Clinical TrialMay 27, 2026, 10:37 AM
Sanofi efdoralprin alfa Phase 2 superior in AATD; fAAT levels normalized
AI Summary
The ElevAATe Phase 2 study demonstrated the superiority of Sanofi's investigational efdoralprin alfa over standard-of-care therapy in achieving and maintaining normalized functional alpha-1 antitrypsin (fAAT) levels in adult patients with AATD-related emphysema. Efdoralprin alfa, dosed every three weeks, achieved mean increases in fAAT trough levels more than three times greater than weekly plasma-derived protein, meeting the primary endpoint. All key secondary endpoints were also met, showing sustained normal fAAT levels for 100% of days during the study, compared to 41% for standard-of-care. The drug was well tolerated with a comparable safety profile.
Key Highlights
- Efdoralprin alfa (Q3W) showed superior fAAT trough level increases (24.1µM) vs. standard-of-care (7.6µM) (p<0.0001).
- Mean change in serum fAAT average concentrations was 32.9µM for efdoralprin alfa (Q3W) vs. 17.9µM for standard-of-care.
- Efdoralprin alfa (Q3W) maintained fAAT levels above normal threshold for 100% of days vs. 40.8% for standard-of-care.
- All key secondary endpoints were met with statistical significance (p<0.0001).
- Efdoralprin alfa was well tolerated with a safety profile comparable to plasma-derived augmentation therapy.
- Incidence of grade "equal or greater than" 2 COPD exacerbations was numerically lower for efdoralprin alfa Q3W (26.8%) vs. pdAAT (44.4%).
- Efdoralprin alfa has US Fast Track and Orphan Drug designations, and EU Orphan designation.
Price Impact
More from SNY