Key Takeaways Sanofi's ElevAATe study showed that efdoralprin alfa met all primary and key secondary endpoints.Efdoralprin alfa achieved higher functional AAT levels versus weekly plasma-derived therapy.The therapy's Q3W and Q4W dosing regimen may enhance convenience for AATD patients.Sanofi (SNY) announced that the phase II ElevAATe study, which evaluated its pipeline candidate, efdoralprin alfa, for the treatment of alpha-1 antitrypsin deficiency (AATD) emphysema, met all primary and key secondary endpoin ...

Core Viewpoint - Sanofi's efdoralprin alfa has shown positive results in the ElevAATe phase 2 study for treating alpha-1 antitrypsin deficiency (AATD) emphysema, meeting all primary and key secondary endpoints, indicating its potential as a new therapeutic option for patients with this rare disease [1][3][5]. Group 1: Study Results - Efdoralprin alfa met all primary and key secondary endpoints when administered every three weeks (Q3W) or four weeks (Q4W) in adults with AATD emphysema [1]. - The treatment demonstrated a statistically significant greater mean increase in functional AAT levels compared to weekly plasma-derived therapy at week 32, with a p-value of less than 0.0001 [1]. - Key secondary endpoints included a superior mean increase in fAAT average concentration and a higher percentage of days above the lower limit of the normal range for both Q3W and Q4W dosing regimens [1][7]. Group 2: Safety and Tolerability - Efdoralprin alfa was well tolerated, exhibiting a similar adverse event profile to plasma-derived therapy [2]. - Additional safety follow-up will be conducted in the ElevAATe OLE phase 2 study [2]. Group 3: Expert Commentary - Christopher Corsico, Global Head of Development at Sanofi, emphasized the significance of achieving higher normal functional AAT levels with less frequent dosing, which could improve the treatment experience for AATD patients [3]. - Igor Barjaktarevic, MD, highlighted the potential of efdoralprin alfa to provide a more convenient treatment option that maintains normal AAT levels without reliance on blood donation programs [3]. Group 4: Regulatory Status and Future Plans - Efdoralprin alfa has received fast track and orphan drug designation from the FDA for AATD emphysema treatment [3][6]. - Sanofi plans to present the study data at an upcoming medical meeting and engage with global regulatory authorities regarding next steps [3]. Group 5: About AATD - AATD is a rare inherited disorder characterized by low or absent levels of AAT, leading to progressive lung and liver damage [4]. - Approximately 235,000 individuals worldwide are affected by AATD, with nearly 100,000 in the US, and about 90% of cases remain undiagnosed [4]. Group 6: About Efdoralprin Alfa - Efdoralprin alfa is a recombinant human AAT-Fc fusion protein being investigated as a restorative therapy for AATD emphysema, with dosing regimens of Q3W or Q4W [5]. - The treatment aims to normalize and maintain functional AAT levels, representing a significant improvement in convenience over weekly plasma-derived therapies [5].