SAN DIEGO, Oct. 2, 2014 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (NASDAQ: NBIX) announced today that it has
initiated a clinical trial of NBI-98854, a proprietary small
molecule Vesicular Monoamine Transporter 2 (VMAT2) inhibitor, in
both children and adolescents with Tourette syndrome.
The T-Force study is an open-label, multi-dose, two-week study
of 36 subjects with Tourette syndrome. Children and adolescents
will receive once-daily dosing of NBI-98854 during a two-week
treatment period to assess both the safety and tolerability of
NBI-98854 in Tourette patients. Additionally, the Yale Global Tic
Severity Scale and the Premonitory Urge for Tics Scale will be
employed during the study to assess the impact of NBI-98854 on the
patients' Tourette symptoms. Data readout from this study is
expected in 2015.
"Advancing NBI-98854 into clinical evaluation of Tourette
syndrome represents another significant achievement for our VMAT2
franchise," said Kevin C. Gorman,
President and Chief Executive Officer of Neurocrine Biosciences.
"An important aspect of this initial Tourette syndrome trial is
that we are exploring NBI-98854 in children age six to eleven, the
target age range for therapy."
T-Force Study Design
The T-Force study is an open-label, multiple ascending dose,
pharmacokinetic and pharmacodynamic, study to evaluate the safety,
tolerability and exposure-response of NBI-98854 in children and
adolescents with Tourette syndrome. A total of 36 patients will be
evaluated over 14 days of once-daily dosing followed by 7 days
off-drug at approximately 10 study centers in the United States. The study will be divided
into two dosing groups consisting of children (ages 6-11) and
adolescents (ages 12-18), and each age group will be further
divided into three dosing cohorts of six patients each. After
completing the initial two weeks of dosing with the first
adolescent cohort, an independent review of both safety and
pharmacokinetic results will occur prior to escalating the dose
level for the second cohort of adolescents. In parallel, while
initiating the second cohort of adolescents, the first cohort of
children (ages 6-11) will also be administered NBI-98854 for a
two-week period. Subsequent dose escalations for children and
adolescents will be based, in part, on the pharmacokinetic and
safety data from the previous cohort in each age group.
Additionally, the patient's Tourette symptoms will be 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 of onset for Tourette syndrome is at
six years, 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.
In addition to this Tourette syndrome study, the Company will
initiate the Phase III pivotal study assessing NBI-98854 in tardive
dyskinesia during the fourth quarter of 2014.
The Company has two distinct Investigational New Drug
Applications, tardive dyskinesia and Tourette syndrome, open with
the Division of Psychiatry Products at the FDA.
About Neurocrine Biosciences
Neurocrine Biosciences, Inc. is a clinical stage drug discovery
company primarily focused on neurological and endocrine based
diseases and disorders. The Company discovers and develops
innovative pharmaceuticals, in diseases with high unmet medical
needs or where the existing drug classes are inadequate, through a
disciplined yet entrepreneurial process. Utilizing a portfolio
approach to drug discovery, Neurocrine has multiple small molecule
drug candidates at various stages of pharmaceutical development.
Neurocrine's two lead late stage clinical programs are elagolix, a
GnRH antagonist for women's health that is partnered with AbbVie
Inc., and a wholly owned VMAT2 inhibitor for the treatment of
movement disorders. 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 the Company's VMAT2 program
and the Company overall. Specifically, the risks and uncertainties
the Company faces with respect to the Company's VMAT2 program
include, but are not limited to; risk that the Company's
VMAT2 Phase III program in tardive dyskinesia will be delayed for
regulatory or other reasons; risk that the guidance provided by the
FDA in the End-of-Phase II meeting may be modified or may not lead
to regulatory approval; risk that the Company will be unable to
complete the T-Force clinical trial in Tourette syndrome for
regulatory or other reasons; risk that NBI-98854 will not proceed
to later stage clinical trials in Tourette syndrome; and risk that
the Company's Phase III or other clinical trials will fail to
demonstrate that NBI-98854 is safe and effective. With respect to
its business overall, the Company faces risk that it will be unable
to raise additional funding required to complete development of all
of its product candidates; risk relating to the Company's
dependence on contractors for clinical drug supply, commercial
manufacturing and marketing and sales activities; uncertainties
relating to patent protection and intellectual property rights of
third parties; risks associated with the Company's dependence on
corporate partners for development, commercial manufacturing and
marketing and sales activities for the Company's partnered
programs; risks and uncertainties relating to competitive products
and technological changes that may limit demand for the Company's
products if approved. The Company also faces the other risks
described in the Company's annual report on Form 10-K for the year
ended December 31, 2013 and quarterly
reports on Form 10-Q for the quarters ended March 31, 2014 and June
30, 2014. Neurocrine undertakes no obligation to update the
statements contained in this press release after the date
hereof.
SOURCE Neurocrine Biosciences, Inc.