SAN RAFAEL, Calif.,
Oct. 26, 2017 /PRNewswire/ --
Financial
Highlights (in millions of U.S. dollars, except per share data,
unaudited)
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Three Months Ended
September 30,
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Nine Months Ended
September 30,
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2017
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2016
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%
Change
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2017
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2016
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%
Change
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|
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Total
Revenues
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$
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334.1
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$
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279.9
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19
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%
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$
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955.3
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$
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816.8
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17
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%
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Aldurazyme Net
Product Revenues
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22.4
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23.7
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(5)
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%
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61.7
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58.8
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5
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%
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Brineura Net Product
Revenues
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3.1
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—
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n/a
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3.4
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—
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n/a
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Kuvan Net Product
Revenues
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105.8
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90.9
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16
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%
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300.1
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257.8
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16
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%
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Naglazyme Net Product
Revenues
|
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72.1
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|
|
77.8
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(7)
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%
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238.4
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221.6
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|
8
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%
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Vimizim Net Product
Revenues
|
|
|
90.3
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|
|
80.9
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|
12
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%
|
|
|
299.3
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|
|
260.3
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|
15
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%
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GAAP Net
Loss
|
|
$
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(12.5)
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|
$
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(37.4)
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|
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|
|
$
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(65.7)
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$
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(539.5)
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|
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GAAP Net Loss per
Share - Basic
|
|
$
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(0.07)
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$
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(0.22)
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|
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$
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(0.38)
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$
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(3.29)
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GAAP Net Loss per
Share - Diluted
|
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$
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(0.07)
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$
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(0.22)
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$
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(0.38)
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$
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(3.30)
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Non-GAAP Income
(Loss) (1)
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$
|
7.8
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$
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2.9
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$
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68.7
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$
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(8.9)
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September 30,
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December 31,
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2017
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2016
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Cash, cash
equivalents and investments
|
|
$
|
1,673.4
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$
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1,362.4
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(1)
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Non-GAAP income
(loss) 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 as detailed
below. Refer to Non-GAAP Information beginning on page 9 of this
press release for a complete discussion of the Company's Non-GAAP
financial information and reconciliations to the comparable GAAP
reported information.
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BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced
financial results for the third quarter ended September 30, 2017. For the quarter ended
September 30, 2017, GAAP Net Loss was
$(12.5) million, or $(0.07) per basic and diluted share, compared to
GAAP Net Loss of $(37.4) million, or
$(0.22) per basic and diluted share
for the quarter ended September 30,
2016. The reduction in GAAP Net Loss year over year was
primarily due to the $31.5 million
net upfront license payment received as a result of the License and
Settlement Agreements entered into with Sarepta Therapeutics Inc.
in July 2017. The decreased GAAP Net
Loss was also driven by increased net product revenues for Kuvan
and Vimizim, partially offset by a decrease in the
Benefit From Income Taxes, and increased Selling, General and
Administrative expenses for Kuvan, Brineura and Vimizim. BioMarin
also announced today that full year GAAP net loss guidance is being
reduced to between ($110) million and ($130)
million.
Non-GAAP Income for the third quarter ended September 30, 2017 was $7.8 million, compared to Non-GAAP Income of
$2.9 million for the quarter ended
September 30, 2016. BioMarin
also announced today that full year Non-GAAP Income guidance
is being increased to between $60 million
and $80 million.
Total Revenues were $334.1 million
for the third quarter of 2017, and were $955.3 million for the nine months ended
September 30, 2017, an increase of
19% and 17% respectively compared to the same periods in 2016. For
the nine months ended September 30,
2017, Kuvan net product revenues increased 16% year over
year. Growth was driven by a 9% increase in the number of
commercial patients on Kuvan therapy in the U.S and the continued
growth in the ex-North American territories acquired in 2016.
For the nine months ended September
30, 2017, Naglazyme net product revenues increased by 8%
year over year, due primarily to an increase of 7% in the number of
Naglazyme commercial patients. Vimizim net product revenues
increased 15% year over year during the nine months ended
September 30, 2017. The number of
Vimizim commercial patients increased 23% year over year.
On October 18, 2017, the Company
commented on its Total Revenue and Non-GAAP Income (Loss) trends
for the third quarter and full-year 2017. In terms of the
overall commercial business, BioMarin stated that sales of products
in markets throughout most of the world are performing at or above
internal expectations. However, the Company said the one
exception is Brazil, where a
slowdown in federal purchasing orders had extended into the third
quarter of this year. As a result, third quarter revenues
were negatively impacted. Since October 18, the Brazilian Ministry of Health has
initiated their purchasing process which is expected to result in
net product revenue from Brazil in
the fourth quarter. Based on this order Total Revenues for
full-year 2017 are confirmed to be within prior guidance.
As of September 30, 2017, BioMarin had cash, cash
equivalents and investments totaling approximately $1.7 billion, as compared to $1.4 billion on December
31, 2016.
Commenting on the quarter, Jean-Jacques Bienaimé, Chairman and
Chief Executive Officer of BioMarin, said, "We achieved a number of
important strategic milestones so far this year, including record
Total Revenues in the third quarter and the go ahead from both U.S.
and U.K. health authorities to begin Phase 3 studies with
valoctocogene roxaparvovec (formerly referred to as BMN 270) gene
therapy program for severe hemophilia A by year-end." Mr. Bienaimé
continued, "We had many significant updates at our recent R&D
Day, including the announcement of our next IND candidate BMN 290
for Freidriech's Ataxia, a rare neurologic disorder that affects
nearly 15,000 people worldwide. We were also pleased to share
that vosoritide for achondroplasia demonstrated a sustained
increase in annualized growth rate at 30 months of treatment.
For pegvaliase, we anticipate FDA action on our Biologics License
Application in the first half of 2018, as well as our planned
submission of the Marketing Authorization Application in
Europe in the first quarter of
2018. With these programs all advancing, supported by our
strong base commercial business, we have reduced our GAAP Net Loss
guidance and increased our Non-GAAP Income guidance for the
full-year 2017."
Revenues (in
millions of U.S. dollars, unaudited)
|
Total
Revenues
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|
Three Months Ended
September 30,
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|
Nine Months Ended
September 30,
|
|
|
|
2017
|
|
|
2016
|
|
|
$
Change
|
|
|
%
Change
|
|
|
2017
|
|
|
2016
|
|
|
$
Change
|
|
|
%
Change
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
Aldurazyme
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$
|
22.4
|
|
|
$
|
23.7
|
|
|
$
|
(1.3)
|
|
|
|
(5)
|
%
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|
$
|
61.7
|
|
|
$
|
58.8
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|
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$
|
2.9
|
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|
|
5
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%
|
Brineura
|
|
|
3.1
|
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|
|
—
|
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|
3.1
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n/a
|
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3.4
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—
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3.4
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n/a
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Firdapse
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5.1
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|
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5.0
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|
|
0.1
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|
|
|
2
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%
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|
14.0
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13.7
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0.3
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2
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%
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Kuvan
(1)
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|
105.8
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|
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90.9
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|
|
14.9
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|
16
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%
|
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|
300.1
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|
|
|
257.8
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|
|
|
42.3
|
|
|
|
16
|
%
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Naglazyme
(2)
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|
72.1
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77.8
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(5.7)
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(7)
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%
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|
238.4
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|
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221.6
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|
16.8
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|
8
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%
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Vimizim
(2)
|
|
|
90.3
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|
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80.9
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|
9.4
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|
12
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%
|
|
|
299.3
|
|
|
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260.3
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|
39.0
|
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|
15
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%
|
Net Product
Revenues
|
|
|
298.8
|
|
|
|
278.3
|
|
|
|
20.5
|
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|
7
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%
|
|
|
916.9
|
|
|
|
812.2
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|
|
|
104.7
|
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|
|
13
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%
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
|
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|
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|
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Royalty and Other
Revenues
|
|
|
35.3
|
|
|
|
1.6
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|
|
|
33.7
|
|
|
|
|
|
|
|
38.4
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|
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4.6
|
|
|
|
33.8
|
|
|
|
|
|
Total
Revenues
|
|
$
|
334.1
|
|
|
$
|
279.9
|
|
|
$
|
54.2
|
|
|
|
19
|
%
|
|
$
|
955.3
|
|
|
$
|
816.8
|
|
|
$
|
138.5
|
|
|
|
17
|
%
|
|
|
(1)
|
Kuvan revenue growth
was driven by a 9% increase in the number of commercial patients on
Kuvan therapy in the U.S. and continued growth in the ex-North
American territories acquired in 2016.
|
(2)
|
Naglazyme and Vimizim
net product revenues experience quarterly fluctuations primarily
due to the timing of government ordering patterns in certain
countries.
|
Details of Net
Product Revenues Attributable to Aldurazyme
|
|
|
|
Three Months Ended
September 30,
|
|
Nine Months Ended
September 30,
|
|
|
|
2017
|
|
2016
|
|
$
Change
|
|
%
Change
|
|
2017
|
|
2016
|
|
$
Change
|
|
%
Change
|
|
Aldurazyme revenue
reported by Genzyme
|
|
$
|
58.4
|
|
$
|
58.9
|
|
$
|
(0.5)
|
|
|
(1)
|
%
|
|
$
|
176.3
|
|
$
|
168.5
|
|
$
|
7.8
|
|
|
5
|
%
|
|
|
Three Months Ended
September 30,
|
|
|
Nine Months Ended
September 30,
|
|
|
2017
|
|
2016
|
|
$
Change
|
|
|
2017
|
|
2016
|
|
$
Change
|
Royalties earned from
Genzyme
|
|
$
|
24.5
|
|
$
|
26.9
|
|
$
|
(2.4)
|
|
|
$
|
74.2
|
|
$
|
71.2
|
|
$
|
3.0
|
Net product transfer
revenues (3)
|
|
|
(2.1)
|
|
|
(3.2)
|
|
|
1.1
|
|
|
|
(12.5)
|
|
|
(12.4)
|
|
|
(0.1)
|
Total Aldurazyme net
product revenues
|
|
$
|
22.4
|
|
$
|
23.7
|
|
$
|
(1.3)
|
|
|
$
|
61.7
|
|
$
|
58.8
|
|
$
|
2.9
|
|
|
(3)
|
To the extent units
shipped to third party customers by Genzyme exceed BioMarin
inventory transfers to Genzyme, BioMarin will record a decrease in
net product revenues from the amounts payable to BioMarin for the
amount of previously recognized product transfer revenue. If
BioMarin inventory transfers exceed units shipped to third party
customers by Genzyme, BioMarin will record incremental net product
transfer revenues for the period. Positive net product transfer
revenues result in the period if BioMarin transferred more units to
Genzyme than Genzyme sold to third-party customers.
|
2017 Financial
Guidance
|
|
Full-year Revenue
Guidance ($ in millions)
|
|
Item
|
|
|
|
|
|
|
Provided August 2,
2017
|
|
Updated October
26, 2017
|
Total
Revenues
|
|
$1,285 to
$1,335
|
|
$1,290 to
$1,320
|
Kuvan Net Product
Revenues
|
|
$380 to
$410
|
|
$400 to
$420
|
Naglazyme Net Product
Revenues
|
|
$300 to
$330
|
|
$310 to
$330
|
Vimizim Net Product
Revenues
|
|
$400 to
$430
|
|
$400 to
$420
|
|
|
Select Full-year
Income Statement Guidance ($ in millions, except
percentages)
|
|
Item
|
|
|
|
|
|
|
|
Provided August 2,
2017
|
|
Updated October
26, 2017
|
Cost of Sales (% of
Total Revenues)
|
|
17.5% to
18.5%
|
|
17.5% to
18.5%
|
Research and
Development Expense
|
|
$610 to
$640
|
|
$600 to
$620
|
Selling, General and
Admin. Expense
|
|
$530 to
$560
|
|
$530 to
$550
|
GAAP Net
Loss
|
|
$(115) to
$(155)
|
|
$(110) to
$(130)
|
Non-GAAP
Income
|
|
$30 to $70
|
|
$60 to $80
|
Key Program Updates at R&D Day October 18, 2017
- BMN 290 for Freidriech's Ataxia (FA): BioMarin announced
that it has selected as its next drug development candidate, BMN
290, a selective chromatin modulation therapy intended for
treatment of FA. FA is a rare autosomal recessive disorder with
worldwide prevalence of approximately 15,000, which results in
disabling neurologic and cardiac progressive decline. Currently
there are no approved disease modifying therapies for FA. In
preclinical models, BMN 290 increases frataxin expression in
affected tissues more than two-fold. BMN 290 is a second-generation
compound derived from a compound the Company acquired from Repligen
Corporation (Repligen) that had human clinical data demonstrating
increases in frataxin in FA patients. The Company selected BMN 290
for its favorable penetration into the central nervous system and
cardiac target tissues, and its preservation of the selectivity of
the original Repligen compound. The Company expects to submit the
IND application for BMN 290 in the second half of 2018.
- Valoctocogene roxaparvovec (formerly referred to as BMN 270)
gene therapy for hemophilia A: BioMarin announced today that it
had been granted Breakthrough Therapy Designation from the U.S.
Food and Drug Administration (FDA) for valoctocogene roxaparvovec.
The designation is intended to expedite the development and review
of medicines to treat a serious disease and preliminary clinical
evidence indicates that the drug may demonstrate substantial
improvement over existing therapies. The Company also announced
that the 6e13 vg/kg dose and 4e13 vg/kg dose had been cleared by
both U.S. and U.K. health authorities to begin Phase 3 studies.
The protocol for each Phase 3 study, one using the 6e13 vg/kg dose
and one using the 4e13 vg/kg dose, will likely include
approximately 40 patients for a duration of 52 weeks per study. The
Company expects to file for approval of valoctocogene roxaparvovec
with 52-week data from the Phase 3 studies. BioMarin expects to
initiate the global Phase 3 program in the fourth quarter of 2017,
complete enrollment of the last patient by the end of 2018 and
provide top-line Phase 3 data by the end of 2019.
At R&D Day, the Company provided an update on the ongoing
open-label Phase 1/2 study of the 4e13 vg/kg dose at up to 36 weeks
of observation at the September 14,
2017 data cut. Since the last data update provided during
the second quarter earnings call on August
2, 2017, five of the six patients at the 4e13 vg/kg dose
tracked to the low range of normal, and the sixth is in the mild
range for Factor VIII levels. Median annualized bleed and factor
VIII use rates for 4e13 and 6e13 vg/kg were zero after Week 4.
The World Health Organization (WHO) has approved, and BioMarin was
issued, the International Nonproprietary Name (INN) "valoctocogene
roxaparvovec" for the Company's gene therapy to treat hemophilia A.
INNs identify pharmaceutical substances or active pharmaceutical
ingredients. Each INN is a unique name that is globally recognized
and is public property. A nonproprietary name is also known as a
generic name.
BioMarin has commissioned its gene therapy manufacturing facility,
located in Novato, California.
Good Manufacturing Practices (GMP) production of valoctocogene
roxaparvovec has commenced and is intended to support clinical
development activities and anticipated commercial demand, upon
product approval. This facility is capable of supporting the
manufacturing of product for approximately 2,000 patients per year,
and the production process was developed in accordance with
International Conference on Harmonisation guidance for
Pharmaceuticals for Human Use facilitating worldwide registration
with health authorities.
- Pegvaliase for phenylketonuria (PKU): BioMarin announced
that the pegvaliase Biologics License Application (BLA) remains on
track for FDA action during the first half of 2018. The Company
plans to submit a Marketing Authorization Application to the
European Medicines Agency in the first quarter of 2018. Pegvaliase
is a PEGylated recombinant phenylalanine ammonia lyase enzyme
product that reduces blood phenylalanine (Phe) levels in adult
patients with PKU who have uncontrolled blood Phe levels on
existing management.
- Vosoritide for achondroplasia: BioMarin provided an
update on its open-label Phase 2 study of vosoritide, an analog of
C-type Natriuretic Peptide (CNP), in children with achondroplasia,
the most common form of disproportionate short stature in
humans.
Vosoritide for achondroplasia has demonstrated sustained increase
in average growth velocity over 30 months of treatment in 10
children, who completed 30 months of daily dosing at 15 µg/kg/day.
Over this period of time, patients experienced mean absolute growth
increase of approximately 4 cm over what their baseline growth
velocity would have predicted.
The sustained increase in annualized growth velocity was
accompanied by sustained improvements over time in height compared
to age- and gender-matched unaffected children as measure by
z-scores. In addition, treatment with vosoritide shows continued
improvement over time in proportionality as measured by a ratio of
the upper and lower body measurements, or U/L ratio.
The ongoing, global Phase 3 study is a randomized,
placebo-controlled study of vosoritide in approximately 110
children with achondroplasia ages 5-14 for 52 weeks. The study will
be followed by a subsequent open-label extension. Children in this
study will have completed a minimum six-month baseline study to
determine their respective baseline growth velocity prior to
entering the Phase 3 study. Vosoritide is being tested in children
in the age range where their growth plates are still open. This is
approximately 25 percent of people with achondroplasia. The Company
expects to complete enrollment of the Phase 3 study in mid-2018 and
provide top-line data in the second half of 2019.
Given the importance of early intervention in this indication, at
R&D Day, the Company announced that it will begin an
infant/toddler study in the first half of 2018 in children ages 0-5
years old.
- BMN 250 for MPS IIIB (Sanfilippo Syndrome, Type B): The
Company discussed preliminary results from the Phase 1/2 trial with
BMN 250 that demonstrated reduced heparan sulfate (HS) levels, a
biomarker in the cerebrospinal fluid (CSF), in the brains of
affected children. BMN 250, is an investigational enzyme
replacement therapy using a novel fusion of recombinant human
alpha-N-acetylglucosaminidase (NAGLU) with a peptide derived from
insulin-like growth factor 2 (IGF2), for the treatment of
Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB).
Discovered by BioMarin, BMN 250 is being studied in a multicenter,
international clinical trial evaluating safety and tolerability, as
well as cognitive function of patients with Sanfilippo B receiving
BMN 250. Designed to restore functional NAGLU activity in the
brain, BMN 250 is administered via intracerebroventricular (ICV)
infusion.
In the completed dose escalation portion of the study (Part 1),
which was primarily designed to determine safety and
pharmacodynamic activity of BMN 250, three patients received
escalating doses (30mg, 100mg, 300mg) of BMN 250 over 9 to12
months. CSF HS levels, which were markedly elevated at baseline,
were reduced to the non-affected or normal range in all three
patients, whether assessed as total or disease-specific HS.
Sanfilippo B patients are missing one of four enzymes for HS
degradation.
In those same patients, abdominal MRI scans showed significantly
enlarged liver size at baseline followed by rapid decreases in
liver size into the normal range for age with BMN 250 treatment,
suggesting that ICV-administered BMN 250 reaches the peripheral
circulation and may have activity in somatic organs. In contrast,
most Sanfilippo B patients enrolled in BioMarin's
concurrently-running observational study (250-901) had increased
liver size at baseline and experienced further increases in liver
size over time. Two of the three treated patients from the dose
escalation arm showed stabilization or some improvement compared to
their pre-dose baselines in cognitive Development Quotient (DQ), a
measure of cognitive function normalized to age. Patients with
untreated Sanfilippo B usually show progressive decline in DQ.
Conference Call Details
BioMarin will host a conference call and webcast to discuss
third quarter 2017 financial results today, Thursday, October 26, 2017 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
International Dial-in Number: 574.990.1362
Conference ID: 96054850
Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 96054850
About BioMarin
BioMarin is a global biotechnology
company that develops and commercializes innovative therapies for
patients with serious and life-threatening rare and ultra-rare
genetic diseases. The Company's portfolio consists of six approved
products and multiple clinical and pre-clinical product candidates.
For additional information, please visit www.biomarin.com.
Forward-Looking Statement
This press release contains
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 and expenses for BioMarin's commercial
products, GAAP Net Loss, Non-GAAP Income (Loss) and other specified
income statement guidance; the financial performance of BioMarin as
a whole; the timing of BioMarin's clinical studies and trials and
announcements of data from those studies and trials, including
BioMarin's Phase 3 program with valoctocogene roxaparvovec; the
ongoing Phase 3 study of vosoritide and the Phase 1/2 study of BMN
250; the continued clinical development and commercialization of
BioMarin's commercial products and product candidates; including
the filing of an IND for BMN 290 in the second half of 2018; the
possible approval and commercialization of BioMarin's product
candidates, including the filing of a Marketing Authorization
Application for pegvaliase in Europe in the first quarter of 2018; the
adequacy of production of valoctocogene roxaparvovec in the
Company's commercial gene therapy manufacturing facility; and
actions by regulatory authorities, including the expected FDA
action on the pegvaliase BLA during the first half of 2018. 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; Genzyme Corporation's success in continuing
the commercialization of Aldurazyme; results and timing of current
and planned preclinical studies and clinical trials, BioMarin's
ability to successfully manufacture its commercial products and
product candidates; the content and timing of decisions by the FDA,
the European Commission and other regulatory authorities concerning
each of the described products and product candidates; the market
for each of these products; actual sales of BioMarin's commercial
products; and those factors detailed in BioMarin's filings with the
Securities and Exchange Commission (SEC), including, without
limitation, the factors contained under the caption "Risk Factors"
in BioMarin's Quarterly Report on Form 10-Q for the quarter ended
June 30, 2017 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®,
Vimizim, Naglazyme®, Kuvan® and
Firdapse® are registered trademarks of BioMarin
Pharmaceutical Inc., or its affiliates. KyndrisaTM
is a trademark of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of
BioMarin/Genzyme LLC.
BIOMARIN
PHARMACEUTICAL INC.
|
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
September 30, 2017
and December 31, 2016
|
(In thousands of
U.S. dollars, except share and per share amounts)
|
|
|
|
September 30,
2017
|
|
|
December 31,
2016(1)
|
ASSETS
|
|
(unaudited)
|
|
|
Current
assets:
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
431,399
|
|
|
$
|
408,330
|
Short-term
investments
|
|
|
825,700
|
|
|
|
381,347
|
Accounts receivable,
net
|
|
|
251,891
|
|
|
|
215,280
|
Inventory
|
|
|
457,393
|
|
|
|
355,126
|
Other current
assets
|
|
|
83,646
|
|
|
|
61,708
|
Total current
assets
|
|
|
2,050,029
|
|
|
|
1,421,791
|
Noncurrent
assets:
|
|
|
|
|
|
|
|
Long-term
investments
|
|
|
416,304
|
|
|
|
572,711
|
Property, plant and
equipment, net
|
|
|
878,624
|
|
|
|
798,768
|
Intangible assets,
net
|
|
|
530,957
|
|
|
|
553,780
|
Goodwill
|
|
|
197,039
|
|
|
|
197,039
|
Deferred tax
assets
|
|
|
484,759
|
|
|
|
446,786
|
Other
assets
|
|
|
22,985
|
|
|
|
32,815
|
Total
assets
|
|
$
|
4,580,697
|
|
|
$
|
4,023,690
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
Accounts payable and
accrued liabilities
|
|
$
|
364,920
|
|
|
$
|
370,505
|
Short-term convertible
debt, net
|
|
|
—
|
|
|
|
22,478
|
Short-term contingent
acquisition consideration payable
|
|
|
52,609
|
|
|
|
46,327
|
Total current
liabilities
|
|
|
417,529
|
|
|
|
439,310
|
Noncurrent
liabilities:
|
|
|
|
|
|
|
|
Long-term convertible
debt, net
|
|
|
1,166,036
|
|
|
|
660,761
|
Long-term contingent
acquisition consideration payable
|
|
|
126,790
|
|
|
|
115,310
|
Other long-term
liabilities
|
|
|
56,780
|
|
|
|
42,034
|
Total
liabilities
|
|
|
1,767,135
|
|
|
|
1,257,415
|
Stockholders'
equity:
|
|
|
|
|
|
|
|
Common stock, $0.001
par value: 500,000,000 shares authorized; 175,495,350 and
172,647,588 shares issued and outstanding
as of September 30, 2017 and December 31, 2016, respectively.
|
|
|
176
|
|
|
|
173
|
Additional paid-in
capital
|
|
|
4,435,449
|
|
|
|
4,288,113
|
Company common stock
held by Nonqualified Deferred Compensation Plan
|
|
|
(14,473)
|
|
|
|
(14,321)
|
Accumulated other
comprehensive income (loss)
|
|
|
(21,434)
|
|
|
|
12,816
|
Accumulated
deficit
|
|
|
(1,586,156)
|
|
|
|
(1,520,506)
|
Total stockholders'
equity
|
|
|
2,813,562
|
|
|
|
2,766,275
|
Total liabilities and
stockholders' equity
|
|
$
|
4,580,697
|
|
|
$
|
4,023,690
|
|
|
|
|
|
|
|
|
(1)
|
December 31, 2016
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, 2016, filed with the U.S. Securities
and Exchange Commission on February 27, 2017.
|
BIOMARIN
PHARMACEUTICAL INC.
|
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
Three and Nine
Months Ended September 30, 2017 and 2016
|
(In thousands of
U.S. dollars, except per share amounts)
|
(Unaudited)
|
|
|
|
Three Months Ended
September 30,
|
|
|
Nine Months Ended
September 30,
|
|
|
2017
|
|
|
2016
|
|
|
2017
|
|
|
2016
|
REVENUES:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net product
revenues
|
|
$
|
298,752
|
|
|
$
|
278,262
|
|
|
$
|
916,868
|
|
|
$
|
812,195
|
Royalty and other
revenues
|
|
|
35,396
|
|
|
|
1,634
|
|
|
|
38,473
|
|
|
|
4,568
|
Total
revenues
|
|
|
334,148
|
|
|
|
279,896
|
|
|
|
955,341
|
|
|
|
816,763
|
OPERATING
EXPENSES:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
|
59,480
|
|
|
|
50,738
|
|
|
|
165,791
|
|
|
|
145,473
|
Research and
development
|
|
|
154,103
|
|
|
|
160,831
|
|
|
|
442,145
|
|
|
|
486,663
|
Selling, general and
administrative
|
|
|
130,532
|
|
|
|
118,758
|
|
|
|
394,056
|
|
|
|
333,635
|
Intangible asset
amortization and contingent consideration
|
|
|
3,760
|
|
|
|
9,654
|
|
|
|
26,096
|
|
|
|
(34,318)
|
Impairment of
intangible assets
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
599,118
|
Total operating
expenses
|
|
|
347,875
|
|
|
|
339,981
|
|
|
|
1,028,088
|
|
|
|
1,530,571
|
LOSS FROM
OPERATIONS
|
|
|
(13,727)
|
|
|
|
(60,085)
|
|
|
|
(72,747)
|
|
|
|
(713,808)
|
Equity in the loss of
BioMarin/Genzyme LLC
|
|
|
(253)
|
|
|
|
(104)
|
|
|
|
(996)
|
|
|
|
(374)
|
Interest
income
|
|
|
3,976
|
|
|
|
1,633
|
|
|
|
10,031
|
|
|
|
4,561
|
Interest
expense
|
|
|
(10,884)
|
|
|
|
(9,980)
|
|
|
|
(31,043)
|
|
|
|
(29,767)
|
Other income,
net
|
|
|
267
|
|
|
|
1,723
|
|
|
|
4,282
|
|
|
|
504
|
LOSS BEFORE INCOME
TAXES
|
|
|
(20,621)
|
|
|
|
(66,813)
|
|
|
|
(90,473)
|
|
|
|
(738,884)
|
Benefit from income
taxes
|
|
|
(8,094)
|
|
|
|
(29,388)
|
|
|
|
(24,823)
|
|
|
|
(199,394)
|
NET
LOSS
|
|
$
|
(12,527)
|
|
|
$
|
(37,425)
|
|
|
$
|
(65,650)
|
|
|
$
|
(539,490)
|
NET LOSS PER
SHARE, BASIC
|
|
$
|
(0.07)
|
|
|
$
|
(0.22)
|
|
|
$
|
(0.38)
|
|
|
$
|
(3.29)
|
NET LOSS PER
SHARE, DILUTED
|
|
$
|
(0.07)
|
|
|
$
|
(0.22)
|
|
|
$
|
(0.38)
|
|
|
$
|
(3.30)
|
Weighted average
common shares outstanding, basic
|
|
|
175,103
|
|
|
|
167,714
|
|
|
|
174,071
|
|
|
|
163,963
|
Weighted average
common shares outstanding, diluted
|
|
|
175,103
|
|
|
|
167,714
|
|
|
|
174,071
|
|
|
|
164,216
|
Non-GAAP Information
The results presented in this press release for the three and
nine months ended September 30, 2017
and 2016 include both GAAP information and Non-GAAP information. As
used in this release, Non-GAAP Income (Loss) is defined by the
Company as GAAP Net Loss excluding net interest expense, provision
for (benefit from) income taxes, depreciation expense, amortization
expense, stock-based compensation expense, contingent consideration
expense and certain other specified items, as detailed below. In
addition, BioMarin includes in this press release the effects of
these adjustments on certain components of GAAP Net Loss for each
of the periods presented. In this regard, Non-GAAP income (loss)
and its components, including Non-GAAP Royalty and Other Revenues,
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 and
Non-GAAP Provision for (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 (Loss) 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.
Non-GAAP Income (Loss) 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 Loss
to Non-GAAP Income (Loss):
Reconciliation of
GAAP Net Loss to Non-GAAP Income (Loss)
|
(In millions of
U.S. dollars)
|
(unaudited)
|
|
|
Three Months
Ended
|
|
|
Nine Months
Ended
|
|
|
Year
Ending
|
|
September
30,
|
|
|
September
30,
|
|
|
December 31,
2017
|
|
2017
|
|
|
2016
|
|
|
2017
|
|
|
2016
|
|
|
Guidance
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP Net
Loss
|
$
|
(12.5)
|
|
|
$
|
(37.4)
|
|
|
$
|
(65.7)
|
|
|
$
|
(539.5)
|
|
|
$(130) -
$(110)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest expense,
net
|
|
6.9
|
|
|
|
8.3
|
|
|
|
21.0
|
|
|
|
25.2
|
|
|
30
|
Benefit from income
taxes
|
|
(8.1)
|
|
|
|
(29.4)
|
|
|
|
(24.8)
|
|
|
|
(199.4)
|
|
|
(20) -
(50)
|
Depreciation
expense
|
|
13.3
|
|
|
|
18.8
|
|
|
|
36.9
|
|
|
|
42.7
|
|
|
45 - 55
|
Amortization
expense
|
|
7.6
|
|
|
|
7.5
|
|
|
|
22.7
|
|
|
|
22.6
|
|
|
30
|
Stock-based
compensation expense
|
|
35.9
|
|
|
|
32.9
|
|
|
|
106.7
|
|
|
|
97.3
|
|
|
130 - 150
|
Contingent
consideration expense (1)
|
|
(3.8)
|
|
|
|
2.2
|
|
|
|
3.4
|
|
|
|
(56.9)
|
|
|
10
|
Impairment charges
(2)
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
599.1
|
|
|
-
|
Royalty and other
revenues (3)
|
|
(31.5)
|
|
|
|
—
|
|
|
|
(31.5)
|
|
|
|
—
|
|
|
(35)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Non-GAAP Income
(Loss)
|
$
|
7.8
|
|
|
$
|
2.9
|
|
|
$
|
68.7
|
|
|
$
|
(8.9)
|
|
|
$60 - $80
|
The following reconciliation of the GAAP reported to Non-GAAP
information provides the details of the effects of the Non-GAAP
adjustments on certain components of the Company's operating
results for each of the periods presented.
Reconciliation Of
Certain GAAP Reported Information To Non-GAAP
Information
|
(In millions of
U.S. dollars)
|
(Unaudited)
|
|
|
Three Months Ended
September 30,
|
|
2017
|
|
|
2016
|
|
|
|
|
|
Adjustments
|
|
|
|
|
|
|
|
|
|
|
Adjustments
|
|
|
|
|
|
GAAP Reported
|
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
|
Royalty and
Other
Revenues,
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
|
Non-
GAAP
|
|
|
GAAP
Reported
|
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
|
Royalty and
Other
Revenues,
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
|
Non-
GAAP
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Royalty and other
revenues (3)
|
$
|
35.4
|
|
|
$
|
—
|
|
|
$
|
(31.5)
|
|
|
$
|
3.9
|
|
|
$
|
1.6
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
1.6
|
Cost of
sales
|
|
59.5
|
|
|
|
—
|
|
|
|
(3.0)
|
|
|
|
56.5
|
|
|
|
50.7
|
|
|
|
—
|
|
|
|
(2.1)
|
|
|
|
48.6
|
Research and
development
|
|
154.1
|
|
|
|
(7.6)
|
|
|
|
(13.8)
|
|
|
|
132.7
|
|
|
|
160.8
|
|
|
|
(11.1)
|
|
|
|
(14.2)
|
|
|
|
135.5
|
Selling, general
and administrative
|
|
130.5
|
|
|
|
(5.7)
|
|
|
|
(19.1)
|
|
|
|
105.7
|
|
|
|
118.8
|
|
|
|
(7.7)
|
|
|
|
(16.6)
|
|
|
|
94.5
|
Intangible asset
amortization and contingent
consideration (1)
|
|
3.8
|
|
|
|
(7.6)
|
|
|
|
3.8
|
|
|
|
—
|
|
|
|
9.7
|
|
|
|
(7.5)
|
|
|
|
(2.2)
|
|
|
|
—
|
Impairment of
intangible assets (2)
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
Interest expense,
net
|
|
(6.9)
|
|
|
|
6.9
|
|
|
|
—
|
|
|
|
—
|
|
|
|
(8.3)
|
|
|
|
8.3
|
|
|
|
—
|
|
|
|
—
|
Benefit from income
taxes
|
|
(8.1)
|
|
|
|
8.1
|
|
|
|
—
|
|
|
|
—
|
|
|
|
(29.4)
|
|
|
|
29.4
|
|
|
|
—
|
|
|
|
—
|
GAAP Net
Loss/Non-GAAP Income
(Loss)
|
|
(12.5)
|
|
|
|
19.7
|
|
|
|
0.6
|
|
|
|
7.8
|
|
|
|
(37.4)
|
|
|
|
5.2
|
|
|
|
35.1
|
|
|
|
2.9
|
|
|
Nine Months Ended
September 30,
|
|
2017
|
|
|
2016
|
|
|
|
|
|
Adjustments
|
|
|
|
|
|
|
|
|
|
|
Adjustments
|
|
|
|
|
|
GAAP Reported
|
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
|
Royalty and
Other
Revenues,
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
|
Non-
GAAP
|
|
|
GAAP Reported
|
|
|
Interest,
Taxes,
Depreciation
and
Amortization
|
|
|
Royalty and
Other
Revenues,
Stock-Based
Compensation,
Contingent
Consideration
and Other
Adjustments
|
|
|
Non-
GAAP
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Royalty and other
revenues (3)
|
$
|
38.5
|
|
|
$
|
—
|
|
|
$
|
(31.5)
|
|
|
$
|
7.0
|
|
|
$
|
4.6
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
4.6
|
Cost of
sales
|
|
165.8
|
|
|
|
—
|
|
|
|
(7.8)
|
|
|
|
158.0
|
|
|
|
145.5
|
|
|
|
—
|
|
|
|
(6.0)
|
|
|
|
139.5
|
Research and
development
|
|
442.1
|
|
|
|
(20.2)
|
|
|
|
(40.0)
|
|
|
|
381.9
|
|
|
|
486.7
|
|
|
|
(23.4)
|
|
|
|
(43.0)
|
|
|
|
420.3
|
Selling, general
and administrative
|
|
394.1
|
|
|
|
(16.7)
|
|
|
|
(58.9)
|
|
|
|
318.5
|
|
|
|
333.6
|
|
|
|
(19.3)
|
|
|
|
(48.3)
|
|
|
|
266.0
|
Intangible asset
amortization and contingent
consideration (1)
|
|
26.1
|
|
|
|
(22.7)
|
|
|
|
(3.4)
|
|
|
|
—
|
|
|
|
(34.3)
|
|
|
|
(22.6)
|
|
|
|
56.9
|
|
|
|
—
|
Impairment of
intangible assets (2)
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
599.1
|
|
|
|
—
|
|
|
|
(599.1)
|
|
|
|
—
|
Interest expense,
net
|
|
(21.0)
|
|
|
|
21.0
|
|
|
|
—
|
|
|
|
—
|
|
|
|
(25.2)
|
|
|
|
25.2
|
|
|
|
—
|
|
|
|
—
|
Benefit from income
taxes
|
|
(24.8)
|
|
|
|
24.8
|
|
|
|
—
|
|
|
|
—
|
|
|
|
(199.4)
|
|
|
|
199.4
|
|
|
|
—
|
|
|
|
—
|
GAAP Net
Loss/Non-GAAP Income
(Loss)
|
|
(65.7)
|
|
|
|
55.8
|
|
|
|
78.6
|
|
|
|
68.7
|
|
|
|
(539.5)
|
|
|
|
(108.9)
|
|
|
|
639.5
|
|
|
|
(8.9)
|
|
|
1.
|
Amounts for the three
and nine months ended September 30, 2016 include $43.8 million and
$21.1 million related to the change in probability of achieving the
Kyndrisa and Reveglucosidase alfa development milestones,
respectively.
|
2.
|
Amounts or the three
and nine months ended September 30, 2016 include $574.1 million and
$25.0 million for the impairment of intangible assets associated
with the discontinuance of the Kyndrisa and Reveglucosidase alpha
development programs, respectively.
|
3.
|
Primarily represents
the one-time upfront payment related to the License and Settlement
Agreement entered into with Sarepta Therapeutics, Inc. in July
2017.
|
Contact:
|
|
|
Investors:
|
|
Media:
|
Traci
McCarty
|
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
|
(415)
455-7451
|
View original content with
multimedia:http://www.prnewswire.com/news-releases/biomarin-announces-third-quarter-2017-financial-results-300544338.html
SOURCE BioMarin Pharmaceutical Inc.