Press ReleaseNeurocrine Announces Expansion Of VMAT2 Inhibitor Program With Initiation Of Tourette Syndrome Clinical Study
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
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
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
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
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
Neurocrine Biosciences, Investor Relations, (858) 617-7600