Carol L. Shields
NEW ORLEANS — Choroidal melanoma has a high unmet medical need, according to Carol L. Shields, MD, and results of an early-stage study of a light-activated viral nanoparticle lend encouragement that a therapy may be developed to meet that need.
At Retina Subspecialty Day preceding the American Academy of Ophthalmology meeting,
Shields delivered interim results from the phase 1b/2 open-label ascending single and repeat dose clinical trial designed to evaluate the safety of AU-011 (Aura Biosciences) for treatment of small to medium choroidal melanoma.
Choroidal melanoma is a rare disease, affecting about 8,000 people worldwide, Shields said. The main treatment option is radiation, which can cause severe vision loss, and metastasis occurs in 25% of patients.
In this study of six patients with 3- and 6-month follow-up, safety as determined by multimodal imaging was the primary endpoint. Secondary endpoint was preliminary efficacy. Both endpoints were met.
Regarding efficacy, “We found stable disease in 83% of patients,” Shields said. “One patient did show tumor growth and required plaque radiotherapy, but keep in mind we’re using subtherapeutic medication as a judgment for safety.”
In rabbits, a 50-µg dose caused complete necrosis of tumors. In this study, three patients received a 20-µg dose intravitreally and three a 40-µg dose.
“We found no serious adverse events. We did find a little bit of inflammation in the anterior and the posterior segment that led to increased intraocular pressure in three patients,” Shields said, attributing the reaction to the possibility of immune stimulation. All patients had preserved vision within five letters of initial vision. – by Patricia Nale, ELS
Shields CL. A phase 1b clinical safety study of a novel tumor targeted therapy (AU-011) for the treatment of primary choroidal melanoma. Presented at AAO Subspecialty Days; Nov. 10-11, 2017; New Orleans.
Disclosure: Shields reports she serves on the advisory board of Aura Biosciences.