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 | Completed | Sponsor: argenx | Recruiting ➔ Completed

Trial completion • Amyotrophic Lateral Sclerosis • CNS Disorders

Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Participants with DOK7 Congenital Myasthenic Syndromes (AAN 2026) - P1 | "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

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

Patient-specific therapeutic benefit of MuSK agonist antibody ARGX-119 in MuSK myasthenia gravis passive transfer models. (PubMed, iScience) - "Patient-specific efficacy could not be explained by titer or competition for ARGX-119 binding, but rather correlated with the presence of MuSK activating antibodies in some patients. This first proof of concept of a MuSK agonist in a clinically relevant MuSK MG model forms a starting point for therapeutic studies toward ARGX-119 efficacy in neuromuscular diseases."

Journal • CNS Disorders • Myasthenia Gravis

Safety, tolerability, efficacy, pharmacokinetics, and immunogenicity of ARGX-119 in patients with amyotrophic lateral sclerosis: a Phase 2a study in progress (ALS-MND 2024) - P2 | "The primary endpoint is safety. Key secondary endpoints are rate of change of MScan-derived motor unit number from baseline to week 24, assessment of pharmacokinetic parameters of ARGX-119, and development of antidrug antibodies against ARGX-119. Multiple exploratory endpoints are included to assess additional neurophysiology endpoints, clinical outcome measures, and mobility at home."

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

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

Enrollment open • Amyotrophic Lateral Sclerosis • CNS Disorders

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 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting | Initiation date: Jun 2024 ➔ Sep 2024

Enrollment open • Trial initiation date • Myasthenia Gravis

ARGX-119 is an agonist antibody for human MuSK that reverses disease relapse in a mouse model of congenital myasthenic syndrome. (PubMed, Sci Transl Med) - "Pharmacokinetic studies in nonhuman primates, rats, and mice revealed a nonlinear PK behavior of ARGX-119, indicative of target-mediated drug disposition and in vivo target engagement. On the basis of this proof-of-concept study, ARGX-119 has the potential to alleviate neuromuscular diseases hallmarked by impaired neuromuscular synaptic function, warranting further clinical development."

Journal • Preclinical • CNS Disorders • Myasthenia Gravis

A Clinical Trial to Investigate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Single and Multiple Ascending Doses of ARGX-119 in Healthy Participants (clinicaltrials.gov) - P1 | N=112 | Completed | Sponsor: argenx | Recruiting ➔ Completed

Trial completion

argenx Reports Half Year 2024 Financial Results and Provides Second Quarter Business Update (GlobeNewswire) - "Generated global net product sales (inclusive of both VYVGART and VYVGART SC) of $478 million in second quarter of 2024...Phase 3 study of empasiprubart for MMN to initiate in fourth quarter of 2024...Phase 1b/2a studies of ARGX-119 to assess early signal detection in patients with CMS and ALS to start by end of 2024."

New P1/2 trial • New P3 trial • Sales • Amyotrophic Lateral Sclerosis • CNS Disorders • Immunology • Myasthenia Gravis

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

New P2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

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 | Not yet recruiting | Sponsor: argenx

New P1 trial • Myasthenia Gravis

A Clinical Trial to Investigate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Single and Multiple Ascending Doses of ARGX-119 in Healthy Participants (clinicaltrials.gov) - P1 | N=116 | Recruiting | Sponsor: argenx | Trial completion date: Apr 2024 ➔ Aug 2024

Trial completion date

Development of ARGX-119, an Agonistic Antibody Targeting MuSK (PEGS 2023) - "Congenital myasthenia (CM) is a devastating neuromuscular disease and mutations in DOK7 are a major cause of CM. We developed agonist antibodies against MUSK and show that these antibodies restored neuromuscular synapse formation and prevented neonatal lethality and late-onset disease in mouse model for DOK7 CM."

CNS Disorders

A Clinical Trial to Investigate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Single and Multiple Ascending Doses of ARGX-119 in Healthy Participants (clinicaltrials.gov) - P1 | N=100 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting | Trial completion date: Jun 2023 ➔ Apr 2024 | Trial primary completion date: Jun 2023 ➔ Apr 2024

Enrollment open • Trial completion date • Trial primary completion date