Neurocrine Biosciences has reported positive results from its phase 2 clinical trial for NBI-1117568, an investigational oral medication targeting schizophrenia.
The trial focused on evaluating the efficacy, safety and tolerability of the drug. The results showed that the once-daily 20 mg dose of NBI-1117568 met its primary endpoint, demonstrating a significant improvement in the Positive and Negative Syndrome Scale (PANSS) total score compared to placebo over six weeks.
The phase 2 trial included 210 adult participants with schizophrenia who were experiencing an acute exacerbation or relapse of symptoms. The 20 mg dose not only achieved a statistically significant reduction in PANSS scores but also showed improvements in other clinical measures, such as the Clinical Global Impression of Severity scale and the Marder Factor Scores for both positive and negative symptoms. The drug was generally well tolerated, with adverse events like somnolence, dizziness, and headache being reported but deemed manageable.
NBI-1117568 works by selectively targeting the M4 muscarinic receptor, a subtype of acetylcholine receptors involved in neurotransmission and brain function. This mechanism of action is designed to modulate psychotic symptoms without the need for combination therapy, potentially offering a safer profile with fewer off-target effects compared to existing treatments. The drug’s ability to act as an orthosteric agonist allows it to achieve efficacy without relying on the presence of acetylcholine, which could lead to more consistent therapeutic outcomes.
Armed with these promising results, Neurocrine plans to advance NBI-1117568 into phase 3 trials in early 2025.
After decades of being frozen in time, schizophrenia treatment barriers are beginning to melt. New drugs are looking to target different neurotransmitter systems, and research into prodrugs and formulations for once-daily dosing or long-acting injectables promise effective treatment with fewer side effects and improved adherence.