-----BEGIN PRIVACY-ENHANCED MESSAGE----- Proc-Type: 2001,MIC-CLEAR Originator-Name: webmaster@www.sec.gov Originator-Key-Asymmetric: MFgwCgYEVQgBAQICAf8DSgAwRwJAW2sNKK9AVtBzYZmr6aGjlWyK3XmZv3dTINen TWSM7vrzLADbmYQaionwg5sDW3P6oaM5D3tdezXMm7z1T+B+twIDAQAB MIC-Info: RSA-MD5,RSA, FaT/aPowZsX4t64g7I/xXLkqXtWxuuXhP1oD+gkJXnLv2n6hHFABc4CzQCmJq+RL 9XqUsa+/j8Z+XYft8Km3mg== 0001048477-03-000016.txt : 20030314 0001048477-03-000016.hdr.sgml : 20030314 20030314154424 ACCESSION NUMBER: 0001048477-03-000016 CONFORMED SUBMISSION TYPE: 8-K PUBLIC DOCUMENT COUNT: 2 CONFORMED PERIOD OF REPORT: 20030331 ITEM INFORMATION: Other events FILED AS OF DATE: 20030314 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: 03604192 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 form8k031403.txt FORM 8-K MARCH 14, 2003 =============================================================================== 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): March 14, 2003 BioMarin Pharmaceutical Inc. (Exact name of registrant as specified in its charter) Delaware 000-26727 68-0397820 (State or other jurisdiction of (Commission (IRS Employer incorporation or organization) File Number) Identification No.) 371 Bel Marin Keys Boulevard, Suite 210, Novato, California 94949 (Address of principal executive offices) (Zip Code) Registrant's telephone number, including area code: (415) 884-6700 Not Applicable ------------------------------------------ (Former name or former address, if changed since last report) =============================================================================== Item 5. Other Events. On March 14 , 2003, BioMarin Pharmaceutical Inc. (the "Registrant"), issued a press release regarding the presentation of data related to the Registrant's Phase II trial of Aryplase for the treatment of MPS VI. The Registrant's press release issued on March 14, 2003 is attached hereto as Exhibit 99.1. Item 7. Financial Statements, Pro Forma Financial Statements and Exhibits. (a) Financial Statements of Business Acquired. Not Applicable. (b) Pro Forma Financial Information. Not Applicable. (c) Exhibits. Exhibit 99.1 Press Release of the Registrant dated March 14, 2003. SIGNATURE 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., a Delaware corporation Date: March 14, 2003 By: /s/ Fredric D. Price ----------------- Fredric D. Price Chairman and Chief Executive Officer EXHIBIT INDEX Exhibit No. Description Exhibit 99.1 Press Release of the Registrant dated March 14, 2003. EX-99 3 pressrelease031403.txt PRESS RELEASE Exhibit 99.1 BioMarin Pharmaceutical NEWS CONTACTS: Joshua Grass (Investors) BioMarin Pharmaceutical Inc. (415) 884-6777 Fredda Malkoff (Media) Feinstein Kean Healthcare (617) 577-8110 For Immediate Release: - ---------------------- BioMarin Presents Positive Phase II Data on Aryplase for MPS VI AryplaseTM Well Tolerated and Associated with Functional Improvements BioMarin to Initiate a Pivotal Phase III Trial in 2003 Novato, CA, March 14, 2003 - BioMarin Pharmaceutical Inc. (Nasdaq and Swiss SWX New Market: BMRN) announced positive results from its Phase II open-label study of Aryplase, recombinant human arylsulfatase B (rhASB), an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). MPS VI (also known as Maroteaux-Lamy Syndrome) is a debilitating, life-threatening genetic disease, for which no drug therapies are currently available, that is caused by a deficiency of the enzyme arylsulfatase B. Dr. Paul Harmatz, M.D. of Children's Hospital Research Center of Oakland, California will present the results today at the American College of Medical Genetics 9th Annual Clinical Genetics Meeting in San Diego, California. The open-label, 10 patient, clinical study of Aryplase was conducted at two sites, one in the United States, and one in Australia. The study evaluated clinical and biochemical measures of safety and efficacy in male and female subjects between the ages of 6 and 22, for a duration of 24 weeks. During the study, subjects received weekly 1.0 mg/kg infusions of Aryplase, a dose selected based on results from both a Phase I study, and the feline MPS VI model. Study Results - ------------- Results from this Phase II study indicate that Aryplase is well tolerated and is associated with improvements in several clinical endpoints. On average, subjects experienced improvements in endurance as measured by the distance walked at the 6 and 12 minute time points of a 12-minute walk test, and the number of stairs climbed in 3 minutes. Functional improvements were also observed in joint pain and stiffness, and in shoulder flexion, extension, and rotation in subjects who exhibited less than 90 degrees shoulder flexion at baseline. Results are provided in more detail below: o The average improvement at the 6-minute time point in the walk test was 64 meters over baseline distances, which ranged from 19 to 247 meters. On average subjects demonstrated a 62% improvement in distance walked at 6 minutes. o The average improvement at 12 minutes in the walk test was 155 meters over baseline distances, which ranged from 33 to 475 meters. On average subjects demonstrated a 98% improvement in distance walked after 12 minutes. o The average improvement in the number of stairs climbed in 3 minutes was 48 stairs over the baseline number, which ranged from 20 to 92 stairs. This increase in the number of stairs climbed represents an average improvement of 110% over baseline. o Subjects also experienced improvement in pain and joint stiffness based on a modified child health assessment questionnaire (CHAQ) in which subjects were asked to score pain levels on a scale from 0 to 100. Joint pain and stiffness scores were reduced on average by 57% and 54% compared to baseline, respectively. o Small improvements in both passive and active measurements of shoulder range of motion for flexion, extension and lateral rotation were observed, with the largest gain achieved in the three subjects who exhibited less than 90 degrees of active shoulder flexion at baseline. In addition to the clinical improvements observed, study participants also demonstrated an average decrease in urinary glycosaminoglycan (GAG) excretion of 71% in 24 weeks, indicating Aryplase reduced carbohydrate storage in MPS VI subjects. As part of the Phase II trial, several other exploratory endpoints were evaluated, but on average did not indicate meaningful changes in the 24-week study period. These include pulmonary function as measured by forced vital capacity, a pinch and grip strength test, physical activity, oxygenation level during sleep, expanded time to get up and go test, and a set of tasks reflecting quality of life. Improvement in these clinical endpoints may be observed with continued infusions over a longer period of time. Results of the study demonstrated that Aryplase was generally well tolerated. Out of 240 infusions over the 24-week period, 5 adverse events were reported during infusion and an additional 8 during the day of infusion, which were most commonly abdominal pain, febrile reactions, and pruritis. Separately, there were 7 serious adverse events, 6 unrelated to drug, and 1 possibly related to the drug. Consistent with infusion of protein drugs, subjects developed antibodies to Aryplase during the course of the study, which did not correlate to reduction in urinary GAG excretion. Mildly reduced complement levels were detected in some patients, which did not have a measurable clinical impact. About MPS VI - ------------ MPS VI (also known as Maroteaux-Lamy Syndrome) is a debilitating, life-threatening genetic disease for which no drug therapies are currently available. MPS VI is caused by a deficiency of the enzyme arylsulfatase B. The deficiency leads to the accumulation of GAGs in the lysosomes, the digestive organelles of the cell. This accumulation in the lysosomes leads to progressive cellular, tissue and organ system dysfunction. Debilitating symptoms can include impaired cardiac and pulmonary function, delayed physical development, skeletal and joint deformities, impaired vision and hearing, sleep apnea, and reduced endurance. The majority of subjects die from disease-related complications between childhood and early adulthood, depending on the severity of the disease. BioMarin has received orphan drug and fast track designations for Aryplase from the U.S. Food and Drug Administration (FDA). In addition, the European Commission has designated Aryplase for the treatment of MPS VI as an orphan medicinal product in the European Community. BioMarin plans to initiate a multi-national, placebo-controlled, double-blind Phase III trial of Aryplase in MPS VI subjects in 2003. 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 about the business prospects of BioMarin Pharmaceutical Inc., including without limitation statements about: expectations concerning the final results of past clinical trials of Aryplase; the expected timing on future clinical trials for Aryplase; and actions by regulatory authorities, including the FDA and the European Commission. 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: the final analysis of results of past clinical trials; results and timing of current and future clinical trials; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning Aryplase; 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 2002 Annual Report on Form 10-K and the factors contained in BioMarin's reports on Forms 10Q and 8K. 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 http://www.biomarinpharm.com. Information on our website is not incorporated by reference into this press release. -----END PRIVACY-ENHANCED MESSAGE-----