Ultragenyx Q2 revenue $166M (+13% YoY); net loss narrows; UX111 BLA receives CRL

Ultragenyx Pharmaceutical Inc.

2025-Q2 EPS reported -$2.73 revenue$305,788,000

Q2 total revenue $166M, Crysvita $120M, Dojolvi $23M; net loss $115M ($1.17/sh) vs $132M loss prior year.
Reaffirms 2025 guidance: total rev $640-670M, Crysvita $460-480M, Dojolvi $90-100M; path to profitability in 2027.
UX111 gene therapy for Sanfilippo type A received FDA CRL for CMC/manufacturing issues; clinical data package no issues; resubmission after Type A meeting.
GTX-102 for Angelman syndrome granted Breakthrough Therapy; Phase 3 Aspire fully enrolled (129 pts); data expected H2 2026.
UX143 Phase 3 final analysis expected end of 2025; DTX401 BLA submission planned Q4 2025; cash $539M as of June 30.

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