Sanofi: Efdoralprin Alfa Meets Primary Endpoint in Phase 2 DAAT Study
Sanofi announced on Monday the results of the phase 2 ElevAATe study evaluating efdoralprin alfa, an experimental treatment for patients with emphysema related to alpha-1 antitrypsin deficiency (AATD). These data were presented at the annual American Thoracic Society conference in Orlando.
Enhanced Efficacy with Reduced Administration Frequency
Efdoralprin alfa, administered every three weeks, demonstrated an increase in residual functional alpha-1 antitrypsin (fAAT) concentrations more than three times higher than that of standard plasma-derived treatments administered weekly. The study achieved its primary endpoint with statistical significance (p < 0.0001). Patients treated every three weeks maintained fAAT levels above the normal threshold for 100% of the days during the 32-week study, compared to 41% of days for patients receiving the standard treatment. All key secondary endpoints were also met (p < 0.0001).
Safety Profile and Regulatory Status
Efdoralprin alfa was well tolerated, with a safety profile comparable to standard treatments. No participants discontinued treatment due to drug-related adverse events. The incidence of severe exacerbations of chronic obstructive pulmonary disease was numerically lower in the efdoralprin alfa arm administered every three weeks (26.8%) than in the comparative arms. The drug candidate has received Fast Track designation and orphan drug designation in the United States, as well as orphan drug designation in the European Union. Sanofi is currently discussing the next steps of development with global regulatory authorities.
