SAN DIEGO, Dec. 16, 2015 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (NASDAQ: NBIX) announced today that it has
successfully completed the Phase Ib T-Force study of NBI-98854
(valbenazine), a once-daily, highly selective, small molecule
Vesicular Monoamine Transporter 2 (VMAT2) inhibitor. The T-Force
Study evaluated several doses of valbenazine in children and
adolescents with Tourette syndrome over two weeks of treatment.
Valbenazine was generally safe and well tolerated. The Yale Global
Tic Severity Scale was also assessed and after two weeks of
treatment showed a mean reduction of 31% from baseline scores, with
over half of the subjects considered clinical responders. The
Company intends to initiate a placebo-controlled Phase II study of
valbenazine in children and adolescents with Tourette syndrome
during the first half of 2016.
"We are very pleased with the pharmacokinetic profile as well as
the safety and tolerability demonstrated by valbenazine in the
T-Force study evaluating children and adolescents with Tourette
syndrome," said Christopher F.
O'Brien, Chief Medical Officer of Neurocrine. "The 31%
reduction in Tourette syndrome symptoms as measured by the Yale
Global Tic Severity Scale after just two weeks of dosing was
corroborated by an improvement in Tourette symptomology as assessed
by the treating physician using the Clinical Global Impression in
Tourette Syndrome Scale. We will use the PK data to support
pediatric dose selection and look forward to initiating a
placebo-controlled Phase II study in children and adolescents with
Tourette's in 2016."
T-Force Study Design
The T-Force study was an open-label, multiple ascending dose,
pharmacokinetic and pharmacodynamic, study that evaluated the
safety, tolerability and exposure-response of valbenazine in
children and adolescents with Tourette syndrome. A total of 28
subjects were evaluated over 14 days of once-daily dosing followed
by seven days off-drug at approximately 10 study centers in
the United States. The study was
divided into two dosing groups consisting of children (ages 6-11)
and adolescents (ages 12-18), and each age group was further
divided into three dosing cohorts. Subsequent dose escalations for
children and adolescents were based, in part, on the
pharmacokinetic and safety data from the previous cohort in each
age group. Additionally, the patients' Tourette symptoms were
evaluated weekly via the Yale Global Tic Severity Scale, the
Premonitory Urge for Tics Scale as well as an overall Clinical
Global Impression in Tourette Syndrome Scale.
About Tourette Syndrome
Tourette syndrome is a neurological disorder that consists of
rapid, non-rhythmic stereotyped motor and vocal tics. Motor tics
are typically characterized by facial grimacing, head jerks,
extremity movements and other dystonic movements. Vocal tics
typically include grunting, throat clearing, and repeating words
and phrases. The average age at onset for Tourette syndrome is at
age six, with symptoms reaching their peak severity at
approximately age ten. Tourette syndrome is more commonly diagnosed
in males than females and may be associated with attention deficit
hyperactivity disorder and obsessive compulsive disorder. There are
approximately 400,000 people with Tourette syndrome in the United States.
About NBI-98854
VMAT2 is a protein concentrated in the human brain that is
primarily responsible for re-packaging and transporting monoamines
(dopamine, norepinephrine, serotonin, and histamine) in
pre-synaptic neurons. NBI-98854, developed in the Neurocrine
laboratories, is a novel, highly-selective VMAT2 inhibitor that
modulates dopamine release during nerve communication, while at the
same time having minimal impact on the other monoamines, thereby
reducing the likelihood of "off-target" side effects. NBI-98854 is
designed to provide low, sustained, plasma and brain concentrations
of active drug to minimize side effects associated with excessive
monoamine depletion.
Modulation of neuronal dopamine levels in diseases such as
tardive dyskinesia, Tourette syndrome, Huntington's chorea,
schizophrenia, and tardive dystonia, which are characterized, in
part, by a hyperdopaminergic state, should provide symptomatic
benefits for patients with these diseases.
Neurocrine has received Breakthrough Therapy Designation from
the FDA for NBI-98854 in the treatment of tardive dyskinesia and
expects to file a New Drug Application for tardive dyskinesia in
2016.
About Neurocrine Biosciences
Neurocrine Biosciences, Inc. discovers and develops innovative
and life-changing pharmaceuticals, in diseases with high unmet
medical needs, through its novel R&D platform, focused on
neurological and endocrine based diseases and disorders. The
Company's two lead late-stage clinical programs are elagolix, a
gonadotropin-releasing hormone antagonist for women's health that
is partnered with AbbVie Inc., and NBI-98854, a vesicular monoamine
transporter 2 inhibitor for the treatment of movement disorders.
Neurocrine intends to maintain certain commercial rights to its
VMAT2 inhibitor for evolution into a fully-integrated
pharmaceutical company.
Neurocrine Biosciences, Inc. news releases are available through
the Company's website via the internet at
http://www.neurocrine.com.
In addition to historical facts, this press release may
contain forward-looking statements that involve a number of risks
and uncertainties. Among the factors that could cause actual
results to differ materially from those indicated in the
forward-looking statements are risks and uncertainties associated
with Neurocrine's business and finances in general, as well as
risks and uncertainties associated with NBI-98854 development.
Specifically, the risks and uncertainties the Company faces include
risks that NBI-98854 development activities for either tardive
dyskinesia or Tourette syndrome, or both, may not be completed on
time or at all; risks that NBI-98854 development activities for
either tardive dyskinesia or Tourette syndrome, or both, may be
delayed for regulatory or other reasons, may not be successful or
replicate previous clinical trial results, may fail to demonstrate
that NBI-98854 is safe and effective for either tardive dyskinesia
or Tourette syndrome, or both,, or may not be predictive of
real-world results or of results in subsequent clinical trials;
risks that NBI-98854 regulatory submissions may not occur or be
submitted in a timely manner; risks that NBI-98854 may not
obtain regulatory approval for either tardive dyskinesia or
Tourette syndrome, or both, or that the U.S. Food and Drug
Administration or regulatory authorities outside the U.S. may make
adverse decisions regarding NBI-98854; risks that NBI-98854 may be
precluded from commercialization by the proprietary rights of third
parties, or have unintended side effects, adverse reactions or
incidents of misuse; risks associated with the Company's dependence
on third parties for development and manufacturing activities
related to NBI-98854; risks that the Company will be unable to
raise additional funding, if required, to complete development of
NBI-98854; risks and uncertainties relating to competitive products
and technological changes that may limit demand for NBI-98854; and
other risks described in the Company's quarterly report on Form
10-Q for the quarter ended September 30,
2015. Neurocrine disclaims any obligation to update the
statements contained in this press release after the date
hereof.
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SOURCE Neurocrine Biosciences, Inc.