Neurocrine Biosciences has announced that FDA has
granted breakthrough therapy designation for NBI-98854 (Vesicular Monoamine
Transporter 2 inhibitor) drug 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.
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.
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.
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.
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.
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
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.
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