Sanofi SA (NASDAQ:SNY) on Wednesday revealed topline data from the ElevAATe phase 2 study of efdoralprin alfa (SAR447537, formerly known as INBRX-101), for alpha-1 antitrypsin deficiency (AATD) emphysema.
AATD causes emphysema when the body doesn’t produce enough of the protective alpha-1 antitrypsin (AAT) protein, leading to a breakdown of the lung’s air sacs (alveoli).
This genetic condition leads to the accumulation of enzymes, such as neutrophil elastase, which damage the lungs, resulting in symptoms like shortness of breath and a chronic cough.
The data showed that efdoralprin alfa met all primary and key secondary endpoints when dosed every three weeks (Q3W) or four weeks (Q4W).
Efdoralprin alfa is an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein.
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It demonstrated a statistically greater mean increase in functional AAT (fAAT) levels within normal range compared to those receiving weekly plasma-derived augmentation therapy at week 32.
The study also met key secondary endpoints, demonstrating 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.
The recombinant efdoralprin alfa was well-tolerated, with a similar adverse event profile to that of plasma-derived therapy.
Sanofi will conduct an additional safety follow-up in the ElevAATe OLE phase 2 study.
Sanofi plans to present the data at a forthcoming medical meeting and engage with global regulatory authorities on the appropriate next steps.
Price Action: SNY stock is up 1.35% at $50.33 at the last check on Wednesday.
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