(NASDAQ: BTAI) BXCL501: An Acute Treatment

(NASDAQ: BTAI) BXCL501: An Acute Treatment January 03, 2019 for Agitation BioXcel Therapeutics, 555 Long Wharf Drive, New Haven, CT 06511 | www.bioxceltherapeutics.com Proprietary & Confidential

Agenda 01 02 BXCL501 PROGRAM OVERVIEW CLINICAL TRIALS RESULTS WITH IV DEXMEDETOMIDINE ALZHEIMER S | SCHIZOPHRENIA 03 04 BXCL501 DEVELOPMENT PLAN IN 2019 SUMMARY ATTENDEES Vimal Mehta, CEO & Member of Board Vincent O'Neill, Chief Medical Officer Frank Yocca, Chief Scientific Officer Robert Risinger, VP, Clinical Development Chetan Lathia, SVP & Head, Translational Medicine Sheldon Preskorn, Member, Clinical Advisory Board Proprietary & Confidential 2

BXCL501: Sublingual Thin Film Dexmedetomidine (Dex) for Acute Treatment of Agitation Rapid clinical development and regulatory approval path (505(b)(2)) Agitation: A growing global healthcare issue ($40B+) Safer, non-invasive anti-agitation treatment needed Current therapies sub-optimal: Dementia: Antipsychotic drugs (black-box warning) for elderly Psychiatric: Invasive with severe side effects BXCL501: An innovative approach Novel mechanism of action (MoA) targets a causal agitation pathway Non-Invasive, easy to administer sublingual film with rapid onset of action 3 Proprietary & Confidential

Dementia: Symptoms of Alzheimer s Disease Nearly half of the Alzheimer s patients have Agitation symptoms every month1 NEUROPSYCHIATRIC5 Agitation Aggression Mood disturbances Wandering COGNITIVE2-4 FUNCTIONAL6 Memory loss Impaired spatial and temporal orientation Language disturbance Agnosia Reduced ability to carry out *ADLs Life example: Dressing Grooming Personal hygiene *ADL= Activity of daily living 1. Ryu SH, Katona C, Rive B, Livingston G, LASER-AD study;2005; 2.Joubert et al. In: Gauthier (ed);2007 3. Rainville et al. In: Gauthier (ed);2007; 4. Alzheimer s Association, Alzheimer's Dement 2016;12(4): 459-509; 5. Gelinas et al. In: Gauthier (ed);2007; 6.Teng & Cummings. In: Gauthier (ed);2007 4 Proprietary & Confidential

BXCL501: Sublingual Thin Film Formulation of Dexmedetomidine (Dex) Dex exerts calming effect at low exposures providing a broad therapeutic index Ideal Pharmaceutical Properties for a Non-invasive Sublingual Film Formulation Film manufacturing completed: Multiple dose strengths ranging from 10 g to 60 g for clinical studies Immediate release film with muco-adhesion properties Proprietary technology delivers low dose ranges The Right Pharmacology and Safety Profile (Precedex IV Dex) For Sedation in ICU Setting Prescribed to 8M+ patients Studied in 120 clinical trials Wide therapeutic index 5 Proprietary & Confidential Loading Dose Maintenance Dose Tolerable Dose 0.5 g/kg 1.6 g/kg >5 g/kg

Dexmedetomidine Mechanism of Action Reduction of hyper-arousal from overactive locus coeruleus neurons in response to stress Hyper-Arousal Physiology Dexmedetomidine MoA Locus Coeruleus (LC) Activation (+) Agitation (-) Agitation Norepinephrine Stress Induced Bipolar Disorder Opiate Withdrawal Schizophrenia Dementia Delirium 6 Proprietary & Confidential

Dexmedetomidine: High Potency and Intrinsic Activity at Alpha2 Receptors Data generated using transfected human Alpha2 receptor Human Alpha2 Receptor Response [Compound] Molar Dexmedetomidine: Pharmacology may translate into greater clinical efficacy 7 Proprietary & Confidential Functional Receptor Response

Human Biology Studies for BXCL501 Differentiate central nervous system (CNS) from peripheral effects EEG Measurements Dex elicits a characteristic change in electroencephalographs (EEG) Plan to use EEG to demonstrate CNS activity that determines therapeutic window Lowest dose with CNS activity Highest dose without excessive drowsiness Determine onset of CNS effect and time course of recovery in agitated patients Akeju O, Kim S-E, Vazquez R, Rhee J, Pavone KJ, Hobbs LE, et al. (2016) Spatiotemporal Dynamics of Dexmedetomidine-Induced Electroencephalogram Oscillations. PLoS ONE 11 (10): e0163431. doi:10.1371/journal. pone.0163431 8 Proprietary & Confidential Scalp Electrodes

IV Dex: Positive Human Proof of Concept in Treating Agitation from Alzheimer's Disease, Schizophrenia and Delirium IV Dex data from 90 subjects: healthy volunteers and three disease pathologies 1 2 ALZHEIMER S DISEASE 14 patient study [10 treatment + 4 placebo} Clinical benefit observed in 7/10 treated **RASS score of -1 No clinically meaningful effects on blood pressure and/or heart rate SCHIZOPHRENIA 14 patient study [10 treatment + 4 placebo] Clinical benefit observed in 9/10 treated RASS score of -1 PEC score of 7 or below No clinically meaningful effects on blood pressure and/or heart rate 90 Subject Experience DELIRIUM 132 patients [46 refractory to haloperidol] 46/46 haloperidol refractory patients responded to IV Dex in reducing agitation HEALTHY ELDERLY VOLUNTEERS 16 subject study [12 treatment + 4 placebo] Mild sedation achieved in 11/12 treated RASS score of -1 No clinically meaningful effects on blood pressure and/or heart rate 4 3 ** Richmond Agitation Sedation Scale * Positive and Negative Symptom Scale-Excitatory Component Carrasco et.al., Critical Care Medicine: July 2016, Vol 44, Issue 7, pp. 1295-1309 9 Proprietary & Confidential

Human Proof of Concept 1: IV Dex Reduces Agitation in Schizophrenia Patients Study results announced Nov 2018: primary endpoint met Study Design Randomized, placebo-controlled dose-ranging study 14 patients [10 treatment + 4 placebo] Primary endpoint: RASS of -1 Secondary endpoint: PEC score of 7 or below PEC Across Time % of Patients Achieving RASS-1 12 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 0% 11 10 9 8 7 6 0 20 40 60 80 100 120 0 20 40 60 80 100 120 Minutes post start of infusion Minutes post start of infusion Dex Dex 9/10 patients achieved PEC score of 7 or below No clinically relevant cardiovascular changes Early PEC reduction before drowsiness 9/10 patients achieved RASS score of -1 10 Proprietary & Confidential PEC Score

Human Proof of Concept 2: IV Dex Reduces Agitation in Alzheimer s Patients Study results announced Jan 2019: primary endpoint met Study Design Randomized, placebo-controlled individual dose-ranging study Infusion initiated at a low rate and increased by 0.1 mcg/kg/h 14 patients [10 treatment + 4 placebo] Primary endpoint: Optimal dose to achieve RASS of -1 Pharmacokinetic/Pharmacodynamic (PK/PD) observed with IV Dex concentrations (pg/mL) Primary endpoint (RASS -1) achieved at a fraction of dose required for surgical sedation Identified a dose range for optimizing film (BXCL501) 1 of 4 Placebo subjects achieved RASS of -1 at 30 mins PK consistent with prior healthy elderly trial No clinically meaningful cardiovascular effects 7/10 Patients Achieved RASS score of -1 No Adverse Events (AE), well-tolerated 11 Proprietary & Confidential Pharmacokinetics (PK) and Clinical Effect % of Patients achieving RASS -1

Human Proof of Concept 3: IV Dex Reduces Agitation in Haloperidol-Refractory Delirium Elderly hyperactive delirium patients refractory to haloperidol are difficult to treat 5 p = .01 4 3 2 1 Titration 0 -1 -2 Minutes -3 0 15 30 45 60 120 240 360 480 600 Start IV Haloperidol IV Haloperidol + IV Dex IV Dex only IV Dex achieved greater time in satisfactory sedation No respiratory or Heart Conduction disturbances BXCL501 MoA shown to treat agitated delirium in elderly 46/46 haloperidol refractory patients calmed by IV Dex * Carrasco et.al., Critical Care Medicine: July 2016, Vol 44, Issue 7, pp. 1295-1309 12 Proprietary & Confidential Richmond Agitation Sedation Scale (RASS) Group Comparison Initial Haloperidol

Sublingual Film (BXCL501) Pharmacokinetic and Safety Study IND-opening study BXCL501-101 Sublingual Pharmacokinetic, Safety and Tolerability Study Placebo-controlled, single ascending dose, pharmacokinetic (PK) study, safety & tolerability of BXCL501 (sublingual film) in healthy adult volunteers ages 18-65 Primary objective Determine PK, safety and tolerability of various film strengths Dosing initiated December 2018 Accrual continues with periodic review between dose escalation Expect dosing completion 1Q2019 13 Proprietary & Confidential

BXCL501 Integrated Clinical Development Plan Acute agitation studies: short with easily measurable clinical endpoints [IND] 2018 PK and Safety Study Multiple Dose Strengths (N=60) Sublingual Thin Film Data readout 1H2019 2019 Registration Trial* (Phase 2/3 Trials) Agitated Schizophrenia and Bipolar Patients Opiate Withdrawal Symptoms Agitated Alzheimer s Patients Hyperactive Delirium Adasuve Approval for Acute Treatment; 2 trials of 2 doses in 300+ patients, one in each indication; Agitated Schizophrenia Patients: 344 Agitated Bipolar Patients: 314 Primary Endpoint: Reduction in PEC score from baseline 2020 [ Submission ] First NDA *Clinical Development plan subject to agreement with FDA 14 Proprietary & Confidential

BXCL501 Program Summary Broad market potential Low doses produce clinically measurable anti-agitation effects On track to initiate a registration trial for an indication for BXCL501 in 2019 with potential NDA filing in 2020 Potential for multiple indications due to BXCL501 s anti-agitation properties across different disease pathologies Well capitalized to achieve multiple value inflection points through 2019 15 Proprietary & Confidential

Dr. Vimal Mehta, CEO BioXcel Therapeutics, New Haven, CT 06511 vmehta@bioxceltherapeutics.com Proprietary & Confidential