ARGX-119 / argenx - LARVOL DELTA

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

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 | Not yet recruiting | Sponsor: argenx

New P1 trial