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Pozelimab + Cemdisiran in PNH with Inadequate Control on C5 Inhibitors
2024-519709-37-00Phase 3Authorised
- Sponsor
- Regeneron Pharmaceuticals
- Start
- Oct 2025
- Modality
- Monoclonal Antibodies → Antibody-Based Therapeutics, Recombinant Proteins and Enzymes, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 8
01
Overview
Phase 3, open-label, single-arm trial of pozelimab and cemdisiran in paroxysmal nocturnal hemoglobinuria (PNH), sponsored by Regeneron Pharmaceuticals. Primary endpoint: Percent change in lactate dehydrogenase (LDH) from baseline to week 28 during treatment period. Planned start Oct 2025; status: authorised.
02
Drugs
pozelimabcemdisiran
03
Conditions
paroxysmal nocturnal hemoglobinuria (PNH)
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