• Full-year 2023 Total Revenues and Earnings Per
Share Financial Guidance Narrowed; VOXZOGO® and
ROCTAVIAN® Net Product Revenue Guidance Adjusted for
Full-year 2023
• VOXZOGO Now Approved for Children without Age
Restrictions in the United States
and for Children Aged 4 Months and Older in Europe
• ROCTAVIAN Reimbursement Network On-track to
Support Global Patient Access and Treatment in 2024 and Beyond;
Final German and Italian Prices Expected in 2023; Over 100
Treatment Eligible Companion Diagnostic Tests Completed
Globally
• For Full-year 2024, BioMarin Expects
Revenues to Approach $3 Billion
Financial Highlights
(in millions of U.S. dollars, except per share data,
unaudited)
|
|
|
Three Months
Ended
September
30,
|
|
Nine Months
Ended
September
30,
|
|
2023
|
|
2022
|
|
%
Change
|
|
2023
|
|
2022
|
|
%
Change
|
|
|
|
|
|
|
|
|
|
|
|
|
Total
Revenues
|
$
581.3
|
|
$
505.3
|
|
15 %
|
|
$ 1,773.0
|
|
$
1,558.5
|
|
14 %
|
|
|
|
|
|
|
|
|
|
|
|
|
Total Enzyme Product
Revenues (1)
|
$
401.5
|
|
$
388.0
|
|
3 %
|
|
$ 1,270.4
|
|
$
1,240.2
|
|
2 %
|
|
|
|
|
|
|
|
|
|
|
|
|
VIMIZIM® Net
Product Revenues
|
$
158.9
|
|
$
155.5
|
|
2 %
|
|
$
525.6
|
|
$
511.7
|
|
3 %
|
VOXZOGO Net Product
Revenues
|
$
123.1
|
|
$
48.3
|
|
155 %
|
|
$
324.2
|
|
$
102.3
|
|
217 %
|
NAGLAZYME®
Net Product Revenues
|
$
108.9
|
|
$
99.5
|
|
9 %
|
|
$
322.0
|
|
$
343.3
|
|
(6) %
|
PALYNZIQ®
Net Product Revenues
|
$
78.9
|
|
$
66.2
|
|
19 %
|
|
$
216.1
|
|
$
182.7
|
|
18 %
|
KUVAN® Net
Product Revenues
|
$
42.9
|
|
$
57.0
|
|
(25) %
|
|
$
144.0
|
|
$
174.0
|
|
(17) %
|
BRINEURA®
Net Product Revenues
|
$
41.0
|
|
$
37.8
|
|
8 %
|
|
$
118.2
|
|
$
111.7
|
|
6 %
|
ALDURAZYME®
Net Product Revenues
|
$
13.8
|
|
$
29.0
|
|
(52) %
|
|
$
88.5
|
|
$
90.8
|
|
(3) %
|
ROCTAVIAN Net Product
Revenues
|
$
0.8
|
|
$
—
|
|
nm
|
|
$
0.8
|
|
$
—
|
|
nm
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net Income (Loss)
(2)
|
$
40.4
|
|
$
(6.7)
|
|
|
|
$
147.3
|
|
$
141.8
|
|
|
Non-GAAP Income
(3)
|
$
89.5
|
|
$
52.0
|
|
|
|
$
310.5
|
|
$
227.6
|
|
|
GAAP Diluted Earnings
(Loss) per Share (EPS)
|
$
0.21
|
|
$
(0.04)
|
|
|
|
$
0.77
|
|
$
0.75
|
|
|
Non-GAAP Diluted EPS
(4)
|
$
0.46
|
|
$
0.27
|
|
|
|
$
1.60
|
|
$
1.20
|
|
|
|
|
September
30,
2023
|
|
December 31,
2022
|
Total cash, cash
equivalents & investments
|
$
1,673.8
|
|
$
1,625.4
|
|
|
(1)
|
Enzyme Products
include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ, and
VIMIZIM.
|
|
|
(2)
|
GAAP Net Income in the
nine months ended September 30, 2022 included a $89.0 million gain,
net of taxes, related to the sale of the Rare Pediatric Disease
Priority Review Voucher (PRV) the company received from the U.S.
Food and Drug Administration (FDA) in connection with U.S. approval
of VOXZOGO.
|
|
|
(3)
|
Non-GAAP Income is
defined by the company as reported GAAP Net Income, excluding
amortization expense, stock-based compensation expense, contingent
consideration, and, in certain periods, certain other specified
items. The company also includes a Non-GAAP adjustment for the
estimated income tax impact of reconciling items. Refer to Non-GAAP
Information beginning on page 9 of this press release for a
complete discussion of the company's Non-GAAP financial information
and reconciliations to the comparable information reported under
U.S. GAAP.
|
|
|
(4)
|
Non-GAAP Diluted EPS is
defined by the company as Non-GAAP Income divided by Non-GAAP
diluted shares outstanding.
|
|
|
nm
|
Not
meaningful
|
SAN
RAFAEL, Calif., Nov. 1, 2023
/PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN)
today announced financial results for the nine months and third
quarter ended September 30, 2023.
"Results in the quarter drove double-digit revenue growth
year-over-year and support BioMarin's full-year 2023 revenue and
profitability objectives, set at the beginning of the year," said
Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of
BioMarin.
"We were very pleased to have recently received FDA approval for
VOXZOGO for children under 5 years of age with achondroplasia,
making it available in the U.S. for children of all ages with open
growth plates. We also recently received approval in Europe to expand VOXZOGO treatment to children
aged 4 months and older. These important age expansions will ensure
that the youngest children treated with VOXZOGO have the
opportunity to experience greater clinical benefit due to a longer
potential treatment window. In parallel with exceptional commercial
and regulatory execution of VOXZOGO during the quarter, we
concluded key reimbursement steps to facilitate ROCTAVIAN treatment
in Europe and the U.S. All
together, these developments drive our expectation that total
BioMarin revenues will approach $3
billion in 2024," said Mr. Bienaimé.
"In Germany, we have tentative agreement with the German Health
Insurance Fund on the final ROCTAVIAN price, which we expect to be
formalized in the coming weeks. With the final German price
expected to be published by year-end, and 60 people in Germany eligible for next steps based on CDx
testing results for AAV5 antibodies, we are encouraged by progress
made in the quarter. We are also pleased to share that price
negotiations with the Italian Medicines Agency are going well and
that we expect a final price by year-end," Mr. Bienaimé added, "In
the U.S. since gaining FDA approval of ROCTAVIAN on June 29, 2023, the commercial team has been
building the reimbursement network to facilitate patient access,
setting the stage for meaningful uptake of ROCTAVIAN in 2024."
Third Quarter Financial Highlights:
- Total Revenues for the third quarter of 2023 were
$581.3 million, an increase of 15%
compared to the same period in 2022. The increase in Total Revenues
was primarily attributed to the following:
- higher VOXZOGO sales volume due to new patients initiating
therapy across all regions;
- higher PALYNZIQ product revenues primarily due to new patients
initiating therapy, particularly in U.S.; partially offset by
- lower ALDURAZYME product revenues primarily driven by
the timing of order fulfillment to Sanofi, and
- lower KUVAN product revenues attributed to increasing generic
competition as a result of the loss of exclusivity in the U.S.
- GAAP and Non-GAAP Net Income of
$40.4 million and $89.5 million resulted in increases of
$47.1 million and $37.5 million for the third quarter of 2023
compared to the same period in 2022, respectively. The increased
net income was primarily due to higher gross profit driven by
increased revenues, as well as lower income tax expense, partially
offset by higher spend in research and development programs to
support both early-stage research and clinical activities and
higher sales and marketing expenses to support the commercial
launch of ROCTAVIAN.
Global Commercial Launches of ROCTAVIAN and
VOXZOGO
- In the U.S., following the June 29,
2023 FDA approval of ROCTAVIAN, a one-time, gene therapy for
the treatment of adults with severe hemophilia A, the company
executed a number of critical steps to drive awareness among
patients, physicians and patient advocates, as well as prepare
Hemophilia Treatment Center (HTC) sites for patient uptake. At the
end of the quarter, payers representing more than 205 million U.S.
lives had published ROCTAVIAN coverage policies. Warranty policies
had been secured by payers representing more than 95 million lives.
As reimbursement and HTC readiness become more connected, U.S.
patient testing and treatment will be more readily accessible.
- Today in Germany, the second
commercial patient was treated with ROCTAVIAN. Over the last
several weeks, the company and the German National Association
of Statuary Health Insurance Funds (GKV) tentatively agreed on a
final ROCTAVIAN price and expect to complete all formalities by
year-end. In Germany,
60 people are eligible for next steps ahead of treatment with
ROCTAVIAN based on CDx testing to determine seronegativity to AAV5.
In Italy, final price negotiations
with the Italian Medicines Agency are going well and are expected
to be formalized by year-end 2023.
- As a result of global delays securing pricing and
reimbursement, and other market preparations for ROCTAVIAN
treatment, and proximity to the holiday season, full-year 2023
guidance has been lowered to less than $10
million.
- At the end of September 2023,
approximately 2,320 children with achondroplasia were being treated
with VOXZOGO across 38 active markets. In the third quarter,
patient growth remained strong worldwide. Based on these trends,
and the expectation that approximately 2,600 children will be
receiving VOXZOGO treatment by year-end, today the company updated
full-year 2023 VOXZOGO guidance to between $435 million and $455
million. Additionally, based on increased fill-finish
manufacturing commitments, VOXZOGO supply is planned to increase
from 2023 levels through the first and second quarters of 2024 and
is expected to be fully unconstrained by mid-year 2024.
- On October 24, 2023, the European
Commission adopted the decision to expand the indication for
VOXZOGO to treat children with achondroplasia aged 4 months and
older with open growth plates. VOXZOGO is approved for the
treatment of children with achondroplasia of all ages with open
growth plates in Japan and the
U.S.
VOXZOGO and ROCTAVIAN Market Expansion
Opportunities
- In the coming weeks, the company plans to begin the pivotal
program with VOXZOGO for the treatment of children with
hypochondroplasia, a condition characterized by impaired bone
growth. Hypochondroplasia is a genetic statural condition caused by
a mutation (gene change) in the fibroblast growth factor receptor-3
(FGFR3) gene. The 6-month observation arm of the study will be
followed by the 52-week randomized, double-blind,
placebo-controlled phase of the 80-participant clinical trial. If
successful, this study is expected to support regulatory approval
in this large indication.
- The company is also preparing to initiate two additional
clinical programs in 2024 with VOXZOGO, one in idiopathic
short stature and one in genetic short stature conditions.
- Additional product expansion opportunities with ROCTAVIAN
continue, including a clinical study investigating ROCTAVIAN
treatment in those with active or prior inhibitors and continued
exploration of methods of administering ROCTAVIAN in people with
pre-existing antibodies against AAV5.
Earlier-stage Development Portfolio On-track; Seven
Product Candidates Advancing
- BMN 255 for hyperoxaluria in chronic liver disease. The
company believes the availability of a potent, orally bioavailable,
small molecule like BMN 255 may be able to significantly reduce
disease and treatment burden in a patient population with
significant unmet need. The company expects to have a determination
of clinical proof of concept in 2024.
- BMN 331 gene therapy for Hereditary Angioedema (HAE) is
in the Phase 1/2 HAERMONY study to evaluate this investigational
AAV5-mediated gene therapy for people living with HAE. The company
expects to dose additional patients with an optimized
corticosteroid regimen with an anticipated clinical proof of
concept determination by 2025.
- BMN 351 for Duchenne Muscular Dystrophy (DMD), is an
antisense oligonucleotide therapy for individuals with exon
51-skip-amenable DMD. The company is currently enabling a global
clinical development plan and expects to have a determination of
clinical proof of concept in 2025.
- BMN 349 for alpha-1 antitrypsin deficiency, an orally
bioavailable, small molecule that preferentially sequesters mutant
protein, preventing polymerization in liver cells that drive the
progressive liver disease form of the illness. The company plans to
initiate a global clinical program with BMN 349 in 2024 and expects
to have a determination of clinical proof of concept in 2025.
- BMN 293 for MYBPC3 hypertrophic cardiomyopathy (HCM).
IND enabling studies are underway and have incorporated pre-IND
feedback from the FDA. The company plans to initiate a global
clinical program with BMN 293 in 2024 and expects to have a
determination of clinical proof of concept in 2026.
- BMN 365 for AAV gene therapy for PKP2 arrhythmogenic
cardiomyopathy. The company is currently conducting IND-enabling
studies and expects to initiate global clinical programs in 2025
with an anticipated clinical proof of concept determination by
2027.
- BMN 355 monoclonal antibody for long-QT syndrome. The
company is currently conducting IND-enabling studies and expects to
initiate global clinical programs in 2025 with an anticipated
clinical proof of concept determination by 2026.
2023 Full-Year
Financial Guidance (in millions, except % and EPS
amounts)
|
|
Item
|
|
Provided July 31,
2023
|
|
Updated November 1,
2023
|
Total
Revenues
|
|
$2,375
|
|
to
|
|
$2,500
|
|
$2,390
|
|
to
|
|
$2,470
|
Enzyme Product
Revenues(1)
|
|
$1,700
|
|
to
|
|
$1,850
|
|
$1,700
|
|
to
|
|
$1,775
|
ROCTAVIAN
Revenues
|
|
$50
|
|
to
|
|
$150
|
|
less than
$10M
|
VOXZOGO
Revenues
|
|
$400
|
|
to
|
|
$440
|
|
$435
|
|
to
|
|
$455
|
|
|
|
|
|
|
|
|
Unchanged
|
Gross Profit
%
|
|
77.5 %
|
|
to
|
|
79 %
|
|
77.8 %
|
|
to
|
|
79 %
|
R&D % of
Revenue
|
|
30 %
|
|
to
|
|
32 %
|
|
Unchanged
|
SG&A % of
Revenue
|
|
35.5 %
|
|
to
|
|
37.5 %
|
|
36 %
|
|
to
|
|
38 %
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net
Income
|
|
$165
|
|
to
|
|
$215
|
|
$170
|
|
to
|
|
$210
|
GAAP Diluted
EPS
|
|
$0.83
|
|
to
|
|
$1.08
|
|
$0.85
|
|
to
|
|
$1.05
|
Non-GAAP
Income
|
|
$370
|
|
to
|
|
$420
|
|
$380
|
|
to
|
|
$410
|
Non-GAAP Diluted
EPS
|
|
$1.85
|
|
to
|
|
$2.10
|
|
$1.90
|
|
to
|
|
$2.05
|
|
|
(1)
|
Enzyme-based Products
include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ and
VIMIZIM.
|
BioMarin will host a conference call and webcast to discuss
third quarter 2023 financial results and full-year financial
guidance today, Wednesday, November 1, 2023, at 4:30 p.m. ET. This event can be accessed through
this link or on the investor section of the BioMarin website
at www.biomarin.com.
U.S./Canada Dial-in
Number: 888-330-3073
|
Replay Dial-in Number:
800-770-2030
|
International Dial-in
Number: 646-960-0683
|
Replay International
Dial-in Number: 647-362-9199
|
No Conference ID:
1816377
|
Conference ID:
1816377
|
About BioMarin
Founded in 1997, BioMarin is a global biotechnology company
dedicated to transforming lives through genetic discovery. The
company develops and commercializes targeted therapies that address
the root cause of genetic conditions. BioMarin's robust research
and development capabilities have resulted in multiple innovative
commercial therapies for patients with rare genetic disorders. The
company's distinctive approach to drug discovery has produced a
diverse pipeline of commercial, clinical, and pre-clinical
candidates that address a significant unmet medical need, have
well-understood biology, and provide an opportunity to be
first-to-market or offer a substantial benefit over existing
treatment options. For additional information, please visit
www.biomarin.com.
Forward-Looking Statements
This press release 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: the expectations of Total
Revenues, Net Product Revenues, Enzyme Product Revenues, Gross
Profit, Research and Development Expense (R&D), Selling,
General and Administrative Expense (SG&A), GAAP Net Income
(Loss), Non-GAAP Income, GAAP Diluted EPS and Non-GAAP Diluted EPS
for the full-year 2023; cash flows from operating activities; the
timing of orders for commercial products; the timing of BioMarin's
clinical development and commercial prospects, including
announcements of data from clinical studies and trials; the
clinical development and commercialization of BioMarin's product
candidates and commercial products, including (i) the potential to
leverage VOXZOGO in conditions beyond achondroplasia, such as
hypochondroplasia, and to expand the indication of VOXZOGO for
children with achondroplasia aged 4 months and older in
Europe, (ii) the results from
clinical studies regarding product expansion opportunities for
ROCTAVIAN, (iii) BioMarin's plans to observe the highest dose of
BMN 255 in a patient population and, if the response is positive,
engage to move to a pivotal Phase 2/3 study in 2024, (iv) with
respect to BMN 331, BioMarin's plan to dose additional patients
with an alternative corticosteroid regimen in late 2023, with an
anticipated proof of concept by 2025, (v) BioMarin's plans to
initiate a global clinical program with BMN 349 in 2024, (vi)
BioMarin's plans to initiate a global clinical program with BMN 293
in 2024, and (vii) BioMarin's plans to initiate global clinical
programs with BMN 365 and BMN 355 in 2025; the potential approval
and commercialization of BioMarin's product candidates, including
commercialization of ROCTAVIAN for the treatment of severe
hemophilia A in the U.S. following FDA approval in June 2023, and the timing of such approval
decisions and product launches, including (i) the anticipated start
and growth of commercial sales of VOXZOGO in additional countries,
and (ii) BioMarin's expectation that EU health authorities take
action on its supplemental marketing application for VOXZOGO in the
coming weeks and the number of additional children that will be
eligible for VOXZOGO in the U.S. and, if such age expansions are
accepted, in Europe; the expected
benefits and availability of BioMarin's product candidates; and
potential growth opportunities and trends, including that BioMarin
expects accelerated growth of VOXZOGO revenues as the product
launch continues in future quarters and that BioMarin expects
growth of ROCTAVIAN revenues as the product's access is expanded in
Europe and following commercial
launch in the U.S.
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: BioMarin's success in the commercialization
of its commercial products, impacts of macroeconomic and other
external factors on BioMarin's operations; results and timing of
current and planned preclinical studies and clinical trials and the
release of data from those 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
each of the described products and product candidates; the market
for each of these products; actual sales of BioMarin's commercial
products; the introduction of generic versions of BioMarin's
commercial products, in particular generic versions of KUVAN; and
those factors detailed in BioMarin's filings with the Securities
and Exchange Commission (SEC), including, without limitation, the
factors contained under the caption "Risk Factors" in BioMarin's
Quarterly Report on Form 10-Q for the quarter ended June 30, 2023 as such factors may be updated by
any subsequent reports. Stockholders 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.
BioMarin®, BRINEURA®, KUVAN®,
NAGLAZYME®, PALYNZIQ®, VIMIZIM®
and VOXZOGO® are registered trademarks of BioMarin
Pharmaceutical Inc., or its affiliates. ROCTAVIAN® is a
trademark of BioMarin Pharmaceutical Inc., with registration in
Europe and pending in the U.S.
ALDURAZYME® is a registered trademark of
BioMarin/Genzyme LLC. All other brand names and service marks,
trademarks and other trade names appearing in this release are the
property of their respective owners.
Contact:
|
|
|
Investors:
|
|
Media:
|
Traci
McCarty
|
|
Marni
Kottle
|
BioMarin
Pharmaceutical Inc.
|
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
|
(650)
374-2803
|
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
September 30,
2023 and December 31, 2022
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
September 30,
2023
|
|
December 31, 2022
⁽¹⁾
|
ASSETS
|
(unaudited)
|
|
|
Current
assets:
|
|
|
|
Cash and cash
equivalents
|
$
785,414
|
|
$
724,531
|
Short-term
investments
|
340,431
|
|
567,006
|
Accounts receivable,
net
|
572,498
|
|
461,316
|
Inventory
|
1,032,159
|
|
894,083
|
Other current
assets
|
224,806
|
|
104,521
|
Total current
assets
|
2,955,308
|
|
2,751,457
|
Noncurrent
assets:
|
|
|
|
Long-term
investments
|
548,002
|
|
333,835
|
Property, plant and
equipment, net
|
1,067,156
|
|
1,073,366
|
Intangible assets,
net
|
302,476
|
|
338,569
|
Goodwill
|
196,199
|
|
196,199
|
Deferred tax
assets
|
1,523,953
|
|
1,505,412
|
Other
assets
|
165,069
|
|
176,236
|
Total
assets
|
$
6,758,163
|
|
$
6,375,074
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
Current
liabilities:
|
|
|
|
Accounts payable and
accrued liabilities
|
$
669,825
|
|
$
572,959
|
Short-term convertible
debt, net
|
493,398
|
|
—
|
Short-term contingent
consideration
|
—
|
|
15,925
|
Total current
liabilities
|
1,163,223
|
|
588,884
|
Noncurrent
liabilities:
|
|
|
|
Long-term convertible
debt, net
|
592,586
|
|
1,083,019
|
Other long-term
liabilities
|
105,457
|
|
100,015
|
Total
liabilities
|
1,861,266
|
|
1,771,918
|
Stockholders'
equity:
|
|
|
|
Common stock, $0.001
par value: 500,000,000 shares authorized; 188,339,482 and
186,250,719 shares issued and outstanding, respectively
|
188
|
|
186
|
Additional paid-in
capital
|
5,542,774
|
|
5,404,895
|
Company common stock
held by the Nonqualified Deferred Compensation Plan (the
NQDC)
|
(10,393)
|
|
(8,859)
|
Accumulated other
comprehensive loss
|
6,257
|
|
(3,867)
|
Accumulated
deficit
|
(641,929)
|
|
(789,199)
|
Total stockholders'
equity
|
4,896,897
|
|
4,603,156
|
Total liabilities and
stockholders' equity
|
$
6,758,163
|
|
$
6,375,074
|
|
|
|
|
(1)
|
December 31, 2022
balances were derived from the audited Consolidated Financial
Statements included in the company's Annual Report on Form 10-K for
the year ended December 31, 2022, filed with the U.S. Securities
and Exchange Commission (SEC) on February 27, 2023.
|
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
Three and
Nine Months Ended September 30, 2023 and
2022
|
(In thousands of
U.S. dollars, except per share amounts)
|
(unaudited)
|
|
|
Three Months
Ended
September
30,
|
|
Nine Months
Ended
September
30,
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
REVENUES:
|
|
|
|
|
|
|
|
Net product
revenues
|
$
568,266
|
|
$
493,348
|
|
$
1,739,390
|
|
$
1,516,533
|
Royalty and other
revenues
|
13,063
|
|
11,996
|
|
33,629
|
|
41,968
|
Total
revenues
|
581,329
|
|
505,344
|
|
1,773,019
|
|
1,558,501
|
OPERATING
EXPENSES:
|
|
|
|
|
|
|
|
Cost of
sales
|
124,745
|
|
116,288
|
|
379,376
|
|
356,379
|
Research and
development
|
191,314
|
|
157,829
|
|
540,523
|
|
476,855
|
Selling, general and
administrative
|
223,928
|
|
216,816
|
|
662,267
|
|
608,270
|
Intangible asset
amortization and contingent consideration
|
15,681
|
|
16,828
|
|
46,975
|
|
50,935
|
Gain on sale of
nonfinancial assets, net
|
—
|
|
—
|
|
—
|
|
(108,000)
|
Total operating
expenses
|
555,668
|
|
507,761
|
|
1,629,141
|
|
1,384,439
|
INCOME (LOSS) FROM
OPERATIONS
|
25,661
|
|
(2,417)
|
|
143,878
|
|
174,062
|
|
|
|
|
|
|
|
|
Interest
income
|
15,740
|
|
4,999
|
|
40,295
|
|
9,324
|
Interest
expense
|
(3,779)
|
|
(4,679)
|
|
(11,237)
|
|
(12,344)
|
Other income (expense),
net
|
4,047
|
|
193
|
|
(3,700)
|
|
(3,908)
|
INCOME (LOSS) BEFORE
INCOME TAXES
|
41,669
|
|
(1,904)
|
|
169,236
|
|
167,134
|
Provision for income
taxes
|
1,291
|
|
4,748
|
|
21,966
|
|
25,324
|
NET INCOME
(LOSS)
|
$
40,378
|
|
$
(6,652)
|
|
$
147,270
|
|
$
141,810
|
EARNINGS (LOSS) PER
SHARE, BASIC
|
$
0.21
|
|
$
(0.04)
|
|
$
0.78
|
|
$
0.77
|
EARNINGS (LOSS) PER
SHARE, DILUTED
|
$
0.21
|
|
$
(0.04)
|
|
$
0.77
|
|
$
0.75
|
Weighted average common
shares outstanding, basic
|
188,219
|
|
185,597
|
|
187,617
|
|
185,009
|
Weighted average common
shares outstanding, diluted
|
191,173
|
|
185,597
|
|
195,042
|
|
192,252
|
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED STATEMENTS OF CASH FLOWS
|
Nine Months Ended
September 30, 2023 and 2022
|
(In thousands of
U.S. dollars)
|
(unaudited)
|
|
|
Nine Months Ended
September 30,
|
|
2023
|
|
2022
|
CASH FLOWS FROM
OPERATING ACTIVITIES:
|
|
|
|
Net income
|
$
147,270
|
|
$
141,810
|
Adjustments to
reconcile net income to net cash used in operating
activities:
|
|
|
|
Depreciation and
amortization
|
77,525
|
|
77,416
|
Non-cash interest
expense
|
3,198
|
|
3,089
|
Amortization of
premium (accretion of discount) on investments
|
(6,781)
|
|
3,741
|
Stock-based
compensation
|
152,244
|
|
149,574
|
Gain on sale of
nonfinancial assets, net
|
—
|
|
(108,000)
|
Loss on equity
investment
|
12,650
|
|
—
|
Deferred income
taxes
|
(20,137)
|
|
(743)
|
Unrealized foreign
exchange loss (gain)
|
5,454
|
|
(16,075)
|
Non-cash changes in
the fair value of contingent consideration
|
—
|
|
2,243
|
Other
|
(224)
|
|
(700)
|
Changes in operating
assets and liabilities:
|
|
|
|
Accounts receivable,
net
|
(131,940)
|
|
(53,752)
|
Inventory
|
(97,948)
|
|
(27,419)
|
Other current
assets
|
(59,389)
|
|
(8,558)
|
Other
assets
|
(20,812)
|
|
12,140
|
Accounts payable and
other short-term liabilities
|
56,333
|
|
(2,398)
|
Other long-term
liabilities
|
14,333
|
|
(3,252)
|
Net cash provided by
operating activities
|
131,776
|
|
169,116
|
CASH FLOWS FROM
INVESTING ACTIVITIES:
|
|
|
|
Purchases of property,
plant and equipment
|
(67,774)
|
|
(85,271)
|
Maturities and sales of
investments
|
751,677
|
|
477,244
|
Purchases of
investments
|
(727,043)
|
|
(457,382)
|
Proceeds from sale of
nonfinancial assets
|
—
|
|
110,000
|
Purchase of intangible
assets
|
(3,141)
|
|
(9,910)
|
Net cash provided by
(used in) investing activities
|
(46,281)
|
|
34,681
|
CASH FLOWS FROM
FINANCING ACTIVITIES:
|
|
|
|
Proceeds from exercises
of awards under equity incentive plans
|
54,548
|
|
43,866
|
Taxes paid related to
net share settlement of equity awards
|
(72,399)
|
|
(50,696)
|
Payments of contingent
consideration
|
(9,475)
|
|
(21,054)
|
Principal repayments of
financing leases
|
(2,241)
|
|
(1,635)
|
Net cash used in
financing activities
|
(29,567)
|
|
(29,519)
|
Effect of exchange
rate changes on cash
|
4,955
|
|
(39)
|
NET INCREASE IN CASH
AND CASH EQUIVALENTS
|
60,883
|
|
174,239
|
Cash and cash
equivalents:
|
|
|
|
Beginning of
period
|
$
724,531
|
|
$
587,276
|
End of
period
|
$
785,414
|
|
$
761,515
|
Non-GAAP Information
The results presented in this press release include both GAAP
information and Non-GAAP information. Non-GAAP Income is defined by
the company as GAAP Net Income (Loss) excluding amortization
expense, stock-based compensation expense, contingent consideration
expense, and, in certain periods, certain other specified items, as
detailed below when applicable. The company also includes a
Non-GAAP adjustment for the estimated tax impact of the reconciling
items. Non-GAAP Diluted EPS is defined by the company as Non-GAAP
Income divided by Non-GAAP diluted shares outstanding
BioMarin regularly uses both GAAP and Non-GAAP results and
expectations internally to assess its financial operating
performance and evaluate key business decisions related to its
principal business activities: the discovery, development,
manufacture, marketing and sale of innovative biologic therapies.
Because Non-GAAP Income, Non-GAAP Diluted EPS and Non-GAAP Diluted
Shares are important internal measurements for BioMarin, the
company believes that providing this information in conjunction
with BioMarin's GAAP information enhances investors' and analysts'
ability to meaningfully compare the company's results from period
to period and to its forward-looking guidance, and to identify
operating trends in the company's principal business. BioMarin also
uses Non-GAAP Income internally to understand, manage and evaluate
its business and to make operating decisions, and compensation of
executives is based in part on this measure.
Non-GAAP Income and its components are not meant to be
considered in isolation or as a substitute for, or superior to
comparable GAAP measures and should be read in conjunction with the
consolidated financial information prepared in accordance with
GAAP. Investors should note that the Non-GAAP information is not
prepared under any comprehensive set of accounting rules or
principles and does not reflect all of the amounts associated with
the company's results of operations as determined in accordance
with GAAP. Investors should also note that these Non-GAAP financial
measures have no standardized meaning prescribed by GAAP and,
therefore, have limits in their usefulness to investors. In
addition, from time to time in the future there may be other items
that the company may exclude for purposes of its Non-GAAP financial
measures; likewise, the company may in the future cease to exclude
items that it has historically excluded for purposes of its
Non-GAAP financial measures. Because of the non-standardized
definitions, the Non-GAAP financial measure as used by BioMarin in
this press release and the accompanying tables may be calculated
differently from, and therefore may not be directly comparable to,
similarly titled measures used by other companies.
The following tables present the reconciliation of GAAP reported
to Non-GAAP adjusted financial information:
Reconciliation of
GAAP Reported Net Income to Non-GAAP
Income(1)
|
(In millions of U.S.
dollars)
|
(unaudited)
|
|
|
Three Months
Ended
September
30,
|
|
Nine Months
Ended
September
30,
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
|
|
|
|
|
|
GAAP Reported Net
Income (Loss)
|
$
40.4
|
|
$
(6.7)
|
|
$
147.3
|
|
$
141.8
|
Adjustments
|
|
|
|
|
|
|
|
Stock-based
compensation expense - COS
|
4.0
|
|
4.4
|
|
13.1
|
|
13.4
|
Stock-based
compensation expense - R&D
|
14.5
|
|
17.1
|
|
49.4
|
|
47.9
|
Stock-based
compensation expense - SG&A
|
29.9
|
|
33.2
|
|
89.8
|
|
88.2
|
Amortization of
intangible assets
|
15.7
|
|
15.9
|
|
47.0
|
|
47.1
|
Contingent
consideration
|
—
|
|
0.9
|
|
—
|
|
3.8
|
Gain on sale of
non-financial assets (2)
|
—
|
|
—
|
|
—
|
|
(108.0)
|
Severance and employee
termination benefits (3)
|
(0.4)
|
|
4.8
|
|
(0.5)
|
|
4.8
|
Loss on investment in
equity securities (4)
|
—
|
|
—
|
|
12.6
|
|
—
|
Income tax effect of
adjustments
|
(14.6)
|
|
(17.6)
|
|
(48.2)
|
|
(11.4)
|
Non-GAAP
Income
|
$
89.5
|
|
$
52.0
|
|
$
310.5
|
|
$
227.6
|
Reconciliation of
Certain GAAP Reported Information to Non-GAAP
Information
|
(in millions, except
per share data)
|
(unaudited)
|
|
|
Three Months
Ended
September
30,
|
|
Nine Months
Ended
September
30,
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
|
|
|
|
|
|
GAAP Diluted
EPS
|
$
0.21
|
|
$
(0.04)
|
|
$
0.77
|
|
$
0.75
|
Adjustments
|
|
|
|
|
|
|
|
Stock-based
compensation expense
|
0.24
|
|
0.28
|
|
0.76
|
|
0.78
|
Amortization of
intangible assets
|
0.08
|
|
0.08
|
|
0.24
|
|
0.24
|
Contingent
consideration
|
—
|
|
—
|
|
—
|
|
0.02
|
Gain on sale of
non-financial assets (2)
|
—
|
|
—
|
|
—
|
|
(0.56)
|
Severance and employee
termination benefits (3)
|
—
|
|
0.02
|
|
—
|
|
0.02
|
Loss on investment in
equity securities (4)
|
—
|
|
—
|
|
0.06
|
|
—
|
Income tax effect of
adjustments
|
(0.07)
|
|
(0.09)
|
|
(0.23)
|
|
(0.05)
|
Non-GAAP Diluted
EPS
|
$
0.46
|
|
$
0.27
|
|
$
1.60
|
|
$
1.20
|
|
|
(1)
|
Certain amounts may not
sum or recalculate due to rounding.
|
|
|
(2)
|
Represents the net gain
in the first quarter of 2022 on the sale to a third party of
the PRV the company received from the FDA in connection with
the U.S. approval of VOXZOGO.
|
|
|
(3)
|
Represents change in
estimates to severance and employee termination benefit charges
in SG&A related to the company's organizational redesign
announced in October 2022. Related to this October 2022
reorganization plan, the company recognized $23.0 million of
expense related to severance and employee termination benefits in
2022, the majority of which was recognized in the fourth
quarter.
|
|
|
(4)
|
Represents the
impairment loss on investment in non-marketable equity securities
recorded in Other income (expense), net in the first quarter of
2023.
|
|
Three Months
Ended
September
30,
|
|
Nine Months
Ended
September
30,
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
|
|
|
|
|
|
GAAP
Weighted-Average Dilutive Shares Outstanding
|
191.2
|
|
185.6
|
|
195.0
|
|
192.3
|
Adjustments
|
|
|
|
|
|
|
|
Issuances under equity
incentive plans
|
—
|
|
3.5
|
|
—
|
|
—
|
Common stock issuable
under Company's convertible debt (1)
|
8.4
|
|
4.0
|
|
4.4
|
|
—
|
Non-GAAP
Weighted-Average Dilutive Shares Outstanding
|
199.6
|
|
193.1
|
|
199.4
|
|
192.3
|
|
|
(1)
|
Common
stock issuable under the company's convertible debt was
excluded from the computation of GAAP Weighted-Average Dilutive
Shares Outstanding when they were anti-dilutive. If converted, the
company would issue 4.0 million shares under the convertible notes
due in 2024 and 4.4 million shares under the convertible notes due
in 2027.
|
|
|
Guidance for the
Year Ended December 31, 2023 (1)(2)
|
|
|
Net
Income
|
|
Diluted
Shares
|
|
Diluted
EPS
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net Income and
Diluted EPS
|
|
$ 170
|
to
|
$ 210
|
|
200
|
|
$0.85
|
to
|
$1.05
|
Amortization of
intangible assets
|
|
60
|
|
|
|
0.30
|
Stock-based
compensation expense
|
|
200
|
|
|
|
1.00
|
Severance and employee
termination benefits
|
|
(0.5)
|
|
|
|
—
|
Loss on investment in
equity securities
|
|
12.6
|
|
|
|
0.06
|
Income tax effect of
adjustments (3)
|
|
(67)
|
|
|
|
(0.34)
|
Non-GAAP Income and
Diluted EPS
|
|
$ 380
|
to
|
$ 410
|
|
200
|
|
$1.90
|
to
|
$2.05
|
|
|
(1)
|
The
adjustments/reconciling items included in this table are presented
to facilitate the reconciliation of Non-GAAP Income and Non-GAAP
Diluted EPS to their closest GAAP financial metrics, GAAP Net
Income and GAAP Diluted EPS. The specific amounts included in each
reconciling line item above represent approximations of the
underlying adjustments from GAAP Net Income to Non-GAAP Income and
from GAAP Diluted EPS to Non-GAAP Diluted EPS. Actual 2023 results
for each reconciling line item may be different, in some cases
materially, than the amounts listed above as a result of
uncertainty regarding, and the potential variability of, those
items.
|
(2)
|
Amounts will sum when
using midpoint of ranges provided
|
(3)
|
Income tax adjustments
represent the estimated income tax impact of each pre-tax non-GAAP
adjustment based on the applicable statutory income tax
rate.
|
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SOURCE BioMarin Pharmaceutical Inc.