BioXcel Therapeutics’ BXCL501 shows positive results in Alzheimer’s agitation

BioXcel Therapeutics announced positive topline results for TRANQUILITY II, a Phase 3 trial of BXCL501, the company’s proprietary, orally dissolving film formulation of dexmedetomidine under investigation for the acute treatment of Alzheimer’s disease-related agitation. The Phase 3 trial met its primary efficacy endpoint with the 60 mcg dose; a statistically significant and clinically meaningful 7.5 point reduction from baseline in Positive and Negative Syndrome Scale-Excitatory Component total score was observed at 2 hours versus 5.4 with placebo. The 60 mcg dose also met the first key secondary endpoint of reducing agitation symptoms at 1 hour during the first episode of agitation but did not meet the other key secondary endpoint of change from baseline in PEC score at 30 minutes. Efficacy for this dose was supported by a number of secondary measures. Most patients – 76% – responded to the first 60 mcg dose and were determined to be “Very Much” or “Much Improved” vs. 50% with placebo. BXCL501 continued to show a PEC reduction over repeated dosing. BXCL501 was well tolerated, with a side effect profile substantially consistent with prior trials of BXCL501 and the current label for IGALMI. The majority of safety events occurring within 24 hours of dosing in this population were mild or moderate in severity and consistent with the current IGALMI label. The company plans to develop a path to potential sNDA submission for the acute treatment of agitation associated with Alzheimer’s disease in H2 2023, subject to further discussions with the FDA.