Bioxcel Therapeutics, Inc. · NASDAQ:BTAI
Bioxcel Therapeutics — Investor Presentation (February 2024)
42 slides from Bioxcel Therapeutics's investor presentation, Feb 14, 2024
Slide 1: AI-Driven Transformative Medicines in Neuroscience
® BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | bioxceltherapeutics.com NASDAQ: BTAI February 2024 AI - Driven Transformative Medicines in Neuroscience
Slide 2: Forward-looking statements
This presentation includes "forward - looking statements" within the meaning of the Private Securities Litigation Reform Act of 19 95. Forward - looking statements in this presentation include but are not limited to: statements regarding BioXcel Therapeutics’ expected timing of, and data res ult s f
Slide 3: Corporate Overview
Ex tncrapeuticr Corporate Overview
Slide 4: About BioXcel Therapeutics
About BioXcel Therapeutics 4 Founded: 2017 IPO: 2018 Ticker: BTAI (Nasdaq) Headquarters: New Haven, CT
Slide 5: Our Mission
® 5 ® Develop transformative medicines in neuroscience utilizing artificial intelligence Our Mission
Slide 6: Strong Value Proposition and Long-Term Growth Potential
Transformative approach leveraging technology, clinical, and commercial expertise Strong Value Proposition and Long - Term Growth Potential 6 Unique Business Model • Employ AI , machine learning, and neuroscience expertise to discover new lead compounds • Re - innovate approved and/or clinically dev
Slide 7: Corporate Growth Drivers
Transformative drug re - innovation approach resulted in rapid development and approval of IGALMI ΠCorporate Growth Drivers Phase 3 TRANQUILITY and SERENITY Programs BXCL502 BXCL503 7 Land Agitation Associated w ith Bipolar Disorders or Schizophrenia Healthcare Provider Supervision The safety and e
Slide 8: R&D Strategy: Build Pipeline Depth with Innovation and Expansion
R&D Strategy: Build Pipeline Depth with Innovation and Expansion Compound Indication/Proposed Indication Preclinical Phase 1 Phase 2 Phase 3 Registration Marketed APPROVED APRIL 5, 2022 Acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults under healthcare
Slide 9: Leadership Expertise
Leadership Expertise 9 Vimal Mehta, Ph.D. Chief Executive Officer & Founder Richard I. Steinhart Senior Vice President & Chief Financial Officer Frank D. Yocca, Ph.D. Senior Vice President & Chief Scientific Officer Vincent J. O’Neill, M.D. Executive Vice President, Chief of Product Development and
Slide 10: TRANQUILITY Program: Agitation in Alzheimer's Dementia
® Acute Treatment of Agitation Associated with Alzheimer’s Dementia (AAD) ® 10 ® TRANQUILITY Program
Slide 11: Potential Sizeable At-Home U.S. Market Opportunity
140M annual episodes * ~$14B+ total market opportunity 6 Agitation associated with Alzheimer’s dementia (AAD) Potential Sizeable At - Home U.S. Market Opportunity 11 1. Alzheimer’s Association. 2023 Alzheimer’s Disease Facts and Figures. Accessed November 14, 2023. https://www.alz.org/media/Document
Slide 12: Favorable Impressions of Target Profile for BXCL501 in AAD
Interest in Target Profile for BXCL501 (% of Total CGs, n=75) 4% 25% 71% Not interested (1 - 2) Neutral (3 - 5) Very interested (6 - 7) Caregivers very interested in blinded target product profile Favorable Impressions of Target Profile for BXCL501 in AAD Source: inVibe Market Research with AAD Care
Slide 13: TRANQUILITY Program Overview
Agitation associated with Alzheimer’s dementia (AAD) TRANQUILITY Program Overview* 13 *An Instructions For Use (IFU) study is being conducted as a brief precursor to at - home trial. TRANQUILITY I TRANQUILITY II TRANQUILITY AT - HOME (planned)* 4 weeks Phase 1b/2 Phase 3 Phase 3 Efficacy, safety, to
Slide 14: TRANQUILITY Program Overview
Agitation associated with Alzheimer’s dementia (AAD) TRANQUILITY Program Overview* 1 *An Instructions For Use (IFU) study is being conducted as a brief precursor to at - home trial. TRANQUILITY I TRANQUILITY II TRANQUILITY AT - HOME (planned)* 4 weeks Phase 1b/2 Phase 3 Phase 3 Efficacy, safety, tol
Slide 15: Planned TRANQUILITY At-Home Pivotal Phase 3 Trial Design
BXCL501 (60 mcg) n=50 Placebo n=50 Screening R Patients with probable Alzheimer’s disease residing at home Primary endpoint: safety and tolerability of BXCL501 4 - week dosing as needed Planned TRANQUILITY At - Home Pivotal Phase 3 Trial Design* 2 Instructions For Use Study • Recruitment Criteria –
Slide 16: BXCL501 Clinical Foundation: Expansion Into At-Home Setting
BXCL501 Clinical Foundation: Expansion Into At - Home Setting 3 • 11 double - blind, placebo - controlled Phase 2 and 3 clinical trials evaluating safety and efficacy • 1,100+ patients enrolled across multiple neuropsychiatric conditions and in healthy volunteers • 273 were over 60 years of age and
Slide 17: SERENITY Program: Agitation in Bipolar Disorders or Schizophrenia
® Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia (at - home setting) ® 4 ® SERENITY Program
Slide 18: Potential Sizeable At-Home U.S. Market Opportunity
40M + annual episodes outside hospital setting * ~$4B+ total market opportunity 5 Agitation associated with bipolar disorders or schizophrenia Potential Sizeable At - Home U.S. Market Opportunity 5 Average ~3 episodes per month 4 ~1.6M Diagnosed Patients with Agitation 1 - 3 1 Wu E. Annual prevalenc
Slide 19: Potential for Patient Use of BXCL501 At Home
80% 33% 17% 14% 13% 6% 4% 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% BXCL501 Anti-anxiety Anti-depressants Mood stabilizers Anti-psychotic Other None Potential Uptake for BXCL501 (if approved for at - home use) (% of Episodes, n=240 ) Q22. You previously indicated that you used the following medications
Slide 20: SERENITY Program Overview
Acute treatment of agitation associated with bipolar disorders or schizophrenia (bipolar disorder - associated agitation) SERENITY Program Overview* 7 PART 1 | CLINIC PART 2 | HOME *Not shown are 2 additional post - marketing requirement studies in schizophrenia & bipolar disorder: • Agitation in Pe
Slide 21: Planned SERENITY At-Home Pivotal Phase 3 Trial Design
• Recruitment Criteria – Patients alone or with informants (as dyads) with at least 1 treated episode of agitation • Treatment – Single dose to treat agitation at levels that typically require intervention – Maximum of 1 dose of study medication within 12 hours Planned SERENITY At - home Pivotal Pha
Slide 22: Upcoming Expected Milestones
® Upcoming Expected Milestones 9 ® 9
Slide 23: Expected BXCL501 Clinical Trial Initiations in H1 2024
Expected BXCL501 Clinical Trial Initiations in H1 2024 TRANQUILITY At Home: highest priority for capital allocation 10 NEUROSCIENCE: BXCL501 Agitation Associated With Bipolar Disorders or Schizophrenia Phase 3 SERENITY At Home Pivotal Trial Topline Data Readout: Expected in Q1 2025* Agitation Associ
Slide 24: Potential Sizeable At-Home U.S. Market Opportunities
Agitation associated with Alzheimer’s dementia (AAD) and bipolar disorders or schizophrenia Potential Sizeable At - Home U.S. Market Opportunities 11 1. Alzheimer’s Association. 2023 Alzheimer’s Disease Facts and Figures. Accessed November 14, 2023. https://www.alz.org/media/Documents/alzheimers - f
Slide 25: IGALMI Commercialization
® IGALMI Œ Commercialization 12 Following commercial field workforce reduction in August 2023
Slide 26: IGALMI (dexmedetomidine) Sublingual Film
First and only orally dissolving sublingual film currently in use under healthcare provider supervision for acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults 1 IGALMI Π(dexmedetomidine) Sublingual Film 13 Noninvasive, self - administered film 1 - 4 cov
Slide 27: Creating Demand in 2024
• Targeting/engaged with key hospital systems for volume - based contracting • J - Code granted by CMS effective Jan. 1, 2024 (streamlines and standardizes reimbursement) • Over 250 hospital P&T approvals to date, with large volume in Q4 2023 • First large academic center approval in Q4 2023 • Activ
Slide 28: Agitation Franchise Expansion Plan
Agitation Franchise Expansion Plan 15 Launch & establish IGALMI Πin hospital setting for bipolar disorder/ schizophrenia Use contracting and pricing to drive familiarity with IGALMI in hospitals 2022 - 2023 2024 2025 2026 Prepare for potential expansion into at - home markets for BXCL501 Leverage p
Slide 29: AI-Driven Drug Re-innovation Platform
® AI - Driven Drug Re - innovation Platform ® ® 16
Slide 30: AI Strategy to Accelerate Drug Re-Innovation Process
From product concept to first - in - human clinical trials using composite AI AI Strategy to Accelerate Drug Re - Innovation Process 17 Concept Generation BioXcel Therapeutics AI and Analytics Natural Language Processing entity - relationship extractions Graph Data Science Machine Learning predictin
Slide 31: NovareAI: Ecosystem for Drug Discovery and Development
Pre - Clinical Platforms Indications Targets Compounds BTAI Re - Innovation Library Opportunity Prioritization Stress Axis Pre - Clinical Validation Mid/Late - Stage Assets BTAI Knowledge Graphs NovareAI: Ecosystem for Drug Discovery and Development AI 18 AI AI AI
Slide 32: Behavioral and Psychological Symptoms in Alzheimer's Disease
Identifying targets and compounds designed to address unmet medical needs in dementia Behavioral and Psychological Symptoms in Alzheimer’s Disease 19 Sleep Disturbances Hallucinations Apathy Depression Agitation/Aggression Anxiety NovareAI 42.3% 38.8% 26.8% 57.4% 27.5% Delusions 24.8% 35.6% BXCL501/
Slide 33: BXCL502: A Novel Agent for Treatment of Chronic Agitation in Dementia
® BXCL502: A Novel Agent for Treatment of Chronic Agitation in Dementia ® 20 ®
Slide 34: BXCL502 Presents a Compelling Value Proposition
Formulation studies are ongoing BXCL502 Presents a Compelling Value Proposition 21 BXCL502 is a novel formulation of latrepirdine and a metabolic stabilizer Potentially blocks excessive signaling mediated by 2 neurotransmitters: serotonin and norepinephrine (noradrenaline) New Chemical Entity Improv
Slide 35: Latrepirdine (Dimebon): Clinical Safety Results and Rationale
Latrepirdine (Dimebon): Clinical Safety Results, Preclinical Confidence in Rationale, and Early Sign of Potential Efficacy 22 CLINICAL Early Sign of Potential Efficacy PRECLINICAL Confidence in Rationale SAFETY RESULTS IN PATIENTS Lower risk VALUE ● Over 1000 patients with AD exposed for 26 weeks an
Slide 36: Recent Examples of Successful CNS Drug Re-Innovation
Recent Examples of Successful CNS Drug Re - Innovation DRUG/COMPANY CHALLENGE SOLUTION STATUS Dextromethorphan Axsome Therapeutics Metabolites cause unwanted side effects Block metabolism with CYP2D6 inhibitor, bupropion Successful clinical trial/depression Xanomeline Karuna Therapeutics Peripheral
Slide 37: Immuno-Oncology: OnkosXcel Therapeutics
Immuno-Oncology OnkosXcel Therapeutics ‘A subsidiary of BioXcel Therapeutics,
Slide 38: BXCL701: Strong Value Proposition in Hard-to-Treat Tumors
BXCL701: Strong Value Proposition in Hard - to - Treat Tumors 25 Novel Mechanism of Action Data Published in JITC Leadership Position in Innate Immunity DPP8/9 Biology Clinical Proof of Concept Cold Tumors Exploring Strategic Options • One of the most clinically advanced oral innate immune activator
Slide 39: Immuno-Oncology Clinical Development
Immuno - Oncology Clinical Development Compound Proposed Indication Preclinical Phase 1 Phase 2 Phase 3 Expected Upcoming Milestone Collaborator BXCL701 Company - sponsored trials Small Cell Neuroendocrine Prostate Cancer (SCNC) FDA Meeting 13 centers US / UK Small Cell Lung Cancer (SCLC) Initiate P
Slide 40: Thank you!
BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | bioxceltherapeutics.com ® Thank you! 27
Slide 41: Appendix
28 Appendix law bioxcel , therapeutics’
Slide 42: IGALMI Indication and Important Safety Information
IGALMI Œ Indication and Important Safety Information 29 INDICATION IGALMI Ρ (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, t ha t is placed under the tongue or behind the lower lip and is used for the acute treatment of agi
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