Bioxcel Therapeutics, Inc. · NASDAQ:BTAI
Bioxcel Therapeutics — Investor Presentation (April 2024)
36 slides from Bioxcel Therapeutics's investor presentation, Apr 22, 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 April 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 1995 . BioXcel Therapeutics, Inc . (“BioXcel” or the “Company”) intends such forward - looking statements to be covered by the safe harbor provisions for forward - looking
Slide 3: Corporate Overview
Corporate Overview hlisStapeutics
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, Proven Business Model • Clinically and commercially validated AI platform • BXCL501 IND to IGALMI Œ approval < 4 years Large U.S. Agitation Market Opp
Slide 7: Corporate Growth Drivers: Land and Expand Strategy
* Land Corporate Growth Drivers to Advance Land and Expand Strategy 7 BXCL501 Phase 3 TRANQUILITY and SERENITY Programs Expand Agitation Associated With Bipolar Disorders or Schizophrenia Medical Supervision Agitation Associated With Alzheimer’s Dementia Agitation Associated With Bipolar Disorders o
Slide 8: Neuroscience Clinical Development
Neuroscience Clinical Development 8 *Government - funded, investigator - sponsored trials The safety and efficacy of investigational agents and/or investigational uses of approved products have not been established Compound Indication/Proposed Indication Preclinical Phase 1 Phase 2 Phase 3 Registrat
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: Acute Treatment of Agitation in Alzheimer's Dementia
® Acute Treatment of Agitation Associated with Alzheimer’s Dementia (AAD) ® 10 ® TRANQUILITY Program
Slide 11: AAD is Debilitating for Patients and a Burden for Caregivers
AAD is Debilitating for Patients and a Burden for Caregivers 11 1. Data on File InVibe Patient and Caregiver Research (n=75) December 2022 2. Alzheimer’s Association. 2023 Alzheimer’s Disease Facts and Figures. Accessed November 14, 2023. https://www.alz.org/media/Documents/alzheimers - facts - and
Slide 12: TRANQUILITY Program Offers Potential Path to sNDA
TRANQUILITY Program Offers Potential Path to sNDA 12 • Plan to discuss details of requirement for long - term safety data at future meeting with FDA** • Company has developed preliminary TRANQUILITY At - Home trial design and is re - evaluating initiation timing * Trial protocol under development, d
Slide 13: Evaluating BXCL501 for AAD in High to Low Care Settings
Clinical trial strategy designed to maximize potential commercial opportunity across patient locations Evaluating BXCL501 for AAD in High to Low Care Settings 13 Skilled Nursing Facilities Memory Care Units Patient Care Intensity TRANQUILITY At - Home Trial* Assisted Living Facilities Home Setting S
Slide 14: TRANQUILITY In-Care Study Design
• Design: Randomized, double - blind, placebo - controlled, parallel group trial • Power: Over 8 0% power • Inclusion Criteria – Patients with probable AD (mild, moderate, or severe, MMSE ≤ 25), who experience agitation, and residing in skilled nursing facilities, memory care units, or assisted livi
Slide 15: Preliminary TRANQUILITY At-Home Study Design
• Study Design: Randomized, double - blind, placebo - controlled, parallel group trial • Primary Objective: Safety and tolerability of BXCL501 60 mcg • Inclusion Criteria – Patients with mild, moderate, or severe probable AD who experience agitation, MMSE ≤ 25 – Patients with not more than three epi
Slide 16: SERENITY Program: Agitation in Bipolar Disorders or Schizophrenia (At-Home)
® Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia (at - home setting) ® 16 ® SERENITY Program
Slide 17: Agitation: Relatively Common and Difficult-to-Manage
Debilitating for Patients and Threatening for Healthcare Providers Agitation: Relatively Common and Difficult - to - Manage 1 17 Characterized by recurring episodes 1 Symptoms differ by patient, vary between episodes, and range from mild to severe 2 - 7 Multi - billion - dollar healthcare burden 8 B
Slide 18: SERENITY Program Seeks Potential IGALMI Label Expansion
SERENITY Program Seeks Potential IGALMI Œ Label Expansion 18 IGALMI APPROVAL Potential opportunity for at - home use in treating agitation associated with bipolar disorders or schizophrenia COMPLETED * SERENITY At - Home represents the redesigned SERENITY III † Trial protocol under development, desi
Slide 19: Planned SERENITY At-Home Study Design
• Recruitment Criteria – Patients alone or with informants with at least 1 treated episode of agitation – Patients with episodes of agitation in the month prior to enrollment • Treatment – Single dose to treat agitation at levels that typically require intervention – Maximum of 1 dose of study medic
Slide 20: Potential for Bipolar/Schizophrenia 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 ) When shown product profile stimulus, patients said they would use a p
Slide 21: IGALMI Commercialization
® IGALMI Œ Commercialization 21 Following commercial field workforce reduction in August 2023
Slide 22: IGALMI Indication and Important Safety Information
IGALMI Œ Indication and Important Safety Information INDICATION IGALMI Ρ (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitatio
Slide 23: Market Conditions Favorable for Continued Demand Growth
• 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 24: Agitation Franchise Expansion Plan
24 Launched & established IGALMI Πin hospital setting for bipolar disorder/ schizophrenia Use contracting and pricing to drive familiarity with IGALMI in hospitals Prepare for potential expansion into in - care and at - home markets for BXCL501 Leverage patient and HCP experiences to prepare for la
Slide 25: AI-Driven Drug Re-innovation Platform
® AI - Driven Drug Re - innovation Platform ® ® 25
Slide 26: 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 26 Concept Generation BioXcel Therapeutics AI and Analytics Natural Language Processing entity - relationship extractions Graph Data Science Machine Learning predictin
Slide 27: 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 AI AI AI 27
Slide 28: 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 28 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 29: BXCL502: Novel Agent for Chronic Agitation in Dementia
® BXCL502: A Novel Agent for Treatment of Chronic Agitation in Dementia ® 29 ®
Slide 30: BXCL502 Presents a Compelling Value Proposition
Formulation studies are ongoing BXCL502 Presents a Compelling Value Proposition 30 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 31: Latrepirdine: Clinical Safety Data and Preclinical Rationale
• Over 1000 patients with AD exposed for 26 weeks and 500 up to 52 weeks (Trials conducted by Pfizer and Medivation) • Showed activity in 5 preclinical models of neuropsychiatric symptoms (Trials conducted by BioXcel Therapeutics) • Secondary efficacy endpoints measuring changes in Neuropsychiatric
Slide 32: Recent Examples of Successful CNS Drug Re-Innovation
Recent Examples of Successful CNS Drug Re - Innovation 32 DRUG/COMPANY CHALLENGE SOLUTION STATUS Dextromethorphan Axsome Therapeutics Low bioavailability & metabolites that may cause side effects Bupropion significantly increases dextromethorphan bioavailability Successful clinical trial/depression
Slide 33: Immuno-Oncology
Immuno-Oncology OnkosXcel Therapeutics A subsidiary of Boel Therapeutics, Ine 33
Slide 34: BXCL701: Strong Value Proposition in Hard-to-Treat Tumors
BXCL701: Strong Value Proposition in Hard - to - Treat Tumors 34 BXCL701 ( talabostat ) is an investigational agent. The safety and efficacy have not been established. mCRPC = metastatic castration - resistant prostate cancer | SCNC = small cell neuroendocrine prostate cancer 1. National Library of
Slide 35: Immuno-Oncology Clinical Development
Immuno - Oncology Clinical Development 35 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) Initiat
Slide 36: Thank you!
BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | bioxceltherapeutics.com ® Thank you!
BioXcel Therapeutics — Investor Presentation (April 2024) on 6ix