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Also known as Pozelimab
Pozelimab+ Cemdisiran · 1 trial · 1 indication
The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability
| Arm | Type | Description |
|---|---|---|
| Group 1 | EXPERIMENTAL | Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP |
| Group 2 | EXPERIMENTAL | Combination regimen throughout the study. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP. |
| Group 3 | EXPERIMENTAL | Cemdisiran throughout the study |
| Group 4 | EXPERIMENTAL | Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP. |
| Name | Type | Description |
|---|---|---|
| Pozelimab + Cemdisiran | DRUG | Subcutaneous administration as described in the protocol |
| Cemdisiran | DRUG | SC administration as described in the protocol |
| Placebo | OTHER | SC administration as described in the protocol |
| Pozelimab | DRUG | SC administration as described in the protocol |
Key Inclusion Criteria: 1. Male or female participants ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older) 2. Participant with documented diagnosis of Myasthenia Gravis (MG) based on medical history and supported by previous evaluations as desc...
Pozelimab is an investigational antibody being studied for CD55-deficient protein-losing enteropathy (CHAPLE disease), age-related macular degeneration, geographic atrophy, sporadic inclusion body myositis, and paroxysmal nocturnal hemoglobinuria. It is also being evaluated in combination with cemdisiran for generalized myasthenia gravis and geographic atrophy. Pozelimab is not approved and remains in clinical development.
Pozelimab is a monoclonal antibody that targets complement component C5, part of the immune system's complement cascade. By binding to C5, it is designed to block the formation of the membrane attack complex and reduce complement-mediated tissue damage. This mechanism is relevant across the diseases being studied, including CHAPLE disease and geographic atrophy.
Pozelimab is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker REGN. Regeneron is conducting clinical trials of Pozelimab both as a monotherapy and in combination with cemdisiran across multiple indications.
Pozelimab is in Phase 3 clinical development. It has completed a Phase 2 study in CD55-deficient protein-losing enteropathy and a Phase 1 study in healthy volunteers. Two Phase 3 trials are ongoing: one in generalized myasthenia gravis and one in geographic atrophy.
Pozelimab has four registered clinical trials. NCT04209634 is a completed Phase 2 study in CHAPLE disease. NCT04940364 is a completed Phase 1 study in healthy Japanese volunteers. NCT05070858 is an active Phase 3 trial in generalized myasthenia gravis. NCT06541704 is a recruiting Phase 3 trial in geographic atrophy.
Pozelimab is a single agent, while Pozelimab/Cemdisiran is a combination therapy that includes both Pozelimab and cemdisiran. The combination is being studied in clinical trials for generalized myasthenia gravis and geographic atrophy. Pozelimab alone has been studied in CHAPLE disease and healthy volunteers.