Sanofi’s efdoralprin alfa (INBRX-101) achieves all endpoints in Phase 2 alpha-1 antitrypsin deficiency (AATD) trial
Sanofi recently acquired Inhibrx, Inc., primarily for the rights to efdoralprin alfa (INBRX-101), a recombinant protein therapy for AATD, in a deal valued at up to $2.2 billion246.
The Phase 2 trial compared efdoralprin alfa (given every 3–4 weeks) to the standard-of-care, plasma-derived therapy (Zemaira, given weekly) in patients with AATD emphysema135.
Efdoralprin alfa met all primary and key secondary endpoints, demonstrating statistically significant increases in functional AAT protein levels over standard therapy, with fewer infusions required135.
Patients on efdoralprin alfa spent more days above the lower limit of normal AAT levels and showed a similar safety profile to plasma therapy13.
Sanofi plans to present detailed data at a future medical meeting and will engage with global regulators to discuss next steps toward potential approval13.
The success positions efdoralprin alfa as a potential best-in-class treatment in a market currently dominated by plasma-derived therapies from companies like CSL, Grifols, and Takeda36.
Sources:
1. https://www.biopharmadive.com/news/sanofi-efdoralprin-alfa-aatd-study-results-inhibrx/803462/
2. https://www.sanofi.com/en/media-room/press-releases/2024/2024-05-30-13-01-38-2890833
3. https://www.fiercebiotech.com/biotech/sanofis-17b-rare-disease-bet-beats-csls-zemaira-phase-2-face
4. https://globalgenes.org/raredaily/sanofi-to-acquire-inhibrx-adding-potential-best-in-class-rare-disease-asset-for-aatd-to-pipeline/
5. https://www.sanofi.com/en/media-room/press-releases/2025/2025-10-22-05-00-00-3170787
6. https://www.biopharmadive.com/news/sanofi-inhibrx-acquire-spinout-alpha-1-antitrypsin/705292/