Neurocrine reports mixed mid-stage schizophrenia study results

Neurocrine Biosciences recently announced mixed outcomes from its Phase II trial of an experimental medication designed for adults with schizophrenia. The trial, which evaluated the effects of varying doses of the oral, muscarinic M4 selective agonist known as NBI-1117568, showed promising results only at the lowest dose. This development led to a 16% drop in the company's share price. Despite this setback, the company is determined to proceed with late-stage development early next year.

The study included 210 adults suffering from schizophrenia, specifically those experiencing an acute exacerbation or relapse of symptoms. Participants were randomly assigned to receive different doses of NBI-1117568 or a placebo. The results revealed that only the once-daily 20-mg dose showed a significant and clinically meaningful reduction in the Positive and Negative Syndrome Scale (PANSS) total score after six weeks. This dose achieved a placebo-adjusted mean reduction of 7.5 points.

Additionally, the 20-mg dose demonstrated statistically significant improvements in several other clinical measures. These included the Clinical Global Impression of Severity (CGI-S) scale, the Marder Factor Score for Positive Symptom Change, and the Marder Factor Score for Negative Symptom Change. Eiry Roberts, the Chief Medical Officer of Neurocrine, commented on the study's findings, emphasizing that the results achieved their goal of identifying a well-tolerated, once-daily dosing regimen with a favorable benefit-risk profile.

Prior to the study's readout, Neurocrine had set an 8-point placebo-adjusted mean reduction in PANSS as the benchmark for success for NBI-1117568. While the 20-mg dose nearly met this target, other doses fell short, with placebo-adjusted mean reductions ranging from 1.9 points for the 40-mg once-daily dose to 5 points for the 30-mg twice-daily dose.

The company also noted that NBI-1117568 was generally safe and well tolerated at all doses examined during the trial. Adverse gastrointestinal events, such as nausea and constipation, were infrequent and occurred at rates similar to those observed with the placebo group.

Despite the mixed results, Neurocrine remains optimistic and plans to advance NBI-1117568 into late-stage development. The company aims to refine the dosing regimen to maximize the drug's therapeutic benefits while minimizing side effects. This ongoing commitment underscores Neurocrine's dedication to developing innovative treatments for schizophrenia, a condition that profoundly impacts the lives of those affected and presents a significant challenge within the field of mental health.

As Neurocrine moves forward, the focus will be on further validating the efficacy and safety of the 20-mg dose. The upcoming stages of development will likely include additional trials designed to confirm these findings and potentially explore the drug's effects over a longer duration or in combination with other therapies. The ultimate goal is to offer a new, effective treatment option for individuals with schizophrenia, providing them with improved symptom management and a better quality of life.

With these next steps, Neurocrine Biosciences continues to contribute to the growing body of research aimed at addressing the complex needs of patients with schizophrenia, demonstrating a commitment to advancing mental health care.