BioMarin's Q3 2025: Contradictions Emerge on Voxzogo Sales, BMN-333 Exposure, and Business Development Strategies

Date of Call: None provided

Financials Results

• Revenue: Raised full-year 2025 total revenue guidance (lower end) to $3.15B; year-to-date total revenue +11% YOY; Voxzogo full-year 2025 revenue reaffirmed at $900M–$935M (midpoint ~25% growth at midpoint).
• EPS: Updated full-year non-GAAP diluted EPS guidance to $3.50–$3.60; Q3 includes $221M IPR&D charge (~$1.10 per share pre-tax) which increased GAAP and non-GAAP R&D and reduced EPS; IPR&D ~ $0.15 per-share impact on non-GAAP EPS noted.
• Operating Margin: Updated full-year non-GAAP operating margin guidance to 26%–27%; Q3 operating margin lower YOY due to $221M IPR&D charge and higher SG&A investments.

Guidance:

• Full-year 2025 total revenue guidance lower end raised to $3.15B; Voxzogo revenue reaffirmed at $900M–$935M.
• Updated full-year non-GAAP operating margin guidance to 26%–27% and non-GAAP EPS to $3.50–$3.60.
• Expect Q4 2025 to be Voxzogo’s highest quarterly revenue due to contracted orders and patient starts.
• Phase‑3 hypochondroplasia data expected H1 2026; potential launch in 2027 if supportive.
• Start BMN‑333 phase 2–3 in H1 2026; Palynziq adolescent label and BMN‑401 phase‑3 readouts expected in 2026.

Business Commentary:

* Strong Financial Performance: - BioMarin Pharmaceutical delivered an 11% year-to-date growth in total revenues, raising the lower end of their full-year 2025 revenue guidance to $3.15 billion. - The growth was driven by the performance of the global enzyme therapies and skeletal conditions business units.

• Voxzogo Revenue and Market Expansion:
• Voxzogo's revenue increased by 24% year-to-date, with an expected full-year 2025 revenue range of $900 million to $935 million.

• This growth was due to strong demand in global markets, with significant contributions from international sales, which comprised 75% of annual revenues.

• Operating Cash Flow and Financial Position:

• BioMarin reported $369 million in operating cash flow in Q3 and $728 million year-to-date.

• The company's cash and investments balance stood at approximately $2 billion at the end of Q3, reflecting strategic investments in business units and potential growth opportunities.

• Strategic Focus and Business Development:

• BioMarin announced plans to divest Roctavian, focusing on core business units aligned with strategic priorities.
• The company emphasized business development as a key strategy, exploring opportunities for growth through collaborations and potential acquisitions.

Sentiment Analysis:

Overall Tone: Positive

• Management raised full‑year revenue guidance midpoint and reaffirmed Voxzogo outlook; reported year‑to‑date revenue growth of 11% and $2B cash and investments; updated non‑GAAP operating margin guidance to 26%–27% and non‑GAAP EPS to $3.50–$3.60; emphasized multiple upcoming data readouts (hypochondroplasia H1 2026, BMN‑333 H1 2026) and active business development.

Q&A:

• Question from Phil Nadeau (TD Cowen): Can you talk a little bit more about the scenarios you see and maybe most specifically, why are you rescinding the guidance now? What has changed over the last year since it was initially issued?
  Response: They replaced a single 2027 number with a range because of substantial uncertainty (potential Voxzogo competition, Enzyme acquisition, Roctavian divestiture); lower‑case assumes two competitors launch and take share, higher‑case assumes delays/IP defenses.

• Question from Selvine Richter (Goldman Sachs): Could you speak to why Voxzogo’s sales were down quarter over quarter? Also, help us understand the business development strategy and when BD levers might emerge to add to the portfolio.
  Response: Q3 Voxzogo dip driven by order timing shifts (Q3→Q4) despite steady patient adds; business development is a top priority and they are pursuing deals across early‑stage to commercial assets, expecting announcements when deals close.

• Question from Corey Kasimov (Evercore): Any qualitative commentary on prior long‑term mid‑2030s guidance for Voxzogo and enzyme therapies CAGR; and given the cash balance, are buybacks being considered versus BD?
  Response: They target high single‑digit sustainable growth for enzyme therapies and continued Voxzogo penetration/indication expansion (but offsets from potential competition are uncertain); capital allocation priority is business development over buybacks.

• Question from Joe Schwartz (Leerink Partners): For BMN‑333 PK data what exposure levels would give confidence for superiority and what minimal annualized growth velocity delta over Voxzogo might be required?
  Response: Phase‑1 met the PK target with ≥3x free CNP AUC in multiple cohorts; they declined to specify an AGV delta but said the program is designed to deliver a clinically meaningful, measurable improvement over Voxzogo tied to functional outcomes.

• Question from Jessica Fye (JP Morgan): What is 2027 FactSet consensus excluding Roctavian; do 40% non‑GAAP operating margin and >$1.25B CFO targets still stand; expectations for BMN‑351 6mg vs 9mg biopsy data; hypochondroplasia powering?
  Response: FactSet 2027 consensus $3.725B including $75M Roctavian → excluding Roctavian = $3.65B; 40% operating margin target for 2026 remains but CFO >$1.25B is top‑line dependent; BMN‑351 six‑ and nine‑mg biopsy and safety data will inform whether ~3–5% at six months predicts ~10% steady‑state dystrophin; hypochondroplasia trial powered to detect an AGV delta similar to Voxzogo effects.

• Question from Julian/Paul (Stifel): How much of your changed outlook reflects commercial competition versus litigation/IP risks? On DMD, why a 10% dystrophin bar and could a lower level still advance?
  Response: Scenario modeling includes both successful competitor launches (lower‑case) and IP defense/delays (higher‑case); the 10% dystrophin target is an ambitious go/no‑go based on models where ~3–5% at six months projects to ~10% steady state and would be expected to deliver meaningful functional benefit.

• Question from Chris Raymond (Raymond James): Are you more concerned about Ascendis (trans‑con‑CNP) vs others and did you assume competitor approvals/timelines (e.g., PDUFA next month)? Also, how is Enzyme/BMN‑401 integration progressing commercially?
  Response: They modeled competitor scenarios using each competitor’s communicated timelines (including upcoming PDUFAs) rather than singling out one rival; Enzyme integration is early (closed July 1) but they expect BioMarin scale to strengthen BMN‑401 execution and plan ENERGY3 preparations for H1 next year.

• Question from Sean Lehman (Morgan Stanley): Latest thoughts on orphan drug exclusivity and likelihood of patient switching; what is balance‑sheet firepower and preferred capital structure for deployment?
  Response: They submitted a petition to the FDA on orphan exclusivity and expect most patients satisfied on Voxzogo to remain on therapy (high adherence); estimated deployable firepower is $4B–$5B and capital will be prioritized toward growth/BD.

• Question from Zaki/Akash (Jefferies): Why disclose the 2027 scenario range now instead of waiting for competitor (BridgeBio) data; does the lower end assume superiority vs BridgeBio and is $4B still the upside?
  Response: They shared the range now due to investor interest and multiple uncertainties; the lower end is a modeled conservative scenario (not necessarily worst‑case) reflecting successful competitor launches, while $4B remains an achievable upside under favorable outcomes (IP wins or portfolio successes).

Contradiction Point 1

Voxzogo Sales and Market Forecasting

It involves differing explanations for the quarter-over-quarter sales performance of Voxzogo, which could impact investor perceptions of the company's financial management and market forecasting abilities.

Why did Voxzogo's sales decline quarter over quarter? What business development strategies are in place for Voxzogo, and when can we expect to see leverage from these efforts? - Selvine Richter (Goldman Sachs)

2025Q3: The slight Q3 decrease was due to timing shifts and market forecasts between Q3 and Q4. - Brian Mueller(CFO)

What factors led to the adjustment in VOXZOGO's revenue guidance? - Tommie Reerink (Goldman Sachs)

2025Q2: Revenue adjustments are due to shifting large international orders, with some moving into early 2026 due to better forecasting. - Cristin Hubbard(CMO)

Contradiction Point 2

BMN-333 Exposure and Efficacy

It concerns the company's stated exposure levels for BMN-333 and the implications for its competitive positioning and clinical efficacy, which are crucial for regulatory approval and market acceptance.

What exposure levels demonstrate clinical superiority over Voxzogo? What minimum annualized growth velocity deltas are needed to drive patient switching? - Joe Schwartz (Leerink Partners)

2025Q3: Exposure levels meeting 3x AUC are required, with multiple dose levels achieving this in our phase one study. - Greg Friberg(CRO)

How do you maintain 3x AUC in patients, and how do AUC levels compare across VOXZOGO doses? - Huidong Wang (Barclays)

2025Q2: We aim for 3x AUC in patients, pending dose-ranging results. - Gregory Friberg(CRO)

Contradiction Point 3

Business Development Priorities

It reflects a shift in strategic focus for business development, which is crucial for future growth and partnerships.

What caused the QoQ decline in Voxzogo sales and when will strategic initiatives show results? - Selvine Richter(Goldman Sachs)

2025Q3: We are exploring deals in early-stage collaborations and phase three, pre-commercial assets. We think assets are more valuable in our hands and see business development as a priority. - Alexander Hardy(CEO)

What is BioMarin's current interest and timeline for BD, and what details will be disclosed about BMN 333 data this year? - Tommie Reerink(Goldman Sachs)

2025Q1: BioMarin continues to focus on BD with a systematic approach and plans for at least one deal this year. - Alexander Hardy(CEO)

Contradiction Point 4

Long-term Growth Rate Target for Enzyme Therapies

It directly impacts the long-term outlook and expectations for the growth of the enzyme therapy segment, influencing investor perceptions and strategic planning.

Can you provide qualitative commentary on your prior mid-2030s guidance for Voxzogo? Have you considered share buybacks given your significant cash balance? - Corey Kasimov (Evercore)

2025Q3: The long-term growth rate target remains high single-digit for enzyme therapies, with Voxzogo's continued growth dependent on competition variables. - Brian Mueller(CFO)

What growth rate do you expect for the enzyme business over the next few years? - Olivia Brayer (Cantor Fitzgerald)

2024Q4: We expect the enzyme business to grow at a high single-digit compound annual growth rate for the foreseeable future. - Brian Mueller(CFO)

Contradiction Point 5

Voxzogo's Revenue Growth Trajectory

It affects the understanding of Voxzogo's revenue growth trajectory and expectations, which are crucial for financial forecasting and investor expectations.

Why were Voxzogo's sales down quarter-over-quarter? What are the business development strategies for Voxzogo, and when will the associated leverage materialize? - Selvine Richter (Goldman Sachs)

2025Q3: Voxzogo revenue remains on track to meet the $900 million to $935 million range. - Brian Mueller(CFO)

VOXZOGO guidance shows growth is slowing. Can you clarify the source of this growth, particularly by region and reimbursement? - Philip Nadeau (TD Cowen)

2024Q4: We expect 2025 to start more strongly than that and then decelerate as we go into the second half of the year as we've previously guided. - Brian Mueller(CFO)