Data from the global ElevAATe phase 2 study (clinical study identifier: NCT05856331) demonstrated superiority of investigational efdoralprin alfa over standard-of-care therapy in achieving and maintaining normalized functional alpha-1 antitrypsin (fAAT) levels in adult patients with alpha-1 antitrypsin deficiency (AATD)-related emphysema. These results are being presented today at the 2026 American Thoracic Society (ATS) International Conference in Orlando, FL, US.

Efdoralprin alfa, dosed every three weeks (Q3W), achieved mean increases in fAAT trough levels more than three times greater than plasma-derived protein (pdAAT) dosed weekly (Q1W), meeting the primary endpoint (p<0.0001). All key secondary endpoints in the study were also met (p<0.0001), highlighting the potential for efdoralprin alfa to be the first therapy to sustain normal fAAT levels for patients and do so with less frequent dosing. In patients dosed Q3W, fAAT levels remained above the normal threshold (23.8 μM) for 100% of days during the 32-week study compared to 41% of days in patients on a standard-of-care augmentation therapy.