Ultragenyx Phase 3 Enh3ance study showed statistically significant results

Ultragenyx (RARE) Pharmaceutical announced positive results from its Phase 3 Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase deficiency. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, DTX301-treated patients demonstrated a statistically significant and clinically meaningful 18% reduction in 24-hour plasma ammonia compared to placebo and maintained average ammonia AUC0-24 in the normal range through Week 36. Eight of nine patients with abnormal ammonia AUC0-24 at baseline, despite optimal current drug treatment and diet restriction, reached normal ammonia levels rapidly, which were generally maintained during this treatment period.