#United States #San Diego #schizophrenia #Neurocrine Biosciences #NBI-1117568

Neurocrine Biosciences Announces Phase 3 Trial for Schizophrenia Treatment NBI-1117568

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) has officially launched a Phase 3 registrational program to investigate the efficacy and safety of NBI-1117568, a novel oral muscarinic M4 selective orthosteric agonist. This comes after promising results from a Phase 2 clinical trial, where significant improvements were observed in adults suffering from schizophrenia.

In the statement released on April 30, 2025, Eiry W. Roberts, M.D., Chief Medical Officer of Neurocrine, emphasized the urgent need for new treatments in the realm of schizophrenia. He mentioned, "Schizophrenia affects millions globally, and with our successful Phase 2 data, we are eager to push forward this innovative therapy that acts selectively on the muscarinic M4 receptor."

The Phase 3 study is designed as a global, double-blind, placebo-controlled trial targeting adults with schizophrenia who are currently experiencing acute symptom relapses. About 280 participants are expected to enroll, and the primary goal will be to evaluate changes in the Positive and Negative Syndrome Scale (PANSS) from their baseline scores. Furthermore, improvements in the Clinical Global Impression of Severity (CGI-S) scale will serve as a key secondary measure.

Overview of Phase 2 Study Results

The motivation behind this new Phase 3 study stems from the encouraging results of the Phase 2 trial that reported key findings.

• - A significant reduction of 7.5 points in the PANSS total score at Week 6 when compared to placebo (p=0.011, effect size of 0.61).
• - Researchers noted an 18.2-point decrease from baseline scores, indicating clinically meaningful improvement.
• - Additionally, improvements were recorded across secondary endpoints, which included enhancements in both the CGI-S and various Marder Factor Scores related to positive and negative symptom changes.
• - The investigational drug was generally well received, showing minimal gastrointestinal and cardiovascular side effects.

NBI-1117568: A Unique Approach

NBI-1117568 sets itself apart as the first oral muscarinic M4 selective orthosteric agonist currently in clinical trials for schizophrenia. With five types of muscarinic acetylcholine receptors influencing neurotransmission, this drug offers a new pathway for treatment. Its targeted action aims to reduce off-target effects often seen with broader antipsychotic medications.

Neurocrine’s Commitment to Neuroscience

Neurocrine Biosciences is dedicated to addressing serious neurological and psychiatric disorders, and the company's robust pipeline includes several other muscarinic receptor-targeting compounds. Along with NBI-1117568, assets such as NBI-1117567, NBI-1117569, and NBI-1117570 are also being developed. Moreover, NBI-1076986, focusing on M4 antagonism for movement disorders, represents their comprehensive strategy in neuroscientific research.

Understanding Schizophrenia

Schizophrenia is a multifaceted disorder that influences approximately 24 million individuals worldwide and comes with a staggering annual economic burden exceeding $150 billion in the United States alone. As one of the leading causes of disability, the condition's impact stretches beyond the individual, affecting families and communities. Current treatments primarily involve antipsychotic medications; however, these come with their own set of challenges, including side effects and varying degrees of efficacy.

Conclusion

Neurocrine Biosciences' Phase 3 study of NBI-1117568 represents a beacon of hope for many who navigate the complexities of schizophrenia. By advancing innovative treatment options with a focus on safety and efficacy, they continue to fulfill their mission of alleviating suffering through neuroscience. As the study progresses, both the company and the medical world will eagerly await the outcomes that could reshape treatment approaches in the field of mental health.