Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — Barclays Global Healthcare Conference 2023
17 slides from Biomarin Pharmaceutical's conference presentation, Mar 15, 2023
Slide 1: Barclays Global Healthcare Conference
Barclays Global Healthcare Conference March 15, 2023 Transforming Lives Through Genetic Discovery Brian Mueller Executive Vice President, Chief Financial Officer
Slide 2: Forward-looking statements
This non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements regarding expectations of growth and profitability, expectations for results of operations and financial guidance
Slide 3: GAAP and Non-GAAP information
This presentation includes both GAAP information and Non-GAAP information. For BioMarin’s 2023 financial guidance, Non-GAAP Income is defined as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense, and certain other specified items. The Company also recently
Slide 4: Solid, Profitable, "IRA-proof" & Growing Base Business
Solid, Profitable, End to End “IRA-proof” & Growing Base Business Business Partnership Transform ▪ Anchoredpartnership to business by durable mindsettherapies enzyme-based with producing nearly $2B of revenue finance acumen and positive cash flows. Steady revenue growth across the portfolio ▪ High b
Slide 5: BioMarin Achieves Record $2.1 Billion in Total Revenues for Full Year 2022
BioMarin Achieves Record $2.1 Billion in Total Revenues for Full Year 2022 Full year sales increased 20%, excluding KUVAN®, and contribute to positive net income for the year Rapid Patient Uptake and Commercial Execution Drives 2022 VOXZOGO® Sales of $169M Expect revenue to more than double in 20231
Slide 6: 2022 Actuals and 2023 Guidance
(In millions, except per share and percentage data) 2022 Actuals 2023 Guidance1 Enzyme Products2 $1,645 $1,700 - $1,850 ROCTAVIANTM -- $100 - $200 VOXZOGO® $169 $330 - $380 Total Revenues $2,096 $2,375 - $2,500 Gross Margin % 76.9% 77.5% - 79.0% R&D % of Revenue 31% 30% - 32% SG&A % of Revenue 41% 3
Slide 7: Anticipated Revenue Growth Enablers
VOXZOGO & ROCTAVIAN >2x $4B - $5B New Base Products $4B-$5B Products ~50% Mid-Decade ~50% $1.85B $2.1B 2021 2022 Mid- End of Decade Decade All other products Anticipated Revenue Growth Enablers Continued growth within Base indication launch of Regulatory approval of U.S. Expansion of VOXZOGO & base
Slide 8: VOXZOGO Achondroplasia Market Opportunity and Launch Excellence
A 1,000+ Additional Children Eligible for Treatment upon Anticipated U.S. and E.U. Age Expansions VOXZOGO Achondroplasia Market Opportunity Launch Excellence 2022 Commercial Execution $3B+ Annual • 1 year anniversary 800 18,000+ Market • Over 1,000 children treated at ~17,000 300 Opportunity year-en
Slide 9: ROCTAVIAN Hemophilia A Market Opportunity
Strategic Market Hemophilia A Base Indicated1 Launch Market Market Access Goal Germany 1Q 2023 $14B+ ~7,000 ~13,000 Cumulative Market Italy 2H 2023 Opportunity France 2H 2023 ~3,000 U.S. 2023 ~3,000 Japan Mid-Decade EMA Indicated U.S. Indicated ROW Indicated Hem A Indicated Hem A Indicated Patients
Slide 10: U.S. Market Access Plans and Regulatory Progress
U.S. Market Access Plans Advancing: • ICER final assessment confirmed assumed value of ROCTAVIAN at $2.5M • Approached by multiple significant U.S. insurers indicating intent to implement a favorable policy for ROCTAVIAN reimbursement • U.S. outcomes-based agreements will be offered in the form of a
Slide 11: VOXZOGO / CNP-Mechanism: Other Genetic Short Stature Conditions
VOXZOGO / CNP-Mechanism: Other Genetic Short Stature (GSS) Conditions 600,000+ GSS conditions currently in the clinic: • Hypochondroplasia • CNP Deficiency • Heterozygous NPR2 mutation • Rasopathy 18,000+ • SHOX Achondroplasia Other GSS • Aggrecan Deficiency Patients Patients (Ages 4-17 years and mo
Slide 12: Research and Early Development
Updates coming at R&D Day September 12, 2023 in New York IND- Therapeutic Focus Product Candidate Research Enabling Phase 1 Phase 2 BMN 255 Hyperoxaluria (Small Molecule) BMN 351 DMD (Exon 51 Oligonucleotide) Research and Early Development Musculoskeletal/ BMN 349 A1ATD (Small Molecule) Metabolic Lo
Slide 13: Products Approved in the Last Years
5 Products Approved in the Last 8 Years Enzyme Replacement Therapies Achondroplasia Phenylketonuria Hemophilia A Gene Therapy 13
Slide 14: Accelerating Revenue Through Mid-Decade & Beyond
Near Term Revenue Growth Enablers Accelerating Revenue Through Mid-Decade & Beyond • Launch VOXZOGO and ROCTAVIAN base indications • Achieve regulatory approval for U.S. $4B - $5B Double-digit ROCTAVIAN Growth $2.1B • Expand VOXZOGO and ROCTAVIAN indications • Continue to grow base portfolio 2022 20
Slide 15: Strategic priorities for growth
Maximize Base • Durable base business and launch of two potential blockbuster Indication Revenue products drive opportunity to double revenue by mid-decade • VOXZOGO beyond Achondroplasia opportunity significantly out- Aggressive sizes base indication Life Cycle Management • Studies underway to purs
Slide 16: 2023 Guidance
(In millions, except per share data) 2023 Guidance1 Net Income Diluted Shares Diluted EPS GAAP Net Income & Diluted EPS $155 to $205 200 $0.78 to $1.03 Amortization expense 60 $0.30 Stock-based compensation expense 207 $1.04 Tax effect of adjustments2 (62) ($0.32) Non-GAAP Income & Diluted EPS $360
Slide 17: Thank You!
Thank You! u BiOMARIN NTT
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