Key Takeaways
Sanofi’s investigational drug efdoralprin alfa showed superiority over standard therapy in a phase 2 trial for a rare lung disease, achieving mean functional alpha-1 antitrypsin (fAAT) trough levels more than three times higher than the current standard of care. The data was presented at the 2026 American Thoracic Society International Conference.
"The ElevAATE data suggest efdoralprin alfa, through its mechanism of action, may be able to restore normal AAT levels and keep patients in that range for longer than the standard-of-care therapy," Igor Barjaktarevic, the principal investigator on the study and an associate professor at UCLA's David Geffen School of Medicine, said.
The ElevAATe study showed that efdoralprin alfa, dosed every three weeks, met its primary endpoint with a mean increase in fAAT trough levels of 24.1 μM. This was significantly higher than the 7.6 μM increase seen in patients receiving the standard weekly plasma-derived therapy (p<0.0001). A key secondary endpoint showed patients on the new drug maintained fAAT levels in the normal range for 100% of days, compared to just 40.8% for the standard treatment.
The results could mark a significant advance for patients with alpha-1 antitrypsin deficiency (AATD), a rare genetic condition that can cause severe lung disease. Efdoralprin alfa could be the first new treatment approach in nearly 40 years, offering a more effective therapy with the convenience of less frequent dosing. Sanofi is now engaging with global regulatory authorities on the next steps for the drug.
Efdoralprin alfa was well tolerated and showed a safety profile comparable to the current plasma-derived therapy. Notably, the incidence of moderate-to-severe COPD exacerbations, a special interest adverse event, was numerically lower in the every-three-week efdoralprin alfa arm (26.8%) compared to the standard care arm (44.4%). The most common adverse events included COPD exacerbations, headache, and COVID-19 infection.
AATD is an underdiagnosed inherited disorder characterized by low levels of the AAT protein, which protects the lungs from inflammation and damage. This deficiency leads to progressive lung deterioration, often resulting in emphysema. An estimated 90% of the approximately 235,000 people worldwide living with AATD are believed to be undiagnosed.
The positive data significantly de-risks the drug's development path and signals a potential major shift in the AATD treatment landscape. Investors will now watch for updates on Sanofi's engagement with global regulatory authorities for the path forward.
This article is for informational purposes only and does not constitute investment advice.
