BioXcel Therapeutics doses first patient in phase 3 Alzheimer’s-associated agitation trial

BioXcel Therapeutics (BioXcel) has announced that the first patient has been dosed in its pivotal phase 3 TRANQUILITY III trial of BXCL501 (dexmedetomidine) sublingual film for the acute treatment of Alzheimer’s-associated agitation.

The company’s TRANQUILITY programme includes two investigational studies, TRANQUILITY II and TRANQUILITY III, to evaluate the safety and efficacy of BXCL501 in patients in assisted living or residential care facilities and nursing homes.

Each trial will enrol approximately 150 patients with dementia ages 65 years and older who will self-administer 40mcg or 60mcg of BXCL501 or placebo under the supervision of a trained research staff member whenever agitation episodes occur over a three-month period.

TRANQUILITY II, which began in May this year, will assess patients in assisted living or residential care facilities requiring minimal assistance with activities of daily living.

Meanwhile, TRANQUILITY III will assess patients residing predominantly in nursing homes with moderate to severe dementia who require moderate or greater assistance with activities of daily living.

Both studies share the same primary efficacy endpoint of change in Positive and Negative Syndrome Scale-Excitatory Component total score from baseline measured at two hours after the initial dose and subsequent doses.

Alzheimer’s disease (AD) is the most prevalent type of dementia in the US and the number of adults over the age of 65 with AD is expected to double from 5.8 million in 2020 to 11.8 million in 2040.

Restlessness and agitation are common behaviours in dementia patients, with approximately 100 million Alzheimer’s-associated agitation episodes reported in the US each year.

The causes of Alzheimer’s-associated agitation varies, but a patient may become agitated suddenly because of a change or specific cause, such as increased noise or not being able to do something they previously could. Patients may also feel a general sense of agitation but not know the cause.

BioXcel believes that BXCL501 potentially targets an important mediator of agitation, and the company has observed anti-agitation results in multiple clinical studies across several neuropsychiatric disorders.

Robert Risinger, chief medical officer, neuroscience of BioXcel, said: “The prevalence of AD is unfortunately increasing and there remains no US Food and Drug Administration-approved product indicated for patients experiencing agitation associated with this condition.

“With two pivotal trials underway in our TRANQUILITY programme, we are aiming to expand BXCL501’s potential to treat the full spectrum of episodic and intermittent chronic agitation market, and address the costly health-care burden related to Alzheimer’s agitation.”