This open-label, multicenter trial is designed to investigate single and multiple ascending doses of light-activated AU-011 in approximately 36 adult subjects with clinically diagnosed primary choroidal melanoma.
The data show that multiple administrations of AU-011 are well tolerated with no related serious adverse events, severe adverse events, or dose-limiting toxicities observed. Drug-related adverse events were all expected and included anterior-chamber inflammation, posterior-chamber inflammation, and increase in IOP, but all were manageable with standard-of-care treatments and resolved without clinical sequelae.
Treatment with AU-011 achieved preservation of BCVA with a mean change of -1.06 letters at 6 months and a mean change of -0.75 letters at 12 months. BCVA was preserved even in high-risk patients with tumors close to the fovea or the optic disk, a factor that typically correlates with a higher risk of irreversible severe vision loss following radioactive treatments. All patients achieved stable disease at the prespecified preliminary efficacy endpoint at 3 months. Biological activity has been confirmed with long-term tumor control in those patients with documented growth before treatment, reduction in tumor thickness, and localized inflammation around the tumor. An expansion cohort of the study is currently under way. The company plans to initiate a pivotal phase 3 clinical program following the phase 1b/2 study.