BioMarin Announces Second Quarter 2009 Financial Results

Net Product Revenue Increase of 35% Drives Profitable Quarter

NOVATO, Calif., July 30 /PRNewswire-FirstCall/ --

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

Item Q2 2009 Q2 2008 Comparison

Total BioMarin Revenue $82.8 29% increase
Total Net Product Revenue $81.5 35% increase
Naglazyme Net Product Revenue $42.9 22% increase
Aldurazyme Net Sales by Genzyme $39.2 1% increase
Aldurazyme BioMarin Net Product
Revenue $21.6 62% increase
Kuvan Net Product Revenue $16.9 41% increase
GAAP Net Income $1.3 $3.8
GAAP Net Income per share $0.01 (basic $0.04 (basic and
and diluted) diluted)
Non-GAAP Net Income $9.0 $9.7
Non-GAAP Net Income per share $0.09 (basic $0.09 (diluted),
and diluted) $0.10 (basic)

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced financial results
for the second quarter ended June 30, 2009. GAAP net income was $1.3 million
($0.01 per diluted share) for the second quarter of 2009, compared to GAAP net
income of $3.8 million ($0.04 per diluted share) for the second quarter of
2008. Non-GAAP net income was $9.0 million ($0.09 per diluted share) for the
second quarter of 2009, compared to non-GAAP net income of $9.7 million ($0.09
per diluted share) for the second quarter of 2008. Non-GAAP net income/loss
excludes non-cash stock compensation expense, certain nonrecurring material
items and the tax effect of the adjustments.

GAAP net loss for the six months ended June 30, 2009 was $11.8 million ($0.12
per diluted share), compared to GAAP net income of $5.5 million ($0.05 per
diluted share) for the six months ended June 30, 2008. Non-GAAP net income
was $18.4 million ($0.18 per diluted share) for the six months ended June 30,
2009, compared to non-GAAP income of $13.8 million ($0.13 per diluted share)
for the six months ended June 30, 2008.

As of June 30, 2009, BioMarin had cash and short and long-term investments
totaling $485.3 million. During the quarter, BioMarin made the final payment
to Medicis of $70.6 million related to the Orapred transaction.

"During the quarter, we reported encouraging results from the Phase I PEG-PAL
study, initiated the Phase I/II study for GALNS for MPS IVA and recently
completed enrollment for this study, a noteworthy milestone for this program.
On the commercial front, we announced earlier today the issuance of patents
covering stable tablet formulation and the once daily dosing regimen for
Kuvan, which we believe will be significant in extending patent protection an
additional ten years beyond orphan drug market exclusivity. In addition,
yesterday, we submitted the Kuvan NDS to Health Canada. With priority review
status, we anticipate marketing approval in the first half of 2010. Also,
during the quarter, we received approval for Naglazyme in Russia, which is
especially significant as Russia influences many countries in Eastern Europe.
In order to support the projected commercial needs for Naglazyme, Aldurazyme,
GALNS and PEG-PAL through at least 2016, we are making significant investments
to double our manufacturing capacity," said Jean-Jacques Bienaime, Chief
Executive Officer of BioMarin. "Based on our performance to date, we feel
confident in meeting our overall top and bottom line financial objectives for
2009 and have narrowed the guidance range on a few items to reflect increased
visibility into the year. Our commercial products are performing well, and we
continue to carefully manage expenses. We are also carefully evaluating both
internal pipeline programs and external product opportunities to maximize
long-term value for both the company and our shareholders."

Net Product Revenue

Net product revenue from Naglazyme (galsulfase), an enzyme replacement therapy
for mucopolysaccharidosis VI (MPS VI), was $42.9 million for the second
quarter of 2009, an increase of 22.3 percent compared to Naglazyme net product
revenue of $35.1 million for the second quarter of 2008. Net product revenue
from Naglazyme for the six months ended June 30, 2009 was $82.3 million, an
increase of 31.0 percent from net product revenue of $62.8 million for the six
months ended June 30, 2008. Changes in foreign currency rates, net of hedges
caused a negative impact to Naglazyme sales of $1.7 million and $3.7 million
in the three and six months ended June 30, 2009, respectively.

Net sales of Aldurazyme (laronidase), an enzyme replacement therapy for
mucopolysaccharidosis I (MPS I) recorded by Genzyme, were $39.2 million for
the second quarter of 2009, an increase of 1.0 percent compared to net sales
by Genzyme of $38.7 million for the second quarter of 2008. Net sales of
Aldurazyme recorded by Genzyme for the six months ended June 30, 2009 were
$76.0 million, compared to net sales of $75.5 million for the six months ended
June 30, 2008. Changes in foreign currency rates caused a negative impact to
Aldurazyme sales by Genzyme of $3.6 million and $7.2 million in the three and
six months ended June 30, 2009, respectively. However, in the second quarter
of 2009, Aldurazyme unit volume increased 9.8 percent compared to the second
quarter of 2008 as the number of patients on therapy worldwide continues to
grow.

Net product revenue to BioMarin related to Aldurazyme was $21.6 million for
the second quarter of 2009, including $6.1 million of incremental product
transfer revenue. This compares to net product revenue to BioMarin of $13.4
million for the second quarter of 2008. During the second quarter of 2009,
BioMarin recorded net product revenue that was higher than the royalty earned
on Genzyme third party sales due to the incremental product transfer revenue
related to net increases in Genzyme Aldurazyme inventory levels during the
period.

Net product revenue from Kuvan (sapropterin dihydrochloride) Tablets, a
product for the treatment of phenylketonuria (PKU), was $16.9 million for the
second quarter of 2009, compared to $12.0 million for the second quarter of
2008. In the second quarter of 2009, net product revenue from Kuvan was
negatively impacted by $0.7 million due to increased and retroactive federal
rebates. Net product revenue from Kuvan for the six months ended June 30,
2009 was $32.5 million, compared to net revenue of $17.8 million for the six
months ended June 30, 2008. The quantity of commercial tablets dispensed to
patients in the U.S., the best metric to track true patient demand, increased
14.8 percent in the second quarter of 2009 compared to the first quarter of
2009.

2009 Guidance

Revenue Guidance ($ in millions)

Item 2009 Guidance Previous 2009 Guidance
Total BioMarin Revenues $311 to $336 $307 to $336
Total Net Product Revenues $304 to $329 $300 to $329
Naglazyme Net Product
Revenue $165 to $175 $160 to $175
Kuvan Net Product Revenue Unchanged $70 to $80
Aldurazyme Net Product
Revenue to BioMarin Unchanged $69 to $74

Selected Income Statement Guidance ($ in millions)

Item Guidance Previous 2009 Guidance
Cost of Sales (% of
Total Revenue) Unchanged 19% to 21%
Selling, General
and Admin. Expense Unchanged $120 to $130
Research and Development
Expense* Unchanged $118 to $128
Interest Income Unchanged $5 to $7
Impairment Loss on La Jolla
and Summit
Investments** $5.9 $7.9
GAAP Net Income (Loss) $(12) to $(6) $(15) to $0
Stock Compensation Expense $34 $32 to $35
Non-GAAP Net Income*** $35.4 to $41.4 $33.7 to $51.7

* Includes upfront research and development expenses of $8.8 million
associated with the La Jolla Pharmaceutical Company transaction.
** Represents impairment losses on investments in La Jolla Pharmaceutical
Company of $4.5 million and Summit plc of $1.4 million during the
first quarter of 2009.
*** Non-GAAP net income excludes non-cash stock compensation expense,
nonrecurring material items and the tax effect of the adjustments.
Please see the table provided at the end of this press release for a
full reconciliation between GAAP and non-GAAP expected net income.

Non-GAAP Financial Information and Reconciliation

The above results for the quarter and six months ended June 30, 2009 and 2008,
full year results for 2008 and financial guidance for 2009 are presented both
as determined in accordance with GAAP and on a non-GAAP basis. As used in
this release, non-GAAP income is calculated in accordance with GAAP, but
excludes non-cash stock compensation expense, certain nonrecurring material
items and the tax effect of the adjustments.

Non-GAAP net income in the second quarter of 2009 and the second quarter of
2008 excluded (1) stock compensation expense of $9.0 million in the second
quarter of 2009 and $5.9 million in the second quarter of 2008; (2) gain on
the sale of equity investments of $1.6 million in the second quarter of 2009
and (3) income tax effect of $0.3 million in the second quarter of 2009.
Non-GAAP net income in the six months ended June 30, 2009 and the six months
ended June 30, 2008 excluded (1) stock compensation expense of $16.8 million
in the six months ended June 30, 2009 and $10.4 million in the six months
ended June 30, 2008; (2) upfront license fees of $8.8 million in the six
months ended June 30, 2009; (3) impairment charges of $5.9 million in the six
months ended June 30, 2009; (4) Aldurazyme transfer revenue of $2.3 million in
the six months ended June 30, 2008; (5) gain on the sale of equity investments
of $1.6 million in the six months ended June 30, 2009 and (6) income tax
effect of $0.3 million and $0.2 million in the six months ended June 30, 2009
and 2008, respectively.

Anticipated non-GAAP net income for the year ended December 31, 2009 and
actual results for the year ended December 31, 2008 exclude (1) stock
compensation expense $34 million for 2009 and $25.3 million for 2008; (2)
upfront license fees of $8.8 million associated with the Riquent transaction
million in 2009 and $1.4 million associated with the Summit transaction in
2008; (3) impairment charges of $5.9 million in 2009 and $4.1 million in 2008;
(4) Kuvan approval milestones of $31.5 million in 2008; (5) the gross margin
of the initial Aldurazyme product transfer to Genzyme of $2.3 million
associated with the restructuring of BioMarin/Genzyme LLC in the first quarter
of 2008; (6) gain on the sale of equity investments of $1.6 million in 2009
and (7) income tax effect of $0.3 million and $2.2 million in 2009 and 2008,
respectively. The reconciliation of these measures to the estimated GAAP net
income is detailed in the table provided at the end of the press release.

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 and Aldurazyme 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.

Research and Development Programs

BioMarin continues to make significant investments in research and development
to ensure continued growth of the company. The current pipeline includes
programs which are in various stages of development and are focused on
treating a range of unmet medical needs. BioMarin is making significant
investments in manufacturing and laboratory facilities to support the
advancement of these programs.

-- PEG-PAL for PKU: BioMarin reported results from the Phase I trial in
early June. The FDA has accepted the design of the Phase II protocol,
and the company expects to initiate the study in the third quarter of
2009, pending IRB approval from the clinical trial sites. Results
from
the Phase II PEG-PAL trial are expected in mid-2010.
-- GALNS for MPS IVA: BioMarin initiated the Phase I/II trial in
mid-April
2009 and completed enrollment in mid-July. The Phase I/II study is an
open-label, within-patient dose escalation trial followed by a
treatment
continuation phase. The company expects to report initial results in
the first half of 2010. Assuming positive results from the Phase I/II
study, BioMarin expects to initiate a pivotal Phase III study in the
second half of 2010.
-- 6R-BH4 for Pulmonary Arterial Hypertension (PAH): The
investigator-sponsored Phase Ib multi-center, open-label
dose-escalation
study in PAH showed that the drug was well-tolerated and improved
six-minute walk distance in patients compared to their pre-treatment
baseline levels. BioMarin expects to communicate a decision on the
future of the 6R-BH4 PAH program in the second half of 2009.
-- BMN-195 - Utrophin upregulator for Duchenne Muscular Dystrophy:
BioMarin
is completing formulation work and toxicology studies and expects to
initiate a Phase I trial by the first quarter of 2010. BMN-195 is an
orally available small molecule which may upregulate utrophin, a
potential replacement for the missing dystrophin protein in DMD
patients.
-- BMN-103 - alpha-glucosidase (GAA) for Pompe Disease: BMN-103 is a
highly
phosphorylated GAA enzyme, which the company believes could result in
more efficient uptake in cells and potentially lead to improved
glycogen
reduction in key affected muscle groups not addressed with current
therapy. BioMarin continues to explore partnering options for this
program.
-- BMN-185 - IgA protease for IgA nephropathy: BioMarin is completing
early
preclinical work and expects to make a decision on the continuation of
the program by the end of 2009. IgA proteases have been shown to
cleave
IgA complexes, the deposition of which causes IgA nephropathy, an
orphan
kidney disorder with few treatment alternatives.
-- Kuvan lifecycle development: Several programs are underway to expand
and protect the market, and improve the ability of healthcare
providers
and patients to better manage their disease. These programs include a
ProDrug form of BH4, as well as a state-of-the-art handheld device to
measure blood Phe levels in PKU patients. Human studies for each of
these are planned for 2010. Regulatory approval and commercial
availability of the handheld blood Phe monitor is expected in the
first
half of 2011.

-- Additional early development candidates: BioMarin is working on
multiple early development opportunities and expects that at least one
new program will be announced by the first quarter of 2010.

Anticipated Upcoming Milestones

3Q09: Initiation of PEG-PAL Phase II trial

3Q09: International Congress of Inborn Errors of Metabolism (ICIEM)
meeting - Data on first cohort of patients from trial evaluating the
impact of Kuvan on executive function

2H09: Decision on 6R-BH4 PAH program

1Q10: Initiation of Phase I trial for BMN-195 for DMD

1Q10: American College of Medical Genetics (ACMG) Meeting - Possible
data from study of Kuvan in institutionalized PKU patients

1H10: Results from Phase I/II trial for GALNS for MPS IVA

Mid-2010: Results from PEG-PAL Phase II trial

Mid-2010: IND filing for existing preclinical candidate

2H10: Initiation of pivotal Phase III trial for GALNS for MPS IVA

1H11: Availability of blood Phe monitor

Conference Call Details

BioMarin will host a conference call and webcast to discuss second quarter
2009 financial results today, Thursday, July 30, at 5:00 p.m. ET (22:00 CET).
This event can be accessed on the investor section of the BioMarin website at
www.BMRN.com.

Date: July 30, 2009
Time: 5:00 p.m. ET (22:00 CET)
U.S. / Canada Dial-in Number: 866.272.9941
International Dial-in Number: 617.213.8895
Participant Code: 45596036
Replay Dial-in Number: 888.286.8010
Replay International Dial-in Number: 617.801.6888
Replay Code: 74141589

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious
diseases and medical conditions. The company's product portfolio comprises
three approved products and multiple clinical and pre-clinical product
candidates. Approved products include Naglazyme(R) (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin; Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a
50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin
dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership
with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other
product candidates include PEG-PAL (PEGylated recombinant phenylalanine
ammonia lyase), which is currently in development for the treatment of PKU and
GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II
clinical development for the treatment of MPS IVA. For additional information,
please visit www.BMRN.com. 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, and Aldurazyme; the financial performance of the BioMarin as a whole;
the timing of BioMarin's clinical trials of PEG-PAL, GALNS and other product
candidates; the continued clinical development and commercialization of
Aldurazyme, Naglazyme, Kuvan, 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 and Kuvan; Genzyme
Corporation's success in continuing the commercialization of Aldurazyme;
results and timing of current and planned preclinical studies and clinical
trials; 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 and Kuvan;
actual sales of Aldurazyme, Naglazyme and Kuvan; 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 2008 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(R), Naglazyme(R) and Kuvan(R) are registered trademarks of BioMarin
Pharmaceutical Inc.

Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.

Contact:

Investors Media
Eugenia Shen Susan Berg
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 506-6570 (415) 506-6594

BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
CONSOLIDATED BALANCE SHEETS
(In thousands, except for share and per share data)

December 31, June 30,
2008 (1) 2009
-------- ----
(unaudited)
ASSETS
Current assets:
Cash and cash equivalents $222,900 $200,050
Short-term investments 336,892 146,341
Accounts receivable, net 54,298 72,576
Inventory 73,162 72,836
Other current assets 50,444 15,178
------ ------
Total current assets 737,696 506,981
Investment in BioMarin/Genzyme LLC 915 462
Long-term investments 1,633 138,863
Property, plant and equipment, net 124,979 159,789
Intangible assets, net 7,626 4,391
Goodwill 21,262 21,262
Other assets 12,584 12,689
------ ------
Total assets $906,695 $844,437
======== ========

LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable and accrued liabilities $59,033 $59,104
Acquisition obligation, net of discount 70,741 -
Deferred revenue 307 929
--- ---
Total current liabilities 130,081 60,033
Convertible debt 497,083 497,083
Other long-term liabilities 2,856 3,887
----- -----
Total liabilities 630,020 561,003
------- -------

Stockholders' equity:
Common stock, $0.001 par value: 250,000,000
shares authorized at December 31, 2008 and
June 30, 2009; 99,868,145 and 100,235,218
shares issued and outstanding at December
31, 2008 and June 30, 2009, respectively 100 100
Additional paid-in capital 852,947 873,378
Company common stock held by deferred
compensation plan (882) (1,708)
Accumulated other comprehensive income 1,106 101
Accumulated deficit (576,596) (588,437)
-------- --------
Total stockholders' equity 276,675 283,434
------- -------
Total liabilities and stockholders' equity $906,695 $844,437
======== ========

(1) December 31, 2008 balances were derived from the audited consolidated
financial statements.

BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
CONSOLIDATED STATEMENTS OF OPERATIONS
For the Three and Six Months Ended June 30, 2008 and 2009
(In thousands, except for per share data, unaudited)

Three Months Ended Six Months Ended
June 30, June 30,
-------- --------
2008 2009 2008 2009
---- ---- ---- ----
Revenues:
Net product
revenues $60,458 $81,472 $118,083 $153,386
Collaborative
agreement
revenues 2,509 868 4,975 1,377
Royalty and license
revenues 1,207 447 1,513 2,004
----- --- ----- -----

Total revenues 64,174 82,787 124,571 156,767
------ ------ ------- -------

Operating expenses:
Cost of sales 9,593 19,848 26,781 34,210
Research and
development 23,755 26,324 41,383 60,682
Selling, general and
administrative 25,203 30,527 48,872 59,095
Amortization of
acquired intangible
assets 1,093 1,775 2,185 2,868
----- ----- ----- -----

Total operating
expenses 59,644 78,474 119,221 156,855
------ ------ ------- -------

Income from operations 4,530 4,313 5,350 (88)
Equity in the loss of
BioMarin/Genzyme LLC (587) (546) (1,120) (1,093)
Interest income 4,101 886 9,750 3,039
Interest expense (4,081) (4,404) (8,193) (8,496)
Impairment loss on
equity investments - - - (5,848)
Net gain from sale of
investments - 1,585 - 1,585
--- ----- --- -----

Income (Loss) before
income taxes 3,963 1,834 5,787 (10,901)
Provision for income
taxes 153 522 291 939
--- --- --- ---
Net income (loss) $3,810 $1,312 $5,496 $(11,840)
====== ====== ====== ========
Net income (loss) per
share, basic $0.04 $0.01 $0.06 $(0.12)
===== ===== ===== =======
Net income (loss) per
share, diluted $0.04 $0.01 $0.05 $(0.12)
===== ===== ===== =======
Weighted average common
shares outstanding,
basic 98,923 100,065 98,285 99,984
====== ======= ====== ======
Weighted average common
shares outstanding,
diluted 104,120 101,217 103,948 100,075
======= ======= ======= =======

Three Months Ended Six Months Ended
June 30, June 30,
-------- --------
2008 2009 2008 2009
---- ---- ---- ----
Cost of sales $392 $1,423 $589 $1,987
Research and
development expense 2,059 2,605 3,617 5,080
Selling, general and
administrative
expense 3,497 4,986 6,206 9,743
----- ----- ----- -----
Total stock-based
compensation
expense $5,948 $9,014 $10,412 $16,810
====== ====== ======= =======

Reconciliation of GAAP Net Income (Loss) to Non-GAAP Net Income
(In millions, except per share data)
(Unaudited)

Three Months Six Months Year
Ended Ended Ended
June 30, June 30, December 31,
-------- -------- ------------
Notes: 2008 2009 2008 2009 2008 2009
------ ---- ---- ---- ---- ---- ----
(forecast)
GAAP Net Income
(Loss) $3.8 $1.3 $5.5 $(11.8) $30.8 $(12.0) to (6.0)
Stock-based
compensation
expense 5.9 9.0 10.4 16.8 25.3 34.0
Upfront
license
fees (1) - - - 8.8 1.4 8.8
Impairment
charges (2) - - - 5.9 4.1 5.9
Kuvan
Approval
Milestones (3) - - - - (31.5) -
Aldurazyme
Transfer
revenue (4) - - (2.3) - (2.3) -
Net gain on
the sale of
equity
investments - (1.6) - (1.6) - (1.6)
Income tax
effect (5) - 0.3 0.2 0.3 2.2 0.3
--- --- --- --- --- ---
Non-GAAP
net income $9.7 $9.0 $13.8 $18.4 $30.0 $35.4 to 41.4

Notes:
(1) Represents upfront license payments related to our collaboration
agreements with Summit Corporation plc and La Jolla Pharmaceutical
Company in 2008 and 2009, respectively.

(2) Includes impairment losses on investments in Summit plc. during the
fourth quarter of 2008 and the first quarter of 2009, and La Jolla
Pharmaceutical Company during the first quarter of 2009.

(3) Represents approval milestones earned in July 2008 of $1.5 million
for the Japanese approval of Kuvan and in December 2008 of $30.0
million for the EMEA approval of Kuvan.

(4) Represents gross margin associated with the initial Aldurazyme
product transfer to Genzyme of $2.3 million associated with the
restructuring of BioMarin/Genzyme LLC in the first quarter of 2008.

(5) Represents the tax effect of the adjustments.

SOURCE BioMarin Pharmaceutical Inc.

Investors, Eugenia Shen, +1-415-506-6570, or Media, Susan Berg,
+1-415-506-6594, both of BioMarin Pharmaceutical Inc.