other material
confidence high
sentiment positive
materiality 0.85
Ultragenyx DTX301 Phase 3 meets primary endpoint: 18% ammonia AUC reduction (p=0.018) in OTC deficiency
Ultragenyx Pharmaceutical Inc.
- DTX301-treated patients (n=18) showed 18% reduction in 24-hour plasma ammonia AUC vs placebo (n=19) at Week 36 (p=0.018).
- 8 of 9 patients with abnormal baseline ammonia reached normal levels; treated patients maintained normal ammonia despite 27% mean reduction in scavenger meds.
- 71% of treated patients reported much improvement on PGIC vs 0% for placebo at Week 24.
- One SAE of acute hepatitis (resolved with steroids); 5 hyperammonemic crises in placebo (1 death) vs 1 in treated (no deaths).
- Study continues to second primary endpoint on treatment burden; data expected H1 2027.