Oral synthetic retinoid replacement compound improves vision in LCA patients

A collaborative research group, led by the McGill University Health Centre, Montreal, Canada, with funding from QLT Inc., (NASDAQ: QLTI; TSX: QLT), have published the results of a Phase 1b clinical study in 14 LCA (Leber’s congenital amaurosis). The patients received an oral dose of a novel retinoid synthetic compound, “QLT091001”, designed to replace the normal functioning of 11-cis retinal. The QLT091001 compound is understood to convert in the body to 9-cis retinal to combine with opsin forming iso-rhodopsin and initiating the phototransduction cascade upon activation by light. In the reported study results, treatment for 7 days with oral QLT091001 showed a restoration of visual function in 11 of 14 LCA patients, measured by improvement in Goldmann Visual Fields (GVF) or visual acuity. In addition, self-reported or parent-reported improvements in daily living activities appeared to support the clinical findings.