BioMarin's 2025 Q3 Earnings Call: Contradictions Emerge on VOXZOGO Sales, 2027 Guidance, Business Development Strategy, and BMN 333 Efficacy

Date of Call: October 27, 2025

Financials Results

• Revenue: Year-to-date total revenue up 11% YOY; updated full-year 2025 total revenue guidance lower end raised to $3.15B (midpoint implies double-digit YOY growth). VOXZOGO 2025 revenue expected $900M–$935M; VOXZOGO YTD revenue +24% and Q3 YOY +15%.
• EPS: $3.50–$3.60 non-GAAP diluted EPS guidance for full year 2025; Q3 included a $221M IPR&D charge (~$1.10 per share pretax) and net ~ $0.15 per share impact to non-GAAP EPS.
• Operating Margin: 26%–27% non-GAAP operating margin guidance for full year 2025; Q3 operating margin down YOY due to the IPR&D charge and higher SG&A investments.

Guidance:

• Full-year 2025 total revenue guidance lower end raised to $3.15B; midpoint implies double-digit YOY growth.
• VOXZOGO 2025 revenue reaffirmed at $900M–$935M; Q4 expected to be the highest VOXZOGO quarter due to contracted orders and patient starts.
• Non-GAAP operating margin guidance 26%–27% and non-GAAP diluted EPS guidance $3.50–$3.60 (net ~ $0.15/share impact from IPR&D).
• 2027 presented as scenario range: lower end aligns with FactSet consensus ~$3.65B excluding ROCTAVIAN; higher-end scenarios can reach ~$4B excluding ROCTAVIAN; no single 2027 point estimate provided.
• Maintain target of ~40% non-GAAP operating margin in 2026 as an objective.

Business Commentary:

* Strong Financial Performance: - BioMarin reported 11% year-to-date total revenue increase compared to the same period in 2024. - The growth was driven by the performance of global enzyme therapies and Skeletal Conditions business units, particularly VOXZOGO's treatment of achondroplasia, which is expected to generate over $900 million in revenues this year.

• VOXZOGO's Market Impact:
• VOXZOGO's year-over-year revenue growth was 24% compared to 2024, with 15% growth in Q3.

• The growth is attributed to significant patient numbers and adherence to therapy worldwide, particularly in international markets where 75% of year-to-date revenue was generated.

• Enzyme Therapies and Skeletal Conditions Business Units:

• The Enzyme Therapies business unit experienced 8% year-to-date growth, with PALYNZIQ's performance resulting in more than 20% growth for the sixth consecutive quarter.

• Growth in these units is driven by increased new patient starts and strong adherence to therapy across all products.

• Guidance and Strategic Initiatives:

• BioMarin raised the lower end of its full year 2025 total revenue guidance, despite a significant $221 million charge related to an acquisition.
• This reflects confidence in continued top-line growth and operational efficiencies, despite uncertainties regarding potential competition in VOXZOGO's market.

Sentiment Analysis:

Overall Tone: Positive

• Management stated they were "very pleased with the strong results" and raised full-year revenue guidance; reported 11% YTD revenue growth, expanding profitability, $369M operating cash flow in Q3 and ~ $2B cash & investments, and reaffirmed VOXZOGO outlook and lifecycle plans — all indicating constructive momentum.

Q&A:

• Question from Philip Nadeau (TD Cowen): My question is on the 2027 guidance. Can you talk a little bit more about the scenarios you see? And maybe more specifically, why are you rescinding the guidance now? What has changed over the last year since it was initially issued?
  Response: Management: They modeled multiple scenarios given new competitive dynamics (potential VOXZOGO competitors), Inozyme acquisition and ROCTAVIAN divestiture; high uncertainty prevents a single 2027 point estimate—lower case assumes two competitors taking significant share, higher case assumes delays/IP wins.

• Question from Salveen Richter (Goldman Sachs): Could you speak to why VOXZOGO sales were down quarter-over-quarter? And then also just help us understand here the business development strategy, given the focus on VOXZOGO and competitive dynamics, when can we start to see BD leverage emerge?
  Response: Management: Q3 VOXZOGO decline was due to timing shifts of large contracted orders (Q4 expected highest VOXZOGO quarter); business development is a top priority—pursuing early-stage through Phase III/pre-commercial/commercial deals and expects to deploy cash to accelerate growth.

• Question from Cory Kasimov (Evercore ISI): Any qualitative commentary on prior long-term mid-2030s guidance re: VOXZOGO opportunity and anticipated CAGR for Enzyme Therapies? And given the cash balance, have you considered buybacks versus BD?
  Response: Management: Enzyme Therapies targeted to sustain high single-digit growth; VOXZOGO outlook remains growth-focused but sensitive to competition so no long-term point estimate given; capital allocation prioritized toward BD over buybacks to maximize top-line growth.

• Question from Joseph Schwartz (Leerink Partners): For upcoming BMN 333 PK data, what exposure levels would give confidence to achieve clinical superiority vs VOXZOGO, and what minimum annualized growth velocity delta might be required?
  Response: Management: Seeking ≥3x increase in free CNP AUC observed in Phase I cohorts; not disclosing a precise AGV delta now, but aiming for a clinically meaningful differentiation that translates to functional/health outcomes beyond linear growth.

• Question from Jessica Fye (JPMorgan): What is the 2027 FactSet consensus excluding ROCTAVIAN? Do longer-term targets (40% operating margin in '26, >$1.25B CFO in '27) still hold? Pipeline: 351 biopsy expectations and hypochondroplasia powering/delta?
  Response: Management: FactSet 2027 consensus $3.725B including $75M ROCTAVIAN, so ~$3.65B excluding ROCTAVIAN; 40% operating margin target for '26 remains the objective and >$1.25B CFO tied to the $4B topline scenario; BMN‑351: 6- and 9‑mg cohorts' biopsies will be reported and target is ~10% dystrophin at steady state (ambitious); hypochondroplasia powered similarly to VOXZOGO achondroplasia effects, with literature suggesting ~1.8 cm/yr growth.

• Question from Julian Pino (Stifel): Can you separate contributions of potential commercial competition versus litigation/IP risks in your scenarios? And on DMD, why set a 10% dystrophin bar when regulatory precedent is lower?
  Response: Management: Scenarios' lower bound assumes two competitors launch successfully; higher bound assumes less competition or IP defense success. For DMD, 10% steady-state dystrophin is set as an ambitious 'true north' based on chemistry and modeling; ~3–5% at 6 months could map to ~10% steady state and is tied to meaningful functional improvement.

• Question from Christopher Raymond (Raymond James): Are you more concerned about TransCon CNP/Ascendis timing and approvals (e.g., PDUFA next month) in your assumptions? And post-Inozyme, how have you improved patient ID/outreach for BMN 401?
  Response: Management: They used mid‑road assumptions reflecting communicated timelines (including upcoming PDUFA) when modeling competitor impact; Inozyme integration is early (<4 months), but BioMarin is already leveraging global commercialization, regulatory and patient‑identification capabilities and plans ENERGY‑3 pivotal work in H1 next year.

• Question from Sean Laaman (Morgan Stanley): Thoughts on orphan drug exclusivity, switching risk for kids <5, and balance sheet/firepower and efficient structure?
  Response: Management: Orphan exclusivity petition filed and outcome tied to PDUFA timing; high adherence and patient satisfaction suggest many on therapy will not switch, while new‑to‑therapy choices differ; estimated deployment firepower ~$4B–$5B (cash ~$2B plus leverage from EBITDA) and capital will be prioritized for BD.

• Question from Unknown Analyst (for Jefferies/Akash Tewari): Why not wait for BridgeBio data before revising scenarios? Is the lower-end case your most conservative view and is $4B the upside?
  Response: Management: They modeled a broad range of scenarios now due to investor interest; lower‑end scenarios (including two competitors launching) still align with consensus, while upside scenarios (including IP wins or portfolio successes) can reach ~$4B excluding ROCTAVIAN; they declined to call any single scenario 'worst' or 'best' and will monitor data as it emerges.

Contradiction Point 1

VOXZOGO Sales Performance and Market Dynamics

It reflects differing explanations for the performance of VOXZOGO sales, which could impact investor perceptions and expectations for the company's revenue growth.

Why did VOXZOGO sales decline quarter-over-quarter? What is your business development strategy? - Salveen Richter (Goldman Sachs)

2025Q3: VOXZOGO sales were down somewhat sequentially, but we do expect to see Q4 be the highest quarter in sales year-to-date. - Brian Mueller(CFO)

What factors contributed to the VOXZOGO revenue guidance adjustment? - Tommie M. Reerink (Goldman Sachs)

2025Q2: We expect full-year VOXZOGO revenue to grow 25% year-over-year. The guidance adjustment reflects shifting large orders outside the U.S. and better visibility on the year's sales, despite bringing down the top end of the range due to timing. - Cristin Hubbard(CMO)

Contradiction Point 2

2027 Revenue Guidance and Competitive Dynamics

It involves changes in financial forecasts, specifically regarding 2027 revenue guidance and competitive dynamics, which are critical indicators for investors.

What scenarios do you see for the 2027 guidance? Why are you rescinding it now? - Philip Nadeau (TD Cowen)

2025Q3: We've decided to clarify that we will not be providing 2027 guidance today in part because we are continuing to weigh various potential competitive dynamics and their impact on our financial outlook. - Brian Mueller(CFO)

What does higher H2 vs. H1 mean for VOXZOGO, and how might revenue trends impact Q2? How might a 25% EU tariff on US pharmaceuticals and a 25% US retaliatory tariff on EU goods impact the business? - Philip Nadeau (TD Cowen)

2025Q1: For 2027, we continue to expect Enzyme Therapy growth to be in the mid-teens range based on the current growth trajectory of the products. - Brian Mueller(CFO)

Contradiction Point 3

Business Development Strategy and Timing

It involves changes in business development strategy and timing, which are critical for strategic growth and investment decisions.

What caused the QoQ decline in VOXZOGO sales? What is the business strategy? - Salveen Richter (Goldman Sachs)

2025Q3: We are focused on early-stage collaborations and Phase III assets. Our strong cash flow lets us prioritize business development. - Alexander Hardy(CEO)

Can you update us on your business development (BD) plans and timeline? When to expect BMN 333 data this year? - Tommie Reerink (Goldman Sachs)

2025Q1: We are excited about BD opportunities, focusing on genetically defined conditions. We plan to complete one deal this year. - Alexander Hardy(CEO)

Contradiction Point 4

Business Development Strategy

It reflects changes in the company's strategic focus and priorities for business development, which are crucial for long-term growth and investment decisions.

Why did VOXZOGO sales decline quarter-over-quarter? What is the business development strategy? - Salveen Richter(Goldman Sachs)

2025Q3: We will be focused on early-stage collaborations and Phase III assets. Both areas require our financial strength and technical expertise. We believe assets are more valuable in our hands. - Alexander Hardy(CEO)

What are your business development priorities for pipeline versus commercial assets and clinical risk appetite, and what trends are expected in the enzyme business for the quarter and year? - Jessica Fye(JPMorgan)

2024Q4: BioMarin is interested in leadership positions in genetically defined conditions, focusing more on preclinical and clinical assets for long-term growth. - Alexander Hardy(CEO)

Contradiction Point 5

BMN 333 Dosing and Efficacy

It involves differing statements about the dosing strategy and expected efficacy of BMN 333, which could impact investor expectations for the product's potential and market positioning.

What exposure levels would demonstrate the superiority of BMN 333 vs. VOXZOGO? - Joseph Schwartz (Leerink Partners)

2025Q3: We aim for clinically meaningful differentiation, considering patient feedback on growth, health, and function. - Gregory Friberg(CDO)

Can you explain the design of the 333 superiority trial and the potential for dosing beyond weekly? - Unidentified Analyst (Jefferies)

2025Q2: We will consider intervals beyond weekly, prioritizing efficacy over dosing frequency. - Gregory Friberg(CDO)