QLT091001: Preliminary Phase Ib data

Preliminary data from an open-label Phase Ib trial in 17 patients with RP due to inherited lecithin-retinol acyltransferase (LRAT) or retinal pigment epithelium-specific protein 65kDa (RPE65) gene mutations showed that once-daily 40 mg/m 2 QLT091001 for 7 days improved mean retinal area from baseline by 22%, 16% and 18% at days 7, 14 and 30, respectively (p=0.03, p=0.13 and p=0.096, respectively). In 14 evaluable patients meeting GVF test criteria as determined by a third-party reader, QLT091001 improved mean retinal area from baseline by 34%, 29% and 23% at days 7, 14 and 30, respectively (p=0.005, p=0.02 and p=0.07, respectively). Furthermore, 9 of 17 patients had their BCVA improve by >=5 letters over baseline in at least 1 eye on the ETDRS eye chart. ...