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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
______________________________________
Form 10-Q
______________________________________
(Mark One)
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QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES
EXCHANGE ACT OF 1934 |
For the quarterly period ended September 30, 2020
Or
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☐ |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES
EXCHANGE ACT OF 1934 |
For the transition period from
to
.
Commission File Number: 000-26727
______________________________________
BioMarin Pharmaceutical Inc.
(Exact name of registrant as specified in its
charter)
______________________________________
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Delaware |
68-0397820 |
(State or other jurisdiction of
incorporation or organization) |
(I.R.S. Employer
Identification No.) |
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770 Lindaro Street |
San Rafael |
California |
94901 |
(Address of principal executive offices) |
(Zip Code) |
(415) 506-6700
(Registrant’s telephone number including area code)
______________________________________
Securities registered pursuant to Section 12(b) of the
Act:
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Title of each class |
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Trading Symbol(s) |
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Name of each exchange on which registered |
Common Stock, par value $0.001 |
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BMRN |
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The Nasdaq Global Select Market |
______________________________________
Indicate by check mark whether the registrant (1) has filed
all reports required to be filed by Section 13 or 15(d) of the
Securities Exchange Act of 1934 during the preceding 12 months (or
for such shorter period that the registrant was required to file
such reports), and (2) has been subject to such filing
requirements for the past 90
days. Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted
electronically every Interactive Data File required to be submitted
pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter)
during the preceding 12 months (or for such shorter period that the
registrant was required to submit such
files). Yes ☒ No ☐
Indicate by check mark whether the registrant is a large
accelerated filer, an accelerated filer, a non-accelerated filer, a
smaller reporting company, or an emerging growth company. See the
definitions of “large accelerated filer,” “accelerated filer,”
“smaller reporting company,” and “emerging growth company” in Rule
12b-2 of the Exchange Act.
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Large Accelerated Filer |
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Accelerated Filer |
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Non-accelerated Filer |
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Smaller Reporting Company |
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Emerging Growth Company |
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If an emerging growth company, indicate by check mark if the
registrant has elected not to use the extended transition period
for complying with any new or revised financial accounting
standards provided pursuant to Section 13(a) of the Exchange
Act. ☐
Indicate by check mark whether the registrant is a shell company
(as defined in Rule 12b-2 of the Exchange
Act.) Yes ☐ No ☒
Applicable only to corporate issuers:
Indicate the number of shares outstanding of each of the issuer’s
classes of common stock, as of the latest practicable date:
181,529,667 shares of common stock, par value $0.001, outstanding
as of October 23, 2020.
BIOMARIN PHARMACEUTICAL INC.
TABLE OF CONTENTS
Unless the context suggests otherwise, references in this Quarterly
Report on Form 10-Q to “BioMarin,” the “Company,” “we,” “us,” and
“our” refer to BioMarin Pharmaceutical Inc. and, where appropriate,
its wholly owned subsidiaries.
BioMarin®,
Brineura®,
Kuvan®,
Naglazyme®,
Palynziq®
and Vimizim®
are our registered trademarks. Aldurazyme®
is a registered trademark of BioMarin/Genzyme LLC. All other brand
names and service marks, trademarks and other trade names appearing
in this report are the property of their respective
owners.
Forward-Looking Statements
This Quarterly Report on Form 10-Q contains “forward-looking
statements” as defined under securities laws. Many of these
statements can be identified by the use of terminology such as
“believes,” “expects,” “intends,” “anticipates,” “plans,” “may,”
“will,” “could,” would,” “projects,” “continues,” “estimates,”
“potential,” “opportunity” or the negative versions of these terms
and other similar expressions. Our actual results or experience
could differ significantly from the forward-looking statements.
Factors that could cause or contribute to these differences include
those discussed in “Risk Factors,” in Part II, Item 1A of this
Quarterly Report on Form 10-Q as well as information provided
elsewhere in this Quarterly Report on Form 10-Q and our Annual
Report on Form 10-K for the year ended December 31, 2019,
which was filed with the Securities and Exchange Commission (the
SEC) on February 27, 2020. You should carefully consider that
information before you make an investment decision.
You should not place undue reliance on these types of
forward-looking statements, which speak only as of the date that
they were made. These forward-looking statements are based on the
beliefs and assumptions of the Company’s management based on
information currently available to management and should be
considered in connection with any written or oral forward-looking
statements that the Company may issue in the future as well as
other cautionary statements the Company has made and may make.
Except as required by law, the Company does not undertake any
obligation to release publicly any revisions to these
forward-looking statements after completion of the filing of this
Quarterly Report on Form 10-Q to reflect later events or
circumstances or the occurrence of unanticipated
events.
The discussion of the Company’s financial condition and results of
operations should be read in conjunction with the Company’s
Condensed Consolidated Financial Statements and the related Notes
thereto included in this Quarterly Report on Form
10-Q.
PART I. FINANCIAL INFORMATION
Item 1. Financial
Statements
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
September 30, 2020 and December 31, 2019
(In thousands, except share amounts)
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September 30,
2020 |
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December 31,
2019
(1)
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ASSETS |
(unaudited) |
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Current assets: |
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Cash and cash equivalents |
$ |
1,015,675 |
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$ |
437,446 |
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Short-term investments |
489,998 |
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316,361 |
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Accounts receivable, net |
411,712 |
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377,404 |
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Inventory |
700,847 |
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680,275 |
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Other current assets |
120,747 |
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130,657 |
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Total current assets |
2,738,979 |
|
|
1,942,143 |
|
Noncurrent assets: |
|
|
|
Long-term investments |
265,122 |
|
|
411,978 |
|
Property, plant and equipment, net |
1,015,062 |
|
|
1,010,868 |
|
Intangible assets, net |
427,172 |
|
|
456,580 |
|
Goodwill |
196,199 |
|
|
197,039 |
|
Deferred tax assets |
1,396,547 |
|
|
549,422 |
|
Other assets |
119,009 |
|
|
122,009 |
|
Total assets |
$ |
6,158,090 |
|
|
$ |
4,690,039 |
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
Current liabilities: |
|
|
|
Accounts payable and accrued liabilities |
$ |
480,403 |
|
|
$ |
570,621 |
|
Short-term convertible debt, net |
374,290 |
|
|
361,882 |
|
Total current liabilities |
854,693 |
|
|
932,503 |
|
|
|
|
|
Noncurrent liabilities: |
|
|
|
Long-term convertible debt, net |
1,074,164 |
|
|
486,238 |
|
Long-term contingent consideration |
54,103 |
|
|
50,793 |
|
Other long-term liabilities |
121,237 |
|
|
98,124 |
|
Total liabilities |
2,104,197 |
|
|
1,567,658 |
|
Stockholders’ equity: |
|
|
|
Common stock, $0.001 par value: 500,000,000 shares authorized;
181,492,344 and 179,838,114 shares issued and outstanding,
respectively.
|
181 |
|
|
180 |
|
Additional paid-in capital |
4,937,791 |
|
|
4,832,707 |
|
Company common stock held by Nonqualified Deferred Compensation
Plan (the NQDC)
|
(10,756) |
|
|
(9,961) |
|
Accumulated other comprehensive income |
10,385 |
|
|
20,164 |
|
Accumulated deficit |
(883,708) |
|
|
(1,720,709) |
|
Total stockholders’ equity |
4,053,893 |
|
|
3,122,381 |
|
Total liabilities and stockholders’ equity |
$ |
6,158,090 |
|
|
$ |
4,690,039 |
|
(1)December 31,
2019 balances were derived from the audited Consolidated Financial
Statements included in the Company’s Annual Report on Form 10-K for
the year ended December 31, 2019, filed with the SEC on
February 27, 2020.
The accompanying notes are an integral part of these Condensed
Consolidated Financial Statements.
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME
(LOSS)
Three and Nine Months Ended September 30, 2020 and
2019
(In thousands, except per share amounts)
(unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
REVENUES: |
|
|
|
|
|
|
|
Net product revenues |
$ |
460,741 |
|
|
$ |
450,900 |
|
|
$ |
1,368,816 |
|
|
$ |
1,224,458 |
|
Royalty and other revenues |
16,043 |
|
|
10,197 |
|
|
39,522 |
|
|
25,147 |
|
Total revenues |
476,784 |
|
|
461,097 |
|
|
1,408,338 |
|
|
1,249,605 |
|
OPERATING EXPENSES: |
|
|
|
|
|
|
|
Cost of sales |
188,793 |
|
|
96,949 |
|
|
398,134 |
|
|
263,567 |
|
Research and development |
147,053 |
|
|
172,963 |
|
|
471,449 |
|
|
542,195 |
|
Selling, general and administrative |
179,450 |
|
|
170,112 |
|
|
542,157 |
|
|
493,024 |
|
Intangible asset amortization and contingent
consideration |
17,429 |
|
|
17,063 |
|
|
48,018 |
|
|
57,114 |
|
Gain on sale of nonfinancial assets |
— |
|
|
— |
|
|
(59,495) |
|
|
(15,000) |
|
Total operating expenses |
532,725 |
|
|
457,087 |
|
|
1,400,263 |
|
|
1,340,900 |
|
INCOME (LOSS) FROM OPERATIONS |
(55,941) |
|
|
4,010 |
|
|
8,075 |
|
|
(91,295) |
|
|
|
|
|
|
|
|
|
Equity in the loss of BioMarin/Genzyme LLC |
(921) |
|
|
(551) |
|
|
(1,077) |
|
|
(780) |
|
Interest income |
4,004 |
|
|
5,340 |
|
|
13,539 |
|
|
17,537 |
|
Interest expense |
(9,597) |
|
|
(2,937) |
|
|
(24,560) |
|
|
(16,530) |
|
Other income, net |
1,239 |
|
|
3,960 |
|
|
1,886 |
|
|
6,038 |
|
INCOME (LOSS) BEFORE INCOME TAXES |
(61,216) |
|
|
9,822 |
|
|
(2,137) |
|
|
(85,030) |
|
Benefit from income taxes |
(846,019) |
|
|
(45,214) |
|
|
(839,138) |
|
|
(46,158) |
|
NET INCOME (LOSS) |
$ |
784,803 |
|
|
$ |
55,036 |
|
|
$ |
837,001 |
|
|
$ |
(38,872) |
|
NET INCOME (LOSS) PER SHARE, BASIC |
$ |
4.33 |
|
|
$ |
0.31 |
|
|
$ |
4.63 |
|
|
$ |
(0.22) |
|
NET INCOME (LOSS) PER SHARE, DILUTED |
$ |
4.01 |
|
|
$ |
0.30 |
|
|
$ |
4.39 |
|
|
$ |
(0.22) |
|
Weighted average common shares outstanding, basic |
181,142 |
|
|
179,289 |
|
|
180,592 |
|
|
178,873 |
|
Weighted average common shares outstanding, diluted |
197,674 |
|
|
185,924 |
|
|
194,959 |
|
|
178,873 |
|
|
|
|
|
|
|
|
|
COMPREHENSIVE INCOME (LOSS) |
$ |
765,138 |
|
|
$ |
74,600 |
|
|
$ |
827,222 |
|
|
$ |
(7,140) |
|
The accompanying notes are an integral part of these Condensed
Consolidated Financial Statements.
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’
EQUITY
Three and Nine Months Ended September 30, 2020 and
2019
(In thousands)
(unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
|
|
|
|
|
|
|
|
Shares of common stock, beginning balances
(1)
|
181,148 |
|
|
179,433 |
|
|
179,838 |
|
|
178,253 |
|
Issuances under equity incentive plans |
344 |
|
|
171 |
|
|
2,172 |
|
|
1,351 |
|
Repurchase of common stock |
— |
|
|
— |
|
|
(518) |
|
|
— |
|
Shares of common stock, ending balances |
181,492 |
|
|
179,604 |
|
|
181,492 |
|
|
179,604 |
|
|
|
|
|
|
|
|
|
Total stockholders' equity, beginning balances
(1)
|
$ |
3,236,679 |
|
|
$ |
2,960,954 |
|
|
$ |
3,122,381 |
|
|
$ |
2,967,940 |
|
Common stock: |
|
|
|
|
|
|
|
Beginning balances
(1)
|
181 |
|
|
179 |
|
|
180 |
|
|
178 |
|
Issuances under equity incentive plans, net of tax |
— |
|
|
1 |
|
|
1 |
|
|
2 |
|
Ending balance |
181 |
|
|
180 |
|
|
181 |
|
|
180 |
|
Additional paid-in capital: |
|
|
|
|
|
|
|
Beginning balance
(1)
|
4,885,637 |
|
|
4,744,316 |
|
|
4,832,707 |
|
|
4,669,926 |
|
Issuances under equity incentive plans, net of tax |
7,319 |
|
|
(1,294) |
|
|
17,601 |
|
|
(19,183) |
|
Stock-based compensation |
44,757 |
|
|
40,144 |
|
|
136,688 |
|
|
122,212 |
|
Repurchase of common stock |
— |
|
|
— |
|
|
(50,000) |
|
|
— |
|
Common stock held by the NQDC |
78 |
|
|
(250) |
|
|
795 |
|
|
(692) |
|
Accounting impact of NQDC Plan change |
— |
|
|
— |
|
|
— |
|
|
10,653 |
|
Ending balance
|
4,937,791 |
|
|
4,782,916 |
|
|
4,937,791 |
|
|
4,782,916 |
|
Treasury stock: |
|
|
|
|
|
|
|
Beginning balance
(1)
|
— |
|
|
— |
|
|
— |
|
|
— |
|
Purchase of treasury stock |
— |
|
|
— |
|
|
(50,000) |
|
|
— |
|
Retirement of treasury stock |
— |
|
|
— |
|
|
50,000 |
|
|
— |
|
Ending balance |
— |
|
|
— |
|
|
— |
|
|
— |
|
Company common stock held by the NQDC: |
|
|
|
|
|
|
|
Beginning balance
(1)
|
(10,678) |
|
|
(10,211) |
|
|
(9,961) |
|
|
(13,301) |
|
Common stock held by the NQDC |
(78) |
|
|
250 |
|
|
(795) |
|
|
692 |
|
Accounting impact of NQDC Plan change |
— |
|
|
— |
|
|
— |
|
|
2,648 |
|
Ending balance |
(10,756) |
|
|
(9,961) |
|
|
(10,756) |
|
|
(9,961) |
|
Accumulated other comprehensive income: |
|
|
|
|
|
|
|
Beginning balance
(1)
|
30,050 |
|
|
17,439 |
|
|
20,164 |
|
|
5,271 |
|
|
|
|
|
|
|
|
|
Other comprehensive income (loss) |
(19,665) |
|
|
19,564 |
|
|
(9,779) |
|
|
31,732 |
|
Ending balance |
10,385 |
|
|
37,003 |
|
|
10,385 |
|
|
37,003 |
|
Accumulated Deficit: |
|
|
|
|
|
|
|
Beginning balance
(1)
|
(1,668,511) |
|
|
(1,790,769) |
|
|
(1,720,709) |
|
|
(1,694,134) |
|
Impact of change in accounting principles |
— |
|
|
— |
|
|
— |
|
|
(2,727) |
|
Net income (loss) |
784,803 |
|
|
55,036 |
|
|
837,001 |
|
|
(38,872) |
|
Ending balance |
(883,708) |
|
|
(1,735,733) |
|
|
(883,708) |
|
|
(1,735,733) |
|
Total stockholders' equity, ending balances
|
$ |
4,053,893 |
|
|
$ |
3,074,405 |
|
|
$ |
4,053,893 |
|
|
$ |
3,074,405 |
|
(1)The
beginning balances for the nine-month periods were derived from the
audited Consolidated Financial Statements included in Company’s
Annual Report on Form 10-K for the years ended December 31,
2019 and 2018, respectively, filed with the SEC on February 27,
2020.
The accompanying notes are an integral part of these Condensed
Consolidated Financial Statements.
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
Nine Months Ended September 30, 2020 and 2019
(In thousands)
(unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
Nine Months Ended September 30, |
|
2020 |
|
2019 |
CASH FLOWS FROM OPERATING ACTIVITIES: |
|
|
|
Net income (loss) |
$ |
837,001 |
|
|
$ |
(38,872) |
|
Adjustments to reconcile net loss to net cash used in operating
activities: |
|
|
|
Depreciation and amortization |
77,814 |
|
|
79,510 |
|
Non-cash interest expense |
14,766 |
|
|
9,395 |
|
Amortization of premium (accretion of discount) on
investments |
175 |
|
|
(1,897) |
|
Stock-based compensation |
142,125 |
|
|
121,763 |
|
Gain on sale of nonfinancial assets |
(59,495) |
|
|
(15,000) |
|
Inventory write-off, net of stock-based compensation |
75,609 |
|
|
— |
|
Deferred income taxes |
(854,199) |
|
|
(59,780) |
|
Unrealized foreign exchange loss |
9,082 |
|
|
784 |
|
Non-cash changes in the fair value of contingent
consideration |
1,352 |
|
|
5,646 |
|
Other |
388 |
|
|
(18) |
|
Changes in operating assets and liabilities: |
|
|
|
Accounts receivable, net |
(32,915) |
|
|
(61,803) |
|
Inventory |
(73,310) |
|
|
(52,571) |
|
Other current assets |
32,848 |
|
|
(13,259) |
|
Other assets |
(5,543) |
|
|
(7,112) |
|
Accounts payable and accrued liabilities |
(76,174) |
|
|
21,747 |
|
Other long-term liabilities |
9,225 |
|
|
2,386 |
|
Net cash provided by (used in) operating activities |
98,749 |
|
|
(9,081) |
|
CASH FLOWS FROM INVESTING ACTIVITIES: |
|
|
|
Purchases of property, plant and equipment |
(83,286) |
|
|
(94,241) |
|
Maturities and sales of investments |
345,224 |
|
|
635,678 |
|
Purchases of available-for-sale securities |
(369,942) |
|
|
(528,497) |
|
Proceeds from sale of nonfinancial assets |
67,159 |
|
|
15,000 |
|
Purchase of intangible assets |
(14,369) |
|
|
(8,323) |
|
Investment in convertible note |
(8,709) |
|
|
— |
|
Other |
(725) |
|
|
(1,747) |
|
Net cash provided by (used in) investing activities |
(64,648) |
|
|
17,870 |
|
CASH FLOWS FROM FINANCING ACTIVITIES: |
|
|
|
Proceeds from exercises of awards under equity incentive
plans |
60,268 |
|
|
21,768 |
|
Taxes paid related to net share settlement of equity
awards |
(42,667) |
|
|
(40,951) |
|
Payment of contingent acquisition consideration |
— |
|
|
(57,508) |
|
Repurchase of common stock |
(50,000) |
|
|
— |
|
Proceeds from convertible senior subordinated note offering,
net |
585,752 |
|
|
— |
|
|
|
|
|
Principal repayments of financing leases |
(6,080) |
|
|
(2,025) |
|
Net cash provided by (used in) financing activities |
547,273 |
|
|
(78,716) |
|
Effect of exchange rate changes on cash |
(3,145) |
|
|
(835) |
|
NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS |
578,229 |
|
|
(70,762) |
|
Cash and cash equivalents: |
|
|
|
Beginning of period |
$ |
437,446 |
|
|
$ |
493,982 |
|
End of period |
$ |
1,015,675 |
|
|
$ |
423,220 |
|
SUPPLEMENTAL CASH FLOW DISCLOSURES: |
|
|
|
Cash paid for income taxes |
$ |
4,355 |
|
|
$ |
6,088 |
|
Cash paid for interest |
$ |
5,615 |
|
|
$ |
5,777 |
|
SUPPLEMENTAL CASH FLOW DISCLOSURES FOR NON-CASH INVESTING AND
FINANCING ACTIVITIES: |
Decrease in accounts payable and accrued liabilities related to
fixed assets |
$ |
(13,281) |
|
|
$ |
(5,801) |
|
Increase (decrease) in accounts payable and accrued liabilities
related to intangible assets |
$ |
(3,043) |
|
|
$ |
2,053 |
|
The accompanying notes are an integral part of these Condensed
Consolidated Financial Statements.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(1) NATURE OF OPERATIONS
BioMarin Pharmaceutical Inc. (the Company) 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.
The Company expects to continue to finance future cash needs that
exceed its operating activities primarily through its current cash,
cash equivalents and investments and through proceeds from debt or
equity offerings, commercial borrowing, or through collaborative
agreements with corporate partners. If the Company elects to
increase its spending on development programs significantly above
current long-term plans or enters into potential licenses and other
acquisitions of complementary technologies, products or companies,
the Company may need additional capital.
(2) BASIS OF PRESENTATION
The accompanying Condensed Consolidated Financial Statements have
been prepared pursuant to United States (U.S.) generally accepted
accounting principles (U.S. GAAP) and the rules and regulations of
the SEC for Quarterly Reports on Form 10-Q and do not include all
of the information and note disclosures required by U.S. GAAP for
complete financial statements, although the Company believes that
the disclosures herein are adequate to ensure that the information
presented is not misleading. The Condensed Consolidated Financial
Statements should therefore be read in conjunction with the
Consolidated Financial Statements and Notes thereto for the fiscal
year ended December 31, 2019 included in the Company’s Annual
Report on Form 10-K. The results of operations for the three and
nine months ended September 30, 2020 are not necessarily
indicative of the results that may be expected for the fiscal year
ending December 31, 2020 or any other period.
On January 1, 2020, the Company adopted Financial Accounting
Standards Board (FASB) Accounting Standards Update (ASU) No.
2016-13,
Financial Instruments-Credit Losses: Measurement of Credit Losses
on Financial Instruments
(ASU 2016-13), as amended, using a modified retrospective approach.
The standard has amended the guidance for measuring and recording
credit losses on financial assets measured at amortized cost by
replacing the incurred-loss model with an expected-loss model. This
new standard also requires that credit losses related to
available-for-sale debt securities be recorded as an allowance
through net income rather than by reducing the carrying amount
under the current, other-than-temporary impairment model. Results
for reporting periods beginning January 1, 2020 are presented under
ASU 2016-13 and the adoption of this standard had no impact on the
Company’s Financial Statements.
U.S. GAAP requires management to make estimates and assumptions
that affect amounts reported in the Condensed Consolidated
Financial Statements and accompanying disclosures. Although these
estimates are based on management’s best knowledge of current
events and actions that the Company may undertake in the future,
actual results may be different from those estimates. The Condensed
Consolidated Financial Statements reflect all adjustments of a
normal, recurring nature that are, in the opinion of management,
necessary for a fair presentation of results for these interim
periods. The full extent to which the novel coronavirus disease
(referred to as COVID-19) pandemic will directly or indirectly
impact the Company’s business, results of operations and financial
condition, including revenues, expenses, reserves and allowances,
manufacturing, clinical trials and research and development costs,
will depend on future developments that are highly uncertain at
this time. As events continue to evolve and additional information
becomes available, the Company’s estimates may change materially in
future periods.
Management performed an evaluation of the Company’s activities
through the date of filing of this Quarterly Report on Form 10-Q,
and has concluded that, except for the maturity and settlement of
our convertible debt discussed in Note 12 -
Debt,
there were no subsequent events or transactions that occurred
subsequent to the balance sheet date prior to filing this Quarterly
Report on Form 10-Q that would require recognition or disclosure in
the Condensed Consolidated Financial Statements.
(3) SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
There have been no material changes to the Company’s significant
accounting policies during the nine months ended September 30,
2020, as compared to the significant accounting policies disclosed
in Note 3 –
Significant Accounting Policies
included in the Company’s Annual Report on Form 10-K for the year
ended December 31, 2019.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(4) RECENT ACCOUNTING PRONOUNCEMENTS
Except as described in Note 2 –
Basis of Presentation,
there have been no new accounting pronouncements adopted by the
Company or new accounting pronouncements issued by the FASB during
the nine months ended September 30, 2020, as compared to the
recent accounting pronouncements described in Note 4 of the
Company’s Annual Report on Form 10-K for the year ended
December 31, 2019, that the Company believes are of
significance or potential significance to the Company.
(5) FINANCIAL INSTRUMENTS
All marketable securities were classified as available-for-sale at
September 30, 2020 and December 31, 2019.
The following tables show the Company’s cash, cash equivalents and
available-for-sale securities by significant investment category
for each period presented:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30, 2020 |
|
Amortized Cost |
|
Gross
Unrealized
Gains |
|
Gross
Unrealized
Losses |
|
Aggregate Fair Value |
|
Cash and Cash Equivalents |
|
Short-term
Marketable
Securities
(1)
|
|
Long-term
Marketable
Securities
(2)
|
Level 1: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash |
$ |
378,705 |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
378,705 |
|
|
$ |
378,705 |
|
|
$ |
— |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Level 2: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Money market instruments |
557,006 |
|
|
— |
|
|
— |
|
|
557,006 |
|
|
557,006 |
|
|
— |
|
|
— |
|
Corporate debt securities |
435,954 |
|
|
4,558 |
|
|
(14) |
|
|
440,498 |
|
|
— |
|
|
280,000 |
|
|
160,498 |
|
U.S. government agency securities |
361,336 |
|
|
2,182 |
|
|
(1) |
|
|
363,517 |
|
|
79,964 |
|
|
199,559 |
|
|
83,994 |
|
Asset-backed securities |
23,430 |
|
|
129 |
|
|
— |
|
|
23,559 |
|
|
— |
|
|
3,639 |
|
|
19,920 |
|
Commercial paper |
6,800 |
|
|
— |
|
|
— |
|
|
6,800 |
|
|
— |
|
|
6,800 |
|
|
— |
|
Foreign and other |
549 |
|
|
161 |
|
|
— |
|
|
710 |
|
|
— |
|
|
— |
|
|
710 |
|
Subtotal |
1,385,075 |
|
|
7,030 |
|
|
(15) |
|
|
1,392,090 |
|
|
636,970 |
|
|
489,998 |
|
|
265,122 |
|
Total |
$ |
1,763,780 |
|
|
$ |
7,030 |
|
|
$ |
(15) |
|
|
$ |
1,770,795 |
|
|
$ |
1,015,675 |
|
|
$ |
489,998 |
|
|
$ |
265,122 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, 2019 |
|
Amortized Cost |
|
Gross
Unrealized
Gains |
|
Gross
Unrealized
Losses |
|
Aggregate Fair Value |
|
Cash and Cash Equivalents |
|
Short-term
Marketable
Securities
(1)
|
|
Long-term
Marketable
Securities
(2)
|
Level 1: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash |
$ |
259,347 |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
259,347 |
|
|
$ |
259,347 |
|
|
$ |
— |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Level 2: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Money market instruments |
173,100 |
|
|
— |
|
|
— |
|
|
173,100 |
|
|
173,100 |
|
|
— |
|
|
— |
|
Corporate debt securities |
518,523 |
|
|
3,575 |
|
|
(12) |
|
|
522,086 |
|
|
— |
|
|
233,294 |
|
|
288,792 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
U.S. government agency securities |
209,633 |
|
|
993 |
|
|
(67) |
|
|
210,559 |
|
|
4,999 |
|
|
83,067 |
|
|
122,493 |
|
Foreign and other |
549 |
|
|
145 |
|
|
(1) |
|
|
693 |
|
|
— |
|
|
— |
|
|
693 |
|
Subtotal |
901,805 |
|
|
4,713 |
|
|
(80) |
|
|
906,438 |
|
|
178,099 |
|
|
316,361 |
|
|
411,978 |
|
Total |
$ |
1,161,152 |
|
|
$ |
4,713 |
|
|
$ |
(80) |
|
|
$ |
1,165,785 |
|
|
$ |
437,446 |
|
|
$ |
316,361 |
|
|
$ |
411,978 |
|
(1) The Company’s short-term marketable
securities mature in one year or less.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(2) The Company’s long-term marketable
securities mature between
one and five years.
As of September 30, 2020, the Company had the ability and
intent to hold all investments that were in an unrealized loss
position until maturity. The Company considered its intent and
ability to hold the securities until recovery of amortized cost
basis, the extent to which fair value is less than amortized cost
basis, conditions specifically related to the security’s industry
and geography, payment structure and history and changes to the
ratings (if any) in determining that the decline in fair value
compared to carrying value is not related to a credit
loss.
The Company has three investments in non-marketable equity
securities, measured using unobservable valuation inputs remeasured
on a nonrecurring basis, which are collectively considered
strategic investments. As of September 30, 2020 and
December 31, 2019, the fair value of the Company’s strategic
investments was $8.0 million and $6.2 million, respectively. These
investments were recorded in Other Assets in the Company’s
Condensed Consolidated Balance Sheets.
(6) GOODWILL AND INTANGIBLE ASSETS
Intangible assets consisted of the following:
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30,
2020 |
|
December 31,
2019 |
Intangible assets: |
|
|
|
Finite-lived intangible assets |
$ |
638,468 |
|
|
$ |
652,734 |
|
Accumulated amortization |
(211,296) |
|
|
(196,154) |
|
Net carrying value |
$ |
427,172 |
|
|
$ |
456,580 |
|
In January 2020, the Company completed the sale of worldwide rights
to Firdapse, the Company's commercial product for the treatment of
Lambert-Eaton myasthenic syndrome, to a third party in exchange for
a one-time cash payment of $67.2 million plus residual
royalties. Under the terms of the agreement, the Company agreed to
provide certain transition services to the third-party purchaser,
such as customer sales and support, for up to 12 months after the
closing of the transaction. During the first quarter of 2020, the
Company recognized a before-tax net gain of $59.5 million
related to the sale of the Firdapse intellectual property (IP) and
existing inventory. Additionally, the Company recognized a
$0.8 million reduction to goodwill and disposed of
$32.2 million in intangible assets, including related
accumulated amortization of $31.6 million, as a result of the
sale of Firdapse.
During the second quarter of 2019, the Company recorded
$15.0 million of gain on sale of intangible assets, reported
in Gain on Sale of Nonfinancial Assets in the Consolidated
Statements of Comprehensive Income (Loss), due to a third party's
achievement of a commercial sales milestone related to a previously
sold intangible asset.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(7) PROPERTY, PLANT AND EQUIPMENT
Property, plant and equipment, net consisted of the
following:
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30,
2020 |
|
December 31,
2019 |
Building and improvements |
$ |
756,479 |
|
|
$ |
725,906 |
|
Manufacturing and laboratory equipment |
402,357 |
|
|
366,951 |
|
Computer hardware and software |
187,722 |
|
|
167,554 |
|
Land |
90,418 |
|
|
90,418 |
|
Leasehold improvements |
52,479 |
|
|
51,324 |
|
Furniture and equipment |
39,360 |
|
|
38,569 |
|
Land improvements |
7,349 |
|
|
7,349 |
|
Construction-in-progress |
92,581 |
|
|
111,897 |
|
|
1,628,745 |
|
|
1,559,968 |
|
Accumulated depreciation |
(613,683) |
|
|
(549,100) |
|
Total property, plant and equipment, net |
$ |
1,015,062 |
|
|
$ |
1,010,868 |
|
The construction-in-progress balance primarily included costs
related to significant in-progress projects at the Company's
facilities in Marin County, California, and Shanbally,
Ireland.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Depreciation expense |
$ |
22,187 |
|
|
$ |
24,238 |
|
|
$ |
65,749 |
|
|
$ |
67,206 |
|
Depreciation capitalized into inventory |
$ |
10,883 |
|
|
$ |
9,807 |
|
|
$ |
34,053 |
|
|
$ |
24,894 |
|
BIOMARIN PHARMACEUTICAL INC.
NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL
STATEMENTS
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(8) SUPPLEMENTAL BALANCE SHEET INFORMATION
Inventory consisted of the following:
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30,
2020 |
|
December 31,
2019 |
Raw materials |
$ |
73,857 |
|
|
$ |
74,442 |
|
Work-in-process |
296,685 |
|
|
349,978 |
|
Finished goods |
330,305 |
|
|
255,855 |
|
Total inventory |
$ |
700,847 |
|
|
$ |
680,275 |
|
Valoctocogene roxaparvovec is an investigational gene therapy
product candidate for the treatment of severe hemophilia A. The
Company must receive marketing approval from the applicable
regulators before the valoctocogene roxaparvovec inventory can be
sold commercially. Starting in the second quarter of 2019, the
Company believed that material uncertainties related to the
ultimate regulatory approval of valoctocogene roxaparvovec for
commercial sale had been significantly reduced and included the
manufacturing-related costs for the commercial production of
valoctocogene roxaparvovec in inventory. A number of factors were
taken into consideration based on the information available at the
time, including the status in the drug development process, pivotal
clinical trial results for the underlying product candidate,
results from meetings with the relevant regulatory authorities
prior to the filing of regulatory applications, historical
experience, as well as potential impediments to the approval
process such as product safety or efficacy, as well as
commercialization and marketplace trends. If the marketing
application is rejected, the manufacturing-related costs for the
commercial production of valoctocogene roxaparvovec will be
expensed to Research and Development (R&D).
In the third quarter of 2020, the Company unexpectedly received a
Complete Response Letter from the U.S. Food and Drug Administration
(FDA) and a Joint Assessment Report from the European Medicines
Agency (EMA) respectively, both indicating that the Company’s
regulatory applications for valoctocogene roxaparvovec could not be
approved in their present form and requested additional safety and
efficacy data from the ongoing Phase 3 study. The Company evaluated
the impact of the new requirement for Phase 3 data that is
currently unknown and determined the value of the pre-launch
inventory was no longer recoverable due to delays in anticipated
regulatory approvals. As a result, the Company adjusted the
pre-launch inventory to zero, its net realizable value, and
recorded $81.2 million to Cost of Sales in the three and nine
months ended September 30, 2020.
Accounts Payable and Accrued Liabilities consisted of the
following:
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30,
2020 |
|
December 31,
2019 |
Accounts payable and accrued operating expenses |
$ |
218,021 |
|
|
$ |
240,981 |
|
Accrued compensation expense |
137,415 |
|
|
192,467 |
|
Accrued rebates payable |
65,999 |
|
|
57,163 |
|
Accrued royalties payable |
20,426 |
|
|
30,797 |
|
Lease liabilities |
10,930 |
|
|
10,700 |
|
Value added taxes payable |
7,222 |
|
|
8,395 |
|
Forward foreign currency exchange contracts |
7,774 |
|
|
10,448 |
|
Deferred revenue |
3,240 |
|
|
13,037 |
|
Other |
9,376 |
|
|
6,633 |
|
Total accounts payable and accrued liabilities |
$ |
480,403 |
|
|
$ |
570,621 |
|
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(9) FAIR VALUE MEASUREMENTS
The Company measures certain financial assets and liabilities at
fair value in accordance with the policy described in Note 3
–
Significant Accounting Policies
included in the Company’s Annual Report on Form 10-K for the year
ended December 31, 2019.
The following tables present the classification within the fair
value hierarchy of financial assets and liabilities not disclosed
elsewhere in these Condensed Consolidated Financial Statements that
are remeasured on a recurring basis as of September 30, 2020
and December 31, 2019. Other than the Company’s
fixed-rate convertible debt disclosed in Note 12 –
Debt,
there were no financial assets or liabilities that were remeasured
using a quoted price in active markets for identical assets (Level
1) as of September 30, 2020 or December 31,
2019.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Fair Value Measurements at September 30, 2020 |
|
|
Significant Other
Observable
Inputs
(Level 2) |
|
Significant
Unobservable
Inputs
(Level 3) |
|
Total |
Assets: |
|
|
|
|
|
|
Other current assets: |
|
|
|
|
|
|
NQDC Plan assets |
|
$ |
1,902 |
|
|
$ |
— |
|
|
$ |
1,902 |
|
|
|
|
|
|
|
|
Other assets: |
|
|
|
|
|
|
NQDC Plan assets |
|
17,652 |
|
|
— |
|
|
17,652 |
|
Restricted investments
(1)
|
|
2,987 |
|
|
— |
|
|
2,987 |
|
Total other assets |
|
20,639 |
|
|
— |
|
|
20,639 |
|
Total assets |
|
$ |
22,541 |
|
|
$ |
— |
|
|
$ |
22,541 |
|
Liabilities: |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
NQDC Plan liability |
|
$ |
1,902 |
|
|
$ |
— |
|
|
$ |
1,902 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other long-term liabilities: |
|
|
|
|
|
|
NQDC Plan liability |
|
17,652 |
|
|
— |
|
|
17,652 |
|
Contingent consideration |
|
— |
|
|
54,103 |
|
|
54,103 |
|
Total other long-term liabilities |
|
17,652 |
|
|
54,103 |
|
|
71,755 |
|
Total liabilities |
|
$ |
19,554 |
|
|
$ |
54,103 |
|
|
$ |
73,657 |
|
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Fair Value Measurements at December 31, 2019 |
|
|
|
Significant Other
Observable
Inputs
(Level 2) |
|
Significant
Unobservable
Inputs
(Level 3) |
|
Total |
Assets: |
|
|
|
|
|
|
|
Other current assets: |
|
|
|
|
|
|
|
NQDC Plan assets |
|
|
$ |
1,177 |
|
|
$ |
— |
|
|
$ |
1,177 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other assets: |
|
|
|
|
|
|
|
NQDC Plan assets |
|
|
16,288 |
|
|
— |
|
|
16,288 |
|
Restricted investments
(1)
|
|
|
3,168 |
|
|
— |
|
|
3,168 |
|
|
|
|
|
|
|
|
|
Total other assets |
|
|
19,456 |
|
|
— |
|
|
19,456 |
|
Total assets |
|
|
$ |
20,633 |
|
|
$ |
— |
|
|
$ |
20,633 |
|
Liabilities: |
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
NQDC Plan liability |
|
|
$ |
1,177 |
|
|
$ |
— |
|
|
$ |
1,177 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other long-term liabilities: |
|
|
|
|
|
|
|
NQDC Plan liability |
|
|
16,288 |
|
|
— |
|
|
16,288 |
|
Contingent consideration |
|
|
— |
|
|
50,793 |
|
|
50,793 |
|
Total other long-term liabilities |
|
|
16,288 |
|
|
50,793 |
|
|
67,081 |
|
Total liabilities |
|
|
$ |
17,465 |
|
|
$ |
50,793 |
|
|
$ |
68,258 |
|
(1) The restricted investments at
September 30, 2020 and December 31, 2019 secure the
Company's irrevocable standby letters of credit obtained in
connection with certain commercial agreements.
There were no transfers between levels during the three and nine
months ended September 30, 2020. The following table
represents a roll forward of contingent consideration.
|
|
|
|
|
|
Contingent consideration at December 31, 2019 |
$ |
50,793 |
|
Changes in fair value of contingent consideration |
1,352 |
|
Foreign exchange remeasurement of Euro denominated contingent
acquisition consideration |
1,958 |
|
Contingent consideration at September 30, 2020 |
$ |
54,103 |
|
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(10) DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES
The Company uses forward foreign currency exchange contracts
(forward contracts) to hedge certain operational exposures
resulting from potential changes in foreign currency exchange
rates. Such exposures result from portions of the Company’s
forecasted revenues and operating expenses being denominated in
currencies other than the U.S. Dollar (USD), primarily the Euro.
Certain of these forward contracts are designated as hedging
instruments and have maturities up to two and a half years. The
Company also enters into forward contracts that are considered to
be economic hedges that are not designated as hedging instruments
and have maturities up to three months. Whether designated or
undesignated, these forward contracts protect against the reduction
in value of forecasted foreign currency cash flows resulting from
product revenues, royalty revenues, operating expenses and asset or
liability positions designated in currencies other than the USD. To
receive hedge accounting treatment, cash flow hedges must be highly
effective in offsetting changes to expected future cash flows on
hedged transactions. The Company does not hold or issue derivative
instruments for trading or speculative purposes.
The Company is exposed to counterparty credit risk on its
derivatives. The Company has established and maintains strict
counterparty credit guidelines and enters into hedging agreements
with financial institutions that are investment grade or better to
minimize the Company’s exposure to potential defaults. The Company
is not required to pledge collateral under these
agreements.
The following table summarizes the aggregate notional amounts for
the Company’s derivatives designated as hedging instruments
outstanding as of the periods presented.
|
|
|
|
|
|
|
|
|
|
|
|
Foreign Exchange Contracts |
September 30, 2020 |
|
December 31, 2019 |
Sell |
$ |
729,998 |
|
|
$ |
820,546 |
|
Purchase |
$ |
163,116 |
|
|
$ |
212,348 |
|
The following table summarizes the aggregate notional amounts for
the Company’s derivatives not designated as hedging instruments
outstanding as of the periods presented.
|
|
|
|
|
|
|
|
|
|
|
|
Foreign Exchange Contracts |
September 30, 2020 |
|
December 31, 2019 |
Sell |
$ |
41,031 |
|
|
$ |
77,335 |
|
Purchase |
$ |
30,031 |
|
|
$ |
30,818 |
|
The fair value carrying amounts of the Company’s derivatives, as
classified within the fair value hierarchy, were as
follows:
|
|
|
|
|
|
|
|
|
|
|
|
Balance Sheet Location |
September 30, 2020 |
|
December 31, 2019 |
Derivatives designated as hedging instruments: |
|
|
|
Asset Derivatives - Level 2
(1)
|
|
|
|
Other current assets |
$ |
13,537 |
|
|
$ |
19,584 |
|
Other assets |
4,621 |
|
|
13,539 |
|
Subtotal |
$ |
18,158 |
|
|
$ |
33,123 |
|
|
|
|
|
Liability Derivatives - Level 2
(1)
|
|
|
|
Accounts payable and accrued liabilities |
$ |
7,704 |
|
|
$ |
8,184 |
|
Other long-term liabilities |
4,406 |
|
|
5,493 |
|
Subtotal |
$ |
12,110 |
|
|
$ |
13,677 |
|
|
|
|
|
Derivatives not designated as hedging instruments: |
|
|
|
Asset Derivatives - Level 2
(1)
|
|
|
|
Other current assets |
$ |
16 |
|
|
$ |
469 |
|
|
|
|
|
|
|
|
|
Liability Derivatives - Level 2
(1)
|
|
|
|
Accounts payable and accrued liabilities |
$ |
70 |
|
|
$ |
2,264 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Total Derivatives Assets |
$ |
18,174 |
|
|
$ |
33,592 |
|
Total Derivatives Liabilities |
$ |
12,180 |
|
|
$ |
15,941 |
|
(1) For additional discussion of fair value
measurements, see Note 3 –
Summary of Significant Accounting Policies
included in the Company’s Annual Report on Form 10-K for the year
ended December 31, 2019.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
The following tables summarize the impact of gains and losses from
the Company's derivatives on its Condensed Consolidated Statements
of Comprehensive Income (Loss) for the periods
presented.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
September 30, 2020 |
|
September 30, 2019 |
Derivatives Designated as Cash Flow Hedging Instruments |
|
|
Cash Flow Hedging Gains (Losses)
Reclassified into Earnings |
|
|
|
Cash Flow Hedging Gains (Losses)
Reclassified into Earnings |
|
|
|
|
|
|
|
|
Net product revenues as reported |
$ |
460,741 |
|
|
$ |
2,693 |
|
|
$ |
450,900 |
|
|
$ |
6,196 |
|
Operating expenses as reported |
$ |
532,725 |
|
|
$ |
(815) |
|
|
$ |
457,087 |
|
|
$ |
(1,388) |
|
|
|
|
|
|
|
|
|
Derivatives Not Designated as Hedging Instruments |
|
|
Gains (Losses) Recognized in Earnings |
|
|
|
Gains (Losses) Recognized in Earnings |
Operating expenses |
|
|
$ |
4,166 |
|
|
|
|
$ |
(1,286) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Nine Months Ended |
|
September 30, 2020 |
|
September 30, 2019 |
Derivatives Designated as Cash Flow Hedging Instruments |
|
|
Cash Flow Hedging Gains (Losses)
Reclassified into Earnings |
|
|
|
Cash Flow Hedging Gains (Losses)
Reclassified into Earnings |
|
|
|
|
|
|
|
|
Net product revenues as reported |
$ |
1,368,816 |
|
|
$ |
17,301 |
|
|
$ |
1,224,458 |
|
|
$ |
11,171 |
|
Operating expenses as reported |
$ |
1,400,263 |
|
|
$ |
(4,274) |
|
|
$ |
1,340,900 |
|
|
$ |
(1,885) |
|
|
|
|
|
|
|
|
|
Derivatives Not Designated as Hedging Instruments |
|
|
Gains (Losses) Recognized in Earnings |
|
|
|
Gains (Losses) Recognized in Earnings |
Operating expenses |
|
|
$ |
9,600 |
|
|
|
|
$ |
(5,182) |
|
As of September 30, 2020, the Company expected to reclassify
unrealized gains of $4.8 million from Accumulated Other
Comprehensive Income (AOCI) to earnings as the forecasted revenue
and operating expense transactions occur over the next 12 months.
For additional discussion of balances in AOCI see Note 13 –
Accumulated Other Comprehensive Income.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(11) LEASES
The following table presents the Company’s right-of-use (ROU)
assets and lease liabilities as of September 30,
2020:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Lease Classification |
|
Classification |
|
September 30,
2020 |
|
December 31,
2019 |
Assets: |
|
|
|
|
|
|
Operating |
|
Other Assets |
|
$ |
45,764 |
|
|
$ |
49,045 |
|
Financing |
|
Other Assets |
|
12,018 |
|
|
10,389 |
|
Total ROU assets |
|
|
|
$ |
57,782 |
|
|
$ |
59,434 |
|
Liabilities: |
|
|
|
|
|
|
Current: |
|
|
|
|
|
|
Operating |
|
Accounts payable and accrued liabilities |
|
$ |
7,813 |
|
|
$ |
7,451 |
|
Financing |
|
Accounts payable and accrued liabilities |
|
3,117 |
|
|
3,249 |
|
Noncurrent: |
|
|
|
|
|
|
Operating |
|
Other long-term liabilities |
|
40,831 |
|
|
44,092 |
|
Financing |
|
Other long-term liabilities |
|
4,570 |
|
|
6,708 |
|
Total lease liabilities |
|
|
|
$ |
56,331 |
|
|
$ |
61,500 |
|
Maturities of lease liabilities as of September 30, 2020 by
fiscal year were as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Maturity of Lease Liabilities |
|
Operating |
|
Financing |
|
Total |
Remainder of 2020 |
|
$ |
3,460 |
|
|
$ |
940 |
|
|
$ |
4,400 |
|
2021 |
|
9,908 |
|
|
3,080 |
|
|
12,988 |
|
2022 |
|
9,095 |
|
|
2,375 |
|
|
11,470 |
|
2023 |
|
8,055 |
|
|
1,797 |
|
|
9,852 |
|
2024 |
|
6,253 |
|
|
48 |
|
|
6,301 |
|
Thereafter |
|
21,714 |
|
|
20 |
|
|
21,734 |
|
Total lease payments |
|
58,485 |
|
|
8,260 |
|
|
66,745 |
|
Less: Interest |
|
(9,841) |
|
|
(573) |
|
|
(10,414) |
|
Present value of lease liabilities |
|
$ |
48,644 |
|
|
$ |
7,687 |
|
|
$ |
56,331 |
|
Lease costs associated with payments under the Company’s leases
were as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
Lease Cost |
|
Classification |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Operating
(1)
|
|
Operating Expenses |
|
$ |
3,227 |
|
|
$ |
3,535 |
|
|
$ |
8,953 |
|
|
$ |
9,946 |
|
Financing: |
|
|
|
|
|
|
|
|
|
|
Amortization |
|
Operating Expenses |
|
841 |
|
|
600 |
|
|
2,307 |
|
|
1,811 |
|
Interest expense |
|
Operating Expenses |
|
111 |
|
|
142 |
|
|
352 |
|
|
455 |
|
Total lease costs |
|
|
|
$ |
4,179 |
|
|
$ |
4,277 |
|
|
$ |
11,612 |
|
|
$ |
12,212 |
|
(1) Includes
short-term leases and variable lease costs, both of which were not
material in the periods presented.
The following table includes the weighted average remaining lease
terms and the weighted average discount rate used to calculate the
present value of the Company’s lease liabilities:
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other Information |
|
September 30,
2020 |
|
September 30,
2019 |
Weighted average remaining lease term (in years): |
|
|
|
|
Operating leases |
|
7.1 |
|
8.0 |
Financing leases |
|
2.8 |
|
4.2 |
|
|
|
|
|
Weighted average discount rate: |
|
|
|
|
Operating leases |
|
5.1 |
% |
|
5.2 |
% |
Financing leases |
|
5.2 |
% |
|
5.4 |
% |
As of September 30, 2020, one
operating lease is expected to commence in the fourth quarter of
2020.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Nine Months Ended September 30, |
Supplemental Cash Flow Information |
|
2020 |
|
2019 |
Cash paid for amounts included in the measurement of lease
liabilities: |
|
|
|
|
Cash used in operating activities: |
|
|
|
|
Operating leases |
|
$ |
7,007 |
|
|
$ |
5,918 |
|
Financing leases |
|
$ |
354 |
|
|
$ |
454 |
|
Cash used in financing activities: |
|
|
|
|
Financing leases |
|
$ |
6,080 |
|
|
$ |
2,027 |
|
ROU assets obtained in exchange for lease obligations: |
|
|
|
|
Operating leases |
|
$ |
3,299 |
|
|
$ |
9,268 |
|
Financing leases |
|
$ |
3,941 |
|
|
$ |
72 |
|
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(12) DEBT
Convertible Notes
As of September 30, 2020, the Company had outstanding
fixed-rate notes with varying maturities for an undiscounted
aggregate principal amount of $1.5 billion (collectively the
Notes). The Notes are senior subordinated convertible obligations,
and interest is payable in arrears, semi-annually. The following
table summarizes information regarding the Company’s convertible
debt:
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30,
2020 |
|
December 31,
2019 |
1.25% senior subordinated convertible notes due in May 2027 (the
2027 Notes)
|
$ |
600,000 |
|
|
$ |
— |
|
Unamortized discount |
(12,792) |
|
|
— |
|
Unamortized deferred offering costs |
(709) |
|
|
— |
|
2027 Notes, net |
586,499 |
|
|
— |
|
|
|
|
|
0.599% senior subordinated convertible notes due in August 2024
(the 2024 Notes)
|
495,000 |
|
|
495,000 |
|
Unamortized discount |
(5,470) |
|
|
(6,533) |
|
Unamortized deferred offering costs |
(1,865) |
|
|
(2,229) |
|
2024 Notes, net |
487,665 |
|
|
486,238 |
|
|
|
|
|
1.50% senior subordinated convertible notes due in October 2020
(the 2020 Notes)
|
374,993 |
|
|
374,993 |
|
Unamortized discount |
(649) |
|
|
(12,078) |
|
Unamortized deferred offering costs |
(54) |
|
|
(1,033) |
|
2020 Notes, net
(1)
|
374,290 |
|
|
361,882 |
|
|
|
|
|
Total convertible debt, net |
$ |
1,448,454 |
|
|
$ |
848,120 |
|
|
|
|
|
Fair value of fixed rate convertible debt
(2):
|
|
|
|
2027 Notes
|
$ |
590,010 |
|
|
$ |
— |
|
2024 Notes
|
517,730 |
|
|
521,839 |
|
2020 Notes
|
374,997 |
|
|
405,679 |
|
Total fair value of fixed rate convertible debt |
$ |
1,482,737 |
|
|
$ |
927,518 |
|
(1) The 2020 Notes are classified as a
current liability in the periods presented because they matured on
October 15, 2020 and were settled in cash for approximately
$375.0 million. No shares were issued in connection with the
settlement as the Company’s share price did not exceed the
conversion price of $94.15, as measured over a 25-day averaging
period, and the capped call transaction entered into concurrently
with the issuance of notes was not triggered. The Company incurred
no gain or loss upon the extinguishment of the 2020
Notes.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
(2) The fair value of the Company’s
fixed-rate convertible debt is based on open market trades and is
classified as Level 1 in the fair value hierarchy. For additional
discussion of fair value measurements, see Note 3 –
Summary of Significant Accounting Policies
included in the Company’s Annual Report on Form 10-K for the year
ended December 31, 2019.
Interest expense on the Company’s convertible debt consisted of the
following:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Accretion of discount on convertible notes |
$ |
4,696 |
|
|
$ |
4,005 |
|
|
$ |
13,201 |
|
|
$ |
11,860 |
|
Coupon interest expense |
4,255 |
|
|
2,271 |
|
|
9,443 |
|
|
6,681 |
|
Amortization of debt issuance costs |
535 |
|
|
508 |
|
|
1,565 |
|
|
1,522 |
|
Total interest expense on convertible debt |
$ |
9,486 |
|
|
$ |
6,784 |
|
|
$ |
24,209 |
|
|
$ |
20,063 |
|
2027 Notes
In May 2020, the Company issued $600.0 million in aggregate
principal amount of senior subordinated unsecured convertible notes
with a maturity date of May 15, 2027. The 2027 Notes were issued to
the public at par value and bear interest at the rate of 1.25% per
annum. Interest is payable semi-annually in cash in arrears on May
15 and November 15 of each year, beginning November 15, 2020. The
2027 Notes are convertible, at the option of the holder into shares
of the Company’s common stock. The initial conversion rate for the
2027 Notes is 7.2743 shares per $1,000 principal amount of the 2027
Notes, which represents a conversion price of approximately $137.47
per share, subject to adjustment under certain conditions.
Following certain corporate transactions, the Company will, in
certain circumstances, increase the conversion rate for a holder
that elects to convert its 2027 Notes in connection with such
corporate transactions by a number of additional shares of the
Company’s common stock. A holder may convert fewer than all of such
holder’s 2027 Notes so long as the amount of the 2027 Notes
converted is an integral multiple of $1,000 principal amount. Net
proceeds from the offering were $585.8 million. In connection with
the issuance of the 2027 Notes, the Company recorded a discount on
the 2027 Notes of $13.5 million, which will be accreted and
recorded as additional interest expense over the life of the 2027
Notes. The Company also incurred $0.7 million of issuance costs,
which were deferred and are being amortized over the life of the
2027 Notes and recorded as additional interest
expense.
The 2027 Notes are senior subordinated, unsecured obligations, and
rank (i) subordinated in right of payment to the prior payment in
full of all of the Company’s existing and future senior debt, (ii)
equal in right of payment with the Company’s existing and future
senior subordinated debt, (iii) senior in right of payment to the
Company’s existing and future indebtedness that is expressly
subordinated in right of payment to the notes, (vi) effectively
subordinated to the Company’s existing and future secured
indebtedness, to the extent of the value of the collateral securing
that indebtedness, and (v) structurally subordinated to all
existing and future indebtedness and other liabilities, including
trade payables, and (to the extent the Company is not a holder
thereof) preferred equity, if any, of the Company’s subsidiaries.
Upon the occurrence of a “fundamental change,” as defined in the
indenture governing the 2027 Notes, the holders may require the
Company to repurchase all or a portion of such holder’s 2027 Notes
for cash at 100% of the principal amount of the 2027 Notes being
purchased, plus any accrued and unpaid interest.
The offer and sale of the 2027 Notes and the shares of the
Company’s common stock issuable upon conversion of the 2027 Notes
have not been registered under the Securities Act or any state
securities laws and the 2027 Notes were offered only to qualified
institutional buyers as defined in Rule 144A under the Securities
Act.
See Note 13 -
Debt
in the Company’s Annual Report on Form 10-K for the year ended
December 31, 2019 for additional information related to the
Company’s convertible debt.
Revolving Credit Facility
In October 2018, the Company entered into an unsecured revolving
credit facility of up to $200.0 million (the 2018 Credit Facility).
The 2018 Credit Facility includes a letter of credit subfacility
and a swingline loan subfacility and is intended to finance ongoing
working capital needs and for other general corporate purposes.
Borrowings under the 2018 Credit Facility bear interest, at the
Company’s option, at a rate equal to either (a) the LIBOR rate
(except that if LIBOR is less than zero it shall be deemed to be
zero for purposes of the 2018 Credit Facility), or LIBOR successor
rate, plus an applicable margin ranging from 1.00% to 1.95% per
annum, based upon the Company’s net leverage ratio and earnings
before interest, taxes, depreciation and amortization (EBITDA) for
each of the two most recently ended four-quarter measurement
periods, or (b) the Base Rate, generally the prime lending rate,
plus an applicable margin ranging from 0.00% to 0.95%, based upon
the Company’s net leverage ratio and EBITDA for each of the two
most recently ended four-quarter measurement periods. Commitment
fees payable on the undrawn amount
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
range from 0.15% to 0.35% per annum based upon the Company’s net
leverage ratio and EBITDA for each of the two most recently ended
four-quarter measurement periods. The Company’s obligations under
the Credit Facility are guaranteed by its direct subsidiary,
California Corporate Center Acquisition LLC, and such obligations
may in the future be guaranteed from time to time by certain other
material domestic subsidiaries. The 2018 Credit Facility matures on
October 19, 2021, at which time all outstanding amounts become due
and payable. The Company incurred approximately $1.0 million of
issuance costs, which are amortized to Interest Expense over the
term of the 2018 Credit Facility. The 2018 Credit Facility contains
financial covenants requiring the Company to maintain a minimum
interest coverage ratio and a minimum liquidity requirement. As of
September 30, 2020, and December 31, 2019, there were no
outstanding amounts due on nor any usage of the 2018 Credit
Facility. As of September 30, 2020, the Company and certain of
its subsidiaries that served as guarantors were in compliance with
all covenants.
(13) ACCUMULATED OTHER COMPREHENSIVE INCOME
The following table summarizes amounts reclassified out of AOCI and
their effect on the Company’s Condensed Consolidated Statements of
Comprehensive Income (Loss) for the three and nine months ended
September 30, 2020 and 2019.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Condensed Consolidated
Statement of Comprehensive Income (Loss)
Classification |
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
|
|
|
2020 |
|
2019 |
|
2020 |
|
2019 |
|
|
|
|
Gains (losses) on cash flow hedges: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Forward contracts
|
Net product revenues |
|
$ |
2,693 |
|
|
$ |
6,196 |
|
|
$ |
17,301 |
|
|
$ |
11,171 |
|
|
|
|
|
Forward contracts
|
Operating expenses |
|
(815) |
|
|
(1,388) |
|
|
(4,274) |
|
|
(1,885) |
|
|
|
|
|
Total gain (loss) on cash flow hedges |
|
|
$ |
1,878 |
|
|
$ |
4,808 |
|
|
$ |
13,027 |
|
|
$ |
9,286 |
|
|
|
|
|
The following tables summarize changes in the accumulated balances
for each component of AOCI, including current period other
comprehensive income (loss) and reclassifications out of AOCI for
the three and nine months ended September 30, 2020 and
2019.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, 2020 |
|
Unrealized Gains
(Losses) on Cash
Flow Hedges |
|
Unrealized Gains
(Losses) on
Available for-Sale
Debt Securities |
|
Other |
|
Total |
AOCI balance at June 30, 2020 |
$ |
22,963 |
|
|
$ |
7,087 |
|
|
$ |
— |
|
|
$ |
30,050 |
|
Other comprehensive income (loss) before
reclassifications
|
(16,094) |
|
|
(2,201) |
|
|
— |
|
|
(18,295) |
|
Less: net gain (loss) reclassified from AOCI |
1,878 |
|
|
— |
|
|
— |
|
|
1,878 |
|
Tax effect |
— |
|
|
508 |
|
|
— |
|
|
508 |
|
Net current-period other comprehensive income (loss) |
(17,972) |
|
|
(1,693) |
|
|
— |
|
|
(19,665) |
|
AOCI balance at September 30, 2020 |
$ |
4,991 |
|
|
$ |
5,394 |
|
|
$ |
— |
|
|
$ |
10,385 |
|
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, 2019 |
|
Unrealized Gains
(Losses) on Cash
Flow Hedges |
|
Unrealized Gains
(Losses) on
Available for-Sale
Debt Securities |
|
Other |
|
Total |
AOCI balance at June 30, 2019 |
$ |
14,181 |
|
|
$ |
3,271 |
|
|
$ |
(13) |
|
|
$ |
17,439 |
|
Other comprehensive income (loss) before
reclassifications |
23,973 |
|
|
522 |
|
|
(3) |
|
|
24,492 |
|
Less: gain (loss) reclassified from AOCI |
4,808 |
|
|
— |
|
|
— |
|
|
4,808 |
|
Tax effect |
— |
|
|
(120) |
|
|
— |
|
|
(120) |
|
Net current-period other comprehensive income (loss) |
19,165 |
|
|
402 |
|
|
(3) |
|
|
19,564 |
|
AOCI balance at September 30, 2019 |
$ |
33,346 |
|
|
$ |
3,673 |
|
|
$ |
(16) |
|
|
$ |
37,003 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Nine Months Ended September 30, 2020 |
|
Unrealized Gains
(Losses) on Cash
Flow Hedges |
|
Unrealized Gains
(Losses) on
Available for-Sale
Debt Securities |
|
Other |
|
Total |
AOCI balance at December 31, 2019 |
$ |
16,614 |
|
|
$ |
3,565 |
|
|
$ |
(15) |
|
|
$ |
20,164 |
|
Other comprehensive income (loss) before
reclassifications |
1,404 |
|
|
2,379 |
|
|
15 |
|
|
3,798 |
|
Less: gain (loss) reclassified from AOCI |
13,027 |
|
|
|
|
— |
|
|
13,027 |
|
Tax effect |
— |
|
|
(550) |
|
|
— |
|
|
(550) |
|
Net current-period other comprehensive income (loss) |
(11,623) |
|
|
1,829 |
|
|
15 |
|
|
(9,779) |
|
AOCI balance at September 30, 2020 |
$ |
4,991 |
|
|
$ |
5,394 |
|
|
$ |
— |
|
|
$ |
10,385 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Nine Months Ended September 30, 2019 |
|
Unrealized Gains
(Losses) on Cash
Flow Hedges |
|
Unrealized Gains
(Losses) on
Available for-Sale
Debt Securities |
|
Other |
|
Total |
AOCI balance at December 31, 2018 |
$ |
7,201 |
|
|
$ |
(1,917) |
|
|
$ |
(13) |
|
|
$ |
5,271 |
|
Other comprehensive income (loss) before
reclassifications |
35,431 |
|
|
7,256 |
|
|
(3) |
|
|
42,684 |
|
Less: gain (loss) reclassified from AOCI |
9,286 |
|
|
— |
|
|
— |
|
|
9,286 |
|
Tax effect |
— |
|
|
(1,666) |
|
|
— |
|
|
(1,666) |
|
Net current-period other comprehensive income (loss) |
26,145 |
|
|
5,590 |
|
|
(3) |
|
|
31,732 |
|
AOCI balance at September 30, 2019 |
$ |
33,346 |
|
|
$ |
3,673 |
|
|
$ |
(16) |
|
|
$ |
37,003 |
|
(14) REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC
INFORMATION
The Company operates in one business segment, which primarily
focuses on the development and commercialization of innovative
therapies for people with serious and life-threatening rare
diseases and medical conditions. The Company considers there to be
revenue concentration risks for regions where Net Product Revenues
exceed 10% of consolidated Net Product Revenues. The concentration
of the Company’s Net Product Revenues within the regions below may
have a material adverse effect on the Company’s revenues and
results of operations if sales in the respective regions experience
difficulties.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
The following table disaggregates Total Revenues from external
customers and collaborative partners by geographic region. Net
product revenues by geographic region are based on patient location
for the Company’s commercial products, except for Aldurazyme, which
is sold exclusively to Sanofi Genzyme (Genzyme) who markets and
sells Aldurazyme world-wide. Aldurazyme revenues earned by the
Company are included in the U.S. region as the transactions are
with Genzyme, whose headquarters is located in the
U.S.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Total revenues by geographic region: |
|
|
|
|
|
|
|
United States |
$ |
252,948 |
|
|
$ |
201,874 |
|
|
$ |
729,672 |
|
|
$ |
562,217 |
|
Europe |
121,158 |
|
|
125,485 |
|
|
375,453 |
|
|
369,585 |
|
Latin America |
44,064 |
|
|
83,799 |
|
|
141,852 |
|
|
164,132 |
|
Rest of world |
58,614 |
|
|
49,939 |
|
|
161,361 |
|
|
153,671 |
|
Total revenues |
$ |
476,784 |
|
|
$ |
461,097 |
|
|
$ |
1,408,338 |
|
|
$ |
1,249,605 |
|
The following table disaggregates Net Product Revenues by
product.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Net product revenues by product: |
|
|
|
|
|
|
|
Vimizim |
$ |
147,891 |
|
|
$ |
163,466 |
|
|
$ |
401,789 |
|
|
$ |
411,953 |
|
Kuvan |
123,993 |
|
|
120,524 |
|
|
368,604 |
|
|
340,771 |
|
Naglazyme |
76,323 |
|
|
94,408 |
|
|
271,585 |
|
|
279,462 |
|
Palynziq |
46,092 |
|
|
24,142 |
|
|
121,354 |
|
|
55,250 |
|
Brineura |
25,455 |
|
|
19,840 |
|
|
75,223 |
|
|
46,815 |
|
Firdapse |
— |
|
|
5,668 |
|
|
1,316 |
|
|
16,262 |
|
Total net product revenues marketed by the Company |
$ |
419,754 |
|
|
$ |
428,048 |
|
|
$ |
1,239,871 |
|
|
$ |
1,150,513 |
|
Aldurazyme net product revenues marketed by Genzyme |
40,987 |
|
|
22,852 |
|
|
$ |
128,945 |
|
|
$ |
73,945 |
|
Total net product revenues |
$ |
460,741 |
|
|
$ |
450,900 |
|
|
$ |
1,368,816 |
|
|
$ |
1,224,458 |
|
The table below disaggregates total Net Product Revenues based on
patient location for products sold directly by the Company, and
global sales of Aldurazyme, which is marketed by
Genzyme.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
United States |
$ |
203,892 |
|
|
$ |
175,127 |
|
|
$ |
582,734 |
|
|
$ |
480,330 |
|
Europe |
115,548 |
|
|
121,363 |
|
|
360,718 |
|
|
358,041 |
|
Latin America |
44,064 |
|
|
83,799 |
|
|
141,852 |
|
|
164,132 |
|
Rest of world |
56,250 |
|
|
47,759 |
|
|
154,567 |
|
|
148,010 |
|
Total net product revenues marketed by the Company |
419,754 |
|
|
428,048 |
|
|
1,239,871 |
|
|
1,150,513 |
|
Aldurazyme net product revenues marketed by Genzyme |
40,987 |
|
|
22,852 |
|
|
128,945 |
|
|
73,945 |
|
Total net product revenues |
$ |
460,741 |
|
|
$ |
450,900 |
|
|
$ |
1,368,816 |
|
|
$ |
1,224,458 |
|
The following table illustrates the percentage of the Company’s
total Net Product Revenues attributed to the Company’s largest
customers for the periods presented.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Customer A |
16 |
% |
|
16 |
% |
|
15 |
% |
|
17 |
% |
Customer B |
14 |
% |
|
13 |
% |
|
14 |
% |
|
12 |
% |
Customer C |
11 |
% |
|
10 |
% |
|
12 |
% |
|
11 |
% |
Customer D |
1 |
% |
|
12 |
% |
|
3 |
% |
|
6 |
% |
Total |
42 |
% |
|
51 |
% |
|
44 |
% |
|
46 |
% |
On a consolidated basis, two customers accounted for 33% and 15% of
the September 30, 2020 accounts receivable balance,
respectively, compared to December 31, 2019, when two
customers accounted for 24% and 16% of the accounts receivable
balance, respectively. As of September 30, 2020, and
December 31, 2019, the accounts receivable balance for Genzyme
included $106.5 million and $60.2 million, respectively, of
unbilled accounts receivable, which becomes payable to the Company
when the product is sold by Genzyme. The Company does not require
collateral from its customers but does perform periodic credit
evaluations of its customers’ financial condition and requires
prepayments in certain circumstances.
The outbreak of COVID-19 continues to affect economies and business
around the world. The Company experienced a modest impact on its
global revenue sources, mostly in the form of demand interruptions
such as missed patient infusions and delayed treatment starts for
new patients, in the three and nine months ended September 30,
2020 and the Company anticipates a continued impact on its
financial results for the remainder of 2020 and into fiscal year
2021. The extent and duration of such effects are highly uncertain
and difficult to predict. The Company is actively monitoring and
managing its response and assessing actual and potential impacts to
its operating results and financial condition, as well as
developments in its business, which could further impact
developments, trends and expectations. See the risk factor related
to the impact of the coronavirus pandemic, “The coronavirus, or
COVID-19, pandemic could materially adversely affect our business,
results of operations and financial condition.” described in “Risk
Factors” in Part II, Item 1A of this Quarterly Report, for
additional details on the impact of the COVID-19
pandemic.
The Company is mindful that conditions in the current macroeconomic
environment could affect the Company’s ability to achieve its
goals. The Company sells its products in countries that face
economic volatility and weakness. Although the Company has
historically collected receivables from customers in certain
countries, sustained weakness or further deterioration of the local
economies and currencies and effects of the impact of the ongoing
COVID-19 pandemic may cause customers in those countries to delay
payment or be unable to pay for the Company’s products. The Company
believes that the allowances for doubtful accounts related to these
countries, if any, are adequate based on its analysis of the
specific business circumstances and expectations of collection for
each of the underlying accounts in these countries. The Company
will continue to monitor these conditions and will attempt to
adjust its business processes, as appropriate, to mitigate
macroeconomic risks to its business.
(15) STOCK-BASED COMPENSATION
The Company has stockholder-approved equity incentive plans that
provide for the granting of service-based restricted stock units
(RSUs), market-based RSUs, performance-based RSUs, stock options
and other types of awards to its employees, officers and
non-employee directors. Compensation expense included in the
Company’s Condensed Consolidated Statements of Comprehensive Income
(Loss) for all stock-based compensation arrangements was as
follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Cost of sales |
$ |
10,342 |
|
|
$ |
4,093 |
|
|
$ |
20,381 |
|
|
$ |
12,629 |
|
Research and development |
15,705 |
|
|
14,261 |
|
|
45,333 |
|
|
43,037 |
|
Selling, general and administrative |
24,122 |
|
|
20,819 |
|
|
76,411 |
|
|
66,097 |
|
Total stock-based compensation expense |
$ |
50,169 |
|
|
$ |
39,173 |
|
|
$ |
142,125 |
|
|
$ |
121,763 |
|
Stock-based compensation of $4.9 million and
$14.9 million was capitalized into inventory for the three and
nine months ended September 30, 2020, respectively.
Stock-based compensation of $5.1 million and $13.2 million was
capitalized into inventory for the three and nine months ended
September 30, 2019, respectively. Capitalized stock-based
compensation is recognized as cost of sales when the related
product is sold.
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
Restricted Stock Unit Awards with Market Conditions
In March 2020, the Compensation Committee and Board approved the
grant of 126,710 RSUs with market-based vesting conditions (base
TSR-RSUs) to certain executives. These base TSR-RSUs vest, if at
all, in full following a three-year service period only if certain
total shareholder return (TSR) results relative to the Nasdaq
Biotechnology Index comparative companies are achieved. The number
of shares that may be earned range between 0% and 200% of the base
TSR-RSUs, with a ceiling achievement level of 100% of the base
TSR-RSUs in the event the Company’s absolute TSR multiplier is
above the 50th percentile but the Company’s TSR multiplier is
negative on an absolute basis. The Company utilized a Monte Carlo
simulation model to determine the grant date fair value of $112.12
per base TSR-RSU. Compensation expense for awards with market
conditions is recognized over the service period using the
straight-line method and is not reversed if the market condition is
not met.
Restricted Stock Unit Awards with Performance
Conditions
In March 2020, the Compensation Committee and Board approved the
grant of 63,400 RSUs with performance-based vesting conditions
(base RSUs) and a grant date fair value of $73.82 per RSU. This
award is contingent upon the achievement of a three-year Non-GAAP
income target and the awarded RSUs, if any, vest ratably over a
three-year service period. The Company evaluated the target in the
context of its current long-range financial plan and determined
that attainment of the target was probable for accounting purposes
commencing in the first quarter of 2020. The number of shares that
may be earned range between 50% and 200% of the base
RSUs.
In March 2020, the Compensation Committee and Board approved the
grant of 63,400 RSUs with performance-based vesting conditions
(base RSUs) and a grant date fair value of $73.82 per RSU. This
award is contingent upon the achievement of a three-year strategic
goal target and the awarded RSUs, if any, vest ratably over a
three-year service period. The Company evaluated the target in the
context of its product candidate development pipeline and planned
regulatory activity and determined that attainment of the target
was probable for accounting purposes commencing in the first
quarter of 2020. The number of shares that may be earned range
between 50% and 200% of the base RSUs.
(16) INCOME TAXES
In the third quarter of 2020, the Company completed an intra-entity
transfer of certain intellectual property rights to an Irish
subsidiary where the Company’s Ex-US regional headquarters are
located and has significant manufacturing and commercial
operations, to better align ownership of intellectual property
rights with how the business operates. The transaction did not
result in a taxable gain; however, the Company’s Irish subsidiary
recognized a deferred tax asset for the book and tax basis
difference of the transferred intellectual property rights. As a
result, the Company recognized a deferred tax asset of
$835.1 million and related tax benefit on its Condensed
Consolidated Financial Statements based on the fair value of the
transferred intellectual property rights. The tax deductions
related to the amortization of these intangible assets will be
recognized in the future and any amortization not deducted for tax
purposes will be carried forward indefinitely under Irish tax laws.
The Company expects to be able to realize the deferred tax asset
resulting from this transaction and has not recorded a valuation
allowance as of September 30, 2020.
(17) NET INCOME (LOSS) PER COMMON SHARE
Potentially issuable shares of common stock include shares issuable
upon the exercise of outstanding employee stock option awards,
common stock issuable under the Company’s Employee Share Purchase
Plan (ESPP), unvested RSUs, common stock held by the NQDC and
contingent issuances of common stock related to the Company’s
convertible debt.
The following table sets forth the computation of basic and diluted
earnings per common share (common shares in
BIOMARIN PHARMACEUTICAL INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS -
(continued)
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Numerator: |
|
|
|
|
|
|
|
Net Income (Loss), basic |
$ |
784,803 |
|
|
$ |
55,036 |
|
|
$ |
837,001 |
|
|
$ |
(38,872) |
|
Add: Interest on convertible notes |
7,070 |
|
|
937 |
|
|
18,333 |
|
|
— |
|
Net Income (Loss), diluted |
$ |
791,873 |
|
|
$ |
55,973 |
|
|
$ |
855,334 |
|
|
$ |
(38,872) |
|
Denominator: |
|
|
|
|
|
|
|
Weighted-average common shares outstanding, basic |
181,142 |
|
|
179,289 |
|
|
180,592 |
|
|
178,873 |
|
Effect of dilutive securities: |
|
|
|
|
|
|
|
Options to purchase common stock |
1,731 |
|
|
1,560 |
|
|
1,728 |
|
|
— |
|
Common stock issuable under the 2020 notes |
3,983 |
|
|
— |
|
|
3,983 |
|
|
— |
|
Common stock issuable under the 2024 notes |
3,970 |
|
|
3,970 |
|
|
3,970 |
|
|
— |
|
Common stock issuable under the 2027 notes |
4,365 |
|
|
— |
|
|
2,373 |
|
|
— |
|
Unvested RSUs |
1,926 |
|
|
669 |
|
|
1,765 |
|
|
— |
|
Common stock potentially issuable for ESPP purchases |
346 |
|
|
231 |
|
|
337 |
|
|
— |
|
Common shares held by the NQDC |
211 |
|
|
205 |
|
|
211 |
|
|
— |
|
Weighted-average common shares outstanding, diluted |
197,674 |
|
|
185,924 |
|
|
194,959 |
|
|
178,873 |
|
Net Income (Loss) per common share, basic |
$ |
4.33 |
|
|
$ |
0.31 |
|
|
4.63 |
|
|
(0.22) |
|
Net Income (Loss) per common share, diluted |
$ |
4.01 |
|
|
$ |
0.30 |
|
|
4.39 |
|
|
(0.22) |
|
In addition to the equity instruments included in the table above,
the table below presents potential shares of common stock that were
excluded from the computation of diluted earnings per common share
as they were anti-dilutive (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Options to purchase common stock |
5,172 |
|
|
5,829 |
|
|
5,175 |
|
|
7,388 |
|
Common stock issuable under the 2020 Notes |
— |
|
|
3,983 |
|
|
— |
|
|
3,983 |
|
Common stock issuable under the 2024 Notes |
— |
|
|
— |
|
|
— |
|
|
3,970 |
|
|
|
|
|
|
|
|
|
Unvested RSUs |
2,900 |
|
|
3,360 |
|
|
3,060 |
|
|
4,029 |
|
Common stock potentially issuable for ESPP purchases |
193 |
|
|
222 |
|
|
204 |
|
|
453 |
|
Common stock held by the NQDC |
— |
|
|
— |
|
|
— |
|
|
205 |
|
Total number of potentially issuable shares |
8,265 |
|
|
13,394 |
|
|
8,439 |
|
|
20,028 |
|
The potential effect of the capped call transactions and potential
shares issuable under the 2020 Notes were excluded from the
calculation of diluted net income (loss) per share in the three and
nine months ended September 30, 2020 and 2019, as the
Company’s closing price on September 30, 2020 and 2019 did not
exceed the conversion price of $94.15 per share. The Company’s 2020
Notes matured on October 15, 2020 and were settled in cash.
The
potential effect of the capped call transactions with respect to
the 2020 Notes was excluded from the diluted net income (loss) per
share as of
September 30, 2020.There
is no similar capped call transaction associated with the 2024
Notes or the 2027 Notes. See Note 12 –
Debt
for additional information on the Company’s 2020 Notes and 2027
Notes. Refer to Note 13 -
Debt
to the Consolidated Financial Statements included in the Company’s
Annual Report on Form 10-K for the year ended December 31,
2019 for additional information related to the 2024 Notes and
details of the capped call transaction.
(18) COMMITMENTS AND CONTINGENCIES
Contingencies
From time to time the Company is involved in legal actions arising
in the normal course of its business. The process of
resolving matters through litigation or other means is inherently
uncertain and it is possible that an unfavorable resolution of
these
matters could adversely affect the Company, its results of
operations, financial condition or cash flows. The Company’s
general
BIOMARIN PHARMACEUTICAL INC.
NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL
STATEMENTS
(In thousands of U.S. Dollars, except per share amounts or as
otherwise disclosed)
practice is to expense legal fees as services are rendered in
connection with legal matters, and to accrue for liabilities when
losses
are probable and reasonably estimable.
Contingent Payments
As of September 30, 2020, the Company was subject to
contingent payments totaling approximately $622.4 million upon
achievement of certain development, regulatory and commercial sales
milestones, if they occur before certain dates in the future. Of
this amount, $235.0 million related to an early stage development
programs licensed from a third party in the second quarter of 2020,
$70.3 million related to the acquisition of certain rights and
other assets with respect to Kuvan and Palynziq from a third party
and $236.0 million related to programs that are no longer being
developed.
As of September 30, 2020, the Company recorded a total of
$54.1 million of contingent liabilities on its
Condensed
Consolidated Balances Sheet. See Note 9 –
Fair Value Measurements
to these Condensed Consolidated Financial Statements for further
information regarding the fair value of the Company’s contingent
consideration.
Other Commitments
In the normal course of business, the Company enters into various
firm purchase commitments primarily related to active
pharmaceutical ingredients, certain inventory-related items and
certain third-party R&D services. As of September 30,
2020, such commitments and other minimum contractual obligations
for clinical and post-marketing services were estimated at
approximately $150.3 million.
Item 2. Management’s Discussion and
Analysis of Financial Condition and Results of
Operations
The following discussion of our financial condition and results of
operations should be read in conjunction with our Condensed
Consolidated Financial Statements and the related Notes thereto
included in this Quarterly Report on Form 10-Q. This discussion
contains forward-looking statements that involve risks and
uncertainties. When reviewing the discussion below, you should keep
in mind the substantial risks and uncertainties that could impact
our business. In particular, we encourage you to review the risk
factor related to the impact of the coronavirus pandemic, “The
coronavirus, or COVID-19, pandemic could materially adversely
affect our business, results of operations and financial
condition.” described in “Risk Factors” in Part II, Item 1A in
this Quarterly Report on Form 10-Q, amongst the other risk factors.
These risks and uncertainties could cause actual results to differ
significantly from those projected in forward-looking statements
contained in this report or implied by past results and trends.
Forward-looking statements are statements that attempt to forecast
or anticipate future developments in our business, financial
condition or results of operations. See the section titled
“Forward-Looking Statements” that appears at the beginning of this
Quarterly Report on Form 10-Q. These statements, like all
statements in this report, speak only as of the date of this
Quarterly Report on Form 10-Q (unless another date is indicated),
and, except as required by law, we undertake no obligation to
update or revise these statements in light of future developments.
Our Condensed Consolidated Financial Statements have been prepared
in accordance with United States (U.S.) generally accepted
accounting principles (U.S GAAP) and are presented in U.S. Dollars
(USD).
Management’s Discussion and Analysis of Financial Condition and
Results of Operations (continued)
(In millions, except as otherwise disclosed)
Overview
We are a global biotechnology company that develops and
commercializes innovative therapies for people with
serious
and life-threatening rare diseases and medical conditions. We
select 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.
Our portfolio consists of several commercial therapies and multiple
clinical and preclinical product candidates. A summary of our major
commercial products, as of September 30, 2020, is provided
below:
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Major Commercial Products |
|
Indication |
|
U.S. Orphan Drug Exclusivity
Expiration
(1)
|
|
U.S. Biologic
Exclusivity
Expiration
(2)
|
|
EU Orphan Drug Exclusivity
Expiration
(1)
|
Aldurazyme (laronidase) |
|
MPS I
(3)
|
|
Expired |
|
Expired |
|
Expired |
Brineura (cerliponase alfa) |
|
CLN2
(4)
|
|
2024 |
|
2029 |
|
2027 |
Kuvan (sapropterin dihydrochloride) |
|
PKU
(5)
|
|
Expired |
|
Not Applicable |
|
2020
(6)
|
Naglazyme (galsulfase) |
|
MPS VI
(7)
|
|
Expired |
|
Expired |
|
Expired |
Palynziq (pegvaliase-pqpz)
(8)
|
|
PKU
|
|
2025 |
|
2030 |
|
2029 |
Vimizim (elosulfase alpha) |
|
MPS IVA
(9)
|
|
2021 |
|
2026 |
|
2024 |
(1)See
“Government Regulation—Orphan Drug Designation” in Part I, Item 1
of our Annual Report on Form 10-K for the year ended
December 31, 2019, filed with the SEC on February 27, 2020
(Annual Report) for further discussion
(2)See
“Government Regulation— Healthcare Reform” in Part I, Item 1 of our
Annual Report for further discussion
(3)For
the treatment of Mucopolysaccharidosis I (MPS I)
(4)For
the treatment of late infantile neuronal ceroid lipofuscinosis type
2 (CLN2)
(5)For
the treatment of phenylketonuria (PKU)
(6)Kuvan,
a small molecule therapy, has been granted orphan drug status in
the European Union (EU), which together with pediatric exclusivity,
confers 12 years of market exclusivity in the EU that expires in
December 2020
(7)For
the treatment of Mucopolysaccharidosis VI (MPS VI)
(8)For
adult patients with PKU
(9)For
the treatment of Mucopolysaccharidosis IV Type A (MPS
IVA)
A summary of our ongoing major development programs, as of
September 30, 2020, is provided below:
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Major Product Candidates
in Development |
|
Target
Indication |
|
U.S. Orphan
Designation |
|
EU Orphan
Designation |
|
Stage |
Valoctocogene roxaparvovec |
|
Severe Hemophilia A |
|
Yes |
|
Yes |
|
Clinical Phase 3 |
Vosoritide |
|
Achondroplasia |
|
Yes |
|
Yes |
|
Clinical Phase 3 |
BMN 307 |
|
PKU |
|
Yes |
|
Yes |
|
Clinical Phase 1/2 |
Uncertainty Relating to the COVID-19 Pandemic
The outbreak of novel coronavirus disease (COVID-19) continues to
affect economies and business around the world. We experienced a
modest impact on our global revenue sources, mostly in the form of
demand interruptions such as missed patient infusions and delayed
treatment starts for new patients, and our overall business
operations during the three and nine months ended
September 30, 2020. We anticipate a continued impact due to
COVID-19 on our financial results for the remainder of 2020 and
into fiscal year 2021. The extent and duration of such effects are
highly uncertain and difficult to predict. We are actively
monitoring and managing our response and assessing actual and
potential impacts to our operating results and financial condition,
as well as developments in our business, which could further impact
the developments, trends and expectations described below. See the
risk factor related to the impact of the coronavirus pandemic, “The
coronavirus, or COVID-19, pandemic could materially adversely
affect our business, results of operations and financial
condition.” described in “Risk Factors” in Part II, Item 1A of
this
Management’s Discussion and Analysis of Financial Condition and
Results of Operations (continued)
(In millions, except as otherwise disclosed)
Quarterly Report, for additional details on the impact of the
COVID-19 pandemic.
Business Developments
We continued to grow our commercial business and advance our
product candidate pipeline during 2020. We believe that the
combination of our internal research programs, acquisitions and
partnerships will allow us to continue to develop and commercialize
innovative therapies for people with serious and life-threatening
rare diseases and medical conditions. Below is a summary of recent
key business developments:
Continued Emphasis on Research and Development
•Valoctocogene
roxaparvovec - We are working with the U.S. Food and Drug
Administration (FDA) to align on steps forward to obtain marketing
approval following the August 18, 2020 Complete Response Letter
(CRL) to our Biologics License Application (BLA) for valoctocogene
roxaparvovec gene therapy for severe hemophilia A. The FDA
recommended that we complete the Phase 3 study and submit two-year
follow-up safety and efficacy data on all study participants. The
Phase 3 study was fully enrolled in November 2019 and will complete
one-year of follow-up in November 2020. We intend to share the
one-year top-line Phase 3 data in the first quarter of
2021.
Additionally, the European Medicines Agency (EMA) recently
requested the 52-week results from the full Phase 3 study cohort of
134 subjects to inform their benefit-risk assessment. To facilitate
this submission within the EMA regulatory framework, we recently
withdrew the Marketing Authorization Application (MAA) and plan to
resubmit the MAA with these data in the second quarter of
2021.
•Vosoritide
- Marketing applications for vosoritide were validated and accepted
by the EMA and FDA, respectively, in the third quarter of 2020. The
Committee for Medicinal Products for human use (CHMP) opinion is
expected in Europe in the second half of 2021. The U.S. New Drug
Application (NDA) for Vosoritide is under Standard review by the
FDA with Prescription Drug User Fee Act (PDUFA) Target Action Date
of August 20, 2021. Vosoritide is an investigational, once daily
injection of an analog of C-type Natriuretic Peptide (CNP) for
children with achondroplasia, the most common form of
disproportionate short stature in humans. Vosoritide has Orphan
Drug designation from the FDA and EMA.
•Palynziq
- In October 2020 we announced that the FDA approved our
supplemental BLA (sBLA) to increase the maximum allowable dose of
Palynziq Injection for treatment of adults with PKU to 60 mg daily.
Previously, the maximum dose was 40 mg daily. In the Phase 3 PRISM
studies, 19% of study participants required a 60 mg dose to achieve
adequate response to Palynziq.
•BMN
307 - In September 2020, we announced that we began dosing
participants in PHEARLESS, the Phase 1/2 study of BMN 307 our gene
therapy candidate for PKU, which has been granted fast track
designation by the FDA. All subjects participating in the PHEARLESS
study will receive product made at commercial scale from our gene
therapy manufacturing facility. In January 2020, both the FDA and
the Medicines and Healthcare Products Regulatory Agency in the
United Kingdom granted BMN 307 Investigational New Drug (IND)
status and approved our Clinical Trial Application (CTA),
respectively. Both the FDA and EMA have granted BMN 307 Orphan Drug
Status.
•BMN
331 - In July 2020, we began Investigational New Drug
(IND)-enabling studies for our third gene therapy product
candidate, BMN 331, for the treatment of hereditary
angioedema.
•Gene
therapy manufacturing facility - In January 2020, we announced that
significant improvements in productivity at our gene therapy
facility had increased capacity for up to 10,000 patients per year,
depending on dose and product mix.
Management’s Discussion and Analysis of Financial Condition and
Results of Operations (continued)
(In millions, except as otherwise disclosed)
Financial Highlights
Key components of our results of operations include the
following:
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Three Months Ended
September 30, |
|
Nine Months Ended
September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Total revenues |
$ |
476.8 |
|