Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — 44th Annual J.P. Morgan Healthcare Conference (January 2026)
26 slides from Biomarin Pharmaceutical's conference presentation, Jan 12, 2026
Slide 1: 44th Annual J.P. Morgan Healthcare Conference
44th Annual J.P. Morgan Healthcare Conference Alexander Hardy Greg Friberg Chief Executive Officer Chief R&D Officer BioMarin Pharmaceutical Inc. January 12, 2026
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 or the company), including, without limitation, statements about: preliminary estimated unaudited fina
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: BioMarin's Unique Capabilities & Profile Unlock Long-term Value
BioMarin’s Unique Capabilities & Profile Unlock Long-term Value Pioneering Innovation in Industry Leading End-to-end Operating Scale Drives Strong Rare Disease Capabilities Financial Outlook Q3’25 YTD Total Revenues 10 80-country presence Marketed products1 $2.3B $0.4B 6 Strong global regulatory app
Slide 5: Our Purpose
OUR PURPOSE Be the biotech leader that translates the promise of genetic discovery into medicines that make a profound impact on the life of each patient. 5
Slide 6: Delivering on our Strategic Priorities over the Last 24 Months
Delivering on our Strategic Priorities over the Last 24 Months VALUE INNOVATION GROWTH COMMITMENT • Executing on full lifecycle • Achieved ~15% total revenue • Two M&A transactions in 20253 innovation opportunities CAGR FY’23 – FY’251 • Increased profitability by > 3x • Accelerated key assets • Sust
Slide 7: 2026 Priorities
2026 Priorities • VOXZOGO for achondroplasia & hypochondroplasia Drive Accelerating Revenue • Enzyme Therapies and PALYNZIQ (12 to 17 y/o) • Amicus Integration • 2 Phase 3 data readouts; 1 filing for Full Approval Deliver a Catalyst-rich Year • 2 label expansions expected • 2 data readouts to suppor
Slide 8: BioMarin Announces Pending Acquisition of Amicus Therapeutics for $4.8 Billion
December 19, 2025: BioMarin Announces Pending Acquisition of Amicus Therapeutics for $4.8 Billion, Expanding Leadership in Rare Diseases 1 Accelerates & Substantially Accretive Exceptional Strategic Fit Diversifies Revenue Beginning in 2027 • Opens access to BioMarin’s 80- • Adds two high-growth pro
Slide 9: Plan to Expand Galafold Market with BioMarin Capabilities
Plan to Expand Galafold Market with BioMarin Capabilities The First and Only Oral Therapy Approved for Fabry Disease Accelerate Penetration Increase Diagnosis and Expand into BioMarin’s into Existing Markets Treatment Rates Global Footprint • Currently approved in > 40 • Focus on diagnosis and treat
Slide 10: Opportunity to Accelerate Global Launch of Pombiliti + Opfolda
Opportunity to Accelerate Global Launch of Pombiliti + Opfolda The Only Two-component Therapy for Pompe Disease Significant Expansion Strengthening Data Drives Exploring Indications to Opportunity Ahead New Starts & Switches Address More Patients • Pombiliti & Opfolda early in global • Growing body
Slide 11: 2025 VOXZOGO Momentum Sets Stage for Skeletal Conditions Outlook
GROWTH 2025 VOXZOGO Momentum Sets Stage for Skeletal Conditions Outlook Skeletal Conditions Lifecycle Strategy to Drive Building on VOXZOGO’s Leadership Long-term Revenue Growth1 in Achondroplasia Skeletal Conditions Revenue1,2 • 10,000 patient years of robust efficacy and safety data; significant e
Slide 12: Accelerating Growth across Enzyme Therapies Business
Accelerating Growth across Enzyme Therapies Business Strong Enzyme Therapies Outlook Key Strategies • Continue to advance PALYNZIQ as a growth Enzyme Therapies Revenue1,2 driver; potential to add adolescent age label expansion to increase eligible patients • Leverage BioMarin’s commercial and manufa
Slide 13: Diverse and Growing Revenues Propel Strong Long-term Outlook
Diverse and Growing Revenues Propel Strong Long-term Outlook Total Revenues1,2 Targeting Sustained Double-Digit Long Term CAGR BMN 401, BMN 333, BMN 3514 Double Digit CAGR ~$3.2B3 $1.9B 2020 2025E 2026E 2030 + 1Chart not drawn to scale; 2Contributions from Galafold & Pombiliti + Opfolda pending tran
Slide 14: Deliver a Catalyst-rich Year: Research and Development Update
2026 Deliver a Catalyst-rich Year Research and Development Update Greg Friberg Chief R&D Officer 14
Slide 15: 2026 Pipeline Highlights Across Therapeutic Areas
2026 Pipeline Highlights Across Therapeutic Areas 3 Major Data Readouts and Planned Submissions for Regulatory Approval 6 New Opportunities across Skeletal Conditions 5 new indications: hypochondroplasia, idiopathic short BMN 401 stature, Noonan syndrome, Turner hypochondroplasia achondroplasia ENPP
Slide 16: BMN 351: Encouraging Dystrophin Responses in First Two Cohorts
BMN 351: Encouraging Dystrophin Responses in First Two Cohorts Study in Duchenne muscular dystrophy (DMD) continues as planned, with 12 mg/kg cohort biopsy data anticipated in 2H’26 Observed and Predicted Dystrophin Expression Study 351-201: Key Highlights LCMS Projections for Absolute Mean Dystroph
Slide 17: BMN 333: Early Results Support Initiation of Phase 2/3 Study
BMN 333: Early Results Support Initiation of Phase 2/3 Study in Children with Achondroplasia Exposure for free CNP (AUC) was increased by over 13X compared to another long-acting CNP agent1 BMN 333 Surpassed 3x Free CNP BMN 333 Phase 2/3 Study Design2 Target in Phase 1 PK Study Enrollment to Begin 1
Slide 18: BMN 401: Potential 1st Disease Modifying Therapy for ENPP1 Deficiency
BMN 401: Potential 1st Disease Modifying Therapy for ENPP1 Deficiency Pivotal Data Anticipated 1H’26 BMN 401 Phase 3 (ENERGY 3) Design Key Highlights Design: Randomized (2:1), Open Label • Registration-enabling study with BMN 401 for ENPP1 deficiency in children 1-12 y/o Randomized Treatment Period
Slide 19: Next Potential VOXZOGO Indication: Hypochondroplasia
Next Potential VOXZOGO Indication: Hypochondroplasia (HCH) Pivotal Data Anticipated 1H’26 Significant Unmet Need in HCH Key Highlights Comorbidity and surgical procedures in individuals • HCH prevalence: 4.2 / 100,000 individuals with HCH vs controls • Total Addressable Patient Population: 14,000 Ov
Slide 20: The Unique Benefits of PALYNZIQ for Adolescents
The Unique Benefits of PALYNZIQ for Adolescents FDA PDUFA Date of Feb. 28, 2026, for Potential Age Expansion (12-17 y/o) PEGASUS Phase 3 Study of PALYNZIQ in Adolescents with PKU Key Highlights Mean % Change from Baseline Following 72 Weeks on Study • PEGASUS Results (week 72): 1500 • Statistically
Slide 21: A Catalyst-rich Outlook for 2026
A Catalyst-rich Outlook for 2026 1H’26 2H’26 BMN 401 PKU PKU ENPP1 deficiency hypochondroplasia hypochondroplasia Adolescents (12-17 y/o) Adolescents (12-15 y/o1) Phase 3 Phase 3 Full Data Submissions for U.S. sBLA PDUFA EU Approval Topline Data (Medical Congress) Regulatory Approval BMN 333 BMN 401
Slide 22: Summary
Summary x g = a x oa Q a
Slide 23: Transformed BioMarin Ready for Next Phase of Growth
Transformed BioMarin Ready for Next Phase of Growth 2024 2025 2026 and Beyond Refreshed Operational Strategic Strategy Implementation Realization Refined Revenue Excellence with Focused Double Digit Revenue Growth Targeting Sustained Double- Digit Revenue CAGR BU Operating Model & Lifecycle As
Slide 24: Thank you. Q&A
iIOMARIN B Thank you. A
Slide 25: Appendix
Appendix Preliminary Financial Update Today
Slide 26: Preliminary Financial Update Today
Preliminary Financial Update Today Item Guidance from Q3’25 Earnings Today’s Update1 FY 2025 Revenue $3,150M - $3,200M ~$3.2B VOXZOGO Revenue $900M - $935M ~$920M Q4 ROCTAVIAN Asset Write Down Not Included ($0.60) – ($0.64)2 (Non-GAAP Diluted EPS Impact) Notes ▪ Q4 Roctavian asset write-downs are es
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