Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — Q2 2025 Earnings Presentation
27 slides from Biomarin Pharmaceutical's earnings presentation, Aug 4, 2025
Slide 1: Second Quarter 2025 Earnings
Second Quarter 2025 Earnings August 4, 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements This non-confidential presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, incl
Slide 3: Non-GAAP Financial Measures
Non-GAAP Financial Measures This presentation includes both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, as
Slide 4: Q2'25 Update
Q2’25 Update: Alexander Hardy, President and CEO • Key Business Updates Brian Mueller, Chief Financial Officer • Financial Results Cristin Hubbard, Chief Commercial Officer • Commercial Update Greg Friberg, Chief R&D Officer • R&D Update Q&A 4
Slide 5: Q2'25 Key Business Updates
Q2'25 Key Business Updates Alexander Hardy President and Chief Executive Officer 5
Slide 6: Strong Q2 Performance Across All Aspects of the Business
Strong Q2 Performance Across All Aspects of the Business Strong Q2’25 revenue growth and • BMN 333 Phase 1 data showed >3x (AUC PK2) significant profitability expansion driven by free CNP levels vs. other long-acting CNP3 execution across the business INNOVATION • Inozyme acquisition completed July
Slide 7: BioMarin's Strong 2H 2025 Outlook
BioMarin’s Strong 2H 2025 Outlook Executing on Key Priorities to Deliver Significant Value Creation in 2025 Continued Strong Growth Progressing Prioritized Pipeline Assets Augment Growth with Business Development 7
Slide 8: Q2'25 Financial Results
Q2’25 Financial Results Brian Mueller Executive Vice President, Chief Financial Officer 8
Slide 9: Double-digit Top-line Growth in Q2'25 Y/Y
Double-digit Top-line Growth in Q2’25 Y/Y Total Revenues ($M) VOXZOGO • Y/Y growth fueled by strong global expansion • 2H’25 poised to deliver higher revenue vs. 1H’25 $825 • Expect $900M to $935M revenue in FY’25 Y/Y $712 Enzyme Therapies 221 +20% 184 • Y/Y growth reflective of strong demand and or
Slide 10: Operating Expenses Expected to Increase in 2H'25 vs. 1H'25
Operating Expenses Expected to Increase in 2H’25 vs. 1H’25 Q2 2025 Results Non-GAAP R&D and SG&A Expenses Y/Y • R&D decreased Y/Y, benefiting from focused R&D Q2’25 investment following 2024 strategic review In millions, except percentages Change • SG&A increased Y/Y to support enterprise resource p
Slide 11: Increasing Profitability Driving Cash Flow and Innovation Expansion
Increasing Profitability Driving Cash Flow and Innovation Expansion GAAP Diluted EPS +124% Non-GAAP Diluted EPS +90% $1.23 $0.55 • Q2 results increased at >3x the rate of revenue $0.29 growth, Y/Y, reflecting implementation of Q2 2023 Q2 2024 Q2 2025 operational efficiencies Non-GAAP Diluted EPS1 •
Slide 12: Updated Full-year 2025 Guidance
Updated Full-year 2025 Guidance Prior Guidance Updated Guidance As of May 1, 2025 As of August 4, 2025 (In millions, except per share and % data) Total Revenues1 $3,100 to $3,200 $3,125 to $3,200 Non-GAAP Operating Margin %2 32% to 33% 33% to 34% Non-GAAP Diluted EPS2,3 $4.20 to $4.40 $4.40 to $4.55
Slide 13: Q2'25 Commercial Update
Q2’25 Commercial Update Cristin Hubbard Executive Vice President, Chief Commercial Officer 13
Slide 14: VOXZOGO: Strong Q2 Contributions; Global Expansion Continues
Skeletal Conditions VOXZOGO: Strong Q2 Contributions; Global Expansion Continues Q2 Revenue ($M) Q2 Y/Y revenue growth driven by strong worldwide demand +20% • As of Q2, children across 51 countries had access to VOXZOGO treatment for achondroplasia (ACH) +62% • Targeting access in more than 60 coun
Slide 15: Next Potential VOXZOGO Indication: Hypochondroplasia (HCH)
Skeletal Conditions Next Potential VOXZOGO Indication: Hypochondroplasia (HCH) VOXZOGO Profile to Address HCH Leveraging Global ACH Experience • HCH is a skeletal condition with significant unmet • Engaging with Skeletal Conditions medical need; no genetically targeted therapies community to advance
Slide 16: Enzyme Therapies: Q2 15% Y/Y Led by PALYNZIQ and VIMIZIM
Enzyme Therapies Enzyme Therapies: Q2 15% Y/Y Led by PALYNZIQ and VIMIZIM Enzyme Therapies Q2 Revenue ($M) +15% Y/Y Strong Growth Across Enzyme Therapies +21% • PALYNZIQ 20% Y/Y growth driven by greater numbers of patients titrating to daily maintenance dose and strong adherence • VIMIZIM 21% Y/Y g
Slide 17: Q2'25 Research & Development Update
Q2’25 Research & Development Update Greg Friberg Executive Vice President, Chief Research & Development Officer 17
Slide 18: New Today: BMN 333 Achieves Free CNP Target in PK Study
Skeletal Conditions New Today: BMN 333 Achieves Free CNP Target in PK Study BMN 333 for Multiple Skeletal Conditions Go-Criteria for Phase 2/3 Reached Healthy volunteer Phase 1 study with BMN 333 demonstrated free CNP levels BMN 333 continues to advance to >3x (area-under-the-curve) those registrati
Slide 19: The Unique Benefits of PALYNZIQ to the Adolescent Population
Enzyme Therapies The Unique Benefits of PALYNZIQ to the Adolescent Population for phenylketonuria (PKU) for adolescents As the only enzyme substitute therapy • Submissions for 12 to 17 year age cohort in U.S. for PKU, PALYNZIQ has demonstrated and Europe planned for 2H’25 the ability to lower blood
Slide 20: BMN 401: Potential First-in-Disease Treatment for ENPP1 Deficiency
Enzyme Therapies BMN 401: Potential First-in-Disease Treatment for ENPP1 Deficiency BMN 401 Advancing in Clinic Preparation for Potential Launch Working with patient and HCP BMN 401 Phase 3 (ENERGY 3) trial communities to identify individuals ongoing for the treatment of children with ENPP1 Deficien
Slide 21: Anticipated Key Pipeline Highlights over Next 1-6 Quarters
Anticipated Key Pipeline Highlights over Next 1-6 Quarters 2H 2025 1H 2026 2H 2026 BMN 333 Ph1 PK presentation BMN 333 Ph1 PK data Skeletal BMN 333 Ph2/3 Conditions registration-enabling study initiation VOXZOGO for HCH Ph3 data VOXZOGO for HCH health authority readout submissions for approval PALYN
Slide 22: IOMARIN
IOMARIN B
Slide 23: Appendix
Appendix Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
Slide 24: Reconciliation of GAAP Reported Net Income to Non-GAAP Income
Reconciliation of GAAP Reported Net Income to Non-GAAP Income(1) (1) Certain amounts may not sum or recalculate due to rounding. (2) Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets. (3) These amounts were in
Slide 25: Reconciliation of GAAP and Non-GAAP R&D and SG&A Expenses
Reconciliation of GAAP and Non-GAAP R&D and SG&A Expenses(1) (1) Certain amounts may not sum or recalculate due to rounding. (3) These amounts were included in SG&A and represent severance and restructuring costs related to the Company's 2024 corporate initiatives and the associated organizational r
Slide 26: Reconciliation of GAAP Income from Operations to Non-GAAP Income from Operations
Reconciliation of GAAP Income from Operations to Non-GAAP Income from Operations(1) (1) Certain amounts may not sum or recalculate due to rounding. (2) Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets. (3) Th
Slide 27: Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS
Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS(1) (1) Certain amounts may not sum or recalculate due to rounding. (2) Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets. (3) These amounts were inclu
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