PARIS (dpa-AFX) - Sanofi SA (SNY) reported positive data from the Phase 2 ElevAATe study of efdoralprin alfa in adult patients with alpha-1 antitrypsin deficiency-related emphysema.
Alpha-1 Antitrypsin Deficiency (AATD) is a rare genetic condition characterized by low levels or absence of alpha-1 antitrypsin, a protein that helps protect the lungs from inflammation-related damage.
The study was designed to evaluate efdoralprin alfa against a standard-of-care augmentation therapy in patients with AATD-related emphysema. Patients were randomized in a 2:2:1 ratio to receive efdoralprin alfa every three or four weeks, or plasma-derived augmentation therapy once weekly.
Efdoralprin alfa administered every three weeks achieved mean increases in functional alpha-1 antitrypsin trough levels more than three times greater than plasma-derived augmentation therapy given weekly, meeting the study's primary endpoint.
Further, patients receiving the every-three-week regimen maintained functional alpha-1 antitrypsin levels above the normal threshold of 23.8 ?M for 100% of days during the 32-week study, compared with 41% of days for patients receiving standard-of-care therapy.
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