BioMarin Announces Fourth Quarter and Full Year 2012 Financial Results

BioMarin Announces Fourth Quarter and Full Year 2012 Financial Results

                  Total Revenue in 2012 Exceeds $500 Million

   PEG-PAL End of Phase 2 Meeting Completed, Phase 3 to Begin Next Quarter

         Conference Call and Webcast to Be Held Today at 5:00 p.m. ET

Financial Highlights ($ in millions, except per share data, unaudited)

                                   FY 2012      FY 2011      Percent Change
Total BioMarin Revenue              $500.7     $441.4     13.4%
Total Net Product Revenue           496.5       437.6       13.5%
Naglayzme Net Product Revenue       257.0       224.9       14.3%
Aldurazyme BioMarin Net Product     82.2        82.8        -0.7%
Kuvan Net Product Revenue           143.1       116.8       22.5%
Firdapse Net Product Revenue        14.2        13.2        7.6%
GAAP Net Loss                       (114.3)     (53.8)      
GAAP Net Loss per Share (basic and  $(0.95)    $(0.48)    
Non-GAAP Adjusted EBITDA Income     $(11.6)    $34.5      
Cash, cash equivalents and short    $566.7     $289.5     95.8%
and long-term investments*
* The cash balance at the end of 2012 includes net proceeds of $235.5 million
from the public offering in June 2012.

SAN RAFAEL, Calif., Feb. 21, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced financial results for the fourth quarter
and full year 2012. GAAP net loss was $53.0 million ($0.43 per share) for the
fourth quarter of 2012, compared to GAAP net loss of $26.7 million ($0.23 per
share) for the fourth quarter of 2011.Non-GAAP adjusted EBITDA was a loss of
$15.5 million for the fourth quarter of 2012, compared to non-GAAP adjusted
EBITDA loss of $3.0 million for the fourth quarter of 2011.The increased GAAP
net loss and the reduced non-GAAP adjusted EBITDA for the fourth quarter of
2012 compared to the fourth quarter of 2011 was primarily due to increased net
product revenue offset by a larger increase in research and development

GAAP net loss for the year ended December 31, 2012 was $114.3 million ($0.95
per share), compared to GAAP net loss of $53.8 million ($0.48 per share) for
the year ended December 31, 2011.Non-GAAP adjusted EBITDA was a loss of $11.6
million for the year ended December 31, 2012, compared to non-GAAP adjusted
EBITDA of $34.5 million for the year ended December 31, 2011. The increased
GAAP net loss and the reduced non-GAAP adjusted EBITDA for the year ended
December 31, 2012 compared to the year ended December 31, 2011 was primarily
due to increased research and development expenses and increased selling,
general and administrative expenses partially offset by increased net product

As of December 31, 2012, BioMarin had cash, cash equivalents and short and
long-term investments totaling $566.7 million, as compared to $533.2 million
on September 30, 2012.

"2012 was a milestone year for BioMarin. Our growing commercial portfolio
helped us surpass $500 million in total revenue and the pipeline continued to
advance, culminating in positive results for the pivotal Phase 3 study for
Vimizim at the end of the year," said Jean-Jacques Bienaimé, Chief Executive
Officer of BioMarin."We are poised for additional clinical milestones in the
first half of 2013 with key data readouts from BMN-701 for Pompe disease and
BMN-673, our PARP inhibitor.We also expect to have our first regulatory
approval for Vimizim by the end of 2013, which we believe will propel the
company into its next stage of growth."

Net Product Revenue (in millions)

          ThreeMonthsEnded December 31,   TwelveMonthsEnded December
          2012      2011     $ Change %      2012     2011     $       %
                                       Change                   Change  Change
Naglazyme  $63.0   $48.1  $14.9  31.0%  $257.0 $224.9 $32.1 14.3%
Kuvan     40.0      30.8     9.2      29.9%  143.1    116.8    26.3    22.5%
Firdapse  3.4       3.3      0.1      3.0%   14.2     13.2     1.0     7.6%

(1) Naglazyme revenues experience quarterly fluctuations due to the timing of
government ordering patterns in certain countries.

            Three Months Ended December    Twelve Months Ended December 31,
            2012    2011    $      %       2012     2011     $ Change %
                             Change Change                            Change
revenue      $53.1 $48.8 $4.3 8.8%    $193.1 $185.2 $7.9   4.3%
reported by
due from     23.9   21.0   2.9          80.4    74.2    6.2     
transfer     0.7    2.8    (2.1)        1.8     8.6     (6.8)   
Aldurazyme   $24.6 $23.8 $0.8        $82.2  $82.8  $(0.6) 
net product
(2) To the extent units shipped to third party customers by Genzyme exceed
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.

2013 Guidance
Revenue Guidance ($ in millions)
Item                                2013 Guidance
Total BioMarin Revenues             $530 to $555
Naglazyme Net Product Revenue       $265 to $285
Kuvan Net Product Revenue           $155 to $170
Selected Income Statement Guidance ($ in millions)
Item                                2013 Guidance
Cost of Sales (% of Total Revenue)  17% to 18%
Selling, General and Admin. Expense $220 to $250
Research and Development Expense*   $340 to $380
GAAP Net Loss                       $(195) to $(170)
Non-GAAP Adjusted EBITDA (loss)     $(75) to $(50)
Cash Balance**                      Over $420

*Research and Development expense guidance includes expenses associated with
the Zacharon acquisition and the University of College London license
** Cash balance includes cash, cash equivalents and short and long term

Anticipated Upcoming Milestones

1Q 2013: File market authorization application for Vimizim (GALNS) for MPS IVA
in the U.S.

1Q 2013: Results for Phase 1/2 trial for BMN-701 for Pompe disease

1Q 2013: Clinical trial application filing for BMN-190 for LINCL (Batten

2Q 2013: File market authorization application for Vimizim (GALNS) for MPS IVA
in the EU

2Q 2013: Initiation of Phase 3 trial for PEG-PAL for PKU

2Q 2013: Presentation on Phase 1/2 BMN-673 solid tumor data at ASCO meeting

Mid 2013: Initiation of Phase 2 trial for BMN-111 for achondroplasia

4Q 2013: Potential initiation of Phase 3 trial for BMN-673 for solid tumors

4Q 2013: Potential FDA approval of Vimizim for MPS IVA

4Q 2013: Potential initiation of Phase 2/3 trial, a key component of the
pivotal program for BMN-701 for Pompe disease

Research and Development Programs

BioMarin continues to make significant investments in research and development
to ensure a strong and profitable pipeline for the company.The current
pipeline includes programs in various stages of development that focus on
treating a range of rare and serious unmet medical needs.

Advanced Clinical Programs

  *Vimizim for MPS IVA: The company is on track to begin submitting market
    authorization application filings by the end of the first quarter of
    2013.The three ancillary studies are ongoing: a clinical trial for
    patients under five years of age (fully enrolled), a cardiopulmonary study
    to more completely document the health benefits ofVimizim (fully
    enrolled), and a study in patients with limited ambulation (enrolling).

Mid-Stage Clinical Programs

  *PEG-PAL for PKU:The company plans to initiate a pivotal Phase 3 study in
    the second quarter of 2013, following a recent end of Phase 2 meeting with
    the FDA.The Phase 3 design includes (1) an open-label study to evaluate
    safety and blood Phe levels in naïve patients and (2) a randomized
    controlled study of the Phase 2 extension study patients to evaluate blood
    Phe levels and neurocognitive endpoints.The FDA indicated the possibility
    for an accelerated approval based on demonstrating sustained reduction in
    Phe levels, though full approval may require demonstration of
    neurocognitive improvement.
  *Kuvan Outcomes Study: The company recently reported top-line results of a
    randomized, placebo-controlled, 13-week Kuvan outcomes study.The primary
    endpoint of the study was an attention deficit hyperactivity rating scale
    (ADHD-RS), a commonly used test to evaluate symptoms of inattentiveness
    and hyperactivity.Kuvan improved the ADHD-RS (p=0.085), driven by a
    statistically significant change in the inattention component of the score
    (p=0.036).The company plans to discuss the submission of this data with
    the FDA for possible inclusion in the Kuvan label.

Early-Stage Clinical Programs

  *BMN-701 for Pompe Disease: Top-line results for the Phase 1/2 trial and a
    program go/no go decision are expected at the end of the first quarter of
    2013.The company recently determinedthat if it proceeds with development
    of BMN-701, it will utilize a new cell line.If the decision is made to
    continue development, the company will be prepared to start a Phase 2/3
    trial, a key component of the pivotal program for BMN-701 with the new
    cell line in the fourth quarter of 2013.
  *BMN-673 (PARP inhibitor):An update on the Phase 1/2 study in solid tumors
    is expected in the second quarter of 2013 at the ASCO Annual Meeting in
    June 2013.A Phase 3 trial in advanced or recurrent solid tumors could
    start enrolling by the end of 2013.
  *BMN-111 for Achondroplasia: The company plans to initiate a Phase 2 trial
    in patients in mid 2013. The primary objective of the clinical proof of
    concept study in pediatric patients will be to evaluate the safety and
    tolerability of daily subcutaneous (sc) injections of BMN-111 administered
    for six months.Secondary objectives of the study will be to assess
    changes in annualized growth velocity, changes in absolute growth and
    changes in body proportions.Other exploratory objectives will also be
  *BMN-190 for LINCL (Batten disease): BioMarin expects to file for its first
    clinical study of BMN-190 in the first quarter of 2013.Pre-CTA/IND
    meetings have already been held with MHRA BfArM, and FDA.BioMarin expects
    to begin enrolling the study mid year 2013.

Preclinical Programs

  *Other early stage programs: BioMarin is working on multiple additional
    early development opportunities, including two new lead optimization
    programs gained through the acquisition of Zacharon Pharmaceuticals:
    inhibition of heparan sulfate synthesis for MPS III and inhibition of
    ganglioside synthesis for diseases such as Tay Sachs and Sandhoff.The
    company also announced today the licensing of a Factor VIII gene therapy
    research program for hemophilia A from University College London and St.
    Jude Children's Research Hospital.

Non-GAAP Financial Information and Reconciliation

The results for the three months and year ended December 31, 2012 and December
31, 2011 are all determined in accordance with GAAP except for non-GAAP
adjusted EBITDA which is determined on a non-GAAP basis. As used in this
release, non-GAAP adjusted EBITDA is based on GAAP earnings before interest,
taxes, depreciation and amortization (EBITDA) and further adjusted to also
exclude certain non-cash stock compensation expense, non-cash contingent
consideration expense and certain other nonrecurring material items (non-GAAP
adjusted EBITDA).

The following table presents the reconciliation of non-GAAP to GAAP financial

Reconciliation of GAAP Net Loss to Non-GAAP Adjusted EBITDA Income (Loss)
(in millions)
                               Three Months Ended     Twelve Months Ended
                                 December 31,          December 31,
                      NOTES     2012        2011       2012        2011
GAAP Net Loss                   $(53.0)   $(26.7)  $(114.3)  $(53.8)
Interest expense, net           1.2        1.1       5.0        5.4
Income tax expense              2.9        3.6       (3.9)      10.2
Depreciation                    6.6        5.7       27.5       24.4
Amortization                    2.6        1.5       17.3       4.4
EBITDA (Loss)                   (39.7)     (14.8)    (68.4)     (9.4)
Stock-based                     12.1       11.2      48.0       43.8
Contingent             (1)      12.1       0.6       8.8        (1.8)
Material non-recurring                                          
Convertible debt       (2)       --        --                  1.9
Non-GAAP Adjusted               $(15.5)   $(3.0)   $(11.6)   $34.5
EBITDA Income (Loss)

(1) Represents the changes in the fair value of contingent acquisition
consideration payable for the period. The change in the current quarter
reflects changes in estimated probabilities and timing of achieving certain
developmental milestones.
(2) Represents debt conversion expense associated with the early conversion of
a portion of our convertible debt in September 2011.

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.

Conference Call Details

BioMarin will host a conference call and webcast to discuss fourth quarter and
full year 2012 financial results today, Thursday, February 21, at 5:00 p.m.
ET. This event can be accessed on the investor section of the BioMarin website

Date: February 21, 2013
Time: 5:00 p.m. ET
U.S. / Canada Dial-in Number: 877.303.6313
International Dial-in Number: 631.813.4734
Conference ID: 49613469

Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 49613469

About BioMarin

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 Vimizim
(N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which
successfully completed Phase III clinical development for the treatment of MPS
IVA, 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-natriuretic peptide, which is currently in Phase I clinical development for
the treatment of achondroplasia. For additional information, please visit Information on BioMarin's website is not incorporated by
reference into this press release.

The BioMarin Pharmaceutical Inc. logo is available at

Forward-Looking Statement

This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., 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 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 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 2011 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^®, Firdapse^® and Vimizim™ are registered
trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme^® is a registered trademark of BioMarin/Genzyme LLC.

December 31, 2012 and December 31, 2011
(In thousands of U.S. dollars, except share and per share amounts)
                                       December 31, 2012 December 31, 2011(1)
ASSETS                                  (unaudited)      
Current assets:                                          
Cash and cash equivalents               $180,527        $46,272
Short-term investments                  270,211          148,820
Accounts receivable, net (allowance for
doubtful accounts: $348 and $513,       109,066          104,839
Inventory                               128,695          130,118
Current deferred tax assets             29,454           21,115
Other current assets                    25,509           18,638
Total current assets                   743,462          469,802
Noncurrent assets:                                       
Investment in BioMarin/Genzyme LLC      1,080            559
Long-term investments                   115,993          94,385
Property, plant and equipment, net      284,473          268,971
Intangible assets, net                  162,980          180,277
Goodwill                                51,543           51,543
Long-term deferred tax assets           225,501          224,677
Other assets                            16,611           15,495
Total assets                            $1,601,643      $1,305,709
Current liabilities:                                     
Accounts payable and accrued            $147,068        $94,125
Convertible debt                        23,365           --
Total current liabilities               170,433          94,125
Noncurrent liabilities:                                  
Long-term convertible debt              324,859          348,329
Long-term contingent acquisition        30,618           33,059
consideration payable
Long-term deferred tax liabilties       33,296           37,155
Other long-term liabilities             26,674           19,993
Total liabilities                       585,880          532,661
Stockholders' equity:                                    
Common stock, $0.001 par value:
250,000,000 shares authorized at
December 31, 2011 and 2012; 125,809,162 126              115
and 114,789,732 shares issued and
outstanding at December 31, 2012 and
2011, respectively.
Additional paid-in capital              1,561,890        1,197,082
Company common stock held by            (6,603)          (3,935)
Nonqualified Deferred Compensation Plan
Accumulated other comprehensive income  (202)            4,887
Accumulated deficit                     (539,448)        (425,101)
Total stockholders' equity              1,015,763        773,048
Total liabilities and stockholders'     $1,601,643      $1,305,709
(1) December 31, 2011 balances were derived from the audited consolidated
financial statements.

Three and Twelve Months Ended December 31, 2012 and 2011
(In thousands of U.S. dollars, except per share amounts)
                     Three Months Ended December Twelve Months Ended December
                      31,                         31,
                     2012          2011          2012           2011
Net product revenues  $130,957    $106,064    $496,497     $437,647
Collaborative         226          93           1,955         468
agreement revenues
Royalty and license   755          1,689        2,271         3,243
Total revenues        131,938      107,846      500,723       441,358
OPERATING EXPENSES:                                           
Cost of sales
amortization of       26,532       21,519       91,830        84,023
certain acquired
intangible assets)
Research and          84,363       57,908       302,218       214,374
Selling, general and  55,049       48,454       198,173       175,423
Intangible asset
amortization and      12,898       1,400        18,717        1,428
Total operating       178,842      129,281      610,938       475,248
LOSS FROM OPERATIONS  (46,904)     (21,435)     (110,215)     (33,890)
Equity in the loss of (253)        (609)        (1,221)       (2,426)
BioMarin/Genzyme LLC
Interest income       765          632          2,584         2,934
Interest expense      (1,930)      (1,878)      (7,639)       (8,409)
Debt conversion       --          --          --           (1,896)
Other income and      (1,772)      174          (1,787)       60
LOSS BEFORE INCOME    (50,094)     (23,116)     (118,278)     (43,627)
Provision for
(benefit from) income 2,918        3,619        (3,931)       10,209
NET LOSS              $(53,012)   $(26,735)   $(114,347)   $(53,836)
NET LOSS PER SHARE,   $(0.43)     $(0.23)     $(0.95)      $(0.48)
Weighted average
common shares         124,575       114,415       120,271        112,122
outstanding, basic
and diluted


Total stock-based compensation expense included in the Condensed Consolidated
Statements of Operations is as follows:
                      ThreeMonthsEnded          TwelveMonthsEnded
                       December 31,                December 31,
                      2012          2011          2012          2011
                      (unaudited)   (unaudited)   (unaudited)   (unaudited)
Cost of sales          $1,355      $1,307      $4,890      $5,171
Research and           5,385         4,295         20,736        16,365
Selling, general and   5,325         5,610         22,346        22,283
                      $12,065     $11,212     $47,972     $43,819

CONTACT: Investors:
         Eugenia Shen
         BioMarin Pharmaceutical Inc.
         (415) 506-6570
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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