Ultragenyx Pharmaceutical Stock Earnings Reports

Ultragenyx reported Q1 2026 total revenue of $136M driven by Crysvita ($93M), Dojolvi ($18M), Evkeeza ($18M) and Mepsevii ($7M). The company reaffirmed 2026 revenue guidance of $730M–$760M (8%–13% growth vs. 2025) and expects Crysvita $500M–$520M and Dojolvi $100M–$110M. Q1 operating expenses were $305M (including $30M non‑cash stock comp and $30M restructuring expenses); net loss was $185M ($1.84/share). Cash and marketable securities were $534M at 3/31/26 and net cash used in operations was $197M (seasonally higher in Q1). Management continues to target a path to profitability in 2027, expects R&D + SG&A to be flat to slightly down in 2026 and to decrease at least 15% in 2027 vs. 2025. Key upcoming catalysts: PDUFA for DTX401 (Aug 23, 2026) and UX111 (Sep 19, 2026), and topline Phase 3 Aspire readout for GTX‑102 (Angelman syndrome) in H2 2026. GTX‑102 Phase 1/2 long‑term data (74 treated; 66 in extension; avg. on therapy ~3 years) show sustained improvements across domains: Bayley‑4 cognitive raw score approaching a ~10‑point mean gain at 12 months (above a 6‑point clinically meaningful threshold) and highly significant MDRI results (p<0.0001 at 12/24/36 months). Manufacturing capacity is being scaled (Bedford, MA facility active for some programs), and launch readiness is underway for potential late‑2026 gene therapy approvals. The company holds two Priority Review Vouchers (PRVs) assumed in modeling at just over $100M each, with upside if market values rise. Management notes most GTX‑102 discontinuations were due to study burden rather than safety.