Celon Pharma S.A. announces robust and positive Phase 2 clinical trial results for its PDE10A inhibitor (CPL?36), a novel, oral, once-daily antipsychotic. Statistically significant and clinically meaningful improvements in the primary endpoint of positive subscale of the PANSS were seen in both tested doses of CPL?36, with a dose-response effect. Management plans to discuss these highly encouraging results with regulatory agencies to advance CPL?36 towards registrational trials and global marketing approvals.

Additionally, CPL?36 is being investigated as a potential treatment for levodopa-induced dyskinesia in Parkinson?s disease, with Phase 2 results expected to be reported in Fourth Quarter 2024. The CPL?36 Phase 2 study was an international, multicenter, randomized, placebo-controlled clinical trial conducted on a group of 189 adult patients hospitalized due to acute schizophrenia. CPL?36 was administered for 4 weeks in two doses of 20 and 40 mg once daily and was placebo-controlled at a ratio of 1:1:1. Patient baseline severity was moderate-severe to severe, as characterized by a PANSS total score of approximately 106.

Patients were screened for up to 10 days and then randomized and treated over a four-week period, at which time the primary endpoint assessment was conducted at Day 28. At Week 4 of treatment, the reduction in positive PANSS subscale score which was the primary endpoint in the trial was 3.7 units from baseline in the 20 mg dose (LS mean difference from placebo, p<0.001, Cohen?s d: 0.73), and 6.3 units in the 40 mg dose (LS Mean difference from placebo, p<0.001, Cohen?s d: 1.38). For total PANSS score at week 4 of treatment (a key secondary endpoint), the 20 mg dose of CPL?36 demonstrated a 9.7 unit reduction from baseline compared to placebo (LS mean difference from placebo, p<0.001, Cohen?s d: 0.77), and 16.4 units in the 40 mg dose (LS mean difference from placebo, p<0.001, Cohen?s d: 1.47).

Other secondary endpoints in the trial included the effects of CPL?36 on overall clinical improvement cognitive performance and functioning such as Clinical Global Impression Scale Improvement (CGI-I), Brief Assessment of Cognition in Schizophrenia (BACS), and number of participants who withdraw due to adverse events (AEs). Results across all of these endpoints were also positive. Drug tolerability was favorable with most treatment emergent adverse events characterized as mild.

Exacerbations of schizophrenia represented the most common severe adverse events that were potentially related to the drug (1.5% in the placebo group, 1.8% in the 20 mg group and 3.1% in the 40 mg group). Treatment discontinuation due to adverse events likely related to the drug occurred in 3.1% patients in the placebo group, 0% patients in the 20 mg group and 7.7% patients in the 40 mg group.