Presentations at WORLD Symposium: Vimizim Phase 3 Results and BMN-701 Phase 1/2 Patient Demographics
SAN RAFAEL, Calif., Feb. 11, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the following presentations will be featured at the 9th Annual WORLD Symposium in Orlando, Florida February 13-15, 2013:
On Friday, February 15th at 9:45 a.m., Dr. Barry Byrne from the University of Florida, Gainesville will make a presentation titled: POM-001 Phase 1/2 Study of BMN-701, GILT-tagged Recombinant Human (rh) GAA in Late-Onset Pompe Disease: Preliminary Report. The presentation will provide background information on BMN-701, review the design of the Phase 1/2 trial and provide information on the patient demographics and baseline characteristics of the 22 patients enrolled in the trial. BioMarin remains on track to report efficacy and safety results for the BMN-701 Phase 1/2 trial by the end of the first quarter of 2013.
On Friday, February 15th at 3:15 p.m., Dr. Christian Hendriksz from the University of Manchester Birmingham Children's Hospital in Lancashire, United Kingdom will make a presentation titled: A Multinational, Randomized, Double–Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of BMN 110 Treatment for Mucopolysaccharidosis IVA (Morquio Syndrome Type A). The presentation will review efficacy and safety data from the Phase 3 study.
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® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include BMN-110 (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
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This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: expectations related to the Zacharon development programs, and the development of BioMarin's other programs, including GALNS; 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: results and timing of current and planned preclinical studies and clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; 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 2011 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®, Naglazyme®, Kuvan® and Firdapse™ are registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
CONTACT: Investors: Eugenia Shen BioMarin Pharmaceutical Inc. (415) 506-6570 Media: Debra Charlesworth BioMarin Pharmaceutical Inc. (415) 455-7451