Neurocrine Biosciences, Inc. (NASDAQ: NBIX) has announced positive top-line data for its phase 2 clinical study of NBI-1117568 (NBI-’568) in adults with schizophrenia. The study showed that the once-daily 20 mg dose of NBI-’568 met the primary endpoint, demonstrating a statistically significant 7.5-point improvement in the PANSS total score compared to placebo at week 6. Additionally, the 20 mg dose also demonstrated statistically significant improvements in the Clinical Global Impression of Severity (CGI-S) scale and Marder Factor Score for positive and negative symptom change.
In terms of safety and tolerability, NBI-’568 was generally well tolerated at all doses studied in the phase 2 clinical trial. Adverse events with the highest incidence were somnolence, dizziness, and headache. Gastrointestinal adverse events, including nausea and constipation, were low in frequency and similar to placebo. Cardiovascular-related events were also low in frequency and were not deemed to have clinical relevance at any dose tested. Furthermore, NBI-’568 was not associated with a greater increase in weight than placebo.
The primary endpoint results at week 6 showed a placebo-adjusted mean reduction of 7.5 points in the PANSS total score, with an 18.2-point reduction from baseline for the 20 mg once-daily dose of NBI-’568. The effect size for this dose was calculated at 0.61.
Dr. Eiry Roberts, Chief Medical Officer at Neurocrine Biosciences, highlighted the significance of the phase 2 dose-finding study, stating that it delivered on the goal of identifying a once-daily, well-tolerated dosing regimen with a compelling and competitive benefit-risk profile. Dr. Maurizio Fava, Psychiatrist-in-Chief at Massachusetts General Hospital of Harvard University, emphasized that NBI-1117568 demonstrated a clinically meaningful and statistically significant reduction in PANSS scores and was well tolerated, with minimal gastrointestinal effects and no weight gain relative to placebo.
Neurocrine Biosciences plans to advance NBI-’568 into phase 3 development early next year based on the positive phase 2 results. The company also has a broad portfolio of assets in clinical development that selectively target muscarinic receptors, including NBI-1117567, NBI-1117569, NBI-1117570, and NBI-1076986.
The phase 2 clinical study was a multicenter, randomized, double-blind, placebo-controlled, multi-arm, multi-stage inpatient dose-finding study designed to assess the efficacy, safety, tolerability, and pharmacokinetics of NBI-’568 compared with placebo in adult subjects with a primary diagnosis of schizophrenia who are experiencing an acute exacerbation or relapse of symptoms. The study enrolled 210 participants.
Neurocrine Biosciences will be hosting a live conference call and webcast to discuss the results at 8:00 a.m. Eastern Time.
Following these announcements, the company's shares moved 0.2%, and are now trading at a price of $152.55. For more information, read the company's full 8-K submission here.
