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BioMarin Presents Data on Duchenne Muscular Dystrophy at the 67th American Academy of Neurology Annual Meeting

Interim analysis of prospective natural history increases understanding of Duchenne muscular dystrophy

BioMarin to present data from seven abstracts on Duchenne and Huntington's disease at AAN 2015

SAN RAFAEL, Calif., April 23, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the results of a natural history study designed to prospectively evaluate the progression of patients with Duchenne muscular dystrophy (DMD) at the 67th American Academy of Neurology (AAN) Annual Meeting in Washington, D.C., April 18-25, 2015. This observational study of nearly 270 pediatric patients (between three and 18 years of age) was designed to characterize the progression of DMD over a three year period to provide information that may help in the development of therapeutic clinical trials. The results of the 12-month interim analysis of 77 ambulatory patients further describes the relationship between baseline characteristics, including age and six-minute walking distance and the trajectory of disease progression. These findings are consistent with that reported in other natural history studies.1

"These initial results are important to help us understand the progression of the disease as boys and young men with Duchenne muscular dystrophy grow and develop. Understanding the clinical course of the disease is critical to the design and analysis of clinical studies that will help make treatment a reality," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "We are grateful to the boys and families who are participating in this ongoing study."

Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12 years. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.

"We appreciate BioMarin's commitment to driving forward meaningful research that will help the Duchenne community better understand the course of this devastating disease," said Valerie A. Cwik, M.D., Executive Vice President and Chief Medical and Scientific Officer at the Muscular Dystrophy Association. "We are optimistic that BioMarin's contribution to the growing body of scientific information could expedite the development of treatment options for boys with Duchenne."

Study Design

PRODMD-01 (NCT01753804) is a prospective, multi-center, exploratory, observational study of physical impairment, activity limitation and quality of life in young males with Duchenne muscular dystrophy, resulting from a mutation of the DMD gene. No medication or treatment are being evaluated in this study. A total of 269 males, between the ages of three and 18 years and a life expectancy of greater than three years, were enrolled in the study. Investigators are monitoring the subjects as they perform various physical tests, and complete quality of life questionnaires to help with determining how this condition progresses over time. Patients are to be assessed every six months over a period of three years. Certain biomarkers are measured in blood and urine samples to investigate a possible relation with disease progression.

BioMarin Poster Presentations at the 2015 AAN Meeting

POSTER SESSION

DATE/TIME
POSTER NUMBER TITLE AUTHORS
II
Tuesday,
April 21
230 Drisapersen: An Overview of the Exon-51 Skipping Antisense Oligonucleotide Clinical Program to Date in Duchenne Muscular Dystrophy (DMD) McDonald C, Goemans N, Voit T,
Wilson R, Wardell C, Campion G
7:30AM-12:00PM
II
Tuesday,
April 21
238 Evaluating the Progression of Physical Impairment, Activity Limitation, and Quality of Life in Duchenne Muscular Dystrophy: A Prospective Natural History Study Goemans N, Wong B, McDonald CM, Jones A, Hall A, Mason C, Campion G
7:30AM-12:00PM
II
Tuesday,
April 21
239 Identification of Serum Biomarkers for Duchenne Muscular Dystrophy Giannakopoulos S, Lourbakos A,
Campion G, de Kimpe S
7:30AM-12:00PM
II
Tuesday,
April 21
240
Pooled Analyses of Efficacy Parameters in Patients with Duchenne Muscular Dystrophy (DMD): Results from the Drisapersen (DRIS) Clinical Trial Program Goemans N, Voit T, McDonald CM,
Hall A, Wilson R, Wardell C, Campion G
7:30AM-12:00PM
V
Wednesday, April 22
297 Therapeutic Benefit of an HTT-Lowering Antisense Oligonucleotide Targeting the CAG-Repeat in the R6/2 Huntington's Disease Mouse Model Datson N, Mulders S, van der Giessen J, Gonzalez A, Campion G, van Deutekom J
2:00PM-6:30PM
VI
Thursday,
April 23
255 Evaluation of Efficacy and Safety Baseline Parameters in Patients with Duchenne Muscular Dystrophy (DMD) from Three Placebo-controlled Studies of Drisapersen (DRIS) Campion G, Voit T, Goemans N,
Wilson R, Wardell C, McDonald CM
7:30AM-12:00PM
VII
Thursday,
April 23
059 Magnetic Resonance Imaging Assessments of Two Doses of Drisapersen in the Treatment of Ambulant Boys with Duchenne Muscular Dystrophy Bishop CA, Janiczek RL, Newbould RD, Dorricott S, Hall A, Campion G
2:00PM - 6:30PM

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises five approved products and multiple clinical and pre-clinical product candidates. Approved products include Vimizim® (elosulfase alfa) for MPS IVA, a product wholly developed and commercialized by BioMarin; Naglazyme® (galsulfase) for MPS VI, a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for MPS I, a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Powder for Oral Solution and 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 drisapersen, an exon skipping oligonucleotide, which is currently undergoing regulatory submission for the treatment of Duchenne muscular dystrophy (exon 51), pegvaliase (PEGylated recombinant phenylalanine ammonia lyase, formerly referred to as BMN 165 or PEG PAL), which is currently in Phase 3 clinical development for the treatment of PKU, talazoparib (formerly referred to as BMN 673), a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, reveglucosidase alfa (formerly referred to as BMN 701), a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase 3 clinical development for the treatment of Pompe disease, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 2 clinical development for the treatment of achondroplasia, BMN 044, BMN 045 and BMN 053, exon skipping oligonucleotides, which are currently in Phase 2 clinical development for the treatment of Duchenne muscular dystrophy (exons 44, 45 and 53), cerliponase alfa (formerly referred to as BMN 190), a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of CLN2 disorder, a form of Batten disease, which is currently in Phase 1, BMN 270, an AAV-factor VIII vector, for the treatment of hemophilia A and BMN 250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of MPS IIIB.

For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

BioMarin®, Naglazyme®, Kuvan®, Firdapse® and VIMIZIM® are registered trademarks of BioMarin Pharmaceutical Inc.

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

1. Pane M, et al. PLOS One 2014; 9:e109205.

CONTACT: Investors: Traci McCarty BioMarin Pharmaceutical Inc. (415) 455-7558 Media: Debra Charlesworth BioMarin Pharmaceutical Inc. (415) 455-7451

Source:BioMarin Pharmaceutical Inc.