BioXcel Reports Positive Correlation Study Results for Efficacy Outcomes in SERENITY Trial

BioXcel Therapeutics reported positive results from a correlation study related to exploratory efficacy outcomes from the SERENITY At-Home trial. The study demonstrated a strong correlation between clinician assessments and patient/caregiver-rated outcomes, providing support for using mCGI-S to assess efficacy in outpatient settings. The results will be included in the sNDA submission planned for Q1 2026.

On October 14, 2025, BioXcel Therapeutics, Inc. (Nasdaq: BTAI) announced positive results from a correlation study related to exploratory efficacy outcomes from the SERENITY At-Home trial. The study demonstrated a strong correlation between clinician assessments and patient/caregiver-rated outcomes, providing support for using the modified Clinical Global Impression – Severity Scale (mCGI-S) to assess efficacy in outpatient settings. The results will be included in the supplemental New Drug Application (sNDA) submission planned for the first quarter of 2026.

The SERENITY At-Home trial assessed the correlation between the Positive and Negative Syndrome Scale – Excited Component (PEC) and mCGI-S in a prospective, open-label, in-clinic trial involving 33 patients. The study found a statistically significant and strong correlation between the clinician assessments and the patient or caregiver (informant) rated outcomes, with a correlation coefficient of ρ=0.89 for patients and ρ=0.88 for informants (p0.0001). This strong correlation supports the use of mCGI-S for assessing efficacy in the outpatient setting, which is particularly relevant for the acute treatment of agitation associated with schizophrenia, bipolar I or II disorder, or Alzheimer’s dementia.

No serious adverse events were reported, and the safety profile remained consistent with the approved label for IGALMI® (dexmedetomidine), the company's sublingual film formulation of dexmedetomidine. The positive results from the SERENITY At-Home trial are part of BioXcel Therapeutics' ongoing efforts to expand the indications for BXCL501, an investigational proprietary, orally dissolving film formulation of dexmedetomidine. The company plans to present additional data and results at upcoming medical meetings and conferences.

BioXcel Therapeutics is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel Therapeutics, focuses on the development of medicines in immuno-oncology. The company's drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications.

The positive results from the SERENITY At-Home trial correlation study are a significant milestone for BioXcel Therapeutics, as they support the company's efforts to expand the indications for BXCL501. The strong correlation between clinician assessments and patient/caregiver-rated outcomes provides valuable data that will be included in the upcoming sNDA submission to the FDA.