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Ultragenyx Pharmaceutical Inc. (RARE)·Q3 2024 Earnings Summary

Executive Summary

  • Q3 revenue was $139.5M (+42% YoY) with Crysvita $97.8M, Dojolvi $21.4M, Evkeeza $10.7M; net loss improved to $(133.5)M and EPS $(1.40) vs $(2.23) in Q3’23 .
  • Sequentially, revenue declined from Q2’s seasonal high ($147.0M) but EPS improved Q/Q from $(1.52), while management reaffirmed full-year revenue guidance of $530–$550M and Crysvita toward the upper end of $375–$400M; Dojolvi $75–$80M; net cash used in ops ≈$400M .
  • Clinical catalysts strengthened: FDA Breakthrough Therapy Designation for setrusumab (UX143) in OI, DTX401 Phase 3 crossover patients showed a 62% mean cornstarch reduction at Week 30, and UX111 moved toward an end-of-year BLA filing path .
  • Management highlighted potential for “up to three near-term BLA submissions and approvals,” positioning revenue acceleration and portfolio breadth as stock drivers into 2025–26 .

What Went Well and What Went Wrong

What Went Well

  • Crysvita revenue grew 31% YoY to $97.8M; LatAm/Turkey product sales were $36.0M (+85% YoY), demonstrating robust international demand and access expansion .
  • DTX401 Phase 3 crossover analysis showed a rapid and larger 62% mean cornstarch reduction by Week 30 vs 41% at Week 48 in the original treatment arm; safety remained acceptable, strengthening regulatory case for mid-2025 BLA .
  • FDA granted Breakthrough Therapy Designation to UX143 based on Phase 2 Orbit and ASTEROID data, potentially expediting review timelines and increasing visibility into OI commercialization .

Quote: “We continue to see substantial year-over-year revenue growth... This growth could accelerate with up to three near-term BLA submissions and approvals” — Emil D. Kakkis, CEO .

What Went Wrong

  • Sequential revenue fell from $147.0M in Q2 to $139.5M in Q3; management cited quarter-to-quarter variability in Latin America ordering, which can affect reported sales cadence .
  • Operating expenses increased Q/Q to $271.5M (from $263.4M in Q2), with R&D $170.1M and SG&A $80.4M; non-cash stock-based comp was $41.6M, continuing to weigh on profitability .
  • Estimates data from S&P Global was unavailable in this session, limiting direct beat/miss analysis versus Street; however, management only reaffirmed guidance, not raised, suggesting cautious trajectory after a strong Q2 .
    (S&P Global consensus unavailable due to data access limits in this session.)

Financial Results

Summary financials (USD Millions except EPS)

MetricQ1 2024Q2 2024Q3 2024
Total Revenues$108.833 $147.026 $139.494
Total Operating Expenses$274.180 $263.387 $271.481
Loss from Operations$(165.347) $(116.361) $(131.987)
Net Loss$(170.684) $(131.598) $(133.516)
Diluted EPS$(2.03) $(1.52) $(1.40)

Q3 YoY comparison

MetricQ3 2023Q3 2024YoY Change
Total Revenues$98.052 $139.494 +42.3%
Net Loss$(159.649) $(133.516) +$26.133M improvement
Diluted EPS$(2.23) $(1.40) +$0.83 improvement

Product and segment revenues

Product/SegmentQ1 2024Q2 2024Q3 2024
Crysvita – Product Sales$36.241 $40.449 $35.604
Crysvita – Profit-Share/NA Royalties/Other$40.402 (profit-share territory) $67.045 (profit-share territory) $55.985 (profit-share territory)
Crysvita – EU Royalties$5.942 $6.176 $6.258
Crysvita – Total$82.585 $113.670 $97.847
Dojolvi$16.362 $19.355 $21.374
Mepsevii$6.611 $6.145 $9.616
Evkeeza$3.275 $7.856 $10.657
Total Revenues$108.833 $147.026 $139.494

KPIs and cash

KPIQ1 2024Q2 2024Q3 2024
Net Cash Used in Operations$(191)M $(77)M $(67)M
Cash, Cash Equivalents & Marketable Securities$568.661M $874.490M $824.694M
Working Capital$350.392M $691.774M $514.600M

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Total RevenueFY 2024$500–$530M (Q1) $530–$550M (Q2 raised; reaffirmed Q3) Raised (Q2); Maintained (Q3)
Crysvita RevenueFY 2024$375–$400M (Q1) Toward upper end of $375–$400M (Q3) Maintained; narrowed to upper end
Dojolvi RevenueFY 2024$75–$80M (Q1) $75–$80M (Q2, Q3) Maintained
Net Cash Used in OperationsFY 2024< $400M (Q1) ≈ $400M (Q3) Maintained (tightened language)

Earnings Call Themes & Trends

TopicPrevious Mentions (Q1 & Q2)Current Period (Q3)Trend
Setrusumab (UX143) OIPhase 3 Orbit/Cosmic fully enrolled; power strong; interims planned; BMD gains and 67% fracture reduction at 14 months FDA Breakthrough Therapy Designation; clarified interim timing/thresholds and commercialization prep Strengthening regulatory momentum
DTX401 GSDIaTopline Phase 3: significant cornstarch reduction at Week 48; preparing filing strategy Crossover patients: 62% mean reduction at Week 30; plan BLA mid-2025; in-house PPQ manufacturing to improve COGS Efficacy signal strengthening; manufacturing leverage
GTX-102 AngelmanEOP2 alignment; Phase 3 design, endpoints, MDRI; global start-up by year-end Long-term data update planned; Phase 3 start confirmed timeline; competitive positioning clarified Advancing to pivotal execution
UX111 MPS IIIAFDA agreement on CSF heparan sulfate surrogate for accelerated approval; pre-BLA in H2 Successful pre-BLA meeting; BLA expected around end of 2024 Approaching filing
UX701 WilsonStage 1 showed tapering off SOC; biomarker improvements; Stage 2 design in H2 Additional Stage 1 cohort planned (higher dose/optimized immunomodulation); aim majority off SOC before Stage 2 Dose optimization for broader response
Commercial traction (Crysvita/Dojolvi/Evkeeza)LatAm patient adds; Q2 seasonal rebound; Evkeeza launch building in EMEA/Japan Continued Crysvita demand; LatAm >700 patients; Evkeeza revenue building; Japan contribution increasing Geographical expansion supports growth
Profitability pathNot detailed timingTarget GAAP profitable quarter by end of 2026 (with PRV monetization and launches) Clearer horizon

Management Commentary

  • “We successfully completed development work across our late-stage programs and now are reaching the submission stage... up to 3 near-term BLA submissions and approvals” — Emil Kakkis .
  • “Crysvita contributed $98M, including $56M from North America, $36M from Latin America and Turkey, and $6M from Europe... Net cash used in operations was $67M” — Howard Horn .
  • “Running [DTX401] PPQ lots in the plant will probably save us around 40%... our own plant substantially reduces our costs” — Emil Kakkis .
  • “We feel like we have the cash... to fund our operating plan that gets us to a GAAP profitable quarter by the end of 2026” — Howard Horn .

Q&A Highlights

  • Setrusumab interims: Extremely stringent first IA (p<0.001) and second IA (p<0.01); if positive, company will only disclose threshold met until database lock; time-to-fracture accrual and powering detailed .
  • DTX401 clarity: Original treatment arm had 41% reduction by Week 48; crossover reached 62% by Week 30 due to titration with glucose visibility; in-house PPQ manufacturing targets ~40% COGS savings .
  • Wilson UX701: Additional Stage 1 cohort at moderately higher dose with optimized immunomodulation to enable majority off standard-of-care; biomarker endpoints include urinary copper, ceruloplasmin activity and liver biopsy substudy .
  • Profitability: Management reiterated plan to achieve a GAAP profitable quarter by end of 2026, aided by PRV monetization and product launches .
  • Commercial readiness: Existing field infrastructure and leadership from Crysvita supports setrusumab launch preparation; Evkeeza outside US building across EMEA/Japan .

Estimates Context

  • S&P Global consensus estimates for Q3 2024 EPS and revenue were not retrievable in this session due to access limits. As a result, we cannot formally assess beat/miss versus Street. Values retrieved from S&P Global were unavailable in this session.
  • Management reaffirmed FY24 revenue guidance ($530–$550M) and product-level ranges (Crysvita toward upper end of $375–$400M; Dojolvi $75–$80M), suggesting internal confidence post-Q3 .

Key Takeaways for Investors

  • Crysvita remains the revenue anchor with robust international growth; Evkeeza and Dojolvi are contributing incremental diversification; expect quarter-to-quarter variability, particularly in LatAm .
  • DTX401’s crossover data are a positive surprise, indicating faster titration and potentially stronger real-world impact; in-house manufacturing could materially improve economics on launch .
  • Setrusumab’s BTD and clear interim framework increase probability of accelerated clinical timelines; launch readiness and manufacturing capacity at 3,000L CMOs support scalability .
  • UX111 MPS IIIA is approaching BLA filing around year-end, adding another near-term catalyst that could expand the commercial base .
  • Cash of ~$825M and full-year net cash use ≈$400M sustains the pipeline; management targets a GAAP profitable quarter by end-2026, hinging on successful filings/launches .
  • With Street estimates unavailable here, trade focus should center on near-term catalysts (setrusumab interims, UX111 BLA timing, DTX401 regulatory interactions) and reaffirmed guidance trajectory .
  • Risk monitoring: trial interims not guaranteed to hit at first cut; quarter-to-quarter revenue cadence may fluctuate due to LatAm ordering patterns; opex and stock comp remain elevated as programs advance .