0001048477-01-500056.txt : 20011107
0001048477-01-500056.hdr.sgml : 20011107
ACCESSION NUMBER: 0001048477-01-500056
CONFORMED SUBMISSION TYPE: 8-K
PUBLIC DOCUMENT COUNT: 2
CONFORMED PERIOD OF REPORT: 20011102
ITEM INFORMATION: Other events
FILED AS OF DATE: 20011102
FILER:
COMPANY DATA:
COMPANY CONFORMED NAME: BIOMARIN PHARMACEUTICAL INC
CENTRAL INDEX KEY: 0001048477
STANDARD INDUSTRIAL CLASSIFICATION: PHARMACEUTICAL PREPARATIONS [2834]
IRS NUMBER: 680397820
STATE OF INCORPORATION: DE
FISCAL YEAR END: 1231
FILING VALUES:
FORM TYPE: 8-K
SEC ACT: 1934 Act
SEC FILE NUMBER: 000-26727
FILM NUMBER: 1773502
BUSINESS ADDRESS:
STREET 1: 371 BEL MARIN KEYS BLVD
STREET 2: STE 210
CITY: NOVATO
STATE: CA
ZIP: 94949
BUSINESS PHONE: 4158846700
MAIL ADDRESS:
STREET 1: 371 BEL MARIN KEYS BLVD
STREET 2: STE 210
CITY: NOVATO
STATE: CA
ZIP: 94949
8-K
1
form8k110201.txt
================================================================================
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
November 2, 2001 (November 2, 2001)
BioMarin Pharmaceutical Inc.
(Exact name of registrant as specified in its charter)
Delaware 000-26727 68-0397820
(State or other jurisdiction (Commission (IRS Employer
of incorporation) File Number) Identification No.)
371 Bel Marin Keys Boulevard, 94949
Suite 210 (Zip Code)
Novato, California
(Address of principal executive offices)
(415) 884-6700
------------------------------------------------
(Registrant's telephone number, including area code)
================================================================================
Item 5. Other Events
On November 2, 2001, we announced the results of our Phase III trial of
Aldurazyme for the treatment of MPS I in a press release dated November 2, 2001.
A copy of the press release is attached hereto as Exhibit 99.1 and incorporated
herein by reference.
Item 7. Financial Statements and Exhibits
EXHIBIT NO. DESCRIPTION OF DOCUMENT
------------ ------------------------------------------------------------------
99.1 Press Release Dated November 2, 2001 Related to the Announcement
of the Results of the Phase III Trial of Aldurazyme for the
Treatment of MPS I.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the
registrant has duly caused this report to be signed on its behalf by the
undersigned hereunto duly authorized.
BioMarin Pharmaceutical Inc.
By: /s/ Raymond W. Anderson
---------------------------------
Raymond W. Anderson
Chief Operating Officer, Chief Financial
Officer, Secretary and Vice President Finance
and Administration
Date: November 2, 2001
Exhibit Index
EXHIBIT NO. DESCRIPTION OF DOCUMENT
------------ ------------------------------------------------------------------
99.1 Press Release Dated November 2, 2001 Related to the Announcement
of the Results of the Phase III Trial of Aldurazyme for the
Treatment of MPS I.
EX-99
2
pressrelease110201.txt
EXHIBIT 99.1
NEWS
Contacts:
For BioMarin: For Genzyme:
Jeremy T. Price Dan Quinn (media)
Financial & Investor Relations (617) 591-5849
BioMarin Pharmaceutical Inc.
(415) 884-6777
Sharon Karlsberg Sally Curley (investors)
Vice President (617) 591-7140
Feinstein Kean Healthcare
(617) 577-8110
For Immediate Release:
BioMarin and Genzyme to File for Marketing Approval
for Aldurazyme(TM) Based on Phase 3 Trial Results
Novato, CA and Cambridge, MA, November 2nd, 2001. - BioMarin Pharmaceutical Inc.
(Nasdaq and Swiss SWX New Market: BMRN) and Genzyme General (Nasdaq: GENZ) today
announced positive results from a preliminary analysis of data from the Phase 3
clinical trial of Aldurazyme(TM) (laronidase), an investigational enzyme
replacement therapy for patients with mucopolysaccharidosis I (MPS I).
Based on the strength of the trial's results, the companies plan to meet with
U.S., Canadian, and European regulatory authorities to discuss applications to
market Aldurazyme.
The Phase 3 trial enrolled 45 patients at five sites in the U.S., Europe, and
Canada in a randomized, double-blind, placebo-controlled study designed to
evaluate the safety and efficacy of Aldurazyme in patients with MPS I. During
the 26-week evaluation period, 22 patients received weekly intravenous infusions
of Aldurazyme and 23 patients received weekly placebo infusions. The 45 patients
ranged in age from 6 to 43, with an average age of 15 years old. All patients
completed the trial and have elected to receive Aldurazyme in an open-label
extension study.
Patients were evaluated at defined intervals to assess progress in meeting two
primary endpoints. The preliminary data analysis showed a statistically
significant increase in pulmonary capacity (p=0.028), and demonstrated a
positive trend in endurance as measured by a six-minute walk test (p=0.066).
Among other endpoints measured in the trial, the main findings of an earlier
open-label study of Aldurazyme were confirmed: a reduction in liver size and a
reduction in excretion of urinary glycosaminoglycans (GAGs), the carbohydrate
substances that accumulate in patients with MPS I. Data from the earlier trial
were published in the New England Journal of Medicine in January, 2001.
The safety profile was comparable between the treatment group and the placebo
group. There were no Aldurazyme-related serious adverse events. The most
commonly reported reactions were fever (14 patients on the placebo and 10 in the
treatment group); headache (16 on placebo and 11 in the treatment group);
rhinitis (10 on placebo and 8 in the treatment group); and rash (5 on placebo
and 8 in the treatment group).
"The data described today confirm and expand upon the results demonstrated in
our first clinical trial," said Richard Moscicki, M.D., senior vice president
and chief medical officer at Genzyme. "We have amassed the body of evidence that
supports the potential benefits of Aldurazyme for patients with MPS I."
"This milestone in the development of Aldurazyme represents a critical step
toward delivering a much-needed therapy to MPS I patients," said Stuart J.
Swiedler, M.D., Ph.D, BioMarin's vice president, clinical affairs. "We look
forward to discussing the next steps with worldwide regulatory agencies."
About MPS I
MPS I (also known as Hurler, Hurler-Scheie, and Scheie syndromes) is a
life-threatening genetic disease caused by a deficiency of the enzyme
alpha-L-iduronidase. The deficiency leads to the accumulation of complex
carbohydrates (GAGs) in the lysosomes of cells, leading to the progressive
dysfunction of cellular, tissue and organ systems. Resulting symptoms can
include impaired cardiac and pulmonary function, delayed physical development,
skeletal and joint deformities, reduced endurance, and in some cases, delayed
mental function. A majority of patients die before adulthood from complications
of the disease.
BioMarin and Genzyme General formed a joint venture in 1998 to develop and
commercialize Aldurazyme worldwide. The companies plan to present the full
details of the data from this trial at a major medical meeting, and to seek
publication in a peer-reviewed medical journal.
Genzyme and BioMarin have received Orphan Drug designation and Fast-Track status
for Aldurazyme for MPS I from the U.S. Food and Drug Administration, and orphan
medicinal product designation in Europe.
Genzyme General develops and markets therapeutic products and diagnostic
products and services. Genzyme General has five therapeutic products on the
market and a strong pipeline of therapeutic products in development focused on
the treatment of genetic diseases and other chronic debilitating disorders with
well-defined patient populations. Genzyme General is a division of Genzyme Corp.
BioMarin Pharmaceutical specializes in the development and commercialization of
therapeutic enzyme products to treat serious, life-threatening diseases and
conditions.
This press release contains forward-looking statements, including statements
regarding: anticipated submissions for regulatory approvals of Aldurazyme in the
U.S. and Europe; the potential benefits of Aldurazyme; the potential market
introduction of Aldurazyme; plans for meetings with regulatory authorities; and
publication plans. Actual results may differ materially from those contained in
these forward-looking statements as a result of a number of factors, including:
the results of the final analysis of the trial data; the ability to manufacture
sufficient amounts of Aldurazyme for development and commercialization
activities; the timing and content of submissions to and decisions made by U.S.,
Canadian, and European regulatory authorities; the continued funding of the
joint venture; the ability to obtain and maintain adequate patent and other
proprietary rights protection for Aldurazyme; the scope, validity and
enforceability of patents and other proprietary rights held by third parties
related to therapies for MPS I and the actual impact of such patents and other
rights on our ability to commercialize Aldurazyme; the competitive environment
for therapies for MPS I; and the risks and uncertainties described in Genzyme's
and BioMarin's reports filed with the Securities and Exchange Commission under
the Securities Exchange Act of 1934, as amended, including, with respect to
Genzyme, Exhibit 99.2 to Genzyme's Annual Report on Form 10-K for the year ended
December 31, 2000, as amended and, with respect to BioMarin, the section of
BioMarin's Annual Report, entitled "Factors that May Affect Future Results" and
the sections of BioMarin's Registration Statements, as amended entitled "Risk
Factors." Genzyme General division common stock is a series of common stock of
Genzyme Corporation. Therefore, holders of Genzyme General division common stock
are subject to all of the risks and uncertainties described in the
aforementioned reports filed by Genzyme Corporation.
Genzyme(R) is a registered trademark of Genzyme Corporation. Aldurazyme(TM) is a
trademark of BioMarin/Genzyme LLC. All rights reserved.