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Sanofi’s Efdoralprin Alfa Shows Superior Biomarker Response to Plasma Therapy in Phase 2 AATD Trial

Next steps include data presentation at a medical meeting followed by talks with regulators.

Overview

  • Sanofi reported that efdoralprin alfa met all primary and key secondary endpoints in the ElevAATe Phase 2 study after eight months of dosing.
  • Efdoralprin alfa given every three or four weeks produced a statistically greater mean increase in functional AAT within the normal range versus weekly plasma-derived augmentation therapy at week 32.
  • The study showed higher average functional AAT concentrations and a greater percentage of days above the lower limit of normal, with a safety profile similar to plasma therapy.
  • Sanofi has not released granular numerical data and plans a full presentation at an upcoming medical meeting, with an additional safety follow-up underway in an open-label extension.
  • Analysts said the readout validates Sanofi’s $2.2 billion 2024 acquisition of Inhibrx and could lift consensus estimates, with the company citing a large, underdiagnosed AATD population.