Tuesday, FibroGen Inc (NASDAQ: FGEN) announced topline data from the Phase 3 LELANTOS-2 trial of pamrevlumab for ambulatory patients with Duchenne muscular dystrophy (DMD) on background systemic corticosteroids.
DMD is an inherited progressive muscular weakness disorder primarily affecting young boys.
The study did not meet the primary endpoint of change in the North Star Ambulatory Assessment (NSAA) total score from baseline to week 52.
NSAA measures functional motor abilities in ambulant children with DMD. It is usually used to monitor the progression of the disease and treatment effects.
Secondary endpoints measured by change from baseline at week 52 in 4-stair climb velocity, 10-meter walk/run test, time to stand, time to loss of ambulation, and proportion of patients with greater than 10 seconds in the 10-meter walk/run test ...