R&D Pipeline Advancing; Many Key Clinical Milestones in the Coming Year
Net Product Revenue Growth of 18 Percent in 2011
Conference Call and Webcast to be Held Today at 5:00 p.m. ET
Financial Highlights ($ in millions, except per share data, unaudited)
|Item||FY 2011||FY 2010 Comparison|
|Total BioMarin Revenue||$441.4||17.3% increase|
|Total Net Product Revenue||$437.6||18.4% increase|
|Naglazyme Net Product Revenue||$224.9||16.7% increase|
|Aldurazyme BioMarin Net Product Revenue*||$82.8||16.3% increase|
|Kuvan Net Product Revenue||$116.8||17.5% increase|
|Firdapse Net Product Revenue||$13.2||106.3% increase|
|GAAP Net Income (Loss)||$(53.8)||$205.8**|
|GAAP Net Income (Loss) per share||$(0.48) (basic and diluted)||$2.00 (basic), $1.73 (diluted)|
|Non-GAAP Adjusted EBITDA||$34.5||$62.0|
* Net product transfer revenue had a positive $8.6 million impact on net Aldurazyme revenue to BioMarin in 2011 and a positive $3.2 million impact on net Aldurazyme revenue to BioMarin in 2010.
** During the third quarter of 2010, the company reversed its deferred tax asset valuation allowance and recorded a one-time benefit of $223.1 million.
NOVATO, Calif., Feb. 16, 2012 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced financial results for the fourth quarter and full year 2011. GAAP net loss was $26.7 million ($0.23 per diluted share) for the fourth quarter of 2011, compared to GAAP net loss of $12.2 million ($0.11 per diluted share) for the fourth quarter of 2010. Non-GAAP adjusted EBITDA was a loss of $3.0 million for the fourth quarter of 2011, compared to non-GAAP adjusted EBITDA of $11.0 million for the fourth quarter of 2010. Non-GAAP adjusted EBITDA excludes depreciation and amortization, contingent consideration expense, interest income and expense, income taxes, stock compensation expense and material non-recurring items. The reconciliation of the non-GAAP measures to the comparable GAAP measure is detailed in the table provided near the end of the press release.
GAAP net loss for the year ended December 31, 2011 was $53.8 million ($0.48 per diluted share), compared to GAAP net income of $205.8 million ($1.73 per diluted share) for the year ended December 31, 2010. During the third quarter of 2010, the company reversed its deferred tax asset valuation allowance and recorded a one-time benefit of $223.1 million. Non-GAAP adjusted EBITDA was $34.5 million for the year ended December 31, 2011, compared to non-GAAP adjusted EBITDA of $62.0 million for the year ended December 31, 2010.
As of December 31, 2011, BioMarin had cash, cash equivalents and short and long-term investments totaling $289.5 million, as compared to $370.0 million on September 30, 2011. The decline in the cash balance is due to a payment of $81.0 million for the purchase of Naglazyme royalty rights from the Adelaide Health Authority in the fourth quarter of 2011.
"The successful execution of our R&D pipeline to deliver therapies that have a meaningful impact in the lives of patients remains the top priority for BioMarin in 2012. Our most advanced clinical program, the Phase 3 trial for MPS IVA, is progressing well, and we anticipate that over 160 patients will be enrolled by mid-March. This is a month earlier than previously communicated thanks to enthusiasm for the study in the MPS IV community," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "Our growing commercial portfolio is funding the majority of our R&D expenses, and we look forward to many key clinical milestones in the coming year, including data readouts for GALNS for MPS IVA, PEG-PAL for PKU, BMN-701 for Pompe disease and BMN-673 for solid tumors."
Net Product Revenue (in millions)
|Three Months Ended December 31,||Years Ended Ended December 31,|
|2011||2010||$ Change||% Change||2011||2010||$ Change||% Change|
|Naglazyme (1)||$ 48.1||$ 45.1||$ 3.0||6.7%||$ 224.9||$ 192.7||$ 32.2||16.7%|
(1) Changes in foreign currency rates, net of hedges, had a negative $1.3 million impact on Naglazyme sales in the fourth quarter of 2011 and negative $0.2 million impact on Naglazyme sales for the year ended December 31, 2011. The number of Naglazyme patients increased 11.9 percent in the fourth quarter of 2011 compared to the fourth quarter of 2010. Naglazyme revenues experience quarterly fluctuations due to the timing of government ordering patterns in certain countries.
(2) The quantity of commercial tablets dispensed to patients in the U.S. increased 10.3 percent in the fourth quarter of 2011 compared to the fourth quarter of 2010.
(3) A product for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS) which was launched in the EU in April 2010.
|Three Months Ended December 31,||Years Ended December 31,|
|2011||2010||$ Change||% Change||2011||2010||$ Change||% Change|
|Aldurazyme revenue reported by Genzyme (4)||$ 48.8||$ 42.5||$ 6.3||14.8%||$ 185.2||$ 166.8||$ 18.4||11.0%|
|Royalties due from Genzyme||21.0||17.8||3.2||74.2||68.0||6.2|
|Incremental product transfer revenues||2.8||5.3||(2.5)||8.6||3.2||5.4|
|Total Aldurazyme net product revenues (5)||$ 23.8||$ 23.1||$ 0.7||$ 82.8||$ 71.2||$ 11.6|
(4) Changes in foreign currency rates caused a decrease to Aldurazyme sales by Genzyme of $0.6 million and an increase to Aldurazyme sales by Genzyme of $5.6 million for the three months and year ended December 31, 2011, respectively.
(5) To the extent units shipped to third party customers by Genzyme exceeded BioMarin inventory transfers to Genzyme, BioMarin will record a decrease in net product revenue from the royalty payable to BioMarin for the amount of previously recognized product transfer revenue. If BioMarin inventory transfers exceed units shipped to third party customers by Genzyme, BioMarin will record incremental net product transfer revenue for the period.
|Revenue Guidance ($ in millions)|
|Item||2012 Guidance||2011 Actual|
|Total BioMarin Revenues||$465 to $510||$441|
|Total Net Product Revenues||$460 to $505||$438|
|Naglazyme Net Product Revenue||$240 to $265||$225|
|Kuvan Net Product Revenue||$126 to $136||$117|
|Aldurazyme Net Product Revenue to BioMarin||$81 to $87||$83|
|Firdapse Net Product Revenue||$13 to $17||$13|
|Selected Income Statement Guidance ($ in millions)|
|Item||2012 Guidance||2011 Actual|
|Cost of Sales (% of Total Revenue)||17% to 18%||19%|
|Selling, General and Admin. Expense||$195 to $205||$175|
|Research and Development Expense||$255 to $265*||$214|
|Amortization and Contingent Consideration||$19||$1|
|Income Tax Expense (Benefit)||$ --||$10|
|GAAP Net Income (Loss)||$(92) to $(82)||$(54)|
|Stock Compensation Expense||$46||$44|
|Non-GAAP Adjusted EBITDA||$15 to $25||$35|
|Non GAAP Net Cash Flow (Usage)||$(30) to $(40)||$(113)**|
|Cash Balance***||$250 to $260||$289.50|
* Approximately $45 million of the $255 million to $265 million is for the production of drug supply for clinical studies.
** Net cash usage in 2011 includes a payment of $81 million for the purchase of Naglazyme royalty rights and a payment of $48.5 million related to the acquisition of the Shanbally manufacturing facility.
*** Cash balance includes cash, cash equivalents and short and long term investments.
Anticipated Upcoming Milestones
|1Q 2012: Initiation of Phase I trial for BMN-111 for achondroplasia in healthy volunteers|
|1Q 2012: Enrollment of targeted 160 patients in the Phase III trial for GALNS for MPS IVA|
|3Q 2012: Results for Phase II trial for PEG-PAL for PKU|
|3Q 2012: Results for Phase I trial for BMN-111 for achondroplasia in healthy volunteers|
|2H 2012: Results for Phase I/II trial for BMN-673 for solid tumors|
|4Q 2012: Results for Phase I/II trial for BMN-701 for Pompe disease|
|4Q 2012: Results for Phase III trial for GALNS for MPS IVA|
|4Q 2012/1Q 2013: Initiation of Phase II trial for BMN-111 for achondroplasia in patients|
|1Q 2013: Market authorization application filings for GALNS for MPS IVA|
|1Q 2013: Results for Phase I/II trial for BMN-673 for hematological malignancies|
|1Q 2013: Initiation of Phase III trial for PEG-PAL for PKU|
|1Q 2013: IND filing for BMN-190 for LINCL (Batten disease)|
|Mid-2013: Results for PKU-016 Kuvan neurocognitive study|
|2H 2013: Potential initiation of Phase III trial for BMN-701 for Pompe disease|
|2H 2013: Potential initiation of Phase III trial for BMN-673 for solid tumors or hematological malignancies|
|4Q 2013: Anticipated FDA approval of GALNS for MPS IVA|
Research and Development Programs
BioMarin continues to make significant investments in research and development to ensure persistent growth for the company. The current pipeline includes programs in various stages of development that are focused on treating a range of serious unmet medical needs.
Advanced Clinical Programs
- GALNS for MPS IVA: BioMarin is on track to enroll the targeted 160 patients in the pivotal Phase III trial in mid-March and to report top-line results in the fourth quarter of 2012. The company expects to submit the first market authorization application filing in the first quarter of 2013.
Mid-Stage Clinical Programs
- PEG-PAL for PKU: The company has initiated Part D of the Phase II trial to investigate the shortest weekly induction regimen to an efficacious maintenance dose. Results from Part D of the Phase II study are expected in the third quarter of 2012, and if successful, the Phase III study is expected to start in the first quarter of 2013 after meetings with regulatory authorities.
- Kuvan Outcomes Study: PKU-016, a randomized, placebo-controlled, 13-week Kuvan outcomes study is ongoing. Endpoints include clinically validated measures of neuropsychiatric symptoms and, if successful, may enable a label amendment. The company expects to report top-line results in mid-2013.
Early-Stage Clinical Programs
- BMN-701 for Pompe Disease: A total of six patients have been enrolled in the 5 mg/kg and 10 mg/kg cohorts, and the first patient in the 20 mg/kg cohort was dosed in January 2012. BMN-701 has been generally well-tolerated with a safety profile consistent with other enzyme replacement therapies. Top-line results for the Phase I/II trial, including data in 15 to 20 patients dosed at 20 mg/kg, are expected in the fourth quarter of 2012.
- BMN-673 (PARP inhibitor): BioMarin has two ongoing trials for BMN-673: a Phase I/II open-label trial of once daily, orally administered BMN-673 for advanced or recurrent solid tumors and a Phase I, two-arm, open-label, dose escalation study for BMN-673 for the treatment of patients with advanced hematological malignancies. Top-line results are expected in the second half of 2012 and the first quarter of 2013, respectively.
- BMN-111 for Achondroplasia: BMN-111 is an analog of C-type Natriuretic Peptide (CNP), a small cyclic peptide that is a positive regulator of bone growth. The company initiated the Phase I study in healthy volunteers in February 2012 and expects results by the third quarter of 2012.
- BMN-190 for LINCL (Batten disease): BioMarin expects to file the IND for BMN-190 in the first quarter of 2013.
- Other early stage programs: BioMarin is working on multiple additional early development opportunities.
Non-GAAP Financial Information and Reconciliation
The above results for the three months and year ended December 31, 2011 and December 31, 2010 and financial guidance for the year ending December 31, 2012 are presented both as determined in accordance with GAAP and on a non-GAAP basis. As used in this release, non-GAAP income is based on GAAP Earnings Before Interest, Taxes, Depreciation and Amortization (EBITDA) adjusted to exclude non-cash stock compensation expense, contingent consideration expense and certain nonrecurring material items (Non-GAAP Adjusted EBITDA).
The following tables detail the reconciliation of non-GAAP to GAAP financial metrics:
|Reconciliation of GAAP Net Income (Loss) to Non-GAAP Adjusted EBITDA (Loss)|
|Three Months Ended|
|Years Ended |
|Year Ending |
|GAAP Net Income (Loss)||$ (26.7)||$ (12.2)||$ (53.8)||$ 205.8||$(92.0) - $(82.0)|
|Interest expense, net||1.1||1.3||5.4||6.2||6.0|
|Income tax expense (benefit)||(1)||3.6||(7.0)||10.2||(227.3)||--|
|EBITDA (Loss)||(14.8)||(11.9)||(9.4)||5.9||(46.0) - (36.0)|
|Gain on sale of equity investments||--||--||--||(0.9)||--|
|Convertible debt exchange||(3)||--||13.7||1.9||13.7||--|
|Non-GAAP Adjusted EBITDA (Loss)||$ (3.0)||$ 11.0||$ 34.5||$ 62.0||$15.0 - $25.0|
|(1) Includes the release of the Company's income tax valuation allowance during 2010.|
|(2) Represents the changes in the fair value of contingent acquisition consideration payable for the period.|
|(3) Represents debt conversion expense associated with the early conversion of a portion of our convertible debt in September 2011 and November 2010.|
|(4) Represents transaction costs associated with the acquisition of ZyStor Therapeutics Inc., during the third quarter of 2010.|
BioMarin believes that this non-GAAP information is useful to investors, taken in conjunction with BioMarin's GAAP information, because it provides additional information regarding the performance of BioMarin's core ongoing business, Naglazyme, Kuvan, Aldurazyme and Firdapse and development of its pipeline. By providing information about both the overall GAAP financial performance and the non-GAAP measures that focus on continuing operations, the company believes that the additional information enhances investors' overall understanding of the company's business and prospects for the future. Further, the company uses both the GAAP and the non-GAAP results and expectations internally for its operating, budgeting and financial planning purposes and uses the Non-GAAP Adjusted EBITDA methodology in establishing corporate goals for internal compensation programs.
Diluted Earnings Per Share Calculation
The following table sets for the computation of GAAP diluted earnings per share:
|GAAP Basic and Diluted Earnings/loss per common share||Three Months Ended December 31,||Years Ended December 31,|
|GAAP Net income (loss), basic||$ (26,735)||$ (12,189)||$ (53,836)||$ 205,819|
|Interest expense on convertible debt||--||--||--||9,977|
|Amortization of deferred offering costs related to convertible debt||--||--||--||1,549|
|GAAP Net income (loss), diluted||$ (26,735)||$ (12,189)||$ (53,836)||$ 217,345|
|Denominator (in thousands of common shares):|
|Basic weighted-average shares outstanding||114,415||107,344||112,122||103,093|
|Effect of dilutuve securities:|
|Potentially issuable shares under equity compensation awards||--||--||--||3,452|
|Common stock issuable under convertible debt||--||--||--||19,129|
|Fully diluted weighted-average shares||114,415||107,344||112,122||125,674|
|GAAP Basic earnings (loss) per common share||$ (0.23)||$ (0.11)||$ (0.48)||$ 2.00|
|GAAP Diluted earnings (loss) per common share||$ (0.23)||$ (0.11)||$ (0.48)||$ 1.73|
Conference Call Details
BioMarin will host a conference call and webcast to discuss fourth quarter and full year 2011 financial results today, Thursday, February 16, at 5:00 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.
|Date: February 16, 2012|
|Time: 5:00 p.m. ET|
|U.S. / Canada Dial-in Number: 866.761.0748|
|International Dial-in Number: 617.614.2706|
|Participant Code: 11776309|
|Replay Dial-in Number: 888.286.8010|
|Replay International Dial-in Number: 617.801.6888|
|Replay Code: 48391178|
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-nutriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
The BioMarin Pharmaceutical Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=11419
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, including, without limitation, statements about: the expectations of revenue and sales related to Naglazyme, Kuvan, Firdapse, and Aldurazyme; the financial performance of the BioMarin as a whole; the timing of BioMarin's clinical trials of GALNS, Firdapse, PEG-PAL, BMN-673, BMN-701, BMN-111 and other product candidates; the continued clinical development and commercialization of Aldurazyme, Naglazyme, Kuvan, Firdapse, and its product candidates; and actions by regulatory authorities. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: our success in the continued commercialization of Naglazyme, Kuvan, and Firdapse; Genzyme Corporation's success in continuing the commercialization of Aldurazyme; results and timing of current and planned preclinical studies and clinical trials, particularly with respect to GALNS, Firdapse, PEG-PAL, BMN-673, BMN-701 and BMN-111; our ability to successfully manufacture our products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products and particularly Aldurazyme, Naglazyme, Kuvan and Firdapse; actual sales of Aldurazyme, Naglazyme Kuvan and Firdapse; Merck Serono's activities related to Kuvan; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2010 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, Naglazyme®, Kuvan® and Firdapse™ are registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
|BIOMARIN PHARMACEUTICAL INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|December 31, 2011 and 2010|
|(In thousands of U.S. dollars, except share and per share amounts)|
|Cash and cash equivalents||$ 46,272||$ 88,079|
|Accounts receivable, net (allowance for doubtful accounts: $513 and $63, respectively)||104,839||86,576|
|Other current assets||39,753||33,874|
|Total current assets||469,802||504,260|
|Investment in BioMarin/Genzyme LLC||559||1,082|
|Property, plant and equipment, net||268,971||221,866|
|Intangible assets, net||180,277||103,648|
|Long-term deferred tax assets||222,649||236,017|
|Total assets||$ 1,303,681||$ 1,262,623|
|LIABILITIES AND STOCKHOLDERS' EQUITY|
|Accounts payable and accrued liabilities||$ 94,125||$ 83,844|
|Total current liabilities||94,125||83,844|
|Other long-term liabilities||88,179||84,001|
|Common stock, $0.001 par value: 250,000,000 shares authorized at December 31, 2011 and 2010: 114,789,732 and 110,634,465 shares issued and outstanding at December 31, 2011 and 2010, respectively||115||111|
|Additional paid-in capital||1,197,082||1,090,188|
|Company common stock held by Nonqualified Deferred Compensation Plan||(3,935)||(1,965)|
|Accumulated other comprehensive income||4,887||188|
|Total stockholders' equity||773,048||717,257|
|Total liabilities and stockholders' equity||$ 1,303,681||$ 1,262,623|
|(1) December 31, 2010 balances were derived from the audited consolidated financial statements.|
|BIOMARIN PHARMACEUTICAL INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|Three and Twelve Months Ended December 31, 2011 and 2010|
|(In thousands of U.S. dollars, except per share amounts)|
|Three Months Ended December 31,||Twelve Months Ended December 31,|
|Net product revenues||$ 106,064||$ 98,477||$ 437,647||$ 369,701|
|Collaborative agreement revenues||93||175||468||682|
|Royalty and license revenues||1,689||2,962||3,243||5,884|
|Cost of sales (excludes amortization of developed product technology)||21,519||20,469||84,023||70,285|
|Research and development||57,908||42,197||214,374||147,309|
|Selling, general and administrative||48,454||42,098||175,423||151,723|
|Intangible asset amortization and contingent consideration||1,400||200||1,428||6,406|
|Total operating expenses||129,281||104,964||475,248||375,723|
|INCOME (LOSS) FROM OPERATIONS||(21,435)||(3,350)||(33,890)||544|
|Equity in the loss of BioMarin/Genzyme LLC||(609)||(797)||(2,426)||(2,991)|
|Debt conversion expense||―||(13,728)||(1,896)||(13,728)|
|Net gain (loss) from sale of investments||―||(25)||―||902|
|INCOME (LOSS) BEFORE INCOME TAXES||(23,116)||(19,238)||(43,627)||(21,490)|
|Provision for (benefit from) income taxes||3,619||(7,049)||10,209||(227,309)|
|NET INCOME (LOSS)||$ (26,735)||$ (12,189)||$ (53,836)||$ 205,819|
|NET INCOME (LOSS) PER SHARE, BASIC||$ (0.23)||$ (0.11)||$ (0.48)||$ 2.00|
|NET INCOME (LOSS) PER SHARE, DILUTED||$ (0.23)||$ (0.11)||$ (0.48)||$ 1.73|
|Weighted average common shares outstanding, basic||114,415||107,344||112,122||103,093|
|Weighted average common shares outstanding, diluted||114,415||107,344||112,122||125,674|
|(1) The totals for the year ended December 31, 2010 were derived from the audited consolidated financial statements.|
|STOCK-BASED COMPENSATION EXPENSE|
|Total stock-based compensation expense included in the Condensed Consolidated Statements of Operations is as follows:|
|Three Months Ended||Years Ended|
|December 31,||December 31,|
|Cost of sales||$ 1,307||$ 1,417||$ 5,171||$ 4,269|
|Research and development||4,295||3,516||16,365||13,760|
|Selling, general and administrative||5,610||4,886||22,283||19,463|
|$ 11,212||$ 9,819||$ 43,819||$ 37,492|
CONTACT: Investors Eugenia Shen BioMarin Pharmaceutical Inc. (415) 506-6570 Media Bob Purcell BioMarin Pharmaceutical Inc. (415) 506-3267