Cemdisiran

Q3 2024 revenues up 11% to $3.72 billion; Dupixent global net sales by Sanofi up 23% to $3.82 billion; U.S. net sales for EYLEA HD and EYLEA up 3% to $1.54 billion; Libtayo global net sales up 24% to $289 million; GAAP diluted EPS up 30% to $11.54; FDA approves Dupixent for COPD and eosinophilic phenotype; positive results for Dupixent in CSU and BP trials.

The NIMBLE phase 3 trial assesses pozelimab and cemdisiran combination therapy in MG patients, with 235 participants. The study includes a 24-week double-blind period, 28-week extension, 68-week open-label, and 52-week off-treatment follow-up. Primary endpoint is change in MG-ADL score. Secondary outcomes include QMG score, MG Composite score, and safety measures. Patients must have MG-ADL scores ≥6, excluding those with MuSK antibodies or thymectomy history.

Phase 3 trial data showed the pozelimab and cemdisiran (poze-cemdi) combination achieved greater control of intravascular hemolysis in PNH patients compared to ravulizumab, with 96% achieving adequate LDH control versus 80% with ravulizumab. Poze-cemdi also demonstrated complete, uninterrupted inhibition of terminal complement, offering potential for less frequent, subcutaneous administration.