Sanofi's Rare Lung Disease Drug Outperforms Standard Therapy in Head-to-Head Trial
London: French drugmaker Sanofi said on Monday a trial showed its rare disease therapy was better than standard care in raising levels of a key protein in patients with a genetic form of lung disease.
The data in the head-to-head study offers a potential boost to Sanofi's $2.2 billion bet on efdoralprin alfa, the experimental therapy it acquired through its 2024 Inhibrx deal in the wake of a run of pipeline setbacks last year.
Sanofi estimates about 235,000 people have the condition and sees an opportunity in switching patients from standard therapy and also in finding those who have not been diagnosed. Sanofi first reported in October that the study had met its main goals.
Investors are looking for evidence that Sanofi's pipeline can support growth beyond Dupixent, its top-selling drug. The drugmaker has hired CEO Belen Garijo to improve R&D productivity and sharpen execution.
Sanofi said the new data, presented at the American Thoracic Society meeting in Orlando, showed that in the 97-patient trial, efdoralprin alfa administered every three weeks raised trough levels for AAT protein by 24.1 micromolar at week 32, versus 7.6 micromolar for weekly plasma-derived standard therapy.
Patients on Sanofi's therapy maintained normal levels of AAT, or alpha-1 antitrypsin, throughout the study, compared with less than half the time for those on standard therapy. Without enough AAT, lungs and liver can both become damaged.
Unlike plasma-derived treatments, efdoralprin alfa is produced using recombinant technology, potentially easing dependence on donated plasma, said Alaa Ahmed, Sanofi's head of medical affairs for rare diseases.
Ahmed said the drug's less frequent dosing could also appeal to patients for whom weekly treatment is a burden.
