QLT's Drug for RP and LCA Improves Vision in 11 Patients

July 25, 2014

A drug developed by the company QLT for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) caused by mutations in the genes RPE65 or LRAT restored visual acuity or visual field in 11 of 14 patients in a Phase 1B clinical trial. Vision improvements for a seven-day course of the drug were sustained for two years for some study participants. No serious adverse events occurred. Results of the trial were published in The Lancet.

After one month of treatment, a 10-year-old girl in the study stopped using her navigational cane and no longer needed her video magnifier to read the blackboard at school. “I am able to walk in the dark and down the stairs by myself and do my own makeup for the first time,” she said.

A 38-year-old female participant who had only light perception upon entering the study reported significant vision improvements nine days after receiving the therapy. “I am able to see and recognize faces for the first time in at least 10 years,” she said. “I can see my face in the mirror, my arm, my cat on the kitchen floor and cars driving by for the first time.”

“These results are encouraging and impressive,” says Patricia Zilliox, Ph.D., chief drug development officer at the Foundation Fighting Blindness Clinical Research Institute. “We are very pleased to see meaningful vision improvements for people with these blinding retinal conditions.”

Known as QLT091001, the drug replaces a retinoid, a form of vitamin A, which is necessary for vision, but is missing in people with RP and LCA caused by RPE65 or LRAT mutations.

“Vitamin A is an essential ingredient in the visual cycle, the biochemical process that makes vision possible,” says Dr. Stephen Rose, the Foundation’s chief research officer. “But for it to work, it needs to get processed and recycled into different forms called retinoids, including the retinoid 11-cis-retinal. QLT’s drug replaces the 11-cis-retinal that’s missing in certain forms of RP and LCA.”

Scientists funded by the Foundation have conducted extensive research to understand the role of LRAT and RPE65 mutations in LCA and RP, giving targets for related treatments to companies such as QLT.

The U.S. Food and Drug Administration has given QLT’s drug a Fast Track Designation. Provided to emerging therapies that address a serious, unmet need, Fast Track Designations can facilitate development and expedite regulatory review, potentially enabling treatments to reach patients sooner.

QLT’s therapy is nearing Phase III clinical development.