argenx (ARGX) investor relations material

Strategic Vision and Innovation

• Aims to reach 50,000 patients, 10 labeled indications, and five new late-stage studies by 2030, leveraging a pipeline-in-a-product approach for neuromuscular and motor neuron diseases.

• Emphasizes a science-based, data-driven approach, leveraging antibody engineering, external academic collaborations, and novel disease biology insights.

• The Immunology Innovation Program (IIP) and collaboration with academic and patient advocacy groups underpin the innovation ecosystem and strategy.

ARGX-119/argenx 119 Development and Clinical Progress

• ARGX-119/argenx 119, a first-in-class, fully human MuSK agonist antibody, is advancing in CMS, ALS, and SMA, with a registrational study in CMS and phase 2/3 studies in ALS and SMA.

• Phase 1b in DOK7 CMS showed proof of biology, favorable safety, and significant improvements in mobility, endurance, and activity, with no serious or grade ≥3 adverse events.

• Digital sensors, actigraphy, and innovative endpoints such as MScan are integrated into trials to capture real-world patient function and enhance data richness.

• Treated patients reported increased home activity and improved mobility in qualitative interviews.

Scientific Rationale and Mechanism of Action

• MuSK activation is critical for neuromuscular junction formation and function; ARGX-119/argenx 119 targets the frizzled-like domain to promote receptor clustering and synaptic transmission.

• Preclinical models demonstrated that ARGX-119/argenx 119 rescues neuromuscular function and survival in DOK7 CMS mice, with effects reversible upon antibody clearance and restored with re-dosing.

• Engineered using patient-derived and synthetic sequences, with collaboration from leading academic centers.

• The approach is expected to benefit a broad range of neuromuscular diseases with defects upstream of MuSK, including certain CMS subtypes, ALS, and SMA.