Bedford-based Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, has dosed the first patient in its Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP).
Nanoscope, a client of TechFW, an incubator / accelerator in Fort Worth, made the announcement on July 19.
“I want to congratulate the Nanoscope team, our clinical partners, and patients participating in the trial. The results from our Phase 2b trial may provide the basis for consideration by the FDA for accelerated approval of MCO-010 for the treatment of RP. We look forward to working with the FDA and other regulatory agencies so that we can bring MCO-010 to RP patients.” said Sulagna Bhattacharya, CEO of Nanoscope.
According to the World Health Organization, more than two billion people suffer from some form of vision impairment, including blindness, and the Centers for Disease Control and Prevention puts the economic costs in the U.S. at more than $51 billion. “If our optogenetic gene monotherapy is eventually approved, it may provide relief to patients with multiple types of retinal degenerative diseases,” Bhattacharya said.
Nasoscope’s optogenetics therapy uses an intravitreally delivered AAV2 vector to deliver Multi-Characteristic Opsin (MCO) genes into retinal cells, where they express polychromatic opsins enabling vision in different color environments. The therapy can be administered in a medical office setting without the need for goggles or other interventions. Based on preliminary evidence from the company’s Phase 1/2a study, MCO-010 is potentially applicable not only for RP, but other degenerative diseases of the eye irrespective of gene mutations. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA.
The Phase 2b will include 27 patients with advanced RP in a randomized, double-blinded, sham-controlled, multi-center trial in the U.S. The treatment will involve a single intravitreal injection of MCO-010 or sham to confirm safety, tolerability and efficacy in improving patients’ vision and visual function. The study is expected to complete enrollment by the end of 2021, with 12-month results available by the end of 2022.
“This is an important milestone for Nanoscope and the field of ophthalmic research. We remain steadfast in our commitment to bring a safe and differentiated optogenetic monotherapy to the ophthalmology community,” said Dr. Mohamed Genead, Chief Development and Chief Medical Officer of Nanoscope. “Accordingly, we are enthusiastic about our MCO-010 development program, which we believe holds significant potential for the treatment of a broad range of retinal diseases.”
A Completed Phase 1/2a open-label study of MCO-010 in 11 patients with RP demonstrated that ambient light activatable optogenetic monotherapy was well-tolerated, with improved quality of life consistent with significant functional vision and visual function improvement in advanced RP patients.
“We are excited by the huge interest received from the RP patients, caregivers and the clinical community. Our team is working hard to further expand the MCO-platform for other retinal degeneration, including Stargardt disease, dry age-related macular degeneration (dAMD), and others,” said Samarendra Mohanty, Ph.D., Nanoscope’s President and Chief Scientific Officer.