Nanoscope Signals BLA Submission for MCO-010 for Retinitis Pigmentosa

Allen C. Ho, MD

Credit: Wills Eye Hospital

Nanoscope Therapeutics has announced plans to submit the Biologics License Application (BLA) for MCO-010 gene therapy for the treatment of severe vision loss due to retinitis pigmentosa (RP) in the first quarter of 2025.¹

Released on October 10, 2024, the announcement followed a productive meeting with the US Food and Drug Administration (FDA) discussing the MCO-010 clinical trial program. The FDA acknowledged the company’s proposal for the next steps for an MCO-010 BLA submission.

“Preservation of baseline visual acuity over several years represents an important treatment effect that deviates from the expected natural history of RP,” said Allen C. Ho, MD, director of retina research and co-director of the retina service at Wills Eye Hospital, and chief medical advisor of Nanoscope.¹ “Feedback from the FDA has informed Nanoscope on its BLA submission plan, thereby presenting the potential for a viable restorative option for patients whose vision has been lost to the array of progression retinal degenerations that comprise RP.”

Retinitis pigmentosa is a group of inherited rare eye diseases impacting the retina, with symptom presentation in childhood, and is a leading cause in working-age adults.² Hundreds of gene mutations have been identified that can cause RP, degrading light-sensing photoreceptor cells. However, there are no approved treatments for severe vision loss due to RP, making slowing or preventing further vision deterioration a notable treatment goal for RP.

Ambient-light activatable MCO optogenetic monotherapy targeting inner retinal neurons can potentially restore vision loss due to advanced RP, with degenerated outer retinal cells.¹ With bipolar cells targeting via mGluR6 promoter-enhancer, the MCO-010 expression cassette has been designed to restore high-quality vision in real-world environments.

Phase 1/2 trial data of MCO-010 in advanced RP showed improvements in vision-guided mobility, shape discrimination, and visual acuity. In the multicenter RESTORE study, a notable population of patients treated with MCO-010 experienced an improvement in best-corrected visual acuity (BCVA) and functional vision, as well as shape discrimination, with a favorable safety profile.

Nanoscope recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt disease, with plans to initiate a Phase 3 registration trial after a productive end-of-phase-2 meeting with the FDA.³

“This productive meeting with the FDA also follows our recent End of Phase 2 meeting for our Stargardt macular degeneration program, which is advancing to a Phase 3 registrational trial,” said Samarendra Mohanty, PhD, president and chief scientific officer.¹ “The evidence of improvements in visual acuity over 3 years across the RESTORE and REMAIN studies reinforces the strength of our commitment to bring this transformative therapy to the patients.”

The company described its plan for a rolling submission of the MCO-010 BLA for severe vision loss due to RP based on its Fast Track Designation granted by the FDA.

“We are pleased with the positive interactions we have had with FDA as a result of the exceptional expertise and tireless commitment of the Nanoscope team,” said Sulagna Bhattacharya, co-founder and chief executive officer of Nanoscope.¹ “Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission.

References

1. _{Nanoscope Therapeutics. Nanoscope announces plans to submit bla for MCO-010 to treat retinitis pigmentosa. Nanoscope Therapeutics. October 10, 2024. Accessed October 10, 2024. https://nanostherapeutics.com/2024/10/10/nanoscope-announces-plans-to-submit-bla-for-mco-010-to-treat-retinitis-pigmentosa/.}
2. _{O'Neal TB, Luther EE. Retinitis Pigmentosa. [Updated 2024 Feb 12]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK519518/}
3. _{Nanoscope Therapeutics. Nanoscope therapeutics announces end-of-phase 2 meeting with U.S. FDA and plan to initiate a phase 3 clinical trial of MCO-010 to treat stargardt macular degeneration. Nanoscope Therapeutics. September 12, 2024. Accessed October 10, 2024. https://nanostherapeutics.com/2024/09/12/nanoscope-therapeutics-announces-end-of-phase-2-meeting-with-u-s-fda-and-plan-to-initiate-a-phase-3-clinical-trial-of-mco-010-to-treat-stargardt-macular-degeneration/.}