QLT Publishes Data From Phase 1b Trial Of QLT091001 In Subjects With Leber Congenital Amaurosis In The Lancet Journal

QLT Inc.

("QLT" or the "Company") today announced the publication of data from its Phase 1b proof-of-concept trial of QLT091001 in subjects with Leber Congenital Amaurosis (LCA) due to inherited genetic mutations in retinal pigment epithelium (RPE65) or lecithin:retinol acyltransferase (LRAT) in The Lancet. The peer-reviewed journal article is available first in the online edition of The Lancet at www.thelancet.com. In the study, treatment for 7 days with oral QLT091001 demonstrated a restoration of clinically meaningful visual function in 11 of the 14 LCA patients, as measured by improvement in Goldmann Visual Fields (GVF) or visual acuity. Self-reported or parent-reported improvements in activities of daily living supported these findings. "Currently, children and adults with LCA due to these genetic mutations progressively lose vision, eventually becoming totally blind as there are no proven therapies that either slow or reverse their condition," stated David A. Saperstein, M.D., a retina physician who serves as Chief Medical Advisor to QLT and is a co-author of the Lancet paper. "The results of this trial are quite promising. If borne out in further clinical trials, QLT091001 would have the potential to become the first oral medication to improve vision and quality of life in patients with these forms of LCA." The trial enrolled 14 patients, ages 6–38, with Leber Congenital Amaurosis due to mutations in RPE65 or LRAT. Patients received 7 days of oral QLT091001 (12 subjects received 40 mg/m2 per day and 2 subjects received 10 mg/m2 per day). Patients were assessed at baseline and days 7, 9, 14, and 30, and every 2 months thereafter, for up to 3 years, for safety outcomes and visual function endpoints, which included GVF, visual acuity, and a functional MRI assessment. In the study, 10 of 14 patients had an improvement of GVF, with a mean increase in retinal area of 28–683%. Six patients had an improvement in visual acuity, with a mean increase of 2–30 letters. After two years, three patients had a sustained GVF response and four had a sustained visual acuity response. Four patients had functional MRI scans, which correlated with visual response or absence of response to treatment. Ten of the responders had self-reported improvements in activities of daily living. No serious adverse events occurred, although transient headaches, photophobia, reduction in serum HDL concentrations, and mild, reversible increases in serum triglycerides and aspartate aminotransferase concentrations were observed. "Publication of this data in a preeminent journal such as The Lancet further highlights the importance of our findings and the potential of QLT091001 to rapidly improve visual function in patients with LCA," stated Jason M. Aryeh, Chairman of the Board. "Together with our Phase 1b retreatment data reported earlier this year, these results underscore QLT091001's value as a potential treatment for inherited retinal diseases due to RPE65 and LRAT mutations."