BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) and IGAN Biosciences announced today that they have initiated a program to develop an IgA protease for treating IgA nephropathy. IgA proteases have been shown to cleave IgA complexes, the deposition of which causes IgA nephropathy, an orphan kidney disorder with few treatment alternatives. BioMarin has executed a research and option agreement with IGAN Biosciences for intellectual property covering IgA proteases for treating IgA nephropathy.
"IgA proteases have the potential to reverse or prevent progression of IgA Nephropathy," said Dr. Andrew Plaut, discoverer of IgA proteases, Professor of Medicine at Tufts University School of Medicine, and Chairman of IGAN Biosciences. "Current treatments are not very effective, and nothing on the horizon looks very promising. BioMarin has a strong track record of aggressively developing disease modifying therapeutics for rare disorders, and I am very pleased that they have made a commitment to further these important efforts."
"Dr. Plaut's laboratory has performed pioneering work in the field of IgA proteases," Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin commented. "These proteases are exceptionally specific for IgA and show promise for treating the underlying pathophysiology of IgA nephropathy. In collaboration with IGAN and the Tufts-New England Medical Center, we look forward to testing these proteases in animal models of IgA nephropathy, and if successful, developing an efficacious therapeutic for the disorder."
"We are pleased to add this program to our growing product development pipeline. The IgA nephropathy indication aligns well with our development and commercial competencies as it is a protein therapeutic for treating a well- defined and relatively large orphan disorder treated by renal specialists," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Well validated markers of renal function and injury will help define a straightforward clinical and regulatory path, and this program also leverages our biologics manufacturing and process development infrastructure and expertise."
IgA nephropathy or Berger's disease is a primary glomerulonephritis characterized by mesangial deposits of IgA complexes. Over time, these deposits damage the kidney, causing 20% of adults with the disorder to progress to end stage renal disease (ESRD). Patients in ESRD require dialysis or a kidney transplant to survive. In the United States, approximately 800 patients per year develop ESRD caused by IgA nephropathy out of the 40,000 patients affected by the disorder.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of two 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, and Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease. For additional information, please visit http://www.bmrn.com/. Information on BioMarin's website is not incorporated by reference into this press release. About IGAN Biosciences
IGAN Biosciences develops novel, safe biotherapeutics for diseases in which current treatments are ineffective. The company's lead compound, a protease designed for treatment of IgA deposition diseases, was based upon research conducted at Tufts New England Medical Center. IGAN Biosciences is based in Boston, Massachusetts. For more information, please visit http://www.iganbio.com/.
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: BioMarin's products and product candidates, commercialization of BioMarin's products; 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: our success in the commercialization of BioMarin's products; 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 2006 Annual Report on Form 10-K, as amended, and the factors contained in BioMarin's reports on Form 10-Q and Form 8-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(R) and Naglazyme(R) are a registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC. Contact: Investors Media Eugenia Shen Susan Berg BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 506-6570 (415) 506-6594 Peter Bonis IGAN Biosciences (781) 626-1453
First Call Analyst:
SOURCE: BioMarin Pharmaceutical Inc.
CONTACT: Investors, Eugenia Shen, +1-415-506-6570, or Media, Susan Berg,
+1-415-506-6594, both of BioMarin Pharmaceutical Inc.; or Peter Bonis of IGAN