"The efficient enrollment of twenty patients is a critical milestone for the MPS IVA program and demonstrates both our commitment to this program and the support and enthusiasm of the MPS IVA patient community. Data generated from this study will be valuable in demonstrating safety and could be instrumental in designing a successful Phase III trial. Assessments from the Phase I/II study such as plasma and urine keratan sulfate levels, pulmonary function and walk tests will be helpful in determining optimal Phase III endpoints," said Henry Fuchs, M.D., Chief Medical Officer of BioMarin. "We appreciate the collaboration of the Morquio community in this important effort, and we hope to develop this new treatment as expeditiously as possible."
The Phase I/II study is designed as an open-label, within-patient dose escalation trial in 20 patients followed by a treatment continuation period. During the dose escalation phase of the study, subjects will receive weekly intravenous infusions of BMN-110 in three consecutive 12-week dosing intervals. The objectives of the Phase I/II study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of GALNS for future studies. BioMarin plans to provide an extension study in which all patients in the Phase I/II study will be eligible to participate.
About MPS IVA
The incidence estimates for MPS IVA vary widely, between one in 200,000 live births to one in 300,000 live births. Approximately 400 patients worldwide have been identified and tracked through the International Morquio Organization (IMO) survey. There are already more MPS IVA patients identified through this registry than there are MPS VI patients being treated with Naglazyme worldwide. Based on the number of identified patients to date, the prevalence of patients with MPS IVA appears similar to that with MPS I.
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 preclinical 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 that was developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of 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 Phase I clinical development for the treatment of PKU. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the development of its program for MPS IVA, and particularly the timing and conduct of clinical trials related thereto, and expectations regarding filings with regulatory agencies. 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 results of current and planned clinical and pre-clinical trials related to the enzyme replacement therapy for MPS IVA; the content and timing of decisions by the U.S. Food and Drug Administration, EMEA and other regulatory agencies, particularly with respect to the enzyme replacement therapy for MPS IVA, 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. 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.
Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.
BioMarin Pharmaceutical Inc.
