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Also known as ARGX-119 IV
ARGX-119 · 2 trials · 3 indications
Adverse Events
Serious Adverse Events
The RHS (Revised Hammersmith Scale) is a validated 36-item scale developed to evaluate the spectrum of gross motor function. Maximum total score; 69 (optimal motor function)
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Before and after the 6MWT assessment, the participant's blood pressure, heart rate, and SPO2 will be recorded, and the participant's perception of fatigue and dyspnea will be measured.
| Arm | Type | Description |
|---|---|---|
| DBTP - ARGX-119 IV | EXPERIMENTAL | Participants receive ARGX-119 IV during the DBTP |
| DBTP - Placebo IV | PLACEBO_COMPARATOR | Participants receive placebo IV during the DBTP |
| ATEP - ARGX-119 IV | PLACEBO_COMPARATOR | Participants receive ARGX-119 IV during the ATEP. Participants from ARGX-119 IV arm in the DBTP will receive placebo once to maintain the DBTP blinding |
| Double-blinded treatment period - ARGX-119 IV | EXPERIMENTAL | Participants receive ARGX-119 during the double-blinded treatment period |
| Double-blinded treatment period - Placebo IV | PLACEBO_COMPARATOR | Participants receive placebo during the double-blinded treatment period |
| Active-treatment period - ARGX-119 IV | EXPERIMENTAL | Participants receive ARGX-119 during the active-treatment period |
| Name | Type | Description |
|---|---|---|
| ARGX-119 IV | BIOLOGICAL | Intravenous infusion of ARGX-119 |
| Placebo IV | OTHER | Intravenous infusion of placebo |
| ARGX-119 | BIOLOGICAL | Intravenous infusion of ARGX-119 |
| Placebo | OTHER | Intravenous infusion of placebo |
Inclusion Criteria: * Is aged ≥5 to \<18 years when completing the informed consent process, defined as providing informed assent according to local regulations and having a parent or guardian sign the ICF, and can comply with protocol * requirements. * Has documented historical genetic diagnosis o...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Biogen Inc. | BIIB | 13 | PHASE3 | Nusinersen, Salanersen |
| Novartis AG Sponsored ADR | NVS | 7 | PHASE3 | Onasemnogene abeparvovec |
| Biohaven Ltd. | BHVN | 1 | PHASE3 | Taldefgrobep Alfa |
| Scholar Rock Holding Corp. | SRRK | 2 | PHASE3 | Apitegromab |
| argenx SE Sponsored ADR | ARGX | 1 | PHASE2 | ARGX-119 |
| Hoffmann-La Roche | RHHBY | 2 | PHASE2 | Risdiplam, RO7204239 |
ARGX-119 is an investigational monoclonal antibody being developed by argenx SE for neuromuscular conditions. It is in clinical development for congenital myasthenic syndrome and spinal muscular atrophy, and it has not been approved by any regulatory authority. It is also referred to as ARGX-119 IV when given intravenously.
ARGX-119 targets MuSK, a muscle-specific kinase protein. MuSK is a receptor tyrosine kinase involved in the formation and maintenance of the neuromuscular junction, the connection between nerve and muscle. By binding MuSK, ARGX-119 is designed to act on this signaling pathway in conditions where neuromuscular transmission is impaired.
ARGX-119 is being developed by argenx SE, a biotechnology company that trades under the ticker ARGX. Argenx is running the clinical program for the antibody across congenital myasthenic syndrome and spinal muscular atrophy.
ARGX-119 is in Phase 1 and Phase 2 clinical development and is not approved for any indication. A Phase 1b study in adults with DOK7-congenital myasthenic syndromes has completed enrollment, and a Phase 2 pediatric study in spinal muscular atrophy is currently recruiting participants.
ARGX-119 is being studied in two trials. NCT06436742 is a Phase 1b study in adults with DOK7-congenital myasthenic syndromes that is active but not recruiting, with 15 participants. NCT07287982 is a recruiting Phase 2 study in children aged 5 to under 18 with spinal muscular atrophy, enrolling 60 participants across the United States, Australia, Canada, and the United Kingdom.
Yes, ARGX-119 IV refers to the intravenous formulation of ARGX-119, the monoclonal antibody developed by argenx SE. The two names refer to the same investigational drug candidate, which targets MuSK and is being studied in congenital myasthenic syndrome and spinal muscular atrophy.