GenSight's GS010 misses in second Phase III for Leber hereditary optic neuropathy

GenSight Biologics S.A. (Euronext:SIGHT) reported that GS010 (rAAV2/2-ND4) missed the primary endpoint of improving visual acuity in the Phase III RESCUE trial to treat Leber hereditary optic neuropathy (LHON).

On the 39-patient trial's primary endpoint, treated eyes achieved an improvement of 13 letters and untreated eyes achieved an improvement of 11 letters in best corrected visual acuity (BCVA) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) eye chart from baseline to week 48. Patients received a single intravitreal injection of GS010 in one eye and sham injection in the other eye...