BioMarin Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syn...
April 21 2016 - 4:30PM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that it
has enrolled the first patient in a Phase 1/2 trial for BMN 250, an
investigational enzyme replacement therapy using a novel fusion of
recombinant human alpha-N-acetyglucosaminidase (NAGLU) with a
peptide derived from insulin-like growth factor 2 (IGF2), for the
treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB
(MPS IIIB). Discovered by BioMarin, BMN 250 is being studied in a
multicenter, international clinical trial evaluating safety and
tolerability, as well as cognitive function of patients with MPS
IIIB receiving BMN 250. Designed to restore functional NAGLU
activity in the brain, BMN 250 is administered via
intracerebroventricular (ICV) infusion.
"Sanfilippo B has long been a difficult condition to treat due
to challenges in effectively delivering a therapy that bypasses the
blood brain barrier to address the underlying cause of the disease.
BMN 250 is designed to overcome these challenges by leveraging our
proprietary technology to deliver enzyme replacement therapies
directly to the brain,” said Hank Fuchs, M.D., Chief Medical
Officer at BioMarin. “BMN 250 is the result of building upon our
almost two decades of experience in developing treatments for MPS
and our most recent experience with an intracerebroventricular
delivery approach, which we have used with another experimental
enzyme replacement therapy delivered directly to the brain to treat
a form of Batten disease.”
The company also announced that BMN 250 has been granted orphan
drug designation by the European Commission. In late 2014, BMN 250
also received orphan drug designation from the U.S. Food and Drug
Administration. BioMarin has three approved therapies to treat
different forms of MPS. BMN 250 is a potential fourth therapy in
development for the treatment of an MPS disorder.
“Children with Sanfilippo syndrome need therapies that target
the root cause of this fatal genetic disease that currently has no
approved treatment options,” said Jill Wood, Co-Founder and
Treasurer, Jonah’s Just Begun Foundation to Cure Sanfilippo.
“We applaud companies like BioMarin who are bringing scientific
research into the clinic. This is a critical first step to
finding potential treatments.”
Study DesignThe BMN 250 development program
consists of two independent and complementary, multicenter,
international studies. BMN 250-901 is an observational study of the
progression of MPS IIIB over time in children. BMN 250-201 is
a Phase 1/2 treatment study conducted in two parts, with Part 1
focused on safety and dose escalation. Part 2 consists of eligible
patients rolling over from the BMN 250-901 observational study in
addition to continued treatment of the patients from Part 1 of the
study. Efficacy will be assessed by comparing changes in disease
progression in the observational BMN 250-901 study vs. changes
observed in Part 2 of the BMN 250-201 Phase 1/2 treatment
study.
About Sanfilippo B Syndrome
Mucopolysaccharidosis IIIB (MPS IIIB) or Sanfilippo B syndrome a
lysosomal storage disease is caused by deficiency in the enzyme
alpha-N-acetyglucosaminidase (NAGLU), one of the four enzymes
required for heparan sulfate (HS) degradation. There are an
estimated 2,000-3,000 patients in existing BioMarin territories who
are living with Sanfilippo B syndrome.
The first symptoms generally appear between the ages of two and
six years old, with behavior disorders, intellectual deterioration,
sleep disorders, and in some cases, very mild dysmorphism. The
neurological involvement becomes more prominent with progressive
loss of motor milestones and communication problems. The prognosis
is poor with death occurring in most cases in the late teens or
early 20s.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for people with serious and
life-threatening rare disorders. The company's portfolio consists
of five commercialized products and multiple clinical and
pre-clinical product candidates.
For additional information, please visit www.BMRN.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about the development plans for BMN
250 and expectations regarding the clinical trials for this product
candidate. 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: the results and timing of current and
planned preclinical and clinical studies; the content and
timing of decisions by the U.S. Food and Drug Administration, the
European Commission and other regulatory authorities; our ability
to successfully manufacture the product candidate for the
preclinical and clinical trials; 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 2015 Annual Report on Form
10-K, and the factors contained in BioMarin's reports on Form 10-Q.
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® is a registered trademarks of BioMarin Pharmaceutical
Inc.
Contact:
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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