Aura Biosciences announced promising safety and efficacy outcomes in a phase 1b/2 trial for AU-011, their first-in-class therapy for primary choroidal melanoma.
“That is an encouraging sign of progress toward developing a new treatment for this cancer that could preserve much more vision than radiotherapy, which is the current standard of care but not FDA approved for this indication,” explained Ivana Kim, MD, co-director of the Ocular Melanoma Center at Massachusetts Eye and Ear.
Last year, AU-011 received fast track and orphan drug designation from the FDA. The intravitreally administered viral nanoparticle selectively binds to ocular melanoma cells and is activated using an ophthalmic laser. This triggers the destruction of AU-011-bound cancer cells—without harming surrounding healthy tissue.
In the current open-label, multicenter trial, the company is assessing single and multiple ascending doses of the therapy in 30 adults with small to medium primary choroidal melanoma.
Patients who were followed for 6 to 12 months successfully maintained their pre-treatment vision. Early efficacy outcomes showed reduced tumor height in 2 participants who received multiple ascending doses. Meanwhile, patients who received a subtherapeutic single ascending dose demonstrated stable disease and vision for up to 12 months.
Encouragingly, there were no severe or serious adverse events, or dose-limiting toxicities. Any observed adverse events were managed with standard care.
“These findings indicate that Aura’s novel, targeted, light-activated treatment could hold real promise for patients with choroidal melanoma,” said Cadmus Rich, MD, chief medical officer of Aura. “Our team is looking forward to continuing our phase 1b/2 study with Dr. Kim and our other collaborators at leading ophthalmology centers across the country.”
The trial will continue to follow patients for 2 years, and is expected to conclude in early 2020.