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BTAI: Pivotal Phase 3 Trials for BXCL501 to Initiate by End of 2019…

By David Bautz, PhD

NASDAQ:BTAI

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Phase 3 Program for BXCL501 in Schizophrenia and Bipolar Disorder to Initiate Before End of 2019

On December 3, 2019, BioXcel Therapeutics, Inc. (NASDAQ:BTAI) announced that following a successful ‘end-of-Phase 2’ meeting with the FDA, the company will be initiating the Phase 3 SERENITY (Sub-Lingual DExmedetomidine in Agitation Associated With SchizophRENIa and Bipolar Disorder STudY) program before the end of 2019.

The SERENITY I trial will be a randomized, double blind, placebo controlled, adaptive trial involving up to 375 patients age 18 to 75 with agitation associated with schizophrenia who will be randomized 1:1:1 to receive either 120 μg BXCL501, 180 μg BXCL501, or placebo. The SERENITY II trial is designed similarly to the SERENITY I trial, but will enroll patients with agitation associated with bipolar disorder.

The primary endpoint of both trials will be a reduction of symptoms of acute agitation using the Positive and Negative Syndrome Scale – Excitatory Component (PEC), a validated regulatory endpoint for quantifying agitation comprised of five elements associated with agitation scored from 1 (minimum) to 7 (maximum) (Montoya et al., 2011), as measured from baseline compared to placebo. A key secondary endpoint will be determining the earliest time where an effect on agitation is apparent by measuring the change in PEC score from baseline. We anticipate topline results in mid-2020. An outline of the SERENITY program is shown below.

Data from Phase 1b Trial in Schizophrenia Patients

In July 2019, BioXcel announced positive top line results from a Phase 1b, randomized, double blind, placebo controlled, multi-center U.S. trial evaluating multiple doses of BXCL501 for the treatment of agitation in 135 schizophrenia patients (NCT04010305). The primary endpoint was the reduction in PEC score from baseline at 120 minutes. Results showed rapid calming without excessive sedation at two hours, with the 80 μg, 120 μg, and 180 μg doses showing reductions in PEC scores of -7.1, -9.2, and -10.8, respectively, compared to -4.5 for placebo. These results were all statistically significant. The following slide shows a rapid and durable response with statistically significant separation from placebo <60 minutes following dosing. The reduction in PEC scores attained by BXCL501 was comparable to levels achieved by IM antipsychotics in other trials (Breier et al., 2002).

A secondary endpoint in the trial included assessment using the Agitation-Calmness Evaluation Scale (ACES), which is a single item that rates the overall agitation and sedation at the time of evaluation, ranging from 1-marked agitation, 2-moderate agitation, 3-mild agitation, 4-normal behavior, 5-mild calmness, 6-moderate calmness, 7-marked calmness, 8-deep sleep, and 9-unarousable. The following graph shows a statistically significant change in ACES from baseline at both 180 μg and 80 μg BXCL501 compared to placebo, consistent with what was seen in the primary endpoint.

BXCL501 was well tolerated with no serious adverse events reported across the entire dose range tested. The most common treatment emergent adverse event was mild somnolence and dry mouth. In addition, all study subjects were able to self-administer the film and complete the study.

Conclusion

We look forward to the results of the upcoming Phase 3 clinical trials of BXCL501 for agitation associated with schizophrenia and bipolar disorder, which we anticipate will be reported in mid-2020. Based upon the Phase 1b results we believe there is a high probability of success in both those trials. Our current valuation is $21.00 per share.

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