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BMB-101

Phase 2

Absence Epilepsy | Small molecule | Neurology |Bright Minds Biosciences Inc.|Last Updated: Dec 5, 2025

Target and mechanism

Molecular target5-HT2C
Target classReceptor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment20

FDA Designations

No designations recorded

Clinical trial landscape

BMB-101 · 3 trials · 6 indications

Phase 2 2Phase 1 1
NCT07266324A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.Prader-Willi Syndrome
NOT YET_RECRUITING16 Analytics
NCT06401538BMB-101 in Absence Epilepsy and DEEAbsence Epilepsy
RECRUITING20 Analytics
PHASE2NOT YET_RECRUITING
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
Prader-Willi SyndromeUnlock trial analytics
PHASE2RECRUITING
BMB-101 in Absence Epilepsy and DEE
Absence EpilepsyUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from Baseline in Hyperphagia Questionnaire for Clinical Trials scores over time in Prader-Willi Syndrome participants.
16 weeks

The Hyperphagia Questionnaire for Clinical Trials consists of 9 items; each rated on a scale from 0 (no symptoms) to 4 (severe symptoms). The total score ranges from 0 to 36, with higher scores indicating worse hyperphagia symptoms. A score of approximately 13 is associated with moderate to severe hyperphagia, and a score of 22 or greater is associated with severe hyperphagia.

Change from baseline in seizure frequency in DEE subjects
10 weeks

Seizure diary

Change from baseline in the number of generalized spike-wave discharges (GSWD) on the 24 hr EEG in Absence subjects.
6 weeks

24 hour EEG

Number of Treatment-emergent Adverse Events
Baseline up to Follow Up/End of Treatment visit, an average of 8 months.

Incidence and severity of adverse events, including serious adverse events and adverse events, clinically significant changes in laboratory testing, vital signs, Holter monitoring, physical examination, and ECGs

Change in Columbia-Suicide Severity Rating Scale (C-SSRS) Response
Administered at each of the following visits in Part 3: Screening, Clinic Discharge, and Follow-up/Early Withdrawal.

Type of Suicidal Ideation, Intensity (1 - 5, with 5 being most severe), Suicidal Behavior

Secondary Endpoints

Change from Baseline in hyperphagia severity score as measured by the Caregiver Global Impression of Severity 7-point scale over time.
16 weeks
Change from Baseline in hyperphagia severity score as measured by the Clinician Global Impression of Severity 7-point scale over time.
16 weeks
Change from Baseline in severity of Prader-Willi Syndrome disease scores as measured by the Clinician Global Impression of Severity 7-point scale over time.
16 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
BMB-101EXPERIMENTALParticipants receive BMB-101 (10mg/mL liquid) orally
PlaceboPLACEBO_COMPARATORParticipants receiving matched placebo orally

Interventions

NameTypeDescription
BMB-101DRUGParticipants will receive weekly ascending oral doses of BMB-101(10 mg/mL) twice daily (BID) for 16 weeks. Doses will be based on weight (kg) and will initially start at 1.67 mg/kg. Doses may be titrated in 0.33 mg/kg increments based on tolerability up to a maximum dose of 2.0 mg/kg.
PlaceboDRUGMatched Placebo
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Participant must be aged 18-65 years (both inclusive). * Genetically confirmed diagnosis of Prader Willi Syndrome via standard DNA testing or other commonly approved methods. * Willing and able to provide voluntary written informed consent, or have a Legally Authorized Represe...

Countries:Australia
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Frequently asked questions about BMB-101

What is BMB-101 used for?

BMB-101 is an investigational small molecule being developed for neurological conditions including Prader-Willi Syndrome, absence epilepsy, and related developmental and epileptic encephalopathies such as Jeavons Syndrome, Dravet Syndrome, and Lennox Gastaut Syndrome. It is currently in clinical development and has not been approved by regulatory authorities.

What does BMB-101 target?

BMB-101 targets the 5-HT2C receptor, a subtype of serotonin receptor involved in neurological signaling. By acting on this receptor, the drug is being studied for its potential effects in conditions like Prader-Willi Syndrome and absence epilepsy, though its exact mechanism of action in these disorders is still under investigation.

Who is developing BMB-101?

BMB-101 is being developed by Bright Minds Biosciences Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol DRUG. The company is conducting clinical trials to evaluate the safety and efficacy of BMB-101 in several neurological indications.

What phase is BMB-101 in?

BMB-101 is in Phase 2 clinical development. It has completed a Phase 1 study in healthy volunteers and is currently being evaluated in Phase 2 trials for absence epilepsy and Prader-Willi Syndrome. The drug remains investigational and has not received FDA approval.

What clinical trials is BMB-101 in?

BMB-101 has been studied in three clinical trials. NCT05397041 was a completed Phase 1 study in healthy volunteers. NCT06401538 is a recruiting Phase 2 trial in absence epilepsy and related conditions. NCT07266324 is a not-yet-recruiting Phase 2 trial in Prader-Willi Syndrome. All trials are being conducted in Australia.

Is BMB-101 the same as other drugs?

BMB-101 is the primary name for this investigational drug and no alternative names have been reported. It is a distinct small molecule targeting the 5-HT2C receptor, developed by Bright Minds Biosciences, and is not known to be identical to any other marketed or investigational medication.