc.
- DTX401 reduced daily cornstarch intake by 41.3% vs 10.3% for placebo at Week 48 (p<0.0001).
- 68% of DTX401 patients achieved ≥30% reduction; 37% achieved ≥50% reduction.
- Secondary endpoint met: 1.1 fewer cornstarch doses/day vs 0.2 for placebo (p=0.0011).
- Safety profile acceptable; no dorsal root ganglion toxicity or thrombotic microangiopathy observed.
- Company plans to discuss results with regulators and submit marketing application in 2025.