Full Press Release Details
SAN RAFAEL, Calif. , Oct. 25, 2018 /PRNewswire/ --
| Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) | ||||||||||||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||||||||||
| 2018 | 2017 | % Change | 2018 | 2017 | % Change | |||||||||||||||||||
| Total Revenues | $ | 391.7 | $ | 334.1 | 17 | % | $ | 1,138.0 | $ | 955.3 | 19 | % | ||||||||||||
| Vimizim Net Product Revenues | 123.3 | 90.3 | 37 | % | 368.0 | 299.3 | 23 | % | ||||||||||||||||
| Kuvan Net Product Revenues | 113.3 | 105.8 | 7 | % | 321.4 | 300.1 | 7 | % | ||||||||||||||||
| Naglazyme Net Product Revenues | 103.1 | 72.1 | 43 | % | 269.2 | 238.4 | 13 | % | ||||||||||||||||
| Aldurazyme Net Product Revenues | 27.6 | 22.4 | 23 | % | 117.7 | 61.7 | 91 | % | ||||||||||||||||
| Brineura Net Product Revenues | 9.9 | 3.1 | 219 | % | 27.7 | 3.4 | 715 | % | ||||||||||||||||
| Palynziq Net Product Revenues | 4.1 | — | n/a | 4.1 | — | n/a | ||||||||||||||||||
| GAAP Net Loss | $ | (12.6) | $ | (12.5) | $ | (73.6) | $ | (65.7) | ||||||||||||||||
| GAAP Net Loss per Share - Basic and Diluted | $ | (0.07) | $ | (0.07) | $ | (0.42) | $ | (0.38) | ||||||||||||||||
| Non-GAAP Income (1) | $ | 60.7 | $ | 7.8 | $ | 101.8 | $ | 68.7 | ||||||||||||||||
| September 30, | December 31, | |||||||||||||||||||||||
| 2018 | 2017 | |||||||||||||||||||||||
| Cash, cash equivalents and investments | $ | 1,648.3 | $ | 1,781.7 |
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) (BioMarin or the Company) today announced financial results for the third quarter ended September 30, 2018 . The financial results that follow represent a comparison of the third quarter of 2018 to the third quarter of 2017. Total revenues were $391.7 million for the quarter ended September 30, 2018 compared to $334.1 million for the quarter ended September 30, 2017 , an increase of 17%. GAAP Net Loss for the quarter ended September 30, 2018 was $12.6 million , or $0.07 loss per basic and diluted share compared to GAAP Net Loss of $12.5 million , or $0.07 loss per basic and diluted share for the quarter ended September 30, 2017 .
Net product revenues for the third quarter of 2018 were $386.3 million , compared to $298.8 million in the third quarter of 2017. The increase in net product revenues was attributed to the following:
The increase in GAAP Net Loss was primarily due to the following:
Non-GAAP Income for the third quarter of 2018 was $60.7 million , compared to Non-GAAP Income of $7.8 million in the third quarter of 2017. The increase in Non-GAAP income in the third quarter of 2018 is primarily attributed to increased gross profit from revenues, offset by higher R&D and SG&A expenses.
As of September 30, 2018 , BioMarin had cash, cash equivalents and investments totaling approximately $1.6 billion , as compared to $1.8 billion on December 31, 2017 . On October 15, 2018 , our 0.75% senior subordinated convertible notes matured and were settled with a combination of $375.0 million in cash for the full principal amount, issuance of common stock for the conversion value in excess of the principal, and cash in lieu of any fractional shares.
Commenting on third quarter results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "BioMarin's achievements this year give us potential for significant growth in 2019. In the second quarter we received FDA approval of Palynziq, an important new therapy that helps address a significant unmet need in adults with phenylketonuria (PKU) who have been unable to control their blood Phe levels with current treatment options. We also filed for approval of Palynziq in Europe and expect to hear the status of that application in the first half of 2019. The market opportunity with Palynziq is significant and we have been very encouraged by the level of enthusiasm from the PKU community since product launch in July. At the end of the third quarter, 124 U.S. commercial patients were being treated with Palynziq and we are on track to have 250 to 300 U.S. commercial patients by year-end."
"In clinical development, we provided two years of clinical data with the 6e13 vg/kg dose of valoctocogene roxaparvovec gene therapy for severe Hemophilia A from the ongoing Phase 1/2 study in at the World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, Scotland during the second quarter. The updated data demonstrated the elimination of need for prophylaxis and no spontaneous bleeds for two years. In addition, we amended the protocol for the global GENEr8-1 (Phase 3) pivotal study to evaluate superiority compared to the current standard of care. The number of participants has been increased to 130 and we now anticipate completing enrollment during the second quarter of 2019. Another significant development in the year was the publication of draft guidance from the FDA for hemophilia products. We are encouraged by the considerations included in the guidance document, particularly in relation to the potential for an accelerated approval, and believe it may be pertinent to valoctocogene roxaparvovec if our program results are supportive."
Mr. Bienaimé continued, "Our annual Research and Development Day will be held in New York City on November 7, 2018 where we look forward to showcasing our capabilities from pre-clinical work through manufacturing. Specifically, we look forward to providing a 42-month update on vosoritide for the treatment of achondroplasia as well as preclinical data with our next gene therapy product candidate, BMN 307, for the treatment of phenylketonuria. With approximately $1.5 billion in revenues anticipated for the full year 2018, two potentially $1.0 billion -plus late-stage clinical product opportunities, and a growing early-stage pipeline, BioMarin is poised for significant growth."
As presented August 2, 2018 Full-Year Financial Guidance ( in millions, except %)
| Item | 2018 Guidance | |
| Total Revenues | $1,470 to $1,530 | |
| Kuvan Net Product Revenues | $440 to $480 | |
| Naglazyme Net Product Revenues | $325 to $355 | |
| Vimizim Net Product Revenues | $460 to $500 | |
| Brineura Net Product Revenues | $35 to $55 | |
| Palynziq Net Product Revenues* | $10 to $14 | |
| Cost of Sales (% of Total Revenues) | 20.0% to 21.0% | |
| Research and Development Expense | $680 to $710 | |
| Selling, General and Admin. Expense | $575 to $615 | |
| GAAP Net Loss | $(115) to $(165) | |
| Non-GAAP Income ** | $100 to $140 |
| * Guidance for Palynziq not previously provided |
| ** All Financial Guidance items are calculated based on US GAAP with the exception of Non-GAAP Income. Refer to Non-GAAP Information beginning on page 9 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the comparable GAAP reported information. |
Select Agenda Items at Upcoming R&D Day, November 7, 2018 in New York City :
Vosoritide for Achondroplasia:
PKU Gene Therapy Data and Program update; Research & Development Pipeline Deep Dive:
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A:
Regulatory Policy Advances at the Food and Drug Administration:
Key Program Highlights
Other Ongoing Clinical Development Programs:
BioMarin will host a conference call and webcast to discuss third quarter 2018 financial results today, Thursday, October 25, 2018 at 4:30 p.m. ET . This event can be accessed on the investor section of the BioMarin website at www.biomarin.com .
| U.S. / Canada Dial-in Number: 866.502.9859 | Replay Dial-in Number: 855.859.2056 |
| International Dial-in Number: 574.990.1362 | Replay International Dial-in Number: 404.537.3406 |
| Conference ID: 6487064 | Conference ID: 6487064 |
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of several approved products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com .
Forward-Looking Statements
BioMarin ® , Brineura ® , Firdapse ® , Kuvan ® , Naglazyme ® , Palynziq ® and Vimizim ® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. Aldurazyme ® is a registered trademark of BioMarin/Genzyme LLC.
| Contact: | ||
| Investors: | Media: | |
| Traci McCarty | Debra Charlesworth | |
| BioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. | |
| (415) 455-7558 | (415) 455-7451 |
| BIOMARIN PHARMACEUTICAL INC. | ||||||||
| CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
| September 30, 2018 and December 31, 2017 | ||||||||
| (In thousands of U.S. dollars, except share and per share amounts) | ||||||||
| September 30, 2018 (1) | December 31, 2017 (2) | |||||||
| ASSETS | (unaudited) | |||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 882,184 | $ | 598,028 | ||||
| Short-term investments | 561,192 | 797,940 | ||||||
| Accounts receivable, net | 384,343 | 261,365 | ||||||
| Inventory | 508,482 | 475,775 | ||||||
| Other current assets | 71,652 | 74,036 | ||||||
| Total current assets | 2,407,853 | 2,207,144 | ||||||
| Noncurrent assets: | ||||||||
| Long-term investments | 204,883 | 385,785 | ||||||
| Property, plant and equipment, net | 924,033 | 896,700 | ||||||
| Intangible assets, net | 494,687 | 517,510 | ||||||
| Goodwill | 197,039 | 197,039 | ||||||
| Deferred tax assets | 429,194 | 399,095 | ||||||
| Other assets | 37,565 | 29,852 | ||||||
| Total assets | $ | 4,695,254 | $ | 4,633,125 | ||||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable and accrued liabilities | $ | 378,226 | $ | 401,921 | ||||
| Short-term convertible debt, net | 374,230 | 360,949 | ||||||
| Short-term contingent acquisition consideration | 86,204 | 53,648 | ||||||
| Total current liabilities | 838,660 | 816,518 | ||||||
| Noncurrent liabilities: | ||||||||
| Long-term convertible debt, net | 826,119 | 813,521 | ||||||
| Long-term contingent acquisition consideration | 58,160 | 135,318 | ||||||
| Other long-term liabilities | 57,370 | 59,105 | ||||||
| Total liabilities | 1,780,309 | 1,824,462 | ||||||
| Stockholders' equity: | ||||||||
| Common stock, $0.001 par value: 500,000,000 shares authorized; 177,902,788 and 175,843,749 shares issued and outstanding, respectively. | 178 | 176 | ||||||
| Additional paid-in capital | 4,620,817 | 4,483,220 | ||||||
| Company common stock held by Nonqualified Deferred Compensation Plan | (13,415) | (14,224) | ||||||
| Accumulated other comprehensive loss | (2,160) | (22,961) | ||||||
| Accumulated deficit | (1,690,475) | (1,637,548) | ||||||
| Total stockholders' equity | 2,914,945 | 2,808,663 | ||||||
| Total liabilities and stockholders' equity | $ | 4,695,254 | $ | 4,633,125 |
| (1) | As of January 1, 2018, the Company adopted the requirements of Accounting Standards Codification 606, Revenue from Contracts with Customers (ASC 606), using the modified retrospective method, and as a result, there is a lack of comparability of certain amounts to the prior periods presented. |
| (2) | December 31, 2017 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, 2017, filed with the U.S. Securities and Exchange Commission on February 26, 2018. |
| BIOMARIN PHARMACEUTICAL INC. | ||||||||||||||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||||||
| Three and Nine Months Ended September 30, 2018 and 2017 | ||||||||||||||||
| (In thousands of U.S. dollars, except per share amounts) | ||||||||||||||||
| (Unaudited) | ||||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2018 (1) | 2017 | 2018 (1) | 2017 | |||||||||||||
| REVENUES: | ||||||||||||||||
| Net product revenues | $ | 386,320 | $ | 298,752 | $ | 1,123,205 | $ | 916,868 | ||||||||
| Royalty and other revenues | 5,386 | 35,396 | 14,793 | 38,473 | ||||||||||||
| Total revenues | 391,706 | 334,148 | 1,137,998 | 955,341 | ||||||||||||
| OPERATING EXPENSES: | ||||||||||||||||
| Cost of sales | 78,893 | 59,480 | 240,245 | 165,791 | ||||||||||||
| Research and development | 161,408 | 154,103 | 520,938 | 442,145 | ||||||||||||
| Selling, general and administrative | 148,566 | 130,532 | 440,182 | 394,056 | ||||||||||||
| Intangible asset amortization and contingent consideration | 18,580 | 3,760 | 42,009 | 26,096 | ||||||||||||
| Gain on sale of intangible assets | — | — | (20,000) | — | ||||||||||||
| Total operating expenses | 407,447 | 347,875 | 1,223,374 | 1,028,088 | ||||||||||||
| LOSS FROM OPERATIONS | (15,741) | (13,727) | (85,376) | (72,747) | ||||||||||||
| Equity in the loss of BioMarin/Genzyme LLC | (468) | (253) | (507) | (996) | ||||||||||||
| Interest income | 6,338 | 3,976 | 17,141 | 10,031 | ||||||||||||
| Interest expense | (12,131) | (10,884) | (35,918) | (31,043) | ||||||||||||
| Other income, net | 2,589 | 267 | 5,266 | 4,282 | ||||||||||||
| LOSS BEFORE INCOME TAXES | (19,413) | (20,621) | (99,394) | (90,473) | ||||||||||||
| Benefit from income taxes | (6,793) | (8,094) | (25,833) | (24,823) | ||||||||||||
| NET LOSS | $ | (12,620) | $ | (12,527) | $ | (73,561) | $ | (65,650) | ||||||||
| NET LOSS PER SHARE, BASIC AND DILUTED | $ | (0.07) | $ | (0.07) | $ | (0.42) | $ | (0.38) | ||||||||
| Weighted average common shares outstanding, basic and dilutive | 177,481 | 175,103 | 176,767 | 174,071 |
Non-GAAP Information
The results presented in this press release include both GAAP information and Non-GAAP information. As used in this release, Non-GAAP Income (Loss) is defined by the Company as GAAP Net Loss excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items, as detailed below when applicable. In addition, BioMarin includes in this press release the effects of these adjustments on certain components of GAAP Net Loss for each of the periods presented. In this regard, Non-GAAP Income (Loss) and its components, including Non-GAAP Cost of Sales, Non-GAAP Research and Development expenses, Non-GAAP Selling, General and Administrative expense, Non-GAAP Intangible Asset Amortization and Contingent Consideration, Non-GAAP Gain on the Sale of Intangible Asset and Non-GAAP Benefit From Income Taxes are statement of operations line items prepared on the same basis as, and therefore components of, the overall Non-GAAP measures.
Non-GAAP Income (Loss) and its components are not meant to be considered in isolation, as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the Company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its Non-GAAP measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP measures. Because of the non-standardized definitions, the Non-GAAP measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.
The following table presents the reconciliation of GAAP Net Loss to Non-GAAP Income:
| Reconciliation of GAAP Net Loss to Non-GAAP Income | |||||||||||||||||
| (In millions of U.S. dollars) | |||||||||||||||||
| (unaudited) | |||||||||||||||||
| Three Months Ended | Nine Months Ended | Year Ending | |||||||||||||||
| September 30, | September 30, | December 31, 2018 | |||||||||||||||
| 2018 | 2017 | 2018 | 2017 | Guidance | |||||||||||||
| GAAP Net Loss | $ | (12.6) | $ | (12.5) | $ | (73.6) | $ | (65.7) | $(115.0) - $(165.0) | ||||||||
| Interest expense, net | 5.8 | 6.9 | 18.8 | 21.0 | 25.0 - 35.0 | ||||||||||||
| Benefit from income taxes | (6.8) | (8.1) | (25.8) | (24.8) | (40.0) - 0.0 | ||||||||||||
| Depreciation expense | 18.6 | 13.3 | 48.1 | 36.9 | 50.0 - 60.0 | ||||||||||||
| Amortization expense | 7.6 | 7.6 | 22.7 | 22.7 | 30.0 | ||||||||||||
| Stock-based compensation expense | 37.1 | 35.9 | 112.3 | 106.7 | 150.0 - 170.0 | ||||||||||||
| Contingent consideration expense | 11.0 | (3.8) | 19.3 | 3.4 | 20.0 - 30.0 | ||||||||||||
| Gain on sale of intangible assets | — | — | (20.0) | — | (20.0) | ||||||||||||
| Royalty and other revenues | — | (31.5) | — | (31.5) | n/a | ||||||||||||
| Non-GAAP Income | $ | 60.7 | $ | 7.8 | $ | 101.8 | $ | 68.7 | $100 - $140 |
The following reconciliation of the GAAP reported to the Non-GAAP information provides the details of the effects of the Non-GAAP adjustments on certain components of the Company's operating results for each of the periods presented.
| Reconciliation Of Certain GAAP Reported Information To Non-GAAP Information | |||||||||||||||||||||||||||||||
| (In millions of U.S. dollars) | |||||||||||||||||||||||||||||||
| (Unaudited) | |||||||||||||||||||||||||||||||
| Three Months Ended September 30, | |||||||||||||||||||||||||||||||
| 2018 | 2017 | ||||||||||||||||||||||||||||||
| Adjustments | Adjustments | ||||||||||||||||||||||||||||||
| GAAP Reported | Interest, Taxes, Depreciation and Amortization | Stock-Based Compensation, Contingent Consideration and Other Adjustments | Non-GAAP | GAAP Reported | Interest, Taxes, Depreciation and Amortization | Stock-Based Compensation, Contingent Consideration and Other Adjustments | Non-GAAP | ||||||||||||||||||||||||
| Royalty and other revenues | $ | 5.4 | $ | — | $ | — | $ | 5.4 | $ | 35.4 | $ | — | $ | (31.5) | $ | 3.9 | |||||||||||||||
| Cost of sales | 78.9 | — | (4.0) | 74.9 | 59.5 | — | (3.0) | 56.5 | |||||||||||||||||||||||
| Research and development | 161.4 | (7.0) | (14.3) | 140.1 | 154.1 | (7.6) | (13.8) | 132.7 | |||||||||||||||||||||||
| Selling, general and administrative | 148.6 | (11.6) | (18.8) | 118.2 | 130.5 | (5.7) | (19.1) | 105.7 | |||||||||||||||||||||||
| Intangible asset amortization and contingent consideration | 18.6 | (7.6) | (11.0) | — | 3.8 | (7.6) | 3.8 | — | |||||||||||||||||||||||
| Gain on sale of intangible assets | — | — | — | — | — | — | — | ||||||||||||||||||||||||
| Interest expense, net | (5.8) | 5.8 | — | — | (6.9) | 6.9 | — | — | |||||||||||||||||||||||
| Benefit from income taxes | (6.8) | 6.8 | — | — | (8.1) | 8.1 | — | — | |||||||||||||||||||||||
| GAAP Net Loss/Non-GAAP Income | (12.6) | 25.2 | 48.1 | 60.7 | (12.5) | 19.7 | 0.6 | 7.8 |
| Nine Months Ended September 30, | |||||||||||||||||||||||||||||||
| 2018 | 2017 | ||||||||||||||||||||||||||||||
| Adjustments | Adjustments | ||||||||||||||||||||||||||||||
| GAAP Reported | Interest, Taxes, Depreciation and Amortization | Stock-Based Compensation, Contingent Consideration and Other Adjustments | Non-GAAP | GAAP Reported | Interest, Taxes, Depreciation and Amortization | Stock-Based Compensation, Contingent Consideration and Other Adjustments | Non-GAAP | ||||||||||||||||||||||||
| Royalty and other revenues | $ | 14.8 | $ | — | $ | — | $ | 14.8 | $ | 38.5 | $ | — | $ | (31.5) | $ | 7.0 | |||||||||||||||
| Cost of sales | 240.2 | — | (10.4) | 229.8 | 165.8 | — | (7.8) | 158.0 | |||||||||||||||||||||||
| Research and development | 520.9 | (25.4) | (43.1) | 452.4 | 442.1 | (20.2) | (40.0) | 381.9 | |||||||||||||||||||||||
| Selling, general and administrative | 440.2 | (22.7) | (58.8) | 358.7 | 394.1 | (16.7) | (58.9) | 318.5 | |||||||||||||||||||||||
| Intangible asset amortization and contingent consideration | 42.0 | (22.7) | (19.3) | — | 26.1 | (22.7) | (3.4) | — | |||||||||||||||||||||||
| Gain on sale of intangible assets | (20.0) | — | 20.0 | — | — | — | — | — | |||||||||||||||||||||||
| Interest expense, net | (18.8) | 18.8 | — | — | (21.0) | 21.0 | — | — | |||||||||||||||||||||||
| Benefit from income taxes | (25.8) | 25.8 | — | — | (24.8) | 24.8 | — | — | |||||||||||||||||||||||
| GAAP Net Loss/Non-GAAP Income (Loss) | (73.6) | 63.8 | 111.6 | 101.8 | (65.7) | 55.8 | 78.6 | 68.7 |
SOURCE BioMarin Pharmaceutical Inc.