BioMarin and IGAN Biosciences to Collaborate on Development of an Enzyme Therapy to Treat IgA Nephropathy, a Rare and Life-Threatening Kidney Disease
NOVATO, Calif., Dec. 3 -- BioMarin Pharmaceutical Inc. 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.
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