41st Annual J.P. Morgan Healthcare Conference - January 9, 2023 Transforming Lives Through Genetic Discovery
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41st Annual J.P. Morgan
Healthcare Conference
January 9, 2023 Transforming Lives Through
Genetic Discovery
J.J. Bienaimé
Chairman and Chief Executive Officer
This non-confidential presentation contains “forward-looking statements” about the business prospects of BioMarin Pharmaceutical
Inc. (BioMarin), including, without limitation, statements about: the expected financial performance of BioMarin, including, without
limitation, preliminary unaudited total revenues and revenue from sales of VOXZOGO for fiscal year 2022, double-digit revenue
growth in 2023, sustainable growth and profitability, profit margin expansion, mid-decade revenues, and other specified financial
guidance; the markets for BioMarin’s products and product candidates, including, without limitation, the addressable patient
populations in BioMarin territories and commercial market opportunities; and BioMarin’s development of product candidates and
commercialization of products, including, without limitation, IND submissions anticipated in 2023, the outcome of BioMarin's BLA
resubmission for ROCTAVIAN to the FDA, and the commercial launch of ROCTAVIAN. These forward-looking statements are
predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and
uncertainties include, among others: completion of BioMarin’s normal quarter and year-end accounting procedures for the quarter
and the year ended December 31, 2022 and completion of the external audit with respect to such periods by BioMarin’s independent
registered public accounting firm; results and timing of current and planned preclinical studies and clinical trials; BioMarin’s ability to
successfully manufacture its commercial products and product candidates; the content and timing of decisions by the FDA, the
European Commission and other regulatory authorities concerning products and product candidates; BioMarin’s success in the
commercialization of its commercial products; and those factors detailed in the press release issued January 8, 2023, as well as
BioMarin’s filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption
“Risk Factors” in BioMarin’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2022 as such factors may be
updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak
only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-
looking statement, whether as a result of new information, future events or otherwise.
2
Scientific Breakthroughs are Foundational to Our Performance
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 barriers to entry results in lower risk of competition and long-lived revenue
streams
New Large-market Launches Are Significant Near-Term Growth Drivers
▪ VOXZOGO® as first treatment for Achondroplasia – BioMarin’s strongest global launch to date
▪ ROCTAVIAN™ poised to revolutionize treatment for Severe Hemophilia A with First Gene
Therapy to launch in a large, chronically managed population with unmet medical needs
▪ Profit margin expansion expected as new product revenue growth leverages world class
infrastructure
Best-In-Class Innovation Capabilities
▪ Industry-leading R&D teams produced suite of eight internally-developed genetic-based commercial therapies
▪ Fully-integrated and scaled biopharma capabilities: Discovery, Clinical, Regulatory and world-class Manufacturing
▪ Leveraging decades of genetic expertise in current age of rapid genetic disease discovery
▪ Exceptional development success rate applied to deep early-stage pipeline with most assets in Company history
4
$ Millions
Reported Revenue Revenue Growth Operating Margin
GAAP Net Income1
Growth Excluding Kuvan® Expansion1
$1,559 11.2%
$1,396 $1,385
$142
$1,179
+12% +17% +12.3 ppts +$148M
(1.2%) ($6)
Q3 YTD 2021 Q3 YTD 2022 Q3 YTD 2021 Q3 YTD 2022 Q3 YTD 2021 Q3 YTD 2022 Q3 YTD 2021 Q3 YTD 2022
1 Operating Margin Expansion and GAAP net income in 2022 includes $108M one-time pretax benefit from sale of priority review voucher (PRV);
5
“YTD” in all instances denotes results through Q3
VOXZOGO &
ROCTAVIAN
>2x $4B - $5B
New Base
Products $4B-$5B Products
~50% Mid-Decade ~50%
$1.85B $2.09B
2021 20221 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 portfolio VOXZOGO & ROCTAVIAN ROCTAVIAN ROCTAVIAN indications
6 1 Unaudited preliminary 2022 revenue estimate
Maximize Base Aggressive Life
Indication Revenue Cycle Management
Sustainable
Growth &
Profitability
Accelerate Clinical Balance Profits with
Pipeline Reinvestments
7
$ Millions
CAGR1
+14% CAGR1 Product 2021
$1,492 2018-21
$1,347
VIMIZIM® $623 +9%
$1,175
$1,015 NAGLAZYME® $380 +3%
PALYNZIQ® $237 +169%
BRINEURA® $128 +48%
ALDURAZYME® $123 (3%)
Core Products $1,492 +14%
2018 2019 2020 2021 2022E (excluding Kuvan)
Core Product Revenue
(excluding Kuvan)
8 1 Compounded annual growth rate 2018-21
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+ • 1 year anniversary
Annual
800 18,000+ Market • Over 1,000 children treated at
~17,000 300 Opportunity year-end
• 32 active markets to date
Approved
age ranges: • $169M1 expected 2022 revenue
• U.S. 5+ 2023 Opportunities
• Japan all
• EMA 2+ • Continued uptake of patients, and
• ROW 2+ expansion into additional new
markets
Initially EMA U.S. Total Potential ACH
Indicated Expansion Expansion Indicated ACH Indicated • Potential age label expansions in
Patients Age < 2 Age < 5 Patients Market Value the U.S. and Europe
(Approximately 1,000 newborns with achondroplasia born each year in BioMarin territories)
9 1 Unaudited preliminary 2022 revenue estimate
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 Patients2 Patients Patients Market Value Brazil Mid-Decade
10 1 Indicated defined as consistent with Phase 3 eligibility criteria 2 U.S. ROCTAVIAN indication as anticipated 1H 2023“The impact of gene therapy on my day to
day, it has been a life change, it has been
a big change...The peace of mind of
living day to day, without being afraid of
everything. That's the 3 years that I've
had so far. A normal life.”
“No bleeding, not even once.”
ROCTAVIAN Clinical Trial PatientOutcomes Based Agreements 2023
(OBAs) Outlook
• First OBA concluded recently representing • First German patients expected to be
multiple German health insurers treated in the coming weeks
• Additional completed OBAs in Germany • Comfortable with ROCTAVIAN full-year
anticipated in the coming weeks 2023 consensus estimates, assuming
March U.S. approval
• Patient CDx testing underway to determine
ROCTAVIAN eligibility • Full-year 2023 ROCTAVIAN revenue
guidance to be provided in February
12~80% of HCPs likely to adopt ROCTAVIAN within 1 year of availability
ROCTAVIAN could capture ~35% of severe adult hemophilia A patients,
per EU HCPs
~75% of HCPs likely to adopt ROCTAVIAN within 1 year of availability
ROCTAVIAN could capture ~40% of severe adult hemophilia A patients,
per US HCPs
Source: BioMarin commissioned market research; Hemophilia A: Global 301 2+ Year Data Impact Assessment, EU (Germany, France, Italy) and US;
13
April 2022U.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 warranty
U.S. Regulatory Progress:
• FDA completed the Pre-License inspection of our Gene Therapy facility in December; Believe
the agency’s comments and observations are addressable and are preparing for launch
• Clinical site inspections by FDA scheduled
• 3-year Phase 3 results to be submitted to FDA in the coming weeks
14Maximize Base Aggressive Life
Indication Revenue Cycle Management
Sustainable
Growth &
Profitability
Accelerate Clinical Balance Profits with
Pipeline Reinvestments
15VOXZOGO / 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 more than 3
Standard Deviations below average)
Phase 2 Results and Next Clinical & Regulatory Steps Expected Mid-2023
16ROCTAVIAN: Hemophilia A
+$3B $32B
+$9B $29B
+$2B $20B
+$4B $18B
$14B
Initial Indicated Prior Inhibitors Active Inhibitors AAV + Age 15-17
(In the clinic) (In the clinic) (In the clinic)
17 1 ROCTAVIAN market represents a one-time treatment per patient and not annual re-occurring opportunityWholly Owned Manufacturing Capabilities Support Rapid Drug Development and Commercialization
Gene Therapy Facilities Biologic Facilities
• 2017: Novato, California facility commissioned (ISPE 2018 • 1999: Novato, California facility opened • 2011: Shanbally, Ireland facility acquired
Facility of the Year – Project Execution) • Footprint: 200,000ft2
• Footprint: 80,000ft2
• Footprint: 50,000ft2 @2000L Bioreactor Scale • Manufactures: BRINEURA, VIMIZIM
• Manufactures: ALDURAZYME,
• Manufactures: ROCTAVIAN Drug Substance + Sterile DP
NAGLAZYME, PALYNZIQ, VIMIZIM,
VOXZOGO Drug Substance • 2023: Sterile Drug Product Capability
• 2020: New Gene Therapy Pilot Plant commissioned
• Footprint: 6,500 ft2 @500L and 2000L Bioreactor Scale
• Manufactures: New Gene Therapy Clinical Candidates
Substantial Track Record of Global Inspections and cGMP Compliance
18Maximize Base Aggressive Life
Indication Revenue Cycle Management
Sustainable
Growth &
Profitability
Accelerate Clinical Balance Profits with
Pipeline Reinvestments
19Phase 3
6e13 vg/kg dose
In Year 3 In Year 4
N=132/112* N=17**
Mean 18.8 15.2
FVIII Activity
ROCTAVIAN Median 8.4 7.4
PHASE 3 Annualized
Mean 1.0 0.8
3-YEAR and 4-Year Bleeding Rate***
(bleeding episodes Median 0.0 0.0
RESULTS per year)
Annualized FVIII Mean 8.4 11.1
Utilization
(infusions per year) Median 0.0 0.0
*N=132 (FVIII Activity); N=112 (ABR and AFR). Two of these patients discontinued from
the study prior to reaching Year 3. FVIII imputed to be 0 IU/dL; no imputation was
carried out for ABR and AFR.
**N=17. One of these patients discontinued from the study prior to reaching Year 4.
FVIII activity imputed to be 0 IU/dL; no imputation was carried out for ABR and AFR.
***Annualized rate is for treated bleeds.
20AAV payload expression in
human cells
• Non-clinical and clinical studies of AAV episomes
indicates that transgene expression is restricted by a
AAV payload transcripts/ng RNA
transcriptional mechanism
• Screen of approved small molecule drugs modulating
this mechanism identified several promising candidates
that increase transgene expression
• Next steps: Investigate in humans the potential of
these compounds to augment initial expression, to
extend or enhance sustained expression and reignite
expression if lost
Concentration of approved drug tested
relative to Cmax of approved dose
21IND-
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 Long Acting CNP (Peptide)
MSK (Oligonucleotide) 3 Candidates
MSK (Gene Therapy)
Non-Oncology HEM (Biologic)
Hematology HEM (Oligonucleotide)
BMN 331 HAE (AAV Gene Therapy)
BMN 293 MYBPC3 HCM (AAV Gene Therapy)
Cardiovascular
CV (AAV Gene Therapy) 4 Candidates
CV mAB
CNS AAV (Gene Therapy) 4 Candidates
CNS (Oligonucleotide) 4 Candidates
Central Nervous System
CNS (Peptide)
CNS (Biologic)
Total Addressable Market For IND Enabling & Phase I Programs $30B+
22AGXT Deficiency Increased Oxalate Recurrent Kidney Stones
In PH1 Inherited Reduced AGXT function Oxalate crystals lead to
mutations disrupt the causes increased recurrent kidney stones
AGXT gene oxalate and kidney damage
23Multi-ascending dose (MAD) Phase 1/2 underway
Single BMN 255 Dose in HHVs
BMN 255 PH1 & Liver -O- BMN 255 (n=6)
Disease
- - Mean of BMN 255
-∆- Placebo (n = 2)
GO AGT
Glycolate Glyoxylate Glycine
---FDA approved
Oxalate product for PH1
24SERPING1 haploinsufficiency Reduced C1-Inhibitor Episodes of Severe Angioedema • Available therapies for HAE cannot provide enduring compensation for loss of C1-inhibitor activity • “Troughs” of activity or missed treatment can leave individuals with HAE exposed to severe attacks • Pre-clinical studies with BMN331 in SerpinG1 mutant mice and NHPs suggest that a 6 E13 dose of BMN331 may provide ample expression of C1-INH within an acceptable range of variation to durably protect recipients from HAE attacks 25
First 6 E13 dosing
(normal range:19-37 mg/dl)
LLN
6e13vg/kg
C1-INH antigen (mg/ dl)
2e13vg/kg
2e13vg/kg
26 C1-INH antigen levels (mg/dL) by visitTherapeutic Focus Product Candidate / Key Attributes
BMN 351 DMD (Exon 51 Oligonucleotide)
• > 10x fold enhanced potency by targeting novel splice enhancer
• At anticipated muscle concentrations > 20% dystrophin predicted
Musculoskeletal/ Metabolic
BMN 349 A1ATD (Small Molecule)
• Rapidly acting, orally bioavailable, binds to mutant A1AT
• Reduces polymer burden and improves liver health in mouse model
BMN 293 MYBPC3 HCM (Gene Therapy)
Cardiovascular • Cardiac specific C3 gene addition delivered by AAV
• Durable rescue of hypertrophy and function in animal disease model
27Addressable
Therapeutic Focus Product Candidate Patient
Population 1
BMN 351 DMD (Exon 51 Oligonucleotide) ~8K
Musculoskeletal / Metabolic BMN 255 Hyperoxaluria (Small Molecule) ~800K
BMN 349 A1ATD (Small Molecule) ~52K
BMN 331 HAE (Gene Therapy) ~13K
Cardiovascular
BMN 293 HCM (Gene Therapy) ~180K
28 1 Clinically addressable patient population across BioMarin territories5 Products Approved in the Last 8 Years
Enzyme Replacement Therapies Achondroplasia
Phenylketonuria Hemophilia A Gene Therapy
29Maximize Base Aggressive Life
Indication Revenue Cycle Management
Sustainable
Growth &
Profitability
Accelerate Clinical Balance Profits with
Pipeline Reinvestments
30Near 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
$2.09B
• Expand VOXZOGO and ROCTAVIAN
indications
• Continue to grow base portfolio
20221 2023E Mid- End of
Decade Decade
Financial Expectations
$4B - $5B of Continuing to Invest in R&D Committing to Large
Revenue in Mid-Decade Pipeline Advancements BioPharma Peer Profit Margins
31 1 Unaudited preliminary 2022 revenue estimateGAAP to Non-GAAP Reconciliation P&L Guidance Measures
GAAP Net Income P&L Line GAAP Non-GAAP
Interest income (expense), net Total Revenue n n
Provision for (benefit from) income
Gross Profit
taxes1 n
% of Revenue
Depreciation expenses
R&D % of
n
Revenue
Amortization expense2 n
SG&A % of
n
Stock-based compensation expense n Revenue
Significant non-recurring items n Net Income (Loss) n n
Non-GAAP Net Income Diluted EPS n n
32 1 2
We will continue to adjust for tax effects of individual non-GAAP adjustments but not the entirety of income taxes To include amortization of intangible assets onlyMaximize 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 pursue ROCTAVIAN indications that more than
double the base indication
Accelerate • Proven track record applied to the most early-stage development
Clinical programs in Company history expected to drive revenue growth
Pipeline throughout decade and beyond
Balance • Plans for sustainable profitability growth and cash accumulation to
Profits With enable a sound capital allocation strategy focused on maximizing
Reinvestment shareholder return
3334
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