Sanofi reported positive Phase 2 ElevAATe results showing investigational efdoralprin alfa significantly improved and sustained functional alpha-1 antitrypsin levels versus standard plasma-derived therapy in adults with AATD-related emphysema.
Written By: Sana Khan, BPharm
Reviewed By: Pharmacally Editorial Team
Sanofi announced positive Phase 2 results from the global ElevAATe study (NCT05856331), presented at the 2026 American Thoracic Society International Conference in Orlando. The investigational recombinant fusion protein efdoralprin alfa demonstrated superior restoration and sustained maintenance of functional alpha-1 antitrypsin (fAAT) levels compared with standard plasma-derived augmentation therapy in adults with AATD-related emphysema. The detailed ATS data build on positive topline ElevAATe results first reported by Sanofi in October 2025.
In the double-blind, randomized trial of 97 patients, efdoralprin alfa administered every three weeks (Q3W) achieved a least square mean increase of 24.1 μM in average serum fAAT trough concentrations at week 32, compared with 7.6 μM for weekly plasma-derived therapy and 16.8 μM for every-four-week dosing.
Patients receiving Q3W dosing maintained fAAT levels above the normal threshold for 100% of days during the study, versus 40.8% with standard therapy. All primary and secondary endpoints were met with statistical significance (p<0.0001).
Efdoralprin alfa was generally well tolerated, with no treatment-emergent adverse events leading to discontinuation. The most common adverse events were COPD exacerbations, headache, and COVID-19 infection. Rates of grade ≥2 COPD exacerbations were numerically lower in the Q3W arm compared with the Q4W and plasma-derived therapy groups.
Igor Barjaktarevic, MD, PhD, principal investigator and Associate Professor at UCLA, said the findings suggest efdoralprin alfa may restore and sustain normal AAT levels longer than current therapies, potentially addressing an unmet need in AATD-related lung disease.
Sanofi said efdoralprin alfa has received Fast Track and Orphan Drug designations in the US and Orphan designation in the EU, and discussions with global health authorities are underway regarding next regulatory steps.
Reference
About the Writer
Sana Jamil Khan (LinkedIn) is a B. Pharm graduate with a strong interest in medical writing and scientific communication. Her work focuses on interpreting clinical research, exploring developments in pharmaceutical science, and presenting complex medical information in a clear and accessible manner. She is particularly interested in topics related to human clinical studies, drug safety observations, and emerging therapeutic research.