Bioxcel sees nothing startling in Tranquility

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One dose too low, one too high, one just right. Such appears to be the result of the phase I/II Tranquility trial of Bioxcel’s BXCL501 in dementia-related agitation. BXCL501, a sublingual film formulation of the sedative dexmedetomidine, was tested in 54 patients in assisted living facilities, 47 of whom had Alzheimer’s disease. Based on topline data the company has sidelined the 90μg dose after some patients experienced orthostasis and dizziness, and higher exposure levels were seen than in earlier trials. The two other doses, 30μg and 60μg, were considered safe, with no severe or serious adverse events reported, but the lower of these failed to produce statistically significant improvements over placebo on three agitation scales. But the middle dose produced “clinically meaningful” reductions within two hours of dosing, according to Bioxcel. Statistically significant separation from placebo in PEC and PAS total scores was observed with this dose at 60 minutes – Mod-CMAI was not measured at this time point, the group said – and lasted until eight hours post-dosing. The company will now discuss pivotal trial plans with the FDA, but it appears that the dose-finding trial has done just that.

Tranquility: topline efficacy results 2hrs post-dose
  BXCL501 60μg (n=20) BXCL501 30μg (n=16) Placebo (n=14)
Safety endpoints
Hypotension 10% 0% 0%
Orthostatic hypotension 5% 6% 0%
Dizziness 5% 6% 0%
Efficacy endpoints
Reduction from baseline in PEC total score -7.1 -5.4 -2.9
    P value vs placebo  0.0011 0.0813  
Response rate* 70% 25% 7%
Reduction from baseline in PAS total score -5.9 -3.9 -2.5
    P value vs placebo  <0.0001 0.0961  
Reduction from baseline in Mod-CMAI total score -14.0 -8.0 -3.2
    P value vs placebo  <0.0001 0.0591  
*Response defined as a reduction of at least 40% in PEC scores. Source: company release.

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