Corporate Presentation

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1 Corporate Presentation September

2 Legal Warning Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our proposed public offering of shares of our common stock, plans with respect to commercializing our product and product candidates, our translational research program, the expected timing of release of additional data for our product candidates, plans to initiate additional studies for product candidates and timing and design of these studies, plans regarding ongoing studies for existing programs, our liquidity position as of the most recent fiscal quarter end, expectations regarding the adequacy of clinical data to support marketing applications and approvals of product candidates, our intent to file marketing applications and the timing of such filings, expectations regarding timing of receiving potential approval of product candidates, and expectations regarding prevalence of patients. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the completion of the proposed public offering on the anticipated terms or at all, our ability to effectively direct the use of the proceeds of our proposed public offering, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of our regulatory filings and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the availability or commercial potential of our product and product candidates, and our ability to integrate acquired businesses, which are more fully described in our most recent Form 10-Q or Form 10-K under the caption Risk Factors and elsewhere in such reports. Any forward-looking statements made by us reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties, and other factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by these forward-looking statements. Accordingly, our actual results may materially differ from our current expectations, estimates, and projections. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Any forward-looking statements made by us in this presentation speak only as of the date of this presentation and represent our estimates and assumptions only as of the date of this presentation. Except as required by law, we assume no obligation, and we disclaim any intent, to update these statements to reflect actual results. This presentation concerns drugs that are under preclinical and/or clinical investigation and which have not yet been approved for marketing by the U.S. Food and Drug Administration (FDA). They are currently limited by Federal law to investigational use, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated. Ultragenyx, Ultragenyx Pharmaceutical, and our logo are our trademarks. Any other trademarks appearing in these slides are the property of their respective holders. 2

3 Our Strategy Rapidly transforming undiscovered discoveries into effective rare disease products CLEAR BIOLOGY HOW HAVE WE DONE IT? RAPID DEVELOPMENT GLOBAL VISION 3

4 Expanding into New Modalities & Capabilities Optimizing solutions to genetic diseases with clear biology Deep Understanding of Genetic Disease Drug Development Small Molecule Traditional Biologics mrna AAV Gene Therapy Advantages Oral delivery Simple manufacturing High specificity targeting Long half life Ability to target undruggable targets Genomics-based discovery process Potential for onetime treatment for monogenic diseases Current Capabilities In late-stage clinical development: Substrate replacement Approved/ In latestage development: Fully human monoclonal antibodies Enzyme replacement therapies In preclinical development: 2 programs using mrna / lipid nanoparticle delivery In early clinical development: AAV gene transfer programs in the clinic 4

5 Two Approved Therapies and Diverse Pipeline Candidate Description Stage -2 to 0 IND Phase 1 Phase 2 Phase 3 Filed Est d Patients in Dev. World Crysvita Anti-FGF23 monoclonal antibody XLH TIO Approved in U.S. and E.U. ~48,000 ~2,000-4,000 CLINICAL TRANSLATIONAL RESEARCH Mepsevii UX007 DTX301 DTX401 DTX201 DTX701 UX068 Arc-#1 UX001P UX053 Undisclosed UX501 UX601 Enzyme replacement Substrate replacement AAV8-OTC Gene Transfer AAV8-G6Pase Gene Transfer AAV-FVIII Gene Transfer AAV-ATP7B Gene Transfer Substrate replacement mrna/ Lipid Substrate replacement mrna/ Lipid Substrate replacement AAV8-PAH Gene Transfer AAV8-ASS1 Gene Transfer MPS 7 LC-FAOD Glut1 DS OTC GSDIa Hemophilia A Wilson Disease Creatine Transporter Deficiency GNE Myopathy GSDIII FSHD PKU Citrullinemia type I Approved in U.S. and E.U. ~200 ~8,000-14,000 ~12,000-28,000 ~10,000 ~6,000 ~144,000 >50,000 ~10,000-50,000 >20,000 ~2,000 >10,000 >50,000 ~50,000 ~2,000 5 Small Molecule Protein biologic RNA Gene Therapy

6 Numerous Catalysts in 2018 Candidate Disease 1H18 2H18 Crysvita Anti-FGF23 monoclonal antibody XLH TIO Positive EC Decision FDA Approval/Launch Pediatric Phase 3 Data Phase 2 48-wk Data Mepsevii Enzyme Replacement MPS7 Positive CHMP Opinion EU Approval UX007 Substrate Replacement LC-FAOD Glut1 DS Pre-NDA meeting with FDA EU filing clarity Phase 3 Movement Disorder Data DTX301 AAV8 gene therapy OTC Deficiency Phase 1/2 Cohort 1 Data Phase 1/2 Cohort 2 Data DTX401 AAV8 gene therapy GSDIa IND Filed Phase 1/2 Cohort 1 Data 6 6 Clinical Regulatory

7 Commercial Progress 7 7

8 Unique Commercial Model Commercial model designed specifically for rare disease environment Early & comprehensive patient diagnosis activities Market access strategy focused on optimizing patient access UltraCare, comprehensive in-house patient support in place Crysvita and Mepsevii launches off to strong start Full prescribing information available at 8

9 Field Teams Are Purpose Built to Support Rare Disease Patients Through Deep Coordination UltraCare Liaison Profile institutional & payer formulary review HCP Center logistical readiness assessment Profile confirmed accounts Patient Diagnosis Liaison Patient diagnosis confirmation Treater insight and referral patterns Medical Science Liaison Academic centers & key payers Disease education Opinion leader development & mapping Medical Affairs Teams 9

10 Crysvita Launch off to a Strong Start First two months of commercial progress As of June 30, completed start forms 60% pediatric; 40% adult 150 unique prescribers across 38 states Policies covering ~100 million lives Positive EC Decision* FDA Approval Estimated Canada Decision Estimated Chile, Mexico, Colombia, Brazil, Argentina, Switzerland*, Decisions Launch* Launch Various EU launches throughout the year* Named Patient Programs Full Crysvita prescribing information available at 10 *Denotes territories where KKI has commercial responsibility

11 Crysvita MAb Against FGF23 for XLH Increased low serum phosphate associated with bone disease XLH: Excess FGF23 1 causes Rickets excess renal phosphate loss Key symptoms: Rickets, deformity, Bowing short stature, fractures, pain, osteomalacia, stiffness CHILDREN Rickets ADULTS Bowing Fractures Conventional Therapy: Oral phosphate + Vitamin D (nephrocalcinosis risk) US prevalence: ~12, Fibroblast growth factor 23

12 Comprehensive Development Program UX023-CL201: Phase 2 (52 children ages 5-12 yrs.) Pediatric (n=126) UX023-CL205: Phase 2 (13 children ages 1-4 yrs.) UX023-CL301: Phase 3 Burosumab vs Conventional Therapy (61 children ages 1-12 yrs.) Adult (n=68) Completed Adult Studies KRN23-US-02 KRN23-INT-001 KRN23-INT-002 KRN UX023-CL203: Phase 2b Long-term Extension (20 adults) UX023-CL303: Phase 3 Placebo-Controlled (134 adults) UX023-Data Monitoring Program (Children & Adults) UX023-CL304: Phase 3 Osteomalacia (14 adults) 12

13 Pediatric Phase 3 Study: Serum Phosphorus Levels in the Normal Range with Crysvita Treatment mg/ml, Mean ± SE Oral Phosphate/Active Vit D Crysvita 3.2 mg/dl LLN Week 13 *P < for change from baseline at Week 40 and change from baseline to Mean post-baseline (Week 1, 4, 8, 16, 24, 32 and 40)

14 Pediatric Phase 3 Study: Substantial Healing of Rickets in 72% of Crysvita Patients LS Mean ± SE Conventional Therapy Week 40 Global Score Crysvita P < Crysvita Conventional Therapy= 1.14 (95% CI 0.83, 1.45) Conventional Therapy (N=32) Substantial Healing (RGI-C +2.0) Crysvita (N=29) 2 (6.3%) 21 (72.4%) Odds Ratio Crysvita vs. Conventional Therapy: % CI: 7.238, P <

15 Pediatric Phase 3 Study: RSS Total 2.8-fold Improvement in Rickets with Crysvita Mean ± SE LS mean change ± SE ± Baseline Week 40 Baseline Week 40 Oral Conventional Phos./Vit. Therapy D LS mean change ± SE ± Burosumab Crysvita Difference (95% CI) Crysvita Conventional Therapy P-Value a (-1.74, -0.94) <

16 Pediatric Phase 3 Study: Safety Profile Generally Consistent with other Pediatric XLH Studies No treatment discontinuations; no deaths reported in the study No treatment-related serious adverse events (SAEs) 3 SAEs in the Crysvita arm (not treatment-related) 1 SAE in conventional therapy arm (not treatment-related) 45% of patients in Crysvita arm had injection site reactions, all but one were mild and none were serious No clinically meaningful changes in mean serum calcium or serum intact parathyroid hormone in either treatment arm No clinically significant changes in renal ultrasounds pre-and post-treatment in either treatment arm 16

17 Pediatric Phase 2 Studies: Normalization of Phosphate & Resolution of Rickets Biomarker Serum Phosphorus, mg/dl Alkaline Phosphatase, U/L Study UX023-CL205 Ages 1-4 (N=13) Normal range lower limit (3.2 mg/dl) Week Week Serum Phosphorus, mg/dl Alkaline Phosphatase, U/L Study UX023-CL201 Ages 5-12 (N=52) Normal range lower limit (3.2 mg/dl) Week Week Q2W (N = 11-13) 201 Q2W (n = 26) Data is presented as mean ± SE; Grey box indicate normal range

18 Pediatrics Phase 2 Studies Crysvita Improved Rickets Total Rickets Severity Score Global RGI-C Mean ± SE *** 59% 61% *** Mean ± SE Q2W (N = 13) 201 Q2W Q2W (n =26) Q2W Q2W 205 Q2W 201 Q2W 201 (N = 13) (n =26) Baseline Week 40 ***For CL201, p based on the Generalized Estimation Equation (GEE) model for the Week 64 subset. For CL205, 2-sided p-values are from the ANCOVA model (includes the change from baseline in RSS as the dependent variable, age and RSS at baseline as covariates) 18

19 Crysvita Improved Bowing After 40 Weeks: Knees closer together and legs straighter 2-year-old Boy 2-year-old Girl Baseline Week 40 Baseline Week 40 19

20 Safety Summary in Pediatrics: Acceptable Safety Over weeks of Treatment in Pediatrics CL201 (Patients Ages 5-12) CL205 (Patients Ages 1-4) 20 Most common treatment-related AE was injection-site reaction (65% of patients); all mild One SAE considered possibly treatment-related Resolved without sequelae and the patient continued in study No deaths or discontinuations No serum phosphorus levels above upper limit of normal No safety concerns associated with intact parathyroid hormone levels, serum or urinary calcium levels, renal ultrasound No patients discontinued therapy One SAE (tooth abscess; not related) Most AEs were Grade 1 or 2 Injection site reactions in 3 of 13 patients (23.1%) Hypersensitivity AEs in 4 of 13 patients (30.8%) Each not likely related to Crysvita No hyperphosphatemia All renal ultrasounds normal

21 Adults Phase 3 Study Sustained Increase in Serum Phosphorus 4 Serum Phosphorus Peak Concentrations 3.5 mg/dl Normal range lower limit (2.5 mg/dl) Week Placebo Placebo-Crysvita Crossover Crysvita Q4W 21

22 Adult Phase 3 Study: Crysvita Improved Fracture/Pseudofracture Healing Healed Active Fractures/Pseudofractures Percentage WEEK 24 Odds Ratio: % CI: 4.93, (p<0.0001) 48 WEEKS WITH CRYSVITA 63.1% Healed 16.9% Partial Healed 80% total WEEKS CROSSOVER 35.2% Healed 39.6% Partial Healed BASELINE Weeks Placebo: 91 Fx/PFx in 38 patients Crysvita: 65 Fx/PFx in 32 patients 75% total 22 Placebo Burosumab Q4W Placebo-Crysvita Crossover

23 Pseudofracture Healing in a 38-year-old Woman Treated with Crysvita Baseline Week 24 Active PFx Healed PFx 23

24 Adults: Crysvita Treatment Improves Bone Quality Bone Quality Study Histomorphometric Results 10 Paired Bone Biopsies Osteoid Volume/ Bone Volume, % Osteoid Thickness, μm Osteoid Surface/ Bone Surface, % Mineralization Lag Time, days ,000 Mean ± SE % % % 1,800 1,600 1,400 1,200 1, Baseline Week 48 24

25 Crysvita Improves Osteomalacia and Heals a Fracture in a 39-year-old Man Baseline Week 12 Week 24 Week 36 Active Fx Partially Healed Fx Partially Healed Fx Healed Fx Histomorphometry Parameters Baseline Week 48 Osteoid Volume/ Bone Volume, % Osteoid Thickness, µm Osteoid Surface/ Bone Surface, % Mineralization lag time, days

26 Crysvita Treatment Continued to Improve Stiffness at Week 48 WOMAC Stiffness Change from Baseline Placebo Numbers on graph are change from baseline 0.25 Weeks Week 24 placebo vs Crysvita p= Placebo-Crysvita Crossover Week 48 change from baseline (Week 24 for placebo) both p< Crysvita Q4W 16

27 Crysvita Treatment Continued to Improve Physical Functioning at Week 48 WOMAC Physical Function Change from Baseline Week 24 placebo vs Crysvita p= Week 48 change from baseline (week 24 for placebo) placebo p<0.001 and Crysvita p= Numbers on graph are change from baseline Weeks Placebo Placebo-Burosumab Crossover Burosumab Q4W 17

28 Crysvita was Associated with a Reduction in Pain 0.5 BPI Worst Pain Change from Baseline Week 24 placebo vs Crysvita p=0.09 Week 48 change from baseline (week 24 for placebo) p< Weeks Placebo Crossover Crysvita 28 Data expressed as mean ± SE

29 Adults: Safety Summary Most common adverse events (>10%) All Patients, n (%) (Combined DB + OL Period; N = 134) Arthralgia 32 (23.9) Nasopharyngitis 30 (22.4) Headache 27 (20.1) Back pain 22 (16.4) Tooth abscess 18 (13.4) Fatigue 18 (13.4) Pain in Extremity 15 (11.2) Hypersensitivity 15 (11.2) Pain 15 (11.2) Toothache 15 (11.2) Muscoloskeletal Pain 14 (10.4) Vitamin D deficiency 14 (10.4) 29 AE profile similar to that previously observed at Week Serious Adverse Events; none treatment-related Improvement (decrease) overall in ipth over time No meaningful changes in ectopic mineralization

30 Disease Burden Survey in Adult XLH Patients Online survey of 165 adults in 13 countries Adults can experience progressively debilitating complications that cause pain and negatively impact functional independence, quality of life despite long-term treatment in childhood with conventional therapy Majority of patients receiving treatment 69% receiving current treatment 50% receiving oral phosphate/vitamin D 71% taking medication for bone or joint pain > 1x/wk 47% had a bone fracture Significant pain, stiffness, physical functioning impairment Bone pain (75%), joint pain (90%), joint stiffness (87%) Restricted range of motion, gait disturbance, muscle pain/weakness (70%) WOMAC pain, stiffness, physical function scores well below normal Calcification outside the bone was commonly reported Osteophytes (46%); enthesopathy (32%); nephrocalcinosis (23%); spinal stenosis (23%); kidney stones (16%) 30

31 UX007 for Long-Chain Fatty Acid Oxidation Disorders* (LC-FAOD) Phase 2 substrate replacement therapy (oral liquid) *Includes VLCAD, LCHAD, CPT-I, CPT-II, TFP, CACT 31

32 UX007 for LC-FAOD Triglyceride of C7: alternative energy source to long-chain fat LC-FAOD: Inability to convert fat into energy MITOCHONDRIAL ENZYME DEFICIENCIES Key symptoms/prognosis: Hypoglycemia, muscle rupture, heart failure Mortality of ~50% 1 ; a cause of SIDS (newborn screened in US) Standard of care: Diet and MCT 2 oil US prevalence: ~2,000 3,500 Status: FDA accepted proposal to submit NDA Pre-NDA meeting with FDA in 2H18 Krebs cycle 1 J Inherit Metab Dis 2013;36: Medium chain triglycerides 32

33 Phase 2 Data Showed Reduction in Frequency and Duration of Major Clinical Events ALL MAJOR CLINICAL EVENTS (MCEs), N=29 Overall annualized event rate Overall annualized duration rate Mean 48.1% * 50.3% ** Median 50.6% 76.7% * p= ** p= Patient 98.4% REDUCTION IN HYPOGLYCEMIA EVENTS Hospitalization Medical Intervention Days Left of Day 0 shows events in 78 weeks pre-ux007 treatment; Right of Day 0 shows events during UX007 treatment 33

34 Safety Profile as Expected from Prior Studies 5/29 patients discontinued 1 due to diarrhea in wk 1 and considered treatment related 4 withdrew consent (wks 1, 8, 8, 78) for reasons not attributed to UX patients (66%) with treatment-related adverse events (most were mild-to-moderate in nature) Most common treatment-related adverse events were GI-related: diarrhea, abdominal/gi pain, and vomiting Some GI events were managed by adjusting dosing or dosing with food No deaths and 1 treatment related SAE of moderate gastroenteritis with vomiting 34

35 UX007 for Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) Phase 3 substrate replacement therapy (oral liquid) 35

36 UX007 for Glut1 Deficiency Syndrome Alternative energy source for the brain Mechanism: Glucose transport defect causes brain energy deficiency Less Glucose Uptake (red color) in Glut1 DS Key symptoms: Seizures, movement disorder, developmental delay Standard of care: Ketogenic diet (70-80% of calories in fat, <10% carbs) U.S. prevalence: ~3,000 7,000 Clinical status: Phase 3 movement disorder study enrollment complete (44 patients); data expected 2H18 Normal Glucose Uptake Glut1 DS Glucose Uptake 36 Ann Neurol 2002

37 Glut1 DS Movement Disorder Ph3 Study Design STUDY DESIGN KEY ENDPOINTS Runin Titration Period Titration Period UX007 Placebo Wash-out Titration Period Titration Period Placebo UX007 UX007 OLE Extension Frequency of disabling paroxysmal movement disorder events: daily electronic diary Week Children and Adults with Glut1 DS 44 patients (1:1 randomization) Experiencing disabling movement disorder events Not typically on KD Duration of disabling paroxysmal movement disorder events: daily electronic diary 37

38 Positive Data in Glut1 DS Movement Disorders Substantial reduction in paroxysmal events 1 Investigator-sponsored trial in six Glut1 DS patients with non-epileptic paroxysmal manifestations Other improvements observed Reduction in duration of motor events Clinical global impression scale Normalization of induction of brain energy metabolism by MRI spectroscopy UX007 was well tolerated Disabling motor events were substantially reduced to a similar extent as the overall total movement events 1 Mochel, J Neurol Neurosurg Psychiatry, Nov Paroxysmal dyskinesia/month % REDUCTION IN PAROXYSMAL MANIFESTATIONS * * * Baseline Treatment Withdrawal Patients completed comprehensive diaries recording all motor and other events during baseline, treatment, and withdrawal phases, which each lasted two months * Treatment

39 DTX301 Program for Ornithine Transcarbamylase (OTC) Deficiency Phase 1/2 study of adeno-associated virus serotype AAV8 vector encoding human OTC

40 DTX301 AAV8 for OTC Deficiency AAV8 gene therapy for stable expression of OTC OTC Deficiency: X-linked urea cycle disorder, genetic defect in ammonia detoxification Key symptoms/prognosis: Acute hyperammonemic episodes that can lead to hospitalization, adverse cognitive & neurological effects, death Treatment limited; only curative approach is liver transplantation WW prevalence: ~10,000, 80% late-onset Status: Cohort 2 ongoing; data expected 2H18 Bowing CHILDREN 40

41 DTX301 AAV8 for OTC Study Design Study Phase Phase 1/2 Study Design Dosing Study Population Sample Size Open-label, multicenter, safety and dose-finding study to determine the safety, tolerability, and efficacy Single intravenous infusion Cohort 1: ^12 GC/kg Cohort 2: ^12 GC/kg Cohort 3: ^13 GC/kg Adults with late-onset OTC deficiency Approximately 6 to 12 patients enrolled sequentially into cohorts 3 patients enrolled in Cohort 1 3 patients will be enrolled in each subsequent cohort Follow-up Period Patients will be followed for 52 weeks after dosing 4-year extension study planned to evaluate the long-term safety and efficacy of DTX301 41

42 Cohort 1: Favorable Initial Safety Profile Safety Summary No infusion-related adverse events No SAEs to date All AE severity graded as 1 or 2 All AEs resolved and unrelated to drug except for elevated liver enzymes (probable relatedness, resolved) Patient 1 Completed 24 weeks Mild elevated liver enzymes between Weeks 4 and 6 (peak 45 IU/L) ALTs returned to normal* after tapering course of corticosteroids 8 AEs (Grade 1 or 2) Patient 2 Completed 20 weeks Mild elevated liver enzymes at Day 36 (peak 118 IU/L) ALTs returned to normal following tapering course of corticosteroids Patient 3 Completed 12 weeks 1 unrelated AE (Grade 1) 42 * Normal ALT levels <40 IU/L

43 Patient 1: Normalization of Ureagenesis and Clinical Stability off Medications Rate of Ureagenesis % of Normal Ureagenesis Activity Prednisone % Normal Ureagenesis ALT (U/L) ALTULN Ureagenesis Screen D0 D1 D5 D9 W2 D18 D22 W4 D32 D36 W6 D46 D50 D54 D58 D62 D66 W10 D74 D78 W12 W16 D126 W20 Na Phenylbutyrate and Na Benzoate discontinued 16 Feb Na Phenylbutyrate 2018 and Na Benzoate discontinued 16 Feb 2018 D154 W24 W Alanine Aminotransferase (U/L) / Prednisone (mg/day) Baseline 200 umol/kg/hr 6 weeks 67% increase from baseline to 335 umol/kg/hr 12 weeks 30% increase from baseline to 261 umol/kg/hr 20 weeks 36% increase from baseline to 273 umol/kg/hr 24 weeks 100.8% increase from baseline to 402 umol/kg/hr Latest ureagenesis at week 36 shows stable effect from Wk24 Per-protocol, all patients remain on prescribed protein restricted diet 36 weeks 94.3% increase from baseline to 389 umol/kg/hr

44 Patients 2 and 3: Ureagenesis and ALT Levels Activity (% of Normal) ALT (U/L) ALT (U/L) % Normal Ureagenesis vs. ALT Patient 2 Patient 3 Activity (% of Normal) 44

45 DTX301 Timeline Cohort 2 Data 2H H 1H 2H Cohort 1 Cohort 2 DMC DMC KEY UPDATES Cohort 2 ongoing Cohort 2 data expected in 2H18 45

46 DTX401 AAV8 for GSDIa GSDIa: Autosomal recessive, inborn error of glucose metabolism; deficient glucose-6-phosphatase (G6Pase) Key symptoms/prognosis: Hypoglycemia leading to significant morbidity and mortality Long-term liver and renal disease Impaired growth and delayed puberty Severe long-term complications in 70-80% patients Limited Treatment Option: Raw cornstarch Glucose GSD I GLYCOGEN Gluc-6-Phos Pyruvate Uric Acid Lactate Prevalence: 6,000 WW Status: First patient dosed July 2018 Cohort 1 data 2H18 Krebs Cycle Acetyl CoA Lipids 46

47 DTX401 AAV8 for GSDIa Second Gene Therapy Program Ongoing H 2H Cohort 1 IND Filing First Patient Dosed DMC KEY UPDATES First patient in cohort 1 dosed July 2018 Cohort 1 data in 2H18 47

48 48 Finance and Business Summary

49 Financial Overview Cash 1 (as of June 30, 2018): $547.1 million Total Revenue (quarter ended June 30, 2018): $12.8 million Operating loss (six months ended June 30, 2018): $22.5 million Cash used in operations (six months ended June 30, 2018): $165.6 million No debt (as of June 30, 2018) 1 Cash, cash equivalents, and investments 49

50 Investment Thesis Diverse, robust company Able to execute Four modalities to optimize treatment of rare diseases Two approved therapies 14+ programs spanning preclinical to commercial Deep domain expertise and high-quality team Rapid execution and innovation in clinical and regulatory strategy Capital-efficient well resourced business model Commercial-stage with global reach 50

51 51 Appendix

52 License and Collaboration with KHK Ultragenyx leads development with costs shared 50/50 US AND CANADA EUROPE LATIN AMERICA Commercialization Ultragenyx launches KHK books sales 50/50 profit share for 5 years then tiered revenue share Shared commercial activities over time KHK commercializes and books sales Ultragenyx commercializes and books sales Royalties After 5 years, tiered revenue share in mid to high 20% range to Ultragenyx after profit share period Up to 10% royalty to Ultragenyx Low single-digit royalty to KHK Commercial supply KHK supplies; price is doubledigit percentage of net sales NA KHK supplies; price is double-digit percentage of net sales 52

53 Key Licenses & Intellectual Property Product License Intellectual Property/Royalties Burosumab (XLH, TIO) Vestronidase Alfa (MPS 7) UX007 (LC-FAOD) UX007 (Glut1 DS) DTX301 (OTC Deficiency) DTX401 (GSDIa) KHK Shared rights to US patents to generic and specific antibodies and use for treatment of XLH ( ) 1 See summary of collaboration St. Louis University Composition and use for treatment of MPS 7 (2035) Low single-digit royalty Baylor Research Institute (BRI) Composition ( ) 2 Use for treatment of LC-FAOD (2020) Mid single-digit royalty BRI & INSERM Composition ( ) 2 Use for treatment of Glut1 DS associated movement disorders (2033) Mid single-digit royalty Sub-License from REGENXBIO of UPENN IP Sub-License from REGENXBIO of UPENN IP Composition and use for treatment of OTC Deficiency ( ) Low to mid single-digit royalty Composition for treatment of GSD1a ( ) Low to mid single-digit royalty Additional internal IP in progress 1 Without patent term extension of up to fourteen years from approval 2 Without patent term extension of up to five years

54 Burosumab MAb Against FGF23 for TIO TIO: Benign tumors cause excess FGF23 and hypophosphatemia MULTIPLE FRACTURES Severe XLH-like disease: Severe osteomalacia, fractures, pain, muscle weakness Standard of care: Resection of tumors (~50% of cases); oral phosphate US prevalence: ~500-1,000 Clinical Status: Phase 2 data second half 2018 (Front) BONE SCAN (Back) 54

55 Phase 2 Study in TIO Screening TC admin/ Bone biopsy Burosumab Treatment Period: 48 weeks Q4W SC administration n ~ 17 TC admin/ Bone biopsy Extension Period 2 years CO-PRIMARY ENDPOINTS Proportion achieving mean serum phosphorus levels above LLN Percent change at 48 wks vs. baseline in excess osteoid based on bone biopsy indices 55

56 Positive Interim Results at 24 Weeks Improved serum phosphorus levels; bone effect observed Mean serum phosphorus levels entered normal range and maintained Significant increase in bone turnover markers P1NP: 51% increase CTx: 38% increase Burosumab improved histomorphometric indices of osteomalacia 3 of 4 patients with bone biopsy data showed improvements; 1 patient did not receive burosumab consistently Statistically significant improvement in PROs Reduction in fatigue Increase in lower limb strength Treatment-related AEs in 7 patients, all mild. Non treatment-related SAEs in 3 patients; and all had a history of tumor progression at baseline 56