QLT has positive outcomes treating LCA

QLT (NASDAQ:QLTI; TSX:QLT) has released “positive outcomes” from interim results of the first three subjects enrolled in a Phase 1b clinical proof-of-concept study of its QLT091001 treatment for Leber congenital amaurosis (LCA), an inherited progressive retinal degenerative disease.

LCA leads to retinal dysfunction and significant visual impairment beginning at birth.  Based on the positive results from the first two pediatric patients, a protocol exception was granted to also treat an adult patient.  QLT091001 is an oral synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of visual function.

QLT said that after seven days of treatment, the first three patients all experienced “clinically relevant improvements” in one or more visual function parameters and “meaningful improvements” in their visual performance related to tasks of daily living.

The onset of visual changes was “rapid and there was progressive improvement beyond the seven days of treatment, with some effects persisting for up to four months after treatment was completed,” the company added.

“These preliminary results are very exciting, are better than expected, and provide a measure of hope that a treatment might be developed for this devastating disease,” Dr. Robert Koenekoop, Director of the McGill Ocular Genetics Laboratory and Chief of Pediatric Ophthalmology at Montreal Children’s Hospital, said in a statement.