NBI-1117568 Phase III Trial

Positive top-line data for the Phase II clinical study in adults with schizophrenia were reported in August 2024.

"There is a significant need for new and innovative medicines to treat schizophrenia, a disorder that impacts millions of people and their families," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "With positive Phase II data in hand, we're excited to advance this investigational novel compound that works directly and selectively at the muscarinic M4 receptor."

The Phase III study is a global double-blind, placebo-controlled trial evaluating NBI 1117568 in adults with a primary diagnosis of schizophrenia who are experiencing an acute exacerbation or relapse of symptoms. The study is expected to enroll approximately 280 patients. The primary endpoint of the study is a reduction from baseline in the Positive and Negative Syndrome Scale (PANSS). The key secondary endpoint is improvement in the Clinical Global Impression of Severity (CGI-S) scale.

Neurocrine is initiating the Phase III study supported by positive top-line data from the Phase II clinical study, which met its primary endpoint for the once-daily 20 mg dose. The study found: i) A clinically meaningful and statistically significant reduction from baseline in the PANSS total score at Week 6 with a placebo-adjusted mean reduction of 7.5 points (p=0.011 and effect size of 0.61) and an 18.2-point reduction from baseline. ii) A statistically significant improvement across several secondary endpoints, including the CGI-S scale, Marder Factor Score – Positive Symptom Change, and Marder Factor Score – Negative Symptom Change. iii) NBI 1117568 was generally safe and well tolerated at all doses studied, with minimal gastrointestinal and cardiovascular adverse events.