Dr. Schefler provided an update on the multicenter trial, which has been designed to evaluate the safety and efficacy of single and multiple ascending doses in 30 adult subjects with clinically diagnosed small to medium primary choroidal melanoma.
Interim data show that, in an expanded set of subjects, AU-011 continues to be well tolerated with no related serious adverse events, no severe adverse events, and no dose-limiting toxicities observed, including the cohorts in the multiple ascending-dose phase of the study. Adverse events were manageable with standard-of-care treatments and there have been no long-term clinical sequelae. Pretreatment visual acuity was maintained in all subjects that have been followed for 6 to 12 months.
Early efficacy results continue to be promising, with several subjects in the multiple-ascending-dose cohorts showing evidence of reduction in tumor height and 100% of the patients meeting the endpoint of stable disease at 3 months.
“All current treatment options for patients with this cancer result in significant vision loss from radiation-related side effects, ” said Dr. Schefler. “A treatment that is office-based, minimally invasive, and has a low risk of side effects would be a dramatic improvement in the therapeutic landscape for this deadly disease.”