BioXcel Therapeutics (BTAI) Study Result summary

Study background and clinical need

• Acute agitation in bipolar disorder and schizophrenia often leads to ED visits and hospitalization, imposing significant burdens on patients, families, and healthcare systems.

• No FDA-approved therapies currently exist for at-home agitation management in these populations.

• IGALMI (BXCL501, dexmedetomidine) sublingual film offers a non-invasive, oral alternative with efficacy comparable to injectable agents.

• The SERENITY At-Home Phase III trial aimed to evaluate the safety and exploratory efficacy of IGALMI for self-administration in the home setting.

• The study supports expanding treatment options for managing agitation outside institutional settings.

Study design and patient population

• Pivotal Phase 3, double-blind, placebo-controlled, 12-week trial evaluated 120 mcg BXCL501 for acute agitation in bipolar disorder or schizophrenia at home.

• 246 patients randomized; 2,628 agitation episodes recorded in 215 patients; average age ~47 years.

• Included adults aged 18–75 with schizophrenia, schizoaffective, schizophreniform, or bipolar I/II disorder, stable on psychotropic regimens, and history of ≥3 agitation episodes in 3 months.

• Excluded those with serious or unstable medical illness, certain personality disorders, or recent moderate/severe substance use disorder.

• Pragmatic design allowed real-world coping strategies and concomitant interventions alongside study medication.

Safety and tolerability results

• BXCL501 was well tolerated with no drug-related serious adverse events, falls, syncopes, or severe treatment-emergent adverse events.

• No patients discontinued due to adverse events in the dexmedetomidine/BXCL501 arm.

• Adverse event profile consistent with prior institutional studies and the approved IGALMI label; most events were mild.

• Most common TEAEs: somnolence (22% IGALMI, 17% placebo at first dose), oral paresthesia/hypoesthesia, dizziness, dry mouth; incidence did not increase with repeat dosing.

• Tolerability remained stable across repeated doses and over time.