Ultragenyx Pharmaceutical (RARE) Corporate presentation summary

Strategic vision and business model

• Focus on developing first-ever treatments for rare diseases, emphasizing potent biology and patient-centric approaches.

• Efficient commercialization with a lean team and adaptive trial designs to expedite development and reduce post-approval R&D costs.

• Four commercial products and a diverse late-stage clinical pipeline targeting high unmet medical needs.

Financial performance and outlook

• Total revenue grew approximately 20% in 2025, reaching $673M, with guidance of $730–760M for 2026.

• Crysvita remains the largest revenue contributor, with $481M in 2025 and projected $500–520M in 2026.

• Strategic restructuring aims for profitability in 2027, with R&D expenses expected to decrease by ~38% and overall expenses down at least 15% by 2027.

• $534M in cash and investments as of March 31, 2026, with planned monetization of PRVs from UX111 and DTX401.

Key clinical programs and catalysts

• UX111 for MPS IIIA: PDUFA date September 19, 2026; showed substantial and durable reductions in CSF HS and improvements in developmental skills.

• DTX401 for GSDIa: PDUFA date August 23, 2026; Phase 3 data showed significant reductions in cornstarch intake and improved glucose control, with acceptable safety profile.

• DTX301 for OTC deficiency: Phase 3 data showed 18% reduction in plasma ammonia AUC at Week 36, with improvements in symptoms and functioning; complete responder data expected 1H 2027.

• GTX-102 for Angelman syndrome: Phase 3 Aspire data expected 2H 2026; no approved therapies currently exist.

• UX701 for Wilson disease: Stage 1 dose finding data expected in 2026; clinical activity observed with some patients off chelation therapy.