QLT to expand phase 1b study of compound for Leber's congenital amaurosis to include patients with retinitis pigmentosa

VANCOUVER, British Columbia — QLT plans to expand a phase 1b proof-of-concept study evaluating an oral medication for Leber's congenital amaurosis to include patients with retinitis pigmentosa, the company announced.

The compound, QLT091001, is an orally administered synthetic retinoid replacement therapy that has shown potential for treating both Leber's congenital amaurosis and retinitis pigmentosa, both of which result from genetic mutations of retinal pigment epithelium protein 65 or lecithin retinol acetyltransferase, according to a press release from the company.

The open-label, single-center phase 1b trial will enroll up to 24 patients diagnosed with Leber's congenital amaurosis or retinitis pigmentosa. All patients will receive two separate daily doses of QLT091001 for 7 days and will be monitored for safety, efficacy and tolerability.

"We are very excited to be expanding the potential uses for QLT091001 into another rare indication, retinitis pigmentosa," Bob Butchofsky, president and CEO of QLT, said in the release. "We continue to enroll [Leber's congenital amaurosis] patients in the trial, with a total of 7 patients currently treated or under treatment. We expect to treat the first [retinitis pigmentosa] patient in the coming weeks."

This follows QLT's April announcement of positive 7-day results among the first three Leber's congenital amaurosis patients treated with the compound. All three patients experienced clinically significant improvements in one or more visual function parameters, with positive vision effects persisting up to 4 months after treatment in some cases.