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Ultragenyx Pharmaceutical Inc. (RARE)·Q3 2025 Earnings Summary

Executive Summary

  • Q3 2025 revenue was $159.93M (+15% YoY), below consensus ($166.79M*) and prior quarter; EPS was -$1.81 vs consensus -$1.24*, driven by expected seasonality in North American Crysvita royalties and elevated pre-launch manufacturing spend .
  • 2025 guidance reaffirmed: Total revenue $640–$670M; Crysvita $460–$480M; Dojolvi $90–$100M; management reiterated path to full-year GAAP profitability in 2027 .
  • Balance sheet bolstered by $400M non-dilutive OMERS royalty financing on North America Crysvita (payments begin Jan 2028; cap 1.55x purchase price), reducing near-term dilution risk and funding up to four launches .
  • Near-term catalysts: UX143 (OI) Phase 3 Orbit+Cosmic topline around Dec/Jan; DTX401 (GSDIa) rolling BLA completion in Q4 2025—both key stock drivers alongside Angelman GTX-102 progress .

What Went Well and What Went Wrong

What Went Well

  • Crysvita and broader portfolio continue to show durable growth: Crysvita revenue $111.94M; Dojolvi $24.28M; Evkeeza $16.72M; Mepsevii $6.99M in Q3 .
  • Commercial execution: ~50 new Crysvita start forms in Latin America, ~875 patients on reimbursed therapy; Evkeeza expanded to ~310 patients across 17 countries YTD .
  • Strategic financing and long-term focus: “We took an important step to strengthen our financial position, receiving $400 million of non-dilutive capital… Payments to OMERS will start in January 2028” — CEO Emil Kakkis .

What Went Wrong

  • Revenue miss vs consensus and sequential decline, largely due to North America Crysvita royalty seasonality; management nonetheless expects strong Q4 royalty revenue .
  • Elevated R&D and pre-launch manufacturing spend: total OpEx $330.82M; CFO highlighted $87M pre-launch inventory manufacturing and R&D $216M for the quarter .
  • Cash burn: net cash used in operations was $91M in Q3 (YTD $366M); cash/cash equivalents $447M pre-OMERS financing .

Financial Results

MetricQ1 2025Q2 2025Q3 2025
Revenue ($USD Millions)$139.29 $166.50 $159.93
Revenue vs Consensus ($USD Millions)-$5.16*+$5.13*-$6.85*
EPS ($USD)$-1.57 $-1.17 $-1.81
EPS vs Consensus ($USD)+$0.08*+$0.13*-$0.57*
EBITDA Margin (%)-96.16%*-59.62%*-101.47%*
EBIT Margin (%)-102.62%*-64.80%*-106.85%*
Net Income Margin (%)-108.46%*-69.04%*-112.81%*

Notes: Consensus from S&P Global; beats/misses bolded in narrative. Values retrieved from S&P Global.*

Segment revenue breakdown:

Segment Revenue ($USD Millions)Q1 2025Q2 2025Q3 2025
Crysvita – Product sales (LatAm & Türkiye)$55.08 $34.73 $47.00
Crysvita – Royalty (U.S. & Canada)$40.85 $79.08 $57.19
Crysvita – Royalty (Europe)$6.93 $6.60 $7.75
Total Crysvita$102.87 $120.41 $111.94
Dojolvi$17.01 $23.21 $24.28
Evkeeza$11.03 $14.57 $16.72
Mepsevii$8.39 $8.31 $6.99
Total Revenues$139.29 $166.50 $159.93

Operational and P&L details:

Metric ($USD Thousands unless noted)Q1 2025Q2 2025Q3 2025
Cost of Sales$28,662 $23,002 $27,991
R&D Expense$165,772 $164,736 $216,212
SG&A Expense$87,797 $86,646 $86,620
Total Operating Expenses$282,231 $274,384 $330,823
Net Loss$(151,080) $(114,951) $(180,413)
EPS (basic & diluted)$-1.57 $-1.17 $-1.81
Cash, cash equivalents & marketable securities (period end)$563,003 $539,039 $447,315
Net Cash Used in Operations (period)$(166,000) $(108,000) $(91,000)

KPI highlights:

KPIQ1 2025Q2 2025Q3 2025
Crysvita LatAm: New start forms~40 ~50 ~50
Crysvita LatAm: Patients on reimbursed therapy~775 ~825 ~875
Dojolvi US: Patients on reimbursed therapy~600 ~600 ~625
Evkeeza: Patients on reimbursed therapy (EMEA)~250 ~285 ~310

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Total RevenueFY 2025$640–$670M $640–$670M Maintained
Crysvita RevenueFY 2025$460–$480M $460–$480M Maintained
Dojolvi RevenueFY 2025$90–$100M $90–$100M Maintained
Net Cash Used in OperationsFY 2025Decrease vs 2024 Modestly increase vs 2024 Raised (cash use)
GAAP ProfitabilityFY 2027Path reaffirmed Path reaffirmed Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q1 & Q2)Current Period (Q3)Trend
Balance sheet & financingNo OMERS deal; discussed cash and ATM; path to 2027 GAAP profitability $400M OMERS royalty financing; payments start 2028; cap 1.55x; option to repurchase Strengthened
UX143 (OI) Phase 3IA2 did not stop; final read expected end of year; confidence in efficacy; negative binomial stats explained Topline expected Dec/Jan; both Orbit & Cosmic together; fracture reduction 40–70% clinically meaningful; superiority vs bisphosphonates discussion On track
GTX-102 (Angelman)BTD granted; ASPIRE fully enrolled; AURORA planned First AURORA patient dosed; ASPIRE data expected 2H26 Advancing
DTX401 (GSDIa)BLA targeted mid-year; PPQ completed Rolling BLA underway; completion expected Q4; 96-week data: ~61% cornstarch reduction; QoL benefits Advancing
UX111 (MPS IIIA)BLA accepted; Priority Review with Aug 18 PDUFA CRL; constructive FDA interactions; plan to resubmit early 2026; up to 6-month review Delayed
Commercial executionCrysvita LatAm growth; Evkeeza Japan/EMEA ramp; Dojolvi steady Continued global growth; Evkeeza ~310 patients; Crysvita seasonality in NA Mixed (steady growth; seasonal variability)

Management Commentary

  • “We received $400 million of non-dilutive capital… Payments to OMERS will start in January 2028… Adding $400 million to our balance sheet will help us deliver on our expected launches, setting us up for our next stage of growth and on our path to profitability in 2027.” — CFO Howard Horn .
  • “Data from the phase III [UX143] studies are on track to read out around the end of the year, which to us means December or January.” — CEO Emil Kakkis .
  • “In the third quarter, we reported $160 million… Crysvita contributed $112 million… Dojolvi $24 million… Evkeeza $17 million… Mepsevii $7 million.” — CFO .
  • “The commercial organization is continuing to successfully launch four products across the globe… approximately 875 Crysvita patients [LatAm]… approximately 310 Evkeeza patients across 17 countries.” — CCO Eric Harris .

Q&A Highlights

  • OI data timing and scope: Both Orbit and Cosmic to be disclosed together; December or January timing due to database cleaning; confidence across age ranges and fracture reduction .
  • OMERS financing & runway: $400M proceeds extend runway; PRV monetization assumptions remain part of 2027 profitability path; aggregate levers to reach profitability .
  • Treatment paradigm: “Bisphosphonates are going to become obsolete” in OI; setrusumab seen as chronic therapy to maintain high-quality bone; key KOL adoption likely in severe/younger patients .
  • DTX401 clinical significance: Durable 96-week reductions in cornstarch (61% mean) and QoL improvements (PGIC 83–95%) underscore clinical value; rolling BLA completion targeted in Q4 .

Estimates Context

  • Q3: Revenue $159.93M vs $166.79M consensus (miss); EPS -$1.81 vs -$1.24 consensus (miss)*.
  • Q2: Revenue $166.50M vs $161.37M consensus (beat); EPS -$1.17 vs -$1.30 consensus (beat)*.
  • Q1: Revenue $139.29M vs $144.46M consensus (miss); EPS -$1.57 vs -$1.65 consensus (beat).
    Analyst participation: EPS estimates (Q3: 19; Q2: 19; Q1: 15); Revenue estimates (Q3: 16; Q2: 18; Q1: 17)    .

Values retrieved from S&P Global.*

Key Takeaways for Investors

  • Near-term event path: UX143 OI data in Dec/Jan is the primary stock driver; superiority and functional benefits vs bisphosphonates will shape adoption and label; management believes a 40–70% fracture reduction is clinically meaningful .
  • Financing de-risks dilution: $400M OMERS deal (payment holiday to 2028; capped) supports multiple potential launches and preserves strategic flexibility for PRV monetization .
  • Q3 miss largely seasonal and investment-driven: Seasonality in North America Crysvita royalties and pre-launch manufacturing expenses weighed on results; management guides to stronger Q4 royalties .
  • DTX401 is advancing toward filing: Compelling 96-week efficacy and QoL data underpin rolling BLA completion in Q4; potential 2026 approval would diversify revenue and support profitability trajectory .
  • Commercial base continues to expand globally: LatAm Crysvita patient adds and Evkeeza uptake across EMEA provide durable growth vectors amid quarter-to-quarter variability .
  • 2025 guidance intact; 2027 GAAP profitability reiterated: Execution on launches (UX111 resubmission early 2026, DTX401 in Q4, potential UX143 approval) and PRV proceeds remain key assumptions .
  • Trading implications: Expect positioning into OI readout; financing reduces near-term equity raise risk; monitor Q4 royalty seasonality recovery and incremental clarity on UX111 resubmission timing .

Appendix: Cross-References and Notes

  • Minor rounding difference: Q3 total OpEx reported at $330.82M in press release; CFO spoke to $331M — driven by pre-launch manufacturing spend and R&D mix .
  • Cash metrics: Q3-end cash $447.32M (pre-OMERS), net cash used in operations $91M; YTD ops cash use $366M .
  • Guidance consistency across quarters: Revenue ranges unchanged from Q1 and Q2 to Q3 .