Neurocrine Biosciences Launches Phase 3 Trials for NBI-1117568 in Schizophrenia Treatment

Neurocrine Biosciences, Inc., based in San Diego, has announced the launch of a Phase 3 clinical trial to assess the potential of NBI-1117568, an experimental oral treatment targeting schizophrenia. This study follows encouraging results from a Phase 2 trial, which were unveiled in August 2024, demonstrating the drug's promise in managing this mental disorder.

Eiry W. Roberts, M.D., who serves as Chief Medical Officer at Neurocrine Biosciences, emphasized the need for groundbreaking treatments for schizophrenia, a condition affecting millions worldwide. The Phase 3 trial will investigate the efficacy, safety, and tolerability of NBI-1117568, specifically focusing on its action at the muscarinic M4 receptor. This receptor plays a vital role in neurotransmission and brain function, marking the drug as a potential new approach to treating schizophrenia.

The upcoming study is designed as a global, double-blind, and placebo-controlled trial, targeting adults diagnosed with schizophrenia who are experiencing acute symptom exacerbations or relapses. Approximately 280 participants are expected to enroll, with the primary aim of observing a reduction in the Positive and Negative Syndrome Scale (PANSS) scores. An additional focus will be on evaluating improvements in the Clinical Global Impression of Severity (CGI-S) scale.

Data from the earlier Phase 2 study indicated significant findings. Notably, the study achieved its primary endpoint with a 20 mg daily dose, showcasing a 7.5-point reduction in PANSS total score compared to placebo, with a total decrease of 18.2 points from baseline after six weeks. The study also recorded substantial improvements in various secondary endpoints, including the CGI-S scale and other symptoms related to schizophrenia. Importantly, NBI-1117568 was well tolerated, with minimal adverse effects on the gastrointestinal and cardiovascular systems.

NBI-1117568 distinguishes itself as the first investigational oral therapy targeting the M4 receptor in schizophrenia treatment. Muscarinic receptors, integral to brain processes, are crucial targets in addressing psychosis and cognitive disorders. NBI-1117568’s distinctive mechanism could lead to a favorable safety profile, minimizing the need for combination therapies that often introduce additional side effects.

Neurocrine Biosciences is also exploring other compounds in its muscarinic receptor-targeted portfolio. This includes NBI-1117567, NBI-1117569, and NBI-1117570, which the company obtained from Nxera Pharma. Additionally, Neurocrine is developing NBI-1076986, an M4 antagonist discovered internally.

Schizophrenia is a complex mental illness characterized by a range of diverse symptoms. The World Health Organization estimates that it affects roughly 24 million individuals globally, with the United States alone incurring annual costs exceeding $150 billion due to the disorder. As one of the major causes of disability around the world, schizophrenia imposes significant challenges both emotionally and functionally on patients and their families. The condition's roots are believed to involve a mixture of genetic and environmental factors, and current treatments predominantly rely on antipsychotic drugs, which can pose long-term health risks.

Neurocrine Biosciences is a leading biopharmaceutical company focused on neuroscience, aiming to create life-changing treatments for neurological, neuroendocrine, and neuropsychiatric conditions. Its diverse portfolio includes FDA-approved therapies for various disorders and a strong pipeline of compounds in advanced stages of development. For three decades, the company has been committed to advancing neuroscience and addressing complex health issues to improve patient lives.