Patients with ocular melanoma have very few treatment options, and those that exist – removal of the eye or plaque radiation – leave them blind within two years. Aura Biosciences has received orphan drug designation from the Food and Drug Administration for its class of intravitreal therapies that target ocular melanoma, founder and CEO Elisabet de los Pinos, PhD, reported at OIS@AAO.
“We have a novel therapy that can be administered in an outpatient procedure with an intravitreal injection activated with an ophthalmic laser that can preserve vision and remove these tumors,” Dr. de los Pinos said.
With the orphan drug designation, Aura expects a rapid development: clinical proof of concept in the first quarter of 2016, a clinical trial in 2016 and an early readout by the second quarter of 2016, and possibly commercial launch in 2017 or 2018 if the trials are successful.
The agent, AU-011, selectively binds tumor cells to kill them. The agent targets cancer cells with a viral-like particle through interaction with heparin sulfate, Dr. de los Pinos said. In preclinical rabbit models, AU-011 was shown to penetrate the entire tumor.
Dr. de los Pinos also noted the company has recruited three expert ocular oncologists to its clinical advisory board: Carol Shields, MD, co-director of the Oncology Service at Wills Eye Hospital, Philadelphia; Evangelos Gragoudas, MD, director of the Retina Service at the Massachusetts Eye and Ear Infirmary of Harvard University, Boston; and Arun D. Singh, MD, director of the Department of Ophthalmic Oncology in the Cole Eye Institute at the Cleveland Clinic.
Aura is also in the early investigation stage of using AU-011 to treat retinoblastoma and is developing a second agent, AU-012, for treatment of bladder and head and neck cancer, Dr. de los Pinos said.
Elisabet de los Pinos, PhD
Elisabet de los Pinos is the Founder and CEO of Aura Biosciences. Eli founded Aura with a vision to transform cancer treatment by developing a novel class of targeted cancer therapy using viral-like particles (VLPs) to deliver drugs selectively to cancer cells, while leaving surrounding tissue unharmed.