SAN RAFAEL,
Calif., Aug. 4, 2020
/PRNewswire/ --
Financial Highlights (in millions of U.S.
dollars, except per share data, unaudited)
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
2020
|
|
2019
|
|
%
Change
|
|
2020
|
|
2019
|
|
%
Change
|
|
|
|
|
|
|
|
|
|
|
|
|
Total
Revenues
|
$
|
429.5
|
|
|
$
|
387.8
|
|
|
11
|
%
|
|
$
|
931.6
|
|
|
$
|
788.5
|
|
|
18
|
%
|
|
|
|
|
|
|
|
|
|
|
|
|
Net Product Revenues
Marketed by BioMarin (1)
|
386.8
|
|
|
373.3
|
|
|
4
|
%
|
|
820.1
|
|
|
722.5
|
|
|
14
|
%
|
|
|
|
|
|
|
|
|
|
|
|
|
Vimizim Net Product
Revenues
|
116.7
|
|
|
122.7
|
|
|
(5)
|
%
|
|
253.9
|
|
|
248.5
|
|
|
2
|
%
|
Kuvan Net Product
Revenues
|
122.6
|
|
|
113.3
|
|
|
8
|
%
|
|
244.6
|
|
|
220.2
|
|
|
11
|
%
|
Naglazyme Net Product
Revenues
|
81.0
|
|
|
98.2
|
|
|
(18)
|
%
|
|
195.3
|
|
|
185.1
|
|
|
6
|
%
|
Palynziq Net Product
Revenues
|
40.7
|
|
|
18.8
|
|
|
116
|
%
|
|
75.3
|
|
|
31.1
|
|
|
142
|
%
|
Brineura Net Product
Revenues
|
25.8
|
|
|
14.8
|
|
|
74
|
%
|
|
49.8
|
|
|
27.0
|
|
|
84
|
%
|
Aldurazyme Net
Product Revenues
|
32.3
|
|
|
5.8
|
|
|
457
|
%
|
|
88.0
|
|
|
51.1
|
|
|
72
|
%
|
GAAP Net Income
(Loss)
|
$
|
(29.2)
|
|
|
$
|
(37.4)
|
|
|
|
|
$
|
52.2
|
|
|
$
|
(93.9)
|
|
|
|
GAAP Net Income
(Loss) per Share – Basic
|
$
|
(0.16)
|
|
|
$
|
(0.21)
|
|
|
|
|
$
|
0.29
|
|
|
$
|
(0.53)
|
|
|
|
GAAP Net Income
(Loss) per Share – Diluted
|
$
|
(0.16)
|
|
|
$
|
(0.21)
|
|
|
|
|
$
|
0.28
|
|
|
$
|
(0.53)
|
|
|
|
Non-GAAP
Income (2)
|
$
|
57.4
|
|
|
$
|
17.1
|
|
|
|
|
$
|
173.9
|
|
|
$
|
42.2
|
|
|
|
|
June 30,
2020
|
|
December
31,
2019
|
Cash, cash
equivalents and investments
|
$
|
1,703.4
|
|
|
$
|
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 9 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 six months
ending June 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 10 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 second quarter
ended June 30, 2020.
Net Product Revenues for the second quarter of 2020 increased to
$419.0 million, compared to
$379.1 million in the second quarter
of 2019. The increase in Net Product Revenues was attributed to the
following:
- Aldurazyme Net Product Revenues increased by $26.5 million due to higher sales volume to
Genzyme;
- Palynziq Net Product Revenues increased by $21.9 million driven by a combination of revenue
from U.S. patients achieving maintenance dosing and new patients
initiating therapy;
- Brineura Net Product Revenues increased by $11.0 million due in large part growth in the
number of patients in all regions; and
- Kuvan Net Product Revenues increased by $9.3 million driven primarily by a U.S. price
increase and Kuvan product mix; partially offset by
- Naglazyme and Vimizim Net Product Revenues combined decreased
by $23.2 million primarily due to
timing of orders as well as the impact of missed infusions
resulting from the COVID-19 pandemic.
The decrease in GAAP Net Loss for the second quarter of 2020,
compared to GAAP Net Loss for the same period in 2019 was primarily
due to the following:
- an increase in gross profit (Total Revenues less Cost of Sales)
of $21.2 million primarily driven by
higher product sales; and
- an increase in the benefit from income taxes; partially offset
by
- the effect of the one-time gain recognized in the second
quarter of 2019 due to a third party's achievement of a commercial
milestone related to previously sold intangible asset; and
- higher selling, general and administrative (SG&A) expense
related to pre-commercialization activities for valoctocogene
roxaparvovec.
Non-GAAP Income for the second quarter of 2020 increased to
$57.4 million, compared to Non-GAAP
Income of $17.1 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, partially offset by higher
SG&A expense.
As of June 30, 2020, BioMarin had cash, cash equivalents
and investments totaling approximately $1.7
billion, as compared to $1.2
billion on December 31, 2019.
Commenting on second quarter 2020 results, Jean-Jacques
Bienaimé, Chairman and Chief Executive Officer of BioMarin, said,
"In the second quarter, BioMarin employees worked collaboratively
to ensure access to our critically-important medicines to the
people we serve, despite the global impact of COVID-19. In these
challenging times, our strong financial results underscore both the
essential nature of our products to patients and our ongoing
efforts to maintain supply around the world."
Mr. Bienaimé continued, "In the second quarter at the World
Federation of Hemophilia Virtual Congress, we were pleased to share
the four-year data update from our ongoing Phase 1/2 study, which
demonstrated sustained clinical benefit following a single
administration of valoctocogene roxaparvovec. All participants in
the study received a single administration of valoctocogene
roxaparvovec in 2016 and remained off exogenous factor prophylaxis
through year four. These data strengthen our confidence in
valoctocogene roxaparvovec and the opportunity to address the unmet
therapeutic needs of people with severe hemophilia A. With our
marketing applications under review in both the United States and Europe, we await the potential approval of
valoctocogene roxaparvovec. We believe each of the submissions
represent the first time a gene therapy product for any type of
hemophilia indication is under review by health authorities. With
the outcome of the Priority Review of our BLA anticipated
August 21, 2020, our commercial team
is preparing to launch what we believe is the most innovative
product yet for people with bleeding disorders."
"Another key milestone in the third quarter of this year,
representing the culmination of years of clinical study and
development, was the July 23
submission of a MAA to the EMA for vosoritide to treat children
with achondroplasia. The company remains on track to submit a NDA
to the FDA later in the third quarter. Our multi-pronged dossier of
data encompasses long-term clinical results in 5 to 18 year-olds
from our Phase 2 study, natural history data, the ongoing study of
newborns through 5 years, and highly statistically significant
placebo-controlled Phase 3 results. The positive results from our
vosoritide clinical programs bolster our confidence in the
potential for this drug to be the first pharmacological treatment
for the underlying cause of achondroplasia. Interest in our
clinical studies with vosoritide has been extremely robust,
demonstrating that families are keen to seek early treatment for
their children."
Mr. Bienaimé concluded, "Despite impact from COVID-19 on our
business in the short-term, we remain focused on working towards
significant achievements that we believe will drive long-term
value. Key milestones for the second half of 2020 include reaching
GAAP profitability for a full year for the first time in our
history, the potential approval of valoctocogene roxaparvovec, and
the pursuit of vosoritide approval. With these exciting
possibilities on the horizon, 2020 has the potential to be the most
momentous year in our 20-year history."
2020 Full-Year Financial Guidance
GAAP Net Income guidance for 2020 has been updated to include
the potential impact of intangible asset transfers between BioMarin
entities. These intangible asset transfers are expected to
occur in the second half of 2020, and are estimated to result in a
one-time, non-cash income tax benefit of approximately $700 million to $900
million. The range acknowledges that the intangible
asset transfers have not yet been completed and therefore the final
value cannot yet be determined with certainty. The final valuation
will be completed when the transactions occur. As a result,
full year GAAP net income guidance has been updated to be in the
range between $720 million and
$980 million. The intangible
asset transfers are not expected to impact Non-GAAP
income.
Item
|
|
Provided April 29,
2020
|
|
Revised August 4,
2020
|
Total Revenues
(1)
|
|
$1,850
|
|
|
to
|
|
$1,950
|
|
|
Unchanged
|
Vimizim Net Product
Revenues
|
|
$530
|
|
|
to
|
|
$570
|
|
|
Unchanged
|
Kuvan Net Product
Revenues
|
|
$430
|
|
|
to
|
|
$480
|
|
|
Unchanged
|
Naglazyme Net Product
Revenues
|
|
$360
|
|
|
to
|
|
$400
|
|
|
Unchanged
|
Palynziq Net Product
Revenues
|
|
$160
|
|
|
to
|
|
$190
|
|
|
Unchanged
|
Brineura Net Product
Revenues
|
|
$85
|
|
|
to
|
|
$115
|
|
|
Unchanged
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of Sales (% of
Total Revenues)
|
|
20
|
%
|
|
to
|
|
21
|
%
|
|
Unchanged
|
Research and
Development Expense
|
|
$675
|
|
|
to
|
|
$725
|
|
|
Unchanged
|
Selling, General and
Administrative Expense
|
|
$780
|
|
|
to
|
|
$830
|
|
|
Unchanged
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net
Income
|
|
$
|
20
|
|
|
to
|
|
$
|
80
|
|
|
$
|
720
|
|
|
to
|
|
$
|
980
|
|
Non-GAAP Income
(2)
|
|
$260
|
|
|
to
|
|
$310
|
|
|
Unchanged
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(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. The revenue guidance assumes
stabilization of such interruptions in late
2020.
|
|
|
(2)
|
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 10 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: On June 17, 2020,
the Company provided a four-year update with the 6e13 vg/kg dose
subjects and a three-year update with the 4e13 vg/kg dose subjects
from the ongoing Phase 2 study in a late-breaking oral presentation
at World Federation of Hemophilia Virtual Summit. The results from
both dose cohorts demonstrated that all subjects had remained off
prophylactic Factor VIII treatment since receiving their single
dose of valoctocogene roxaparvovec. In addition, cumulative mean
annualized bleed rates (ABR) remained less than one in both cohorts
and below pre-treatment baseline levels. In the six study
participants who were previously on Factor VIII prophylaxis in the
6e13 vg/kg cohort, the data showed substantial and sustained
reductions in bleeding that required Factor VIII infusions. During
the four years following treatment with valoctocogene roxaparvovec,
the cumulative mean ABR was 0.8, which represents a 95% reduction
from baseline. In the fourth year, the mean ABR was 1.3 and the
median was zero. There was a 96% reduction in mean Factor VIII
usage to 5.4 infusions per year cumulatively over four years from
the baseline of 135.6 infusions per year on Standard of Care
prophylaxis.
- Similarly, in the six study participants in the 4e13 vg/kg
cohort, the data showed substantial and sustained reductions in
bleeding requiring Factor VIII infusions following treatment with
valoctocogene roxaparvovec. The cumulative mean ABR was reduced by
93% to 0.9 with continued absence of target joint bleeds in 5 of 6
subjects during the three years observed, which represents a 93%
reduction from baseline. During the third year of follow-up, the
mean ABR was 0.5 and the median ABR was zero, and 67% or four out
of six, of the study participants were bleed-free. Five out of six
participants had no spontaneous bleeds. There was a 96% reduction
in mean Factor VIII usage to 5.7 infusions per year cumulatively
over three years from the baseline of 142.8 infusions per year. The
FDA review of the BLA, under Priority Review, for valoctocogene
roxaparvovec is on-track with a PDUFA target action date of
August 21, 2020. In Europe, the MAA filing remains under
accelerated assessment at this time. However, as communicated on
the first quarter results call, the review procedure was extended
by at least 3 months due to COVID-19 delays. Further, as is the
case with most filings that initially receive accelerated
assessment, there is a high possibility that the MAA will revert to
a standard review procedure from accelerated assessment. Based on
these assumptions, the CHMP opinion is expected in late 2020/early
2021.
- Vosoritide for children with achondroplasia: On
July 23, 2020, the Company announced
that it had submitted the MAA for vosoritide for the treatment of
children with achondroplasia to the EMA. Vosoritide is an
investigational, once daily injection of an analog of C-type
Natriuretic Peptide (CNP). BioMarin plans to submit a vosoritide
marketing application to the FDA later in the third quarter of
2020.
The applications include positive
final results from its randomized, double-blind, placebo-controlled
Phase 3 study evaluating the efficacy and safety of vosoritide. The
placebo-adjusted increased change from baseline in growth velocity
after one year of treatment with vosoritide, the primary endpoint,
was 1.6 cm/yr (p<0.0001). An ongoing, open-label, Dose Finding
Phase 2 study of vosoritide for achondroplasia demonstrated over 54
months that children in cohort 3 (N=10) of the study, at a dose of
15 µg/kg/day, achieved a statistically significant (p< 0.005)
cumulative mean additional height gain of 9.0 cm compared to
children, matched for age and gender, in a new natural history
achondroplasia dataset (N=619). The study enrolled 121 children
aged 5 to 14 with achondroplasia, the most common form of
disproportionate short stature. The results were consistent across
the broad patient population studied. Vosoritide was generally well
tolerated with mild-to-moderate injection site reactions being the
most frequent adverse event; there were no clinically significant
blood pressure decreases.
- BMN 307 gene therapy product candidate for phenylketonuria
(PKU): On January 13, 2020 the
Company announced that both the FDA and the Medicines and
Healthcare Products Regulatory Agency (MHRA) in the U.K. have
granted the Company Investigational New Drug (IND) status and
approved its Clinical Trial Application (CTA), respectively, for
BMN 307.
Depending on the ongoing impact of
COVID-19, the Company currently believes that dosing in Phearless,
the Phase 1/2 study of BMN 307, could begin later in the third
quarter. In the meantime, sites are being prepared to open and
enroll patients. All subjects participating in the Phearless study
will receive product made at commercial scale from BioMarin's
award-winning gene therapy manufacturing facility. Both the FDA and
EMA have granted BMN 307 Orphan Drug Status.
- DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM):
On May 3, 2020 the Company announced
that it had entered into a preclinical collaboration and license
agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform
company, to develop novel gene therapies to treat rare genetic
cardiomyopathies. DiNAQOR will receive 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.
- BMN 331 gene therapy product candidate for Hereditary
Angioedema (HAE): The Company began IND-enabling studies in
July with its third gene therapy candidate, BMN 331, 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.
- Vosoritide for the treatment of Genetic Causes of Short
Stature (GSS): In July, an investigator-initiated trial with
vosoritide for GSS began enrolling participants. As previously
announced, the Company plans to study vosoritide for treatment of
broader genetic statural abnormalities starting with genetic short
stature (GSS). This study is part of a research collaboration with
Children's National Hospital in Washington, D.C.
BioMarin will host a conference call and webcast to discuss
second quarter 2020 financial results today, Tuesday,
August 4, 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:
3285215
|
Conference ID:
3285215
|
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, other specified income statement
guidance for the full-year 2020, and our belief that 2020 has the
potential to be the most momentous year in BioMarin's 20-year
history; the financial performance of BioMarin as a whole,
including (i) that BioMarin expects to become profitable on a GAAP
basis for the first time in 2020 and (ii) that the COVID-19-related
demand interruptions will normalize in late 2020; the timing of
BioMarin's clinical development and commercial prospects, including
(i) the Company's expectation that it will start dosing patients in
the PHEARLESS study in the second half of 2020, the Company's
expectation that it will begin IND-enabling studies for BMN 331 for
HAE in mid-2020, and the Company's expectation that vosoritide will
be studied in broader genetic statural deficiencies with GSS, (ii)
BioMarin's clinical studies and trials, (iii) completion of
enrollment of those studies and trials, and (iv) announcements of
data from those studies and trials; the clinical development and
commercialization of BioMarin's product candidates and commercial
products, including (i) BioMarin's remaining on track to submit an
NDA to the FDA later in the third quarter for vosoritide for
children with achondroplasia ,and (ii) the potential approval and
commercialization of BioMarin's product candidates, including
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 Annual Report on Form 10-Q for the quarter
ended March 31, 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.
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
June 30, 2020
and December 31, 2019
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
June 30,
2020
|
|
December 31,
2019(1)
|
ASSETS
|
(unaudited)
|
|
|
Current
assets:
|
|
|
|
Cash and cash
equivalents
|
$
|
818,900
|
|
|
$
|
437,446
|
|
Short-term
investments
|
561,472
|
|
|
316,361
|
|
Accounts receivable,
net
|
381,215
|
|
|
377,404
|
|
Inventory
|
743,852
|
|
|
680,275
|
|
Other current
assets
|
142,127
|
|
|
130,657
|
|
Total current
assets
|
2,647,566
|
|
|
1,942,143
|
|
Noncurrent
assets:
|
|
|
|
Long-term
investments
|
323,058
|
|
|
411,978
|
|
Property, plant and
equipment, net
|
1,010,917
|
|
|
1,010,868
|
|
Intangible assets,
net
|
433,381
|
|
|
456,580
|
|
Goodwill
|
196,199
|
|
|
197,039
|
|
Deferred tax
assets
|
555,137
|
|
|
549,422
|
|
Other
assets
|
135,852
|
|
|
122,009
|
|
Total
assets
|
$
|
5,302,110
|
|
|
$
|
4,690,039
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
Current
liabilities:
|
|
|
|
Accounts payable and
accrued liabilities
|
$
|
443,202
|
|
|
$
|
570,621
|
|
Short-term convertible
debt, net
|
370,100
|
|
|
361,882
|
|
Total current
liabilities
|
813,302
|
|
|
932,503
|
|
Noncurrent
liabilities:
|
|
|
|
Long-term convertible
debt, net
|
1,073,202
|
|
|
486,238
|
|
Long-term contingent
consideration
|
50,216
|
|
|
50,793
|
|
Other long-term
liabilities
|
128,711
|
|
|
98,124
|
|
Total
liabilities
|
$
|
2,065,431
|
|
|
$
|
1,567,658
|
|
Stockholders'
equity:
|
|
|
|
Common stock, $0.001
par value: 500,000,000 shares authorized; 181,148,332 and
179,838,114 shares issued and outstanding, respectively.
|
181
|
|
|
180
|
|
Additional paid-in
capital
|
4,885,637
|
|
|
4,832,707
|
|
Company common stock
held by Nonqualified Deferred Compensation Plan
|
(10,678)
|
|
|
(9,961)
|
|
Accumulated other
comprehensive income
|
30,050
|
|
|
20,164
|
|
Accumulated
deficit
|
(1,668,511)
|
|
|
(1,720,709)
|
|
Total stockholders'
equity
|
3,236,679
|
|
|
3,122,381
|
|
Total liabilities and
stockholders' equity
|
$
|
5,302,110
|
|
|
$
|
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
|
Six Months Ended
June 30, 2020 and 2019
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
|
(unaudited)
|
|
(unaudited)
|
|
(unaudited)
|
|
(unaudited)
|
REVENUES:
|
|
|
|
|
|
|
|
Net product
revenues
|
$
|
419,032
|
|
|
$
|
379,075
|
|
|
$
|
908,075
|
|
|
$
|
773,558
|
|
Royalty and other
revenues
|
10,453
|
|
|
8,688
|
|
|
23,479
|
|
|
14,950
|
|
Total net
revenues
|
429,485
|
|
|
387,763
|
|
|
931,554
|
|
|
788,508
|
|
OPERATING
EXPENSES:
|
|
|
|
|
|
|
|
Cost of
sales
|
97,967
|
|
|
77,436
|
|
|
209,341
|
|
|
166,618
|
|
Research and
development
|
182,139
|
|
|
185,641
|
|
|
324,396
|
|
|
369,232
|
|
Selling, general and
administrative
|
175,412
|
|
|
160,754
|
|
|
362,707
|
|
|
322,912
|
|
Intangible asset
amortization and contingent consideration
|
14,912
|
|
|
20,286
|
|
|
30,589
|
|
|
40,051
|
|
Gain on sale of
nonfinancial assets
|
—
|
|
|
(15,000)
|
|
|
(59,495)
|
|
|
(15,000)
|
|
Total operating
expenses
|
470,430
|
|
|
429,117
|
|
|
867,538
|
|
|
883,813
|
|
INCOME (LOSS) FROM
OPERATIONS
|
(40,945)
|
|
|
(41,354)
|
|
|
64,016
|
|
|
(95,305)
|
|
|
|
|
|
|
|
|
|
Equity in the loss of
BioMarin/Genzyme LLC
|
(79)
|
|
|
(44)
|
|
|
(156)
|
|
|
(229)
|
|
Interest
income
|
4,291
|
|
|
5,899
|
|
|
9,535
|
|
|
12,197
|
|
Interest
expense
|
(8,048)
|
|
|
(6,866)
|
|
|
(14,963)
|
|
|
(13,593)
|
|
Other income,
net
|
2,508
|
|
|
470
|
|
|
647
|
|
|
2,078
|
|
INCOME (LOSS)
BEFORE INCOME TAXES
|
(42,273)
|
|
|
(41,895)
|
|
|
59,079
|
|
|
(94,852)
|
|
Provision for
(benefit from) income taxes
|
(13,090)
|
|
|
(4,460)
|
|
|
6,881
|
|
|
(944)
|
|
NET INCOME
(LOSS)
|
(29,183)
|
|
|
(37,435)
|
|
|
52,198
|
|
|
(93,908)
|
|
NET INCOME (LOSS)
PER SHARE, BASIC
|
$
|
(0.16)
|
|
|
$
|
(0.21)
|
|
|
$
|
0.29
|
|
|
$
|
(0.53)
|
|
NET INCOME (LOSS)
PER SHARE, DILUTED
|
$
|
(0.16)
|
|
|
$
|
(0.21)
|
|
|
$
|
0.28
|
|
|
$
|
(0.53)
|
|
Weighted average
common shares outstanding, basic
|
180,729
|
|
|
179,048
|
|
|
180,314
|
|
|
178,662
|
|
Weighted average
common shares outstanding, diluted
|
180,729
|
|
|
179,048
|
|
|
184,344
|
|
|
178,662
|
|
The following table presents Net Product Revenues by
Product:
Net Product
Revenues by Product
|
(In millions of
U.S. dollars)
|
|
|
Three Months
Ended
June 30,
|
|
Six Months
Ended
June 30,
|
|
2020
|
|
2019
|
|
%
Change
|
|
2020
|
|
2019
|
|
%
Change
|
|
(unaudited)
|
|
(unaudited)
|
|
|
|
(unaudited)
|
|
(unaudited)
|
|
|
Brineura
|
$
|
25.8
|
|
|
$
|
14.8
|
|
|
74
|
%
|
|
$
|
49.8
|
|
|
$
|
27.0
|
|
|
84
|
%
|
Firdapse
(1)
|
—
|
|
|
5.5
|
|
|
(100)
|
%
|
|
1.2
|
|
|
10.6
|
|
|
(89)
|
%
|
Naglazyme
|
81.0
|
|
|
98.2
|
|
|
(18)
|
%
|
|
195.3
|
|
|
185.1
|
|
|
6
|
%
|
PKU
franchise
|
163.3
|
|
|
132.1
|
|
|
24
|
%
|
|
319.9
|
|
|
251.3
|
|
|
27
|
%
|
Vimizim
|
116.7
|
|
|
122.7
|
|
|
(5)
|
%
|
|
253.9
|
|
|
248.5
|
|
|
2
|
%
|
Net Product Revenues
Marketed by BioMarin
|
386.8
|
|
|
373.3
|
|
|
|
|
820.1
|
|
|
722.5
|
|
|
|
Aldurazyme Net
Product Revenues Marketed by Genzyme
|
32.3
|
|
|
5.8
|
|
|
457
|
%
|
|
88.0
|
|
|
51.1
|
|
|
72
|
%
|
Total Net Product
Revenues
|
$
|
419.1
|
|
|
$
|
379.1
|
|
|
|
|
$
|
908.1
|
|
|
$
|
773.6
|
|
|
|
|
|
(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 six months ending
June 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
June 30,
|
|
Six Months
Ended
June 30,
|
|
2020
|
|
2019
|
|
%
Change
|
|
2020
|
|
2019
|
|
%
Change
|
|
(unaudited)
|
|
(unaudited)
|
|
|
|
(unaudited)
|
|
(unaudited)
|
|
|
Kuvan
|
$
|
122.6
|
|
|
113.3
|
|
|
8
|
%
|
|
$
|
244.6
|
|
|
220.2
|
|
|
11
|
%
|
Palynziq
|
40.7
|
|
|
18.8
|
|
|
116
|
%
|
|
75.3
|
|
|
31.1
|
|
|
142
|
%
|
Total PKU
franchise
|
$
|
163.3
|
|
|
$
|
132.1
|
|
|
24
|
%
|
|
$
|
319.9
|
|
|
$
|
251.3
|
|
|
27
|
%
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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
June 30,
|
|
Six Months
Ended
June 30,
|
|
Guidance
Year Ending
|
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
|
December 31,
2020
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net Income
(Loss)
|
$
|
(29.2)
|
|
|
$
|
(37.4)
|
|
|
$
|
52.2
|
|
|
$
|
(93.9)
|
|
|
$
|
720.0
|
|
—
|
$
|
980.0
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest expense,
net
|
3.7
|
|
|
1.0
|
|
|
5.4
|
|
|
1.4
|
|
|
3.0
|
|
—
|
2.0
|
|
|
Provision for
(benefit from) income taxes
|
(13.1)
|
|
|
(4.5)
|
|
|
6.9
|
|
|
(0.9)
|
|
|
(721.8)
|
|
—
|
(915.8)
|
|
|
Depreciation
expense
|
9.7
|
|
|
12.9
|
|
|
20.0
|
|
|
27.9
|
|
|
50.0
|
|
—
|
47.0
|
|
|
Amortization
expense
|
15.5
|
|
|
13.4
|
|
|
31.2
|
|
|
20.9
|
|
|
63.0
|
|
—
|
61.0
|
|
|
Stock-based
compensation expense
|
45.1
|
|
|
39.8
|
|
|
92.0
|
|
|
82.6
|
|
|
177.0
|
|
—
|
167.0
|
|
|
Contingent
consideration expense
|
(0.6)
|
|
|
6.9
|
|
|
(0.6)
|
|
|
19.2
|
|
|
2.0
|
|
—
|
2.0
|
|
|
Gain on sale of
nonfinancial assets
|
—
|
|
|
(15.0)
|
|
|
(59.5)
|
|
|
(15.0)
|
|
|
(59.5)
|
|
—
|
(59.5)
|
|
|
Licensed In-Process
R&D (1)
|
26.3
|
|
|
—
|
|
|
26.3
|
|
|
—
|
|
|
26.3
|
|
—
|
26.3
|
|
|
Non-GAAP
Income
|
$
|
57.4
|
|
|
$
|
17.1
|
|
|
$
|
173.9
|
|
|
$
|
42.2
|
|
|
$
|
260.0
|
|
—
|
$
|
310.0
|
|
|
|
|
(1)
|
Represents the one
time 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
June 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
|
$
|
97.9
|
|
|
$
|
—
|
|
|
$
|
(4.9)
|
|
|
$
|
93.0
|
|
|
$
|
77.4
|
|
|
$
|
—
|
|
|
$
|
(3.7)
|
|
|
$
|
73.7
|
|
Research and
development
|
182.1
|
|
|
(4.6)
|
|
|
(42.3)
|
|
|
135.2
|
|
|
185.6
|
|
|
(7.3)
|
|
|
(14.9)
|
|
|
163.4
|
|
Selling, general and
administrative
|
175.4
|
|
|
(5.1)
|
|
|
(24.2)
|
|
|
146.1
|
|
|
160.8
|
|
|
(5.6)
|
|
|
(21.2)
|
|
|
134.0
|
|
Intangible asset
amortization and contingent consideration
|
14.9
|
|
|
(15.5)
|
|
|
0.6
|
|
|
—
|
|
|
20.3
|
|
|
(13.4)
|
|
|
(6.9)
|
|
|
—
|
|
Gain on sale of
nonfinancial assets
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(15.0)
|
|
|
—
|
|
|
15.0
|
|
|
—
|
|
Interest expense,
net
|
(3.7)
|
|
|
3.7
|
|
|
—
|
|
|
—
|
|
|
(1.0)
|
|
|
1.0
|
|
|
—
|
|
|
—
|
|
Provision for
(benefit from) income taxes
|
(13.1)
|
|
|
13.1
|
|
|
—
|
|
|
—
|
|
|
(4.5)
|
|
|
4.5
|
|
|
—
|
|
|
—
|
|
GAAP Net Income
(Loss)/Non-GAAP Income
|
$
|
(29.2)
|
|
|
$
|
15.8
|
|
|
$
|
70.8
|
|
|
$
|
57.4
|
|
|
$
|
(37.4)
|
|
|
$
|
22.8
|
|
|
$
|
31.7
|
|
|
$
|
17.1
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Six months ended
June 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
|
$
|
209.3
|
|
|
$
|
0.0
|
|
|
$
|
(10.0)
|
|
|
$
|
199.3
|
|
|
$
|
166.6
|
|
|
$
|
0.0
|
|
|
(8.5)
|
|
|
$
|
158.1
|
|
Research and
development
|
324.4
|
|
|
(9.4)
|
|
|
(56.0)
|
|
|
259.0
|
|
|
369.2
|
|
|
(16.7)
|
|
|
(28.8)
|
|
|
323.7
|
|
Selling, general and
administrative
|
362.7
|
|
|
(10.6)
|
|
|
(52.3)
|
|
|
299.8
|
|
|
322.9
|
|
|
(11.2)
|
|
|
(45.3)
|
|
|
266.4
|
|
Intangible asset
amortization and contingent consideration
|
30.6
|
|
|
(31.2)
|
|
|
0.6
|
|
|
—
|
|
|
40.1
|
|
|
(20.9)
|
|
|
(19.2)
|
|
|
—
|
|
Gain on sale of
nonfinancial assets
|
(59.5)
|
|
|
—
|
|
|
59.5
|
|
|
—
|
|
|
(15.0)
|
|
|
—
|
|
|
15.0
|
|
|
—
|
|
Interest expense,
net
|
(5.4)
|
|
|
5.4
|
|
|
—
|
|
|
—
|
|
|
(1.4)
|
|
|
1.4
|
|
|
—
|
|
|
—
|
|
Provision for
(benefit from) income taxes
|
6.9
|
|
|
(6.9)
|
|
|
—
|
|
|
—
|
|
|
(0.9)
|
|
|
0.9
|
|
|
—
|
|
|
—
|
|
GAAP Net
Loss/Non-GAAP Income
|
52.2
|
|
|
63.5
|
|
|
58.2
|
|
|
173.9
|
|
|
(93.9)
|
|
|
49.3
|
|
|
86.8
|
|
|
42.2
|
|
Contact:
|
|
|
Investors:
|
|
Media:
|
Traci
McCarty
|
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
|
(415)
455-7451
|
View original content to download
multimedia:http://www.prnewswire.com/news-releases/biomarin-announces-second-quarter-2020-total-revenue-growth-of-11-to-430-million-301106049.html
SOURCE BioMarin Pharmaceutical Inc.