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Ultragenyx Pharmaceutical Inc. (RARE)·Q1 2025 Earnings Summary

Executive Summary

  • Q1 2025 revenue was $139.3M, up 28% year over year; EPS was ($1.57), an improvement from ($2.03) in Q1 2024. Segment detail: Crysvita $102.9M (+25% YoY), Dojolvi $17.0M, Evkeeza $11.0M, Mepsevii $8.4M .
  • Versus S&P Global consensus, revenue missed ($139.3M vs $144.5M*), while EPS beat (($1.57) vs ($1.65)). EBITDA also beat (actual ($133.9M) vs consensus ($139.0M)) . Values marked with * retrieved from S&P Global.
  • Full-year 2025 guidance reaffirmed: total revenue $640–$670M; Crysvita $460–$480M; Dojolvi $90–$100M; expected 14–20% YoY revenue growth and reduced operating cash burn versus 2024 .
  • Near-term catalysts include UX111 priority review with PDUFA on Aug 18, 2025 and UX143 (setrusumab) Phase 3 Orbit/Cosmic interim analyses in mid-2025, both cited as on track by management .

What Went Well and What Went Wrong

What Went Well

  • Strong commercial execution: Crysvita revenue rose 25% YoY to $102.9M, with Latin America & Türkiye product sales up 52% YoY to $55.1M; Evkeeza reached $11.0M as launches outside the U.S. progressed .
  • Guidance confidence and cash runway: FY25 revenue guidance reaffirmed and management expects reduced net cash used in operations in 2025; quarter-end cash, equivalents, and marketable debt securities were $563.0M .
  • Pipeline momentum: UX111 BLA mid-cycle review completed; inspections underway; UX143 databases being cleaned for IA2; DTX401 BLA filing targeted mid-2025 after successful PPQ runs .

What Went Wrong

  • Top-line miss vs consensus: Q1 revenue of $139.3M missed S&P Global consensus ($144.5M*), driven in part by uneven ordering patterns (management cautions on LatAm variability) . Values marked with * retrieved from S&P Global.
  • Operating intensity still high: Total operating expenses were $282.2M, up vs Q3 2024 and only modestly lower than Q4 2024; operating loss was ($142.9M) .
  • Net cash used in operations was $166M for Q1 (seasonally higher due to bonuses and milestone payments), underscoring continued cash consumption prior to anticipated pipeline inflections .

Financial Results

Quarterly Performance vs Prior Periods

MetricQ3 2024Q4 2024Q1 2025
Revenue ($USD Millions)$139.5 $164.9 $139.3
Net Loss ($USD Millions)($133.5) ($133.4) ($151.1)
EPS (Basic & Diluted, $USD)($1.40) ($1.39) ($1.57)
Loss from Operations ($USD Millions)($132.0) ($122.3) ($142.9)

Year-over-Year and Quarter-over-Quarter

ComparisonRevenueEPSNet Loss
Q1 2025 vs Q1 2024+28.0% (139.3 vs 108.8) Improved to ($1.57) vs ($2.03) Improved to ($151.1) vs ($170.7)
Q1 2025 vs Q4 2024-15.5% (139.3 vs 164.9) Worsened to ($1.57) vs ($1.39) Worsened to ($151.1) vs ($133.4)

Margins

Margin MetricQ3 2024Q4 2024Q1 2025
EBIT Margin % (Loss from Ops / Revenue)-94.7% -74.2% -102.6%
Net Income Margin % (Net Loss / Revenue)-95.7% -80.9% -108.4%

Q1 2025 Actual vs S&P Global Consensus

MetricActualConsensusSurprise
Revenue ($USD Millions)$139.3 $144.5*-$5.2M (miss)*
EPS ($USD)($1.57) ($1.65)*+$0.08 (beat)*
EBITDA ($USD Millions)($133.9) ($139.0)*+$5.1M (beat)*

Values marked with * retrieved from S&P Global.

Segment Breakdown

SegmentQ1 2024 ($USD Thousands)Q1 2025 ($USD Thousands)
Crysvita – Product sales (LatAm & Türkiye)$36,241 $55,080
Crysvita – Royalty (U.S. & Canada)$40,402 $40,853
Crysvita – Royalty (Europe)$5,942 $6,932
Total Crysvita$82,585 $102,865
Dojolvi$16,362 $17,009
Evkeeza$3,275 $11,031
Mepsevii$6,611 $8,387
Total Revenues$108,833 $139,292

KPIs (Commercial Execution)

KPIQ1 2025
Crysvita (LatAm) new start forms → reimbursed therapy~40 forms → ~40 patients
Crysvita patients on commercial product (LatAm)~775 patients
Dojolvi new start forms / new reimbursed patients~30 / ~25
Dojolvi total patients since 2020~600
Dojolvi unique prescribers (U.S.)~270
Dojolvi treated (EMEA, named patient)>260 patients; majority in France; growing Middle East requests
Evkeeza treated (EMEA & JP & CA progress)~250 patients; +50 YTD; 15 countries; JP building; CA private payers secured

Guidance Changes

MetricPeriodPrevious Guidance (as of Q4 2024)Current Guidance (Q1 2025)Change
Total RevenueFY 2025$640–$670M $640–$670M Maintained
Crysvita RevenueFY 2025$460–$480M $460–$480M Maintained
Dojolvi RevenueFY 2025$90–$100M $90–$100M Maintained
YoY Revenue GrowthFY 2025~14–20% ~14–20% Maintained
Net Cash Used in OperationsFY 2025Lower vs 2024 Lower vs 2024 Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q3 2024, Q4 2024)Current Period (Q1 2025)Trend
UX143 (OI)BTD granted; Phase 3 progressing to IA2 mid-2025 Databases being cleaned; IA2 in mid-2025; detailed discussion of p-value thresholds and variability Building confidence; execution on track
UX111 (MPS IIIA)Pre-BLA meeting success; BLA filed end-2024 Priority Review; mid-cycle completed; inspections underway; PDUFA Aug 18, 2025 On-schedule regulatory review
DTX401 (GSDIa)Positive Phase 3; crossover ~62% reduction; planning BLA mid-2025 PPQ completed; stronger clinical/CMC package; BLA mid-2025 Steady toward filing
UX701 (Wilson)Stage 1 activity; responders tapered SOC; cohort 4 planned Cohort 4 enrolling at 4e13; enhanced immunomodulation; Stage 2 protocol amended (open-label, active control) Optimizing to broaden response
Dojolvi commercialGrowth in U.S. & EMEA named patient; PMDA CEA alignment (Japan) New start forms; prescriber base growing; >260 EMEA patients; JP continuing launch momentum Solid, steady growth
Tariffs/MacroNot highlightedCFO monitoring; no material exposure expected currently Neutral

Management Commentary

  • “Our commercial team delivered a strong quarter that puts us in a position to have another year with meaningful revenue growth… we are preparing to launch our next set of programs” — Emil Kakkis (CEO) .
  • “Crysvita contributed $103 million, including $41 million from North America, $55 million from Latin America and Turkey and $7 million from Europe… Evkeeza contributed $11 million” — Howard Horn (CFO) .
  • “Our interaction with the FDA on the UX111 BLA review thus far remain on track… mid-cycle review meeting… inspections… on track for the PDUFA action date of August 18” — Emil Kakkis .
  • “We recently successfully completed our process performance qualification runs… an even stronger clinical and CMC filing package… submit to the FDA midyear” — Eric Crombez (CMO) on DTX401 .

Q&A Highlights

  • Setrusumab IA2 and statistics: Management detailed variability considerations, negative binomial approach, and strict p-value thresholds (IA2 at <0.01; final at <0.04); if IA2 narrowly misses, study continues without revealing p-value .
  • Regulatory environment: Discussion on CBER leadership dynamics; management remains confident in UX111 given clinical efficacy data beyond biomarkers .
  • Angelman (GTX-102) competitive landscape and enrollment: Expect full pivotal enrollment in 2025; management argues for potency and long-term data differentiation vs peers .
  • Commercial drivers: LatAm Crysvita growth propelled by reimbursement in Brazil/Mexico and physician experience; adult uptake strengthening .

Estimates Context

  • Q1 2025 vs consensus: Revenue $139.3M vs $144.5M* (miss); EPS ($1.57) vs ($1.65)* (beat); EBITDA ($133.9M) vs ($139.0M)* (beat). Values marked with * retrieved from S&P Global.
  • Near-term consensus trajectory: Q3 2025 revenue $166.8M*, EPS ($1.24); Q4 2025 revenue $188.0M, EPS ($1.21); Q1 2026 revenue $172.4M, EPS ($1.43)*. Values retrieved from S&P Global.
  • With guidance maintained and upcoming clinical/regulatory catalysts, revisions will likely hinge on IA2 outcome and PDUFA progress, with Evkeeza ramp as a secondary tailwind .

Key Takeaways for Investors

  • Mixed print with a top-line miss but EPS/EBITDA beats; underlying demand remains strong, particularly in LatAm Crysvita, while management reiterates FY25 revenue and segment guidance . Values vs consensus retrieved from S&P Global.
  • UX111 PDUFA (Aug 18, 2025) and UX143 IA2 mid-2025 are the principal stock catalysts; both are reported on schedule with proactive data/CMC preparation .
  • Expect quarter-to-quarter revenue variability, especially in LatAm Crysvita ordering patterns; but trajectory points to sustained growth as reimbursement expands .
  • Cash of $563.0M and anticipated lower FY25 operating cash usage support execution through key launches/readouts; watch milestone payments’ impact on quarterly cash flows .
  • Evkeeza outside-U.S. ramp (EMEA, JP) is becoming more meaningful and diversifying revenue base; named-patient dynamics in EMEA add upside as pricing matures .
  • Regulatory clarity: management disclosed mid-cycle completion and inspections underway for UX111; confidence anchored in clinical endpoints and biomarker correlation .
  • Trading stance: IA2 outcome and any top-line disclosure will likely drive near-term volatility; positive interim would reset probability of success for setrusumab and could prompt estimate/target price revisions.

Notes: All consensus estimate values marked with * are retrieved from S&P Global.