Exhibit 99.1
Contacts:
Joshua Grass Fredda Malkoff
Manager, Investor and Financial Relations Account Director
BioMarin Pharmaceutical Inc. Feinstein Kean Healthcare
(415) 884-6777 (617) 577-8110
jgrass@biomarinpharm.com fmalkoff@fkhealth.com
For Immediate Release:
BioMarin Announces Second Quarter 2002 Financial Results
Conference Call and Webcast to be Held Today at 4:15 PM ET (2215 CET)
Novato, CA, August 1, 2002 - BioMarin Pharmaceutical Inc. (Nasdaq and Swiss SWX
New Market: BMRN) today announced financial results for its second quarter ended
June 30, 2002. The net loss was $14.4 million ($0.27 per share) for the second
quarter of 2002 compared to $12.0 million ($0.30 per share) for the second
quarter of 2001. The net loss was $41.0 million ($0.77 per share) for the six
months ended June 30, 2002 compared to $21.7 million ($0.57 per share) for the
six months ended June 30, 2001. BioMarin had $100.6 million in cash, cash
equivalents and short-term investments as of June 30, 2002.
The net loss in the second quarter of 2002 was primarily due to the continued
development of the Company's lead product programs: AldurazymeTM for MPS I,
AryplaseTM for MPS VI, and NeutralaseTM for heparin reversal.
Recent Achievements
Fredric D. Price, BioMarin's Chairman and Chief Executive Officer, commented,
"Since May, we have made significant progress in our clinical development
programs. I am pleased to highlight the following recent achievements and
upcoming events:"
o BioMarin received a U.S. patent covering AldurazymeTM for the treatment
of MPS I. Aldurazyme is a specific form of purified recombinant human
alpha-L-iduronidase that is being investigated as an enzyme replacement
therapy for MPS I, a life-threatening genetic disease for which no
specific drug treatments currently exist. The patent claims unique
characteristics of the pharmaceutical composition of Aldurazyme. This
patent, which protects a highly purified form of alpha-L-iduronidase,
supports the intellectual property position for using Aldurazyme to
treat MPS I.
o BioMarin and Genzyme submitted the final portion of their "rolling"
Biologics License Application (BLA) for Aldurazyme to the U.S. Food and
Drug Administration (FDA). The final portion of the BLA includes
clinical data from the six-month, placebo-controlled Phase 3 trial of
Aldurazyme, six months of data from the ongoing open-label Phase 3
extension study, and three years of data from the Phase 1 trial and
extension study. As part of the BLA submission, BioMarin and Genzyme
have formally requested Priority Review.
o BioMarin appointed a new Chief Financial Officer and elected three new
members to its Board of Directors. Louis Drapeau joined BioMarin as
Vice President, Finance & Chief Financial Officer, and Secretary. In
addition, Franz Cristiani, retired Partner of Arthur Andersen, LLP,
Elaine Heron, PhD, Chairman and CEO of Picoliter Inc., and Vijay
Samant, President and CEO of Vical Incorporated, were elected to the
Board.
o BioMarin established a Science Advisory Board (SAB) comprised of
leading scientists and physicians who will provide management with
critical scientific and strategic input as the Company continues to
develop its pipeline of enzyme therapeutics.
o The principal investigator from the Phase 1 clinical trial of Aryplase
for MPS VI presented positive findings from the trial's extension study
that indicate Aryplase continues to be well-tolerated at both dose
levels by the five patients who have received treatment for a total of
48 weeks. In addition, the 1.0 mg/kg dose continued to produce a
greater sustained reduction than the 0.2 mg/kg dose in the excretion of
urinary glycosaminoglycans (GAGs) over 48 weeks. The reduction in
urinary GAGs indicates that Aryplase is breaking down the complex
carbohydrate materials that otherwise accumulate in patients with MPS
VI and lead to the debilitating and life-threatening symptoms of the
disease.
o Investigators from the Phase 3 clinical trial of Aldurazyme for MPS I
presented detailed results from the double-blind portion of the trial
and preliminary six-month findings from the trial's ongoing open-label
extension study. Data from the extension study indicate patients who
received Aldurazyme for twelve months continued to improve upon the
results seen in the first six months of treatment.
o BioMarin commenced preclinical studies of NeuroTransTM and initiated
key steps to continue building the technology's intellectual property
position. NeuroTrans is the proprietary brain delivery technology that
BioMarin is developing to treat neurological problems associated with
many lysosomal storage disorders.
Upcoming Events
o BioMarin will hold its annual stockholders' meeting on August 13, 2002
at 10:00 a.m. The annual meeting will be held at the Embassy Suites
Hotel, located at 101 McInnis Parkway in San Rafael, CA. At this year's
annual meeting, BioMarin stockholders will vote on, among other items,
the Company's planned acquisition of all the outstanding shares of
Glyko Biomedical Ltd. Glyko Biomedical's principal asset is an
approximate 21% ownership interest in the capital stock of BioMarin.
o BioMarin and Genzyme expect a decision regarding FDA's acceptance of
the Aldurazyme BLA and its Priority Review status by the end of
September 2002.
o BioMarin plans to initiate the first of two Phase 3 clinical trials of
NeutralaseTM for the reversal of heparin following Coronary Artery
Bypass Graft (CABG) surgery during the third quarter of this year.
BioMarin will host a conference call and webcast to discuss second quarter
financial results today at 4:15 PM ET (2215 CET). This event can be accessed on
the BioMarin website at: http://investor.biomarinpharm.com.
Date: August 1st, 2002
Time: 4:15 PM ET (2215 CET)
U.S. & Canada Toll-free Dial in #:1-800-915-4836
International Dial in #: 1-973-317-5319
Replay Toll-free Dial in #: 1-800-428-6051
Replay International Dial in #: 973-709-2089
Replay Code #: 254590
BioMarin specializes in the development and commercialization of therapeutic
enzyme products to treat serious, life-threatening diseases and other
conditions.
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including the following potential
future products: Aldurazyme for the treatment of MPS I, Neutralase for the
reversal of anticoagulants, Aryplase for the treatment of MPS VI, and NeuroTrans
for the delivery of enzymes across the blood-brain barrier. These
forward-looking statements are predictions and involve risks and uncertainties
such that actual results may differ materially from these statements. Results
may differ materially depending on the progress of BioMarin's product programs,
the actual results of the current and planned clinical trials, actions of
regulatory authorities, including actions related to the Biologics License
Application and the Marketing Authorization Application, the closing of the
planned acquisition of Glyko Biomedical Ltd., availability of capital, future
actions in the pharmaceutical market and developments by competitors, and those
factors detailed in BioMarin's filings with the Securities and Exchange
Commission such as 10Q, 10K and 8K reports. 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.
AldurazymeTM is a trademark of BioMarin/Genzyme LLC. All rights reserved.
BioMarin Pharmaceutical Inc.
Condensed Consolidated Balance Sheets
(in thousands)
December 31, June 30,
2001 2002
--------------- -------------
(unaudited)
Assets
Current assets:
Cash and cash equivalents $ 12,528 $ 19,363
Short-term investments 118,569 81,195
Due from BioMarin/Genzyme LLC 3,096 2,135
Current assets of discontinued operations of Glyko, Inc. 668 687
Other current assets 1,922 2,175
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Total current assets 136,783 105,555
Investment in BioMarin/Genzyme LLC 1,145 2,356
Property, plant and equipment, net 32,560 32,704
Other non-current assets 1,323 1,358
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Total assets $ 171,811 $ 141,973
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Liabilities and Stockholders' Equity
Current liabilities:
Accounts payable $ 4,284 $ 2,014
Accrued liabilities 2,198 3,348
Current liabilities of discontinued operations of Glyko, Inc. 229 158
Other short-term liabilities 1,591 2,211
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Total current liabilities 8,302 7,731
Long-term liabilities 3,961 3,463
--------------- -------------
Total liabilities 12,263 11,194
--------------- -------------
Stockholders' equity:
Common stock 52 53
Additional paid-in capital 305,230 316,813
Common stock warrants 5,134 5,219
Deferred compensation (699) (281)
Notes receivable from stockholders (2,037) (2,108)
Accumulated other comprehensive income (13) 216
Accumulated deficit (148,119) (189,133)
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Total stockholders' equity 159,548 130,779
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Total liabilities and stockholders' equity $ 171,811 $ 141,973
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BioMarin Pharmaceutical Inc.
Consolidated Statements of Operations
(in thousands, except per share data)
(unaudited)
Three Months Ended June 30, Six Months Ended June 30,
--------------------------- -------------------------
2001 2002 2001 2002
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Revenues
Revenues from BioMarin/Genzyme LLC $ 2,852 $ 3,423 $ 5,542 $ 7,215
Revenues - other 160 - 160 -
------------- ------------- ------------ ------------
Total revenues 3,012 3,423 5,702 7,215
Operating Costs and Expenses
Research and development 11,506 13,336 21,163 26,554
General and administrative 1,536 3,062 3,010 6,988
In-process research and development - - - 11,223
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Total operating costs and expenses 13,042 16,398 24,173 44,765
Loss from operations (10,030) (12,975) (18,471) (37,550)
Interest income 436 1,024 904 1,404
Interest expense (1) (161) (3) (252)
Equity in loss of BioMarin/Genzyme LLC (1,736) (2,461) (2,844) (4,759)
------------- ------------- ------------ -----------
Net loss from continuing operations (11,331) (14,573) (20,414) (41,157)
Income (loss) from discontinued operations (638) 172 (1,255) 294
Loss from disposal of discontinued operations - (10) - (151)
------------- ------------- ------------ -----------
Net loss $ (11,969) $ (14,411) $ (21,669) $(41,014)
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Net loss per share, basic and diluted
Loss from continuing operations $ (0.28) $ (0.27) $ (0.54) $ (0.78)
------------- ------------- ------------ -----------
Income (loss) from discontinued operations $ (0.02) $ - $ (0.03) $ 0.01
------------- ------------- ------------ -----------
Loss on disposal of discontinued operations $ - $ - $ - $ -
------------- ------------- ------------ -----------
Net loss $ (0.30) $ (0.27) $ (0.57) $ (0.77)
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Weighted average common shares outstanding 39,587 53,407 38,313 52,973
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