BioMarin Announces Second Quarter 2013 Financial Results

BioMarin Announces Second Quarter 2013 Financial Results

                   Total RevenueGrowth Exceeds 10 Percent

                      Phase 3 Trial to Start in 3Q 2013

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

Financial Highlights ($ in millions, except per share data, unaudited)
                                                  Q2 2013   Q2 2012   Percent
Total BioMarin Revenue                             $136.8  $124.0  10.3%
Total BioMarin Revenue (excluding Aldurazyme Net   140.5    120.6    16.5%
Product Transfer Revenue) - non-GAAP
Naglazyme Net Product Revenue                      69.9     62.9     11.1%
Aldurazyme BioMarin Net Product Revenue           17.5     21.8     -19.7%
Aldurazyme Royalty Revenue (excluding Net Product  21.2     18.4     15.2%
Transfer Revenue) - non-GAAP
Kuvan Net Product Revenue                          40.9     34.7     17.9%
Firdapse Net Product Revenue                       4.1      3.6      13.9%
Net Loss                                          (21.5)   (32.0)   
Net Loss per Share (basic)                         $(0.15) $(0.27) 
Net Loss per Share (diluted)                       $(0.16) $(0.27) 
Adjusted EBITDA Loss - non-GAAP                    $0.0    $(7.3)  
Cash, cash equivalents and short and long-term     $524.4  $524.6  0.0%

SAN RAFAEL, Calif., July 25, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced financial results for its second quarter
ended June 30, 2013. GAAP net loss was $21.5 million ($0.16 per diluted share)
for the second quarter of 2013, compared to GAAP net loss of $32.0 million
($0.27 per diluted share) for the second quarter of 2012. GAAP net loss for
the six months ended June 30, 2013 was $61.3 million ($0.46 per diluted
share), as compared to GAAP net loss of $56.0 million ($0.48 per diluted
share) for the six months ended June 30, 2012.Non-GAAP adjusted EBITDA was
$0.0 million for the second quarter of 2013, compared to non-GAAP adjusted
EBITDA loss of $7.3 million for the second quarter of 2012.Non-GAAP adjusted
EBITDA was a loss of $8.0 million for the six months ended June 30, 2013, as
compared to a loss of $7.2 million for the six months ended June 30, 2012.The
decreased GAAP net loss and the decreased non-GAAP adjusted EBITDA loss for
the second quarter of 2013 compared to the second quarter of 2012 was
primarily due to higher total revenue and a smaller increase in operating

As of June 30, 2013, BioMarin had cash, cash equivalents and short and
long-term investments totaling $ 524.4 million, as compared to $525.7 million
on March 31, 2013.

"In the second quarter, we continued to execute on our development goals as we
head into the second half of the year," said Jean-Jacques Bienaimé, Chief
Executive Officer of BioMarin."We made good progressin a number of important
areas including commercial, R&D and regulatory.Highlights included initiation
of the Phase 3 PEG-PAL trial for PKU, confirmation of priority review status
for Vimizim in the U.S., validation of the MAA by the EMA, submission of the
Vimizim MA in Brazil and a positive update of the ongoing BMN 673 trial for
genetically-defined cancers at the ASCO annual meeting.We are pleased with
the momentum of the BMN 673 program because of maturing and improving
responses, completed positive regulatory discussions and earlier Phase 3
timelines than originally anticipated.We remain on track to meet our upcoming
goals for the remainder of the year."

Net Product
Revenue (in                                                       
             Three Months Ended June 30,      Six Months Ended June 30,
             2013     2012     $       %      2013     2012     $       %
                                Change  Change                   Change  Change
Naglazyme     $69.9  $62.9  $7.0  11.1%  $139.3 $131.5 $7.8  5.9%
Kuvan         40.9    34.7    6.2    17.9%  78.5    66.7    11.8   17.7%
Firdapse      4.1     3.6     0.5    13.9%  7.7     7.2     0.5    6.9%
Aldurazyme    17.5    21.8    (4.3)  -19.7% 34.2    33.8    0.4    1.2%
Net Product   132.4   123.0   9.4    7.6%   259.7   239.2   20.5   8.6%
agreement     0.9     0.4     0.5          1.0     0.6     0.4    
Royalty and
license       3.5     0.6     2.9          4.0     0.9     3.1    
BioMarin      136.8   124.0   12.8   10.3%  264.7   240.7   24.0   10.0%
Revenues -
Net Product   (3.7)   3.4     (7.1)        (6.3)   (3.0)   (3.3)  
Net Product
Revenue) -    $140.5 $120.6 $19.9 16.5%  $271.0 $243.7 $27.3 11.2%

Reconciliation of Aldurazyme Revenue (in millions)
            Three Months Ended June 30,       Six Months Ended June 30,
            2013    2012    $ Change  %        2013     2012   $      %
                                       Change                  Change Change
revenue      $53.6  $45.8  $7.8     17.0%    $102.1  $91.7 $10.4 11.3%
reported by
due from     21.2   18.4   2.8              40.5    36.8  3.7   
Genzyme -
transfer     (3.7)  3.4    (7.1)            (6.3)   (3.0) (3.3) 
net product  $17.5   $21.8  $(4.3)           $34.2   $33.8 $0.4  
revenues -
(1) Naglazyme revenues experience quarterly fluctuations due to the timing of
government ordering patterns in certain countries.

(2) 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 business.
By providing information about both the GAAP and non-GAAP revenue measures,
the company believes that the additional information enhances investors'
overall understanding of the company's business and in particular allows for
more consistent period to period evaluation of the revenue.

(3) 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 Full Year Financial Guidance                      
Revenue Guidance ($ in millions)                       
Item                                   2013 Guidance    Previous 2013 Guidance
Total BioMarin Revenues                $530 to $555     Unchanged
Naglazyme Net Product Revenue          $265 to $285     Unchanged
Kuvan Net Product Revenue              $155 to $170     Unchanged
Selected Income Statement Guidance ($                  
in millions)
Item                                   2013 Guidance    Previous 2013 Guidance
Cost of Sales (% of Total Revenue)     17% to 18%       Unchanged
Selling, General and Admin. Expense    $220 to $240     $220 to $250
Research and Development Expense       $340 to $380     Unchanged
GAAP Net Loss                          $(185) to $(160) $(195) to $(170)
Non-GAAP Adjusted EBITDA (loss)        $(65) to $(40)   $(75) to $(50)
Cash Balance*                          Over $440        Over $420
* Cash balance includes cash, cash equivalents and short and long term

Anticipated Upcoming Milestones

3Q 2013: Initiation of Phase 1/2 trial for BMN 190 for Batten disease
3Q 2013: Initiation of Phase 3 trial for BMN 673 for BRCA breast cancer
4Q 2013: Initiation of Phase 2/3 switching trial for BMN 701 for Pompe disease
4Q 2013: Potential CHMP opinion for Vimizim for MPA IVA
4Q 2013/1Q 2014: Initiation of Phase 1/2 trial for BMN 111 for achondroplasia
1Q 2014: PDUFA date for Vimizim for MPS IVA
1Q 2014: Potential launch of Vimizim for MPS IVA
4Q 2014: Top-line results for Phase 3 trial for PEG-PAL for PKU

Research and Development Programs

BioMarin continues to make significant progress in research and development to
ensure a strong 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.

Programs Under Regulatory Review for Approval

  *Vimizim for MPS IVA: During the second quarter, regulatory advancements
    were made worldwide.The FDA granted Vimizim priority review designation
    and assigned a PDUFA date of February 28, 2014.The EMA has validated the
    Vimizim MAA and has granted accelerated review status, which could lead to
    a CHMP opinion before year end.During the second quarter, the company
    also submitted the MA in Brazil.Ongoing production of bulk drug substance
    and drug product will allow for a global launch following approvals in
    multiple countries.Both the FDA and EMA have conducted or scheduled
    pre-approval inspections associated with the review of the marketing

Advanced Clinical Programs

  *PEG-PAL for PKU:The company initiated a pivotal Phase 3 study in the
    second quarter of 2013.The Phase 3 program 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, which is expected to be initiated in the third
    quarter of 2013.The company expects top-line results for the Phase 3
    study in the fourth quarter of 2014.
  *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 and
    the respiratory parameter MIP as the primary endpoint.The company has
    completed a full scale production campaign using its new higher producing
    cell line and has verified the production yields.Characterization testing
    is currently ongoing, and it is expected that regulatory filings to
    support the introduction of this material into clinical studies will be
    submitted in the fourth quarter of 2013.
  *BMN 673 (PARP inhibitor):As of July 24, the RECIST response rate from the
    ongoing trial is nine out of 18 breast cancer patients, or 50 percent,
    including one confirmed complete response.This response rate includes
    three additional confirmed responses since the last update at the ASCO
    Annual Meeting, and two new patients are yet to be confirmed.Four
    patients are ongoing with stable disease with potential for additional
    responses.All patients have been on treatment for at least twelve
    weeks.The study is still ongoing, and the company will provide additional
    updates later this year, including data in ovarian cancer, Ewing's sarcoma
    and small cell lung cancer.BioMarin now expects to initiate a Phase 3
    trial for BMN 673 in deleterious gBRCA mutation metastatic breast cancer
    in late third quarter of 2013, earlier than previously announced at the
    ASCO Annual Meeting in early June.

Early-Stage Clinical Programs

  *BMN 111 for Achondroplasia: The company is modifying its clinical program
    based on a request from the Food and Drug Administration (FDA) for more
    clinical pharmacokinetic and safety data in children with the
    disease.BioMarin previously completed a Phase 1 study in adult healthy
    volunteers. Although the FDA acknowledges that the company has identified
    a range of doses that are relatively well-tolerated short–term, it has now
    requested additional PK and safety data in children with achondroplasia
    before proceeding to extended dosing in children.The FDA has placed the
    BMN 111 program on partial clinical hold.The company will work with the
    appropriate health authoritieson the implications on the program.The
    company expects to initiate its first study in pediatric patients in the
    fourth quarter of 2013 or the first quarter of 2014.
  *BMN 190 for LINCL (Batten disease): BioMarin filed a CTA in the first
    quarter of 2013 and expects to begin enrolling the study in the third
    quarter of 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 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 Adjusted EBITDA Reconciliation

The results for the three and six months ended June 30, 2013 and June 30, 2012
and financial guidance for the year ending December 31, 2013 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 Loss
(in millions)

                      ThreeMonths Ended    SixMonths Ended      Ending
                        June 30,             June 30,             December
                NOTES  2013       2012       2013       2012       2013
GAAP Net Loss          $(21.5)  $(32.0)  $(61.3)  $(56.0)  -
Interest               --       1.4       1.0       2.8       0.7
expense, net
Provision for
(benefit from)         1.2       (0.4)     (3.5)     (0.4)     (14.6)
income taxes
Depreciation           6.6       7.2       12.7      14.5      27.0
Amortization           3.6       2.7       6.2       12.0      11.5
EBITDA Loss            (10.1)    (21.1)    (44.9)    (27.1)    (160.4) -
compensation           13.9      12.6      25.5      23.8      70.0
consideration    (1)   (3.8)     1.2       1.0       (3.9)     15.0
Debt conversion  (2)    --       --       10.4      --       10.4
Non-GAAP                                                            $(65.0) -
Adjusted EBITDA        $0.0     $(7.3)   $(8.0)   $(7.2)   $(40.0)
(1) Represents the expense associated with the changein 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 second quarter
2013 financial results today, Thursday, July 25, 2013 at 5:00 p.m. ET. This
event can be accessed on the investor section of the BioMarin website at

Date: July 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: 13894023

Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 13894023

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 III 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 Vimizim and
our other product candidates; and actions by regulatory authorities,
particularly actions related to Vimizim. 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 Vimizim and each of the other 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.

June 30, 2013 and December 31, 2012
(In thousands of U.S. dollars, except share and per share amounts)
                                                  June 30, 2013 December 31,
ASSETS                                             (unaudited)  
Current assets:                                                 
Cash and cash equivalents                          $175,445     $180,527
Short-term investments                             225,116      270,211
Accounts receivable, net (allowance for doubtful   115,063      109,066
accounts: $376 and $348, respectively)
Inventory                                          142,296      128,695
Current deferred tax assets                        29,474       29,454
Other current assets                               33,526       25,509
Total current assets                              720,920      743,462
Noncurrent assets:                                              
Investment in BioMarin/Genzyme LLC                 1,001        1,080
Long-term investments                              123,905      115,993
Property, plant and equipment, net                 282,002      284,473
Intangible assets, net                             168,434      162,980
Goodwill                                           54,975       51,543
Long-term deferred tax assets                      235,400      225,501
Other assets                                       14,883       16,611
Total assets                                      $1,601,520   $1,601,643
LIABILITIES AND STOCKHOLDERS' EQUITY                            
Current liabilities:                                            
Accounts payable and accrued liabilities           $142,241     $147,068
Convertible debt                                   --          23,365
Total current liabilities                          142,241      170,433
Noncurrent liabilities:                                         
Long-term convertible debt                         109,822      324,859
Long-term contingent acquisition consideration     23,261       30,618
Long-term deferred tax liabilities                 37,182       33,296
Other long-term liabilities                        30,756       26,674
Total liabilities                                  343,262      585,880
Stockholders' equity:                                           
Common stock, $0.001 par value: 250,000,000 shares
authorized at June 30, 2013 and December 31, 2012;
140,050,009 and 125,809,162 shares issued and      140          126
outstanding atJune 30, 2013 and December 31,
2012, respectively.
Additional paid-in capital                         1,863,961    1,561,890
Company common stock held by Nonqualified Deferred (7,493)      (6,603)
Compensation Plan
Accumulated other comprehensive income (loss)      2,441        (202)
Accumulated deficit                                (600,791)    (539,448)
Total stockholders' equity                         1,258,258    1,015,763
Total liabilities and stockholders' equity         $1,601,520   $1,601,643
(1) December 31, 2012 balances were derived from the audited consolidated
financial statements.


Three and Six Months Ended June 30, 2013 and 2012
(In thousands of U.S. dollars, except per share amounts)
                                  Three Months Ended    SixMonths Ended
                                   June 30,              June 30,
                                  2013       2012       2013       2012
Net product revenues               $132,400  $122,991  $259,744  $239,230
Collaborative agreement revenues   889       423       1,024     519
Royalty and license revenues       3,521     605       3,970     919
Total revenues                     136,810   124,019   264,738   240,668
OPERATING EXPENSES:                                              
Cost of sales (excludes
amortization of certain acquired   22,567    23,574    43,067    40,679
intangible assets)
Research and development           85,661    77,812    169,404   151,646
Selling, general and               50,656    51,539    101,706   96,787
Intangible asset amortization and  (2,022)   2,048     3,534     4,376
contingent consideration
Total operating expenses           156,862   154,973   317,711   293,488
LOSS FROM OPERATIONS               (20,052)  (30,954)  (52,973)  (52,820)
Equity in the income (loss) of     (163)     102       (564)     (632)
BioMarin/Genzyme LLC
Interest income                    650       536       1,368     1,041
Interest expense                   (603)     (1,925)   (2,328)   (3,872)
Debt conversion expense            --       --       (10,420)  --
Other income (expense)             (123)     (176)     105       (140)
LOSS BEFORE INCOME TAXES           (20,291)  (32,417)  (64,812)  (56,423)
Provision for (benefit from)       1,242     (411)     (3,469)   (445)
income taxes
NET LOSS                           $(21,533) $(32,006) $(61,343)  $(55,978)
NET LOSS PER SHARE, BASIC          $(0.15)   $(0.27)   $(0.46)    $(0.48)
NET LOSS PER SHARE, DILUTED        $(0.16)   $(0.27)   $(0.46)   $(0.48)
Weighted average common shares     139,400    117,912    133,716    116,496
outstanding, basic
Weighted average common shares     139,596    117,912    133,716    116,496
outstanding, diluted
COMPREHENSIVE LOSS                $(20,247) $(29,868) $(58,700) $(56,215)

Total stock-based compensation expense included in the Condensed Consolidated
Statements of Comprehensive Loss is as follows:
                      ThreeMonthsEnded          SixMonthsEnded
                      June 30,                   June 30,
                      2013          2012          2013          2012
                      (unaudited)   (unaudited)   (unaudited)   (unaudited)
Cost of sales          $1,130        $1,335        $2,174        $2,207
Research and           6,381         5,468         11,705        10,290
Selling, general and   6,418         5,834         11,615        11,268
                      $13,929       $12,637       $25,494       $23,765

CONTACT: Investors and Media:
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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