BioMarin Announces First Quarter 2013 Financial Results

BioMarin Announces First Quarter 2013 Financial Results

 Total Revenue Continues Steady Growth of 10 Percent; Progress on All Aspects
                               of the Business

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

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

                                           Q1 2013   Q1 2012   Percent Change
Total BioMarin Revenue                      $127.9  $116.6  9.7%
Total Net Product Revenue                   127.3    116.2    9.6%
Naglayzme Net Product Revenue               69.4     68.6     1.2%
Aldurazyme BioMarin Net Product Revenue     16.7     12.0     39.2%
Kuvan Net Product Revenue                   37.6     32.0     17.5%
Firdapse Net Product Revenue                3.6      3.6      0.0%
GAAP Net Loss                               (39.8)   (24.0)   
GAAP Net Loss per Share (basic and diluted) $(0.31) $(0.21) 
Non-GAAP Adjusted EBITDA Loss               $(8.0)  $(0.1)  
Cash, cash equivalents and short and        $525.7  $287.7  82.7%
long-term investments

SAN RAFAEL, Calif., April 25, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced financial results for its first quarter
ended March 31, 2013. GAAP net loss was $39.8 million ($0.31 per share) for
the first quarter of 2013, compared to GAAP net loss of $24.0 million ($0.21
per share) for the first quarter of 2012.Non-GAAP adjusted EBITDA was a loss
of $8.0 million for the first quarter of 2013, compared to non-GAAP adjusted
EBITDA loss of $0.1 million for the first quarter of 2012.The increased GAAP
net loss and the increased non-GAAP adjusted EBITDA loss for the first quarter
of 2013 compared to the first quarter of 2012 was primarily due to increased
net product revenue offset by a larger increase in research and development

As of March 31, 2013, BioMarin had cash, cash equivalents and short and
long-term investments totaling $525.7 million, as compared to $566.7 million
on December 31, 2012.

"In the first quarter, we continued to execute on our development goals as we
head into another potentially transformative year for the company," said
Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin."We made good
progress on a number of important fronts including commercial, R&D, regulatory
and business development.Highlights included positive results for the Phase
1/2 trial for BMN-701for Pompe disease, submission of the BLA for Vimizim in
the U.S. and the MAA for Vimizim in the EU, the acquisition of Zacharon
Pharmaceuticals and the licensing of the Factor VIII program for Hemophilia
A.We hit all of our stated milestones for the quarter and remain on track to
meet our upcoming goals for the remainder of the year."

              ThreeMonthsEnded March 31,
              2013    2012 $ Change % Change
Naglazyme ^(1) $69.4 68.6 $0.8   1.2%
Kuvan         37.6    32.0 5.6      17.5%
Firdapse      3.6     3.6  0.0      0.0%

(1) Naglazyme revenues experience quarterly fluctuations due to the timing of
government ordering patterns in certain countries.There were no abnormal
ordering patterns in the first quarter of 2013.In the first quarter of 2012,
there was a delayed order from the fourth quarter of 2011.There was a 10.2
percent increase in Naglazyme revenue in the first quarter of 2013 over the
fourth quarter of 2012 and an 11.0 percent increase in patients on therapy as
compared to the first quarter of 2012, which is consistent with that of
previous quarters.

                                          Three Months Ended March 31,
                                          2013    2012    $ Change % Change
Aldurazyme revenue reported by Genzyme     $48.4 $45.9 $2.5   5.4%
Royalties due from Genzyme                 19.3   18.4   0.9     
Incremental product transfer revenues ^(2) (2.6)  (6.4)  3.8     
Total Aldurazyme net product revenues      $16.7 $12.0 $4.7   

(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.

BioMarin Reaffirms 2013 Full Year Financial 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 licensing of the Factor VIII program for
Hemophilia A from the University College London and St. Jude Children's
Research Hospital

** Cash balance includes cash, cash equivalents and short and long term

Anticipated Upcoming Milestones

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
Mid 2013: Initiation of Phase 1/2 trial for BMN-190 for Batten disease
4Q 2013: Initiation of Phase 3 trial for BMN-673
4Q 2013: Potential FDA approval of Vimizim for MPS IVA
4Q 2013: Initiation of Phase 2/3 switching trial 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 submitted the BLA for Vimizim at the end
    of the first quarter of 2013 and the market authorization application
    filing in the EU earlier this week.Vimizim has received accelerated
    review status from the EMA.

Mid-Stage Clinical Programs

  *PEG-PAL for PKU:The company plans to initiate a pivotal Phase 3 study in
    the second quarter of 2013.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 and
    patients from the open label trial 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

Early-Stage Clinical Programs

  *BMN-701 for Pompe Disease: The company plans to initiate a Phase 2/3
    switching trial by the end of 2013 in late-onset Pompe patients who have
    previously been treated with alglucosidase alfa.Subject to discussions
    with health authorities, the proposed study design is a single arm trial,
    with treatment at 20 mg/kg administered every other week for 24 weeks.The
    company intends to use the respiratory parameter MIP as the primary
    endpoint.Secondary objectives include MEP and six-minute walk test, as
    well as safety.The study will be conducted with full scale material from
    a revised manufacturing process, which has improved process robustness and
    increased productivity.
  *BMN-673 (PARP inhibitor):An update on the Phase 1/2 study in solid tumors
    is expected at the ASCO Annual Meeting in June 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 filed a CTA in the first
    quarter of 2013 and expects to begin enrolling the study in mid
    2013.Orphan drug designation has been granted in both the U.S. and EU.

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 has an ongoing 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 ended March 31, 2013 and March 31, 2012 and
financial guidance for the year ending December 31, 2013are 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 Loss
(in millions)
                                     Three Months Ended         Year Ending
                                   March 31,                 December 31,
                          NOTES     2013          2012         Guidance
GAAP Net Loss                       $(39.8)     $(24.0)    $(195.0) -
Interest expense, net               1.0          1.4         0.7
Benefit from Income taxes          (4.7)        --         (14.6)
Depreciation expense                6.1          7.3         25.0
Amortization expense                2.6          9.3         10.5
EBITDA Loss                         (34.8)       (6.0)       (173.4) -
Stock-based compensation            11.6         11.1        70.0
Contingent consideration   (1)      4.8          (5.2)       18.0
Material non-recurring:                                      
Debt conversion expense    (2)       10.4         --         10.4
Non-GAAP Adjusted EBITDA            $(8.0)      $(0.1)     $(75.0) -
Loss                                                            $(50.0)
(1) Represents the expense 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 2017 convertible notes in March 2013.

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 first quarter 2013
financial results today, Thursday, April 25, 2013 at 5:00 p.m. ET. This event
can be accessed on the investor section of the BioMarin website at

Date: April 25, 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: 29712683

Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 29712683

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.

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; our expectations regarding the progress and 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; our
expectations regarding the timing of our regulatory filings for our 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 risks that are discussed in BioMarin's filings with the
Securities and Exchange Commission, including, without limitation, BioMarin's
2012 Annual Report on Form 10-K, and our periodic reports on Form 10-Q and
Form 8-K. 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.

Vimizim™ is our trademark, and BioMarin^®, Naglazyme^®, Kuvan^®, Firdapse^®
are registered trademarks of BioMarin Pharmaceutical Inc.

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

March 31, 2013 and December 31, 2012
(In thousands of U.S. dollars, except share and per share amounts)
                                          March 31, 2013 December 31, 2012(1)
ASSETS                                     (unaudited)   
Current assets:                                          
Cash and cash equivalents                  $154,380     $180,527
Short-term investments                     247,502       270,211
Accounts receivable, net (allowance for
doubtful accounts: $376 and $348,          120,345       109,066
Inventory                                  135,822       128,695
Current deferred tax assets                29,474        29,454
Other current assets                       35,501        25,509
Total current assets                      723,024       743,462
Noncurrent assets:                                       
Investment in BioMarin/Genzyme LLC         679           1,080
Long-term investments                      123,819       115,993
Property, plant and equipment, net         281,865       284,473
Intangible assets, net                     172,016       162,980
Goodwill                                   54,975        51,543
Long-term deferred tax assets              226,757       225,501
Other assets                               15,158        16,611
Total assets                               $1,598,293   $1,601,643
Current liabilities:                                     
Accounts payable and accrued liabilities   $144,008     $147,068
Convertible debt                           --           23,365
Total current liabilities                  144,008       170,433
Noncurrent liabilities:                                  
Long-term convertible debt                 109,849       324,859
Long-term contingent acquisition           27,224        30,618
consideration payable
Long-term deferred tax liabilities         37,521        33,296
Other long-term liabilities                29,539        26,674
Total liabilities                          348,141       585,880
Stockholders' equity:                                    
Common stock, $0.001 par value:
250,000,000 shares authorized at March 31,
2013 and December 31, 2012;                139           126
138,873,207and 125,809,162 shares issued
and outstanding at March 31, 2013 and
December 31, 2012, respectively.
Additional paid-in capital                 1,833,831     1,561,890
Company common stock held by Nonqualified  (5,715)       (6,603)
Deferred Compensation Plan
Accumulated other comprehensive income     1,155         (202)
Accumulated deficit                        (579,258)     (539,448)
Total stockholders' equity                 1,250,152     1,015,763
Total liabilities and stockholders' equity $1,598,293   $1,601,643

(1) December 31, 2012 balances were derived from the audited consolidated
financial statements.

Three Months Ended March 31, 2013 and 2012
(In thousands of U.S. dollars, except per share amounts)
                                                      2013       2012
Net product revenues                                   $127,344  $116,239
Collaborative agreement revenues                       135        96
Royalty and license revenues                           449        314
Total revenues                                         127,928    116,649
OPERATING EXPENSES:                                               
Cost of sales (excludes amortization of certain        20,500     17,105
acquired intangible assets)
Research and development                               83,743     73,834
Selling, general and administrative                    51,050     45,248
Intangible asset amortization and contingent           5,556      2,328
Total operating expenses                               160,849    138,515
LOSS FROM OPERATIONS                                   (32,921)   (21,866)
Equity in the loss of BioMarin/Genzyme LLC             (401)      (734)
Interest income                                        718        505
Interest expense                                       (1,725)    (1,947)
Debt conversion expense                                (10,420)   --
Other income                                           228        36
LOSS BEFORE INCOME TAXES                               (44,521)   (24,006)
Provision for income taxes                             (4,711)    (34)
NET LOSS                                               $(39,810) $(23,972)
NET LOSS PER SHARE, BASIC AND DILUTED                 $(0.31)   $(0.21)
Weighted average common shares outstanding, basic and  127,969     115,070
COMPREHENSIVE LOSS                                    $(38,452) $(26,347)

Total stock-based compensation expense included in the Condensed Consolidated
Statements of Comprehensive Loss is as follows:
                                   March 31,
                                   2013                  2012
                                   (unaudited)           (unaudited)
Cost of sales                       $1,044              $873
Research and development            5,324                 4,695
Selling, general and administrative 5,197                 5,566
                                   $11,565             $11,134

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

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