BioXcel Therapeutics (BTAI) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase 2024 summary

Strategic clinical development plans

• Preparing to initiate two pivotal phase III trials for BXCL501, targeting at-home use for schizophrenia/bipolar agitation and a new indication for Alzheimer's-related agitation.

• SERENITY At-Home trial focuses on safety, using an approved IGALMI dose, with efficacy as an exploratory endpoint.

• TRANQUILITY In-Care trial for Alzheimer's agitation has substantial FDA alignment on design, endpoints, and sample size.

• Real-world patient populations, including those on background therapies, will be included to reflect actual clinical settings.

• Trials are designed to accommodate a spectrum of agitation severity and care settings, aiming for broad label expansion.

Regulatory and risk considerations

• Multiple recent FDA interactions have led to strong alignment on trial designs and endpoints.

• FDA collaboration is high, with a focus on demonstrating continued efficacy and societal impact.

• Breakthrough Therapy Designation received for acute agitation in Alzheimer's, with no prior acute approvals in this space.

• Safety in elderly populations is supported by existing data, with no significant new safety concerns identified.

• Independent audit confirmed data integrity after a site event in TRANQUILITY II.

Operational and financial strategy

• Operational focus is on executing the two pivotal trials, with resources concentrated on clinical development.

• Financing options under evaluation include equity, royalty monetization, and strategic partnerships.

• Commercialization of IGALMI is maintained with a minimal sales force, while pricing strategies for at-home use are under review, referencing neuropsych drug benchmarks.

• Supportive relationships with major investors and ongoing amendments to meet business needs.

• Ex-U.S. strategic partnerships are being considered, with flexibility in deal structure based on partner fit.