Neurocrine Initiates Tourette Syndrome Clinical Trial

Neurocrine Biosciences, Inc.

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.