SAN RAFAEL, Calif.,
April 29, 2020 /PRNewswire/ --
Financial Highlights (in millions of U.S. dollars, except per
share data, unaudited)
|
Three Months Ended
March 31,
|
|
2020
|
|
2019
|
|
%
Change
|
|
|
|
|
|
|
Total
Revenues
|
$
|
502.1
|
|
|
$
|
400.7
|
|
|
25
|
%
|
|
|
|
|
|
|
Net Product Revenues
Marketed by BioMarin (1)
|
433.3
|
|
|
349.2
|
|
|
24
|
%
|
|
|
|
|
|
|
Vimizim Net Product
Revenues
|
137.2
|
|
|
125.8
|
|
|
9
|
%
|
Kuvan Net Product
Revenues
|
122.0
|
|
|
106.9
|
|
|
14
|
%
|
Naglazyme Net Product
Revenues
|
114.3
|
|
|
86.9
|
|
|
32
|
%
|
Palynziq Net Product
Revenues
|
34.6
|
|
|
12.3
|
|
|
181
|
%
|
Brineura Net Product
Revenues
|
24.0
|
|
|
12.2
|
|
|
97
|
%
|
|
|
|
|
|
|
Aldurazyme Net
Product Revenues
|
55.7
|
|
|
45.3
|
|
|
23
|
%
|
|
|
|
|
|
|
GAAP Net Income
(Loss)
|
$
|
81.4
|
|
|
$
|
(56.5)
|
|
|
|
GAAP Net Income
(Loss) per Share – Basic
|
$
|
0.45
|
|
|
$
|
(0.32)
|
|
|
|
GAAP Net Income
(Loss) per Share – Diluted
|
$
|
0.44
|
|
|
$
|
(0.32)
|
|
|
|
Non-GAAP
Income (2)
|
$
|
116.5
|
|
|
$
|
24.8
|
|
|
|
|
|
|
|
|
|
March
31,
2020
|
|
December
31,
2019
|
|
Cash, cash
equivalents and investments
|
$
|
1,149.2
|
|
|
$
|
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 will be reflected in the
Company's consolidated financial statements for the three months
ending 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, 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 first quarter
ended March 31, 2020.
Total Revenues increased 25% to $502.1
million. The increase in Total Revenues was primarily
attributed to increased Net Product Revenues which were
$489.0 million in the first quarter
of 2020, compared to $394.5 million
for the first quarter of 2019. The increase in Net Product Revenues
was attributed to the following:
- Naglazyme Net Product Revenues increased by $27.4 million, or 32%, primarily due to orders
from Russia and Brazil;
- Palynziq Net Product Revenues increased by $22.3 million or 181%, driven by combination of
revenue from U.S. patients achieving maintenance dosing and new
patients initiating therapy;
- Kuvan Net Product Revenues increased by $15.1 million, or 14%, primarily driven by
patient growth in North
America;
- Brineura Net Product Revenues increased by $11.8 million, or 97%, due in large part to
global patient growth;
- Vimizim Net Product Revenues increased by $11.4 million, or 9%, driven primarily due to
orders from Brazil; and
- Aldurazyme Net Product Revenues increased $10.4 million, or 23%, due to higher sales volume
to Sanofi Genzyme.
The increase in GAAP Net Income for the first quarter of 2020,
compared to GAAP Net Loss for the same period in 2019 was primarily
due to the following:
- increased gross profits of $79.1
million primarily driven by increased product sales;
- a net gain on the sale of nonfinancial assets of $59.5 million due to the divestiture and sale of
the Firdapse business; and
- decreased research and development (R&D) expenses;
partially offset by
- higher selling, general and administrative (SG&A) expense
related to pre-commercialization activities for valoctocogene
roxaparvovec, commercialization activities in support of the EU
commercial launch and continued U.S. expansion of Palynziq, and
foreign currency exchange losses.
Non-GAAP Income for the first quarter of 2020 increased to
$116.5 million, compared to Non-GAAP
Income of $24.8 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 higher gross
profit and decreased R&D expense, partially offset by higher
SG&A expense.
As of March 31, 2020, BioMarin had cash, cash equivalents
and investments totaling approximately $1.1
billion, as compared to $1.2
billion on December 31, 2019.
Commenting on first quarter 2020 results, Jean-Jacques Bienaimé,
Chairman and Chief Executive Officer of BioMarin, said, "With the
arrival of COVID-19 to the many regions where we do business,
BioMarin employees performed in unprecedented ways to ensure the
continued supply of our critically-important medicines to the
people we serve. I am proud of the commitment and dedication
demonstrated by our colleagues in these challenging times. Our
strong financial results in the first quarter underscore both the
essential-nature of our products to patients and the extraordinary
efforts made to maintain supply around the world. In the face of
the many challenges of COVID-19, our regulatory team further
progressed our next two potential commercial products. The
Biologics License Application (BLA) for valoctocogene roxaparvovec
for severe hemophilia A was accepted for Priority Review from the
FDA with an action date of August 21,
2020. This milestone represents a tremendous achievement for
BioMarin, but the potential approval of the first gene therapy in
any type of hemophilia is an even greater triumph for the
hemophilia community. They have been waiting decades for this
groundbreaking advancement and we are honored to be on this journey
together. With an approval decision for valoctocogene roxaparvovec
expected later this year, our commercial team prepares eagerly to
launch what we believe is the most innovative product yet for
people with bleeding disorders."
Mr. Bienaimé continued, "Based on positive interactions with
U.S. and European regulatory authorities in the quarter, we plan to
submit marketing applications in both regions for vosoritide to
treat children with achondroplasia in the third quarter of this
year. Our multi-pronged dossier of data encompasses long-term
clinical results in 5 to 18 year-olds, natural history data, the
ongoing study of newborns through 5 years, and highly statistically
significant placebo-controlled Phase 3 results. The positive and
significant results from our vosoritide clinical programs have led
us to believe that this potential drug could 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, "2020 is expected to be a
transformational year for BioMarin, despite impact from COVID-19 in
the near-term. The agility demonstrated by BioMarin employees in
the face of this global pandemic has enabled the continued supply
of our essential medicines to the patients who need them. And while
we expect minor financial impact in the near-term, our business is
well-positioned to weather such challenges. Our first quarter
revenue growth and improvement in profitability support our belief
that 2020 continues to look poised to be one of our most
significant value-creating years to date."
2020 Full-Year Financial Guidance
Due to the uncertainty surrounding the COVID-19 pandemic and the
potential impact on its business, BioMarin is reducing its guidance
for Total Revenues and Net Product Revenues for Vimizim, Naglazyme
and Palynziq for 2020.
Item
|
Provided February
26, 2020
|
|
Updated April 29,
2020
|
Total Revenues
(1)
|
$1,950
|
|
|
to
|
|
$2,050
|
|
|
$1,850
|
|
|
to
|
|
$1,950
|
Vimizim Net Product
Revenues
|
$560
|
|
|
to
|
|
$610
|
|
|
$530
|
|
|
to
|
|
$570
|
Kuvan Net Product
Revenues
|
$430
|
|
|
to
|
|
$480
|
|
|
Unchanged
|
Naglazyme Net Product
Revenues
|
$380
|
|
|
to
|
|
$420
|
|
|
$360
|
|
|
to
|
|
$400
|
Palynziq Net Product
Revenues
|
$180
|
|
|
to
|
|
$210
|
|
|
$160
|
|
|
to
|
|
$190
|
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
(2)
|
$20
|
|
|
to
|
|
$80
|
|
|
Unchanged
|
Non-GAAP Income
(3)
|
$260
|
|
|
to
|
|
$310
|
|
|
Unchanged
|
(1)
|
Updated 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 updated revenue guidance
assumes stabilization of such interruptions in the second half of
2020.
|
|
|
(2)
|
2020 GAAP Net Income
guidance does not reflect the potential impact on non-cash GAAP
income tax associated with the tax effects of potential
intra-entity intangible asset transfers between BioMarin entities
as a result of changing international tax laws. Any such changes,
if implemented, are not expected to have an impact on operations or
cash flows in 2020 but may have an impact on GAAP Net Income in the
form of an income tax benefit of potentially greater than $500
million.
|
|
|
(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 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: The FDA review of the BLA, under Priority
Review, for valoctocogene roxaparvovec is on-track with a PDUFA
action date of August 21, 2020. On
December 23, 2019, the Company
announced that the European Medicines Agency (EMA) validated the
Company's Marketing Authorization Application (MAA) for
valoctocogene roxaparvovec which has been in review under
accelerated assessment since January. Although the MAA
remains under accelerated assessment at this time, the Company
expects the review procedure to be extended by at least 3 months
due to COVID-19 delays. Further, the Company believes there
is a high possibility that the MAA will revert to the standard
review procedure, as is the case with most filings that initially
receive accelerated assessment. Because of the combination of
these events, the Company expects an opinion from the CHMP in late
2020/early 2021.
The Company recently received EMA licensure of its gene therapy
manufacturing facility for the production of valoctocogene
roxaparvovec, an important step in obtaining regulatory approval of
the product in the EU. The Health Products Regulatory Authority
(HPRA) of Ireland conducted, on
behalf of EMA, a pre-approval inspection in the first quarter and
issued a cGMP certification in the second quarter. The inspection
of the facility by FDA is expected to be complete during the second
quarter, which would allow for potential licensure of the facility
in the U.S. consistent with the August
21st PDUFA date.
The marketing applications are based on the Phase 3 interim
analysis and the updated three-year Phase 1/2 data from patients
treated with valoctocogene roxaparvovec. The Company believes that
both submissions represent the first time a gene therapy product
for any type of hemophilia indication is under review for marketing
authorization by health authorities.
BioMarin has dosed 134 study participants in the full GENEr8-1
Phase 3 study with 52-week results expected in the first quarter of
2021. Although the trial is open label, BioMarin has implemented a
data access plan designed to substantially mirror a blinded trial.
This plan restricts the release of any ongoing data to a small
group of medical personnel monitoring and managing the trial, and
then, only to the extent necessary to perform their monitoring
responsibilities.
BioMarin intends to provide 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 mid-2020.
- Vosoritide for children with achondroplasia: On
April 6, 2020, the Company announced
that based on recent meetings with health authorities in the U.S.
and Europe, it plans to submit
marketing applications to the FDA and EMA in the third quarter of
2020. The marketing applications are based on 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). Vosoritide is an investigational, once
daily injection analog of C-type Natriuretic Peptide (CNP). 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 no clinically
significant blood pressure decreases.
On November 14, 2019, the Company
provided an update on the ongoing Phase 2 study of vosoritide which
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
comprehensive natural history achondroplasia dataset (N=619). 2.2
cm of this additional increase occurred in the last 12 months of
treatment further informing our understanding of vosoritide's
ongoing treatment impact. These data are expected to corroborate
maintenance of effect at the time of anticipated marketing
application submissions later this year.
The vosoritide development program includes four distinct areas of
focus to support global approval. In addition to the completed
Phase 3 study and ongoing Phase 2 study in children ages 5-14
years, the global program includes a large contemporaneous natural
history study which is underway.
The fourth component of the Company's global development program
with vosoritide, includes a large Phase 2 study in infants and
young children (newborn to 60 months old) with achondroplasia, to
determine the impact of treatment in this age group. Cohorts 1 and
2 include children from ages 6 months through 5 years of age, and
has completed enrollment. Completion of enrollment of cohort 3,
which includes children ages 0 to 6 months old, is expected to be
somewhat delayed due to impact from COVID-19.
- 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.
The impact of COVID-19 has created uncertainty about when it will
be safe for patients to be dosed in PHEARLESS, our Phase 1/2 study
of BMN 307. The Company currently estimates that dosing will begin
in the second half of 2020. In the meantime, new sites are
currently 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.
Preclinical data with BMN 307 demonstrated a lifetime Phe
correction sustained at 80 weeks in mouse models. BMN 307 is an AAV
vector containing the DNA sequence that codes for the phenylalanine
hydroxylase enzyme that is deficient in people with PKU.
- BMN 331 gene therapy product candidate for Hereditary
Angioedema (HAE): On November 14,
2019, the Company announced its third gene therapy
candidate, BMN 331, for the treatment of Hereditary Angioedema
(HAE). BioMarin plans to build on its ever wider and deeper
expertise in developing gene therapies for severe hemophilia A and
PKU to improve efficiencies in the development process, and to
optimize capsid and transgene design. The Company is monitoring
developments surrounding COVID-19 but expects to begin IND-enabling
studies in mid-2020.
- Vosoritide for the treatment of Dominantly Inherited Short
Stature (DISS): On November 14,
2019, the Company announced that vosoritide will be studied
in broader genetic statural abnormalities starting with dominantly
inherited short stature (DISS), as part of a research collaboration
with Children's National Hospital. The Company plans to build on
its learnings with vosoritide in achondroplasia and look for
efficiencies in the development process, particularly around
pre-clinical research and manufacturing. The Company is monitoring
developments surrounding COVID-19 but currently expects the trial
with vosoritide for DISS to begin in the second half of 2020.
- Gene Therapy manufacturing productivity increases
capacity: On January 13,
2020, the Company announced that significant improvements in
productivity in the gene therapy facility had increased capacity
for up to 10,000 patients per year, depending on dose and product
mix.
BioMarin will host a conference call and webcast to discuss
first quarter 2020 financial results today, Wednesday,
April 29, 2020 at 4:15 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: 833.360.0852
|
Replay Dial-in
Number: 855.859.2056
|
International Dial-in
Number: 630.652.5841
|
Replay International
Dial-in Number: 404.537.3406
|
Conference ID:
6194595
|
Conference ID:
6194595
|
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 2020 being poised to be one of
the Company's most significant value-creating years to date; the
financial performance of BioMarin as a whole, including (i)
BioMarin's expectation of a minor financial impact in the near
term, (ii) BioMarin's business being well positioned to weather
such challenges (iii) that BioMarin will become profitable on a
GAAP basis for the first time in 2020 and (iv) that the
COVID-19-related demand interruptions such as missed patient
infusions and delayed treatment starts for new patients will
stabilize in the second half of 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 DISS as
part of a research collaboration with Children's National Hospital,
with a clinical trial expected to begin the second half of 2020,
(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, including BioMarin's Phase 2
program with valoctocogene roxaparvovec; the clinical development
and commercialization of BioMarin's product candidates and
commercial products, including (i) BioMarin's plans to submit
marketing applications for vosoritide for children with
achondroplasia in the third quarter of 2020, (ii) the productivity,
capacity and licensure of its gene therapy manufacturing facility,
including the timing of such licensure, and (iii) 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-K for the year
ended December 31, 2019 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,
2020 and December 31, 2019
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
March 31,
2020
|
|
December 31,
2019 (1)
|
ASSETS
|
(unaudited)
|
|
|
Current
assets:
|
|
|
|
Cash and cash
equivalents
|
$
|
476,632
|
|
|
$
|
437,446
|
|
Short-term
investments
|
381,764
|
|
|
316,361
|
|
Accounts receivable,
net
|
396,384
|
|
|
377,404
|
|
Inventory
|
705,652
|
|
|
680,275
|
|
Other current
assets
|
155,817
|
|
|
130,657
|
|
Total current
assets
|
2,116,249
|
|
|
1,942,143
|
|
Noncurrent
assets:
|
|
|
|
Long-term
investments
|
290,796
|
|
|
411,978
|
|
Property, plant and
equipment, net
|
1,009,972
|
|
|
1,010,868
|
|
Intangible assets,
net
|
443,717
|
|
|
456,580
|
|
Goodwill
|
196,199
|
|
|
197,039
|
|
Deferred tax
assets
|
539,990
|
|
|
549,422
|
|
Other
assets
|
125,918
|
|
|
122,009
|
|
Total
assets
|
$
|
4,722,841
|
|
|
$
|
4,690,039
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
Current
liabilities:
|
|
|
|
Accounts payable and
accrued liabilities
|
$
|
454,506
|
|
|
$
|
570,621
|
|
Short-term convertible
debt, net
|
365,964
|
|
|
361,882
|
|
Total current
liabilities
|
820,470
|
|
|
932,503
|
|
Noncurrent
liabilities:
|
|
|
|
Long-term convertible
debt, net
|
486,713
|
|
|
486,238
|
|
Long-term contingent
consideration
|
50,524
|
|
|
50,793
|
|
Other long-term
liabilities
|
125,172
|
|
|
98,124
|
|
Total
liabilities
|
$
|
1,482,879
|
|
|
$
|
1,567,658
|
|
Stockholders'
equity:
|
|
|
|
Common stock, $0.001
par value: 500,000,000 shares authorized; 180,761,969
and 179,838,114 shares issued and
outstanding, respectively.
|
181
|
|
|
180
|
|
Additional paid-in
capital
|
4,854,814
|
|
|
4,832,707
|
|
Company common stock
held by Nonqualified Deferred Compensation Plan
|
(9,832)
|
|
|
(9,961)
|
|
Accumulated other
comprehensive income
|
34,127
|
|
|
20,164
|
|
Accumulated
deficit
|
(1,639,328)
|
|
|
(1,720,709)
|
|
Total stockholders'
equity
|
3,239,962
|
|
|
3,122,381
|
|
Total liabilities and
stockholders' equity
|
$
|
4,722,841
|
|
|
$
|
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
|
Three Months Ended
March 31, 2020 and 2019
|
(In thousands of
U.S. dollars, except per share amounts)
|
|
|
Three Months Ended
March 31,
|
|
2020
|
|
2019
|
|
(unaudited)
|
|
(unaudited)
|
REVENUES:
|
|
|
|
Net product
revenues
|
$
|
489,043
|
|
|
$
|
394,483
|
|
Royalty and other
revenues
|
13,026
|
|
|
6,262
|
|
Total net
revenues
|
502,069
|
|
|
400,745
|
|
OPERATING
EXPENSES:
|
|
|
|
Cost of
sales
|
111,374
|
|
|
89,182
|
|
Research and
development
|
142,257
|
|
|
183,591
|
|
Selling, general and
administrative
|
187,295
|
|
|
162,158
|
|
Intangible asset
amortization and contingent consideration
|
15,677
|
|
|
19,765
|
|
Gain on sale of
nonfinancial assets
|
(59,495)
|
|
|
—
|
|
Total operating
expenses
|
397,108
|
|
|
454,696
|
|
INCOME (LOSS) FROM
OPERATIONS
|
104,961
|
|
|
(53,951)
|
|
|
|
|
|
Equity in the loss of
BioMarin/Genzyme LLC
|
(77)
|
|
|
(185)
|
|
Interest
income
|
5,244
|
|
|
6,298
|
|
Interest
expense
|
(6,915)
|
|
|
(6,727)
|
|
Other income,
net
|
(1,861)
|
|
|
1,608
|
|
INCOME (LOSS)
BEFORE INCOME TAXES
|
101,352
|
|
|
(52,957)
|
|
Provision for income
taxes
|
19,971
|
|
|
3,516
|
|
NET INCOME
(LOSS)
|
81,381
|
|
|
(56,473)
|
|
NET INCOME (LOSS)
PER SHARE, BASIC
|
$
|
0.45
|
|
|
$
|
(0.32)
|
|
NET INCOME (LOSS)
PER SHARE, DILUTED
|
$
|
0.44
|
|
|
$
|
(0.32)
|
|
Weighted average
common shares outstanding, basic
|
179,898
|
|
|
178,271
|
|
Weighted average
common shares outstanding, diluted
|
187,163
|
|
|
178,271
|
|
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,
|
|
2020
|
|
2019
|
|
%
Change
|
|
(unaudited)
|
|
(unaudited)
|
|
|
Brineura
|
$
|
24.0
|
|
|
$
|
12.2
|
|
|
97
|
%
|
Firdapse
(1)
|
1.2
|
|
|
5.1
|
|
|
(76)
|
%
|
Naglazyme
|
114.3
|
|
|
86.9
|
|
|
32
|
%
|
PKU
franchise
|
156.6
|
|
|
119.2
|
|
|
31
|
%
|
Vimizim
|
137.2
|
|
|
125.8
|
|
|
9
|
%
|
Net Product Revenues
Marketed by BioMarin
|
433.3
|
|
|
349.2
|
|
|
|
Aldurazyme Net
Product Revenues Marketed by Genzyme
|
55.7
|
|
|
45.3
|
|
|
23
|
%
|
Total Net Product
Revenues
|
$
|
489.0
|
|
|
$
|
394.5
|
|
|
|
(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 months ending March 31, 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
March
31,
|
|
2020
|
|
2019
|
|
%
Change
|
|
(unaudited)
|
|
(unaudited)
|
|
|
Kuvan
|
$
|
122.0
|
|
|
106.9
|
|
|
14
|
%
|
Palynziq
|
34.6
|
|
|
12.3
|
|
|
181
|
%
|
Total PKU
franchise
|
$
|
156.6
|
|
|
$
|
119.2
|
|
|
31
|
%
|
|
|
|
|
|
|
|
|
|
|
|
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
March
31,
|
|
Guidance
Year
Ending
|
|
2020
|
|
2019
|
|
December 31,
2020
|
GAAP Net Income
(Loss)
|
$
|
81.4
|
|
|
$
|
(56.5)
|
|
|
$
|
20.0
|
|
|
—
|
|
$
|
80.0
|
|
|
|
|
|
|
|
|
|
|
Interest expense,
net
|
1.7
|
|
|
0.4
|
|
|
3.0
|
|
|
—
|
|
2.0
|
|
Provision for income
taxes
|
20.0
|
|
|
3.5
|
|
|
4.5
|
|
|
—
|
|
10.5
|
|
Depreciation
expense
|
10.3
|
|
|
14.9
|
|
|
50.0
|
|
|
—
|
|
47.0
|
|
Amortization
expense
|
15.7
|
|
|
7.5
|
|
|
63.0
|
|
|
—
|
|
61.0
|
|
Stock-based
compensation expense
|
46.9
|
|
|
42.7
|
|
|
177.0
|
|
|
—
|
|
167.0
|
|
Contingent
consideration expense
|
—
|
|
|
12.3
|
|
|
2.0
|
|
|
—
|
|
2.0
|
|
Gain on sale of
nonfinancial assets
|
(59.5)
|
|
|
—
|
|
|
(59.5)
|
|
|
—
|
|
(59.5)
|
|
Non-GAAP
Income
|
$
|
116.5
|
|
|
$
|
24.8
|
|
|
$
|
260.0
|
|
|
—
|
|
$
|
310.0
|
|
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,
|
|
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
|
$
|
111.4
|
|
|
$
|
—
|
|
|
$
|
(5.1)
|
|
|
$
|
106.3
|
|
|
$
|
89.2
|
|
|
$
|
—
|
|
|
$
|
(4.8)
|
|
|
$
|
84.4
|
|
Research and
development
|
142.3
|
|
|
(4.8)
|
|
|
(13.7)
|
|
|
123.8
|
|
|
183.6
|
|
|
(9.3)
|
|
|
(13.8)
|
|
|
160.5
|
|
Selling, general and
administrative
|
187.3
|
|
|
(5.5)
|
|
|
(28.1)
|
|
|
153.7
|
|
|
162.2
|
|
|
(5.6)
|
|
|
(24.1)
|
|
|
132.5
|
|
Intangible asset
amortization and contingent consideration
|
15.7
|
|
|
(15.7)
|
|
|
—
|
|
|
—
|
|
|
19.8
|
|
|
(7.5)
|
|
|
(12.3)
|
|
|
—
|
|
Gain on sale of
nonfinancial assets
|
(59.5)
|
|
|
—
|
|
|
59.5
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
Interest expense,
net
|
(1.7)
|
|
|
1.7
|
|
|
—
|
|
|
—
|
|
|
(0.4)
|
|
|
0.4
|
|
|
—
|
|
|
—
|
|
Provision for income
taxes
|
20.0
|
|
|
(20.0)
|
|
|
—
|
|
|
—
|
|
|
3.5
|
|
|
(3.5)
|
|
|
—
|
|
|
—
|
|
GAAP Net Income
(Loss)/Non-GAAP Income
|
$
|
81.4
|
|
|
$
|
47.7
|
|
|
$
|
(12.6)
|
|
|
$
|
116.5
|
|
|
$
|
(56.5)
|
|
|
$
|
26.3
|
|
|
$
|
55.0
|
|
|
$
|
24.8
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Contact:
|
|
|
Investors:
|
|
Media:
|
Traci
McCarty
|
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
|
(415)
455-7451
|
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SOURCE BioMarin Pharmaceutical Inc.