Nanoscope Therapeutics Announces First Patient Dosed in Phase 2b Clinical Trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa
BEDFORD, Texas, July 19, 2021 / Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, today announced that the first patient had been dosed in its Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP).
“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, Chief Executive Officer of Nanoscope.
Nanoscope’s optogenetics therapy uses an intravitreally delivered proprietary 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 trial (NCT04945772) will include 27 participants in a randomized, double-blinded, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. 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 both Nanoscope and the field of ophthalmic research. We remain steadfast in our commitment to bringing 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 the 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 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.
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina. When activated by light, these opsins trigger the physiological process of vision. RP, linked to over 60 different gene mutations, encompasses a group of rare genetic disorders where photoreceptors degrade gradually, leading to impaired vision and eventual blindness.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. The company’s pipeline includes optogenetics-based retinal regeneration therapies for patients with RP, Stargardt disease, and dAMD.