BioMarin Presents Data on Duchenne Muscular Dystrophy at the 67th American Academy of Neurology Annual Meeting
April 23 2015 - 4:51PM
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
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
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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 |
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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 |
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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 |
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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 |
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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 |
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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 |
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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 |
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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
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