Positive preliminary results reported in phase 1b re-treatment trial of QLT091001

Clinically meaningful improvements in visual fields and visual acuity have been reported in preliminary results from a phase 1b clinical trial of repeated treatments of oral QLT091001 in subjects with Leber congenital amaurosis or retinitis pigmentosa, according to a press release from QLT.

Specifically, the international multicenter trial targeted Leber congenital amaurosis or retinitis pigmentosa due to inherited genetic mutations in retinal pigment epithelium (RPE65) or lecithin:retinol acyltransferase (LRAT), the release said. The extension study included patients who been treated with a single course of QLT091001 in a previous phase 1b study.

To date, 19 of 27 subjects (70%) had an increase in visual fields in the retinal area from baseline of 20% or more, while 70% of subjects had an increase in visual acuity from baseline of five letters or more. The improvements were seen in at least one eye at two consecutive visits within 6 months from the start of any QLT091001 treatment course, according to the release.

Dosing in the study is completed, and follow-up is continuing. Final clinical data, including duration of response and other evaluations, are expected to be released in the third quarter.