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BIOMARIN PHARMACEUTICAL INC (BMRN)·Q1 2025 Earnings Summary

Executive Summary

  • Q1 2025 delivered solid growth and margin expansion: total revenue $745.1M (+15% y/y), GAAP EPS $0.95 (+107% y/y), and non-GAAP EPS $1.13 (+59% y/y), with non-GAAP operating margin at 35.7% (+1,190 bps y/y) .
  • Results modestly beat S&P Global consensus: revenue $745.1M vs $738.7M* and non-GAAP EPS $1.13 vs $0.95*; strength was driven by 40% y/y VOXZOGO revenue growth and lower operating expenses from 2024 cost transformation and portfolio reprioritization .
  • Guidance reaffirmed: FY25 total revenue $3.10–$3.20B, non-GAAP op margin 32–33%, non-GAAP EPS $4.20–$4.40; VOXZOGO FY25 revenue expected at $900–$950M .
  • Management flagged order timing and H2 weighting (VOXZOGO revenues to be more weighted to H2), implying potential Q2 volatility despite sustained patient growth—an important near-term trading dynamic .

Consensus estimates marked with * Values retrieved from S&P Global.

What Went Well and What Went Wrong

What Went Well

  • VOXZOGO momentum: revenue up 40% y/y to $214M, driven by global demand and expanded access to 49 countries; management expects H2 weighting as ordering patterns normalize across OUS markets .
  • Profitability outpaced revenue growth: GAAP operating margin 30.0% (+1,640 bps y/y) and non-GAAP operating margin 35.7% (+1,190 bps y/y), reflecting 2024 cost transformation and R&D reprioritization .
  • Cash generation: operating cash flow $174M (+271% y/y) and total cash/investments ~$1.8B, supporting reinvestment in pipeline and commercialization .
    “Non-GAAP operating margin reached 35.7%… driven by strong revenue performance and a spending reset as we prepare to invest more aggressively across R&D and SG&A in the coming quarters.” — CFO Brian Mueller .

What Went Wrong

  • Sequential VOXZOGO revenue disconnect: management highlighted flattish revenue trends from Q4 → Q1 and potentially into Q2, despite increasing patients due to multi-market order timing—creating near-term optical pressure .
  • KUVAN headwind: product revenue fell 31% y/y to $25M due to ongoing generic competition post loss of exclusivity .
  • Tax rate impact: higher tax provision associated with increased taxable income partially offset profit growth .

Financial Results

Consolidated Results vs Prior Quarters and Consensus

MetricQ3 2024Q4 2024Q1 2025
Total Revenues ($USD Millions)$745.7 $747.3 $745.1
GAAP Diluted EPS ($USD)$0.55 $0.64 $0.95
Non-GAAP Diluted EPS ($USD)$0.91 $0.92 $1.13
GAAP Operating Margin %15.3% 21.6% 30.0%
Non-GAAP Operating Margin %27.7% 31.1% 35.7%
Revenue Consensus Mean ($USD Millions)*$703.3*$711.0*$738.7*
Primary EPS Consensus Mean ($USD)*$0.79*$0.74*$0.95*

Consensus estimates marked with * Values retrieved from S&P Global.

Key implications: Q1 2025 was a clean beat on both revenue and non-GAAP EPS versus consensus, with margin expansion the principal driver of EPS outperformance .

Product Revenue Breakdown

Product ($USD Millions)Q1 2024Q4 2024Q1 2025
VOXZOGO$153 $208 $214
VIMIZIM$193 $191 $188
NAGLAZYME$106 $110 $114
PALYNZIQ$76 $100 $93
ALDURAZYME$35 $39 $49
BRINEURA$39 $48 $40
Total Enzyme Therapies$449 $488 $484
KUVAN$36 $28 $25
ROCTAVIAN$1 $11 $11

Observations: VOXZOGO grew both y/y and sequentially; enzyme therapies rose 8% y/y with notable PALYNZIQ and ALDURAZYME growth; KUVAN continued to decline .

KPIs

KPIQ3 2024Q4 2024Q1 2025
Operating Cash Flow ($USD Millions)$221 N/A$174
Total Cash & Investments ($USD Billions)~$1.5 $1.659 $1.779
VOXZOGO Countries (#)N/A47 49
VOXZOGO OUS Revenue Share (%)N/A74% ~75% (management commentary)

Guidance Changes

MetricPeriodPrevious Guidance (Feb 19, 2025)Current Guidance (May 1, 2025)Change
Total RevenuesFY 2025$3.10B–$3.20B $3.10B–$3.20B Maintained
Non-GAAP Operating Margin %FY 202532%–33% 32%–33% Maintained
Non-GAAP Diluted EPSFY 2025$4.20–$4.40 $4.20–$4.40 Maintained
VOXZOGO RevenueFY 2025$900M–$950M $900M–$950M Maintained

Note: Guidance reflects impacts of currently enacted U.S. tariffs but excludes potential future pharmaceutical tariffs; management characterizes current exposure as immaterial to China, Mexico, and Canada .

Earnings Call Themes & Trends

TopicPrevious Mentions (Q3 2024, Q4 2024)Current Period (Q1 2025)Trend
VOXZOGO demand and ordering patternsRapid adoption; >3,800 patients; strong OUS contribution; progress on hypochondroplasia enrollment; noted bolus orders affecting quarterly dynamics H1 flattish revenue vs rising patients; H2 revenue weighting expected; 49 countries live Stable demand; revenue more back-half weighted
U.S. vs OUS mixVOXZOGO FY24 24% U.S. / 76% OUS; Q4 26% / 74% OUS ~75% of VOXZOGO; long-term mix ~1/3 U.S., 2/3 OUS Mix stable; OUS remains majority
Tariffs/macro exposureLong-term guardrails framed at Investor Day; diversified revenue base Immaterial exposure to enacted tariffs; modeling scenarios; net U.S. exporter via CA manufacturing Monitoring; limited near-term impact
BD strategyMethodical approach; alignment with core strengths and BU focus Targeting at least one BD deal in 2025; clinical-stage, genetically defined assets Increasing focus; near-term catalyst
BMN 333 (long-acting CNP)First-in-human initiation planned; preclinical sustained free CNP FDA agreement on overall plan; aiming for superiority vs VOXZOGO in comparative study; Phase II/III start in 2026 Advancing; ambitious efficacy bar
PALYNZIQ adolescentsEnrollment complete; data expected 2025 Pivotal met primary endpoint; label expansion submissions in H2 2025 Positive inflection
Margin trajectoryYTD 2024 margins expanding via cost program Non-GAAP margin 35.7% in Q1; expect slightly lower margins Q2–Q4 as spend steps up; FY in line with 32–33% Strong start; planned reinvestment ahead

Management Commentary

  • “Non-GAAP EPS of $1.13… reflects significant profitability expansion, nearly 4x the rate of top line growth… paving the way for record full year performance in 2025.” — CEO Alexander Hardy .
  • “We expect total revenues to be higher in the second half… VOXZOGO revenues looking back to Q4 2024 and anticipated trends through Q2 2025 have revenues slightly disconnected from patient growth and appearing relatively similar quarter-to-quarter.” — CFO Brian Mueller .
  • “We continue to focus on doing at least one business development deal this year… clinical-stage assets aligned with our business unit structure.” — CEO Alexander Hardy .
  • “We reached agreement with the FDA on an overall clinical development plan for BMN 333… we are looking for superiority in growth dynamics from a higher exposure of CNP.” — CMO Gregory Friberg .

Q&A Highlights

  • VOXZOGO quarterly pattern and H2 weighting: revenues may be flattish Q1–Q2 despite rising patients; H2 expected stronger due to global order timing .
  • Tariffs exposure: immaterial impact from enacted tariffs; company is a net U.S. exporter and ~2/3 of revenue ex-U.S.; scenario planning underway for potential future tariffs .
  • BD pipeline: aiming to execute at least one BD deal in 2025 aligned to genetically defined conditions and BU strengths .
  • BMN 333 program: FDA-aligned combined Phase II/III with comparative effectiveness vs VOXZOGO; explicit intent to demonstrate superiority .
  • U.S./OUS VOXZOGO mix: ~75% OUS currently; long-term target ~2/3 OUS / 1/3 U.S. .

Estimates Context

Metric (Q1 2025)Consensus*ActualSurprise
Revenue ($USD Millions)$738.7*$745.1 +$6.4M / +0.9%
Primary EPS ($USD)$0.95*$1.13 +$0.18 / +18.9%

Consensus estimates marked with * Values retrieved from S&P Global.

Implications: The revenue beat was modest, but EPS outperformance was significant, driven by margin expansion and lower OpEx following 2024 portfolio optimization and cost initiatives . Given management’s guidance for Q2–Q4 reinvestment and H2 weighting, quarterly models may need to smooth earnings and shift revenue to H2 .

Key Takeaways for Investors

  • Q1 was a quality beat on EPS with broad-based margin expansion; watch for planned reinvestment to temper margins near term while FY margin targets remain intact .
  • VOXZOGO remains the core growth engine; expect H2 revenue weighting and potential Q2 optical softness due to multi-market order timing—manage near-term expectations accordingly .
  • Pipeline catalysts in 2025: PALYNZIQ adolescent sBLA submissions (H2), BMN 351 dystrophin data (H2), BMN 333 initial PK data (YE), and potential BD transaction—each a stock narrative driver .
  • OUS-heavy revenue base and net U.S. export manufacturing provide some insulation to tariff scenarios; current tariff exposure characterized as immaterial .
  • Enzyme Therapies contribute solidly (8% y/y) with PALYNZIQ +22% y/y; KUVAN decline persists due to generics—mix improving toward durable assets .
  • Liquidity supports continued investment: $1.779B cash/investments and strong cash generation underpin commercialization and R&D execution .
  • Guidance reaffirmation post-Q1 reduces downside risk; monitor H2 execution and margin cadence against 32–33% FY non-GAAP margin and $4.20–$4.40 EPS .

Appendix: Non-GAAP Adjustments and Notes

  • Non-GAAP EPS and operating margin exclude stock-based compensation, intangible amortization, and certain specified items; reconciliations provided in the press release .
  • Q1 tax provision increased with higher taxable income; a headwind to net income vs operating trends .
  • VOXZOGO OUS order timing dynamics likely to increase back-half revenue weighting in 2025 .