Intra-Cellular Therapeutics has announced positive topline results from its Phase III Study 304 (NCT04959032), evaluating Caplyta (lumateperone) for the prevention of relapse in patients with schizophrenia. The trial met its primary endpoint, demonstrating a statistically significant delay in time to relapse during the 26-week double-blind treatment phase. Caplyta reduced the risk of relapse by 63% compared to placebo.
The multi-center, parallel-group Phase III study enrolled 228 patients. Participants initially received 42mg of Caplyta during an 18-week open-label period. Subsequently, patients were randomized to receive either Caplyta or placebo in the double-blind treatment phase. The primary endpoint was the time to relapse, which was significantly longer in the Caplyta group compared to the placebo group.
The relapse rate in the Caplyta group was 16.4%, compared to 38.6% in the placebo group. The trial also met its secondary endpoint, with Caplyta demonstrating better treatment discontinuation rates during the double-blind phase. These findings support the potential for continued long-term treatment with Caplyta.
Caplyta's Market Performance and Future Expansion
Caplyta, a butyrophenone antipsychotic, was initially approved by the FDA in 2019 for the treatment of schizophrenia in adults. Its approval was expanded in 2022 to include the treatment of depressive episodes associated with bipolar I or II disorder. Intra-Cellular Therapeutics anticipates Caplyta sales to reach between $665 million and $685 million in 2024. GlobalData projects that Caplyta sales could exceed $3 billion by 2030.
Expert Commentary
"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 Caplyta" said Dr. Suresh Durgam, executive VP and chief medical officer at Intra-Cellular Therapies.
Planned sNDA Submission and Ongoing Studies
Intra-Cellular Therapeutics plans to submit a supplemental New Drug Application (sNDA) to the FDA in Q4 2024, seeking approval for Caplyta as an adjunct treatment for major depressive disorder (MDD). The company is also evaluating Caplyta in pediatric patients with schizophrenia and bipolar disorders in an ongoing Phase III trial (NCT06229210), which is currently recruiting participants.
