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ML-004

Phase 2

Autism Spectrum Disorder | Small molecule | Psychiatry |MapLight Therapeutics, Inc.|Last Updated: Jul 20, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment288

FDA Designations

No designations recorded

Clinical trial landscape

ML-004 · 2 trials · 1 indication

Phase 2 2
NCT05889273ML-004 Open-Label Extension Study in Adults and Adolescents With Autism Spectrum Disorders (ASD)Autism Spectrum Disorder
ACTIVE NOT_RECRUITING120 Analytics
NCT05081245ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD)Autism Spectrum Disorder
COMPLETED168 Analytics
PHASE2ACTIVE NOT_RECRUITING
ML-004 Open-Label Extension Study in Adults and Adolescents With Autism Spectrum Disorders (ASD)
Autism Spectrum DisorderUnlock trial analytics
PHASE2COMPLETED
ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD)
Autism Spectrum DisorderUnlock trial analytics

Study Endpoints

Primary Endpoints

Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Baseline up to Day 362
Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score
Baseline up to Day 110

Change from baseline in the ABI-Social Communication Domain Score will be reported. The ABI is a 62-item questionnaire for reporting the behaviors of subjects (ages: 3 years-adulthood) diagnosed with ASD. The tool is suitable for completion by parents or care/study partners of people with ASD. Each item assesses either quality (from not at all to without help) or frequency (never to very often) of a particular behavior. The Social Communication domain score is the sum of the scores in the social communication domain divided by the number of items in the domain.

Secondary Endpoints

Frequency of occurrence of Serious Adverse Events (SAEs)
Baseline up to Day 362
Frequency of occurrence of TEAEs leading to discontinuation.
Baseline up to Day 362
Frequency of occurrence of TEAEs arising from clinically important changes in other safety assessments.
Baseline up to Day 362
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ML-004 (IR)/(ER) tabletEXPERIMENTALML-004 is a bilayer immediate-release (IR)/extended-release (ER, gastroretentive) oral tablet formulation. ML-004 is taken orally once daily and provided as a tablet in two dose strengths of 12 mg (3 mg IR/9 mg ER) and 24 mg tablet (6 mg IR/18 mg ER). Dose levels for this study are 12 mg (provided as one 12 mg tablet), 24 mg (one 24 mg tablet), 48 mg (two 24 mg tablets), and 72 mg (three 24 mg tablets).
ML-004 PlaceboPLACEBO_COMPARATORMatched placebo oral tablets will consist of same excipients as the investigational drug but without any ML-004. Placebo taken orally once daily to match tablet number of ML-004 dose levels for 12 mg (1 tablet), 24 mg (1 tablet), 48 mg (2 tablets), and 72 mg (3 tablets).

Interventions

NameTypeDescription
ML-004 (IR)/(ER) tabletDRUGParticipants will receive ML-004 once daily.
ML-004 PlaceboDRUGParticipants will receive matching placebo once daily.
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Eligibility Criteria

Age Range12 Years to 46 Years
SexALL
Healthy VolunteersNo
Study Sites21

Inclusion Criteria: * Has completed Study ML-004-002 within the past 90 days * Age 12 years to 46 years at screening * Has a designated care/study partner who can reliably report on symptoms * Has a diagnosis of Autism Spectrum Disorder (ASD) * Has a body mass index (BMI) ≥18 kg/m² * Psychoactive m...

Countries:United StatesAustraliaCanada
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Recent Changes (Last 90 Days)

MEDIUMJul 20, 2026NCT05889273Status: ENROLLING_BY_INVITATION → ACTIVE_NOT_RECRUITING
MEDIUMJul 20, 2026NCT05889273Status: ENROLLING_BY_INVITATION → ACTIVE_NOT_RECRUITING
MEDIUMJun 19, 2026NCT05081245TRIAL_REMOVED: changed
MEDIUMJun 19, 2026NCT05081245TRIAL_REMOVED: changed
MEDIUMJun 19, 2026NCT05081245TRIAL_REMOVED: changed

Frequently asked questions about ML-004

What is ML-004 used for?

ML-004 is an investigational small molecule being developed for Autism Spectrum Disorder (ASD). It is currently in Phase 2 clinical development and is not yet approved by the FDA. The drug is being studied in adolescents and adults with ASD.

Who makes ML-004?

ML-004 is being developed by MapLight Therapeutics, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol MPLT. MapLight is conducting clinical trials to evaluate the safety and efficacy of ML-004 in patients with Autism Spectrum Disorder.

What phase is ML-004 in?

ML-004 is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug is being studied in two Phase 2 trials for Autism Spectrum Disorder, one of which has been completed and one that is active but not recruiting.

What clinical trials is ML-004 in?

ML-004 has been studied in two clinical trials. The first, NCT05081245, was a Phase 2 study in adolescents and adults with Autism Spectrum Disorder, which has been completed with 168 participants. The second, NCT05889273, is an open-label extension study with 120 participants, currently active but not recruiting. Both trials were conducted in the United States, Australia, and Canada.

Is ML-004 the same as ML-004 (IR)/(ER) tablet?

Yes, ML-004 is also known as ML-004 (IR)/(ER) tablet. The drug is being developed in immediate-release and extended-release tablet formulations. This alternative name may be used in clinical trial documentation and other references to the drug.