© 2023 BioMarin. All rights reserved.
BioMarin Pharmaceutical Inc.
As of March 31, 2004, BioMarin had cash, cash equivalents, and short-term investments of approximately $191.2 million.
The net loss in the first quarter of 2004 was due primarily to: the continued clinical development of Phase 3 product candidate Aryplase(TM) for the treatment of mucopolysaccharidosis VI (MPS VI); BioMarin's share of the loss from BioMarin/Genzyme LLC related to Aldurazyme(R); and expenses related to the advancement of the company's phenylketonuria (PKU) clinical development program.
Fredric D. Price, Chairman and Chief Executive Officer of BioMarin stated, "We are pleased with the progress we have made this year in our transformation to a fully integrated, pediatric-focused biopharmaceutical company with commercial operations in the U.S. We anticipate completion of the recently announced Ascent Pediatrics transaction soon, and in this current quarter we will announce the results of the just completed Phase 3 trial of Aryplase for MPS VI. If the results from that trial are positive, we anticipate filing applications for approval of Aryplase in the U.S. and the E.U. in the fourth quarter of 2004."
First Quarter Highlights
Ascent Pediatrics Transaction
-- On April 20, 2004, BioMarin announced the signing of a definitive
agreement with Medicis Pharmaceutical Corporation to obtain its
pediatric business, Ascent Pediatrics, Inc. The transaction, which is
expected to close in the second quarter of 2004, brings with it
Orapred(R), a U.S. Food and Drug Administration (FDA) approved,
patent-protected drug to treat asthma in children, two additional
proprietary formulations of Orapred, and a U.S. pediatric-focused sales
organization.
-- Excluding the non-cash charges associated with the transaction,
BioMarin expects that the net loss for 2004 will be less than the
previously announced guidance of a loss between $68 million to
$70 million. The company expects that the net sales of Orapred will be
approximately $42 million for the 12 months ending December 31, 2004.
BioMarin will record all Orapred revenues beginning with the
transaction closing.
-- BioMarin expects the loss for 2005 will decrease significantly due to
the anticipated profits from Orapred and Aldurazyme, an enzyme
replacement therapy for the treatment of mucopolysaccharidosis I
(MPS I), marketed on a worldwide basis by Genzyme Corporation.
BioMarin will provide more specific revised guidance for 2004 and 2005
during the third quarter of this year and at the end of this year,
respectively.
Aldurazyme for MPS I
-- Sales of Aldurazyme by BioMarin/Genzyme LLC were $7.4 million for the
first quarter of 2004. Previously announced sales guidance for the
12 months ending December 31, 2004, provided by Genzyme Corporation,
remains unchanged at between $40 million and $44 million. Aldurazyme
received marketing approval from the FDA and the European Commission in
the second quarter of 2003 and since then, Aldurazyme has been approved
in Norway, Iceland, Israel, and the Czech Republic, with additional
applications pending in Canada, Australia, New Zealand, Russia, Korea,
and Bulgaria.
Aryplase for MPS VI
-- BioMarin recently completed the Phase 3 trial of Aryplase for MPS VI
and expects to announce the results in the second quarter of 2004,
following analysis of the data from the six clinical sites. If the
data are positive, the company plans to file simultaneous applications
for approval in the U.S. and European Union in the fourth quarter of
2004.
Phenoptin(TM) for PKU
-- On February 23, 2004, BioMarin announced the initiation of its PKU
clinical program beginning with a pilot trial of tetrahydrobiopterin
(6R-BH4). The company expects to announce data from this trial in the
third quarter and, pending positive trial results, to initiate an
additional clinical trial in late 2004.
Upcoming Presentations at Medical Conferences
Investigators will present data from ongoing studies of Aldurazyme for MPS I and Aryplase for MPS VI at the following three conferences:
3rd International Conference: Prospects in the Treatment of Rare Diseases, May 19-22, 2004, Trieste, Italy
Aryplase/ MPS VI
-- Enzyme Replacement Therapy for Skeletal Disease in
Mucopolysaccharidosis Type VI
Immune Tolerance
-- Prospects for Treating Null-Genotyped Patients with Neurologic Disease
Using Immune Tolerance and Intrathecal Enzyme Therapy
8th International Symposium on MPS and Related Diseases, June 10 - 13, 2004, Mainz, Germany
Aldurazyme/ MPS I
-- Aldurazyme(R) (laronidase) Enzyme Replacement Therapy in MPS I:
Preliminary Data in Children Less Than Five Years of Age
-- The Attenuated ('Non-Hurler') Phenotype of Mucopolysaccharidosis: A
Review of the Clinical Features in 29 Patients
-- MPS I Characteristics: Description of First Patients Entered into the
MPS I Registry
-- Update on Clinical Trial Results in MPS I
-- The MPS I Registry: Centralised Data Collection to Outline the Natural
History and Treatment of MPS I Disease
-- Successful Treatment of the Brain and Meninges with Immune Tolerance in
Canine MPS I
Aryplase/ MPS VI
-- Update on Phase 1, 2, and 3 Clinical Trial Results of Aryplase in MPS
VI
-- A Threshold Effect of Urinary Glycosaminoglycans and the Walk Test as
Indicators of Disease Progression in a Survey of Subjects with
Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)
-- Long-Term Combined Therapy with Recombinant Human
N-acetylgalactosamine-4-Sulfatase for Degenerative Joint Disease in
Mucopolysaccharidosis VI Cats
-- Flexible Endoscopy in a 13-Year-Old Boy with MPS VI and Tracheostomy
Because of Upper Airway Obstruction: Changes After 91 and 146 Weeks of
a Phase I/II Enzyme Replacement Therapy with Recombinant Human
Arylsulfatase B
36th European Human Genetics Conference, June 12 - 15, 2004, Munich, Germany
Aryplase/ MPS VI
-- Update on Phase 1, 2, and 3 Clinical Trial Results of Aryplase in MPS
VI
-- A Threshold Effect of Urinary Glycosaminoglycans and the Walk Test as
Indicators of Disease Progression in a Survey of Subjects with
Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)
Upcoming Corporate Presentations
BioMarin will present an overview of its business and product development programs at the following upcoming healthcare and biotechnology investment conferences:
-- Rodman & Renshaw Global Healthcare Conference, May 12-14, 2004, London,
United Kingdom
-- BioEquity Europe 2004, May 17-18, 2004, Edinburgh, United Kingdom
-- UBS 2004 Global Specialty Pharmaceutical Conference, May 24-25, 2004,
New York City, NY
-- Pacific Growth Equities Life Sciences Growth Conference, June 9-10,
2004, San Francisco, CA
-- Needham & Co. Third Annual Biotechnology Conference, June 16-17, 2004,
New York City, NY
BioMarin will host a conference call and webcast to discuss first quarter financial results today at 12:00 p.m. EDT (18:00 CEST). This event can be accessed on the BioMarin website at: http://investor.biomarinpharm.com/ .
Date: May 4, 2004 Time: 12:00 p.m. EDT (18:00 CEST) U.S. & Canada Toll-free Dial in #: 1-800-915-4836 International Dial in #: 973-317-5319 Replay Toll-free Dial in #: 1-800-428-6051 Replay International Dial in #: 973-709-2089 Replay Code #: 346222
BioMarin Pharmaceutical Inc. develops innovative biopharmaceuticals and commercializes therapeutics for serious pediatric diseases.
Forward-Looking Statement
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the possible acquisition of the Ascent Pediatrics, Inc. business and the financial performance of that business if the transaction is completed; the financial performance of Aldurazyme and the company as a whole; the continued development and commercialization of Aldurazyme, Aryplase and Phenoptin; 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: actual completion of the proposed transaction; the successful integration of the sales force and the Orapred business into BioMarin's organization; results and timing of current and planned preclinical studies and clinical trials; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning each of the described products; the market for each of these products and particularly Aldurazyme and Orapred; actual sales of Aldurazyme and Orapred; the possible development of competing products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Factors That May Affect Future Results" in BioMarin's 2003 Annual Report on Form 10-K and the factors contained in BioMarin's reports on Forms 10-Q and 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.
BioMarin's press releases and other company information are available online at www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
NOTE: Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC. All rights reserved.
Orapred(R) is a registered trademark of Ascent Pediatrics, Inc., a wholly owned subsidiary of Medicis Pharmaceutical Corporation.
CONTACT: Joshua A. Grass, Sr. Manager, Investor and Financial Relations, +1-415-506-6777, or Susan Ferris Manager, Corporate Communications, +1-415-506-6701, both of BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc. and Subsidiaries
Consolidated Balance Sheets
(In thousands, except share and per share data)
December 31, March 31,
2003 (1) 2004
(unaudited)
Assets
Current assets:
Cash and cash equivalents $121,406 $96,199
Short-term investments 84,951 94,999
Investment in and advances to
BioMarin/Genzyme LLC 16,058 15,149
Other current assets 2,854 2,478
Total current assets 225,269 208,825
Property and equipment, net 25,154 24,447
Other assets 5,917 6,024
Total assets $256,340 $239,296
Liabilities and Stockholders' Equity
Current liabilities:
Accounts payable and accrued liabilities $10,098 $12,850
Other current liabilities 2,717 2,311
Total current liabilities 12,815 15,161
Convertible debt 125,000 125,000
Other long-term liabilities 672 363
Total liabilities 138,487 140,524
Stockholders' equity:
Common stock, $0.001 par value:
150,000,000 shares authorized;
64,156,285 and 64,273,601 shares issued
and outstanding December 31, 2003 and
March 31, 2004, respectively 64 64
Additional paid-in capital 414,110 414,929
Warrants 5,219 5,219
Deferred compensation (145) (101)
Accumulated other comprehensive loss (17) (16)
Accumulated deficit (301,378) (321,323)
Total stockholders' equity 117,853 98,772
Total liabilities and stockholders'
equity $256,340 $239,296
(1) December 31, 2003 balances were derived from the audited consolidated
financial statements.
BioMarin Pharmaceutical Inc. and Subsidiaries
Consolidated Statements of Operations
For the Three Months Ended March 31, 2003 and 2004
(In thousands, except per share data, unaudited)
Three Months Ended
March 31,
2003 2004
Operating expenses:
Research and development $10,991 $13,887
General and administrative 2,799 3,689
Equity in the loss of BioMarin/Genzyme LLC 6,753 1,759
Total operating expenses 20,543 19,335
Loss from operations (20,543) (19,335)
Interest income 413 761
Interest expense (130) (1,371)
Net loss from continuing operations (20,260) (19,945)
Gain on disposal of discontinued operations 577 --
Net loss $(19,683) $(19,945)
Net loss per share, basic and diluted:
Net loss from continuing operations $(0.36) $(0.31)
Gain on disposal of discontinued operations 0.01 --
Net loss $(0.35) $(0.31)
Weighted average common shares outstanding 56,964 64,225
SOURCE: BioMarin Pharmaceutical Inc.
CONTACT: Joshua A. Grass, Sr. Manager, Investor and Financial Relations,
+1-415-506-6777, or Susan Ferris Manager, Corporate Communications,
+1-415-506-6701, both of BioMarin Pharmaceutical Inc.
Web site: http://www.bmrn.com/