Sanofi has reported positive results from its global phase-II ElevAATe study (NCT05856331), demonstrating that Efdoralprin Alfa (SAR447537, formerly INBRX-101) met all primary and key secondary endpoints in adults with Alpha-1 antitrypsin deficiency (AATD) emphysema. The investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, administered every three or four weeks, achieved a statistically significant increase in functional AAT levels within the normal range compared to weekly plasma-derived augmentation therapy at week 32 [p<0.0001]. The study also showed superior mean increases in average fAAT concentrations and a higher percentage of days above the lower limit of the normal range across both dosing regimens.

The recombinant Efdoralprin Alfa was well tolerated with a similar Adverse Event (AE) profile to plasma-derived therapy. Additional safety follow-up will be assessed in the ElevAATe OLE phase-II study (clinical study identifier: NCT05897424).

  “These data demonstrate that Efdoralprin Alfa achieved consistently higher normal functional AAT levels, with less frequent dosing, compared to a current standard of care. This is particularly meaningful as maintaining protective protein levels is the cornerstone of pulmonary management of AATD and currently available treatments require weekly therapeutic infusions. The ElevAATe results represent the potential for Efdoralprin Alfa to be a restorative recombinant therapeutic option for the AATD community, reinforcing our commitment to develop treatments for both rare and respiratory conditions with great unmet medical need,” said Christopher Corsico, Global Head—Development, Sanofi.

Efdoralprin Alfa was previously granted fast track and Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of AATD Emphysema. It is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. Sanofi plans to present the data at a forthcoming medical meeting and engage with global regulatory authorities on the appropriate next steps.

 “AATD is a debilitating condition that can be challenging to treat. Achieving and maintaining normal AAT levels with less frequent dosing and with complete independence from blood donation programmes would be a welcome change to the current treatment experience for people living with AATD. With the current standard of care, patients reach but do not maintain normal protein levels between the infusions, leaving a remaining unmet need. I’m encouraged by the ElevAATe trial results and what Efdoralprin Alfa could mean for the AATD community,” said Igor Barjaktarevic, MD, PhD, Associate Professor, David Geffen School of Medicine at UCLA and primary investigator on the ElevAATe phase-II study.