Ultragenyx reported positive 36-week results from the Phase 3 Enh3ance trial of DTX301, an investigational AAV8 gene therapy for ornithine transcarbamylase deficiency. In the randomized, double-blind, placebo-controlled period, DTX301 (n=18) reduced 24-hour plasma ammonia AUC0-24 by 18% versus placebo (n=19) at Week 36 (p=0.018). The company also reported a mean 27% reduction in ammonia scavenger medications at Week 36 among treated patients. The study is continuing to a second primary endpoint evaluating treatment burden through 64 weeks of follow-up, with data expected in the first half of 2027.
Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ultragenyx Pharmaceutical Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: 202603120830PRIMZONEFULLFEED9670725) on March 12, 2026, and is solely responsible for the information contained therein.
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