Recent Updates
Recently added Catalysts

Brexanolone

Phase 2

Alcohol Use Disorder (AUD) | Small molecule | Psychiatry |United Therapeutics Corporation|Last Updated: May 31, 2025

Target and mechanism

Molecular targetGABRA1, GABRB2, GABRG2, GABRB3, GABRA4, GABRG3
Target classPositive Allosteric Modulator
ModalitySmall molecule

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment40

FDA Designations

No designations recorded

Clinical trial landscape

Brexanolone · 1 trial · 2 indications

Phase 2 1
NCT06580444Brexanolone to Target Concurrent PTSD and Stress Induced Alcohol Use in Veterans: A Dose Finding StudyAlcohol Use Disorder (AUD)
NOT YET_RECRUITING40 Analytics
PHASE2NOT YET_RECRUITING
Brexanolone to Target Concurrent PTSD and Stress Induced Alcohol Use in Veterans: A Dose Finding Study
Alcohol Use Disorder (AUD)Unlock trial analytics

Study Endpoints

Primary Endpoints

Severity and numbers of treatment-emergent AEs
From onset of Brexanolone administration until 30-day follow up.

The number and proportion of individuals experiencing any adverse event, any SAE, and any related adverse event overall will be summarized using frequency tables. No formal statistical tests will be conducted, rather the safety will be evaluated from a risk/benefit perspective. Any p-values reported will be for descriptive purposes only.

Change in Heart Rate from onset of Brexanolone administration until discharge from the drug administration session.
An average of 22 hours (From the start of Brexanolone administration on day 1 at 10 am until 7 am on day 2)

The change in heart rate a physiological safety/stress measure are continuous and obtained multiple times after study drug administration which occurs on day 1. This measure will be analyzed using descriptive statistics and graphical summaries including time-specific summary statistics (e.g., box and whisker plots).

Change in Blood Pressure from onset of Brexanolone administration until discharge from the drug administration session.
An average of 22 hours (From the start of Brexanolone administration on day 1 at 10 am until 7 am on day 2)

The change in blood pressure a physiological safety/stress measure are continuous and obtained multiple times after study drug administration which occurs on day 2. This measure will be analyzed using descriptive statistics and graphical summaries including time-specific summary statistics (e.g., box and whisker plots).

Change in Cortisol from onset of Brexanolone administration until discharge from the drug administration session.
FAn average of 22 hours (From the start of Brexanolone administration on day 1 at 10 am until 7 am on day 2)

The change in Cortisol a physiological safety/stress measure are continuous and obtained multiple times after study drug administration which occurs on day 2. This measure will be analyzed using descriptive statistics and graphical summaries including time-specific summary statistics (e.g., box and whisker plots).

Change in percentage of milliliters of alcohol consumed during the 2-hour ad-libitum drinking paradigm between laboratory sessions (stress vs. neutral).
During the 2-hour ad-libitum drinking paradigm between lab sessions (stress Vs Neutral)

A set of dose-response models will be tested response using the Multiple Comparisons Procedure Modifications (MCP-Mod) methodology including change in percentage of milliliters consumed during the 2-hour ad-libitum drinking paradigm within laboratory sessions (stress and neutral) as dependent variable, the treatment as factor (90, 60, 30 and 0 mcg/kg/h), and baseline dependent value. These dose-response models with repeated measures will be used to test the effects of the regimens of 90, 60, and 30 mcg/kg/h doses of brexanolone on alcohol consumption in self-administration.

Change in Posttraumatic Stress Disorder Checklist for DSM-5 (PCL-5) score from intake until 30-day follow-up
From intake, day 0 through day 37 (30-day follow-up).

Another set of dose-response models will test Multiple Comparisons Procedure Modifications (MCP-Mod) methodology for the 30-day follow-up in PCL-5 score as dependent variable, the treatment as factor (90, 60, 30 and 0 mcg/kg/h), and baseline dependent value. These dose-response models will be used to test the effects of the regimens of 90, 60, 30 and 0 mcg/kg/h doses of brexanolone on PTSD symptoms. Scale PCL-5 is 0-4 with 0 being not at all and 4 being extreme; the higher score is worse outcome

Change in Change in Posttraumatic Stress Disorder Checklist for DSM-5 (PCL-5) score from intake until end of dosing.
An average of 22 hours (From intake, day 0 through end of infusion on Day 2)

Another set of dose-response models will test Multiple Comparisons Procedure Modifications (MCP-Mod) methodology including change from intake to end of dosing in PCL-5 score as dependent variable, the treatment as factor (90, 60, 30 and 0 mcg/kg/h), and baseline dependent value. The same modeling will be repeated for the 30-day follow-up timepoint. These dose-response models will be used to test the effects of the regimens of 90, 60, 30 and 0 mcg/kg/h doses of brexanolone on PTSD symptoms. Scale PCL-5 is 0-4 with 0 being not at all and 4 being extreme; the higher score is worse outcome

Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeOTHER

Treatment Arms

ArmTypeDescription
PlaceboNO_INTERVENTIONA 0.9% bag of sodium chloride will be administered intravenously. Participant will be given a self-administered alcoholic drink of their choice that is individually calculated, and they will receive money up to $15 for every minute they resist drinking.
90mcg/kg/h dose of BrexanoloneACTIVE_COMPARATORBrexanolone will be diluted with sterile water and 0.9% sodium chloride and will be administered intravenously. Participant will be given a self-administered alcoholic drink of their choice that is individually calculated, and they will receive money up to $15 for every minute they resist drinking.
60mcg/kg/h dose of BrexanoloneACTIVE_COMPARATORBrexanolone will be diluted with sterile water and 0.9% sodium chloride and will be administered intravenously. Participant will be given a self-administered alcoholic drink of their choice that is individually calculated, and they will receive money up to $15 for every minute they resist drinking.
30mcg/kg/h dose of BrexanoloneACTIVE_COMPARATORBrexanolone will be diluted with sterile water and 0.9% sodium chloride and will be administered intravenously. Participant will be given a self-administered alcoholic drink of their choice that is individually calculated, and they will receive money up to $15 for every minute they resist drinking.

Interventions

NameTypeDescription
BrexanoloneDRUGThe participant will receive either a placebo, or 90, 60, or 30 mcg/kg/hr of Brexanolone over a 20-hour period intravenous infusion.
Unlock Study Design Details

Eligibility Criteria

Age Range21 Years to 70 Years
SexALL
Healthy VolunteersNo

Inclusion Criteria: 1. Provision of signed and dated informed consent form. 2. Stated willingness to comply with all study procedures and availability for the duration of the study. 3. Male or female, aged 21-70 years old, either active duty or with a history of US military service. 4. Current DSM-...

Unlock Eligibility Criteria

Frequently asked questions about Brexanolone

What is Brexanolone used for in Alcohol Use Disorder?

Brexanolone is an investigational small molecule being studied for Alcohol Use Disorder (AUD), specifically in patients with concurrent post-traumatic stress disorder (PTSD). It is being evaluated in a Phase 2 dose-finding study targeting stress-induced alcohol use in veterans. The drug is not yet approved for this indication.

What does Brexanolone target?

Brexanolone is a small molecule that modulates the GABA-A receptor, a key target in the central nervous system involved in inhibitory neurotransmission. By acting on this receptor, it aims to address stress-induced alcohol use in patients with AUD and PTSD. This mechanism is being studied in a Phase 2 clinical trial.

Who makes Brexanolone?

Brexanolone is being developed by United Therapeutics Corporation, a biopharmaceutical company listed on NASDAQ under the ticker UTHR. The company is conducting a Phase 2 study of brexanolone for Alcohol Use Disorder and PTSD in veterans.

What phase is Brexanolone in?

Brexanolone is in Phase 2 clinical development for Alcohol Use Disorder (AUD) with concurrent PTSD. It is currently being studied in a dose-finding trial that is not yet recruiting participants. The drug is investigational and has not been approved for this use.

What clinical trials is Brexanolone in?

Brexanolone is being studied in a Phase 2 clinical trial with the identifier NCT06580444. This randomized, double-blind, active-controlled study aims to evaluate brexanolone for targeting concurrent PTSD and stress-induced alcohol use in veterans. The trial plans to enroll 40 participants aged 21 years and older.

Is Brexanolone the same as Zulresso?

Brexanolone is the same drug as Zulresso, which is the brand name for brexanolone. Zulresso is approved for postpartum depression, while brexanolone is now being investigated for Alcohol Use Disorder and PTSD. The drug is being developed by United Therapeutics for this new indication.