Slide 1: SERENITY At-Home Pivotal Phase 3 Safety Trial Topline Results
® Agitation Associated with Bipolar Disorders or Schizophrenia in the At-Home Setting SERENITY At-Home Pivotal Phase 3 Safety Trial Topline Results August 27, 2025 BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | www.bioxceltherapeutics.com Nasdaq: BTAI
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. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 2
Slide 3: Agitation: A Clinical Burden With No Clear Solution
3 Agitation: A Clinical Burden With No Clear Solution1 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 • Medic
Slide 4: At-Home Intervention: Early Intervention May Prevent Severity Escalation
4 At-Home Intervention: Early Intervention May Prevent Severity Escalation Potentially reduce ER visits, hospitalizations, or first-responder interventions 23 million1 57-77M EPISODES1-3 AT HOME SETTING AGITATION AGITATION PRE-AGITATION PRE-AGITATION MILD MILD MODERATE MODERATE SEVERE SEVERE INITIAT
Slide 5: SERENITY Program Seeks IGALMI Label Expansion
5 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 sND
Slide 6: IGALMI (dexmedetomidine) Sublingual Film 120 mcg
6 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 7: Positive Topline Results: SERENITY At-Home Trial
7 Positive Topline Results: SERENITY At-Home Trial Results are supportive of sNDA submission for label expansion of IGALMI® • Data collected from 2,628 agitation episodes – 2,437 episodes were treated in 208 patients – Patients self-administered the film for all treated episodes • Well tolerated wit
Slide 8: SERENITY At-Home Trial Design
8 SERENITY At-Home Trial Design ®
Slide 9: SERENITY At-Home Safety Trial Design
9 SERENITY At-Home Safety Trial Design Pragmatic study reflecting real-world patient behavior Primary Objective: Safety and tolerability of BXCL501 12-week at-home PRN* treatment Agitation associated with bipolar disorder / BXCL501 120 mcg N=100 1:1 schizophrenia Screening R Placebo N=100 Patients r
Slide 10: Defining The At-Home Study Population
10 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 11: SERENITY At-Home Topline Results
11 SERENITY At-Home Topline Results ®
Slide 12: Baseline Demographics and Disease Characteristics
12 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 13: Agitation Episodes and Dosing Frequency
13 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 14: BXCL501 was Well Tolerated
14 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 15: BXCL501 Tolerability Profile Consistent With IGALMI Label
15 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 16: BXCL501 Tolerability Profile Consistent With Repeat Dosing
16 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 17: BXCL501 Tolerability Profile Consistent Over the Trial Duration
17 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 18: Exploratory Assessments of Treatment Benefit
18 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 19: Clinical Summary
19 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 20: Planned Next Steps
20 Planned Next Steps Scientific conference sNDA preparation and Define commercialization data presentation submission strategy
Slide 21: Thank you!
Thank you! ® BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | www.bioxceltherapeutics.com
Slide 22: Appendix
Appendix ® BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | www.bioxceltherapeutics.com
Slide 23: Change in PEC Score from Baseline
SERENITYNEW I & II TRIAL 23 Building on Proven Efficacy of 120 mcg Dose SERENITY SERENITYI:I:Change ChangeininPEC PECScore Scorefrom fromBaseline Baseline SERENITY II: Change in PEC Score from Baseline 0 0 * p < 0.025 Placebo * p < 0.025 Placebo *** p < 0.0001 120 mcg *** p < 0.0001 120 mcg Change i
Slide 24: Safety Profile in Initial Pivotal Clinical Trials
24 Safety Profile in Initial Pivotal Clinical Trials1-3 Adverse reactions reported in ≥2% of IGALMI-treated patients and greater than placebo in SERENITY I and II1 IGALMI IGALMI Placebo Adverse reaction 180 mcg 120 mcg (n=252) (n=252) (n=255) • The majority of adverse reactions Somnolence* 23% 22% 6