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BioMarin Announces Third Quarter 2012 Financial Results

BioMarin Announces Third Quarter 2012 Financial Results

             Steady Commercial Growth Supports Advancing Pipeline
                  GALNS Phase 3 Results Expected in 4Q 2012

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

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

                                           Q3 2012   Q3 2011   Percent Change
Total BioMarin Revenue                      $128.1  $113.4  13.0%
Total Net Product Revenue                   126.3    112.9    11.9%
Naglayzme Net Product Revenue               62.5     55.9     11.8%
Aldurazyme BioMarin Net Product Revenue     23.8     23.0     3.5%
Kuvan Net Product Revenue                   36.4     30.5     19.3%
Firdapse Net Product Revenue                3.6      3.5      2.9%
GAAP Net Loss                               (5.4)    (17.7)   
GAAP Net Loss per Share (basic and diluted) $(0.04) $(0.16) 
Non-GAAP Adjusted EBITDA Income             11.1     4.3      

SAN RAFAEL, Calif., Oct. 25, 2012 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced financial results for the third quarter of
2012. GAAP net loss was $5.4 million ($0.04 per share) for the third quarter
of 2012, compared to GAAP net loss of $17.7 million ($0.16 per share) for the
third quarter of 2011.Non-GAAP adjusted EBITDA was $11.1 million for the
third quarter of 2012, compared to non-GAAP adjusted EBITDA of $4.3 million
for the third quarter of 2011.The reduced GAAP net loss and the increased
non-GAAP adjusted EBITDA for the third quarter of 2012 compared to the third
quarter of 2011 was primarily due to increased net product revenue partially
offset by a smaller increase in research and development expenses.

GAAP net loss for the nine months ended September 30, 2012 was $61.3 million
($0.52 per share), compared to GAAP net loss of $27.1 million ($0.24 per
share) for the nine months ended September 30, 2011.Non-GAAP adjusted EBITDA
was $3.9 million for the nine months ended September 30, 2012, compared to
non-GAAP adjusted EBITDA of $37.4 million for the nine months ended September
30, 2011. The increased GAAP net loss and the reduced non-GAAP adjusted EBITDA
for the first three quarters of 2012 compared to the first three quarters of
2011 was primarily due to increased research and development expenses and
increased selling, general and administrative expenses partially offset by
increased net product revenue.

As of September 30, 2012, BioMarin had cash, cash equivalents and short and
long-term investments totaling $533.2 million, as compared to $524.6 million
on June 30, 2012.

"During the third quarter, the commercial portfolio showed continued steady
growth, and we announced the advancement of two important pipeline
programs.PEG-PAL will advance as our next pivotal Phase 3 program in the
second quarter of next year and BMN-111 for achondroplasia will advance to a
Phase 2 program in mid 2013," said Jean-Jacques Bienaimé, Chief Executive
Officer of BioMarin."The next major inflection point for the company is the
readout of the GALNS pivotal Phase 3 study.We also expect to release Phase 2
results for our BMN-701 Pompe program in the first quarter of 2013 and
additional data on our BMN-673 PARP-inhibitor program before year end."

Net Product Revenue (in millions)
                                                              
            Three Months Ended September 30, Nine Months Ended September 30,
            2012  2011    $ Change  % Change 2012   2011    $ Change % Change
Naglazyme ^  $    $ 55.9  $ 6.6     11.8%    $     $ 176.8 $ 17.1   9.7%
(1)          62.5                             193.9
Kuvan ^ (2)  36.4  30.5    5.9       19.3%    103.1  86.0    17.1     19.9%
Firdapse     3.6   3.5     0.1       2.9%     10.8   9.8     1.0      10.2%
                                                              
(1) Changes in foreign currency rates, net of hedges, had a negative $0.2
million and a negative $1.4 million impact on Naglazyme sales in the three and
nine months ended September 30, 2012, compared to the same periods in 2011,
respectively.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 16.5 percent in the third quarter of 2012 compared to the third
quarter of 2011.

           Three Months Ended September    Nine Months Ended September 30,
            30,
           2012   2011   $       %       2012    2011    $        %
                            Change Change                   Change  Change
Aldurazyme
revenue
reported by $48.3  $46.3 $2.0  4.3%    $140.0 $136.4 $3.6   2.6%
Genzyme
^(3)
                                                               
                                                               
Royalties
due from    19.7   19.0   0.7           56.6    53.0    3.6     
Genzyme
Incremental
product
transfer    4.1    4.0    0.1           1.1     6.0     (4.9)   
revenues
^(4)
Total
Aldurazyme  $23.8 $23.0 $0.8         $57.7  $59.0  $(1.3) 
net product
revenues
                                                               
(3) The total number of Aldurazyme patients increased 9.5 percent in the
third quarter of 2012 as compared to the third quarter of 2011.
                                                               
(4) 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.

                                                      
2012 Guidance                                         
Revenue Guidance ($ in millions)                       
Item                                   2012 Guidance    Previous 2012 Guidance
Total BioMarin Revenues                $475 to $510     Unchanged
Total Net Product Revenues             $470 to $505     Unchanged
Naglazyme Net Product Revenue          $250 to $265     Unchanged
Kuvan Net Product Revenue              $130 to $140     Unchanged
Aldurazyme Net Product Revenue to      $81 to $87       Unchanged
BioMarin
Firdapse Net Product Revenue           $13 to $17       Unchanged
                                                      
Selected Income Statement Guidance ($                  
in millions)
Item                                   2012 Guidance    Previous 2012 Guidance
Cost of Sales (% of Total Revenue)     17% to 18%       Unchanged
Selling, General and Admin. Expense    $195 to $205     Unchanged
Research and Development Expense       $285 to $295     Unchanged
Amortization and Contingent            $19              Unchanged
Consideration
Income Tax Expense                     $(5)             $ --
GAAP Net (Loss)                        $(100) to $(110) $(105) to $(115)
Stock Compensation Expense             $50              Unchanged
Non-GAAP Adjusted EBITDA               $(5) to $5       Unchanged
                                                      
Non GAAP Net Cash Flow (Usage)         $(20) to $(30)   $(40) to $(50)
Cash Balance*                          $495 to $505     $475 to $485
                                                      
* Cash balance includes cash, cash equivalents and short and long term
investments
                                                      
Anticipated Upcoming Milestones                        
                                                      
4Q 2012: Results for Phase 3 trial for GALNS for MPS IVA
4Q 2012: Update on Phase 1/2 trial for BMN-673 for solid tumors
1Q 2013: Market authorization application filing for GALNS for MPS IVA
1Q 2013: Results for Phase 1/2 trial for BMN-701 for Pompe disease
1H 2013: IND filing for BMN-190 for LINCL (Batten disease)
2Q 2013: Initiation of Phase 3 trial for PEG-PAL for PKU
2Q 2013: Results for PKU-016 Kuvan neurocognitive study
Mid 2013: Initiation of Phase 2 trial for BMN-111 for achondroplasia
2H 2013: Potential initiation of Phase 3 trial for BMN-673 for solid tumors
4Q 2013: Potential FDA approval of GALNS for MPS IVA

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

  *GALNS for MPS IVA: The company is on track to report top-line results in
    the current quarter.Two of the three ancillary studies are fully
    enrolled: a clinical trial for patients under five years of age and a
    cardiopulmonary study to more completely document the health benefits of
    GALNS.A study in patients with limited ambulation is expected to complete
    enrollment this quarter.

Mid-Stage Clinical Programs

  *PEG-PAL for PKU:The company expects to initiate a pivotal Phase 3 study
    in the second quarter of 2013, following an anticipated end of Phase 2
    meeting with the FDA in the first quarter of 2013.The preliminary Phase 3
    design includes an open-label study to evaluate safety and blood Phe
    levels in naïve patients and a randomized controlled study of the Phase 2
    extension study patients to evaluate blood Phe levels and psychiatric and
    executive function endpoints.
    
  *Kuvan Outcomes Study: PKU-016, a randomized, placebo-controlled, 13-week
    outcomes study in patients treated with Kuvan is fully enrolled.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 the second quarter of 2013.

Early-Stage Clinical Programs

  *BMN-701 for Pompe Disease: The company has completed the enrollment of 16
    patients in the 20 mg/kg cohort.Top-line results for the Phase 1/2 trial
    are expected in the first quarter of 2013.
    
  *BMN-673 (PARP inhibitor):Over 20 patients have been dosed in the Phase
    1/2 open-label trial on once daily, orally administered BMN-673 for
    advanced or recurrent solid tumors.An update on this program is expected
    in the fourth quarter of 2012. A Phase 3 trial in advanced or recurrent
    solid tumors could start enrolling by the end of 2013.
    
  *BMN-111 for Achondroplasia: The company expects to initiate a Phase 2
    trial in patients in mid 2013.

Preclinical Programs

  *BMN-190 for LINCL (Batten disease): BioMarin expects to file the IND for
    BMN-190 in the first half 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 and nine months ended September 30, 2012 and
September 30, 2011 and financial guidance for the year ending December 31,
2012 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 income 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
metrics:

Reconciliation of GAAP Net Loss to Non-GAAP Adjusted EBITDA Income (Loss)
(in millions)
(unaudited)
                                                                 
                          Three Months Ended   Nine Months Ended   
                            September 30,       September 30,
                                                                     Year
                                                                     Ending
                   NOTES   2012       2011      2012      2011      December
                                                                     31, 2012
                                                                     Guidance
                                                               
                                                                     $(110.0)
GAAP Net Loss              $(5.4)   $(17.7) $(61.3) $(27.1) --
                                                                     $(100.0)
                                                               
Interest expense,          1.1       1.4      3.9      4.2      5.0
net
Income tax expense         (6.4)     (2.1)    (6.8)    6.6      (5.0)
(benefit)
Depreciation               6.4       6.0      21.0     18.7     30.0
Amortization               2.7       1.0      14.5     2.9      11.0
EBITDA Income              (1.6)     (11.4)   (28.7)   5.3      (69.0) -
(Loss)                                                               (59.0)
                                                               
Stock-based                12.1      11.6     35.9     32.6     50.0
compensation
Contingent          (1)    0.6       2.2      (3.3)    (2.4)    14.0
consideration
                                                               
Material                                                        
non-recurring
Convertible debt    (2)     --       1.9               1.9      
exchange
                                                               
Non-GAAP Adjusted                                                    $(5.0) --
EBITDA Income              $11.1    $4.3    $3.9    $37.4   $5.0
(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 and
uses the non-GAAP adjusted EBITDA methodology in establishing corporate goals
for internal compensation programs.

Conference Call Details

BioMarin will host a conference call and webcast to discuss third quarter 2012
financial results today, Thursday, October 25, at 5:00 p.m. ET. This event can
be accessed on the investor section of the BioMarin website at www.BMRN.com.

Date: October 25, 2012
Time: 5:00 p.m. ET
U.S. / Canada Dial-in Number: 877.303.6313
International Dial-in Number: 631.813.4734
Conference ID: 38161058

Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 38161058

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 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

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 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 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^® 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
September 30, 2012 and December 31, 2011
(In thousands of U.S. dollars, except share and per share amounts)
                                                               
                                              September 30, 2012 December 31,
                                                                  2011(1)
ASSETS                                         (unaudited)        
Current assets:                                                  
Cash and cash equivalents                      $ 181,330         $ 46,272
Short-term investments                         245,125           148,820
Accounts receivable, net (allowance for
doubtful accounts: $339 and $513,              117,290           104,839
respectively)
Inventory                                      120,825           130,118
Other current assets                           54,816            39,753
Total current assets                           719,386           469,802
Investment in BioMarin/Genzyme LLC             848               559
Long-term investments                          106,741           94,385
Property, plant and equipment, net             273,724           268,971
Intangible assets, net                         165,624           180,277
Goodwill                                       51,543            51,543
Long-term deferred tax assets                  229,771           222,649
Other assets                                   19,739            15,495
Total assets                                   $ 1,567,376       $ 1,303,681
LIABILITIES AND STOCKHOLDERS' EQUITY                             
Current liabilities:                                             
Accounts payable and accrued liabilities       $ 111,489         $ 94,125
Convertible debt                               23,440            --
Total current liabilities                      134,929           94,125
Long-term convertible debt                     324,861           348,329
Other long-term liabilities                    92,392            88,179
Total liabilities                              552,182           530,633
Stockholders' equity:                                            
Common stock, $0.001 par value: 250,000,000
shares authorized at September 30, 2012 and
December31, 2011: 123,790,150 and 114,789,732 124               115
shares issued and outstanding at September 30,
2012 and December31, 2011, respectively.
Additional paid-in capital                     1,505,776         1,197,082
Company common stock held by Nonqualified      (6,603)           (3,935)
Deferred Compensation Plan
Accumulated other comprehensive income         2,333             4,887
Accumulated deficit                            (486,436)         (425,101)
Total stockholders' equity                     1,015,194         773,048
Total liabilities and stockholders' equity     $ 1,567,376       $ 1,303,681
                                                                
                                                               
(1) December 31, 2011 balances were derived from the audited consolidated
financial statements.



BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
Three and Nine Months Ended September 30, 2012 and 2011
(In thousands of U.S. dollars, except per share amounts)
(Unaudited)
                                                              
                           Three Months Ended       Nine Months Ended
                            September 30,            September 30,
                           2012        2011         2012         2011
REVENUES:                                                      
Net product revenues        $ 126,310  $ 112,891  $ 365,540  $ 331,583
Collaborative agreement     1,210      97          1,729       375
revenues
Royalty and license         597        437         1,516       1,554
revenues
Total revenues              128,117    113,425     368,785     333,512
OPERATING EXPENSES:                                            
Cost of sales (excludes
amortization of certain     24,619     22,445      65,298      62,504
acquired intangible assets)
Research and development    66,209     58,577      217,855     156,466
Selling, general and        46,337     44,880      143,124     126,969
administrative
Intangible asset
amortization and contingent 1,443      3,040       5,819       28
consideration
Total operating expenses    138,608    128,942     432,096     345,967
LOSS FROM OPERATIONS        (10,491)   (15,517)    (63,311)    (12,455)
Equity in the loss of       (336)      (608)       (968)       (1,817)
BioMarin/Genzyme LLC
Interest income             778        722         1,819       2,302
Interest expense            (1,837)    (2,168)     (5,709)     (6,531)
Debt conversion expense     --        (1,896)     --         (1,896)
Other income and (expense)  125        (264)       (15)        (114)
LOSS BEFORE INCOME TAXES    (11,761)   (19,731)    (68,184)    (20,511)
Provision for (benefit      (6,404)    (2,078)     (6,849)     6,590
from) income taxes
NET LOSS                    $ (5,357) $ (17,653) $ (61,335) $ (27,101)
NET LOSS PER SHARE, BASIC   $ (0.04)  $ (0.16)   $ (0.52)   $ (0.24)
AND DILUTED
                                                              
Weighted average common
shares outstanding, basic   123,434     112,290      118,810      111,358
and diluted
                                                              
COMPREHENSIVE LOSS         $ (7,674) $ (10,426) $ (63,889) $ (25,985)




STOCK-BASED COMPENSATION EXPENSE
                                                               
                              Three Months Ended      Nine Months Ended
                              September 30,           September 30,
                              2012        2011        2012        2011
                              (unaudited) (unaudited) (unaudited) (unaudited)
Cost of sales                  $ 1,327     $ 1,334     $ 3,535     $ 3,864
Research and development       5,060       4,372       15,351      12,070
Selling, general and           5,752       5,912       17,021      16,673
administrative
                              $ 12,139    $ 11,618    $ 35,907    $ 32,607

CONTACT: Investors and Media
         Eugenia Shen
         BioMarin Pharmaceutical Inc.
         (415) 506-6570

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