Medication for Agitation in Dementia and Alzheimer Disease Patients Moves Forward

January 6, 2021
Leah Kuntz

A new formulation of dexmedetomidine meets primary and secondary endpoints in recently completed study.

BioXcel Therapeutics recently announced that its orally dissolving, thin-film formulation of dexmedetomidine, BXCL501, met the primary and secondary endpoints of the TRANQUILITY trial at the 60 mcg dose level.

“We are very encouraged by the promising topline results from the TRANQUILITY study, which was designed to identify a recommended dose of BXCL501 for a potential pivotal study in dementia patients suffering from agitation. Following decades of research, there are still no effective treatments that directly target agitation commonly seen with dementia patients, and we are thrilled by the potential of being the first to develop a therapy designed to address this significant patient and caregiver need,” Vimal Mehta, Chief Executive Officer of BioXcel, said to the press. “Based on the results observed, we believe BXCL501 has broad potential in treating the full spectrum of agitation in patients with dementia. We look forward to advancing BXCL501 into a late-stage study this year following dialogue with the FDA.”1

BXCL501 is a selective alpha-2a receptor agonist for the treatment of agitation and opioid withdrawal symptoms. BioXcel believes that BXCL501 directly targets an agitation mechanism and has observed anti-agitation results in a number of clinical studies across several neuropsychiatric disorders.

The US Food and Drug Administration (FDA) granted BXCL501 Fast Track Designation for the acute treatment of agitation in patients with schizophrenia, bipolar disorders, and dementia. Two different phase 3 trials (SERENITY I and II) studied BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders.

The TRANQUILITY study consisted of 54 participants in assisted living facilities with agitation related to dementia, 87% of whom had Alzheimer disease. Participants received BXCL501 at either 30 mcg, 60 mcg, 90 mcg, or a placebo. Participants reported no severe or serious adverse events, and the primary safety and tolerability endpoints were met. Adverse events in the trial included hypotension, orthostatic hypotension, and dizziness, with the most common adverse event being mild or moderate somnolence.

BioXcel also shared plans for a phase 2 trial studying hospitalized patients who have agitation symptoms associated with delirium, intended to begin within the next several months.

Reference

1. BioXcel Therapeutics. BioXcel Therapeutics announces BXCL501 met the primary and all secondary endpoints in the TRANQUILITY phase 1b/2 study for the acute treatment of agitation in dementia, including Alzheimer disease. News release. GlobeNewswire. January 5, 2021. https://www.globenewswire.com/news-release/2021/01/05/2153316/0/en/BioXcel-Therapeutics-Announces-BXCL501-Met-the-Primary-and-All-Secondary-Endpoints-in-the-TRANQUILITY-Phase-1b-2-Study-for-the-Acute-Treatment-of-Agitation-in-Dementia-including-Al.html