SAN RAFAEL, Calif.,
April 29, 2021 /PRNewswire/ --
Financial Highlights (in millions of U.S. dollars, except per
share data, unaudited)
|
|
Three Months Ended
March 31,
|
|
|
2021
|
|
2020
|
|
%
Change
|
|
|
|
|
|
|
|
Total
Revenues
|
|
$
|
486.0
|
|
|
$
|
502.1
|
|
|
(3)
|
%
|
|
|
|
|
|
|
|
Net Product Revenues
Marketed by BioMarin (1)
|
|
$
|
417.8
|
|
|
$
|
433.3
|
|
|
(4)
|
%
|
|
|
|
|
|
|
|
Vimizim Net Product
Revenues
|
|
$
|
158.4
|
|
|
$
|
137.2
|
|
|
15
|
%
|
Naglazyme Net Product
Revenues
|
|
$
|
107.3
|
|
|
$
|
114.3
|
|
|
(6)
|
%
|
Kuvan Net Product
Revenues
|
|
$
|
70.8
|
|
|
$
|
122.0
|
|
|
(42)
|
%
|
Palynziq Net Product
Revenues
|
|
$
|
54.0
|
|
|
$
|
34.6
|
|
|
56
|
%
|
Brineura Net Product
Revenues
|
|
$
|
27.3
|
|
|
$
|
24.0
|
|
|
14
|
%
|
|
|
|
|
|
|
|
Aldurazyme Net
Product Revenues
|
|
$
|
50.0
|
|
|
$
|
55.7
|
|
|
(10)
|
%
|
|
|
|
|
|
|
|
GAAP Net
Income
|
|
$
|
17.4
|
|
|
$
|
81.4
|
|
|
|
GAAP Net Income per
Share – Basic
|
|
$
|
0.10
|
|
|
$
|
0.45
|
|
|
|
GAAP Net Income per
Share – Diluted
|
|
$
|
0.09
|
|
|
$
|
0.44
|
|
|
|
Non-GAAP
Income (2)
|
|
$
|
104.4
|
|
|
$
|
116.5
|
|
|
|
|
March 31,
2021
|
|
December
31,
2020
|
|
Cash, cash
equivalents and investments
|
$
|
1,408.6
|
|
|
$
|
1,350.9
|
|
|
|
|
(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. 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 first quarter ended March 31, 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/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. Refer to Non-GAAP
Information beginning on page 11 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 first quarter
ended March 31, 2021.
"We mark the start of 2021 with strong financial results, and a
number of exciting regulatory decisions ahead this year," said
Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of
BioMarin. "The potential approval in Europe this summer of vosoritide, the first
potential therapeutic option for children with achondroplasia, will
set the stage for our next significant phase of growth, especially
considering the EMEA region is 3 times larger than the U.S. market.
To date, the interactions with the review team at the European
Medicines Agency have been very positive. Next, we plan to
re-submit the application for valoctocogene roxaparvovec gene
therapy, to treat hemophilia A, to the EMA in the second quarter,
to be followed by potential approval of vosoritide in the United States later this year."
Mr. Bienaimé continued, "We are well positioned to deliver
strong results for the full-year 2021, and reaffirm our financial
guidance provided early this year. This takes into consideration
the uneven ordering patterns in our dynamic and global commercial
business, as well as continued uncertainty outside of the United States from COVID-19, and further
underscores the essential nature of our products to the people who
rely on them. Taken together, our financial strength, including
positive cash flows from operations of $113.5 million in the first quarter, combined
with near-term opportunities from potential new products and a
robust early-stage pipeline, we believe that 2021 will be pivotal
to BioMarin's evolution."
Financial Highlights:
- Net Product Revenues for the first quarter of 2021
decreased to $467.8 million, compared
to $489.0 million for the same period
in 2020. The decrease in Net Product Revenues was primarily
attributed to the following:
-
- Lower Kuvan product revenues primarily due to known generic
competition in the U.S.; partially offset by
- Higher Vimizim product revenues primarily due to timing of
orders from Europe, Middle East and North Africa; and
- Higher Palynziq product revenues primarily driven by a
combination of revenue from more U.S. patients achieving
maintenance dosing and new patients initiating therapy.
- GAAP Net Income decreased $64.0
million to $17.4 million for
the first quarter of 2021 compared to $81.4
million for the same period in 2020. The decrease was
primarily due to the absence of the gain on sale the Firdapse
commercial assets totaling $59.5
million in the first quarter of 2020 and a decrease in gross
profits due to the lower Kuvan product revenues due to generic
competition in the U.S. This decrease was partially offset by lower
selling, general and administrative (SG&A) expenses primarily
due to a decrease in foreign currency exchange losses in the first
quarter of 2021.
- Non-GAAP Net Income for the first quarter of 2021
decreased to $104.4 million compared
to Non-GAAP Income of $116.5 million
for the same period in 2020. The decline in Non-GAAP Income for the
quarter, compared to the same period in 2020, was primarily
attributed to lower gross profits, partially offset by lower
SG&A expenses primarily due to a decrease in foreign currency
exchange losses in the first quarter of 2021.
Commercial Portfolio (Naglazyme, VIMIZIM, Brineura,
Palynziq, Kuvan and Aldurazyme)
- Naglazyme, VIMIZIM and Brineura maintained robust patient
compliance in the quarter and continue to grow based on strong
underlying demand.
-
- Patients on commercial Naglazyme and Vimizim therapy both
increased approximately 10% year-over-year.
- Patients on commercial Brineura therapy increased by more than
30% year-over-year.
- Order timing for both Naglazyme and VIMIZIM are expected to be
more concentrated in the first half of 2021 as compared to the
second half of 2021.
- Palynziq top-line results in 2021 are expected to increase
approximately 35% based on the mid-point of full-year 2021 guidance
as compared to full-year 2020 results.
- Palynziq growth in European, Middle
East and African regions (EMEA) has been impacted by ongoing
challenges due to COVID-19 with Palynziq revenue from EMEA expected
to increase when PKU clinics have more freedom to operate and start
additional patients.
-
- The number of U.S. Patients on commercial Palynziq therapy
increased more than 20% year-over-year and is expected to continue
to grow as U.S. PKU clinics re-open over the coming quarters.
- Loss of Kuvan U.S. market share, due to the introduction of
generics following BioMarin's loss of U.S. market exclusivity, is
consistent with expectations.
- Aldurazyme contributions were driven by the timing of product
released and transfer of control to Genzyme, who is responsible for
marketing Aldurazyme worldwide.
Late-stage Regulatory Portfolio (Vosoritide and
valoctocogene roxaparvovec)
- In Europe, with respect to
vosoritide for the treatment of achondroplasia, BioMarin is in the
final stages of the review procedure ahead of the anticipated
June 2021 Committee for Medicinal
Products for Human Use (CHMP) opinion. Assuming a positive CHMP
opinion, the European Commission could potentially grant marketing
authorization for vosoritide in the third quarter of 2021.
- In Europe, with respect to
valoctocogene roxaparvovec for the treatment of severe hemophilia
A, based on positive pre-submission feedback in the current
quarter, BioMarin reaffirms its plan to submit the Marketing
Authorization Application with one-year results from the Phase 3
GENEr8-1 study to the European Medicines Agency (EMA) in
June 2021.
- In the U.S., BioMarin provided the U.S. Food and Drug
Administration (FDA) with the two-year results from the Phase 3
extension study with vosoritide to supplement the New Drug
Application (NDA) already under review. As anticipated, the FDA
designated this submission as a major amendment to the application,
thus extending the Prescription Drug User Fee Act (PDUFA) target
action date by three months to November 20,
2021 to provide time for a full review of the
submission.
-
- Also in the first quarter of 2021, the FDA pre-approval
inspection of BioMarin's Novato facility for the manufacture of
vosoritide drug substance was completed, representing another
important milestone as vosoritide advances through the review
process.
- During the current quarter, the FDA reiterated their
recommendation that BioMarin submit two-year follow-up safety and
efficacy data on all study participants from the GENEr8-1 study to
support their benefit/risk assessment of valoctocogene
roxaparvovec. BioMarin is targeting a Biologics License Application
(BLA) submission in the second quarter of 2022 assuming favorable
results, followed by an expected 6-month review procedure by the
FDA.
-
- The FDA granted Regenerative Medicine Advanced Therapy (RMAT)
designation to valoctocogene roxaparvovec. The RMAT designation is
complementary to Breakthrough Therapy Designation, which BioMarin
received in 2017.
Earlier-stage Development Portfolio (BMN 307, BMN 255, BMN
331, DiNA-001, Allen Institute Collaboration)
- BMN 307: Dose escalation in PHEarless, the Phase 1/2 study of
BMN 307 continues based on encouraging Phe lowering and safety
profile observed in study participants who were treated with the
lowest dose.
- BMN 255 for a subset of chronic renal disease: On January 11, 2021 BioMarin announced that it had
filed an IND in 2020 for BMN 255, a small molecule for a subset of
chronic renal disease. BMN 255 was driven by genetic discoveries
for both mechanism and for identifying individuals for
treatment.
- BMN 331 gene therapy product candidate for Hereditary
Angioedema (HAE): IND-enabling studies are ongoing with BMN 331 for
the treatment of HAE, BioMarin's third gene therapy candidate.
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. BioMarin is on track to file
an IND for BMN 331 mid-year 2021.
- BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling
studies are underway with BMN 351, an antisense oligonucleotide
therapy that has demonstrated dystrophin expression levels of
30-50% of wild-type levels in the quadriceps in a DMD mouse model
treated at 18.7 mg/kg/week for 13 weeks (measured 2 weeks following
last administration). If results from the ongoing pre-clinical
studies are supportive, BioMarin anticipates filing an IND for BMN
351 in the first half of 2022.
- DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM):
Pre-clinical studies are underway with DiNA-001 following a
collaboration announced in 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.
- On April 28, 2021, BioMarin and
the Allen Institute announced a collaboration to create new gene
therapies aimed at rare genetic diseases of the central nervous
system (CNS). The goal is to combine the Allen Institute's
leadership in large-scale genomic science in the CNS therapeutic
area with BioMarin's expertise in developing transformational gene
therapies.
2021 Full-Year Financial Guidance (in millions, except
%)
Item
|
|
2021 Guidance *
(reaffirmed)
|
Total
Revenues
|
|
$1,750
|
|
to
|
|
$1,850
|
Vimizim Net Product
Revenues
|
|
$570
|
|
to
|
|
$610
|
Kuvan Net Product
Revenues
|
|
$250
|
|
to
|
|
$290
|
Naglazyme Net Product
Revenues
|
|
$365
|
|
to
|
|
$395
|
Palynziq Net Product
Revenues
|
|
$210
|
|
to
|
|
$250
|
Brineura Net Product
Revenues
|
|
$120
|
|
to
|
|
$140
|
|
|
|
|
|
|
|
Cost of Sales (% of
Total Revenues)
|
|
23%
|
|
to
|
|
25%
|
Research and
Development Expense
|
|
$645
|
|
to
|
|
$695
|
Selling, General and
Administrative Expense
|
|
$725
|
|
to
|
|
$775
|
|
|
|
|
|
|
|
GAAP Net
Loss
|
|
($130)
|
|
to
|
|
($80)
|
Non-GAAP Income
(1)
|
|
$170
|
|
to
|
|
$220
|
|
|
|
|
|
|
|
|
*2021 Guidance takes
into consideration ongoing expected impact from the COVID-19
pandemic in 2021 assuming consistent trends experienced during
2020.
|
|
|
(1)
|
All Financial
Guidance items are calculated based on U.S. GAAP with the exception
of Non-GAAP Income. Refer to Non-GAAP Information beginning on
page11 of this press release for a complete discussion of the
Company's Non-GAAP financial information and reconciliations to the
corresponding GAAP reported information.
|
BioMarin will host a conference call and webcast to discuss
first quarter and full-year 2021 financial results today, Thursday,
April 29, 2021 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:
2396463
|
Conference ID:
2396463
|
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 Loss, Non-GAAP Income, and other specified income statement
guidance for the full-year 2021; cash flows from operating
activities; 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) BioMarin's plan to submit complete one-year Phase 3 data for
valoctocogene roxaparvovec to the EMA in June 2021, (ii) BioMarin's plan to re-submit its
MAA for valoctocogene roxaparvovec to the EMA in June 2021, (iii) BioMarin's plans to re-submit a
BLA for valoctocogene roxaparvovec to the FDA with two-year
follow-up results from all the subjects from the Phase 3 GENEr8-1
study in the second quarter of 2022, (iv) that the CHMP opinion for
vosoritide is expected in Europe
in June 2021, (v) the extended PDUFA
target action date with respect to vosoritide of November 20, 2021, (vi) BioMarin's anticipated
IND submission for BMN 351 in the first half of 2022, and (vii)
BioMarin and Allen Institute collaborating to create new gene
therapies; 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; and the expected benefits and availability of BioMarin's
product candidates, including that vosoritide would be the first
potential therapeutic option for children with achondroplasia; and
potential growth opportunities and trends, including (i) that
vosoritide would set the stage for the Company's next significant
stage of growth if approved and (ii) growth expectations regarding
Palynziq.
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, including BioMarin's projected impact
of the COVID-19 pandemic on its global revenue sources, including
due to demand interruptions such as missed patient infusions and
delayed treatment starts for new patients; 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-K
for the year ended December 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
|
March 31,
2021 and December 31, 2020
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
March 31,
2021
|
|
December 31,
2020(1)
|
ASSETS
|
(unaudited)
|
|
|
Current
assets:
|
|
|
|
Cash and cash
equivalents
|
$
|
667,313
|
|
|
$
|
649,158
|
|
Short-term
investments
|
420,178
|
|
|
416,228
|
|
Accounts receivable,
net
|
396,097
|
|
|
448,351
|
|
Inventory
|
713,929
|
|
|
698,548
|
|
Other current
assets
|
91,249
|
|
|
129,934
|
|
Total current
assets
|
2,288,766
|
|
|
2,342,219
|
|
Noncurrent
assets:
|
|
|
|
Long-term
investments
|
321,127
|
|
|
285,473
|
|
Property, plant and
equipment, net
|
1,022,474
|
|
|
1,032,471
|
|
Intangible assets,
net
|
405,903
|
|
|
417,271
|
|
Goodwill
|
196,199
|
|
|
196,199
|
|
Deferred tax
assets
|
1,429,386
|
|
|
1,432,150
|
|
Other
assets
|
141,198
|
|
|
142,237
|
|
Total
assets
|
$
|
5,805,053
|
|
|
$
|
5,848,020
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
Current
liabilities:
|
|
|
|
Accounts payable and
accrued liabilities
|
$
|
399,225
|
|
|
$
|
492,548
|
|
Short-term contingent
consideration
|
30,810
|
|
|
—
|
|
Total current
liabilities
|
430,035
|
|
|
492,548
|
|
Noncurrent
liabilities:
|
|
|
|
Long-term convertible
debt, net
|
1,076,127
|
|
|
1,075,145
|
|
Long-term contingent
consideration
|
29,153
|
|
|
60,130
|
|
Other long-term
liabilities
|
107,728
|
|
|
114,195
|
|
Total
liabilities
|
$
|
1,643,043
|
|
|
$
|
1,742,018
|
|
Stockholders'
equity:
|
|
|
|
Common stock, $0.001
par value: 500,000,000 shares authorized; 182,670,614 and
181,740,999 shares issued and outstanding, respectively.
|
183
|
|
|
182
|
|
Additional paid-in
capital
|
5,010,619
|
|
|
4,993,407
|
|
Company common stock
held by Nonqualified Deferred Compensation Plan
|
(9,558)
|
|
|
(9,839)
|
|
Accumulated other
comprehensive income (loss)
|
5,004
|
|
|
(16,139)
|
|
Accumulated
deficit
|
(844,238)
|
|
|
(861,609)
|
|
Total stockholders'
equity
|
4,162,010
|
|
|
4,106,002
|
|
Total liabilities and
stockholders' equity
|
$
|
5,805,053
|
|
|
$
|
5,848,020
|
|
|
|
|
|
(1)
|
December 31, 2020
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, 2020, filed with the U.S. Securities
and Exchange Commission (SEC) on February 26, 2021.
|
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
Three Months Ended
March 31, 2021 and 2020
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
|
Three Months Ended
March 31,
|
|
|
2021
|
|
2020
|
|
|
(unaudited)
|
|
(unaudited)
|
REVENUES:
|
|
|
|
|
Net product
revenues
|
|
$
|
467,769
|
|
|
$
|
489,043
|
|
Royalty and other
revenues
|
|
18,261
|
|
|
13,026
|
|
Total net
revenues
|
|
486,030
|
|
|
502,069
|
|
OPERATING
EXPENSES:
|
|
|
|
|
Cost of
sales
|
|
120,166
|
|
|
111,374
|
|
Research and
development
|
|
148,725
|
|
|
142,257
|
|
Selling, general and
administrative
|
|
174,318
|
|
|
187,295
|
|
Intangible asset
amortization and contingent consideration
|
|
17,735
|
|
|
15,677
|
|
Gain on sale of
nonfinancial assets
|
|
—
|
|
|
(59,495)
|
|
Total operating
expenses
|
|
460,944
|
|
|
397,108
|
|
INCOME FROM
OPERATIONS
|
|
25,086
|
|
|
104,961
|
|
|
|
|
|
|
Equity in the loss of
BioMarin/Genzyme LLC
|
|
(1,351)
|
|
|
(77)
|
|
Interest
income
|
|
2,439
|
|
|
5,244
|
|
Interest
expense
|
|
(3,804)
|
|
|
(6,915)
|
|
Other income,
net
|
|
858
|
|
|
(1,861)
|
|
INCOME BEFORE
INCOME TAXES
|
|
23,228
|
|
|
101,352
|
|
Provision for income
taxes
|
|
5,857
|
|
|
19,971
|
|
NET
INCOME
|
|
$
|
17,371
|
|
|
$
|
81,381
|
|
NET INCOME PER
SHARE, BASIC
|
|
$
|
0.10
|
|
|
$
|
0.45
|
|
NET INCOME PER
SHARE, DILUTED
|
|
$
|
0.09
|
|
|
$
|
0.44
|
|
Weighted average
common shares outstanding, basic
|
|
181,772
|
|
|
179,898
|
|
Weighted average
common shares outstanding, diluted
|
|
184,365
|
|
|
187,163
|
|
BIOMARIN
PHARMACEUTICAL INC.
|
CONDENSED
CONSOLIDATED STATEMENTS OF CASH FLOWS
|
Three Months Ended
March 31, 2021 and 2020
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
Three Months Ended
March 31,
|
|
2021
|
|
2020
|
|
(unaudited)
|
|
(unaudited)
|
CASH FLOWS FROM
OPERATING ACTIVITIES:
|
|
|
|
Net income
|
$
|
17,371
|
|
|
$
|
81,381
|
|
Adjustments to
reconcile net income to net cash used in operating
activities:
|
|
|
|
Depreciation and
amortization
|
27,983
|
|
|
25,964
|
|
Non-cash interest
expense
|
1,043
|
|
|
4,618
|
|
Amortization of
premium (accretion of discount) on investments
|
673
|
|
|
60
|
|
Stock-based
compensation
|
49,503
|
|
|
46,994
|
|
Gain on sale of
nonfinancial assets
|
—
|
|
|
(59,495)
|
|
Deferred income
taxes
|
3,335
|
|
|
10,603
|
|
Unrealized foreign
exchange loss
|
3,950
|
|
|
9,400
|
|
Non-cash changes in
the fair value of contingent consideration
|
2,255
|
|
|
(4)
|
|
Other
|
(871)
|
|
|
(383)
|
|
Changes in operating
assets and liabilities:
|
|
|
|
Accounts receivable,
net
|
40,294
|
|
|
(31,898)
|
|
Inventory
|
(6,425)
|
|
|
(20,706)
|
|
Other current
assets
|
42,784
|
|
|
8,302
|
|
Other
assets
|
1,617
|
|
|
(441)
|
|
Accounts payable and
accrued liabilities
|
(72,304)
|
|
|
(94,733)
|
|
Other long-term
liabilities
|
2,304
|
|
|
5,144
|
|
Net cash provided by
(used in) operating activities
|
113,512
|
|
|
(15,194)
|
|
CASH FLOWS FROM
INVESTING ACTIVITIES:
|
|
|
|
Purchases of
property, plant and equipment
|
(25,507)
|
|
|
(40,554)
|
|
Maturities and sales
of investments
|
194,637
|
|
|
94,701
|
|
Purchases of
available-for-sale securities
|
(237,171)
|
|
|
(40,104)
|
|
Proceeds from sale of
nonfinancial assets
|
—
|
|
|
67,159
|
|
Purchase of
intangible assets
|
(2,747)
|
|
|
(3,463)
|
|
Other
|
—
|
|
|
(335)
|
|
Net cash provided by
(used in) investing activities
|
(70,788)
|
|
|
77,404
|
|
CASH FLOWS FROM
FINANCING ACTIVITIES:
|
|
|
|
Proceeds from
exercises of awards under equity incentive plans
|
5,817
|
|
|
10,116
|
|
Taxes paid related to
net share settlement of equity awards
|
(29,097)
|
|
|
(28,844)
|
|
Principal repayments
of financing leases
|
(1,084)
|
|
|
(943)
|
|
Net cash used in
financing activities
|
(24,364)
|
|
|
(19,671)
|
|
Effect of exchange
rate changes on cash
|
(205)
|
|
|
(3,353)
|
|
NET INCREASE IN
CASH AND CASH EQUIVALENTS
|
$
|
18,155
|
|
|
$
|
39,186
|
|
Cash and cash
equivalents:
|
|
|
|
Beginning of
period
|
$
|
649,158
|
|
|
$
|
437,446
|
|
End of
period
|
$
|
667,313
|
|
|
$
|
476,632
|
|
The following table presents Net Product Revenues by
Product:
Net Product
Revenues by Product
|
(In millions of
U.S. dollars)
|
|
|
Three Months
Ended
March 31,
|
|
2021
|
|
2020
|
|
%
Change
|
|
(unaudited)
|
|
(unaudited)
|
|
|
PKU
franchise
|
$
|
124.8
|
|
|
$
|
156.6
|
|
|
(20)
|
%
|
Vimizim
|
158.4
|
|
|
137.2
|
|
|
15
|
%
|
Naglazyme
|
107.3
|
|
|
114.3
|
|
|
(6)
|
%
|
Brineura
|
27.3
|
|
|
24.0
|
|
|
14
|
%
|
Firdapse
(1)
|
—
|
|
|
1.2
|
|
|
(100)
|
%
|
Net Product Revenues
Marketed by BioMarin
|
$
|
417.8
|
|
|
$
|
433.3
|
|
|
|
Aldurazyme Net
Product Revenues Marketed by Genzyme
|
50.0
|
|
|
55.7
|
|
|
(10)
|
%
|
Total Net Product
Revenues
|
$
|
467.8
|
|
|
$
|
489.0
|
|
|
|
|
|
(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 months ending March
31, 2020; and as a result of the transaction BioMarin no longer
generates Net Product Revenues from Firdapse.
|
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)
|
|
|
|
Three Months
Ended
March
31,
|
|
|
2021
|
|
2020
|
|
%
Change
|
|
|
(unaudited)
|
|
(unaudited)
|
|
|
Kuvan
|
|
$
|
70.8
|
|
|
$
|
122.0
|
|
|
(42)
|
%
|
Palynziq
|
|
54.0
|
|
|
34.6
|
|
|
56
|
%
|
Total PKU
franchise
|
|
$
|
124.8
|
|
|
$
|
156.6
|
|
|
(20)
|
%
|
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 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
March 31,
|
|
Guidance
Year
Ending
|
|
|
2021
|
|
2020
|
|
December 31,
2021
|
|
|
|
|
|
|
|
|
|
GAAP Net Income
(Loss)
|
|
$
|
17.4
|
|
|
$
|
81.4
|
|
|
$
|
(130.0)
|
|
—
|
$
|
(80.0)
|
|
|
|
|
|
|
|
|
|
|
Interest expense,
net
|
|
1.4
|
|
|
1.7
|
|
|
11.0
|
|
—
|
11.0
|
|
Provision for income
taxes
|
|
5.9
|
|
|
20.0
|
|
|
(16.0)
|
|
—
|
(16.0)
|
|
Depreciation
expense
|
|
12.5
|
|
|
10.3
|
|
|
48.0
|
|
—
|
48.0
|
|
Amortization
expense
|
|
15.4
|
|
|
15.7
|
|
|
62.0
|
|
—
|
62.0
|
|
Stock-based
compensation expense
|
|
49.5
|
|
|
46.9
|
|
|
186.0
|
|
—
|
186.0
|
|
Contingent
consideration expense
|
|
2.3
|
|
|
—
|
|
|
9.0
|
|
—
|
9.0
|
|
Gain on sale of
nonfinancial assets
|
|
—
|
|
|
(59.5)
|
|
|
—
|
|
—
|
—
|
|
Non-GAAP
Income
|
|
$
|
104.4
|
|
|
$
|
116.5
|
|
|
$
|
170.0
|
|
—
|
$
|
220.0
|
|
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
March 31,
|
|
2021
|
|
2020
|
|
|
|
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
|
$
|
120.2
|
|
|
$
|
—
|
|
|
$
|
(6.5)
|
|
|
$
|
113.7
|
|
|
$
|
111.4
|
|
|
$
|
—
|
|
|
$
|
(5.1)
|
|
|
$
|
106.3
|
|
Research and
development
|
148.7
|
|
|
(7.5)
|
|
|
(17.5)
|
|
|
123.7
|
|
|
142.3
|
|
|
(4.8)
|
|
|
(13.7)
|
|
|
123.8
|
|
Selling, general and
administrative
|
174.3
|
|
|
(5.0)
|
|
|
(25.5)
|
|
|
143.8
|
|
|
187.3
|
|
|
(5.5)
|
|
|
(28.1)
|
|
|
153.7
|
|
Intangible asset
amortization and contingent consideration
|
17.7
|
|
|
(15.4)
|
|
|
(2.3)
|
|
|
—
|
|
|
15.7
|
|
|
(15.7)
|
|
|
0.0
|
|
|
—
|
|
Gain on sale of
nonfinancial assets
|
0.0
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(59.5)
|
|
|
—
|
|
|
59.5
|
|
|
—
|
|
Interest expense,
net
|
(1.4)
|
|
|
1.4
|
|
|
—
|
|
|
—
|
|
|
(1.7)
|
|
|
1.7
|
|
|
—
|
|
|
—
|
|
Provision for income
taxes
|
5.9
|
|
|
(5.9)
|
|
|
—
|
|
|
—
|
|
|
20.0
|
|
|
(20.0)
|
|
|
—
|
|
|
—
|
|
GAAP Net Income /
Non-GAAP Income
|
$
|
17.4
|
|
|
$
|
35.2
|
|
|
$
|
51.8
|
|
|
$
|
104.4
|
|
|
$
|
81.4
|
|
|
$
|
47.7
|
|
|
$
|
(12.6)
|
|
|
$
|
116.5
|
|
Contact:
|
|
|
Investors:
|
|
Media:
|
Traci
McCarty
|
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
|
(415)
455-7451
|
View original content to download
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SOURCE BioMarin Pharmaceutical Inc.