Nanoscope Announces Plans to Submit BLA for Retinitis Pigmentosa Treatment

Nanoscope Therapeutics Inc. recently announced a productive FDA meeting for its clinical program evaluating MCO-010 for the treatment of retinitis pigmentosa (RP). Based on the regulatory feedback provided in the meeting, Nanoscope will commence with submission of a Biologics License Application (BLA) in Q1 2025.

In connection with the meeting, the FDA acknowledged Nanoscope’s proposed next steps to facilitate a BLA submission for MCO-010 for the treatment of severe vision loss due to RP. Nanoscope 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.

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 still endure a lifetime of progressive vision impairment, thereby dramatically impacting quality of life.

“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, FACS, FASRS, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and Chief Medical Advisor of Nanoscope. “Feedback from the FDA has informed Nanoscope’s BLA submission plan, for a viable, restorative option for patients whose vision has been lost due to the array of progressive retinal degenerations that comprise RP.”

“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,” added Sulagna Bhattacharya, Co-founder and Chief Executive Officer of Nanoscope. “Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission. 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.”

“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,” also added Samarendra Mohanty, PhD, President and Chief Scientific Officer of Nanoscope. “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 patients.”

Retinitis pigmentosa, is a group of rare eye diseases that affect the retina, that presents symptoms in childhood and is a leading cause of blindness in working-age adults. It is an inherited disease, meaning it is passed on to children via faulty genes from their parents. There are more than several hundred gene mutations identified that can cause RP. In patients with RP, the light-sensing photoreceptors cells degrade leading to loss of vision. Currently, there are no approved treatments for severe vision loss due to RP. Slowing or preventing further deterioration of vision for patients with RP is a treatment goal.

MCO-010 (sonpiretigene isteparvovec, suspension for intravitreal injection) is the only broadband, fast, and most sensitive opsin currently in clinical trials. Current gene therapies are aimed at treating patients with specific genetic mutations in the outer retinal cells, while ambient-light activatable MCO optogenetic monotherapy targeting abundant inner retinal neurons has the potential to restore vision loss due to advanced RP, with degenerated outer retinal cells. With bipolar cell targeting via mGluR6 promoter-enhancer, the MCO-010 expression cassette is designed for restoring high-quality vision in real-world environments. The proprietary AAV2 vector allows robust transduction of MCO-010 in bipolar cells upon intravitreal injection. The Phase 1/2 trial of MCO-010 in advanced RP patients demonstrated improvement in vision-guided mobility, shape discrimination, and visual acuity. A significant proportion of patients treated with MCO-010 in the randomized double-masked multicenter RESTORE trial exhibited improvements in best-corrected visual acuity as well as functional vision assessed by vision-guided mobility, and shape discrimination, along with a favorable safety profile.

The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025.

Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company’s lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.