Interim results from QLT's Phase 1b study of QLT091001 for Leber congenital amaurosis

QLT Inc. (Nasdaq:QLTI) (TSX:QLT) ("QLT" or the "Company") today announced interim results from the first 3 subjects enrolled in a Phase 1b clinical proof-of-concept study of QLT091001 in the treatment of Leber congenital amaurosis (LCA), an inherited progressive retinal degenerative disease that leads to retinal dysfunction and significant visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of visual function.

The Phase 1b trial is a short-term, open-label, single-center study to evaluate the safety profile and effects on retinal function in 8 pediatric subjects (aged 5 to 14 years) diagnosed with LCA due to inherited deficiency of retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT). Based on the positive results from the first 2 pediatric patients, a protocol exception was granted to also treat an adult patient. Subjects receive daily oral doses of QLT091001 for 7 days at the Montreal Children's Hospital at the McGill University Health Centre, Montreal, Canada under the supervision of the trial's principal investigator, Robert K. Koenekoop, M.D., Ph.D. Patients were monitored to ensure overall safety. Efficacy assessments included several visual function parameters including best-corrected visual acuity and visual field testing.