- Clinical-stage biopharma Neurocrine Biosciences signaled optimism in one of its central nervous system drugs on Tuesday despite the candidate failing a Phase 2 that tested it as a treatment for Tourette syndrome.
- While the drug, Ingrezza (valbenazine), alleviated the motor tics characteristic of Tourette better than placebo, it did not meet the T-Forward trial's primary endpoint. Neurocrine didn't give additional details, though, in order to avoid a potential assessment bias, according to a statement.
- Ingrezza inhibits a protein called vesicular monoamine transporter 2 (VMAT2), which moves neurotransmitters such as dopamine across cells. Though the exact cause of Tourette isn't known, research suggests the regulation of neurotransmitters plays some role in the disease's development.
The only Food and Drug Administration-approved treatment for the motor tics characteristic of Tourette are the antipsychotics haloperidol, pimozide and aripiprazole, according to the Tourette Association of America.
The relatively open market has not gone unnoticed, with big players such as Teva jumping at the chance to get in on the action. The Israeli drugmaker proved this in early 2015, dropping $3.2 billion to acquire Auspex Pharmaceuticals and access to the target's central nervous system drugs aimed at Huntington's disease, tardive dyskinesia and Tourette.
T-Forward analyzed the effects of Ingrezza on 124 adults with severe Tourette syndrome over the course of 10 weeks using the Yale Global Tic Severity Scale. While the drug didn't demonstrate a significant improvement from baseline by week eight — the primary endpoint of the study — Neurocrine remained optimistic about its prospects for other diseases.
"Through this initial placebo-controlled study we gained valuable insights into the conduct of clinical trials in Tourette patients, including patient identification methods and appropriate study inclusion/exclusion criteria, the company's Chief Medical Officer Chris O'Brien said a statement.
The company submitted a new drug application (NDA) for Ingrezza as a tardive dyskinesia treatment in mid-October. The FDA granted the medication priority review, meaning a decision on its approval should come no later than March. Neurocrine is also collaborating on an endometriosis drug with AbbVie, and plans to submit an NDA for it early this year.