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Investigational Schizophrenia Drug Hits Primary Endpoint in Phase 2 Trial
A once-daily, 20 mg dose of an investigational oral muscarinic M4 selective agonist in development for adults with schizophrenia achieved its primary endpoint in a phase 2 trial, maker Neurocrine Biosciences announced.
The randomized, double-blind inpatient study included 210 adults with schizophrenia experiencing an acute exacerbation or relapse of symptoms. Once-daily, 20 mg NBI-1117568 demonstrated a statistically significant 7.5-point improvement, compared with placebo, in Positive and Negative Syndrome Scale (PANSS) total score at week 6, the company reported. The improvement from baseline in PANSS total score was 18.2 points with the 20-mg dose.
Additionally, the 20 mg dose demonstrated statistically significant improvement in additional endpoints, including the Clinical Global Impression of Severity scale, Marder Factor Score – Positive Symptom Change, and Marder Factor Score – Negative Symptom Change.
NBI-1117568 was generally safe and well tolerated at all doses in the study, which included once-daily 20 mg, once-daily 40 mg, once-daily 60 mg, and twice-daily 30 mg. Discontinuation due to adverse events was similar between NBI-1117568 and placebo. Somnolence, dizziness, and headache were the most common adverse events. Gastrointestinal adverse events were low in frequency, and cardiovascular-related events were low in frequency and determined not to have clinical relevance at any dose. Few extrapyramidal symptom adverse events were reported, and NBI-1117568 was not linked with a greater increase in weight than placebo.
“This phase 2 dose-finding study delivered on our goal of identifying a once-daily, well-tolerated dosing regimen with a compelling and competitive benefit-risk profile,” said Eiry W. Roberts, MD, chief medical officer at Neurocrine Biosciences. “We recognize the significant need for new and innovative medicines to treat schizophrenia and look forward to advancing NBI-'568 [NBI-1117568], the first M4 selective agonist, into phase 3 development early next year.”
Previously, Neurocrine Biosciences had considered an 8-point placebo-adjusted improvement in PANSS as a marker of success for NBI-1117568, FirstWord Pharma reported. The publication added that, according to biotechnology analyst Brian Abrahams, MD, of RBC Capital Markets, New York, New York, achieving the best results with the lowest dose studied is “not something that is necessarily typical in this class — and something that is likely to call into question the replicability of the results in phase 3 (trials).”
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