Bioxcel Therapeutics, Inc. · NASDAQ:BTAI

Bioxcel Therapeutics — Investor Presentation (April 2020)

30 slides from Bioxcel Therapeutics's investor presentation, Apr 15, 2020

1. Slide 1: Next Wave of Medicines Utilizing AI

   NASDAQ: BTAI Next Wave April 2020 of Medicines Utilizing AI BioXcel Therapeutics, 555 Long Wharf Drive, New Haven, CT 06511 | www.bioxceltherapeutics.com

2. Slide 2: Forward-Looking Statements

   Forward-Looking Statements This presentation includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this presentation include, but are not limited to, statements that relate to the advancement and development of

3. Slide 3: Overview

   Overview SERENITY Program Pivotal Readouts Expected in Mid-2020 Neuro Program BXCL501—Sublingual Thin Film for Acute Treatment of Agitation AI-Powered Drug Development • Phase 3 schizophrenia and bipolar trials (SERENITY I & II) initiated; readouts expected mid-2020 Phase 1b/2 dementia trial (TRANQU

4. Slide 4: AI Platform May Reduce Development Timelines and Cost

   AI Platform May Reduce Development Timelines and Cost 4–5 Year Development Cycle Multiple Candidates Screened By AI Selection of Best Candidates Translational Team Candidate Validation Clinical Development Team & Regulatory Team Human Proof of Concept & Registration Trials N D A S u b m i s s i o n

5. Slide 5: Pipeline

   Pipeline Neuropsychiatry Acute agitation in dementia Opioid withdrawal Immuno-oncology Neuroendocrine Prostate Cancer (tNEPC) Double Combination Advanced Solid Tumor Types (MD Anderson Led) Pancreatic Cancer Triple Combo *Regulatory path to be determined; device + drug combination to be evaluated af

6. Slide 6: BXCL501: Potential First in Class Sublingual Thin Film Dexmedetomidine for Acute Treatment of Agitation

   BXCL501: Potential First in Class Sublingual Thin Film Dexmedetomidine (Dex) for Acute Treatment of Agitation 6 © BioXcel Therapeutics, Inc.

7. Slide 7: Agitation: A Common Phenomenon

   Agitation: A Common Phenomenon Psychiatric Conditions Associated with (1) • 9.7 million suffer each year ~9.7M • • • • Schizophrenia/bipolar: 3.1M Dementia: 4M Opioid withdrawal: 1.6M zophrenia lar Disorder (2) Delirium: 1M Dementia • • • Patients experience multiple episodes per year $40 billion pe

8. Slide 8: Broad Market Potential Across Centers Where Neuropsychiatric Patients are Treated

   Broad Market Potential Across Centers Where Neuropsychiatric Patients are Treated Urgent Care Centers Hospitals Medical + Neuro + Psych Wards Psychiatric Hospitals Mental Clinics Neurology/Mental Health Crisis Care Emergency Departments General, Psychiatric Nursing Homes Assisted Living 8 © BioXcel

9. Slide 9: Current Therapies Are Suboptimal

   Current Treatment Options Fail to Address the Underlying Condition • • • • Verbal de-escalation is used as first line treatment Injectables are invasive with severe side effects Antipsychotic drugs have black-box warning for elderly Restraining can damage the caregiver/patient relationship • Require

10. Slide 10: BXCL501: Proprietary Sublingual Thin Film of Dex Designed to Block Driver of Agitation

    BXCL501: Proprietary Sublingual Thin Film of Dex* Designed to Block Driver of Agitation Target Causal Agitation •Dex activates at the alpha-2a receptor preventing the release of norepinephrine ✔ Easy to administer, sublingual formulation ✔ Non-traumatic ✔ Rapid onset of action, without excessive sed

11. Slide 11: Proprietary, Easy-to-Administer Formulation

    Proprietary, Easy-to-Administer Formulation • Muco-adhesion properties designed for optimizing compliance Adaptable technology enables broad dose range Flexible for potential combination of multiple drugs on a single film Expected patent term until 2039 -2041 • • • • • Manufacturing Phase 3/registra

12. Slide 12: Successful Phase 1b Clinical Trial in Agitated Schizophrenia Patients

    Successful Phase 1b Clinical Trial in Agitated Schizophrenia Patients Assessing Agitation Episodes in Schizophrenia Evaluation Period (6 hours) 0 min 60 min 120 min 180 min 240 min 300 min 360 min * R * Patients Dosed 12 © BioXcel Therapeutics, Inc. Primary Endpoint: Change from Baseline in PEC Scor

13. Slide 13: Primary Endpoint: Statistically Significant Change in PEC Score

    Primary Endpoint: Statistically Significant Change in PEC Score Clinically Meaningful, Rapid and Durable Responses 0 -2 -4 Placebo 80 mcg 180 mcg -6 * * -8 * -10 * = p <0.05 ** = p <0.001 *** = p < 0.0001 *** -12 0 60 120 180 240 300 360 Time Post Dose (Minutes) * The lowest dose tested, 20 mcg (not

14. Slide 14: Safety Results in Phase 1b Study

    Safety Results in Phase 1b Study Well-tolerated with no serious or severe adverse events Most common adverse events: mild somnolence and dry mouth Maximum tolerated dose was not reached All subjects (100%) were able to self-administer the film 14 © BioXcel Therapeutics, Inc.

15. Slide 15: SERENITY Phase 3 Pivotal Trials Initiated: Adaptive Design

    SERENITY Phase 3 Pivotal Trials Initiated: Adaptive Design Evaluation Period (6 hours) 0 min 60 min 120 min 180 min 240 min 300 min 360 min * Agitated R * Patients Dosed 15 © BioXcel Therapeutics, Inc. Primary Endpoint: Change from Baseline in PEC Score (PANSS-Excitatory Component) at 2 Hours SERENI

16. Slide 16: TRANQUILITY Phase 1b/2 Trial Initiated – Dementia

    TRANQUILITY Phase 1b/2 Trial Initiated – Dementia Assessing Agitation Episodes in Dementia Evaluation Period (24 hours) R Mid-2020 • • • • • Dementia (all forms including AD) PAS: Pittsburgh Agitation Scale Clinically significant agitation PEC: PANSS Excitatory Component CMAI: Cohen-Mansfield Agitat

17. Slide 17: RELEASE Phase 1b/2 Trial – Opioid Withdrawal

    RELEASE Phase 1b/2 Trial – Opioid Withdrawal Evaluation Period (5 Days) R • • • Pharmacokinetics, Safety and Tolerability • Street/prescription opiate abusers with signs and symptoms of opiate withdrawal before entry COWS: Clinical Opiate Withdrawal Scale • Double-blinded, placebo-controlled dose es

18. Slide 18: Neuroscience Program Strategy

    Neuroscience Program Strategy 18 © BioXcel Therapeutics, Inc. Preagitation Mild to Moderate Agitation Severe Agitation/Aggression of Agitation Delirium (sNDA) Opioid Withdrawal (sNDA) Dementia (sNDA) Prophylaxis or Prevention IM Formulation Development Schizophrenia/Bipolar (NDA)

19. Slide 19: BXCL501 Product Development Milestones

    BXCL501 Product Development Milestones 19 © BioXcel Therapeutics, Inc. 4Q2019 1Q2020 Mid-2020 2021 SERENITY I & II Trials (Schizophrenia/Bipolar) Phase 3 Pivotal Trial Initiation Expected Phase 3 Data Readout Expected NDA Submission TRANQUILITY Trial (Dementia) Phase 1b/2 Trial Initiation Expected P

20. Slide 20: BXCL701 Potential First-in-Class Oral IO Therapy

    BXCL701 Potential First-in-Class Oral IO Therapy 20 © BioXcel Therapeutics, Inc.

21. Slide 21: Orally Administered Investigational Innate Immunity Pathway Activator

    Orally Administered Investigational Immunity Pathway Activator of Systemic Innate Dual MoA designed to inhibit DPP 8/9 & FAP BXCL701 • Current approved immunotherapies struggle to address tumors that appear “cold” or uninflamed • BXCL701 is designed to stimulate the innate immune system, facilitatin

22. Slide 22: BXCL701 Clinical Development Strategy

    BXCL701 Clinical Development Strategy Multiple Opportunities to Evaluate Patient Outcomes Treatment Emergent Prostate Cancer (tNEPC): Phase 1b/2 trial of BXCL701 and KEYTRUDA 1 • • • Pancreatic Cancer Melanoma Acute Myeloid Leukemia Pancreatic Cancer: Triple combination (BXCL701, bempegaldesleukin a

23. Slide 23: Phase 1b/2: In Combination with KEYTRUDA to Treat tNEPC

    Phase 1b/2: In Combination with KEYTRUDA to Treat tNEPC 1 • • • Dose finding study in combination with KEYTRUDA® Preliminary pharmacokinetics of BXCL701 are within expectations based on prior data Initial data readout expected 1H 2020 Simon 2-stage: 15+15 Primary Endpoint: Composite response rate: T

24. Slide 24: Pancreatic Cancer: Triple Combination Trial

    Pancreatic Cancer: Triple Combination Trial 2 • Simon 2-stage: 15+15 Primary Endpoint: ORR Combination Secondary Endpoint: DoR, PFS, OS Exploratory Endpoint: Effect on immune cells (MDSC, T-cells, neutrophils) • • • *BXCL701 phase expected to be initiated following Nektar and Pfizer’s safety run-in

25. Slide 25: Expanding Study of BXCL701: Open-label Basket Trial with Keytruda

    Expanding Study of BXCL701: Open-label Basket Trial with Keytruda 3 • Cohort A: evaluating patients who are naïve to checkpoint therapy Cohort B: evaluating patients who have failed or • are refractory to checkpoint therapy Outcome measures: ORR, progression-free-survival, overall survival, duration

26. Slide 26: BXCL701 Program Milestones

    BXCL701 Program Milestones Trial Initiation Trial Ongoing 26 © BioXcel Therapeutics, Inc. 1H2019 2H2019 1H2020 2H2020 Neuroendocrine Prostate Cancer (tNEPC) Site Activation & Recruitment Ongoing Expected Initial Data Readout Pancreatic Cancer (PDA) Mechanism Trial Initiated Double Combination Expect

27. Slide 27: Thank You!

    Thank You! Dr. Vimal Mehta, CEO BioXcel Therapeutics, New Haven, CT vmehta@bioxceltherapeutics.com 06511 27 © BioXcel Therapeutics, Inc.

28. Slide 28: Appendix

    Appendix 28 © BioXcel Therapeutics, Inc.

29. Slide 29: Predictable and Dose Proportional PK Observed in Phase I Study

    Predictable and Dose Proportional PK Observed in Phase I Study Phase I Clinical Studies in 42 Healthy Volunteers 1000 100 40 µg 20 µg 10 µg 10 10 0.1 0 1 2 3 4 5 6 7 8 Time (Hours) 29 © BioXcel Therapeutics, Inc. Mean Concentration (ng/L) 28 active : 14 placebo

30. Slide 30: Secondary Evaluation: Change in ACES from Baseline

    Secondary Evaluation: Change in ACES from Baseline Consistent with Primary Endpoint p = <0.0001 5 4 p = <0.0156 3 2 1 * The lowest dose tested, 20 mcg (not shown) was repeated in subjects who did not achieve response criterion 0 The ACES consists of a single item that rates overall agitation and sed

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