Phase III trial of BXCL 501 meets endpoints in patients with agitation and schizophrenia and bipolar disorder.- BioXcel Therapeutics

BioXcel Therapeutics announced that BXCL 501 (sublingual dexmedetomidine), met the primary and secondary endpoints of SERENITY I and SERENITY II, demonstrating a robust treatment effect in the trials with patients with agitation and schizophrenia and bipolar disorder. Results demonstrated that BXCL501 was well tolerated, with rapid and durable reductions in agitation. In patients with schizophrenia (SERENITY I) and a second study of bipolar disorder (SERENITY II), highly statistically significant and clinically meaningful reductions in the Positive and Negative Syndrome Scale, Excitatory Component (“PEC”) score at two hours, the primary endpoint, were reported for both high and low dose cohorts of BXCL501 compared to placebo (p<0.0001). both studies also met the key secondary endpoint, demonstrating improvement in pec scores beginning as early as 20 minutes in patients with bipolar disorder, at both dose levels, and as early as 20 minutes in patients with schizophrenia for the 180 mcg dose level. exploratory efficacy endpoints confirmed the primary endpoint, with duration of response lasting at least four hours after treatment. the secondary endpoint was highly statistically significant at 30 minutes, 45 minutes, 60 minutes, and 90 minutes in both studies. at 20 minutes, both doses were statistically significant in patients with bipolar disorder (p><0.025), and in patients with schizophrenia who received the higher 180 mcg dose. efficacy was further evaluated using two additional measures of agitation—the agitation and calmness evaluation scale (aces), and clinical global impression – improvement scale (cgi-i)—each of which showed statistically significant improvements for both doses of bxcl501 compared to placebo. bxcl501 was well tolerated in both serenity trials. overall, the most commonly reported adverse events from both trials were somnolence (22% for 180 mcg dose arms, 21% for 120 mcg dose arms and 6% for placebo arms;>75% of these events were classified as mild), dry mouth (4.4%, 7.5% and 1.2%, respectively), and dizziness (6.0%, 3.9%, and 0.8%, respectively). All adverse events were mild to moderate in severity, with none categorized as severe or requiring further intervention or monitoring. Few subjects discontinued the trials due to adverse events (SERENITY I: 0 for 180 mcg dose, 2 for 120 mcg dose and 0 for placebo arm; SERENITY II: 0, 1, and 0, respectively).