Ultragenyx Pharmaceutical Key Takeaways From 47th Annual Global Healthcare Conference

Ultragenyx management expressed confidence in the Phase 3 transition for GTX-102 in Angelman syndrome and emphasized the company's path to profitability by FY27.

Key Takeaways

• 1.Management is optimistic that Ph1/2 data for GTX-102 in Angelman syndrome will translate to the Phase 3 study.
• 2.Ultragenyx expects to reach GAAP profitability in FY27, driven by two near-term gene therapy launches.

Table of Contents

• Key Takeaways
• Valuation/Risks
• Disclosure Appendix

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