Ultragenyx Discontinues GTX-102 and UX701 Programs Following Clinical Trial Outcomes

Ultragenyx will discontinue GTX-102 for Angelman syndrome after it failed to demonstrate efficacy in a Phase 3 trial, and end UX701 for Wilson disease, which did not advance to Phase 3. The company plans to redirect R&D resources toward programs including DTX301 and UX143. Management said the changes support its goal of achieving GAAP profitability by 2027; the article reports no financial figures or stock-price impact.