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BPL-003

Phase 2

Alcohol Use Disorder | Small molecule | Psychiatry |AtaiBeckley Inc.|Last Updated: Jul 6, 2026

Target and mechanism

Molecular target5-HT2AR
Target classReceptor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment13

FDA Designations

BREAKTHROUGH_THERAPY

Clinical trial landscape

BPL-003 · 4 trials · 3 indications

Phase 2 3Phase 1 1
NCT05870540BPL-003 Efficacy and Safety in Treatment Resistant DepressionTreatment Resistant Depression
COMPLETED196 Analytics
NCT05674929An Open-Label, Single Dose Study in Patients With Alcohol Use DisorderAlcohol Use Disorder
COMPLETED13 Analytics
NCT05660642An Open-Label Study to Evaluate the Safety, Tolerability and Pharmacodynamics of BPL-003 in Patients With Treatment Resistant DepressionTreatment Resistant Depression
RECRUITING72 Analytics
PHASE2COMPLETED
BPL-003 Efficacy and Safety in Treatment Resistant Depression
Treatment Resistant DepressionUnlock trial analytics
PHASE2COMPLETED
An Open-Label, Single Dose Study in Patients With Alcohol Use Disorder
Alcohol Use DisorderUnlock trial analytics
PHASE2RECRUITING
An Open-Label Study to Evaluate the Safety, Tolerability and Pharmacodynamics of BPL-003 in Patients With Treatment Resistant Depression
Treatment Resistant DepressionUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from baseline in Montgomery-Asberg Depression Rating Scale (MADRS)
4 weeks

High compared to low dose of BPL-003. The MADRS is a 10-item diagnostic questionnaire used to measure the severity of depressive episodes in participants with mood disorders. A higher MADRS score indicates more severe depression, and each item yields a score of 0-6.

OLE Primary Safety Outcome Measure
8 weeks

To determine the safety of a second dose of BPL-003 given with psychological support to participants with TRD as assessed by treatment-emergent adverse events.

Percentage of Participants With Treatment Emergent Adverse Events
Up to 12 weeks

The number (%) of participants with at least one treatment-emergent adverse event is presented. More detailed information is provided in the dedicated 'Adverse Events' section

Percentage of Participants With Clinically Significant Abnormal Laboratory Tests
Up to 12 weeks

The number (%) of participants with any clinically significant abnormal laboratory tests (routine haematology, clinical chemistry and coagulation) is presented

Percentage of Participants With Clinically Significant Abnormal Vital Signs
Up to 12 weeks

The number (%) of participants with any clinically significant abnormal vital signs (blood pressure, heart rate and temperature) result is presented

Number of Participants With Post-baseline Suicidal Ideation or Behaviour Based on C-SSRS Score
Up to 12 weeks

The C-SSRS was performed at screening, on Day 0 (dosing day), and on Days 1, 7, and 84 post-dose. Participants were counted if they answered 'yes' to any question. At baseline, the C-SSRS assessed the worst-point suicidal ideation experienced during the participant's lifetime. Beyond baseline, suicidal ideation and behaviour since last visit was assessed.

Time to Readiness for Discharge Post-dose Using the Readiness for Discharge Questionnaire (RDQ)
1 Day

The RDQ was a brief assessment scale to ensure that ahead of discharge after BPL-003 dosing, participants: * Were fully responsive, aware of their surroundings, and reacted adequately * The acute psychedelic effects of the drug had completely subsided * Were fully orientated (name, location, time) * Had normal (or only slightly elevated) blood pressure and pulse rate, and normal breathing frequency and body temperature * Had a stable gate, normal muscle coordination, and could walk safely * Had only mild to moderate potential side affects that did not need to be medically monitored * Had no acute suicidal ideations or suicidal intentions * Possible distress or feelings of overwhelm had sufficiently subsided and the participant felt safe to be discharged. Readiness for discharge was assessed at 90 minutes post-dose and then every 30 minutes until the participant was deemed ready for discharge (eg, the answer was 'yes' to all of the above items).

Percentage of Participants With Occurrence of Reactivation Using the Reactivation Questionnaire (ReAQ)
Up to 12 weeks

The occurrence and (if applicable) frequency, emotional valence, and functional impact of any reactivation events was determined. To determine if reactivation had occurred, participants were asked if they had any flashbacks or recurrence of any effects of the study drug experience.

1. To assess the safety and tolerability of single or multiple intranasal doses of BPL-003 in patients with treatment resistant depression
Baseline to 12 weeks post dose

* Percentage of patients with treatment emergent adverse events * Percentage of patients with clinically significant abnormal laboratory tests * Percentage of patients with clinically significant abnormal vital signs * Percentage of patients with clinically significant findings in physical examination * Percentage of patients with clinically significant ECG parameters or cardiac telemetry abnormalities (Part 1 Arm B only) * Percentage of patients with suicidal ideation or behaviour

Percentage of subjects with treatment emergent AEs (TEAES)
From screening through to the follow up visit, up to 65 days

Secondary Endpoints

Change from baseline in Montgomery-Asberg Depression Rating Scale (MADRS)
1 week
Safety of BPL-003 given with psychological support as assessed by number and percentage of participants with adverse events
8 weeks
Safety of BPL-003 given with psychological support as assessed by percentage of participants with clinically significant abnormal laboratory tests
8 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Low doseEXPERIMENTALActive placebo comparator
Medium doseEXPERIMENTAL -
High doseEXPERIMENTAL -
MonophasicEXPERIMENTAL -
BiphasicEXPERIMENTAL -
BPL-003 armEXPERIMENTAL -
Arm AEXPERIMENTAL -
Arm BEXPERIMENTAL -
Placebo armPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
BPL-003DRUGA single dose administered intranasally
PlaceboOTHERA single dose of placebo will be administered intranasally
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites42

Inclusion Criteria: 1. At least moderate major depressive disorder. 2. Diagnosed with TRD defined as failure to respond to an adequate dose and duration of at least 2 pharmacological treatments based on the MGH ATRQ assessment. 3. Hamilton Depression Rating Scale score ≥19 at Screening and Baseline...

Countries:United StatesAustraliaGermanyPolandSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWAug 1, 2026NCT05660642Enrollment: 64 → 72
LOWAug 1, 2026NCT05660642Enrollment: 64 → 72
LOWAug 1, 2026NCT05660642Enrollment: 64 → 72

Frequently asked questions about BPL-003

What is BPL-003 used for?

BPL-003 is an investigational small molecule being studied for psychiatric conditions, including treatment resistant depression and alcohol use disorder. It is also being evaluated in pharmacokinetic studies in healthy adults. BPL-003 is not approved and remains in clinical development.

What does BPL-003 target?

BPL-003 targets the 5-HT2A receptor, a subtype of serotonin receptor. This receptor is the molecular target for the drug's mechanism of action in the central nervous system.

Who is developing BPL-003?

BPL-003 is being developed by AtaiBeckley Inc., a biopharmaceutical company. The company's stock is listed under the ticker symbol ATAI.

What phase is BPL-003 in?

BPL-003 is in Phase 2 clinical development. It has completed a Phase 1 study and multiple Phase 2 studies, with one Phase 2 trial currently recruiting participants. The drug has not received FDA approval.

What clinical trials is BPL-003 in?

BPL-003 has been studied in several clinical trials. NCT05347849 was a completed Phase 1 single ascending dose study in healthy subjects. NCT05660642 is a recruiting Phase 2 open-label study in treatment resistant depression. NCT05674929 was a completed Phase 2 study in alcohol use disorder, and NCT05870540 was a completed Phase 2 efficacy and safety study in treatment resistant depression.

Is BPL-003 the same as any other drug?

BPL-003 is also known by the name BPL003. It is a single investigational compound and is not the same as any other marketed drug.