Nanoscope to Submit BLA for MCO-010 to Treat Retinitis Pigmentosa

Nanoscope Therapeutics Inc., a clinical-stage biotechnology company based in Dallas, reported a highly productive meeting with the U.S. Food and Drug Administration (FDA) regarding its MCO-010 therapy aimed at treating retinitis pigmentosa (RP). This positive interaction has allowed Nanoscope to move forward with plans to submit a Biologics License Application (BLA) in the first quarter of 2025.

During the meeting, the FDA recognized Nanoscope's proposed steps toward submitting the BLA for MCO-010, which has been designated Fast Track status due to the severe vision impairment it addresses. The company's plan involves a rolling submission process, an approach facilitated by the Fast Track designation.

Retinitis pigmentosa is a group of inherited eye diseases that damage the retina, leading to severe vision loss and eventually blindness. Despite the focus on vision rehabilitation, patients with RP often experience a lifetime of progressive vision decline, severely affecting their quality of life. Current treatment efforts aim to preserve existing vision and slow further deterioration.

Dr. Allen C. Ho, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and Chief Medical Advisor of Nanoscope, emphasized the significance of preserving baseline visual acuity over several years, a positive deviation from the typical progression of RP. He highlighted the potential of MCO-010 to offer a restorative treatment option for patients suffering from various forms of RP.

Sulagna Bhattacharya, Co-founder and CEO of Nanoscope, expressed satisfaction with the productive discussions with the FDA, crediting the expertise and dedication of the Nanoscope team. She reiterated the company's goal to change lives by advancing MCO-010 to regulatory submission and emphasized the focus on patients awaiting meaningful sight restoration.

Samarendra Mohanty, Ph.D., President and Chief Scientific Officer of Nanoscope, noted that the positive FDA meeting follows the recent End of Phase 2 meeting for their Stargardt macular degeneration program, which is advancing to a Phase 3 registrational trial. He pointed to evidence from the RESTORE and REMAIN studies, which showed improvements in visual acuity over three years, as reinforcing the company’s commitment to bringing transformative therapy to patients.

Retinitis pigmentosa is a significant cause of blindness among working-age adults. It is an inherited condition caused by various gene mutations, leading to the degradation of light-sensing photoreceptor cells in the retina and consequent vision loss. Currently, no approved treatments exist for severe vision loss due to RP, and treatment goals focus on slowing or preventing further vision deterioration.

MCO-010, known scientifically as sonpiretigene isteparvovec, is a novel gene therapy currently undergoing clinical trials. Unlike existing gene therapies targeting specific genetic mutations, MCO-010 is designed to restore vision by targeting inner retinal neurons. Using a proprietary AAV2 vector, MCO-010 achieves robust transduction in bipolar cells, essential for restoring high-quality vision in everyday settings. Clinical trials have shown significant improvements in vision-guided mobility, shape discrimination, and visual acuity, along with a favorable safety profile.

The company has also completed the Phase 2 STARLIGHT trial for MCO-010 in Stargardt patients and plans to initiate a Phase 3 registrational trial in the first quarter of 2025.

Nanoscope Therapeutics is dedicated to developing optogenetic therapies to restore sight for millions of patients suffering from retinal degenerative diseases. Their leading asset, MCO-010, has received FDA Fast Track and orphan drug designations for both RP and Stargardt's disease. In addition to MCO-010, Nanoscope is advancing preclinical assets, including the non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.