BUCK INSTITUTE PARTNERS WITH BIOMARIN TO EXPAND EARLY RESEARCH ON NOVEL TREATMENT FOR FAMILIAL ALZHEIMER’S DISEASE

The Buck Institute for Age Research (Buck) announced today that it has entered into a research collaboration with BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to evaluate therapeutic approaches for treatment of early onset Familial Alzheimer’s Disease (eFAD), an autosomal dominant form of Alzheimer’s disease that typically presents at 30 – 65 years of age. eFAD is an orphan disorder with a prevalence of approximately 34,000 in the US. Targeting eFAD represents a good strategic fit with BioMarin’s core competencies in developing protein-based therapeutics for rare genetic diseases.

AD Research at the Buck Institute focuses on signal transduction pathways that may explain all of the different, yet seemingly opposed theories of AD, namely that AD is caused by either an overabundance of amyloid-β (Aβ) peptide or neurofibrillary tangles that build up inside the nerve cells.  Current studies on AD at the Buck take place in the laboratory of Dale Bredesen, MD, where the researchers have shown in both cell culture and mice the inhibition of the production of amyloid-β (Aβ) peptides while simultaneously facilitating the growth and preservation of nerve fibers in the brain. As part of this agreement, BioMarin has agreed to fund a joint research collaboration for up to two years with the Buck and the Bredesen laboratory.

“With our resources and the support from BioMarin, we are well positioned to extend our studies into the mechanisms and potential treatments for AD, and to hopefully realize our shared goal of developing drugs to bring new hope to patients suffering from AD, the most feared of all age-related diseases,” said Brian K. Kennedy, PhD, CEO of the Buck Institute. “Aging research has made great progress and moving forward, this collaboration with BioMarin supports our efforts to translate our basic science into effective therapies.  This is the core of our mission to extend the healthy years of life.”

About the Buck Institute

The Buck Institute is an independent non-profit organization dedicated to extending the healthspan, the healthy years of each individual’s life.  Buck Institute scientists work in an innovative, interdisciplinary setting to understand the mechanisms of aging and to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimer’s and Parkinson’s disease, cancer, stroke, and arthritis.  Collaborative research at the Institute is supported by genomics, proteomics and bioinformatics technology.  For more information: www.buckinstitute.org.

About BioMarin Pharmaceutical Inc.

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 PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU; and GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in clinical development for the treatment of MPS IVA. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.