BioMarin Initiates Phase 3b Study to Evaluate the Effects of Kuvan on Neurophychiatric Symptoms in Subjects with PKU
NOVATO, Calif., Aug 17, 2010 /PRNewswire via COMTEX/ -- BioMarin Pharmaceutical Inc. announced today that the first subject has initiated treatment in a Phase 3b study (PKU-016) to evaluate the effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (PKU). BioMarin expects to report results in the first half of 2012.
"We hope that this trial will provide quantitative data regarding the neuropsychiatric effects of Kuvan for PKU patients, and if successful, will continue to build its value proposition and to drive the continued expansion of the market," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Although we have heard anecdotal accounts from patients and physicians regarding Kuvan's impact beyond blood Phe level reduction, BioMarin has never formally evaluated these endpoints in a company trial due to the extremely short development time for Kuvan. We are excited to have this important study underway, and if the results are positive, we plan to file for a label amendment. We also look forward to upcoming data from several investigator-sponsored studies on the neurocognitive effects of Kuvan at the SSIEM meeting in Istanbul in late August."
The Phase 3b study is a double-blind, placebo-controlled, randomized 28-week study (2-week screening period, 13-week randomized treatment period, 13-week open-label treatment period) to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU. The study will enroll approximately 200 subjects ages twelve and above at approximately 40 sites in the U.S. and Canada.
The primary efficacy variable for symptoms of attention deficit hyperactivity disorder (ADHD) will be evaluated using the Attention-Deficit Hyperactivity Disorder Rating Scale (ASRS) in subjects twelve to 17 years of age and the Adult ADHD Self-Report Scale (ASRS) in subjects ages 18 and above. The Clinical Global Impression of Improvement (CGI-I) scale will be assessed to determine improvement in global impression of behavior, symptoms and functioning relative to treatment as the primary efficacy endpoint for global function.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical 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 which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse(TM) (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Other product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in clinical development for the treatment of MPS IVA and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II 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 expectations about the timing, conduct and impact of the clinical trial of Kuvan to evaluate neurophychiatric symptoms in subjects with PKU. 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 enrolment of such trial; the data generated from such trial; the content and timing of decisions by the U.S. Food and Drug Administration and other regulatory authorities; 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 2009 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. 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(R), Naglazyme(R) and Kuvan(R) are registered trademarks of BioMarin Pharmaceutical Inc.
Firdapse(TM) is a trademark of BioMarin Huxley Ltd.
Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.
Eugenia Shen Susan Berg
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc
(415) 506-6570 (415) 506-6594