Bilateral visual acuity gains reported in phase 3 trial of LHON gene therapy treatment
SAN FRANCISCO — A gene therapy treatment candidate for Leber hereditary optic neuropathy bilaterally improved best corrected visual acuity during a phase 3 trial, according to a speaker here.
“We saw bilateral improvement in both the treated and the sham eyes,” Patrick Yu-Wai-Man, FRCOphth, MBBS, PhD, said at the American Academy of Ophthalmology annual meeting.
Yu-Wai-Man presented the 96-week data from the phase 3 RESCUE trial, which included 39 patients with Leber hereditary optic neuropathy (LHON) treated with either GS010 (rAAV2/2-ND4) from GenSight Biologics or sham. The trial included patients who experienced vision loss due to LHON within 6 months. At week 96, data were available for 34 eyes that received GS010 and 34 that received sham.
Eyes treated with GS010 experienced continuous bilateral best corrected visual acuity from week 48 to week 96. From nadir, defined as the worst BCVA from baseline to week 96, GS010-treated eyes experienced a mean 26.3 ETDRS letter equivalent improvement compared with a mean 22.8 ETDRS letter equivalent for the sham-treated eyes, Yu-Wai-Man said. – by Robert Linnehan
Yu-Wai-Man P, et al. Efficacy of rAAV2/2-ND4 gene therapy for Leber hereditary optic neuropathy: 96-week data from the phase 3 RESCUE trial. Presented at: American Academy of Ophthalmology annual meeting; October 11-15, 2019; San Francisco.
Disclosure: Yu-Wai-Man reports he is a consultant for and receives research support from GenSight Biologics and receives research support from Santhera Pharmaceuticals and Stealth BioTherapeutics.