Retinitis Pigmentosa Optogenetic Therapy Gets FDA Fast Track Designation


The therapy was previously granted orphan drug designation for both RP and Stargardt disease.

Nanoscope Therapeutics’ MCO-010, an investigational ambient-light activatable multi-characteristic opsin (MCO) optogenetic monotherapy, has received fast track designation (FTD) from the FDA for the treatment of retinitis pigmentosa (RP) via intravitreal injection.1

MCO-010 is intended to restore vision in blind patients. It previously demonstrated promising efficacy and safety in a phase 1/2 clinical trial (NCT04919473) for patients with RP, data from which were presented at the 2021 Annual Meeting of the American Society of Retina Specialists (ASRS), October 8-12.2 It is currently being investigated in 2 additional trials, including a phase 2 clinical trial (RESTORE; NCT04945772) for patients with RP and a separate phase 2 study (STARLIGHT; NCT05417126) for patients with Stargardt disease. Nanoscope announced that the first patient in the STARLIGHT trial, which has reached full enrollment, was dosed in July of this year.3,4 It was previously announced that the therapy was granted orphan drug designation for both RP and Stargardt disease by the FDA in January 2022.5

"The FDA's decision to grant FTD underscores the importance of MCO-010 to address a serious unmet need and validates its potential as an effective therapeutic for patients with RP," Sulagna Bhattacharya, chief executive officer, Nanoscope Therapeutics, said in a statement.1 "We are proud to have the support of the FDA and look forward to collaboratively interacting with FDA to assess next steps in the clinical development and future regulatory review of MCO-010."

The double-masked, sham-controlled RESTORE trial enrolled 27 patients with RP with advanced vision loss in January 2022. Participants are required to be 18 years of age or older, to have had a best-corrected visual acuity (BCVA) worse than 1.9 LogMAR in the study eye at screening and 1.6 LogMAR or worse in the fellow eye, and to have had retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening. Patients who pass mobility testing at 0.3 or 1 lux will be excluded from the study. Patients with narrow iridocorneal angles contraindicating pupillary dilation, retinal detachment in the study eye affecting central vision, or significant vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole in the study eye significantly affecting central vision will also be excluded from the study. Additional exclusion criteria relate to patient health status, including specific pre-existing conditions in the eyes, current treatments, and treatment history.

The study will divide participants into 3 arms with 9 patients each. The medium dose level arm will receive 0.9x1011 gc/eye of MCO-010 and the high dose level arm will receive 1.2x1011 gc/eye of MCO-010. Patients in the third arm will receive a sham injection. Doses of MCO-010 will be delivered via a single intravitreal injection. The primary end points for the study are the change from baseline in the Y-Mobility Test at 52 weeks and the type, severity, and incidence of ocular and systemic adverse events. Secondary end points include changes from baseline in shape recognition assay, determination of optical flow direction, Freiburg Visual Acuity, pupillary light reflex, light sensitivity, visual field, functional vision outcomes measured by the National Eye Institute Visual Function Questionnaire 25, and pharmacokinetic parameters. The study has an estimated completion date of March 1, 2024. Nanoscope Therapeutics expects topline data from RESTORE to be announced in the first half of next year.

1. Nanoscope Therapeutics receives fast track designation by the FDA for MCO-010 for the treatment of retinitis pigmentosa. News release. Nanoscope Therapeutics Inc. October 10, 2022. 
2. Positive data from Nanoscope's phase 1/2a trial of gene therapy to restore vision in patients blinded by retinitis pigmentosa to be featured at 2021 ASRS meeting. News release. Nanoscope Therapeutics. October 8, 2021.
3. Nanoscope Therapeutics announces first patient dosed in STARLIGHT phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease. News release. Nanoscope Therapeutics. July 25, 2022.
4. Nanoscope Therapeutics announces completion of enrollment in STARLIGHT phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease. News release. Nanoscope Therapeutics Inc. September 13, 2022. 
5. NanoscopeTherapeutics announces FDA clearance of IND for MCO-010 gene therapy in Stargardt macular degeneration patients. News release. Nanoscope Therapeutics. January 25, 2022. 
Recent Videos
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary
Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials
Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics
Zheng-Yi Chen, DPhil, on Looking Deeper Into Effects of Gene Therapy on OTOF Deafness
Arshad Khanani, MD
Alfonso Sabater, MD, PhD
Shankar Musunuri, PhD
Ula V. Jurkunas, MD
Alfonso Sabater, MD, PhD, on Bringing Gene Therapy to Ophthalmology
© 2024 MJH Life Sciences

All rights reserved.