Biomarin Pharmaceutical Inc. · NASDAQ:BMRN
Biomarin Pharmaceutical — Investor Day (September 2023)
73 slides from Biomarin Pharmaceutical's investor day presentation, Sep 12, 2023
Slide 1: Welcome R&D Day 2023
WELCOME R&D DAY 2023 September 12, 2023
Slide 2: Welcome and Opening Remarks
Welcome and Opening Remarks Jean-Jacques Bienaimé Chairman and Chief Executive Officer 2
Slide 3: Delivering top and bottom-line growth while fueling a sustainable pipeline
Delivering top and bottom-line growth while fueling a sustainable pipeline of innovative products Optimizing Prioritizing Life Revenue Cycle Management Focusing on Proof of Balancing Profits & Concepts and the Reinvestments Early-Stage Pipeline 3
Slide 4: History of Innovation & Success; Breakthrough Possibilities; Investment in Future Growth
HISTORY OF INNOVATION & B R E A K T H R OU G H INVESTMENT IN FUTURE SUCCESS P OS S I B I L I TIE S G R OW T H Industry Leading Track Record Profitable Enzyme Business Plus Ability to Achieve Goal of Both of Translating Genetic Launches of Two Potential Profit Margin Expansion & Discoveries Into Bloc
Slide 5: History of R&D Success
Four for Four on Major History of R&D Success De-risking Events Over the 8 Approved Products Last Two Years *Market Opportunities > $1 billion annual revenue VOXZOGO 100% EU Approval VOXZOGO Commercialization rate for US Approval BioMarin Phase 3 assets ROCTAVIAN EU Approval ROCTAVIAN US Approval 5
Slide 6: R&D Day 2023: Sustaining the R&D Engine
R&D Day 2023 sustaining the R&D engine Hank Fuchs, MD President, Worldwide Research and Development 6
Slide 7: Agenda
Welcome and Opening Remarks Jean-Jacques Bienaimé – Chairman and Chief Executive Officer Sustaining the R&D Engine Hank Fuchs, MD – President, Worldwide Research & Development ROCTAVIAN Tara Robinson, MD, PhD – Senior Medical Director ROCTAVIAN Panel Drs. Johnny Mahlangu and Guy Young, Professor Ami
Slide 8: BioMarin's approach to genetically enabled development
Leveraging genetic discoveries BioMarin can develop and tools, BioMarin has a clear a targeted therapy that directly or understanding of the underlying proximally addresses the disease mechanism fundamental defect of the disease Study designs use readily The medicine has a assessable biomarkers/ tra
Slide 9: What's Next: BioMarin's Foundation
What’s Next Amplifying value: BioMarin at Present • Growing leverage from genomics Honed and Scaled: revolution • Leverageable base indications, breadth of • Larger clinical portfolio expansion opportunities • Integrated evidence to support value • Genetics enabling more than just perpetual influx o
Slide 10: New Investigational Products
2023 2024 2025 2026 TBA01* BMN 355 LQTS TBA02* New BMN 351 DMD BMN 365 ACM BMN 331 HAE BMN 365 ACM Investigational BMN 349 AATD BMN 333 LA CNP BMN 351 DMD BMN 355 LQTS Products BMN 293 HCM BMN 255 RSF BMN 349 AATD BMN 293 HCM Near- to Longer-Term Progress ROCTAVIAN: Japan, Adolescents, Prior inhibit
Slide 11: Pipeline growth through expansion opportunities
ROCTAVIAN and VOXZOGO base indications feed near-term revenues and fuel 01 pipeline growth through expansion opportunities and leveraged data Next wave of candidate programs genetically enabled, targeted, readily assessable 02 and potentially transformative Genomic insights and tools sustain the inf
Slide 12: ROCTAVIAN program update
ROCTAVIAN program update Tara Robinson, MD, PhD Senior Medical Director, Late-Stage Clinical Development 12
Slide 13: ROCTAVIAN Approved
+) ROCTAVIAN APPROVED! • Approved in 2 major markets – Approved in EU, US • Global commercialization underway in key markets • International expansion underway – Taiwan, Brazil, and Mexico marketing authorizations submitted, and Australia submission by EOY • Japan Clinical Trial Notification submitt
Slide 14: Factor VIII Activity, chromogenic assay
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW Eleven of 13 participants remain free of prophylactic therapy and with accept
Slide 15: Prior and Active Inhibitors, Japan, Adolescents, AAV5+, HDACi
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi Prior and Active HDAC Inhibitors Adolescents Inhibition Japan AAV5+ FVIII GENEr8-
Slide 16: Inhibitor (+): Status and Next Steps
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW Demonstration of Tolerance in Canine Status and Next Steps Model of FVIII inh
Slide 17: Japan: Rationale, Approach, Status and Next Steps
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW Rationale and Approach Status and Next Steps • 5k+ Japanese patients diagnose
Slide 18: Adolescents: Development Rationale and Approach
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW Development Rationale & Approach FVIII levels in mice at equivalent human age
Slide 19: AAV5(+) Participants Show Lower Expression
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW AAV5(+) Participants ( ) Show Lower- to No Expression Rationale and Approach
Slide 20: Plasmapheresis and Antibody Cleaving Enzyme Data
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW Rationale Plasmapheresis: FIX DNA increases Pretreatment with Antibody Cleavi
Slide 21: Prophylactic Corticosteroid Study
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW • Single arm prophylactic corticosteroid (CS) study: CS administered Day 1 th
Slide 22: HDACi Increases Transgene Expression
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi NEW Rationale and Approach Status and Next Steps • While most patients have durab
Slide 23: ROCTAVIAN Program Update Summary
ROCTAVIAN APPROVED ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY ROCTAVIAN OPPORTUNITY US EU Base Indication I n h i b i t o r (+) Japan Adolescents A A V 5 (+) Steroids, HDACi ROCTAVIAN program update summary • Phase 2 study data demonstrate that the trend
Slide 24: ROCTAVIAN Panel
Moderated by: Professor Johnny Mahlangu, Doctor Guy Young, MD Professor Amit Nathwani, Hank Fuchs, MD MBBCh, MMed, FCPath MBChB, FRCP, RCPath, Director, Hemostasis and President, Worldwide Research PhD Director Haemophilia Thrombosis Center and Clinical and Development Comprehensive Care, Charlotte
Slide 25: VOXZOGO program update
VOXZOGO program update Elena Fisheleva MD Executive Medical Director Late-Stage Clinical Development 25
Slide 26: VOXZOGO Approved
+) VOXZOGO APPROVED! • FDA PDUFA (under 5 years) 2023 • CHMP opinion (under 2 years) 2023 • Approved in Japan for treatment from birth; in Brazil and Australia from age 2 • Pursuing regulatory approvals globally – Marketing applications under review in Argentina, Taiwan, Israel, Mexico, Chile, Colom
Slide 27: VOXZOGO Opportunities
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions Hypochondroplasia Select Short Stature Infants with Genetic Conditions Achondroplasia Idiopathic
Slide 28: Annualized Growth Velocity in Infants with Achondroplasia
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions Mean 12-month Interval AGV in Vosoritide-Treated Children is Restoration of substantial proportio
Slide 29: Causes of Short Stature
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions Achondroplasia (ACH) Indication expansion 2023 FGFR3 Hypochondroplasia (HCH) Pivotal study 2024 C
Slide 30: Hypochondroplasia: Rationale and Next Steps
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions NEW Rationale and Approach Status and Next Steps • Pathogenesis similar to achondroplasia caused
Slide 31: Idiopathic Short Stature: Rationale and Waning Effect of Growth Hormone
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions • In short stature conditions without growth hormone (GH) Waning Effect of Growth Hormone over Ti
Slide 32: Idiopathic Short Stature: Status and Next Steps
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions NEW Rationale and Approach Status and Next Steps • ~0.6% of population have a height < -2.5 SD •
Slide 33: Select Genetic Short Stature Conditions
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions NEW Rationale and Approach Status and Next Steps • Noonan's Syndrome, Turner's Syndrome and • Con
Slide 34: VOXZOGO Program Update Summary
VOXZOGO APPROVED VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY VOXZOGO OPPORTUNITY Achondroplasia Infants Hypochondroplasia Idiopathic Short Stature Select Genetic Short Stature Conditions VOXZOGO program update summary • Label expansion in ACH into infants and younger children – Durab
Slide 35: VOXZOGO Panel
Moderated by: Andrew Dauber, MD Melita Irving, MBBS Bradley S. Miller, MD, PhD Jonathan Day, MBBS, PhD Chief of Endocrinology, Consultant in Clinical Director, Division of Group Vice President, Children’s National Hospital Genetics, Guy’s and St. Endocrinology, M Health Fairview Late-Stage Clinical
Slide 36: BMN 255 for Hyperoxaluria in NAFLD
BMN 255 for Hyperoxaluria in NAFLD Dave Jacoby, MD, PhD Group Vice President Head of Experimental Medicine and Early Clinical Development 36
Slide 37: BMN 255: Development Rationale and Transformative Potential
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 38: BMN 255: Evidence to Date and Next Steps
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 39: BMN 331 for Hereditary Angioedema
BMN 331 for Hereditary Angioedema Dave Jacoby, MD, PhD Group Vice President Head of Experimental Medicine and Early Clinical Development 39
Slide 40: BMN 331: Development Rationale and Transformative Potential
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 41: BMN 331: Evidence to Date and Next Steps
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 42: BMN 351 for Duchenne's Muscular Dystrophy
BMN 351 for Duchenne’s Muscular Dystrophy Dave Jacoby, MD, PhD Group Vice President Head of Experimental Medicine and Early Clinical Development 42
Slide 43: BMN 351: Development Rationale and Transformative Potential
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 44: BMN 351: Evidence to Date and Next Steps
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 45: BMN 349 for Alpha-1 Antitrypsin Deficiency
BMN 349 for Alpha-1 Antitrypsin Deficiency Dave Jacoby, MD, PhD Group Vice President Head of Experimental Medicine and Early Clinical Development 45
Slide 46: BMN 349: Development Rationale and Transformative Potential
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 47: BMN 349: Evidence to Date and Next Steps
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 48: BMN 293 for MYBPC3 Hypertrophic Cardiomyopathy
BMN 293 for MYBPC3 Hypertrophic Cardiomyopathy Kevin Eggan, PhD Chief Scientific Officer 48
Slide 49: BMN 293: Development Rationale and Transformative Potential
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 50: BMN 293: Evidence to Date and Next Steps
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 51: BMN 365 for PKP2 Arrhythmogenic Cardiomyopathy
BMN 365 for PKP2 Arrhythmogenic Cardiomyopathy Kevin Eggan, PhD Chief Scientific Officer 51
Slide 52: BMN 365: Development Rationale and Transformative Potential
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 53: BMN 365: Evidence to Date and Next Steps
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 54: BMN 355 for Long-QT Syndrome
BMN 355 for Long-QT Syndrome Kevin Eggan, PhD Chief Scientific Officer 54
Slide 55: BMN 355: Development Rationale and Transformative Potential
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 56: BMN 355: Evidence to Date and Next Steps
BMN 255 BMN 331 BMN 351 BMN 349 BMN 293 BMN 365 BMN 355 s m all m olecule AAV gene therapy oligonucleotide s m all m olecule AAV gene therapy AAV g e n e t h e r a py m onoclonal antibody for NAFLD with AGT for hereditary angioedema for exon51 Duchenne’s for alpha-1 antitrypsin for MYBPC3 hypertroph
Slide 57: R&D Day 2023: Sustainable Pipeline
R&D Day 2023 sustainable pipeline Kevin Eggan, PhD Chief Scientific Officer 57
Slide 58: Human genetic information is growing faster than it is understood
• The catalog of human genetic information is growing faster than it is being understood, creating opportunities for therapeutic application and discovery • Determining the impact of genetic variation on patients is allowing BioMarin to better identify more patients to treat with our pipeline and po
Slide 59: Number of human single nucleotide sequence variants
1200 Number of human single nucleotide sequence 1000 800 variants (millions) 600 400 200 0 2005 05/06 10/05 2006 03/07 2007 2008 2009 10/07 04/08 2010 05/09 04/10 2011 09/10 08/11 11/11 2012 06/12 2013 2014 04/13 05/14 2015 10/14 06/15 2016 11/15 04/16 2017 11/16 02/17 10/17 2021 06/21 Date 59 BioMa
Slide 60: Number of Variants in MYBPC3
• Wider use of diagnostic exome sequencing and machine learning (ML) approaches has increased: – Number of known mutations in MYBPC3 Number of Variants – Number of likely addressable patients Mutation that clearly damages protein and may be used for diagnosis in future Mutation with unclear impact o
Slide 61: Accuracy of Stature Prediction
Number of Genes Known to be Associated Accuracy of Stature Prediction with Human Height over Time Increased with Polygenic Score 8000 7209 Accuracy of Stature Prediction (%) 7000 Combining 6000 polygenic 5000 scores with 4000 parental 3290 height twice 3000 as accurate as 2000 widely-used 1000 423 p
Slide 62: Number of human genetic variants identified
300 Pathogenic to gene function Likely pathogenic Benign to gene function Number of human genetic variants identified (100s) Likely benign 200 Unknown impact Identification of DNA sequence variation happening much faster 100 than its function can be reported 2015 2016 2017 2018 2019 2020 62 Year Fay
Slide 63: CNP receptor variants and adult height
Lab quantification of DNA variant impact on pathway CNP receptor variants predictive of adult height 2 activity by location on CNP receptor gene sequence1 4 3 Increase in function 2 Height (SDs) 1 0 -1 Decrease in function -2 -3 -4 Modest Large Decrease of Non-variant Increase of increase of functio
Slide 64: Genes associated with kidney stone hospitalization
Number of genes known to be associated with kidney stone hospitalization over time 48 Number of Genes 20 14 1 1 3 2 2009 2010 2012 2015 2019 2023 Urabe et al Oddson et al Tanikawa et al 64 Thorleifsson et al Howles et al BioMarin Gudbjartsson et al
Slide 65: Translating Genetic Discoveries into Transformative Medicines
Translating Genetic Discoveries into Transformative Medicines Pipeline Sustainability High PTS Candidates Transformative Medicines via Genetically- via Expertly-Informed using the Optimal Enabled Discovery Screening and Modality Development Virtually limitless influx Proven excellence in the of pote
Slide 66: Genetically enabled pipeline expansion by modality and area
Achondroplasia Hypochondroplasia Select genetic short Idiopathic short stature conditions stature Gene Therapy BMN 331 BMN 293 BMN 365 Central Nervous CLN 2 Genetic epilepsies System Developmental Disorders 66
Slide 67: Research and Early Development
IND- Product Candidate Research Enabling Phase 1 Phase 2 BMN 255 Hyperoxaluria (Small Molecule) BMN 351 DMD (Exon 51 Oligonucleotide) Research and Early Development BMN 349 A1ATD (Small Molecule) BMN 333 Long Acting CNP (Peptide) MSK (Oligonucleotide) 4 Candidates MSK (Gene Therapy) Metabolic (Biolo
Slide 68: Understanding of genetic variation and its impact on new medicines
Understanding of genetic variation and its impact on new medicines 5-10 Increasing use of whole- years Amount of DNA sequencing genome and other sequencing technologies from each person 3-5 Deeper sequencing years with current methods More people of More people of Today western descent diverse ances
Slide 69: Sustainable Growth & Profitability
Sustainable Growth & Profitability Brian Mueller Executive Vice President Chief Financial Officer 69
Slide 70: Sustainable Growth through Strong Cash Generation and Cash Management
Ability to fund investments in Prioritizing R&D spend to focus Leveraging a resilient, indication expansion on opportunities with highest diversified business model and opportunities and largest R&D potential of scientific and mature global infrastructure to pipeline in BioMarin history commercial s
Slide 71: Post Launch Revenue Trajectories
120 Post Launch Revenue Trajectories 100 REVENUE (USD, M) 80 60 40 20 Launch Qtr Q1 Q2 Q3 Q4 Q5 Q6 Q7 Q8 QUARTERS POST LAUNCH 71
Slide 72: Near Term Financial Execution Priorities
Near Term Financial Execution Accelerating Revenue Through Mid-Decade & Beyond Priorities 2017-2021 2022-2023 Mid-Decade Investment Years Sustainable GAAP Profitability Strong Double-Di git Revenue 1 Growth Commit ti ng to Large BioPharma 2 Peer Profit Margins 0% Op Margin % Significant Investme nts
Slide 73: Thank you
Thank you Q&A
BioMarin Pharmaceutical — Investor Day (September 2023) on 6ix