BioXcel Therapeutics begins phase 3 trial for acute agitation in Alzheimer’s patients

BioXcel Therapeutics has announced that the first patient involved in the phase 3 TRANQUILITY 2 study of BXCL501 has received a dose for the acute treatment of agitation in patients with Alzheimer’s disease.

BXCL501 – the company’s orally dissolving, thin film formulation of dexmedetomidine – has been assessed as part of a pivotal phase 3 TRANQUILITY programme.

The programme began in December 2021 and is made up of two studies, TRANQUILITY 2 and TRANQUILITY 3.

Each study has been designed to evaluate the safety and effectiveness of BXCL501 in adults aged 65 years and older who are in assisted living, residential facilities or nursing homes.

Alzheimer’s disease is the most common form of dementia in the US, and it is expected that by 2040, approximately 12 million Americans aged 65 years and over may be impacted by the disease. Of these patients, up to 70% experience agitation, while around 100 million agitation episodes happen in the US annually. The knock-on effect of these episodes on both patients and their caregivers can be devastating.

As yet, there are no approved therapeutic options available for the acute treatment of agitation related to dementia, including Alzheimer’s disease.

BioXcel’s decision to progress with the assessment of both the 40 and 60mcg dosing regimens in the TRANQUILITY 2 and 3 pivotal trials is reinforced by results from a recent, multicentre, placebo-controlled study of 46 patients. The study evaluated the effectiveness, safety and tolerability of BXCL501 40mcg dose in patients with agitation associated with dementia.

BXCL501 was granted an earlier Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for the acute treatment of agitation in dementia, and showed a statistically significant decrease in agitation with both the 30 and 60mcg doses, with no severe or serious adverse events reported.

Dr Robert Risinger, chief medical officer of BioXcel Therapeutics, said: “We believe the recent FDA approval of BXCL501 for the acute treatment of agitation associated with schizophrenia or bipolar 1 or 2 disorder in adults has laid a strong foundation for pursuing this Alzheimer’s-related agitation programme to potentially address this debilitating symptom for patients.

He added: “Importantly, we are also expanding TRANQUILITY 2 to more than ten clinical trial sites in the US and with no current FDA approved treatments for agitation associated with this disease, we are making strong and swift efforts to potentially bring BXCL501 and its proven ability to address agitation to this large market.”