BioXcel completes two Phase 2 dosing cohorts of BXCL501

NEW HAVEN, Conn.—BioXcel Therapeutics, Inc. (BTI) announced today that it has completed dosing of the first two cohorts of agitated schizophrenia patients in its Phase 2 efficacy trial of BXCL501, the company’s proprietary sublingual thin film formulation of dexmedetomidine (Dex). BTI plans to use the results from this study to pursue two planned Phase 3 pivotal trials in agitated schizophrenia and bipolar disorder patients, which are currently expected to begin in the fourth quarter of 2019.

The first two cohorts in this double-blind, placebo-controlled Phase 2 efficacy trial totaling 54 patients were completed within three weeks of study initiation. BTI currently plans to enroll approximately 200 agitated schizophrenia patients. The primary endpoint of the study is change from baseline measured by the total score of Positive and Negative Symptom Scale - Excitatory Component scale (PEC). The secondary endpoints include assessment using the Agitation Calmness Evaluation Scale (ACES).

“We are pleased with the progress of our Phase 2 efficacy study with BXCL501 in agitated schizophrenia patients, a study we believe will support the launch of our pivotal Phase 3 studies in late 2019,” said Vimal Mehta, Ph.D., chief executive officer of BTI. “We have now treated 96 subjects with BXCL501 to date, in addition to the positive data from our human proof-of-concept studies in multiple neuropsychiatric conditions with intravenous (“IV”) Dex. In parallel, we are accelerating clinical development plans for agitated dementia, opioid withdrawal symptoms and hyperactive delirium outside the currently on-going schizophrenia study. We continue to believe that agitation represents a multi-billion-dollar burden to the healthcare system and is severely lacking effective and safe non-invasive treatment options.”

BXCL501 is a potential first-in-class, proprietary sublingual thin film of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism, and the company has shown anti-agitation effects in multiple clinical studies across multiple neuropsychiatric indications. 

A Phase 1 pharmacokinetic and safety study of BXCL501 has yielded positive topline data. The company plans to unveil future development plans for BXCL501 in agitated dementia, opioid withdrawal symptoms and hyperactive delirium through 2019.

“We are delighted to be advancing the efficacy trial of our potent selective alpha-2 agonist, BXCL501. With successful completion of dosing in the first two patient cohorts, we are excited and looking forward to the results from this efficacy trial that are expected in 3Q 2019,” added Robert Risinger, M.D., vice president of Clinical Development for BTI. “Data from the Phase 1 pharmacokinetic (bioavailability) and safety study of BXCL501 exhibited clinically favorable features by achieving targeted exposure levels and the study met both its primary and secondary endpoints. We believe that results from our two BXCL501 clinical studies will lay a solid foundation to launch several Phase 3 pivotal trials across multiple neuro-psychiatric conditions with high unmet medical needs.”