BioMarin Announces Program for BMN-111 for the Treatment of Achondroplasia

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Oct 19, 2010, 08:00 ET

NOVATO, Calif., Oct. 19 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced its program for BMN-111, a peptide therapeutic for the treatment of achondroplasia. BioMarin plans to file an IND in the fourth quarter of 2011 and to initiate a Phase 1 clinical trial by the first quarter of 2012.

BMN-111, for the treatment of achondroplasia, is an analog of C-type Natriuretic Peptide (CNP), a small cyclic peptide that is a positive regulator of bone growth. It is produced and has a receptor in the growth plate, and along with the fibroblast growth factor receptor 3 (FGFR3), regulates normal bone growth. In addition to short stature, there are complications in achondroplasia that are related to bone compression (e.g. foramen magnum narrowing, spinal stenosis, upper respiratory narrowing) of nervous tissues or other tissues.

"This program is a perfect fit in our growing pipeline. It is an unmet medical need in an orphan disease population and can leverage our expertise in biologic manufacturing," said Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer of BioMarin. "Proof-of-concept studies demonstrate that CNP can reverse the achondroplastic phenotype in mice. Additionally, in both mice and primates, BMN-111 was able to penetrate the growth plate and accelerate growth at hemodynamically acceptable doses. We have a proven track record of expeditiously bringing life-altering therapeutics to patients and are determined to do the same with this program. We look forward to updating you on advancements in this and other programs in our product pipeline."

Additional details of the achondroplasia program, along with an overview of BioMarin's product portfolio and advancements in the research and development pipeline will be provided today at BioMarin's R&D Day program in New York City. Interested parties may access a live audio webcast of the presentation via the investor section of the BioMarin website, http://www.BMRN.com. A replay of the presentation will be archived on the site for at least one week following the presentation.

About Achondroplasia

Achondroplasia is caused by an autosomal dominant activating mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of bone growth. Eighty percent of cases are the result of a spontaneous mutation, and ninety-eight percent of those cases have a G380R mutation. Achondroplasia is the most common form of human dwarfism and is characterized by failure of normal conversion of cartilage into bone. Clinical manifestations of the disease include short stature, craniomedullary compression, apnea, bowed legs, frontal bossing and midface hypoplasia, permanent sway of the lower back, spinal stenosis, recurrent ear infections and obesity.

The rate of incidence of achondroplasia is one in 15,000 to one in 40,000 live births, with approximately 18,000 to 24,000 patients in the U.S. and Europe combined.

About BioMarin

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 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.

Forward-Looking Statement

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 achondroplasia, 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 trials related to the peptide therapeutic for achondroplasia; the content and timing of decisions by the U.S. Food and Drug Administration and other regulatory agencies, particularly with respect to the peptide therapeutic for achondroplasia, 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. 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® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.

Firdapse™ is a trademark of BioMarin Huxley Ltd.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.