Intra-Cellular Therapies, Inc. announced the publication in JAMA Psychiatry of Caplyta trial (ITI-007-301) in adult patients with schizophrenia.

Intra-Cellular Therapies, Inc. announced the publication of results from its Caplyta (lumateperone) clinical trial (ITI-007-301) in adult patients with schizophrenia. In this trial, Caplyta 42 mg met the primary endpoint and demonstrated antipsychotic efficacy with statistically significant superiority over placebo at Week 4 as measured by the change from baseline on the Positive and Negative Syndrome Scale (PANSS) total score (drug-placebo difference = -4.2 points). Caplyta 42 mg also met the key secondary endpoint of statistically significant improvement on the Clinical Global Impression Scale for Severity of Illness. Caplyta 42 mg showed significant antipsychotic efficacy as early as week 1, which was maintained at every time point throughout the study. The most commonly reported adverse events occurring for Caplyta 42 mg in greaater than 5% of patients and more than 2 times the rate in the placebo group were somnolence (Caplyta 42 mg, 17.3% vs placebo, 4.0%), sedation (12.7% vs 5.4%), fatigue (5.3% vs 1.3%), and constipation (6.7% vs 2.7%). No significant differences were observed compared to placebo on metabolic parameters, including weight, cholesterol, triglycerides, glucose and insulin, or on prolactin levels. No extrapyramidal symptoms (EPS)–related TEAEs occurred in 5% or more of patients in any treatment arm. Intra-Cellular Therapies is also presenting additional analyses of Study ITI-007-301 in a poster at the International Society for CNS Clinical Trials and Methodologies (ISCTM) Annual Scientific Meeting that further demonstrate the strength of the Study 301 results. This poster presents a post-hoc sensitivity analysis of Study ITI-007-301 that assesses the sensitivity of the prospective Mixed-effect Model for Repeated Measures (MMRM) based analysis, which assumes missing data as missing-at-random (MAR), under the alternative assumption that missing data were missing-not-at-random (MNAR). There was a higher rate of early discontinuations due to lack of efficacy in the placebo group compared with the lumateperone-treated group, suggesting that the MAR assumption that underlies the MMRM analysis may result in an underestimation of the drug-placebo difference by at least 32%. Using 3 other appropriate methods for dealing with imbalances in discontinuations due to lack of efficacy, lumateperone 42 mg was found to have substantially greater benefit compared with placebo than that obtained using the primary MMRM analysis, with drug-placebo differences of -6.2, -6.5, and -7.3 points on the PANSS in these analyses compared with -4.2 points in the primary analysis. Results from these additional sensitivity analyses confirm the efficacy of lumateperone 42 mg compared with placebo in Study ‘301. See-January 8, 2020- "Efficacy and Safety of Lumateperone for Treatment of Schizophrenia A Randomized Clinical Trial" -Christoph U. Correll, MD; Robert E. Davis, PhD; Michal Weingart, PhD; et al JAMA Psychiatry. Published online January 8, 2020. doi:10.1001/jamapsychiatry.2019.4379.