Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — 43rd Annual J.P. Morgan Healthcare Conference (January 2025)
26 slides from Biomarin Pharmaceutical's conference presentation, Jan 13, 2025
Slide 1: 43rd Annual J.P. Morgan Healthcare Conference
43rd Annual J.P. Morgan Healthcare Conference BioMarin Pharmaceutical Inc. Alexander Hardy, President and Chief Executive Officer January 13, 2025
Slide 2: Forward Looking Statements
Forward Looking Statements This 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, including the expecta
Slide 3: Non-GAAP Financial Measures
Non-GAAP Financial Measures This presentation includes both GAAP information and Non-GAAP information. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense, and, in certain periods
Slide 4: BioMarin is the global leader in treating genetically defined conditions
BioMarin is the SCIENTIFIC ACCELERATED EXCELLENCE INNOVATION global leader in Uncovering and solving for the root cause of Rapidly transforming insights into category-defining conditions medicines treating genetically defined conditions. IN-HOUSE GLOBAL EXPERTISE DELIVERY Driving the development Ens
Slide 5: Building on Strong Execution in 2024
Building on Strong Execution in 2024 3Q’24 Year-to-date Financials New Strategy to Drive Value Creation ~$2.1B 27.7% $2.60 +19% +7.6 ppts +63% VALUE INNOVATION GROWTH Total Non-GAAP Non-GAAP COMMITMENT Revenues Operating Diluted Margin1 Earnings Per Share1 Milestones Anticipated over Next 18 Months
Slide 6: Building on Strong Execution in 2024
Building on Strong Execution in 2024 3Q’24 Year-to-date Financials New Strategy to Drive Value Creation ~$2.1B 27.7% $2.60 +19% +7.6 ppts +63% VALUE INNOVATION GROWTH COMMITMENT Total Non-GAAP Non-GAAP Revenues Operating Diluted Margin1 Earnings Per Share1 Milestones Anticipated over Next 18 Months
Slide 7: Building on Strong Execution in 2024
Building on Strong Execution in 2024 3Q’24 Year-to-date Financials New Strategy to Drive Value Creation ~$2.1B 27.7% $2.60 +19% +7.6 ppts +63% VALUE INNOVATION GROWTH Total Non-GAAP Non-GAAP COMMITMENT Revenues Operating Diluted Margin1 Earnings Per Share1 Milestones Anticipated over Next 18 Months
Slide 8: Building on Strong Execution in 2024
Building on Strong Execution in 2024 3Q’24 Year-to-date Financials New Strategy to Drive Value Creation ~$2.1B 27.7% $2.60 +19% +7.6 ppts +63% VALUE INNOVATION GROWTH Total Non-GAAP Non-GAAP COMMITMENT Revenues Operating Diluted Margin1 Earnings Per Share1 Milestones Anticipated over Next 18 Months
Slide 9: VOXZOGO Presents Attractive Global Expansion Opportunities
VOXZOGO Presents Attractive Global Expansion Opportunities with 90% of Total Addressable Patient Population (TAPP) Outside the U.S. Percentage of Infants and Children with Achondroplasia Eligible for VOXZOGO Treatment; (24K Global TAPP) Europe, Japan, Australia, Canada: ~22% of VOXZOGO TAPP Highly P
Slide 10: Plans for Strong Growth in the U.S. with VOXZOGO in Achondroplasia
Plans for Strong Growth in the U.S. with VOXZOGO in Achondroplasia U.S. Achondroplasia TAPP: ~2,400 VOXZOGO U.S. Strategy • Expected to be the only approved product for achondroplasia, from birth, through 2027 U.S. ACH TAPP • Majority of new patients are in 0-5 age group since label expansion in 202
Slide 11: New Publications Highlight Importance of Early VOXZOGO Treatment
New Publications Highlight Importance of Early VOXZOGO Treatment VOXZOGO Treated Children Demonstrated New International Treatment Guidelines Statistically Significantly Improvements in Recommend Early Diagnosis and Treatment Proportionality vs. Untreated Control Arm with VOXZOGO New International T
Slide 12: VOXZOGO for Hypochondroplasia Demonstrated Increase in Annualized Growth Velocity
VOXZOGO for Hypochondroplasia (HCH) Demonstrated a 1.81cm Increase in Annualized Growth Velocity in Phase 2 Potential First-in-class Approved Treatment Option; Tracking to Expected 2027 Launch Annualized Growth Velocity Clearly Defined Go-to-market Strategy • Rapid recruitment of pivotal study, expe
Slide 13: Building Leadership in Skeletal Conditions
Building Leadership in Skeletal Conditions 2025 2026 2027 VOXZOGO expected Continued Global to be available in Commercial Expansion >60 countries VOXZOGO for ACH New guidelines, emerging datasets, and broad label support early treatment with VOXZOGO Anticipated Clinical ✓ first-in-human BMN 333 init
Slide 14: Underlying, Established Strengths of the Enzyme Therapies Business
Underlying, Established Strengths of the Enzyme Therapies Business ~$1.4B Enzyme Therapies Revenues 3Q’24 Year-to-date High Patient Reach and Challenging Path for First-in-category Medicines Treatment Continuity Biosimilars • Built and created global leadership in each therapeutic area • 20-year tra
Slide 15: Strategy to Drive Long-Term Growth across Enzyme Therapies Business
Strategy to Drive Long-Term Growth across Enzyme Therapies Business Targeting High Single-digit CAGR (2023-2034) Find & Start New Patients Enzyme Therapies Revenue • Expand access to diagnostic tools, including gene panels • Implement initiatives to increase diagnosis, including cascade 8% CAGR scre
Slide 16: External Innovation Strategy to Complement Revenue Growth
External Innovation Strategy to Complement Revenue Growth Rich Environment for Business Development ▪ Scientific momentum allows clearly defined drug development ▪ Rich biotech arena with many small companies at R&D stage Leadership in ▪ Biotech remains capital constrained Development & Commercializ
Slide 17: BMN 333: Opportunity to Optimize PK and Clinical Profile in ACH
BMN 333: Opportunity to Optimize PK and Clinical Profile in ACH BMN 333 – Longer-Acting CNP SQ dosing of BMN 333 releases CNP in NHPs 30 ug/kg/wk 150 ug/kg/wk 1000000 1000000 dose dose dose dose dose • Half-life prolongation chemistry similar to (pM) Prodrug Plasma Conc (pM) Conc (pM) 100000 100000
Slide 18: BMN 351 for Duchenne Muscular Dystrophy; Targeting 2025 POC
BMN 351 for Duchenne Muscular Dystrophy; Targeting 2025 POC • Oligonucleotide for Exon-51 Duchenne Preclinical Evidence Published Muscular Dystrophy 1. Muscle cell penetration ✓ WMS 2020 • Novel splice site for exon-51 skip induction, 2. Binding at novel splice site ✓ FUTURES 2022 targeting >10% dys
Slide 19: Anticipated Near-Term R&D Milestones
Anticipated Near-Term R&D Milestones Candidate Condition 18-Month Look-Ahead VOXZOGO Hypochondroplasia 1H25: Pivotal trial fully enrolled VOXZOGO Idiopathic Short Stature 1H25: Phase 2 study enrolling Skeletal Conditions VOXZOGO Noonan/Turner/SHOX Syndrome 1H25: Phase 2 study enrolling BMN 333 ACH &
Slide 20: PURPOSE-DRIVEN. PATIENT-FOCUSED. FUTURE-READY.
PURPOSE-DRIVEN. PATIENT-FOCUSED. FUTURE-READY. 20
Slide 21: Thank you.
ZOMARIN une 5) Q&A Thank you.
Slide 22: Appendix
Appendix Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
Slide 23: Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information(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 amo
Slide 24: Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information(1) Three Months Ended Nine Months Ended Three Nine Months Ended September 30, September30, September 30, September 30, Percent Percent Percent Percent Percent Percent Percent Percent of GAAP of GAAP ofGAAP of GAAP ofGAAP of GA
Slide 25: Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information(1) Three Months Ended Nine Months Ended Three Months Ended Nine Months September 30, September 30, September 30, September 2024 2023 2024 2023 2024 2023 2024 P Diluted EPS GAAP Diluted EPS$ 0.55 $ 0.21 $ 1.56 $ 0.77 $ 0.55 $ 0
Slide 26: Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information(1) Three Months Ended Nine Months Ended Three Nine Months Ended September 30, September 30, September 30, September 30, 2024 2023 2024 2024 2023 2023 2024 2023 AAP Weighted-Average Diluted Shares Outstanding GAAP Weighted-Aver
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