SAN DIEGO, Oct. 30, 2014 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (NASDAQ: NBIX) announced today that the U.S. Food
and Drug Administration (FDA) has granted Breakthrough Therapy
Designation for its Vesicular Monoamine Transporter 2 inhibitor,
NBI-98854, in tardive dyskinesia. A breakthrough therapy
designation is granted for a drug that is intended to treat a
serious or life-threatening disease or condition and preliminary
clinical evidence indicates that the drug may demonstrate
substantial improvement on clinically significant endpoints over
available therapies. The breakthrough designation also allows
intensive discussions with the FDA which are intended to expedite
the development and review process of eligible drugs.
"We are pleased that after reviewing our dataset the FDA
recognizes that NBI-98854 is potentially an important therapy for
the treatment of tardive dyskinesia, and we look forward to working
closely with the Division of Psychiatry to advance our development
program," said Christopher F.
O'Brien, Chief Medical Officer of Neurocrine
Biosciences.
The Breakthrough Therapy Designation was granted, in part, based
on the results of the Phase IIb Kinect studies of NBI-98854 in
approximately 220 patients with tardive dyskinesia. Data from
the Phase IIb program were presented in June
2014 at the Annual Congress of Parkinson's Disease and
Movement Disorders in Stockholm,
Sweden.
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.
The Company recently initiated a Phase III study of NBI-98854 in
tardive dyskinesia, the Kinect 3 study. The Kinect 3 study,
along with the previous efficacy studies of NBI-98854, is designed
to complete the placebo-controlled clinical efficacy evaluation of
NBI-98854 for tardive dyskinesia. The Company also intends to
conduct a separate one-year open-label safety study of NBI-98854 to
support the anticipated 2016 filing of a New Drug Application with
the FDA in tardive dyskinesia.
In addition to the tardive dyskinesia clinical effort, the
Company has recently initiated a clinical study assessing NBI-98854
in children and adolescents with Tourette syndrome.
About Tardive Dyskinesia
Tardive dyskinesia is characterized by involuntary, repetitive
movements of the extremities: lip smacking, grimacing, tongue
protrusion, facial movements or blinking, puckering and pursing of
the lips, or involuntary movements of the limbs. These
symptoms are rarely reversible and there are currently no approved
treatments.
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 a wholly owned 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 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 clinical program will fail to demonstrate that
NBI-98854 is safe and effective; risk that the VMAT2 Phase III
program will be delayed for regulatory or other reasons; risk that
the Breakthrough Therapy designation may not result in an expedited
development and review process for NBI-98854 and may not lead to
regulatory approval; and risk that the Company will not be able to
submit an NDA filing for NBI-98854 tardive dyskinesia as planned;
and risk that the Company will be unable to complete the Tourette
syndrome Phase I clinical trial for regulatory or other reasons.
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.