Bioxcel Therapeutics Announced Results From The Correlation Study Related To Exploratory Efficacy Outcomes From SERENITY At-home Trial. The Results, Along With The Data From The Serenity At-home Trial, Will Be Included In The Supplemental New Drug Application Submission Planned For 1Q26

The standard method for measuring acute agitation associated with schizophrenia and bipolar disorder is the Positive and Negative Syndrome Scale – Excited Component (PEC) administered by a trained clinician, which was used in the Serenity I & II Pivotal Trials. In order to evaluate BXCL501 for continued clinical effect with repeat dosing in the at-home setting using an exploratory efficacy measurement, the Company, in consultation with FDA, developed the modified CGI-S (mCGI-S) scale, which can be scored by patients and/or caregivers. The study assessed the correlation between PEC and mCGI-S in this prospective, open label, in-clinic trial in 33 patients.

The results demonstrated a strong correlation between the clinician assessments and the patient or caregiver (informant) rated outcomes, providing support for using mCGI-S to assess efficacy in the outpatient setting. A statistically significant and strong correlation between the PEC and mCGI-S with a correlation of ρ=0.89; p<0.0001 for patients and ρ=0.88; p<0.0001 for informants was observed.

"We are pleased with the strong and significant correlation observed with clinician assessment for both patients and informants, providing support for our exploratory efficacy outcomes in the SERENITY At-Home study." said Dusan Kostic, Ph. D. Senior Vice President Medical Affairs and Clinical Development. "We plan to include these findings as a part of our upcoming sNDA package."

There were no serious adverse events reported and the safety profile remains consistent with the IGALMI® label.

Additional data and results will be presented at upcoming medical meetings and conferences.

About Modified Clinical Global Impression – Severity Scale (mCGI-S)

The original clinician rated CGI-S scale describes an 8-point rating of agitation symptoms where zero means there was no assessment performed and scores of 1-7 rate increasing severities of the symptoms being assessed. For ease of implementation by the patient and informant in the home setting this scale was modified in consultation with FDA. The modified CGI-S is a 4-point scale where 0 is no agitation, and scores of 1-3 describe increasing severities of agitation (mild, moderate, or severe).

About the Positive and Negative Syndrome Scale - Excited Component (PEC)

The PEC is a 5-item assessment for rating acute psychomotor agitation within the following domains: excitement, hostility, tension, uncooperativeness, and poor impulse control (Montoya et al. 2011). Each item utilizes a 7-point scale, with domain-specific descriptions of the signs the rater may observe for each severity rating: 1 = Absent, 2 = Minimal, 3 = Mild, 4 = Moderate, 5 = Moderate Severe, 6 = Severe, and 7 = Extreme. The total PEC score is the sum of the five domain scores, ranging from 5 to 35. A PEC score of 14 is the threshold needed to treat an acute agitation episode.