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BXCL501

Phase 3

Agitation | Small molecule | Neurology |BioXcel Therapeutics, Inc.|Last Updated: Aug 11, 2026

Target and mechanism

ModalitySmall molecule

Also known as BXCL501 (180 micrograms)

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment151

FDA Designations

BREAKTHROUGH_THERAPYFAST_TRACK

Clinical trial landscape

BXCL501 · 3 trials · 10 indications

Phase 3 2Phase 1 1
NCT05658510Dexmedetomidine in the Treatment of Agitation Associated With Schizophrenia and Bipolar Disorder (SERENITY III)Agitation,Psychomotor
COMPLETED452 Analytics
NCT05271552Dexmedetomidine in the Treatment of Agitation Associated With Dementia (TRANQUILITY II)Agitation
COMPLETED151 Analytics
PHASE3COMPLETED
Dexmedetomidine in the Treatment of Agitation Associated With Schizophrenia and Bipolar Disorder (SERENITY III)
Agitation,PsychomotorUnlock trial analytics
PHASE3COMPLETED
Dexmedetomidine in the Treatment of Agitation Associated With Dementia (TRANQUILITY II)
AgitationUnlock trial analytics

Study Endpoints

Primary Endpoints

Part 1: Change from baseline in Positive and Negative Syndrome Scale - Excited (PEC) total score
2 hours

The Positive and Negative Syndrome Scale - Excited Component (PEC) comprises 5 items associated with agitation: poor impulse control, tension, hostility, uncooperativeness, and excitement; each scored 1 (minimum) to 7 (maximum). The PEC, the sum of these 5 subscales, thus ranges from 5 (absence of agitation) to 35 (extremely severe)

Part 2: The incidence of SAEs and TEAEs compared with placebo.
Through study completion, an average of 12 weeks

To assess the safety of 120 mcg BXCL501 when used in an at-home environment based on serious adverse events (SAE) and treatment emergent adverse events (TEAEs).

Absolute Change From Baseline in Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score for the First Episode of Agitation
120 minutes

The Positive and Negative Syndrome Scale - Excited Component (PEC) comprises 5 items associated with agitation: poor impulse control, tension, hostility, uncooperativeness, and excitement; each scored 1 (minimum) to 7 (maximum). The PEC, the sum of these 5 subscales, thus ranges from 5 (absence of agitation) to 35 (extremely severe)

Safety of BXCL501 versus Placebo and Lofexidine
Study Days 1-7

Establish the safety of BXCL501 relative to placebo and lofexidine in subjects with OUD who are physically dependent on opioids as measured by the frequency of adverse events.

Secondary Endpoints

Part 1: Clinical Global Impression - Improvement (CGI-I)
2 hours
Part 1: Change in Modified Clinical Global Impression - Severity (mCGI-S) scores from Baseline
2 hours
Part 1:The number of responders based on the Modified Clinical Global Impression - Severity (mCGI-S) score
2 hours
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1: 60 mcg of BXCL501EXPERIMENTALSublingual film containing 60 Micrograms Dexmedetomidine
Part 1: Matching PlaceboPLACEBO_COMPARATORSublingual Placebo film
Part 2: 120 mcg of BXCL501EXPERIMENTALSublingual film containing 120 Micrograms Dexmedetomidine
Part 2: Matching PlaceboPLACEBO_COMPARATORSublingual Placebo film
Cohort 1- 40 MicrogramsEXPERIMENTALSublingual film containing 40 Micrograms Dexmedetomidine
Cohort 2- 60 MicrogramsEXPERIMENTALSublingual film containing 60 Micrograms Dexmedetomidine
PlaceboPLACEBO_COMPARATORSublingual Placebo film
BXCL501 (180 micrograms)EXPERIMENTALThe current study is a randomized, double-blind, double-dummy inpatient study comparing BXCL501 (180 and 240 ug BID), lofexidine (as a positive control), and placebo.
BXCL501 (240 micrograms)EXPERIMENTALThe current study is a randomized, double-blind, double-dummy inpatient study comparing BXCL501 (180 and 240 ug BID), lofexidine (as a positive control), and placebo.
Lofexidine (Positive Control)ACTIVE_COMPARATORThe current study is a randomized, double-blind, double-dummy inpatient study comparing BXCL501 (180 and 240 ug BID), lofexidine (as a positive control), and placebo.

Interventions

NameTypeDescription
BXCL501DRUGSublingual Film
Matching PlaceboDRUGSublingual Placebo Film
BXCL501 (180 micrograms)DRUGThroughout the 7-day inpatient study (Figure 1; Table 1), participants will receive sublingual BXCL501 or placebo twice daily and lofexidine or placebo 4 times daily using a double-blind, double-dummy design.
BXCL501 (240 micrograms)DRUGThroughout the 7-day inpatient study (Figure 1; Table 1), participants will receive sublingual BXCL501 or placebo twice daily and lofexidine or placebo 4 times daily using a double-blind, double-dummy design.
PlaceboOTHERThroughout the 7-day inpatient study (Figure 1; Table 1), participants will receive sublingual BXCL501 or placebo twice daily and lofexidine or placebo 4 times daily using a double-blind, double-dummy design.
Lofexidine (Positive Control)OTHERThroughout the 7-day inpatient study (Figure 1; Table 1), participants will receive sublingual BXCL501 or placebo twice daily and lofexidine or placebo 4 times daily using a double-blind, double-dummy design.
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites24

A patient may enroll in only one part of the study; either Part 1 or Part 2. Inclusion Criteria: * Male and female patients between the ages of 18 to 75 years, inclusive * Patients who can read, understand and provide written informed consent. * Patients who have met Diagnostic and Statistical Man...

Countries:United States
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Recent Changes (Last 90 Days)

MEDIUMSep 7, 2026NCT05658510TRIAL_REMOVED: changed
MEDIUMSep 7, 2026NCT05658510TRIAL_REMOVED: changed
MEDIUMSep 7, 2026NCT05658510TRIAL_REMOVED: changed
MEDIUMSep 7, 2026NCT05658510TRIAL_REMOVED: changed

Frequently asked questions about BXCL501

What is BXCL501 used for?

BXCL501 is an investigational small molecule being developed for the treatment of agitation associated with dementia, schizophrenia, and bipolar disorder, as well as for opioid withdrawal. It is a sublingual formulation of dexmedetomidine, currently in Phase 3 clinical trials for agitation indications.

What does BXCL501 target?

BXCL501 contains dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. By activating these receptors, it is thought to produce sedative and anxiolytic effects without causing respiratory depression, which is why it is being studied for acute agitation and opioid withdrawal.

Who makes BXCL501?

BXCL501 is being developed by BioXcel Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol BTAI. The company is focused on utilizing artificial intelligence to identify and develop therapies in neuroscience and immuno-oncology.

What phase is BXCL501 in?

BXCL501 is in Phase 3 clinical development for agitation associated with schizophrenia, bipolar disorder, and dementia. It has received Breakthrough Therapy and Fast Track designations from the FDA. It is not yet approved and remains investigational.

What clinical trials is BXCL501 in?

BXCL501 has been studied in several clinical trials, including NCT05271552 (TRANQUILITY II) for agitation in dementia, NCT05658510 (SERENITY III) for agitation in schizophrenia and bipolar disorder, and NCT05712707 for opioid withdrawal. These trials are completed or active, with a total enrollment of 452 participants.

Is BXCL501 the same as dexmedetomidine?

BXCL501 is a sublingual formulation of dexmedetomidine, a medication already approved intravenously for sedation. BXCL501 is designed for acute treatment of agitation and is being investigated as a potential at-home treatment option.