Intra-Cellular Therapies (ITCI) has announced positive topline results from Study 304, a Phase 3 trial evaluating CAPLYTA (lumateperone) for the prevention of relapse in patients with schizophrenia. The study demonstrated a statistically significant longer time to relapse in patients treated with lumateperone compared to placebo (p=0.0002). Only 16.4% of lumateperone patients relapsed compared to 38.6% in the placebo group, representing a 63% reduction in relapse risk. The drug was generally well-tolerated, with headache being the most common adverse event. The study also met its key secondary endpoint of time to all-cause discontinuation (p=0.0007).
Key Findings from Study 304
The Phase 3 trial was a multicenter, multi-national, randomized, double-blind, placebo-controlled, parallel-group study. It included an 18-week open-label phase where patients with schizophrenia were treated with lumateperone 42 mg per day. Patients who met the stabilization criteria during the open-label period were then randomized to either continue on lumateperone 42 mg (N=114) or switch to placebo (N=114) for up to 26 weeks or until relapse. The primary endpoint was time to first symptom relapse, and the key secondary endpoint was time to all-cause discontinuation during the double-blind phase.
Dr. Suresh Durgam, Executive Vice President and Chief Medical Officer of Intra-Cellular Therapies, stated, "The control of symptoms and the prevention of relapses is critical to improving long-term patient outcomes. We are very pleased that the results from Study 304, a randomized withdrawal trial, demonstrated efficacy along with favorable safety and tolerability which support the benefit of continued long-term treatment with lumateperone."
Clinical Implications and Market Impact
The results of Study 304 represent a significant advancement in the maintenance treatment of schizophrenia. The 63% reduction in relapse risk is clinically meaningful and supported by strong statistical significance (p=0.0002). This data strengthens CAPLYTA's position in the schizophrenia market, valued at $6.3 billion. The favorable safety profile, with headache as the most notable side effect, gives CAPLYTA a competitive advantage over other antipsychotics known for metabolic and movement-related adverse effects.
The maintenance therapy indication could expand prescription duration and potentially increase the lifetime value per patient. Analysts anticipate that these results will likely drive significant revenue growth for ITCI. The maintenance therapy indication expands CAPLYTA's addressable market and should increase prescription persistence. The strong efficacy data strengthens CAPLYTA's competitive position against established antipsychotics.
Safety and Tolerability
In this study, lumateperone was generally safe and well-tolerated. In the double-blind phase, the most commonly reported adverse event that was observed at a rate greater than or equal to 5% and twice the rate of placebo was headache.
About CAPLYTA (lumateperone)
CAPLYTA 42 mg is an oral, once-daily atypical antipsychotic approved in adults for the treatment of schizophrenia and depressive episodes associated with bipolar I or II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate. The efficacy of CAPLYTA could be mediated through a combination of antagonist activity at central serotonin 5-HT2A receptors and postsynaptic antagonist activity at central dopamine D2 receptors.
