Nanoscope to submit BLA for MCO-010 to treat retinitis pigmentosa

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Nanoscope Therapeutics Inc announced a productive FDA meeting for its clinical program evaluating MCO-010 for the treatment of retinitis pigmentosa (RP).

According to the company, based on the regulatory feedback provided in the meeting, it will commence Biologics License Application (BLA) submission in Q1 2025.

MCO-010 is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation.¹

Sulagna Bhattacharya, co-founder and CEO of Nanoscope, said the company was pleased with the positive interactions it has had with FDA.

“Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission,” Bhattacharya said in a statement. “With every step forward, we are focused on the patients who are waiting for meaningful sight restoration. Our team looks forward to continuing the important work we have begun, along with our partners, to bring this therapy to patients who have significant unmet need.”

The FDA has acknowledged Nanoscope’s proposed next steps to facilitate a BLA submission for MCO-010 for the treatment of severe vision loss due to RP. The company outlined its plans for a rolling submission of the MCO-010 BLA for the treatment of severe vision loss due to RP based on its Fast Track Designation.

According to the company, severe vision loss is often the outcome for individuals diagnosed with RP, ultimately leading to blindness. The current treatment paradigm focuses on vision rehabilitation, but patients diagnosed with RP often go through a lifetime of progressive vision issues, which impacts their quality of life.

Allen C. Ho, MD, FACS, FASRS, director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and chief medical advisor of Nanoscope, said in a statement the preservation of baseline visual acuity over several years represents an important treatment effect that deviates from the expected natural history of RP.

“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 progressive retinal degenerations that comprise RP,” he said.

Samarendra Mohanty, PhD, president and chief scientific officer of Nanoscope, noted the meeting with the FDA also follows the company’s recent End of Phase 2 meeting for its

Stargardt macular degeneration program, which is advancing to a Phase 3 registrational trial.

“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,” Mohanty said.

The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently unveiled plans to start a Phase 3 registrational trial during the first quarter of 2025.¹

Reference:

1. 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. Prnewswire.com. Published September 12, 2024. Accessed October 9, 2024. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-announces-end-of-phase-2-meeting-with-us-fda-and-plan-to-initiate-a-phase-3-clinical-trial-of-mco-010-to-treat-stargardt-macular-degeneration-302247017.html