Sanofi has announced encouraging results from a Phase 2 clinical trial of efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the liver and lungs. As reported by BioPharmaDive, the drug, acquired through Sanofi’s $2.2 billion purchase of Inhibrx last year, met all primary and key secondary endpoints in the study.

Participants receiving efdoralprin alfa every three to four weeks showed greater increases in protective AAT protein levels compared to those on standard weekly plasma-derived treatments. The therapy also maintained functional protein levels above the lower limit of normal for longer durations, with a safety profile comparable to existing options.

AATD, which can lead to conditions like emphysema and cirrhosis, remains underdiagnosed and underserved, with an estimated 235,000 global cases, 90% of which are unidentified. Sanofi’s recombinant protein approach offers a potential alternative to plasma-based therapies, aiming to reduce treatment frequency and reliance on blood donations.

The company plans to present full trial data at an upcoming medical conference and engage with regulators to determine next steps. Analysts suggest the results could significantly boost expectations for the drug’s commercial potential.