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Ultragenyx Pharmaceutical Inc. (RARE)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 total revenue was $164.9M (+29% y/y), driven by Crysvita $115.4M (+23% y/y), Dojolvi $31.1M (+34% y/y), and Evkeeza $10.4M; FY2024 revenue reached $560.2M, exceeding the company’s raised guidance range from Q3 2024. Net loss was $133.4M ($1.39 per share) in Q4 .
  • FY2025 guidance: total revenue $640–$670M, Crysvita $460–$480M, Dojolvi $90–$100M; management expects reduced net cash used in operations versus 2024 ($414M) .
  • Key 2025 catalysts: UX111 (Sanfilippo) BLA accepted with PDUFA decision expected H2 2025; DTX401 (GSDIa) BLA mid-2025; OI program (UX143) Phase 3 Orbit/Cosmic interim analyses mid-2025 with potential filing thereafter .
  • Wall Street consensus (S&P Global) for Q4 2024 EPS/Revenue was unavailable due to a provider rate limit; estimates context is noted below (no numeric beat/miss shown).

What Went Well and What Went Wrong

What Went Well

  • Strong commercial execution: Q4 revenue +29% y/y; Crysvita +23% y/y; Dojolvi +34% y/y; Evkeeza demand building ex-U.S. .
  • Guidance beat/raise cadence: FY2024 revenue $560M exceeded the upper end of prior guidance ($530–$550M) and FY2025 revenue guided to +14–20% y/y growth .
  • Pipeline momentum with near-term approvals: “We have created a next-generation rare disease company on a pathway to profitability… pending PDUFA… second gene therapy BLA… pivotal Phase 3 OI… Phase 3 Angelman” (CEO) .

What Went Wrong

  • Continued losses as R&D and SG&A support pipeline: Q4 net loss $133.4M; Q4 operating expenses $287.2M (R&D $187.8M; SG&A $82.5M) .
  • Q1 cash flow seasonality and milestone payments to weigh on 2025 Q1 operating cash ($45M total for Angelman Phase 3 initiation and Evkeeza sales milestone) .
  • Variability in LatAm Crysvita ordering continues to drive quarter-to-quarter revenue volatility (management reminder) .

Financial Results

MetricQ2 2024Q3 2024Q4 2024
Total revenues ($USD Millions)$147.0 $139.5 $164.9
Net loss ($USD Millions)$(131.6) $(133.5) $(133.4)
Diluted EPS ($USD)$(1.52) $(1.40) $(1.39)
Total operating expenses ($USD Millions)$263.4 $271.5 $287.2
Cost of sales ($USD Millions)$21.3 $21.0 $16.9
Cash, cash equivalents & marketable debt securities ($USD Millions)$874.5 (as of 6/30) $824.7 (as of 9/30) $745.0 (as of 12/31)

Product revenue breakdown

MetricQ2 2024Q3 2024Q4 2024
Crysvita total revenue ($USD Millions)$113.7 $97.8 $115.4
Dojolvi revenue ($USD Millions)$19.4 $21.4 $31.1
Evkeeza revenue ($USD Millions)$7.9 $10.7 $10.4
Mepsevii revenue ($USD Millions)$6.1 $9.6 $8.0
Total revenues ($USD Millions)$147.0 $139.5 $164.9

KPIs

KPIQ2 2024Q3 2024Q4 2024
Crysvita patients on reimbursed therapy in LatAm (approx.)>620 ~700 ~750
Dojolvi reimbursed patients since launch (approx.)~520 ~550 ~575
Evkeeza patients treated across EMEA/Japan (countries, approx.)n/aPatients across major EU countries; Japan launched ~190 pts across 14 countries; Japan building

Year-over-year Q4 highlights

  • Total revenue +29%, Crysvita +23%, Dojolvi +34% y/y; Evkeeza $10.4M as adoption builds ex-U.S. .

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Total RevenueFY2024$530–$550M Actual $560.2M Beat
Crysvita RevenueFY2024$375–$400M (upper end) Actual $409.5M Beat
Dojolvi RevenueFY2024$75–$80M Actual $88.2M Beat
Net Cash Used in OperationsFY2024Around $400M Actual $414M Slightly above
Total RevenueFY2025n/a$640–$670M New
Crysvita RevenueFY2025n/a$460–$480M New
Dojolvi RevenueFY2025n/a$90–$100M New
Net Cash Used in OperationsFY2025n/aLower than 2024 Lower

Earnings Call Themes & Trends

TopicPrevious Mentions (Q2 & Q3)Current Period (Q4 2024)Trend
UX111 (Sanfilippo, gene therapy)Agreement with FDA that CSF HS can support accelerated approval; pre-BLA planned BLA submitted Dec 2024; PDUFA decision and potential launch H2 2025 Advancing toward approval
UX143 (Setrusumab for OI)Phase 2 14-month data: 67% fracture reduction; Breakthrough Therapy Designation; Phase 3 Orbit interim plan IA2 mid-2025 (p<0.01) with high confidence; file quickly if Orbit hits; Cosmic interim in parallel De-risked path, timing clarified
GTX-102 (Angelman ASO)EOP2 meeting positive; Phase 3 Aspire to start by YE2024; Aurora planned Aspire enrollment began Dec 2024; complete in H2 2025; Aurora to initiate in 2025; sham-control rationale Execution underway
DTX401 (GSDIa, gene therapy)Phase 3 topline: 41% mean reduction at Week 48; positive endpoints Crossover pts show 62% mean reduction by Week 30; BLA mid-2025; manufacturing insourcing expected to lower COGS ~40% Strengthening commercial case
DTX301 (OTC deficiency, gene therapy)Phase 3 dosing ongoing Phase 3 enrollment complete (n=37); protocol amended for 36-week blinded ammonia endpoint; unblinded removal of meds/diet Study optimized for patient-centricity
Evkeeza (HoFH, ex-U.S. launch)Launch momentum in EMEA & Japan; country-by-country reimbursement ~190 patients across 14 countries; Japan to contribute more meaningfully Building adoption
Japan strategy (Dojolvi)Conditional Early Approval path; J-NDA mid-2025 Reiterated as part of 2025 focus On track

Management Commentary

  • “We have created a next-generation rare disease company on a pathway to profitability with meaningful revenue growth from multiple global products and a series of potential new drug approvals.” – Emil D. Kakkis, CEO .
  • “Total revenue is expected to be between $640 million and $670 million, which represents 14% to 20% growth over 2024.” – Howard Horn, CFO .
  • On OI interim timing: “We feel confident one way or another the trial is going to end and will be successful this year... we have more confidence in the second interim than we did in the first.” – CEO .
  • On UX111 launch readiness: “We will get our work with payers upfront… urgency and time really matters to these kids… I would look at UX111 as being more like Zolgensma in SMA.” – CEO .

Q&A Highlights

  • OI (UX143) interim confidence and timing: IA2 mid-2025 seen as most likely stop; if hit, filing follows quickly; if not, final analysis in Q4 2025 .
  • Angelman Phase 3 design: Sham vs placebo chosen for ethical/practical reasons; 48-week primary endpoint to capture broader gains; Aurora to support broader labeling .
  • OTC (DTX301) protocol: 36-week blinded ammonia endpoint; unblinded medication/diet removal improves power, reduces cost, addresses patient concern .
  • Manufacturing economics: In-house PPQ lots for DTX401 expected to reduce costs by ~40%, improving margins for gene therapy portfolio .
  • Commercial leverage: Existing IEM and bone franchises position field teams for upcoming launches (UX111, DTX401, UX143) .

Estimates Context

  • S&P Global consensus for Q4 2024 (Revenue, EPS, EBITDA) was unavailable at the time of retrieval due to provider rate limits; no numeric beat/miss vs Wall Street is shown. Values from S&P Global were not retrieved; please note unavailability.

Where estimates may need to adjust:

  • FY2025 revenue guidance ($640–$670M) embeds Crysvita growth (+12–17%) and Dojolvi (+2–14%), plus ex-U.S. Evkeeza expansion; consensus models should reflect stronger LatAm Crysvita growth and the ex-U.S. Evkeeza trajectory .
  • Pipeline catalysts (UX111 PDUFA H2 2025; DTX401 BLA mid-2025; OI IA2 mid-2025) may shift 2H 2025 launch timing and contribution assumptions .

Key Takeaways for Investors

  • Commercial engine accelerating: Q4 revenue re-accelerated to $164.9M; FY2024 beat prior guidance; FY2025 guide implies double-digit growth with diversified drivers (LatAm Crysvita, Dojolvi, Evkeeza) .
  • Catalysts concentrated in 2025: UX143 Phase 3 IA2 midyear, UX111 PDUFA H2 2025, DTX401 BLA mid-2025—each with potential to add meaningful optionality and de-risk the path to profitability .
  • Expense discipline with launch focus: Despite high R&D/SG&A, management targets lower operating cash burn in 2025 and highlighted manufacturing insourcing to lift gene therapy margins longer term .
  • Latin America and ex-U.S. momentum: Crysvita patient adds and Dojolvi named-patient demand drive regional growth; Evkeeza adoption broadening across 14 countries and in Japan .
  • OI narrative supportive: Phase 2 durability (67% fracture reduction) and BTD + statistically stringent interim plan underpin confidence; filing path defined if IA2 hits .
  • Trading setup: Near-term stock sensitivity likely tied to UX143 IA2 outcome timing and any UX111 BLA/PDUFA updates; variance in LatAm ordering could introduce quarterly revenue noise (watch disclosures) .
  • Estimates monitoring: With FY2025 guide now set, watch for Street upward revisions on Crysvita/Dojolvi and inclusion of potential late-2025 contributions from pipeline launches; note S&P Global estimate data was unavailable to confirm current consensus.