#United States #Scottsdale #schizophrenia #Neurocrine Biosciences #NBI-1117568

Promising Results for NBI-1117568 in Schizophrenia Treatment

SAN DIEGO, May 28, 2025 – Neurocrine Biosciences, Inc. (Nasdaq: NBIX) recently presented encouraging data from its Phase 2 trial involving NBI-1117568, an innovative treatment for schizophrenia, during the American Society of Clinical Psychopharmacology 2025 Annual Meeting in Scottsdale, Arizona. The findings indicate a noteworthy improvement in the symptoms and severity of schizophrenia in adult participants.

NBI-1117568 is recognized as the first and only oral muscarinic M4 selective orthosteric agonist that is currently under investigation for schizophrenia treatment. This drug has shown significant results in managing symptoms, and the data highlights additionally its safety and tolerability profile, marking a hopeful step forward in schizophrenia management.

Dr. Eiry W. Roberts, Chief Medical Officer at Neurocrine Biosciences, emphasized the challenges that traditional treatment pathways impose on patients, stating, “Conventional therapies often lead to substantial short- and long-term complications, prompting many patients to discontinue treatment. Our goal is to present new, effective, and well-tolerated options for these individuals.” He further noted that NBI-1117568 selectively targets muscarinic M4 receptors, which play a critical role in regulating neurotransmitters affected by schizophrenia.

In the dose-finding study, conducted among adults aged 18 to 55 diagnosed with schizophrenia, participants were randomly assigned to receive either NBI-1117568 (doses of 20 mg, 40 mg, 60 mg once daily; or 30 mg twice daily) or a placebo. Notably, other antipsychotic drugs were prohibited throughout the trial's duration. The design featured a six-week double-blind period followed by a two-week safety assessment.

Findings indicated that NBI-1117568 was generally safe and well-tolerated across all doses evaluated, with treatment discontinuation rates due to adverse events mirroring those of the placebo group. The most common side effects observed were somnolence (10.7% for NBI-1117568 vs. 2.9% for placebo) and dizziness (9.3% vs. 1.4%, respectively). Importantly, any occurrence of increased heart rate was temporary and diminished over the treatment period, without presenting clinical significance. Furthermore, there was no reported weight gain associated with the drug compared to the placebo.

The primary endpoint was the change in the Positive and Negative Syndrome Scale (PANSS) score, measured from baseline to Week 6. Results demonstrated statistically significant reductions in PANSS total scores, particularly with the 20 mg dosage of NBI-1117568 observed as early as Week 3 and sustained through Week 6. An improvement was also noted by Week 2 in the Clinical Global Impression of Severity (CGI-S) scale, continuing through subsequent assessments.

Encouraged by these positive results, Neurocrine has embarked on a Phase 3 registrational program to further assess the clinical efficacy and safety of NBI-1117568 in schizophrenia treatment. This Phase 3 study is a global, randomized, double-blind, placebo-controlled trial, focusing on adults exhibiting an acute exacerbation or relapse of symptoms. Approximately 280 patients will be enrolled, targeting a reduction in the PANSS from the baseline as the main outcome, while the key secondary endpoint will assess improvements in the CGI-S scale.

For more details regarding the ongoing NBI-1117568 Phase 3 study, interested parties are encouraged to visit ClinicalTrials.gov.

Additional presentations at the ASCP 2025 annual event also spotlighted advancements including the efficacy of valbenazine in treating tardive dyskinesia, with findings elucidated in various posters including results from the Phase 4 KINECT-PRO study and the long-term effects of valbenazine treatment.

About NBI-1117568: This investigational compound stands out as the first muscarinic M4 selective orthosteric agonist undergoing clinical development for schizophrenia treatment. Muscarinic receptors, vital for neurotransmission in the brain, have been validated as therapeutic targets for psychosis and cognitive disorders. Through its selectivity, NBI-1117568 promises to provide an innovative therapeutic mechanism coupled with enhanced safety, negating the need for combination therapies that could bear unwanted side effects.

About Schizophrenia: Schizophrenia is a multifaceted disorder characterized by diverse symptoms, impacting around 24 million individuals globally according to the World Health Organization. In the United States, the financial burden associated with schizophrenia surpasses $150 billion annually. Traditional treatments heavily employing antipsychotic medication often result in substantial health implications, thus creating an urgent need for effective alternatives.

About Neurocrine Biosciences: Neurocrine Biosciences is a premier biopharmaceutical entity with a focus on neuroscience. Our mission centers around delivering transformative therapies for patients facing serious neurological, neuroendocrine, and neuropsychiatric conditions. Our portfolio comprises FDA-approved treatments alongside a robust pipeline, reflecting our commitment to advancing brain health through innovative scientific endeavors. For further information, visit neurocrine.com.