French healthcare company Sanofi S.A. (Euronext Paris:SAN) (Nasdaq:SNY) on Wednesday announced positive topline results from its global ElevAATe phase 2 study evaluating efdoralprin alfa (SAR447537) in adults with alpha-1 antitrypsin deficiency (AATD) emphysema.

The investigational recombinant human alpha-1 antitrypsin-Fc fusion protein met all primary and key secondary endpoints across both three-week and four-week dosing regimens.

In a head-to-head comparison, efdoralprin alfa demonstrated statistically significant superiority over standard plasma-derived augmentation therapy, achieving normal functional AAT levels at steady state. The therapy also maintained higher average AAT concentrations and more days within the normal range than the comparator.

Efdoralprin alfa was well tolerated, with a safety profile consistent with plasma-derived treatments. Further safety evaluation is ongoing in the ElevAATe OLE phase 2 extension study.

The therapy has received Fast Track and Orphan Drug designations from the US Food and Drug Administration (FDA) and remains under clinical investigation. Sanofi intends to present full results at an upcoming medical meeting and consult regulatory authorities on next steps.

AATD is a rare, inherited disorder that causes progressive deterioration of the tissue of the lungs and liver.