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Adimanebart

Phase 3

Congenital Myasthenic Syndrome | Monoclonal antibody | Neurology |argenx SE|Last Updated: Aug 4, 2026

Target and mechanism

ModalityMonoclonal antibody

Also known as Adimanebart IV

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment105

FDA Designations

No designations recorded

Clinical trial landscape

Adimanebart · 2 trials · 4 indications

Phase 3 1Phase 1 1
NCT07746089A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)Congenital Myasthenic Syndrome
NOT YET_RECRUITING105 Analytics
PHASE3NOT YET_RECRUITING
A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)
Congenital Myasthenic SyndromeUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from baseline at week 24 in 6MWT distance
Up to 24 weeks

The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.

Incidence of AEs and SAEs
up to 104 weeks

AE: adverse event; SAE: serious adverse event

AUC0-inf
Up to 20 weeks

AUC0-inf = area under the concentration-time curve from time 0 to infinity

Secondary Endpoints

Change from baseline in 6MWT distance over time
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in PROMIS PF-10b T-score over time
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
Change from baseline in QMG key component composite score over time
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Double-blinded treatment period (DBTP) - Adimanebart IVEXPERIMENTALParticipants randomized to receive Adimanebart IV
Double-blinded treatment period (DBTP) - Placebo IVPLACEBO_COMPARATORParticipants randomized to receive Placebo IV
Open-label extension (OLE) - Adimanebart IVEXPERIMENTALParticipants receive Adimanebart IV
Part 1 - Adimanebart IVEXPERIMENTALParticipants randomized to receive adimanebart IV
Part 1 - Adimanebart SC (formulation A)EXPERIMENTALParticipants randomized to receive adimanebart SC
Part 2 - Adimanebart SC (formulation A) in lower body weightsEXPERIMENTALParticipants with body weight \< 60 kg will receive adimanebart SC
Part 2 - Adimanebart SC (formulation A) in higher body weightsEXPERIMENTALParticipants with body weight between 100 and 130 kg will receive adimanebart SC
Part 3 - Adimanebart SC (formulation B)EXPERIMENTALParticipants randomized to receive adimanebart SC formulation B
Part 3 - Adimanebart SC (formulation C)EXPERIMENTALParticipants randomized to receive adimanebart SC formulation C

Interventions

NameTypeDescription
Adimanebart IVBIOLOGICALIntravenous infusion of Adimanebart
Placebo IVOTHERIntravenous infusion of Placebo
Adimanebart SC - formulation ABIOLOGICALSubcutaneous administration of Adminanebart SC
Adimanebart SC - formulation BBIOLOGICALSubcutaneous administration of Adminanebart SC
Adimanebart SC - formulation CBIOLOGICALSubcutaneous administration of Adminanebart SC
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Eligibility Criteria

Age Range12 Years to N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: DBTP: * At least 12 years of age. * Has a diagnosis of DOK7-, MUSK-, AGRN-, or LRP4-CMS with documented mutations. * Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) or other CMS medication must have been receiving the medication for at least 6 mont...

Countries:Canada
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Recent Changes (Last 90 Days)

LOWAug 4, 2026NCT07746089NEW_TRIAL: changed
LOWAug 4, 2026NCT07746089NEW_TRIAL: changed
LOWJun 29, 2026NCT07673601NEW_TRIAL: changed
LOWJun 29, 2026NCT07673601NEW_TRIAL: changed

Frequently asked questions about Adimanebart

What is Adimanebart used for?

Adimanebart is an investigational monoclonal antibody being studied for congenital myasthenic syndrome (CMS), specifically in adult and pediatric participants with DOK7-, MUSK-, AGRN-, or LRP4- CMS. It is also being studied in healthy volunteers to assess its bioavailability and how it moves through the body.

What does Adimanebart target?

Adimanebart is a monoclonal antibody, but its specific molecular target has not been disclosed in the available information. It is being investigated for congenital myasthenic syndrome, a group of inherited neuromuscular disorders.

Who makes Adimanebart?

Adimanebart is being developed by argenx SE, a biopharmaceutical company traded on the stock exchange under the ticker ARGX. The company is conducting clinical trials to evaluate the drug's safety and efficacy.

What phase is Adimanebart in?

Adimanebart is in Phase 3 clinical development for congenital myasthenic syndrome. It is also being studied in a Phase 1 trial in healthy volunteers. The drug is investigational and has not been approved by regulatory authorities.

What clinical trials is Adimanebart in?

Adimanebart is being studied in two clinical trials. NCT07673601 is a Phase 1 study in healthy volunteers in Canada, assessing absolute bioavailability. NCT07746089 is a Phase 3 study in patients with congenital myasthenic syndrome, evaluating efficacy and safety.

Is Adimanebart the same as Adimanebart IV?

Yes, Adimanebart is also known as Adimanebart IV. The name Adimanebart IV refers to the intravenous formulation of the drug, which is being studied in clinical trials for congenital myasthenic syndrome.