Ultragenyx Pharmaceutical Inc. Q1 FY2025 earnings call

Commercial Team Execution

• Crysvita in Latin America generated ~40 new start forms leading to ~40 reimbursed patients, with ~775 patients on commercial product; growth expected to continue with reimbursement negotiations in Brazil and Mexico. In the U.S., Kyowa Kirin leads Crysvita commercialization, with Q1 2025 revenue supported by increasing new starts and patients. Dojolvi in the U.S. had ~30 new start forms and ~25 new reimbursed patients since launch in 2020, with ~600 total reimbursed patients; over 260 patients treated in EMEA via named patient sales. Evkeeza in EMEA has ~250 patients on reimbursed therapy across 15 countries, with ongoing country-by-country pricing negotiations; in Japan, building on launch momentum; in Canada, negotiating with health authorities.

Development Pipeline Updates

• UX143 in osteogenesis imperfecta: Phase 3 ORBIT study patients enrolled for at least a year, databases being cleaned/locked for second interim analysis. GTX-102 for Angelman syndrome: Phase 3 enrolling efficiently in multiple countries, data expected in 2026. DTX301 for OTC: Phase 3 study completed enrollment, on track to read out data in next year. UX701 for Wilson disease: Enrolling fourth dose finding cohort for dose selection. BLAs: DTX401 BLA submission on track for mid-2025; UX111 BLA under review by FDA progressing on schedule with mid-cycle review meeting on track, PDUFA action date August 18.

Manufacturing and Regulatory

• Manufacturing facility in Bedford, Massachusetts qualified after PPQ runs. Regulatory interactions on UX111 BLA remain on track with mid-cycle review and inspections ongoing.

In the first quarter of 2025, Ultragenyx reported revenue of $139 million, representing a 28% growth over the first quarter of 2024. Crysvita contributed $103 million, with $41 million from North America, $55 million from Latin America and Turkey, and $7 million from Europe, showing 25% growth over 2024; Latin America and Turkey specifically had 52% growth. Dojolvi contributed $17 million, consistent with its steady growth trajectory. Evkeeza contributed $11 million as demand builds outside the U.S. Mepsevii contributed $8 million, treating patients in an ultra-rare indication.

Revenue Guidance for 2025

• Total revenue expected to be between $640 million and $670 million, representing 14% to 20% growth over 2024. Crysvita revenue expected to be between $460 million and $480 million, 12% to 17% growth over 2024. Dojolvi revenue expected to be between $90 million and $100 million, 2% to 14% growth over 2024. Tariffs: No material exposure expected for products including Crysvita.

• CBER nomination: Uncertain impact on rare disease approvals, but Ultragenyx's UX111 program has substantial clinical data. - Regulatory uncertainties: Variation in trial results and potential delays in regulatory processes, though UX111 BLA review is on track. - Trial variation: Variability in patient fracture rates and other baseline characteristics in OI trials could impact study outcomes.

Q: Yaron Werber from TD Cowen asked about dispersion in the UX143 study and fracture reduction at 14 months.

A: Emil Kakkis explained about variation in baseline fracture rates, stratification by age, and that at 14 months, there was a 67% reduction in fracture frequency median with a P value of 0.0014.

Q: Tazeen Ahmad from Bank of America inquired about the likelihood of success if the study moves to a third interim.

A: Emil Kakkis stated confidence in hitting the study's goal either at the second interim or the end, citing variation but strong positive outlook.

Q: Gena Wang from Barclays asked about CBER nomination impact and washout period in UX143.

A: Emil Kakkis noted concerns about CBER nomination but confidence in UX111's data, and on washout period, explained that placebo patients' fracture rate decline is less impactful than setrusumab's effect.

Q: Lydia Erdman from Goldman Sachs asked about messaging and timing of UX143 data.

A: Emil Kakkis said data release timing depends on DMC results, with faster clean/lock and data disclosure expected compared to prior interim.

Q: Malcolm Kuno from JPMorgan asked about enrollment in the Angelman program.

A: Eric Crombez responded that they are fully enrolling the study globally with active sites.

Q: Kristen Kluska from Cantor asked about age baseline in UX143.

A: Emil Kakkis said majority of patients are pediatric with limited older patients included for adult labeling.

Q: Yigal Nochomovitz from Citi asked about OI type distribution and P value tolerance.

A: Emil Kakkis discussed OI type distribution in the study and that a P value less than 0.01 is needed for success at the second interim, with a reasonable chance of hitting the 0.01 threshold.

Q: Joseph Schwartz from Leerink asked about Angelman market evolution.

A: Emil Kakkis stated confidence in their ASO being the leader, citing long-term data and patient progress.

Q: Liisa Bayko from Evercore ISI asked about UX143 data timing and disclosure.

A: Emil Kakkis explained that clean/lock of the database takes ~8 weeks, with sooner top line data expected than prior interim.

Q: Luca Issi from RBC Capital asked about UX143 PRV and sunset.

A: Emil Kakkis said they have PIG designation and aim to get approved by October 2026, with confidence in being a leader in OI.

Q: Mehdi Goudarzi from Evercore ISI asked about setrusumab's MOA on OI types.

A: Emil Kakkis explained setrusumab benefits all OI types as it strengthens bones regardless of collagen mutation.

Q: Maury Raycroft from Jefferies asked about effect size range for UX143 success.

A: Emil Kakkis discussed clinically meaningful AFR reduction as at least 30%, with slopes linear and separation occurring within 2-3 months.

Q: Dylan from Wedbush asked about Crysvita growth drivers in LatAm.

A: Emil Kakkis and Eric Crombez mentioned word of mouth, doctors' positive feedback on patient outcomes, and reimbursement in Brazil and Mexico driving growth.

Q: Jack Allen from Baird asked about UX143 patient baseline and pain impact.

A: Emil Kakkis explained majority of patients had less than a year of exposure at first interim, and Eric Crombez noted pain is a key focus in evaluating the trial, with improvement seen in Phase 2 data.