Ultragenyx Pharmaceutical Inc. · NASDAQ:RARE
Ultragenyx Pharmaceutical — Q4 2025 Earnings Presentation
46 slides from Ultragenyx Pharmaceutical's earnings presentation, Feb 12, 2026
Slide 1: Corporate Presentation
Corporate Presentation February 2026 Confidential and Proprietary
Slide 2: Forward looking statements
Forward looking statements Cautionary note regarding forward-looking statements: This presentation contains forward-looking development goals in the expected time frames, risks related to reliance on third parties to statements, including, but not limited to, statements regarding our expectations, e
Slide 3: Leading the future of rare disease
Ultragenyx founded to develop first-ever treatments Leading the future of rare disease Aly lives with X-Linked Hypophosphatemia 3 Confidential and Proprietary
Slide 4: Our differentiated approach to rare diseases
Our differentiated approach to rare diseases Research Development Commercial Pursue high potential programs Accelerate to drive value Patient-centric approach • Potent biology in severe diseases • Adaptive trial designs • Lean commercial team • Treating underlying cause • Novel endpoints • Emphasize
Slide 5: Creating a successful and profitable rare disease company
Creating a successful and profitable rare disease company 4 commercial products Diverse clinical pipeline Potential Phase 2/3 Mason lives with Angelman syndrome 5 programs 2 approvals in 2026 5 Confidential and Proprietary
Slide 6: Focused on three therapeutic areas
Focused on three therapeutic areas Late-stage pipeline will leverage successful global commercial organization BONE-ENDOCRINE INBORN ERRORS OF METABOLISM NEUROGENETIC COMMERCIAL LC-FAOD MPS VII XLH & TIO HoFH Ph 3: UX143 for OI Ph 3: UX111 for MPS IIIA Ph 3: GTX-102 for AS CLNICAL PROGRAMS LATE STAG
Slide 7: Diverse late-stage clinical pipeline
Diverse late-stage clinical pipeline Inborn Errors of Therapeutic Area: Bone/Endo Neurogenetic Metabolism Candidate Description Phase 1 Phase 2 Phase 3 Route of Admin Prevalence1 ASO activating Intrathecal (IT) GTX-102 paternal expression of Angelman Syndrome (AS) Infusion ~60,000 UBE3A AAV9 SGSH UX
Slide 8: Clinical Programs
Clinical Programs Confidential and Proprietary
Slide 9: UX111 for Sanfilippo syndrome (MPS IIIA)
UX111 for Sanfilippo syndrome (MPS IIIA) AAV9 gene therapy MPS IIIA: Fatal lysosomal storage disease of CNS − Early childhood onset − Rapid neurodegeneration • Treatment: No approved therapies • Prevalence*: ~3,000 to 5,000 * Prevalence in commercially accessible geographies Sadie lives with MPSIIIA
Slide 10: UX111 for MPS IIIA: Substantial and durable reduction in CSF HS exposure
UX111 for MPS IIIA: Substantial and durable reduction in CSF HS1 exposure regardless of age or stage of disease Rapid and sustained reduction in CSF HS1 Key Takeaways up to 7.76 years • > 80% of participants reduced CSF HS by 50% in efficacy set CSF HS Exposure (%) • Median CSF HS exposure One patie
Slide 11: UX111 for MPS IIIA: Bayley raw scores improved or stabilized
UX111 for MPS IIIA: Bayley raw scores on all 5 subtests were improved or stabilized in mITT set compared to natural history Bayley-III Raw Score Bayley-III Raw Score Bayley-III Raw Score 23.2 (3.3) p <0.0001 11.1 (2.9) 8.1 (2.8) p=0.0008 p=0.0076 Cognitive Receptive Communication Expressive Communic
Slide 12: UX111 for MPS IIIA: Conclusions
UX111 for MPS IIIA: Conclusions • Substantial and durable reductions in CSF HS exposure – Regardless of dose, age or stage of disease at time of intervention • Developmental skills durably improved or stabilized relative to declining natural history – Meaningful differences with persuasive statistic
Slide 13: DTX401 for glycogen storage disease type Ia (GSDIa)
DTX401 for glycogen storage disease type Ia (GSDIa) AAV8 gene therapy; PDUFA action date set for August 23, 2026 GSDIa: Life-threatening defect in liver’s ability to release glucose due to G6Pase deficiency Daily cornstarch consumption − Severe hypoglycemia − Long-term liver and renal disease • Trea
Slide 14: DTX401 for GSDIa: Phase 3 successful across primary and key secondary endpoints
DTX401 for GSDIa: Phase 3 successful across primary and key secondary endpoints p-value Week 48 Takeaways Primary • GSDIa is a severe, life-threatening %∆ daily cornstarch intake <0.0001 metabolic disease, with long term Endpoint complications due to inability to control glucose # of total daily dos
Slide 15: DTX401 for GSDIa: Phase 3 crossover and original arm continued reducing cornstarch
DTX401 for GSDIa: Phase 3 crossover and original treatment arm continued reducing daily cornstarch through Week 96 Statistically significant and clinically meaningful cornstarch Week 96 reductions continued in crossover period Daily cornstarch (CS) reductions • 61% reduction in daily CS at Week 96 i
Slide 16: DTX401 for GSDIa: Reductions in day and nighttime CS vs placebo
DTX401 for GSDIa: Statistically significant reductions in frequency and quantity of day and nighttime CS vs placebo at Week 48 that continued to Week 96 Total Daily Cornstarch (CS) Doses Nighttime Cornstarch (CS) Doses and Grams Placebo / Placebo / Total Daily CS Doses (n) DTX4011 Crossover2 p-value
Slide 17: DTX401 for GSDIa: Patients reported meaningful improvements
DTX401 for GSDIa: Patients reported meaningful improvements in disease condition at Week 48 and Week 96 Patient Global Impression of Change (PGIC) • Week 48: 79% (15/19) reported improved GSDIa in DTX401-treated group versus 52% (12/23) Placebo • Week 96: (end of Double-blind Crossover Period) impro
Slide 18: GTX-102 for Angelman syndrome (AS)
GTX-102 for Angelman syndrome (AS) Antisense oligonucleotide; Phase 3 Aspire data expected in 2H-2026 Angelman Syndrome: Loss-of-function of maternal UBE3A gene − Cognitive, communication, motor, behavior, and sleep impairment and seizures − Requires continuous care • Treatment: No approved therapie
Slide 19: GTX-102 for AS: Two global studies enrolling broad age range and genotypes
GTX-102 for AS: Two global studies enrolling broad age range and genotypes Aspire Phase 3 Sham-Controlled Study Aurora Open Label Study Cohort A GTX-102 Open Label Extension Cohort B Sham Open Label Extension Screening Cohort C Screening 129 Patients; 48-week primary 1:1 Randomization efficacy perio
Slide 20: UX701 for Wilson disease (WD)
UX701 for Wilson disease (WD) AAV9 gene therapy; Stage 1, dose finding data expected in 2026 Wilson disease: Life-threatening defect in liver’s ability to metabolize copper due to ATP7B mutation − Liver failure − Neurologic deterioration − Death, if untreated • Treatment: Modified diet, chelation th
Slide 21: UX701 for WD: Clinical activity observed in Stage 1
UX701 for WD: Clinical activity observed in Stage 1 with 6 of 15 patients completely off chelators and/or zinc therapy1 • Clinical activity observed across all three dose cohorts in Stage 1 – 6 of 15 patients completely off chelators and/or zinc therapy – 1 additional patient tapering standard of ca
Slide 22: Financials and 2026 Catalysts
Financials and 2026 Catalysts Confidential and Proprietary
Slide 23: Total revenue grew approximately 20% in 2025
Total revenue grew approximately 20% in 2025 800 Total Revenue* $730 - 760M 2025 2026 700 $673M Product Actuals Guidance 600 $560M Total $673M $730-760M revenue1 Revenue ($M) 500 20% 8-13% $433M 400 $356M $481M $500-$520M Crysvita2 17% 4-8% 300 $266M TIO $96M $100-$110M 200 $182M Dojolvi 9% 4-14% $1
Slide 24: Strategic restructuring plan supports path to profitability in 2027
Strategic restructuring plan supports path to profitability in 2027 Profitability assumptions • Revenue: Continued growth from current products, plus contribution from upcoming launches • Expected combined R&D and SG&A expenses* compared to 2025: • 2026: flat to down low-single digits • 2027: decrea
Slide 25: Key clinical and regulatory catalysts
Key clinical and regulatory catalysts PROGRAM OBJECTIVE ANTICIPATED TIMING DTX401 BLA filing GSDIa PDUFA decision date August 23, 2026 UX111 BLA resubmission TBD Sanfilippo syndrome UX701 Stage 1 dose finding data (Cohorts 1-4) 2026 Wilson disease GTX-102 Phase 3 Aspire data 2H-2026 Angelman syn
Slide 26: We are leading the future of rare disease with first ever treatments
We are leading the future of rare disease with first ever treatments History of consistent and ongoing revenue growth from base business Near-term catalysts from 5 Phase 2/3 programs and 2 potential approvals Revenue growth and new launches plus expense management to reach profitability in 2027 and
Slide 27: Appendix
Appendix Confidential and Proprietary
Slide 28: Key licenses & intellectual property – clinical programs
Key licenses & intellectual property – clinical programs Product License US Intellectual Property Rights/Royalties + Milestones • Setrusumab antibody (2028) UX143 • Use of anti-sclerostin antibodies including setrusumab for treatment of OI (2037) (Osteogenesis Mereo Biopharma • Tiered double-digit r
Slide 29: Crysvita partnership revenue recognition
Crysvita partnership revenue recognition Product Sales: Revenue in Profit-Share Royalty revenue in Latin America & Türkiye Territory: U.S. and Canada European Territory Commercialization Ultragenyx KKC KKC KKC books sales and pays Ultragenyx books sales and revenue share calculated KKC books sales a
Slide 30: Illustration of Crysvita Royalty Monetization
Illustration of Crysvita Royalty Monetization • RARE receives annual tiered royalty up to 30% on KKC’s U.S./Canada revenue Royalty Interest % of RARE • RARE received $500M from OMERS for 30% of RARE royalties up to 1.45x OMERS = +25% • Payments began April 2023 U.S. / Canada OMERS = 30% • RARE recei
Slide 31: CRYSVITA exclusivity summary
CRYSVITA® exclusivity summary XLH Orphan TIO Orphan Biologics Crysvita CoM Q2W Exclusivity Exclusivity Exclusivity Patent Dosing Patent United States 2025 2027 2030 2032* 2035 2018 2020 2022 2024 2026 2028 2030 2032 2034 2036 2028 2033* 2035 XLH Orphan + Crysvita CoM Q2W Europe D&M Exclusivity Paten
Slide 32: DOJOLVI exclusivity summary
DOJOLVI® exclusivity summary NCE LC-FAOD Orphan Dojolvi CoM Ultrapure Dojolvi Exclusivity Exclusivity Patent (Pending; Allowed) United States 2025 2027 2029* 2034 2018 2020 2022 2024 2026 2028 2030 2032 2034 2036 2034 Ultrapure Dojolvi Europe (Pending) *Includes US PTE award Regulatory Exclusivity I
Slide 33: MEPSEVII exclusivity summary
MEPSEVII® exclusivity summary MPS7 Orphan Biologics Mepsevii Exclusivity Exclusivity CoM Patent United States 2024 2029 2035 2018 2020 2022 2024 2026 2028 2030 2032 2034 2036 2028 2035 MPS7 Orphan + Mepsevii Europe D&M Exclusivity CoM Patent Regulatory Exclusivity Issued Patents 33 Confidential and
Slide 34: EVKEEZA exclusivity summary
EVKEEZA® exclusivity summary Evkeeza Atherosclerosis Formulation Data & Marketing CoM Treatment Patent Exclusivity Patent Patent (Pending) Europe 2031 2036* 2037 2041 2022 2024 2026 2028 2030 2032 2034 2036 2038 2040 *Includes EU SPC award Regulatory Exclusivity Issued Patents Pending Patent Applica
Slide 35: UX143 (setrusumab) for Osteogenesis Imperfecta
UX143 (setrusumab) for Osteogenesis Imperfecta Confidential and Proprietary
Slide 36: UX143 for osteogenesis imperfecta Phase 3 results
UX143 for osteogenesis imperfecta Phase 3 results Neither study achieved primary endpoint of reduction in AFR1 compared to placebo (Orbit) or bisphosphonates (Cosmic) Both studies demonstrated statistically significant increases in bone mineral density (BMD) Additional data shows reduction in verteb
Slide 37: Key licenses & intellectual property – commercial products
Key licenses & intellectual property – commercial products Product License United States Intellectual Property Rights/Royalties • Anti-FGF23 antibodies and use for treatment of XLH and TIO (2028-2032)1 CRYSVITA® Kyowa Kirin Co. (KKC) • Q2W dosing for treatment of FGF23-associated hypophosphatemic di
Slide 38: Two randomized Phase 3 studies provide large data set
Two randomized Phase 3 studies provide large data set controlled with placebo or active Orbit Phase 3 Placebo-Controlled Study Design Cosmic Phase 3 Active-Controlled Study Design Setrusumab (n=107) Setrusumab (n=34) Open Label Extension Open Label Extension Placebo (n=52) Screening IV-BP (n=35) Scr
Slide 39: Baseline fractures are comparable between groups in both studies
Baseline fractures are comparable between groups in both studies Orbit: more severe type III/IV patients exited placebo via rescue criteria Orbit Phase 3 Baseline Historical Fractures1 Cosmic Phase 3 Baseline Historical Fractures1 Setrusumab Placebo Setrusumab IV-Bisphos. Mean / Median 3.2 / 2.0 3.3
Slide 40: Setrusumab is substantially more effective in increasing BMD
Setrusumab is substantially more effective in increasing BMD Orbit: UX143 demonstrated clinically and statistically Cosmic: UX143 demonstrated clinically and statistically significant increases in BMD vs placebo significant increases in BMD vs active comparator 1.0 UX143* 0.70 (0.08) 1.4 Placebo* -0
Slide 41: Orbit: fracture rates in UX143 vs placebo
Orbit: UX143 patients showed an increase in fractures over a low placebo rate, but were the same as placebo when all fractures were considered (p=ns) Primary Endpoint (Excl. mVFTFS) 160 Cumulative Distribution of Confirmed fractures by Primary Endpoint1 Key Secondary 140 Excluding mVFTFS x-ray and s
Slide 42: Orbit: disease severity reduced and pain/comfort & activity improved
Orbit: In setrusumab patients, disease severity (PGIS) in peds/teens reduced and pain/comfort & sports/activity improved Peds/Teens patients constitute 85% of subjects in Orbit Ph3 study (135/159) Patient Global Impression Pain/Comfort Sports/Activity Scale of Severity (PGIS) POSNA-PODCI POSNA-PODCI
Slide 43: Cosmic: Setrusumab treatment shows reduced fractures over IV-BP
Cosmic: Setrusumab treatment shows reduced fractures over IV-BP (p=ns) 140 Confirmed fractures by Primary Endpoint Key Secondary1 Total Fractures 120 x-ray and skeletal survey Total fractures Excl. mVFTFS Cumulative Distribution of 100 Primary Endpoint # of fractures 102 82 80 UX143 AFR Mean (SD, SE
Slide 44: Cosmic: Large reduction in vertebral fractures on setrusumab
Cosmic: Large reduction in vertebral fractures on setrusumab (p-value 0.081) Despite MORE severe type III/IV patients on UX143 (65% UX143 vs 54% to IV-BP) Radiographically confirmed fractures Radiographically Confirmed Fracture Type All Vertebral Fractures (comparing 19 vs 46 vertebral fractures*) T
Slide 45: No new safety concerns identified, TEAEs consistent with safety profile
No new safety concerns identified, reported TEAEs are consistent with the anticipated safety profile for setrusumab Orbit* Cosmic* Treatment emergent adverse events (TEAE) Treatment emergent adverse events (TEAE) • No serious-related TEAE’s • No serious related TEAE’s • Low incidence (<2%) severe-re
Slide 46: Overall data suggest an impact of setrusumab on OI but missed primary AFR
Overall data suggest an impact of setrusumab on OI disease but missed primary AFR endpoints are a challenge The largest BMD improvements found in the lumbar spine BMD are associated with reduced vertebral fractures and improved pain and functional outcomes in pediatric patients Improved Lumbar Spine
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