Full Press Release Details
• Full-year 2023 Total Revenues and Earnings Per Share Financial Guidance Narrowed; VOXZOGO ® and ROCTAVIAN ® Net Product Revenue Guidance Adjusted for Full-year 2023
• VOXZOGO Now Approved for Children without Age Restrictions in the United States and for Children Aged 4 Months and Older in Europe
• ROCTAVIAN Reimbursement Network On-track to Support Global Patient Access and Treatment in 2024 and Beyond; Final German and Italian Prices Expected in 2023; Over 100 Treatment Eligible Companion Diagnostic Tests Completed Globally
• For Full-year 2024, BioMarin Expects Revenues to Approach $3 Billion
| Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) | |||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||
| 2023 | 2022 | % Change | 2023 | 2022 | % Change | ||||||
| Total Revenues | $ 581.3 | $ 505.3 | 15 % | $ 1,773.0 | $ 1,558.5 | 14 % | |||||
| Total Enzyme Product Revenues (1) | $ 401.5 | $ 388.0 | 3 % | $ 1,270.4 | $ 1,240.2 | 2 % | |||||
| VIMIZIM ® Net Product Revenues | $ 158.9 | $ 155.5 | 2 % | $ 525.6 | $ 511.7 | 3 % | |||||
| VOXZOGO Net Product Revenues | $ 123.1 | $ 48.3 | 155 % | $ 324.2 | $ 102.3 | 217 % | |||||
| NAGLAZYME ® Net Product Revenues | $ 108.9 | $ 99.5 | 9 % | $ 322.0 | $ 343.3 | (6) % | |||||
| PALYNZIQ ® Net Product Revenues | $ 78.9 | $ 66.2 | 19 % | $ 216.1 | $ 182.7 | 18 % | |||||
| KUVAN ® Net Product Revenues | $ 42.9 | $ 57.0 | (25) % | $ 144.0 | $ 174.0 | (17) % | |||||
| BRINEURA ® Net Product Revenues | $ 41.0 | $ 37.8 | 8 % | $ 118.2 | $ 111.7 | 6 % | |||||
| ALDURAZYME ® Net Product Revenues | $ 13.8 | $ 29.0 | (52) % | $ 88.5 | $ 90.8 | (3) % | |||||
| ROCTAVIAN Net Product Revenues | $ 0.8 | $ — | nm | $ 0.8 | $ — | nm | |||||
| GAAP Net Income (Loss) (2) | $ 40.4 | $ (6.7) | $ 147.3 | $ 141.8 | |||||||
| Non-GAAP Income (3) | $ 89.5 | $ 52.0 | $ 310.5 | $ 227.6 | |||||||
| GAAP Diluted Earnings (Loss) per Share (EPS) | $ 0.21 | $ (0.04) | $ 0.77 | $ 0.75 | |||||||
| Non-GAAP Diluted EPS (4) | $ 0.46 | $ 0.27 | $ 1.60 | $ 1.20 |
| September 30, 2023 | December 31, 2022 | ||
| Total cash, cash equivalents & investments | $ 1,673.8 | $ 1,625.4 |
| (1) | Enzyme Products include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ, and VIMIZIM. |
| (2) | GAAP Net Income in the nine months ended September 30, 2022 included a $89.0 million gain, net of taxes, related to the sale of the Rare Pediatric Disease Priority Review Voucher (PRV) the company received from the U.S. Food and Drug Administration (FDA) in connection with U.S. approval of VOXZOGO. |
| (3) | Non-GAAP Income is defined by the company as reported GAAP Net Income, excluding amortization expense, stock-based compensation expense, contingent consideration, and, in certain periods, certain other specified items. The company also includes a Non-GAAP adjustment for the estimated income tax impact of reconciling items. Refer to Non-GAAP Information beginning on page 9 of this press release for a complete discussion of the company's Non-GAAP financial information and reconciliations to the comparable information reported under U.S. GAAP. |
| (4) | Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP diluted shares outstanding. |
| nm | Not meaningful |
SAN RAFAEL, Calif. , Nov. 1, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) today announced financial results for the nine months and third quarter ended September 30, 2023.
"Results in the quarter drove double-digit revenue growth year-over-year and support BioMarin's full-year 2023 revenue and profitability objectives, set at the beginning of the year," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.
"We were very pleased to have recently received FDA approval for VOXZOGO for children under 5 years of age with achondroplasia, making it available in the U.S. for children of all ages with open growth plates. We also recently received approval in Europe to expand VOXZOGO treatment to children aged 4 months and older. These important age expansions will ensure that the youngest children treated with VOXZOGO have the opportunity to experience greater clinical benefit due to a longer potential treatment window. In parallel with exceptional commercial and regulatory execution of VOXZOGO during the quarter, we concluded key reimbursement steps to facilitate ROCTAVIAN treatment in Europe and the U.S. All together, these developments drive our expectation that total BioMarin revenues will approach $3 billion in 2024," said Mr. Bienaimé.
"In Germany, we have tentative agreement with the German Health Insurance Fund on the final ROCTAVIAN price, which we expect to be formalized in the coming weeks. With the final German price expected to be published by year-end, and 60 people in Germany eligible for next steps based on CDx testing results for AAV5 antibodies, we are encouraged by progress made in the quarter. We are also pleased to share that price negotiations with the Italian Medicines Agency are going well and that we expect a final price by year-end," Mr. Bienaimé added, "In the U.S. since gaining FDA approval of ROCTAVIAN on June 29, 2023 , the commercial team has been building the reimbursement network to facilitate patient access, setting the stage for meaningful uptake of ROCTAVIAN in 2024."
Third Quarter Financial Highlights:
Global Commercial Launches of ROCTAVIAN and VOXZOGO
VOXZOGO and ROCTAVIAN Market Expansion Opportunities
Earlier-stage Development Portfolio On-track; Seven Product Candidates Advancing
| 2023 Full-Year Financial Guidance (in millions, except % and EPS amounts) | ||||||||||||
| Item | Provided July 31, 2023 | Updated November 1, 2023 | ||||||||||
| Total Revenues | $2,375 | to | $2,500 | $2,390 | to | $2,470 | ||||||
| Enzyme Product Revenues (1) | $1,700 | to | $1,850 | $1,700 | to | $1,775 | ||||||
| ROCTAVIAN Revenues | $50 | to | $150 | less than $10M | ||||||||
| VOXZOGO Revenues | $400 | to | $440 | $435 | to | $455 | ||||||
| Unchanged | ||||||||||||
| Gross Profit % | 77.5 % | to | 79 % | 77.8 % | to | 79 % | ||||||
| R&D % of Revenue | 30 % | to | 32 % | Unchanged | ||||||||
| SG&A % of Revenue | 35.5 % | to | 37.5 % | 36 % | to | 38 % | ||||||
| GAAP Net Income | $165 | to | $215 | $170 | to | $210 | ||||||
| GAAP Diluted EPS | $0.83 | to | $1.08 | $0.85 | to | $1.05 | ||||||
| Non-GAAP Income | $370 | to | $420 | $380 | to | $410 | ||||||
| Non-GAAP Diluted EPS | $1.85 | to | $2.10 | $1.90 | to | $2.05 |
BioMarin will host a conference call and webcast to discuss third quarter 2023 financial results and full-year financial guidance today, Wednesday, November 1, 2023, at 4:30 p.m. ET . This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.
| U.S./Canada Dial-in Number: 888-330-3073 | Replay Dial-in Number: 800-770-2030 |
| International Dial-in Number: 646-960-0683 | Replay International Dial-in Number: 647-362-9199 |
| No Conference ID: 1816377 | Conference ID: 1816377 |
Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's robust research and development capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit www.biomarin.com .
Forward-Looking Statements
BioMarin ® , BRINEURA ® , KUVAN ® , NAGLAZYME ® , PALYNZIQ ® , VIMIZIM ® and VOXZOGO ® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ROCTAVIAN ® is a trademark of BioMarin Pharmaceutical Inc., with registration in Europe and pending in the U.S. ALDURAZYME ® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.
| Contact: | ||
| Investors: | Media: | |
| Traci McCarty | Marni Kottle | |
| BioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. | |
| (415) 455-7558 | (650) 374-2803 |
| BIOMARIN PHARMACEUTICAL INC. | |||
| CONDENSED CONSOLIDATED BALANCE SHEETS | |||
| September 30, 2023 and December 31, 2022 | |||
| (In thousands of U.S. dollars, except per share amounts) | |||
| September 30, 2023 | December 31, 2022 ⁽¹⁾ | ||
| ASSETS | (unaudited) | ||
| Current assets: | |||
| Cash and cash equivalents | $ 785,414 | $ 724,531 | |
| Short-term investments | 340,431 | 567,006 | |
| Accounts receivable, net | 572,498 | 461,316 | |
| Inventory | 1,032,159 | 894,083 | |
| Other current assets | 224,806 | 104,521 | |
| Total current assets | 2,955,308 | 2,751,457 | |
| Noncurrent assets: | |||
| Long-term investments | 548,002 | 333,835 | |
| Property, plant and equipment, net | 1,067,156 | 1,073,366 | |
| Intangible assets, net | 302,476 | 338,569 | |
| Goodwill | 196,199 | 196,199 | |
| Deferred tax assets | 1,523,953 | 1,505,412 | |
| Other assets | 165,069 | 176,236 | |
| Total assets | $ 6,758,163 | $ 6,375,074 | |
| LIABILITIES AND STOCKHOLDERS' EQUITY | |||
| Current liabilities: | |||
| Accounts payable and accrued liabilities | $ 669,825 | $ 572,959 | |
| Short-term convertible debt, net | 493,398 | — | |
| Short-term contingent consideration | — | 15,925 | |
| Total current liabilities | 1,163,223 | 588,884 | |
| Noncurrent liabilities: | |||
| Long-term convertible debt, net | 592,586 | 1,083,019 | |
| Other long-term liabilities | 105,457 | 100,015 | |
| Total liabilities | 1,861,266 | 1,771,918 | |
| Stockholders' equity: | |||
| Common stock, $0.001 par value: 500,000,000 shares authorized; 188,339,482 and 186,250,719 shares issued and outstanding, respectively | 188 | 186 | |
| Additional paid-in capital | 5,542,774 | 5,404,895 | |
| Company common stock held by the Nonqualified Deferred Compensation Plan (the NQDC) | (10,393) | (8,859) | |
| Accumulated other comprehensive loss | 6,257 | (3,867) | |
| Accumulated deficit | (641,929) | (789,199) | |
| Total stockholders' equity | 4,896,897 | 4,603,156 | |
| Total liabilities and stockholders' equity | $ 6,758,163 | $ 6,375,074 |
| BIOMARIN PHARMACEUTICAL INC. | |||||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | |||||||
| Three and Nine Months Ended September 30, 2023 and 2022 | |||||||
| (In thousands of U.S. dollars, except per share amounts) | |||||||
| (unaudited) | |||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||
| 2023 | 2022 | 2023 | 2022 | ||||
| REVENUES: | |||||||
| Net product revenues | $ 568,266 | $ 493,348 | $ 1,739,390 | $ 1,516,533 | |||
| Royalty and other revenues | 13,063 | 11,996 | 33,629 | 41,968 | |||
| Total revenues | 581,329 | 505,344 | 1,773,019 | 1,558,501 | |||
| OPERATING EXPENSES: | |||||||
| Cost of sales | 124,745 | 116,288 | 379,376 | 356,379 | |||
| Research and development | 191,314 | 157,829 | 540,523 | 476,855 | |||
| Selling, general and administrative | 223,928 | 216,816 | 662,267 | 608,270 | |||
| Intangible asset amortization and contingent consideration | 15,681 | 16,828 | 46,975 | 50,935 | |||
| Gain on sale of nonfinancial assets, net | — | — | — | (108,000) | |||
| Total operating expenses | 555,668 | 507,761 | 1,629,141 | 1,384,439 | |||
| INCOME (LOSS) FROM OPERATIONS | 25,661 | (2,417) | 143,878 | 174,062 | |||
| Interest income | 15,740 | 4,999 | 40,295 | 9,324 | |||
| Interest expense | (3,779) | (4,679) | (11,237) | (12,344) | |||
| Other income (expense), net | 4,047 | 193 | (3,700) | (3,908) | |||
| INCOME (LOSS) BEFORE INCOME TAXES | 41,669 | (1,904) | 169,236 | 167,134 | |||
| Provision for income taxes | 1,291 | 4,748 | 21,966 | 25,324 | |||
| NET INCOME (LOSS) | $ 40,378 | $ (6,652) | $ 147,270 | $ 141,810 | |||
| EARNINGS (LOSS) PER SHARE, BASIC | $ 0.21 | $ (0.04) | $ 0.78 | $ 0.77 | |||
| EARNINGS (LOSS) PER SHARE, DILUTED | $ 0.21 | $ (0.04) | $ 0.77 | $ 0.75 | |||
| Weighted average common shares outstanding, basic | 188,219 | 185,597 | 187,617 | 185,009 | |||
| Weighted average common shares outstanding, diluted | 191,173 | 185,597 | 195,042 | 192,252 |
| BIOMARIN PHARMACEUTICAL INC. | |||
| CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS | |||
| Nine Months Ended September 30, 2023 and 2022 | |||
| (In thousands of U.S. dollars) | |||
| (unaudited) | |||
| Nine Months Ended September 30, | |||
| 2023 | 2022 | ||
| CASH FLOWS FROM OPERATING ACTIVITIES: | |||
| Net income | $ 147,270 | $ 141,810 | |
| Adjustments to reconcile net income to net cash used in operating activities: | |||
| Depreciation and amortization | 77,525 | 77,416 | |
| Non-cash interest expense | 3,198 | 3,089 | |
| Amortization of premium (accretion of discount) on investments | (6,781) | 3,741 | |
| Stock-based compensation | 152,244 | 149,574 | |
| Gain on sale of nonfinancial assets, net | — | (108,000) | |
| Loss on equity investment | 12,650 | — | |
| Deferred income taxes | (20,137) | (743) | |
| Unrealized foreign exchange loss (gain) | 5,454 | (16,075) | |
| Non-cash changes in the fair value of contingent consideration | — | 2,243 | |
| Other | (224) | (700) | |
| Changes in operating assets and liabilities: | |||
| Accounts receivable, net | (131,940) | (53,752) | |
| Inventory | (97,948) | (27,419) | |
| Other current assets | (59,389) | (8,558) | |
| Other assets | (20,812) | 12,140 | |
| Accounts payable and other short-term liabilities | 56,333 | (2,398) | |
| Other long-term liabilities | 14,333 | (3,252) | |
| Net cash provided by operating activities | 131,776 | 169,116 | |
| CASH FLOWS FROM INVESTING ACTIVITIES: | |||
| Purchases of property, plant and equipment | (67,774) | (85,271) | |
| Maturities and sales of investments | 751,677 | 477,244 | |
| Purchases of investments | (727,043) | (457,382) | |
| Proceeds from sale of nonfinancial assets | — | 110,000 | |
| Purchase of intangible assets | (3,141) | (9,910) | |
| Net cash provided by (used in) investing activities | (46,281) | 34,681 | |
| CASH FLOWS FROM FINANCING ACTIVITIES: | |||
| Proceeds from exercises of awards under equity incentive plans | 54,548 | 43,866 | |
| Taxes paid related to net share settlement of equity awards | (72,399) | (50,696) | |
| Payments of contingent consideration | (9,475) | (21,054) | |
| Principal repayments of financing leases | (2,241) | (1,635) | |
| Net cash used in financing activities | (29,567) | (29,519) | |
| Effect of exchange rate changes on cash | 4,955 | (39) | |
| NET INCREASE IN CASH AND CASH EQUIVALENTS | 60,883 | 174,239 | |
| Cash and cash equivalents: | |||
| Beginning of period | $ 724,531 | $ 587,276 | |
| End of period | $ 785,414 | $ 761,515 |
Non-GAAP Information
The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization expense, stock-based compensation expense, contingent consideration expense, and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP diluted shares outstanding
Non-GAAP Income and its components are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.
The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:
| Reconciliation of GAAP Reported Net Income to Non-GAAP Income (1) | |||||||
| (In millions of U.S. dollars) | |||||||
| (unaudited) | |||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||
| 2023 | 2022 | 2023 | 2022 | ||||
| GAAP Reported Net Income (Loss) | $ 40.4 | $ (6.7) | $ 147.3 | $ 141.8 | |||
| Adjustments | |||||||
| Stock-based compensation expense - COS | 4.0 | 4.4 | 13.1 | 13.4 | |||
| Stock-based compensation expense - R&D | 14.5 | 17.1 | 49.4 | 47.9 | |||
| Stock-based compensation expense - SG&A | 29.9 | 33.2 | 89.8 | 88.2 | |||
| Amortization of intangible assets | 15.7 | 15.9 | 47.0 | 47.1 | |||
| Contingent consideration | — | 0.9 | — | 3.8 | |||
| Gain on sale of non-financial assets (2) | — | — | — | (108.0) | |||
| Severance and employee termination benefits (3) | (0.4) | 4.8 | (0.5) | 4.8 | |||
| Loss on investment in equity securities (4) | — | — | 12.6 | — | |||
| Income tax effect of adjustments | (14.6) | (17.6) | (48.2) | (11.4) | |||
| Non-GAAP Income | $ 89.5 | $ 52.0 | $ 310.5 | $ 227.6 |
| Reconciliation of Certain GAAP Reported Information to Non-GAAP Information | |||||||
| (in millions, except per share data) | |||||||
| (unaudited) | |||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||
| 2023 | 2022 | 2023 | 2022 | ||||
| GAAP Diluted EPS | $ 0.21 | $ (0.04) | $ 0.77 | $ 0.75 | |||
| Adjustments | |||||||
| Stock-based compensation expense | 0.24 | 0.28 | 0.76 | 0.78 | |||
| Amortization of intangible assets | 0.08 | 0.08 | 0.24 | 0.24 | |||
| Contingent consideration | — | — | — | 0.02 | |||
| Gain on sale of non-financial assets (2) | — | — | — | (0.56) | |||
| Severance and employee termination benefits (3) | — | 0.02 | — | 0.02 | |||
| Loss on investment in equity securities (4) | — | — | 0.06 | — | |||
| Income tax effect of adjustments | (0.07) | (0.09) | (0.23) | (0.05) | |||
| Non-GAAP Diluted EPS | $ 0.46 | $ 0.27 | $ 1.60 | $ 1.20 |
| (1) | Certain amounts may not sum or recalculate due to rounding. |
| (2) | Represents the net gain in the first quarter of 2022 on the sale to a third party of the PRV the company received from the FDA in connection with the U.S. approval of VOXZOGO. |
| (3) | Represents change in estimates to severance and employee termination benefit charges in SG&A related to the company's organizational redesign announced in October 2022. Related to this October 2022 reorganization plan, the company recognized $23.0 million of expense related to severance and employee termination benefits in 2022, the majority of which was recognized in the fourth quarter. |
| (4) | Represents the impairment loss on investment in non-marketable equity securities recorded in Other income (expense), net in the first quarter of 2023. |
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||
| 2023 | 2022 | 2023 | 2022 | ||||
| GAAP Weighted-Average Dilutive Shares Outstanding | 191.2 | 185.6 | 195.0 | 192.3 | |||
| Adjustments | |||||||
| Issuances under equity incentive plans | — | 3.5 | — | — | |||
| Common stock issuable under Company's convertible debt (1) | 8.4 | 4.0 | 4.4 | — | |||
| Non-GAAP Weighted-Average Dilutive Shares Outstanding | 199.6 | 193.1 | 199.4 | 192.3 |
| Guidance for the Year Ended December 31, 2023 (1)(2) | ||||||||||
| Net Income | Diluted Shares | Diluted EPS | ||||||||
| GAAP Net Income and Diluted EPS | $ 170 | to | $ 210 | 200 | $0.85 | to | $1.05 | |||
| Amortization of intangible assets | 60 | 0.30 | ||||||||
| Stock-based compensation expense | 200 | 1.00 | ||||||||
| Severance and employee termination benefits | (0.5) | — | ||||||||
| Loss on investment in equity securities | 12.6 | 0.06 | ||||||||
| Income tax effect of adjustments (3) | (67) | (0.34) | ||||||||
| Non-GAAP Income and Diluted EPS | $ 380 | to | $ 410 | 200 | $1.90 | to | $2.05 |
| (1) | The adjustments/reconciling items included in this table are presented to facilitate the reconciliation of Non-GAAP Income and Non-GAAP Diluted EPS to their closest GAAP financial metrics, GAAP Net Income and GAAP Diluted EPS. The specific amounts included in each reconciling line item above represent approximations of the underlying adjustments from GAAP Net Income to Non-GAAP Income and from GAAP Diluted EPS to Non-GAAP Diluted EPS. Actual 2023 results for each reconciling line item may be different, in some cases materially, than the amounts listed above as a result of uncertainty regarding, and the potential variability of, those items. |
| (2) | Amounts will sum when using midpoint of ranges provided |
| (3) | Income tax adjustments represent the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable statutory income tax rate. |
SOURCE BioMarin Pharmaceutical Inc.