SAN RAFAEL, Calif.,
Nov. 5, 2020 /PRNewswire/ --
Financial Highlights (in millions of U.S.
dollars, except per share data, unaudited)
|
Three Months Ended
September 30,
|
|
Nine Months Ended
September 30,
|
|
2020
|
|
2019
|
|
%
Change
|
|
2020
|
|
2019
|
|
%
Change
|
|
|
|
|
|
|
|
|
|
|
|
|
Total
Revenues
|
$
|
476.8
|
|
|
$
|
461.1
|
|
|
3
|
%
|
|
$
|
1,408.3
|
|
|
$
|
1,249.6
|
|
|
13
|
%
|
|
|
|
|
|
|
|
|
|
|
|
|
Net Product Revenues
Marketed by BioMarin (1)
|
419.8
|
|
|
428.1
|
|
|
(2)
|
%
|
|
1,239.9
|
|
|
1,150.6
|
|
|
8
|
%
|
|
|
|
|
|
|
|
|
|
|
|
|
Vimizim Net Product
Revenues
|
147.9
|
|
|
163.5
|
|
|
(10)
|
%
|
|
401.8
|
|
|
412.0
|
|
|
(2)
|
%
|
Kuvan Net Product
Revenues
|
124.1
|
|
|
120.6
|
|
|
3
|
%
|
|
368.7
|
|
|
340.8
|
|
|
8
|
%
|
Naglazyme Net Product
Revenues
|
76.3
|
|
|
94.4
|
|
|
(19)
|
%
|
|
271.6
|
|
|
279.5
|
|
|
(3)
|
%
|
Palynziq Net Product
Revenues
|
46.1
|
|
|
24.1
|
|
|
91
|
%
|
|
121.4
|
|
|
55.2
|
|
|
120
|
%
|
Brineura Net Product
Revenues
|
25.4
|
|
|
19.8
|
|
|
28
|
%
|
|
75.2
|
|
|
46.8
|
|
|
61
|
%
|
|
|
|
|
|
|
|
|
|
|
|
|
Aldurazyme Net
Product Revenues
|
40.9
|
|
|
22.8
|
|
|
79
|
%
|
|
128.9
|
|
|
73.9
|
|
|
74
|
%
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net Income
(Loss)
|
$
|
784.8
|
|
|
$
|
55.0
|
|
|
|
|
$
|
837.0
|
|
|
$
|
(38.9)
|
|
|
|
GAAP Net Income
(Loss) per Share – Basic
|
$
|
4.33
|
|
|
$
|
0.31
|
|
|
|
|
$
|
4.63
|
|
|
$
|
(0.22)
|
|
|
|
GAAP Net Income
(Loss) per Share – Diluted
|
$
|
4.01
|
|
|
$
|
0.30
|
|
|
|
|
$
|
4.39
|
|
|
$
|
(0.22)
|
|
|
|
Non-GAAP
Income (2)
|
$
|
98.7
|
|
|
$
|
78.1
|
|
|
|
|
$
|
272.6
|
|
|
$
|
120.1
|
|
|
|
|
September
30,
2020
|
|
December
31,
2019
|
Cash, cash
equivalents and investments
|
$
|
1,770.8
|
|
|
$
|
1,165.8
|
|
|
|
(1)
|
Net Product Revenues
Marketed by BioMarin is the sum of revenues from Vimizim, Kuvan,
Naglazyme, Palynziq, Brineura and Firdapse, each calculated in
accordance with Generally Accepted Accounting Principles in the
United States (U.S. GAAP). Sanofi Genzyme (Genzyme) is BioMarin's
sole customer for Aldurazyme and is responsible for marketing and
selling Aldurazyme to third parties. Refer to page 8 for a table
showing Net Product Revenues by product, including Firdapse. In
January 2020, BioMarin divested the Firdapse assets to a third
party in a sale transaction. The sale is reflected in the Company's
consolidated financial statements for the three and nine months
ending September 30, 2020; as a result of the transaction
BioMarin will not recognize Net Product Revenues from Firdapse in
the future.
|
(2)
|
Non-GAAP Income is
defined by the Company as reported GAAP Net Income, excluding net
interest expense, provision for (benefit from) income taxes,
depreciation expense, amortization expense, stock-based
compensation expense, contingent consideration expense and, in
certain periods, certain other specified 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.
|
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the
Company) today announced financial results for the third quarter
ended September 30, 2020.
Net Product Revenues for the third quarter of 2020 increased to
$460.7 million, compared to
$450.9 million in the third quarter
of 2019. The increase in Net Product Revenues was primarily
attributed to the following:
- Palynziq Net Product Revenues increased by $22.0 million driven by a combination of revenue
from U.S. patients achieving maintenance dosing and new patients
initiating therapy;
- Aldurazyme Net Product Revenues increased by $18.1 million due to higher sales volume to
Genzyme;
- Naglazyme and Vimizim Net Product Revenues decreased by an
aggregate of $33.7 million primarily
due to timing of orders placed from Latin
America as well as the impact of missed infusions resulting
from the COVID-19 pandemic.
The increase in GAAP Net Income for the third quarter of 2020,
compared to the same period in 2019 was primarily due to the
following:
- an increase in the benefit from income taxes of $800.8 million primarily due the completion of an
intra-entity transfer of certain intellectual property (IP) rights
to an Irish subsidiary where the Company's Ex-US regional
headquarters are located and has significant manufacturing and
commercial operations, to better align ownership of IP rights with
how the business operates resulting in a tax benefit of
$835.1 million based on the fair
value of the transferred IP rights; and
- decreased research and development (R&D) expense primarily
resulting from decreased clinical manufacturing costs for BMN 307
and lower clinical activity spend for valoctocogene roxaparvovec
gene therapy programs; partially offset by
- an increase in Cost of Sales of $91.8
million primarily attributed to the $81.2 million reserve of valoctocogene
roxaparvovec pre-launch inventory due to delays in anticipated
regulatory approvals; and
- higher selling, general and administrative (SG&A) expense
related to pre-commercialization activities for valoctocogene
roxaparvovec.
Non-GAAP Income for the third quarter of 2020 increased to
$98.7 million, compared to Non-GAAP
Income of $78.1 million for the same
period in 2019. The increase in Non-GAAP Income for the quarter,
compared to the same period in 2019, was attributed to decreased
R&D expense and higher gross profit, excluding the $81.2 million pre-launch inventory charge,
partially offset by higher SG&A expense.
As of September 30, 2020, BioMarin had cash, cash
equivalents and investments totaling approximately $1.8 billion, which includes net proceeds of
$535.8 million from the Company's
May 2020 convertible debt offering,
as compared to $1.2 billion on
December 31, 2019. On October 15,
2020, the Company's 1.50% senior subordinate convertible
notes matured and were settled in cash for approximately
$375.0 million.
Commenting on third quarter 2020 results, Jean-Jacques Bienaimé,
Chairman and Chief Executive Officer of BioMarin, said, "While the
impact of COVID-19 continued through the third quarter, BioMarin
employees remained focused on our mission to serve patients and
ensure the steady supply of our critically-important medicines. In
these continued unpredictable times, the essential nature of our
products to the people who rely on them remains constant."
Mr. Bienaimé continued, "In the third quarter we received
unexpected news on the status of our application with valoctocogene
roxaparvovec for hemophilia A from health authorities, which
resulted in a delay in potential approval timelines. However, we
remain confident in our valoctocogene roxaparvovec gene therapy and
its potential to redefine the treatment paradigm for people with
hemophilia A. We continue to work with the health authorities to
align on next steps toward approval. Our 134-subject Phase 3 study
with valoctocogene roxaparvovec will complete one-year of
observation in all subjects later this month, and we anticipate
sharing top-line results comprising 1 to 2 years of follow-up from
that study, in the first quarter of 2021. We also plan to submit
the complete one-year Phase 3 data to the EMA in the second quarter
of 2021."
"Vosoritide for the treatment of achondroplasia is advancing as
planned with applications under review in both the U.S. and
Europe with potential regulatory
approvals anticipated in 2021. The significant unmet medical need
for children with achondroplasia has enabled BioMarin to build a
multi-pronged dossier of clinical studies. It includes the highly
statistically significant placebo-controlled Phase 3 results,
long-term clinical results in 5 to 18 year-olds from our Phase 2
study, natural history data, and the ongoing study of newborns
through 5 years, which is nearing enrollment completion. The
positive results from our vosoritide clinical programs bolster our
confidence in the potential for this drug to be the first
pharmacological treatment to address the underlying cause of
achondroplasia. Interest in our clinical studies with vosoritide
has been extremely robust, demonstrating that many families are
keen to seek early treatment for their children."
Mr. Bienaimé concluded, "Despite the impact from COVID-19 and
the timing set-back on the potential approval of valoctocogene
roxaparvovec, we remain confident in our business. BioMarin
fundamentals are strong, driven by our global base business of
essential medicines and cash position, but our people and pursuit
and development of innovative therapies will always be our most
important assets."
2020 Full-Year Financial Guidance (in millions, except
%)
Item
|
|
Provided August 4,
2020
|
|
Revised November
5, 2020
|
Total Revenues
(1)
|
|
$1,850
|
to
|
$1,950
|
|
$1,810
|
to
|
$1,870
|
Vimizim Net Product
Revenues
|
|
$530
|
to
|
$570
|
|
$515
|
to
|
$545
|
Kuvan Net Product
Revenues
|
|
$430
|
to
|
$480
|
|
Unchanged
|
Naglazyme Net Product
Revenues
|
|
$360
|
to
|
$400
|
|
$370
|
to
|
$400
|
Palynziq Net Product
Revenues
|
|
$160
|
to
|
$190
|
|
Unchanged
|
Brineura Net Product
Revenues
|
|
$85
|
to
|
$115
|
|
$90
|
to
|
$110
|
|
|
|
|
|
|
|
|
|
Cost of Sales (% of
Total Revenues) (2)
|
|
20%
|
to
|
21%
|
|
26%
|
to
|
28%
|
Research and
Development Expense
|
|
$675
|
to
|
$725
|
|
$630
|
to
|
$670
|
Selling, General and
Administrative Expense
|
|
$780
|
to
|
$830
|
|
$725
|
to
|
$765
|
|
|
|
|
|
|
|
|
|
GAAP Net
Income
|
|
$720
|
to
|
$980
|
|
$760
|
to
|
$820
|
Non-GAAP Income
(3)
|
|
$260
|
to
|
$310
|
|
$280
|
to
|
$330
|
|
|
(1)
|
Revenue guidance
reflects BioMarin's projected impact of the COVID-19 pandemic on
its global revenue sources, mostly in the form of demand
interruptions such as missed patient infusions and delayed
treatment starts for new patients. Total Revenue guidance also
reflects the impact of the valoctocogene roxaparvovec FDA Complete
Response Letter whereby the Company no longer expects any revenue
from valoctocogene roxaparvovec in 2020 and the previously
anticipated October 2020 loss of market exclusivity for Kuvan in
the U.S. Management also notes that the impact of COVID-19 on
revenues is expected to persist into 2021 due primarily to the
effect of delays in new patients initiating therapy.
|
(2)
|
Revised Cost of Sales
guidance for 2020 reflects the incremental charge of $81.2 million
during the third quarter of 2020 related to valoctocogene
roxaparvovec pre-launch inventory reserves.
|
(3)
|
All Financial
Guidance items are calculated based on U.S. GAAP with the exception
of Non-GAAP Income/Loss. 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
corresponding GAAP reported information.
|
Key Program Highlights
- Valoctocogene roxaparvovec gene therapy for severe
hemophilia A: BioMarin is working with the U.S. Food and Drug
Administration to align on steps forward to obtain marketing
approval following the August 18,
2020 Complete Response Letter to the Company's Biologics
License Application for valoctocogene roxaparvovec gene therapy for
severe hemophilia A. The FDA recommended that the Company complete
the Phase 3 study and submit two-year follow-up safety and efficacy
data on all study participants. The Phase 3 study was fully
enrolled in November 2019 and will
complete one-year of follow-up of all patients in November 2020. The Company intends to share the
one-year top-line Phase 3 data in the first quarter of 2021.
Additionally, the EMA recently requested the 52-week results from
the full Phase 3 study cohort of 134 subjects to inform their
benefit-risk assessment. To facilitate this submission within the
EMA regulatory framework, BioMarin recently withdrew the MAA and
plans to resubmit the MAA with these data to the EMA in the second
quarter of 2021.
- Vosoritide for children with achondroplasia: Marketing
applications for vosoritide were recently validated and accepted,
by EMA and FDA, respectively. The CHMP opinion is expected in
Europe in the second half of 2021.
The U.S. New Drug Application for vosoritide is under Standard
review by the FDA with a Prescription Drug User Fee Act target
action date of August 20, 2021.
Vosoritide is an investigational, once daily injection of an analog
of C-type Natriuretic Peptide. If approved, vosoritide would be the
only therapeutic treatment available for children with
achondroplasia.
- Palynziq for Phenylketonuria (PKU): On October 7, 2020 the Company announced that the
FDA approved the supplemental Biologics License Application (sBLA)
to increase the maximum allowable dose of Palynziq
(pegvaliase-pqpz) Injection for treatment of adults with
Phenylketonuria (PKU) to 60 mg daily. Previously, the maximum dose
was 40 mg daily. In the Phase 3 PRISM studies, 19% of study
participants required a 60 mg dose to achieve adequate response to
Palynziq.
Palynziq is indicated to reduce blood Phe concentrations in adults
with phenylketonuria (PKU), who have uncontrolled blood Phe
concentrations greater than 600 μmol/L on existing management.
Palynziq, a PEGylated recombinant phenylalanine ammonia lyase
enzyme, is the first and only approved enzyme substitution therapy
to target the underlying cause of PKU by helping the body to break
down Phe.
- BMN 307 gene therapy product candidate for phenylketonuria
(PKU): On September 24, 2020, the
Company announced that it began dosing participants in PHEARLESS,
the Phase 1/2 study of BMN 307. Both the FDA and EMA granted BMN
307 Orphan Drug Status. Additionally, the FDA has granted Fast
Track status to BMN 307. Product for use in the Phase 1/2 study was
made at commercial scale from BioMarin's award-winning gene therapy
manufacturing facility.
- BMN 331 gene therapy product candidate for Hereditary
Angioedema (HAE): IND-enabling studies are ongoing with BMN
331, BioMarin's third gene therapy candidate, for the treatment of
Hereditary Angioedema (HAE). BioMarin plans to leverage its broad
expertise in developing gene therapies for severe hemophilia A and
PKU to improve efficiencies in the development process of BMN
331.
- DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM):
Pre-clinical studies are underway with DiNA-001 following a
collaboration announced in May 2020
with DiNAQOR, a gene therapy platform company, to develop novel
gene therapies to treat rare genetic cardiomyopathies. DiNAQOR
received an undisclosed upfront payment and is eligible to receive
development, regulatory and commercial milestones on product sales
in addition to tiered royalties on worldwide sales.
BioMarin will host a conference call and webcast to discuss
third quarter 2020 financial results today, Thursday,
November 5, 2020 at 4:30 p.m.
ET. This event can be accessed on the investor section of
the BioMarin website at www.biomarin.com.
U.S./Canada Dial-in
Number: 866.502.9859
|
Replay Dial-in
Number: 855.859.2056
|
International Dial-in
Number: 574.990.1362
|
Replay International
Dial-in Number: 404.537.3406
|
Conference ID:
3291898
|
Conference ID:
3291898
|
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for people with serious and
life-threatening rare diseases and medical conditions. The Company
selects product candidates for diseases and conditions that
represent a significant unmet medical need, have well-understood
biology and provide an opportunity to be first-to-market or offer a
significant benefit over existing products. The Company's portfolio
consists of several commercial therapies and multiple clinical and
preclinical product candidates.
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, Research and Development Expense,
Selling, General and Administrative Expense, Cost of Sales, GAAP
Net Income, Non-GAAP Income, and other specified income statement
guidance for the full-year 2020; the timing of BioMarin's clinical
development and commercial prospects, including announcements of
data from clinical studies and trials, including that the Company
anticipates sharing top-line results from its Phase 3 study with
valoctocogene roxaparvovec in early 2021; the clinical development
and commercialization of BioMarin's product candidates and
commercial products, including (i) BioMarin's plan to submit
complete one-year Phase 3 data to the EMA in the second quarter of
2021, (ii) BioMarin's plan to resubmit its MAA for valoctocogene
roxaparvovec to the EMA in the second quarter of 2021,and (iii) the
potential approval and commercialization of BioMarin's product
candidates, including vosoritide for the treatment of
achondroplasia and valoctocogene roxaparvovec for the treatment of
severe hemophilia A, including timing of such approval
decisions.
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; results and timing of current and
planned preclinical studies and clinical trials, as well as the
potential impact of the COVID-19 pandemic on (i) BioMarin's ability
to continue such preclinical studies and clinical trials and (ii)
the timing of such 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, including
the potential impact of the COVID-19 pandemic on the regulatory
authorities' abilities to issue such decisions and the timing of
such decisions; the market for each of these products; actual sales
of BioMarin's commercial products and the impact that the COVID-19
pandemic may have on such sales; 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, 2020 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® and Vimizim® are registered trademarks of
BioMarin Pharmaceutical Inc., or its affiliates.
Aldurazyme® is a registered trademark of BioMarin/Genzyme
LLC.
Contact:
|
|
|
Investors:
|
|
Media:
|
Traci
McCarty
|
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
|
(415)
455-7451
|
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
September 30,
2020 and December 31, 2019
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
September 30,
2020
|
|
December 31,
2019(1)
|
ASSETS
|
(unaudited)
|
|
|
Current
assets:
|
|
|
|
Cash and cash
equivalents
|
$
|
1,015,675
|
|
|
$
|
437,446
|
|
Short-term
investments
|
489,998
|
|
|
316,361
|
|
Accounts receivable,
net
|
411,712
|
|
|
377,404
|
|
Inventory
|
700,847
|
|
|
680,275
|
|
Other current
assets
|
120,747
|
|
|
130,657
|
|
Total current
assets
|
2,738,979
|
|
|
1,942,143
|
|
Noncurrent
assets:
|
|
|
|
Long-term
investments
|
265,122
|
|
|
411,978
|
|
Property, plant and
equipment, net
|
1,015,062
|
|
|
1,010,868
|
|
Intangible assets,
net
|
427,172
|
|
|
456,580
|
|
Goodwill
|
196,199
|
|
|
197,039
|
|
Deferred tax
assets
|
1,396,547
|
|
|
549,422
|
|
Other
assets
|
119,009
|
|
|
122,009
|
|
Total
assets
|
$
|
6,158,090
|
|
|
$
|
4,690,039
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
Current
liabilities:
|
|
|
|
Accounts payable and
accrued liabilities
|
$
|
480,403
|
|
|
$
|
570,621
|
|
Short-term convertible
debt, net
|
374,290
|
|
|
361,882
|
|
Total current
liabilities
|
854,693
|
|
|
932,503
|
|
Noncurrent
liabilities:
|
|
|
|
Long-term convertible
debt, net
|
1,074,164
|
|
|
486,238
|
|
Long-term contingent
consideration
|
54,103
|
|
|
50,793
|
|
Other long-term
liabilities
|
121,237
|
|
|
98,124
|
|
Total
liabilities
|
$
|
2,104,197
|
|
|
$
|
1,567,658
|
|
Stockholders'
equity:
|
|
|
|
Common stock, $0.001
par value: 500,000,000 shares authorized; 181,492,344 and
179,838,114 shares issued and outstanding, respectively.
|
181
|
|
|
180
|
|
Additional paid-in
capital
|
4,937,791
|
|
|
4,832,707
|
|
Company common stock
held by Nonqualified Deferred Compensation Plan
|
(10,756)
|
|
|
(9,961)
|
|
Accumulated other
comprehensive income
|
10,385
|
|
|
20,164
|
|
Accumulated
deficit
|
(883,708)
|
|
|
(1,720,709)
|
|
Total stockholders'
equity
|
4,053,893
|
|
|
3,122,381
|
|
Total liabilities and
stockholders' equity
|
$
|
6,158,090
|
|
|
$
|
4,690,039
|
|
|
|
(1)
|
December 31, 2019
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, 2019, filed with the U.S. Securities
and Exchange Commission (SEC) on February 27, 2020.
|
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
Nine Months Ended
September 30, 2020 and 2019
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
Three Months
Ended
September 30,
|
|
Nine Months
Ended
September 30,
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
|
(unaudited)
|
|
(unaudited)
|
|
(unaudited)
|
|
(unaudited)
|
REVENUES:
|
|
|
|
|
|
|
|
Net product
revenues
|
$
|
460,741
|
|
|
$
|
450,900
|
|
|
$
|
1,368,816
|
|
|
$
|
1,224,458
|
|
Royalty and other
revenues
|
16,043
|
|
|
10,197
|
|
|
39,522
|
|
|
25,147
|
|
Total net
revenues
|
476,784
|
|
|
461,097
|
|
|
1,408,338
|
|
|
1,249,605
|
|
OPERATING
EXPENSES:
|
|
|
|
|
|
|
|
Cost of
sales
|
188,793
|
|
|
96,949
|
|
|
398,134
|
|
|
263,567
|
|
Research and
development
|
147,053
|
|
|
172,963
|
|
|
471,449
|
|
|
542,195
|
|
Selling, general and
administrative
|
179,450
|
|
|
170,112
|
|
|
542,157
|
|
|
493,024
|
|
Intangible asset
amortization and contingent consideration
|
17,429
|
|
|
17,063
|
|
|
48,018
|
|
|
57,114
|
|
Gain on sale of
nonfinancial assets
|
—
|
|
|
—
|
|
|
(59,495)
|
|
|
(15,000)
|
|
Total operating
expenses
|
532,725
|
|
|
457,087
|
|
|
1,400,263
|
|
|
1,340,900
|
|
INCOME (LOSS) FROM
OPERATIONS
|
(55,941)
|
|
|
4,010
|
|
|
8,075
|
|
|
(91,295)
|
|
|
|
|
|
|
|
|
|
Equity in the loss of
BioMarin/Genzyme LLC
|
(921)
|
|
|
(551)
|
|
|
(1,077)
|
|
|
(780)
|
|
Interest
income
|
4,004
|
|
|
5,340
|
|
|
13,539
|
|
|
17,537
|
|
Interest
expense
|
(9,597)
|
|
|
(2,937)
|
|
|
(24,560)
|
|
|
(16,530)
|
|
Other income,
net
|
1,239
|
|
|
3,960
|
|
|
1,886
|
|
|
6,038
|
|
INCOME (LOSS)
BEFORE INCOME TAXES
|
(61,216)
|
|
|
9,822
|
|
|
(2,137)
|
|
|
(85,030)
|
|
Benefit from income
taxes
|
(846,019)
|
|
|
(45,214)
|
|
|
(839,138)
|
|
|
(46,158)
|
|
NET INCOME
(LOSS)
|
784,803
|
|
|
55,036
|
|
|
837,001
|
|
|
(38,872)
|
|
NET INCOME (LOSS)
PER SHARE, BASIC
|
$
|
4.33
|
|
|
$
|
0.31
|
|
|
$
|
4.63
|
|
|
$
|
(0.22)
|
|
NET INCOME (LOSS)
PER SHARE, DILUTED
|
$
|
4.01
|
|
|
$
|
0.30
|
|
|
$
|
4.39
|
|
|
$
|
(0.22)
|
|
Weighted average
common shares outstanding, basic
|
181,142
|
|
|
179,289
|
|
|
180,592
|
|
|
178,873
|
|
Weighted average
common shares outstanding, diluted
|
197,674
|
|
|
185,924
|
|
|
194,959
|
|
|
178,873
|
|
The following table presents Net Product Revenues by
Product:
Net Product
Revenues by Product
|
(In millions of
U.S. dollars)
|
|
|
Three Months
Ended
September 30,
|
|
Nine Months
Ended
September 30,
|
|
2020
|
|
2019
|
|
%
Change
|
|
2020
|
|
2019
|
|
%
Change
|
|
(unaudited)
|
|
(unaudited)
|
|
|
|
(unaudited)
|
|
(unaudited)
|
|
|
PKU
franchise
|
$
|
170.2
|
|
|
$
|
144.7
|
|
|
18
|
%
|
|
$
|
490.1
|
|
|
$
|
396.0
|
|
|
24
|
%
|
Vimizim
|
147.9
|
|
|
163.5
|
|
|
(10)
|
%
|
|
401.8
|
|
|
412.0
|
|
|
(2)
|
%
|
Naglazyme
|
76.3
|
|
|
94.4
|
|
|
(19)
|
%
|
|
271.6
|
|
|
279.5
|
|
|
(3)
|
%
|
Brineura
|
25.4
|
|
|
19.8
|
|
|
28
|
%
|
|
75.2
|
|
|
46.8
|
|
|
61
|
%
|
Firdapse
(1)
|
—
|
|
|
5.7
|
|
|
(100)
|
%
|
|
1.2
|
|
|
16.3
|
|
|
(93)
|
%
|
Net Product Revenues
Marketed by BioMarin
|
419.8
|
|
|
428.1
|
|
|
|
|
1,239.9
|
|
|
1,150.6
|
|
|
|
Aldurazyme Net
Product Revenues
Marketed by Genzyme
|
40.9
|
|
|
22.8
|
|
|
79
|
%
|
|
128.9
|
|
|
73.9
|
|
|
74
|
%
|
Total Net Product
Revenues
|
$
|
460.7
|
|
|
$
|
450.9
|
|
|
|
|
$
|
1,368.8
|
|
|
$
|
1,224.5
|
|
|
|
|
|
(1)
|
In January
2020, BioMarin divested the Firdapse assets to a third party in a
sale transaction. The sale is reflected in the Company's
consolidated financial statements for the three and nine months
ending September 30, 2020; and as a result of the transaction
BioMarin will not recognize Net Product Revenues from Firdapse in
the future.
|
The following table presents Net Product Revenues for the PKU
Franchise by Product:
Net Product
Revenues by Product for the PKU Franchise
|
(In millions of
U.S. dollars)
|
(unaudited)
|
|
|
Three Months
Ended
September 30,
|
|
Nine Months
Ended
September 30,
|
|
2020
|
|
2019
|
|
%
Change
|
|
2020
|
|
2019
|
|
%
Change
|
|
(unaudited)
|
|
(unaudited)
|
|
|
|
(unaudited)
|
|
(unaudited)
|
|
|
Kuvan
|
$
|
124.1
|
|
|
120.6
|
|
|
3
|
%
|
|
$
|
368.7
|
|
|
340.8
|
|
|
8
|
%
|
Palynziq
|
46.1
|
|
|
24.1
|
|
|
91
|
%
|
|
121.4
|
|
|
55.2
|
|
|
120
|
%
|
Total PKU
franchise
|
$
|
170.2
|
|
|
$
|
144.7
|
|
|
18
|
%
|
|
$
|
490.1
|
|
|
$
|
396.0
|
|
|
24
|
%
|
Non-GAAP Information
The results presented in this press release include both GAAP
information and Non-GAAP information. As used in this release,
Non-GAAP Income is defined by the Company as GAAP Net Income/Loss
excluding net interest expense, provision for (benefit from) income
taxes, depreciation expense, amortization expense, stock-based
compensation expense, contingent consideration expense and, in
certain periods, certain other specified items, as detailed below
when applicable. In addition, BioMarin includes in this press
release the effects of these adjustments on certain components of
GAAP Net Income/Loss for each of the periods presented. In this
regard, Non-GAAP Income and its components, including Non-GAAP Cost
of Sales, Non-GAAP Research and Development expenses, Non-GAAP
Selling, General and Administrative expense, Non-GAAP Intangible
Asset Amortization and Contingent Consideration, Non-GAAP Gain on
the Sale of Intangible Asset and Non-GAAP Benefit From Income Taxes
are statement of operations line items prepared on the same basis
as, and therefore components of, the overall Non-GAAP measures.
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 and its components 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, 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 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 measures;
likewise, the Company may in the future cease to exclude items that
it has historically excluded for purposes of its Non-GAAP measures.
Because of the non-standardized definitions, the Non-GAAP 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 table presents the reconciliation of GAAP Net
Income (Loss) to Non-GAAP Income:
Reconciliation of
GAAP Net Income (Loss) to Non-GAAP Income
|
(In millions of
U.S. dollars)
|
(unaudited)
|
|
|
Three Months
Ended
September 30,
|
|
Nine Months
Ended
September 30,
|
|
Guidance
Year Ending
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
|
December 31,
2020
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net Income
(Loss)
|
$
|
784.8
|
|
|
$
|
55.0
|
|
|
$
|
837.0
|
|
|
$
|
(38.9)
|
|
|
$
|
760.0
|
|
—
|
$
|
820.0
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest expense,
net
|
5.6
|
|
|
(2.4)
|
|
|
11.0
|
|
|
(1.0)
|
|
|
13.0
|
|
—
|
11.0
|
|
Benefit from income
taxes
|
(846.0)
|
|
|
(45.2)
|
|
|
(839.1)
|
|
|
(46.2)
|
|
|
(845.8)
|
|
—
|
(838.8)
|
|
Depreciation
expense
|
11.1
|
|
|
14.4
|
|
|
31.1
|
|
|
42.3
|
|
|
50.0
|
|
—
|
47.0
|
|
Amortization
expense
|
15.5
|
|
|
16.3
|
|
|
46.7
|
|
|
37.2
|
|
|
63.0
|
|
—
|
61.0
|
|
Stock-based
compensation expense
|
50.2
|
|
|
39.2
|
|
|
142.2
|
|
|
121.8
|
|
|
195.4
|
|
—
|
185.4
|
|
Contingent
consideration expense
|
1.9
|
|
|
0.8
|
|
|
1.3
|
|
|
19.9
|
|
|
2.0
|
|
—
|
2.0
|
|
Provision for
inventory reserve, net (1)
|
75.6
|
|
|
—
|
|
|
75.6
|
|
|
—
|
|
|
75.6
|
|
—
|
75.6
|
|
Gain on sale of
nonfinancial assets
|
—
|
|
|
—
|
|
|
(59.5)
|
|
|
(15.0)
|
|
|
(59.5)
|
|
—
|
(59.5)
|
|
Licensed In-Process
R&D (2)
|
—
|
|
|
—
|
|
|
26.3
|
|
|
—
|
|
|
26.3
|
|
—
|
26.3
|
|
Non-GAAP
Income
|
$
|
98.7
|
|
|
$
|
78.1
|
|
|
$
|
272.6
|
|
|
$
|
120.1
|
|
|
$
|
280.0
|
|
—
|
$
|
330.0
|
|
|
|
(1)
|
Represents a $81.2
million charge related to pre-launch valoctocogene roxaparvovec
inventory, net of stock-based compensation, as a result of the
unexpected delays in anticipated regulatory approvals.
|
(2)
|
Represents the
upfront license fee paid to a third party and recognized as R&D
expense in the second quarter of 2020.
|
The following reconciliation of the GAAP reported to the
Non-GAAP information provides the details of the effects of the
Non-GAAP adjustments on certain components of the Company's
operating results for each of the periods presented.
Reconciliation of
Certain GAAP Reported Information to Non-GAAP
Information
|
(In millions of
U.S. dollars)
|
(unaudited)
|
|
|
Three months ended
September 30,
|
|
2020
|
|
2019
|
|
|
|
Adjustments
|
|
|
|
|
|
Adjustments
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP
Reported
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
Non-GAAP
|
|
GAAP
Reported
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
Non-GAAP
|
Cost of
sales
|
$
|
188.8
|
|
|
$
|
—
|
|
|
$
|
(86.0)
|
|
|
$
|
102.8
|
|
|
$
|
96.9
|
|
|
$
|
—
|
|
|
$
|
(4.1)
|
|
|
$
|
92.8
|
|
Research and
development
|
147.1
|
|
|
(5.8)
|
|
|
(15.7)
|
|
|
125.6
|
|
|
173.0
|
|
|
(6.4)
|
|
|
(14.3)
|
|
|
152.3
|
|
Selling, general and
administrative
|
179.5
|
|
|
(5.3)
|
|
|
(24.1)
|
|
|
150.1
|
|
|
170.1
|
|
|
(8.0)
|
|
|
(20.8)
|
|
|
141.3
|
|
Intangible asset
amortization and contingent consideration
|
17.4
|
|
|
(15.5)
|
|
|
(1.9)
|
|
|
—
|
|
|
17.1
|
|
|
(16.3)
|
|
|
(0.8)
|
|
|
—
|
|
Gain on sale of
nonfinancial assets
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
Interest expense,
net
|
(5.6)
|
|
|
5.6
|
|
|
—
|
|
|
—
|
|
|
2.4
|
|
|
(2.4)
|
|
|
—
|
|
|
—
|
|
Benefit from income
taxes
|
(846.0)
|
|
|
846.0
|
|
|
—
|
|
|
—
|
|
|
(45.2)
|
|
|
45.2
|
|
|
—
|
|
|
—
|
|
GAAP Net Income
/Non-GAAP Income
|
$
|
784.8
|
|
|
$
|
(813.8)
|
|
|
$
|
127.7
|
|
|
$
|
98.7
|
|
|
$
|
55.0
|
|
|
$
|
(16.9)
|
|
|
$
|
40.0
|
|
|
$
|
78.1
|
|
|
|
|
Nine months ended
September 30,
|
|
2020
|
|
2019
|
|
|
|
Adjustments
|
|
|
|
|
|
Adjustments
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP
Reported
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
Non-GAAP
|
|
GAAP
Reported
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
Non-GAAP
|
Cost of
sales
|
$
|
398.1
|
|
|
$
|
0.0
|
|
|
$
|
(96.0)
|
|
|
$
|
302.1
|
|
|
$
|
263.6
|
|
|
$
|
0.0
|
|
|
(12.6)
|
|
$
|
251.0
|
Research and
development
|
471.4
|
|
|
(15.2)
|
|
|
(71.7)
|
|
|
384.5
|
|
|
542.2
|
|
|
(23.1)
|
|
|
(43.1)
|
|
476.0
|
Selling, general and
administrative
|
542.2
|
|
|
(15.9)
|
|
|
(76.4)
|
|
|
449.9
|
|
|
493.0
|
|
|
(19.2)
|
|
|
(66.1)
|
|
407.7
|
Intangible asset
amortization and contingent consideration
|
48.0
|
|
|
(46.7)
|
|
|
(1.3)
|
|
|
—
|
|
|
57.1
|
|
|
(37.2)
|
|
|
(19.9)
|
|
—
|
Gain on sale of
nonfinancial assets
|
(59.5)
|
|
|
—
|
|
|
59.5
|
|
|
—
|
|
|
(15.0)
|
|
|
—
|
|
|
15.0
|
|
—
|
Interest expense,
net
|
(11.0)
|
|
|
11.0
|
|
|
—
|
|
|
—
|
|
|
1.0
|
|
|
(1.0)
|
|
|
—
|
|
—
|
Benefit from income
taxes
|
(839.1)
|
|
|
839.1
|
|
|
—
|
|
|
—
|
|
|
(46.2)
|
|
|
46.2
|
|
|
—
|
|
—
|
GAAP Net Income
(Loss)/Non-GAAP Income
|
837.0
|
|
|
(750.3)
|
|
|
185.9
|
|
|
272.6
|
|
|
(38.9)
|
|
|
32.3
|
|
|
126.7
|
|
120.1
|
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SOURCE BioMarin Pharmaceutical Inc.