adimanebart (ARGX-119) / argenx - LARVOL DELTA

ReALiSe: A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2 | N=60 | Terminated | Sponsor: argenx | Active, not recruiting ➔ Terminated; The study was terminated following review of interim data indicating that continuation was unlikely to demonstrate a clinically meaningful treatment effect. The decision was made to minimize unnecessary participant burden.

Trial termination • Amyotrophic Lateral Sclerosis • CNS Disorders

CoMetS: A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS) (clinicaltrials.gov) - P3 | N=105 | Not yet recruiting | Sponsor: argenx

New P3 trial • Pediatrics • AGRN • LRP4

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) (clinicaltrials.gov) - P1 | N=15 | Active, not recruiting | Sponsor: argenx | Recruiting ➔ Active, not recruiting

Enrollment closed

Phase 1b Study of Safety and Efficacy of Adimanebart (ARGX-119) in DOK7 Congenital Myasthenic Syndromes (ICNMD 2026) - P1 | "Results from this phase 1b study demonstrate proof-of-biology for adimanebart in participants with DOK7-CMS, which may correlate to real-world activity."

Clinical • P1 data • CNS Disorders • Genetic Disorders • Myasthenia Gravis

Adimanebart (ARGX-119): A First-in-Class, Humanized, Agonistic Monoclonal Antibody With Therapeutic Potential in Neuromuscular Junction Disorders (ICNMD 2026) - P1, P2 | "By stabilizing and preserving NMJ and motor neuron function, facilitating synaptic transmission and promoting motor endplate integrity, adimanebart has therapeutic potential to improve muscle function in patients with CMS, ALS, and SMA."

Amyotrophic Lateral Sclerosis • Cardiovascular • CNS Disorders • Genetic Disorders • Movement Disorders • Rare Diseases • LRP4 • SMA4 • SMN1

In Vivo Effects of a MuSK Agonist Antibody Against Mutation-Specific Congenital Myasthenic Syndromes (ICNMD 2026) - "Administration of 3B2 prevented mortality, restored body weight and muscle fiber size to wild-type levels, and improved muscle strength in an Agrn-CMS mouse model, but did not rescue the CMS phenotype in mouse models of ColQ and AChR deficiency. These data suggest differential effects of adimanebart, dependent upon pathophysiology of CMS subtypes and the expected adimanebart mechanism of action."

Preclinical • AGRN

A Study to Assess the Absolute Bioavailability and How Adimanebart SC Moves Through the Body in Healthy Adult Participants (clinicaltrials.gov) - P1 | N=52 | Recruiting | Sponsor: argenx

New P1 trial

A Phase 2a Study To Assess The Safety, Tolerability, Efficacy, Pharmacokinetics, And Immunogenicity Of Adimanebart In Adults With Amyotrophic Lateral Sclerosis: Study Results From ReALiSe (ENCALS 2026) - P2 | "MScan-MUN was an insightful measurement that correlated with clinical endpoints, including ALSFRS-R total score and its subdomains absolute values and change from baseline. Conclusion Although adimanebart was well tolerated with a favorable safety profile, results do not support further development of adimanebart in ALS due to the absence of treatment effect across secondary and exploratory efficacy endpoints."

Clinical • P2a data • PK/PD data • Amyotrophic Lateral Sclerosis • CNS Disorders • NEFL

Safety and Efficacy of ARGX-119 in DOK7 Congenital Myasthenic Syndromes: A Phase 1b Study (JSNE 2026) - No abstract available

Clinical • P1 data

First-in-Human Dose Selection and Safety, Tolerability, Pharmacokinetics, and Immunogenicity of the Muscle-Specific Kinase Agonist Adimanebart (ARGX-119). (PubMed, J Clin Pharmacol) - "There was no apparent impact of anti-drug antibodies on adimanebart pharmacokinetics or safety. This study supports investigation of adimanebart as an agonistic muscle-specific kinase monoclonal antibody treatment for neuromuscular junction disorders."

Clinical • First-in-human • Journal • P1 data • PK/PD data • CNS Disorders

Gene-specific response to muscle specific kinase agonist antibody in the treatment of congenital myasthenic syndromes. (PubMed, Brain Commun) - "Recently, a MuSK agonist antibody (ARGX-119) with high affinity for the Frizzled-like domain showed remarkable rescue of a Dok7-CMS mouse model...Our findings suggest that MuSK agonists may benefit patients with AGRN-CMS, which should be tested in clinical trials. Our study emphasizes that effective CMS treatment is gene-dependent and relies on an accurate genetic diagnosis."

Journal • CNS Disorders • Rare Diseases • AGRN

Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Participants with DOK7 Congenital Myasthenic Syndromes (AAN 2026) - P1 | "Ambulatory (no wheelchair use at baseline) participants receiving ARGX-119 demonstrated coherence in response across most endpoints measuring leg function; this coherence in response did not occur in participants in the placebo arm. Conclusions Results demonstrate proof-of-biology for ARGX-119 in participants with DOK7-CMS."

Clinical • P1 data • PK/PD data • CNS Disorders • Genetic Disorders • Myasthenia Gravis

ReALiSe: A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2 | N=60 | Active, not recruiting | Sponsor: argenx | Completed ➔ Active, not recruiting | Trial completion date: Jul 2027 ➔ Aug 2026 | Trial primary completion date: Jul 2027 ➔ Aug 2026

Enrollment closed • Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

ReALiSe: A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2 | N=60 | Completed | Sponsor: argenx | Recruiting ➔ Completed

Trial completion • Amyotrophic Lateral Sclerosis • CNS Disorders

argenx to Present New Data at 2026 AAN Annual Meeting that Continue to Transform Patient Outcomes in MG and CIDP and Build Upon Strength of Pipeline (argenx Press Release) - "Positive results from Phase 3 ADAPT OCULUS study show VYVGART’s potential as the first targeted treatment for patients living with ocular MG; Additional data from ADAPT SERON – the largest study of patients with gMG who do not have detectable AChR-Ab – demonstrate VYVGART’s efficacy and safety across subtypes; New biomarker analysis, real-world evidence and post-hoc insights highlight VYVGART’s expanding treatment approach in CIDP....Additional results from the ARGX-119 Phase 1b trial evaluating adimanebart in patients with DOK7 congenital myasthenic syndromes (CMS) will also be shared..."

Clinical data • Genetic Disorders • Immunology • Inflammation • Myasthenia Gravis

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) (clinicaltrials.gov) - P1 | N=16 | Recruiting | Sponsor: argenx | Trial completion date: Nov 2025 ➔ Jan 2028 | Trial primary completion date: Nov 2025 ➔ Aug 2027 | Active, not recruiting ➔ Recruiting

Enrollment open • Trial completion date • Trial primary completion date

Sparkle: A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Movement Disorders • Muscular Atrophy • Pediatrics • Rare Diseases

Sparkle: A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy (clinicaltrials.gov) - P2 | N=60 | Not yet recruiting | Sponsor: argenx

New P2 trial • Genetic Disorders • Movement Disorders • Muscular Atrophy • Pediatrics • Rare Diseases

ReALiSe: A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2 | N=60 | Active, not recruiting | Sponsor: argenx | Recruiting ➔ Active, not recruiting

Enrollment closed • Amyotrophic Lateral Sclerosis • CNS Disorders

ARGX-119 Development (argenx Press Release) - "CMS registrational study on track to start in 2026; Phase 2a proof-of-concept study ongoing in ALS; topline results expected in first half of 2026; SMA proof-of-concept study on track to start by end of 2025."

New trial • P2a data • Amyotrophic Lateral Sclerosis

ARGX-119 Development (GlobeNewswire) - "Registrational study to start in CMS in 2026 following positive Phase 1b proof-of-concept data; Phase 2a proof-of-concept study ongoing in ALS; topline results expected in first half of 2026; SMA proof-of-concept study on track to start by end of year; ARGX-119 R&D webinar to be hosted on September 16, 2025."

New trial • P1 data • P2a data • Amyotrophic Lateral Sclerosis • Genetic Disorders • Muscular Atrophy

argenx Advances Clinical Development of ARGX-119 in Congenital Myasthenic Syndromes (GlobeNewswire) - "argenx SE...announced its plan to advance the clinical development of ARGX-119, a first-in-class agonist antibody to muscle-specific kinase (MuSK), to a registrational study in patients with congenital myasthenic syndromes (CMS) following the analysis of topline data from the Phase 1b study...The decision to advance the development of ARGX-119 in CMS is supported by the results of the Phase 1b study. ARGX-119 demonstrated a favorable safety and tolerability profile, which was the primary endpoint. Efficacy was evaluated across multiple secondary and exploratory endpoints, including Six-Minute Walk Test (6MWT), Quantitative Myasthenia Gravis (QMG) score and Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Consistent improvements were observed in treated DOK7-CMS patients through the 12-week study across multiple efficacy scores."

New trial • P1 data • Genetic Disorders

First-in-Human Dose Selection and Pharmacokinetics, Safety, Tolerability, and Immunogenicity of ARGX-119, an Agonist Antibody for Human Muscle-Specific Kinase (AAN 2025) - P1, P2 | "ARGX-119 was well tolerated and has a favorable safety profile in healthy participants at the doses investigated in single- and multiple-dose cohorts in this FIH study. ARGX-119 is currently being evaluated in a phase 1b study in adult participants with DOK7-CMS (NCT06436742) and a phase 2a study in adult participants with ALS (NCT06441682)."

Clinical • P1 data • PK/PD data • Amyotrophic Lateral Sclerosis • CNS Disorders • Myasthenia Gravis

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants with DOK7-Congenital Myasthenic Syndromes (CMS) (clinicaltrials.gov) - P1 | N=16 | Active, not recruiting | Sponsor: argenx | Recruiting ➔ Active, not recruiting

Enrollment closed • Myasthenia Gravis

argenx Reports Full Year 2024 Financial Results and Provides Fourth Quarter Business Update (GlobeNewswire) - "ARGX-119 is being evaluated in congenital myasthenic syndromes (CMS), amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA). Phase 1b proof-of-concept study ongoing in CMS; topline results expected in second half of 2025. Phase 2a proof-of-concept study ongoing in ALS; topline results expected in first half of 2026. SMA proof-of-concept study on track to start in 2025."

New trial • P1 data • P2a data • Amyotrophic Lateral Sclerosis • CNS Disorders