Igalmi (dexmedetomedine sublingual) / BioXcel - LARVOL DELTA

Dexmedetomidine in the Treatment of Agitation Associated With Schizophrenia and Bipolar Disorder (SERENITY III) (clinicaltrials.gov) - P3 | N=452 | Active, not recruiting | Sponsor: BioXcel Therapeutics Inc | Recruiting ➔ Active, not recruiting

Enrollment closed • Bipolar Disorder • CNS Disorders • Mood Disorders • Psychiatry • Schizophrenia • Schizophreniform Disorder

BASIS: BXCL501 After Stress to Increase Recovery Success (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: University of North Carolina, Chapel Hill

New P2 trial • CNS Disorders • Mood Disorders • Post-traumatic Stress Disorder

BioXcel Therapeutics Strengthens Cash Position to Advance SERENITY At-Home Pivotal Phase 3 Safety Trial for Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia (GlobeNewswire) - "BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that, following the successful raise of $14 million gross proceeds in an equity financing that closed on March 4, 2025, it has approximately $35 million in cash. The strengthened cash position will support continued advancement of the Company’s pivotal Phase 3 SERENITY At-Home trial....'Patient enrollment is continuing and topline data results expected in the second half of 2025 are intended to support a potential sNDA submission to expand the label for IGALMI in the at-home setting'."

Financing • P3 data: top line • Bipolar Disorder • Schizophrenia

BioXcel Therapeutics Announces 33% Enrollment in SERENITY At-Home Pivotal Phase 3 Safety Trial for Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia (GlobeNewswire) - "BioXcel Therapeutics, Inc...today announced it has reached 33% enrollment in its 200-patient SERENITY At-Home trial. The pivotal Phase 3 trial is designed to evaluate the safety of BXCL501, the Company’s investigational, proprietary, orally dissolving film formulation of dexmedetomidine, in the at-home setting for the acute treatment of agitation associated with bipolar disorders or schizophrenia. Topline data results, which are expected in the second half of 2025, are intended to support a potential supplemental new drug application (sNDA) submission to expand the label of the FDA-approved IGALMI (dexmedetomidine) sublingual film."

Enrollment status • P3 data: top line • Bipolar Disorder • Schizophrenia

Sublingual Dexmedetomidine for Treating Opioid Withdrawal (clinicaltrials.gov) - P1/2 | N=160 | Recruiting | Sponsor: New York State Psychiatric Institute | Trial completion date: Apr 2025 ➔ Jul 2025 | Trial primary completion date: Jan 2025 ➔ Apr 2025

Trial completion date • Trial primary completion date • Substance Abuse

BioXcel Therapeutics Announces FDA Closed its Inspection of Site for Phase 3 TRANQUILITY II Trial for Acute Treatment of Agitation Associated with Alzheimer’s Dementia (GlobeNewswire) - "BioXcel Therapeutics, Inc...today announced that the U.S. Food and Drug Administration (FDA) has concluded that the inspection of a single site in its TRANQUILITY II Phase 3 trial is closed under 21 C.F.R.20.64(d)(3) and released the Establishment Inspection Report. The FDA has designated 'Voluntary Action Indicated' for the site."

FDA event • Alzheimer's Disease • Dementia

BioXcel Therapeutics Provides Clinical and Business Update (GlobeNewswire) - "BioXcel Therapeutics, Inc...today provided an update on the progress of its late-stage clinical programs for lead neuroscience asset BXCL501, as well as its recent steps to enhance operational and financial flexibility and strengthen its Board leadership...The majority of trial sites have been opened and patient enrollment is progressing with the SERENITY At-Home trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia."

Trial status • Depression • Schizophrenia

Dementia Care Research and Psychosocial Factors. (PubMed, Alzheimers Dement) - "There are currently no FDA-approved treatments for acute management of agitation in elderly patients with dementia. In this study, BXCL501 60 µg and 40 µg significantly reduced the symptoms of agitation at 2 hours after administration in this patient population, as measured by PEC. At those doses, the treatments were relatively well tolerated."

Journal • Alzheimer's Disease • Cardiovascular • CNS Disorders • Dementia • Hypotension

Dexmedetomidine in the Treatment of Agitation Associated With Schizophrenia and Bipolar Disorder (SERENITY III) (clinicaltrials.gov) - P3 | N=450 | Recruiting | Sponsor: BioXcel Therapeutics Inc | Active, not recruiting ➔ Recruiting | Trial completion date: Mar 2025 ➔ Sep 2025 | Trial primary completion date: Mar 2025 ➔ Sep 2025

Enrollment open • Trial completion date • Trial primary completion date • Bipolar Disorder • CNS Disorders • Mood Disorders • Psychiatry • Schizophrenia • Schizophreniform Disorder

Determining Efficacy and Safety of BXCL501 in Agitation Associated With Pediatric Schizophrenia and Bipolar Disorder (clinicaltrials.gov) - P1 | N=140 | Recruiting | Sponsor: BioXcel Therapeutics Inc | Trial completion date: Jul 2024 ➔ Dec 2026 | Trial primary completion date: Jul 2024 ➔ Jul 2026

Trial completion date • Trial primary completion date • Bipolar Disorder • CNS Disorders • Mood Disorders • Pediatrics • Psychiatry • Schizophrenia • Schizophreniform Disorder

A Phase 3 Study of Sublingual Dexmedetomidine for Episodic Treatment of Agitation Associated with Alzheimer's Dementia (CTAD 2024) - No abstract available

Late-breaking abstract • P3 data • Alzheimer's Disease • CNS Disorders • Dementia

BioXcel Therapeutics Announces Clinical Prioritization and Update on BXCL501 Late-Stage Programs for Agitation (GlobeNewswire) - "BioXcel Therapeutics, Inc...today announced its clinical prioritization and an update on its late-stage development programs for the treatment of agitation with BXCL501. The clinical prioritization is intended to optimize resource allocation and focus on the development of its lead neuroscience asset into potential new markets, while deprioritizing sales efforts for its approved drug IGALM (dexmedetomidine) sublingual film...'We are prioritizing our late-stage clinical programs for BXCL501 in...Alzheimer’s disease and placing greater emphasis on advancing these trials,'...The Company plans to continue to supply IGALMI to current and future customers through existing distribution channels, without commercial support."

Pipeline update • Alzheimer's Disease • CNS Disorders

BioXcel advances Alzheimer’s agitation treatment (Investing.com) - "BioXcel Therapeutics, Inc., a biopharmaceutical company, has announced the submission of its phase 3 trial protocol for BXCL501 to the U.S. Food and Drug Administration (FDA) on Thursday. The study, named TRANQUILITY In-Care, is pivotal in evaluating the efficacy and safety of a 60 mcg dose of BXCL501, specifically for agitation associated with Alzheimer’s dementia."

Clinical protocol • FDA event • Alzheimer's Disease • CNS Disorders

Dexmedetomidine HCL (BXCL501) as a potential treatment for alcohol use disorder and comorbid PTSD: A phase 1b, placebo-controlled crossover laboratory study. (PubMed, Am J Addict) - "This is the first study to provide scientific support for BXCL501's potential to treat PTSD and comorbid AUD."

Clinical • Journal • P1 data • Addiction (Opioid and Alcohol) • Anesthesia • CNS Disorders • Mood Disorders • Post-traumatic Stress Disorder • Psychiatry

Effect of Sublingual Formulation of Dexmedetomidine Hydrochloride (HCl) (BXCL501) - Outpatient Study (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: Pharmacotherapies for Alcohol and Substance Use Disorders Alliance | Initiation date: Apr 2024 ➔ Aug 2024

Trial initiation date • Addiction (Opioid and Alcohol) • CNS Disorders • Mood Disorders • Post-traumatic Stress Disorder

A Phase Ib/II Study of BXCL501 in Agitation Associated with Dementia (AAIC 2024) - "There are currently no FDA-approved treatments for acute management of agitation in elderly patients with dementia. In this study, BXCL501 60 µg and 40 µg significantly reduced the symptoms of agitation at 2 hours after administration in this patient population, as measured by PEC. At those doses, the treatments were relatively well tolerated."

P1/2 data • Alzheimer's Disease • Cardiovascular • CNS Disorders • Dementia • Hypotension

BXCL501 for Agitation in Schizophrenia (clinicaltrials.gov) - P2 | N=50 | Recruiting | Sponsor: Yale University | Trial completion date: Dec 2024 ➔ Jun 2025 | Trial primary completion date: Jun 2024 ➔ Dec 2024

Trial completion date • Trial primary completion date • CNS Disorders • Psychiatry • Schizophrenia

BioXcel Therapeutics to Present at Jefferies Global Healthcare Conference (GlobeNewswire) - "BioXcel Therapeutics, Inc...announced that CEO Vimal Mehta, Ph.D., will present at the Jefferies Global Healthcare Conference in New York City. The presentation is set for Wednesday, June 5 at 3 p.m. ET, and will highlight BioXcel Therapeutics’ late-stage clinical programs with BXCL501 for the acute treatment of agitation associated with Alzheimer’s dementia, bipolar disorders, and schizophrenia. Dr. Mehta will be joined by Vincent J. O’Neill, M.D., Executive Vice President, Chief of Product Development and Medical Officer."

Clinical • Alzheimer's Disease • Bipolar Disorder • CNS Disorders • Dementia • Schizophrenia

BioXcel Therapeutics Announces Oral and Poster Presentations at the 2024 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting (GlobeNewswire) - "BioXcel Therapeutics, Inc...today announced that Rob Risinger, M.D., Chief Medical Officer of Neuroscience, will deliver oral and poster presentations at the 2024 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting."

P1/2 data • CNS Disorders • Dementia

Dexmedetomidine Sublingual Film for the Management of Agitation in Delirium: Safety and Preliminary Efficacy (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Jeff C. Huffman, MD | N=80 ➔ 0 | Unknown status ➔ Withdrawn

Enrollment change • Trial withdrawal • CNS Disorders

Tachyphylaxis, Tolerance, & Withdrawal Post Treatment With Igalmi for Agitation in Schizophrenia or Bipolar Disorder (clinicaltrials.gov) - P4 | N=23 | Completed | Sponsor: BioXcel Therapeutics Inc | Recruiting ➔ Completed

Trial completion • Bipolar Disorder • CNS Disorders • Mood Disorders • Psychiatry • Schizophrenia • Schizophreniform Disorder

A Phase Ib/II Multicenter, Randomized, Double Blind, Placebo Controlled, Ascending Dose Finding Study of BXCL501 in Agitation Associated with Dementia (ASCP 2024) - "There are currently no FDA-approved treatments for acute management of agitation in elderly patients with dementia. In this study, BXCL501 60 µg and 40 µg significantly reduced the symptoms of agitation at 2 hours after administration in this patient population, as measured by PEC. At those doses, the treatments were relatively well tolerated."

Clinical • P1/2 data • Alzheimer's Disease • Bipolar Disorder • CNS Disorders • Cognitive Disorders • Dementia • Mood Disorders • Psychiatry • Schizophrenia

An Exploratory Comparison of Sublingual Dexmedetomidine with Quetiapine in Healthy Elderly Subjects (ASCP 2024) - "In this exploratory study, observations correspond to those expected for the types of medications studied: sleepiness and orthostatic hypotension. Of note, sleepiness was less pronounced with BXCL501 60 µg compared with quetiapine 25-100 mg. The limitation of the study was the small sample size; these results should thus be considered preliminary."

Clinical • Alzheimer's Disease • CNS Disorders • Dementia

Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of BXCL501 with Concomitant Treatment with Anti-Depressant in Healthy Volunteers (ASCP 2024) - "Dex is more potent with greater intrinsic activity than other alpha2-adrenergic agonists (e.g. clonidine, guanfacine and lofexidine)...The most frequent TEAEs related to duloxetine were hypotension and orthostatic hypotension (together 31%) and abdominal pain and constipation (together 15%)... This study confirmed the safety, tolerability and PK profile of BXCL501 with repeated dosing across a range of doses. Concomitant administration of BXCL501 (split doses) with an anti-depressant which increases both serotonergic and noradrenergic signaling was well tolerated. These data provide initial support that adjunctive BXCL501 could be a potential treatment option for acute conditions of distress (e.g."

Clinical • ADHD (Impulsive Aggression) • Alzheimer's Disease • Attention Deficit Hyperactivity Disorder • Bipolar Disorder • CNS Disorders • Dementia • Depression • Mood Disorders • Pain • Psychiatry • Schizophrenia

BioXcel Therapeutics Announces TRANQUILITY In-Care Pivotal Phase 3 Trial Plan With BXCL501 for Agitation Associated With Alzheimer’s Dementia (GlobeNewswire) - "BioXcel Therapeutics, Inc...announced details regarding the planned design of its upcoming TRANQUILITY In-Care Phase 3 trial to evaluate BXCL501, the company’s investigational proprietary, orally dissolving film formulation of dexmedetomidine, as a potential acute treatment for agitation associated with Alzheimer’s dementia (AAD) in the care setting. The Company’s plan to conduct this trial is based on feedback received from the U.S. Food and Drug Administration (FDA), following the recent receipt of minutes from the Type B/Breakthrough Therapy designation meeting held with the agency on February 20, 2024....The Company expects to generate additional Phase 3 efficacy and safety data in the TRANQUILITY....The Company also plans to discuss the details of the requirement for long-term safety data at a future meeting with the FDA."

Clinical protocol • FDA event • Alzheimer's Disease • CNS Disorders • Dementia