Bioxcel Therapeutics, Inc. · NASDAQ:BTAI
Bioxcel Therapeutics — Investor Presentation (November 2025)
45 slides from Bioxcel Therapeutics's investor presentation, November 2025
Slide 1: Corporate Presentation
Nasdaq: BTAI ® Corporate Presentation November 2025 BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | www.bioxceltherapeutics.com
Slide 2: Forward-Looking Statements
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
Slide 3: Our Mission
3 Our Mission Develop transformative medicines in neuroscience utilizing artificial intelligence ®
Slide 4: IGALMI and BXCL501 Market Expansion Potential
4 IGALMI® and BXCL501 Market Expansion Potential Land and expand strategy with novel agitation treatment option for multiple conditions & settings LONGER-TERM POTENTIAL SHORT-TERM POTENTIAL CURRENT Agitation Agitation Associated Agitation Associated with Associated with with Bipolar Disorders Alzhei
Slide 5: BXCL501 Has Broad Potential in Neuropsychiatric Disorders
5 BXCL501 Has Broad Potential in Neuropsychiatric Disorders Numerous opportunities to address significant unmet acute and chronic conditions ~$12.5M in Grants Awarded Acute Chronic (Company Sponsored) (Externally Sponsored) 9 million patients diagnosed with OUD, with over 5 million patients ~60-93 m
Slide 6: Key Company Milestones
6 Key Company Milestones Positive Business Momentum Serenity At-Home safety and exploratory efficacy data announced • Analyses ongoing for clinical study reports sNDA preparation in progress • Content and format of sNDA aligned with FDA in August • sNDA non-clinical section completed, clinical and C
Slide 7: IGALMI Indication and Important Safety Information
7 IGALMI® (dexmedetomidine) Sublingual Film IGALMI® Indication and Important Safety Information INDICATION IGALMI® (dexmedetomidine) sublingual film is a prescription medicine, administered Approved for acute treatment of agitation associated with under the supervision of a health care provider, tha
Slide 8: IGALMI (dexmedetomidine) Sublingual Film
8 IGALMI® (dexmedetomidine) Sublingual Film Approved for acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults under healthcare provider supervision • Patient administered under the supervision of a healthcare provider who should monitor vital signs and ale
Slide 9: Agitation Mechanism and BXCL501 (Dexmedetomidine) MOA
9 Agitation Mechanism and BXCL501 (Dexmedetomidine) MOA Disorders like schizophrenia, bipolar, and dementia Dexmedetomidine is one of the most potent, selective, full can affect the hyperarousal pathway, causing LC agonists at Alpha-2A receptors neurons to fire in a tonic mode causing fight-or-fligh
Slide 10: Our Neuroscience Development Portfolio
Our Neuroscience Development Portfolio 10 Compound Indication/Proposed Indication Preclinical Phase 1 Phase 2 Phase 3 Registration Marketed Acute treatment of agitation associated IGALMI® with schizophrenia or bipolar I or II (dexmedetomidine) disorder in adults under sublingual film healthcare prov
Slide 11: Substantial Intellectual Property and Patent Protection
11 Substantial Intellectual Property and Patent Protection* • 30+ granted or allowed patents and 140 additional patent applications in prosecution • 13 currently listed U.S. patents for IGALMI® in FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) with patent protect
Slide 12: Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia
12 Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia (at-home setting) SERENITY Program ®
Slide 13: Agitation is Relatively Common and Difficult-to-Manage
13 Agitation is Relatively Common and Difficult-to-Manage1 Debilitating for patients and threatening for healthcare providers Characterized by Best-practice guidelines recommend recurring episodes1 agitation be treated by: • Behavioral calming techniques • Verbal de-escalation Symptoms differ by • M
Slide 14: The At-Home Market for BXCL501 in Bipolar Disorders and Schizophrenia: Summary
14 The At-Home Market for BXCL501 in Bipolar Disorders and Schizophrenia: Summary The potential for BXCL501 in the at-home setting reflects significant unmet need and attractiveness to patients • Our market research indicates approximately 3 episodes of agitation per month in the home setting – Prev
Slide 15: Episode Frequency in Patients Experiencing Agitation
15 Episode Frequency in Patients Experiencing Agitation Agitation episodes expected to occur 3-4 times per month based on multiple data sources Primary Market Research (N=80)1 Published Survey (N=583)2 Severity Number of episodes/year 15% 36% Mild 22.4 Schizophrenia (n=20) 49% Moderate 15.4 35 Episo
Slide 16: Physician Market Research Cites Need for Effective, Fast-acting, More Tolerable Treatment Options
16 Physician Market Research Cites Need for Effective, Fast-acting, More Tolerable Treatment Options Key unmet needs in bipolar disorders Key unmet needs in schizophrenia View lack of current effective and fast-acting treatments to be the primary unmet need 38% View lack of current effective and fas
Slide 17: Potential Patient Use of BXCL501 At Home
17 Potential Patient Use of BXCL501 At Home When shown product profile stimulus, patients stated they would use a product with the BXCL501 target profile for 80% of their bipolar/schizophrenia agitation episodes Potential Uptake for BXCL501 (if approved for at-home use) (% of Episodes, N=240) 90% 80
Slide 18: At-Home Intervention: Early Intervention May Prevent Severity Escalation
18 At-Home Intervention: Early Intervention May Prevent Severity Escalation Potentially reduce ER visits, hospitalizations, or first-responder interventions 23M EPISODES1 57-77M EPISODES1-3 AT HOME SETTING AGITATION AGITATION PRE-AGITATION PRE-AGITATION MILD MILD MODERATE MODERATE SEVERE SEVERE INIT
Slide 19: SERENITY Program Seeks IGALMI Label Expansion
19 SERENITY Program Seeks IGALMI® Label Expansion No FDA-approved therapies for agitation associated with bipolar disorders or schizophrenia in the at-home setting SERENITY Program Journey IGALMI® APPROVAL SERENITY SERENITY PLANNED SERENITY I SERENITY II with HCP supervision AT-HOME AT-HOME TRIAL sN
Slide 20: SERENITY At-Home Topline Results
20 SERENITY At-Home Topline Results ®
Slide 21: SERENITY At-Home Trial Design
21 SERENITY At-Home Trial Design Topline data released in Q3 2025 Primary Objective: Safety and tolerability of BXCL501 12-week at-home PRN treatment Agitation associated with bipolar disorder / 1:1 BXCL501 120 mcg N=100 schizophrenia Screening R Placebo N=100 Patients residing at home • Design: dou
Slide 22: Defining The At-Home Study Population
22 Defining The At-Home Study Population INCLUDED PATIENTS EXCLUDED PATIENTS • Male and female patients ages • Unstable or serious medical illness 18–75 years with bipolar I or II (e.g., CHF, recent MI, hepatic disorder, schizophrenia, disease) schizoaffective, or schizophreniform disorder • Diagnos
Slide 23: Baseline Demographics and Disease Characteristics
23 Baseline Demographics and Disease Characteristics IGALMI 120 MCG PLACEBO OVERALL* PARAMETER (N=102) (N=106) (N=246) Age, years, Mean (SD) 47.5 (12.4) 47.2 (13.0) 47.0 (12.9) Female, n (%) 48 (47.1%) 60 (56.6%) 120 (48.8%) Race, n (%) White 52 (51.0%) 59 (55.7%) 131 (53.3%) Black or African Americ
Slide 24: Agitation Episodes and Dosing Frequency
24 Agitation Episodes and Dosing Frequency Large safety database generated to support sNDA submission • 2,437 agitation episodes were treated in 208 patients – 168 (81%) treated patients completed the full 12-week study – Average of 11.7 agitation episodes were recorded per treated patient • Data co
Slide 25: BXCL501 was Well Tolerated
25 BXCL501 was Well Tolerated Primary objective of the pivotal safety trial was met • No patients discontinued due to TEAEs in the BXCL501 arm • Adverse event profile consistent with approved IGALMI® Label and other clinical trials in institutional setting – No drug-related serious adverse events (S
Slide 26: BXCL501 Tolerability Profile Consistent With IGALMI Label
26 BXCL501 Tolerability Profile Consistent With IGALMI® Label Most common treatment-emergent adverse events (TEAEs) in Serenity At-Home trial and comparison with same TEAEs in the existing label SERENITY I & II SERENITY AT-HOME (IGALMI® Label1) Adverse Event by Dose (Episode)1 Treatment-Emergent SIN
Slide 27: BXCL501 Tolerability Profile Consistent With Repeat Dosing
27 BXCL501 Tolerability Profile Consistent With Repeat Dosing Incidence of TEAEs did not increase with additional dosing TEAEs: Events per dosed episode by dose numbers After Doses 1-3 After doses 4 to 12 After doses 13 and beyond Treatment-Emergent Adverse Event BXCL501 PLACEBO BXCL501 PLACEBO BXCL
Slide 28: BXCL501 Tolerability Profile Consistent Over the Trial Duration
28 BXCL501 Tolerability Profile Consistent Over the Trial Duration Incidence of TEAEs did not increase over time TEAEs: Events per dosed episode by week in trial Weeks 1 to 4 Weeks 5 to 8 Weeks 9 to 12 Treatment-Emergent Adverse Event BXCL501 PLACEBO BXCL501 PLACEBO BXCL501 PLACEBO (N=454)1 (N=474)1
Slide 29: Exploratory Assessments of Treatment Benefit
29 Exploratory Assessments of Treatment Benefit Similar reductions in symptom severity with repeat dosing over a 12-week period • The efficacy of 120 mcg dose for a single dose administration proven and approved as IGALMI® • The primary objective of this trial was safety and the exploratory objectiv
Slide 30: The Benefit of BXCL501 Over Placebo Across Episode Severities
30 The Benefit of BXCL501 Over Placebo Across Episode Severities PERCENT OF FULLY RESOLVED EPISODES BY BASELINE SEVERITY P<0.0001 70% BXCL501 Placebo P<0.0001 P<0.0001 Resolved Episodes 60% P<0.0005 50% 40% 30% 20% 10% 0% No. of episodes 178 217 665 704 315 349 1158 1270 Severe Moderate Mild Overall
Slide 31: Clinical Summary
31 Clinical Summary • Well tolerated with repeat dosing across the trial duration • Safety profile consistent with prior inpatient SERENITY trials – No discontinuations due to TEAEs in the BXCL501 arm – No serious drug-related adverse events – No falls or syncopes • All patients successfully self-ad
Slide 32: Provisional Targeting Model
32 Provisional Targeting Model Among the highest deciles of Bipolar & Schizophrenia patient Dx volume, 70% are managed by psychiatrists, with 8.1k total HCP targets. 50-70 discrete field FTEs can cover these targets BP/SCZ Patient Dx Volume (Decile >5) National Distribution of 8.1k HCP Targets by De
Slide 33: BXCL501 for Agitation Associated with Alzheimer's Dementia (AAD)
33 BXCL501 for Agitation Associated with Alzheimer’s Dementia (AAD) ®
Slide 34: AAD is Debilitating for Patients and a Burden for Caregivers
34 AAD is Debilitating for Patients and a Burden for Caregivers Agitation is the primary reason for patients to live in long-term care facilities versus a home setting • Nearly 7 million Alzheimer’s dementia patients in the U.S., with approximately 50% suffering from agitation. • AD-related agitatio
Slide 35: Strategic Opportunity for BXCL501 in AAD Treatment Market
Strategic Opportunity for BXCL501 in AAD Treatment Market 35 PRN therapy could potentially address breakthrough episodes – favorable chronic treatment companion Perceptual Map No Boxed Warning • Brexpiprazole – After treatment, the mean CMAI scores indicated still clinically significant agitation du
Slide 36: TRANQUILITY II: Multi-Center, Randomized, Double-Blind, Placebo-controlled Efficacy and Safety Study
36 TRANQUILITY II: Multi-Center, Randomized, Double-Blind, Placebo-controlled Efficacy and Safety Study PRN dosing of BXCL501 over a 12-week period in subjects with agitation associated with dementia Major Findings • Treatment with 60 μg BXCL501 showed efficacy in reducing symptoms of agitation. • B
Slide 37: Change From Baseline in PEC Total Score Compared to Placebo for the First Episode
Change From Baseline in PEC Total Score Compared to Placebo 37 for the First Episode of Agitation BXCL501 60 mcg BXCL501 40 mcg PLACEBO ENDPOINT (N=50) (N=48) (N=51) Change from baseline [LS Mean (SE)] @ 1 hour -6.2 (0.6) | p = 0.0185 - 4.2 (0.6) | p = 0.9590 - 4.2 (0.6) Change from baseline [LS Mea
Slide 38: BXCL501 Was Well Tolerated
38 BXCL501 Was Well Tolerated Treatment-emergent adverse events of special interest (AESI) occurring within 24 hours after first dose and over entire trial period AEs during 12-week trial AEs within 24 hours of first dose (443 doses) BXCL501 BXCL501 BXCL501 BXCL501 Placebo Placebo Adverse Event Seve
Slide 39: TRANQUILITY Program Offers Potential Opportunity to Address a Significant Unmet Need in AAD
39 TRANQUILITY Program Offers Potential Opportunity to Address a Significant Unmet Need in AAD TRANQUILITY Program Journey CONFIRMATORY PHASE 3 BXCL501 BREAKTHROUGH TRANQUILITY POTENTIAL TRANQUILITY I THERAPY DESIGNATION TRANQUILITY II IN-CARE TRIAL1,2 sNDA SUBMISSION COMPLETED MARCH 2021 COMPLETED
Slide 40: Evaluating BXCL501 for AAD in High to Low Care Settings
40 Evaluating BXCL501 for AAD in High to Low Care Settings Clinical trial strategy designed to maximize potential commercial opportunity across patient locations TRANQUILITY In-Care Trial TRANQUILITY At-Home Trial* Efficacy and Safety Safety Skilled Nursing Facilities Memory Care Units Patient Care
Slide 41: Second Phase 3 Trial: TRANQUILITY In-Care Study Design
41 Second Phase 3 Trial: TRANQUILITY In-Care Study Design* Primary Endpoint: Change from baseline PEC total score at Agitated patients with 2 hours for first dose dementia and probable Alzheimer’s disease BXCL501 (60 mcg) N=75 Screening R Patients residing in 1:1 care facilities Placebo N=75 • Desig
Slide 42: Preliminary TRANQUILITY At-Home Study Design
42 Preliminary TRANQUILITY At-Home Study Design* Primary Objective: Safety Agitated patients with probable Alzheimer’s dementia BXCL501 (60 mcg) N=50 Screening R Patients residing at home 1:1 Placebo N=50 • Study Design: Randomized, double-blind, placebo-controlled, parallel group trial • Primary Ob
Slide 43: Caregiver Impressions of BXCL501 Target Product Profile Have Been Favorable
Caregiver Impressions of BXCL501 Target Product Profile Have 43 Been Favorable >70% of caregivers expressed high interest in BXCL501 Interest in BXCL501 (% of Total CGs, n=75) 4% 25% 71% Very interested (6-7) Neutral (3-5) Not interested (1-2) Source: inVibe Market Research with ADA Caregivers (n=75
Slide 44: Key Company Milestones
44 Key Company Milestones Positive Business Momentum Serenity At-Home safety and exploratory efficacy data announced • Data analysis ongoing for clinical study reports sNDA preparation in progress • Content and format of sNDA aligned with FDA in August • sNDA non-clinical section completed, clinical
Slide 45: Thank you!
45 Thank you! ® BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | bioxceltherapeutics.com
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