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BIOMARIN PHARMACEUTICAL INC

For the period ended Sep 30, 2024. Filed Oct 31, 2024.

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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Sep 30, 2024 · Filed Oct 31, 2024

BMRN$57.18+2.1%Stored close · as of Nov 9, 2026

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Item 1. Financial Statements

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME

Three and Nine Months Ended September 30, 2024 and 2023

(In thousands, except per share amounts)

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
REVENUES:
Net product revenues$733,867$568,266$2,073,811$1,739,390
Royalty and other revenues11,87313,06332,79133,629
Total revenues745,740581,3292,106,6021,773,019
OPERATING EXPENSES:
Cost of sales188,457128,041444,096394,132
Research and development184,901191,314573,675540,523
Selling, general and administrative253,480215,768742,418632,894
Intangible asset amortization5,00915,68133,60646,975
Gain on sale of nonfinancial assets——(10,000)—
Total operating expenses631,847550,8041,783,7951,614,524
INCOME FROM OPERATIONS113,89330,525322,807158,495
Interest income18,05315,74057,20340,295
Interest expense(2,968)(3,779)(10,089)(11,237)
Other income (expense), net5,463(817)2,203(18,317)
INCOME BEFORE INCOME TAXES134,44141,669372,124169,236
Provision for income taxes28,3611,29170,20821,966
NET INCOME$106,080$40,378$301,916$147,270
EARNINGS PER SHARE, BASIC$0.56$0.21$1.59$0.78
EARNINGS PER SHARE, DILUTED$0.55$0.21$1.56$0.77
Weighted average common shares outstanding, basic190,429188,219189,806187,617
Weighted average common shares outstanding, diluted197,147191,173196,683195,042
COMPREHENSIVE INCOME$83,931$65,252$335,354$157,394

CONDENSED CONSOLIDATED BALANCE SHEETS

September 30, 2024 and December 31, 2023

(In thousands, except share amounts)

September 30, 2024December 31, 2023 ⁽¹⁾
ASSETS · (unaudited)
Current assets:
Cash and cash equivalents$675,448$755,127
Short-term investments254,996318,683
Accounts receivable, net777,547633,704
Inventory1,179,3391,107,183
Other current assets169,260141,391
Total current assets3,056,5902,956,088
Noncurrent assets:
Long-term investments561,985611,135
Property, plant and equipment, net1,045,4081,066,133
Intangible assets, net260,920294,701
Goodwill196,199196,199
Deferred tax assets1,530,7791,545,809
Other assets199,314171,538
Total assets$6,851,195$6,841,603
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable and accrued liabilities$715,658$683,147
Short-term convertible debt, net—493,877
Total current liabilities715,6581,177,024
Noncurrent liabilities:
Long-term convertible debt, net594,627593,095
Other long-term liabilities127,514119,935
Total liabilities1,437,7991,890,054
Stockholders’ equity:
Common stock, $0.001 par value: 500,000,000 shares authorized; 190,553,918 and 188,598,154 shares issued and outstanding, respectively191189
Additional paid-in capital5,739,9105,611,562
Company common stock held by the Nonqualified Deferred Compensation Plan(11,717)(9,860)
Accumulated other comprehensive income (loss)4,650(28,788)
Accumulated deficit(319,638)(621,554)
Total stockholders’ equity5,413,3964,951,549
Total liabilities and stockholders’ equity$6,851,195$6,841,603

(1)December 31, 2023 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, 2023, filed with the SEC on February 26, 2024.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

Three and Nine Months Ended September 30, 2024 and 2023

(In thousands)

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
Shares of common stock, beginning balances (1)190,356188,152188,598186,251
Issuances under equity incentive plans1981871,9562,088
Shares of common stock, ending balances190,554188,339190,554188,339
Total stockholders' equity, beginning balances (1)$5,286,299$4,782,827$4,951,549$4,603,156
Common stock:
Beginning balances (1)190188189186
Issuances under equity incentive plans, net of tax1—22
Ending balances191188191188
Additional paid-in capital:
Beginning balances (1)5,696,7015,493,9565,611,5625,404,895
Issuances under equity incentive plans, net of tax(2,688)(1,827)(33,031)(19,778)
Stock-based compensation45,85350,645159,522156,123
Change in Common stock held by the Nonqualified Deferred Compensation plan (NQDC)44—1,8571,534
Ending balances5,739,9105,542,7745,739,9105,542,774
Company common stock held by the NQDC:
Beginning balances (1)(11,673)(10,393)(9,860)(8,859)
Common stock held by the NQDC(44)—(1,857)(1,534)
Ending balances(11,717)(10,393)(11,717)(10,393)
Accumulated other comprehensive income (loss):
Beginning balances (1)26,799(18,617)(28,788)(3,867)
Other comprehensive income (loss)(22,149)24,87433,43810,124
Ending balances4,6506,2574,6506,257
Accumulated Deficit:
Beginning balances (1)(425,718)(682,307)(621,554)(789,199)
Net income106,08040,378301,916147,270
Ending balances(319,638)(641,929)(319,638)(641,929)
Total stockholders' equity, ending balances$5,413,396$4,896,897$5,413,396$4,896,897

(1)The beginning balances for the nine-month periods 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, 2023 filed with the SEC on February 26, 2024.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

Nine Months Ended September 30, 2024 and 2023

(In thousands)

Nine Months EndedSeptember 30,
20242023
CASH FLOWS FROM OPERATING ACTIVITIES:
Net income$301,916$147,270
Adjustments to reconcile net income to net cash provided by operating activities:
Depreciation and amortization72,81977,525
Non-cash interest expense2,6993,198
Accretion of discount on investments(6,619)(6,781)
Stock-based compensation149,652152,244
Gain on sale of nonfinancial assets(10,000)—
Impairment of assets and other non-cash adjustments19,88912,650
Deferred income taxes13,709(20,137)
Unrealized foreign exchange loss (gain)(22,352)5,454
Other(1,254)(224)
Changes in operating assets and liabilities:
Accounts receivable, net(130,456)(131,940)
Inventory(29,259)(97,948)
Other current assets(19,939)(59,389)
Other assets(31,839)(20,812)
Accounts payable and other short-term liabilities68,01956,333
Other long-term liabilities10,22914,333
Net cash provided by operating activities387,214131,776
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property, plant and equipment(65,894)(67,774)
Maturities and sales of investments478,436751,677
Purchases of investments(352,371)(727,043)
Proceeds from sale of nonfinancial assets10,000—
Purchase of intangible assets(11,225)(3,141)
Other1,141—
Net cash provided by (used in) investing activities60,087(46,281)
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from exercises of awards under equity incentive plans41,41554,548
Taxes paid related to net share settlement of equity awards(72,651)(72,399)
Payments of contingent consideration—(9,475)
Repayments of convertible debt(494,987)—
Other(3,083)(2,241)
Net cash used in financing activities(529,306)(29,567)
Effect of exchange rate changes on cash2,3264,955
NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS(79,679)60,883
Cash and cash equivalents:
Beginning of period$755,127$724,531
End of period$675,448$785,414
SUPPLEMENTAL CASH FLOW DISCLOSURES:
Cash paid for interest$6,654$6,640
Cash paid for income taxes$36,088$51,687
SUPPLEMENTAL CASH FLOW DISCLOSURES FOR NON-CASH INVESTING AND FINANCING ACTIVITIES:
Increase in accounts payable and accrued liabilities related to fixed assets$8,457$7,977
Increase (decrease) in accounts payable and accrued liabilities related to intangible assets$(9,059)$6,449

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(1)BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES

Nature of Operations

BioMarin Pharmaceutical Inc. (the Company or BioMarin) is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin pursues treatments that offer new possibilities for patients and families around the world navigating rare or difficult to treat genetic conditions.

Basis of Presentation

These Condensed Consolidated Financial Statements have been prepared pursuant to 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 management 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, 2023 included in the Company’s Annual Report on Form 10-K. The Condensed Consolidated Financial Statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions have been eliminated. The results of operations for the three and nine months ended September 30, 2024 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2024 or any other period.

Change in Presentation

Effective January 1, 2024, the Company changed its presentation for foreign currency transaction gains and losses resulting from remeasurement and idle plant costs within its Condensed Consolidated Statements of Comprehensive Income. Effective with this change in presentation, foreign currency transaction gains and losses resulting from remeasurement are presented in Other Income (Expense), Net and idle plant costs are presented in Cost of Sales. Prior to this change in presentation, both foreign currency transaction gains and losses resulting from remeasurement and idle plant costs were presented in Selling, General and Administrative (SG&A) expense. The Company believes that this change in presentation is preferable because the revised presentation is more consistent with how management measures the Company’s operating performance.

Prior period amounts were revised to conform to current period presentation. The following table reflects the impacts of the change in presentation for the prior periods presented. The change in presentation had no impact to Net Income, Total Stockholders’ Equity or earnings per share for the three and nine months ended September 30, 2023.

Three Months ended September 30, 2023Nine Months Ended September 30, 2023
Previously ReportedAdjustmentsAs Reported (after adjustments)Previously ReportedAdjustmentsAs Reported (after adjustments)
Cost of sales$124,745$3,296$128,041$379,376$14,756$394,132
SG&A$223,928$(8,160)$215,768$662,267$(29,373)$632,894
Total operating expenses$555,668$(4,864)$550,804$1,629,141$(14,617)$1,614,524
Other income (expense), net$4,047$(4,864)$(817)$(3,700)$(14,617)$(18,317)

Use of Estimates

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.

Management performed an evaluation of the Company’s activities through the date of filing of this Quarterly Report on Form 10-Q to determine if there were any subsequent events that occurred subsequent to the balance sheet date and prior to filing this Quarterly Report on Form 10-Q, that would require recognition or disclosure in the Condensed Consolidated Financial Statements.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

Significant Accounting Policies

There have been no material changes to the Company’s significant accounting policies during the nine months ended September 30, 2024, as compared to the significant accounting policies disclosed in Note 1 –Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023.

Recent Accounting Pronouncements

There have been no new accounting pronouncements adopted by the Company or new accounting pronouncements issued by the Financial Accounting Standards Board (FASB) during the nine months ended September 30, 2024, as compared to the recent accounting pronouncements described in Note 1 to the Company’s Consolidated Financial Statements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, that the Company believes are of significance or potential significance to the Company. The following paragraphs discuss new accounting pronouncements issued by the FASB, but not yet adopted by the Company.

In November 2023, the FASB issued Accounting Standards Update (ASU) 2023-07, Segment Reporting Topic 280, Improvements to Reportable Segment Disclosures, to improve reportable segment disclosure requirements through enhanced disclosures about significant segment expenses. ASU 2023-07 expands public entities’ segment disclosures by requiring disclosure of significant segment expenses that are regularly provided to the chief operating decision maker and included within each reported measure of segment profit or loss, an amount and description of its composition for other segment items and interim disclosures of a reportable segment’s profit or loss and assets. The effective date for the update is for fiscal years beginning after December 15, 2023 and interim periods within fiscal years beginning after December 15, 2024 and should be applied on a retrospective basis to all periods presented. The Company is currently evaluating the effect of adopting the update on its related disclosures.

In December 2023, the FASB issued ASU 2023-09, Income Taxes Topic 740, Improvements to Income Tax Disclosures. The guidance requires disclosure of disaggregated information about the Company’s effective tax rate reconciliation as well as information on income taxes paid. The disclosure requirements will be applied on a prospective basis, with the option to apply it retrospectively. The effective date for the update is for fiscal years beginning after December 15, 2024. The Company is currently evaluating the effect of the update on its related disclosures.

(2)FINANCIAL INSTRUMENTS

All marketable securities were classified as available-for-sale as of September 30, 2024 and December 31, 2023.

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, 2024
Amortized CostGrossUnrealizedGainsGrossUnrealizedLossesAggregate Fair ValueCash and Cash EquivalentsShort-termMarketableSecurities (1)Long-termMarketableSecurities (2)
Level 1:
Cash$381,781$—$—$381,781$381,781$—$—
Level 2:
Money market instruments288,687——288,687288,687——
Corporate debt securities551,5456,154(114)557,585—178,968378,617
U.S. government agency securities172,6061,372(49)173,9294,98075,13493,815
Asset-backed securities89,866588(7)90,447—89489,553
Subtotal1,102,7048,114(170)1,110,648293,667254,996561,985
Total$1,484,485$8,114$(170)$1,492,429$675,448$254,996$561,985
December 31, 2023
Amortized CostGrossUnrealizedGainsGrossUnrealizedLossesAggregate Fair ValueCash and Cash EquivalentsShort-termMarketableSecurities (1)Long-termMarketableSecurities (2)
Level 1:
Cash$229,676$—$—$229,676$229,676$—$—
Level 2:
Money market instruments499,483——499,483499,483——
Corporate debt securities587,8963,476(1,996)589,376—193,251396,125
U.S. government agency securities251,952556(1,140)251,36819,976111,343120,049
Commercial paper20,0765—20,0815,99214,089—
Asset-backed securities94,744351(134)94,961——94,961
Subtotal1,454,1514,388(3,270)1,455,269525,451318,683611,135
Total$1,683,827$4,388$(3,270)$1,684,945$755,127$318,683$611,135

(1) The Company’s short-term marketable securities mature inone year or less.

(2) The Company’s long-term marketable securities mature betweenone andfive years.

As of September 30, 2024, 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 certain investments in non-marketable equity securities, measured using unobservable valuation inputs and remeasured on a nonrecurring basis, which are collectively considered strategic investments. As of September 30, 2024 and December 31, 2023, the fair value of the Company’s strategic investments was $6.8 million and $11.3 million, respectively. These investments were recorded to Other Assets in the Company’s Condensed Consolidated Balance Sheets. In the second quarter of 2024, based on new developments, the Company became aware of factors that indicated a $4.5 million decline in the fair value of one of its strategic investments. In the first quarter of 2023, the Company concluded that factors existed indicating it would no longer realize a $12.6 million equity investment in its non-marketable securities. The losses on the Company’s non-marketable equity investments were recorded to Other Income (Expense), Net on the Company’s Condensed Consolidated Statements of Comprehensive Income for the respective periods. See Note 1 -Business Overview and Significant Accounting Policies included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 for additional information related to the Company’s non-marketable securities policy.

(3)SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION

Supplemental Balance Sheet Information

Inventory consisted of the following:

September 30,2024December 31,2023
Raw materials$144,484$155,704
Work-in-process499,069571,107
Finished goods535,786380,372
Total inventory$1,179,339$1,107,183

NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

Property, Plant and Equipment, Net consisted of the following:

September 30,2024December 31,2023
Property, plant and equipment, gross$1,914,616$1,933,222
Accumulated depreciation(869,208)(867,089)
Total property, plant and equipment, net$1,045,408$1,066,133

Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2024 was $10.9 million and $35.0 million, respectively. Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2023 was $9.9 million and $29.6 million, respectively. See Note 10 –Restructuring for impairments relating to the Company’s organizational redesign efforts.

Intangible Assets, Net consisted of the following:

September 30,2024December 31,2023
Finite-lived intangible assets$714,015$710,011
Accumulated amortization(453,095)(415,310)
Net carrying value$260,920$294,701

In the first quarter of 2024, the Company received $10.0 million due to the achievement of a regulatory approval milestone by a third party related to previously sold intangible assets, which the Company recorded as a Gain on Sale of Nonfinancial Assets in the Condensed Consolidated Statements of Comprehensive Income.

Accounts Payable and Accrued Liabilities consisted of the following:

September 30,2024December 31,2023
Accounts payable and accrued operating expenses$312,321$315,509
Accrued compensation expense200,149201,067
Accrued rebates payable141,85696,179
Accrued income taxes21,0402,651
Foreign currency exchange forward contracts14,30133,853
Lease liability8,0608,779
Accrued royalties payable7,49114,299
Deferred revenue2,3464,620
Other8,0946,190
Total accounts payable and accrued liabilities$715,658$683,147

(4)FAIR VALUE MEASUREMENTS

The Company measures certain financial assets and liabilities at fair value in accordance with the policy described in Note 1– Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023.

Other than the Company’s fixed-rate convertible debt disclosed in Note 6 –Debt, there wereno financial assets or liabilities that were remeasured using quoted prices in active markets for identical assets (Level 1) as of September 30, 2024 or

December 31, 2023. The Company hadno financial assets or liabilities that are remeasured on a recurring basis using unobservable inputs that reflect estimates and assumptions (Level 3) as of September 30, 2024 or December 31, 2023.

Level 2 assets and liabilities that are remeasured using significant observable inputs consisted of the following:

September 30, 2024December 31, 2023
Assets:
Other current assets:
NQDC Plan assets$3,113$2,026
Other assets:
NQDC Plan assets35,08528,119
Restricted investments (1)6392,393
Total other assets35,72430,512
Total assets$38,837$32,538
Liabilities:
Current liabilities:
NQDC Plan liability$3,113$2,026
Other long-term liabilities:
NQDC Plan liability35,08528,119
Total liabilities$38,198$30,145

(1) The restricted investments as of September 30, 2024 and December 31, 2023 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, 2024.

(5)DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES

The Company uses foreign currency exchange forward contracts (forward contracts) to protect against the impact of changes in the value of forecasted foreign currency cash flows resulting from revenues and operating expenses denominated in currencies other than the U.S. Dollar (USD), primarily the Euro. Certain of these forward contracts are designated as cash flow hedges and have maturities of up to 24 months. The Company also enters into forward contracts to manage foreign exchange risk related to asset or liability positions denominated in currencies other than USD. Such forward contracts are considered to be economic hedges, are not designated as hedging instruments and have maturities of up tothree months. The Company does not use derivative instruments for speculative trading 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 outstanding as of the periods presented.

Forward ContractsSeptember 30,2024December 31,2023
Derivatives designated as hedging instruments:
Sell$1,343,097$1,249,662
Purchase$272,070$198,408
Derivatives not designated as hedging instruments:
Sell$320,017$350,269
Purchase$46,656$90,102

The fair value carrying amounts of the Company’s derivatives, which are classified as Level 2 within the fair value hierarchy, were as follows:

Balance Sheet LocationSeptember 30,2024December 31,2023
Derivatives designated as hedging instruments:
Asset Derivatives
Other current assets$12,630$6,663
Other assets4,4441,855
Subtotal$17,074$8,518
Liability Derivatives
Accounts payable and accrued liabilities$13,147$30,005
Other long-term liabilities5,3198,171
Subtotal$18,466$38,176
Derivatives not designated as hedging instruments:
Asset Derivatives
Other current assets$2,248$547
Liability Derivatives
Accounts payable and accrued liabilities$1,154$3,848
Total Derivatives Assets$19,322$9,065
Total Derivatives Liabilities$19,620$42,024

For additional discussion of fair value measurements, see Note 1 –Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023.

The following tables summarize the impact of gains and losses from the Company's derivatives on its Condensed Consolidated Statements of Comprehensive Income for the periods presented.

Three Months Ended September 30,Nine Months Ended September 30,
2024202320242023
Derivatives Designated as Cash Flow Hedging InstrumentsCash Flow Hedging Gains (Losses) Reclassified into EarningsCash Flow Hedging Gains (Losses) Reclassified into EarningsCash Flow Hedging Gains (Losses) Reclassified into EarningsCash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues$3,365$(4,636)$5,617$(489)
Operating expenses$172$908$486$426
Derivatives Not Designated as Hedging InstrumentsGains (Losses) Recognized in EarningsGains (Losses) Recognized in EarningsGains (Losses) Recognized in EarningsGains (Losses) Recognized in Earnings
Operating expenses$(12,094)$6,559$9,447$3,165

As of September 30, 2024, the Company expects to reclassify unrealized losses of $0.6 million from Accumulated Other Comprehensive Income (Loss) (AOCI) to earnings as the forecasted revenues and operating expense transactions occur over the next twelve months. For additional discussion of balances in AOCI see Note 7 –Accumulated Other Comprehensive Income.

(6)DEBT

Convertible Notes

As of September 30, 2024, the Company had outstanding fixed-rate convertible notes for an undiscounted aggregate principal amount of $600.0 million. The convertible notes detailed below 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 30,2024December 31,2023
0.599% senior subordinated convertible notes due in August 2024 (the 2024 Notes)$—$495,000
Unamortized discount net of deferred offering costs—(1,123)
2024 Notes, net (1)—493,877
1.25% senior subordinated convertible notes due in May 2027 (the 2027 Notes)600,000600,000
Unamortized discount net of deferred offering costs(5,373)(6,905)
2027 Notes, net594,627593,095
Total convertible debt, net$594,627$1,086,972
Fair value of fixed-rate convertible debt (2):
2024 Notes$—$488,288
2027 Notes571,998619,260
Total fair value of fixed-rate convertible debt$571,998$1,107,548

(1) The Company’s convertible notes due in 2024 matured on August 1, 2024. Substantially all holders of the 2024 Notes were repaid with cash, totaling approximately $495.0 million. No gain or loss was incurred upon the extinguishment.

(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 1 –Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023.

Interest expense on the Company’s convertible debt consisted of the following:

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
Coupon interest expense$2,456$2,616$7,689$7,849
Accretion of discount on convertible notes6068412,2902,519
Amortization of debt issuance costs67148364445
Total interest expense on convertible debt$3,129$3,605$10,343$10,813

See Note 10 -Debt to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 for additional information related to the Company’s convertible debt.

Revolving Credit Facility

In August 2024, the Company entered into an unsecured revolving credit facility providing for $600.0 million in revolving loan commitments. The credit facility is intended to finance ongoing working capital needs and for other general corporate purposes. The credit facility contains financial covenants including a maximum total net leverage ratio and a minimum interest coverage ratio. The credit facility matures in August 2029. As of September 30, 2024, there wereno amounts outstanding under the credit facility and the Company was in compliance with all covenants.

(7)ACCUMULATED OTHER COMPREHENSIVE INCOME

The following tables summarize changes in the accumulated balances for each component of AOCI, including current-period other comprehensive income and reclassifications out of AOCI, for the periods presented.

Three Months Ended September 30, 2024
Unrealized Gains(Losses) on CashFlow HedgesUnrealized Gains(Losses) onAvailable-for-SaleDebt SecuritiesTotal
AOCI balance as of June 30, 2024$28,187$(1,388)$26,799
Other comprehensive income (loss) before reclassifications(26,099)9,752(16,347)
Less: gain (loss) reclassified from AOCI3,537—3,537
Tax effect—(2,265)(2,265)
Net current-period other comprehensive income (loss)(29,636)7,487(22,149)
AOCI balance as of September 30, 2024$(1,449)$6,099$4,650
Three Months Ended September 30, 2023
Unrealized Gains(Losses) on CashFlow HedgesUnrealized Gains(Losses) onAvailable-for-SaleDebt SecuritiesTotal
AOCI balance as of June 30, 2023$(10,035)$(8,582)$(18,617)
Other comprehensive income (loss) before reclassifications19,8921,63421,526
Less: gain (loss) reclassified from AOCI(3,728)—(3,728)
Tax effect—(380)(380)
Net current-period other comprehensive income (loss)23,6201,25424,874
AOCI balance as of September 30, 2023$13,585$(7,328)$6,257
Nine Months Ended September 30, 2024
Unrealized Gains(Losses) on CashFlow HedgesUnrealized Gains(Losses) onAvailable-for-SaleDebt SecuritiesTotal
AOCI balance as of December 31, 2023$(29,658)$870$(28,788)
Other comprehensive income (loss) before reclassifications34,3126,82641,138
Less: gain (loss) reclassified from AOCI6,103—6,103
Tax effect—(1,597)(1,597)
Net current-period other comprehensive income (loss)28,2095,22933,438
AOCI balance as of September 30, 2024$(1,449)$6,099$4,650
Nine Months Ended September 30, 2023
Unrealized Gains(Losses) on CashFlow HedgesUnrealized Gains(Losses) onAvailable-for-SaleDebt SecuritiesTotal
AOCI balance as of December 31, 2022$8,226$(12,093)$(3,867)
Other comprehensive income (loss) before reclassifications5,2966,22011,516
Less: gain (loss) reclassified from AOCI(63)—(63)
Tax effect—(1,455)(1,455)
Net current-period other comprehensive income (loss)5,3594,76510,124
AOCI balance as of September 30, 2023$13,585$(7,328)$6,257

For additional discussion of reclassifications from AOCI see Note 5 – Derivative Instruments and Hedging Strategies.

(8)REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION

The Company operates inone business segment, which focuses on the development and commercialization of innovative therapies for patients with serious and life-threatening rare diseases and medical conditions.

The following table presents Total Revenues and disaggregates Net Product Revenues by product.

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
VOXZOGO$189,785$122,973$526,597$324,146
VIMIZIM178,158158,869548,694525,453
NAGLAZYME131,906108,916369,584322,040
PALYNZIQ90,63478,885254,634216,105
ALDURAZYME71,02913,812144,84988,533
BRINEURA37,00541,042121,361118,244
KUVAN28,11542,94792,576144,047
ROCTAVIAN7,23582215,516822
Total net product revenues733,867568,2662,073,8111,739,390
Royalty and other revenues11,87313,06332,79133,629
Total revenues$745,740$581,329$2,106,602$1,773,019

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. The table below disaggregates total Net Product Revenues by geographic region, which is based on patient location for the Company's commercial products sold directly by the Company, except for ALDURAZYME, which is marketed and sold exclusively by Sanofi worldwide.

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
United States$235,515$194,745$655,963$553,184
Europe191,017168,784607,755512,910
Latin America111,90881,524274,920224,716
Rest of world124,398109,401390,324360,047
Total net product revenues marketed by the Company$662,838$554,454$1,928,962$1,650,857
ALDURAZYME net product revenues marketed by Sanofi71,02913,812144,84988,533
Total net product revenues$733,867$568,266$2,073,811$1,739,390

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.

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
Customer A12%13%11%12%
Customer B11151315
Customer C10101010
Total33%38%34%37%

On a consolidated basis, one customer accounted for 18% of the Company’s September 30, 2024 accounts receivable balance compared to December 31, 2023, when two customers accounted for 15% and 12% of the accounts receivable balance, respectively. As of September 30, 2024, and December 31, 2023, the accounts receivable balance for Sanofi included $94.2 million and $63.4 million, respectively, of unbilled accounts receivable, which becomes payable to the Company when the product is sold through by Sanofi. 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 Company is mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, and supply chain disruptions, could affect the Company’s ability to achieve its goals. In addition, 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 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.

(9)STOCK-BASED COMPENSATION

The Company has stockholder-approved equity incentive plans that provide for the granting of restricted stock units (RSUs) and stock options as well as other forms of equity compensation to its employees, officers and non-employee directors. The Company also has an Employee Stock Purchase Plan (ESPP). Compensation expense included in the Company’s Condensed Consolidated Statements of Comprehensive Income for all stock-based compensation arrangements was as follows:

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
Cost of sales$5,092$4,042$12,110$13,094
Research and development11,49614,49045,10849,373
Selling, general and administrative26,90129,85592,43489,777
Total stock-based compensation expense$43,489$48,387$149,652$152,244

(10)RESTRUCTURING

During 2024, in connection with the discontinuation of certain research and development programs and organizational redesign efforts centered around the Company’s strategic priorities, including the acceleration and maximization of VOXZOGO, establishing the ROCTAVIAN opportunity, and focusing R&D activities on the assets that management believes will be the most productive and accelerate earnings per share through expanding margins, the Company committed to plans in the second and third quarters of 2024, to reduce its global workforce by approximately 170 and 225 employees, respectively. These workforce reductions are expected to be substantially completed by the end of 2024.

The restructuring plan includes severance and employee-related costs, asset impairments, and other costs. The asset impairment charges were for abandoned assets-in-progress and a Right-of-Use asset (ROU Asset) related to leased office space the Company decided to exit and sub-lease. The Company utilized the discounted cash flow approach to determine the fair value of the ROU Asset. The ROU Asset impairment is the difference between the existing lease terms and rates and the expected sub-lease terms and rates available in the market. The Other category includes restructuring-related costs, which are expensed as incurred, as well as other obligations related to the leased office space that will be satisfied over the remainder of the lease term.

The restructuring charges and adjustments were included in Selling, General, and Administrative in the Condensed Consolidated Statements of Comprehensive Income. Restructuring expenses consisted of the following:

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
20242024
Severance and one-time employee benefits$28,080$53,355
Asset Impairments6,77716,448
Other8,66816,187
$43,525$85,990

The following unpaid balance as of September 30, 2024 was recorded to Accounts Payable and Accrued Liabilities on the Condensed Consolidated Balance Sheet:

Severance and related costsOtherTotal
Balance as of December 31, 2023$—$—$—
Charges and Adjustments53,35516,187$69,542
Payments(25,535)(5,237)$(30,772)
Balance as of September 30, 2024$27,820$10,950$38,770

(11)EARNINGS 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 ESPP, unvested RSUs 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 thousands):

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
Numerator:
Net Income, basic$106,080$40,378$301,916$147,270
Add: Interest expense, net of tax, on the Company's convertible debt1,832—5,4952,810
Net Income, diluted$107,912$40,378$307,411$150,080
Denominator:
Weighted-average common shares outstanding, basic190,429188,219189,806187,617
Effect of dilutive securities:
Common stock issuable under the Company's equity incentive plans2,3532,9542,5123,455
Common stock issuable under the Company’s convertible debt(1)4,365—4,3653,970
Weighted-average common shares outstanding, diluted197,147191,173196,683195,042
Earnings per common share, basic$0.56$0.21$1.59$0.78
Earnings per common share, diluted$0.55$0.21$1.56$0.77

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 EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
Common stock issuable under the Company's equity incentive plans9,4148,5479,2558,045
Common stock issuable under the Company’s convertible debt (1)—8,335—4,365
Total number of potentially issuable shares9,41416,8829,25512,410

(1) If converted, the Company would issue 4.0 million shares under the 2024 Notes and 4.4 million shares under the 2027 Notes. The Company’s 2024 Notes matured in August 2024 and substantially all holders were repaid in cash. For additional discussion of the Company’s convertible debt, see Note 6 -Debt.

(12)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 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 based on existing information. The Company accrues for the best estimate of a loss within a range; however, if no estimate in the range is better than any other, then the minimum amount in the range is accrued. Liabilities are evaluated and refined each reporting period as additional information is known. Any receivables for insurance recoveries for these liability claims are recorded as assets when it is probable that a recovery will be realized.

As previously disclosed, the Company received a subpoena from the U.S. Department of Justice (DOJ) requesting that the Company produce certain documents regarding sponsored testing programs relating to VIMIZIM and NAGLAZYME. The Company has produced the requested documents in response to the subpoena and is cooperating fully. The Company is unable to make any assurances regarding the outcome of the investigation by the DOJ, or the impact, if any, that such investigation may have on the Company’s business, Condensed Consolidated Balance Sheets, Condensed Consolidated Statements of Comprehensive Income or Condensed Consolidated Statements of Cash Flows.

Contingent Payments

As of September 30, 2024, the Company was subject to contingent payments, primarily comprised of development, regulatory and commercial milestones. Those considered reasonably possible totaled $196.1 million, of this amount the Company may pay up to $4.1 million over the next 12 months if certain contingencies are met.

Other Commitments

The Company uses experts and laboratories at universities and other institutions to perform certain R&D activities. These amounts are recorded as R&D expense as services are provided. In the normal course of business, the Company enters into various firm purchase commitments primarily to procure active pharmaceutical ingredients, certain inventory-related items and certain third-party R&D services, production services and facility construction services. The Company also has commitments related to enterprise resource planning (ERP) system implementation costs for which the Company is committed. As of September 30, 2024, such commitments were estimated at $474.1 million, of which $269.8 million is expected to be paid in 2024 as underlying goods and services are received. The Company has also licensed technology from third parties, for which it is required to pay royalties upon future sales, subject to certain annual minimums.

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 risks and uncertainties described in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q. 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).

(In millions, except as otherwise disclosed)

Overview

We are a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. Our San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we pursue treatments that offer new possibilities for patients and families around the world navigating rare or difficult to treat genetic conditions.

A summary of our commercial products, as of September 30, 2024, is provided below:

Commercial ProductsIndication
VOXZOGO (vosoritide)Achondroplasia
VIMIZIM (elosulfase alpha)Mucopolysaccharidosis (MPS) IVA
NAGLAZYME (galsulfase)MPS VI
PALYNZIQ (pegvaliase-pqpz)Phenylketonuria (PKU)
ALDURAZYME (laronidase)MPS I
BRINEURA (cerliponase alfa)Neuronal ceroid lipofuscinosis type 2 (CLN2)
KUVAN (sapropterin dihydrochloride)PKU
ROCTAVIAN (valoctocogene roxaparvovec)Severe Hemophilia A

Financial Highlights

Key components of our results of operations include the following:

Three Months EndedSeptember 30,Nine Months EndedSeptember 30,
2024202320242023
Total revenues$745.7$581.3$2,106.6$1,773.0
Cost of sales$188.5$128.0$444.1$394.1
Research and development (R&D)$184.9$191.3$573.7$540.5
Selling, general and administrative (SG&A)$253.5$215.8$742.4$632.9
Net income$106.1$40.4$301.9$147.3

See “Results of Operations” below for discussion of our results for the periods presented.

Uncertainty Relating to Macroeconomic Environment

Conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, and supply chain disruptions, could impact our global revenue sources and our overall business operations. The extent and duration of such effects remain 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, “Our business is affected by macroeconomic conditions.” described in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q.

Recent Developments

We continued to grow our commercial business and advance our product candidate pipeline during 2024. We believe that the combination of our internal research programs and partnerships will allow us to continue to develop and commercialize innovative therapies for patients with serious and life-threatening rare diseases and medical conditions.

In 2024, we focused on value creation through working to accelerate growth, optimize efficiencies and drive operational excellence, including progress in executing on the key strategic priorities of accelerating and maximizing VOXZOGO, establishing the ROCTAVIAN opportunity, and focusing R&D activities on the assets management believes will be the most productive and

(In millions, except as otherwise disclosed)

accelerate earnings per share through expanding margins. We also completed a strategic portfolio assessment of R&D programs to determine which we believe have the strongest combination of scientific merit, opportunity for commercial success and potential value creation for stockholders. We determined that BMN 333, a long-acting C-type natriuretic peptide for multiple growth disorders, BMN 349, a potential best-in-class, oral therapeutic for liver disease associated with Alpha-1 Antitrypsin Deficiency, and BMN 351, our next generation oligonucleotide for Duchenne Muscular Dystrophy, all met criteria for advancement. Based on such strategic portfolio assessment, certain programs have also been discontinued. In connection with the strategic portfolio assessment and organizational redesign efforts, we have committed to plans in the second and third quarters of 2024 to reduce our global workforce by an aggregate of approximately 395 employees. Further, we initiated the execution of our financial and operational strategy that improved operating results and is expected to continue to improve our results of operations going forward. In August 2024, we announced our ROCTAVIAN strategy, which includes focusing on the U.S., Germany, and Italy, where ROCTAVIAN is approved and reimbursed, and reducing additional investments in development and manufacturing, that we anticipate will enable ROCTAVIAN to contribute to our long-term profitability. See the risk factor, “Our success depends on our ability to manage our growth.” described in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q.

Change in Presentation

On January 1, 2024, we changed our presentation of foreign currency transaction gains and losses resulting from remeasurement and idle plant costs within our Condensed Consolidated Statements of Comprehensive Income. See Note 1 to our accompanying Condensed Consolidated Financial Statements for additional details.

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