Q&A: Kenneth Fan on MCO-010 trials for Stargardt disease

Kenneth C. Fan, MD, MBA, of Retina Consultants of Texas, highlighted findings from the STARLIGHT study at the American Academy of Ophthalmology 2025 annual meeting, held October 18-10, in Orlando, Florida, United States. He focused on the use of MCO-010 optogenetic therapy in the treatment of Stargardt disease, an inherited retinal dystrophy for which no approved therapies currently exist. The study represents an important step in the exploration of gene-based and novel therapeutic interventions for inherited retinal diseases (IRDs).

Note: The following conversation has been lightly edited for clarity.

Ophthalmology Times Europe: What visual improvements were observed across the different assessments in patients treated with MCO-010, and how do these compare to baseline measurements?

Kenneth C. Fan, MD, MBA: MCO-010 was used in the treatment of Stargardt disease in the STARLIGHT study, which is a Phase 2 open-label study. The clinical trial end points were safety, but also looking at efficacy, and the most pronounced improvements of visual acuity were noted in 3 of the patients with a macular phenotype, wherein significant meaningful gains in vision were noted at 48 weeks with and without the wearable magnifier.

OTE: What safety and tolerability findings emerged from the study, and how might they impact the future development of MCO-010 for Stargardt disease?

Fan: Safety and tolerability is one of the most important things in a Phase 2 trial. In this study, there were no incidents of retinitis or vasculitis noted throughout the study period. There were a few incidences of transient vitreous cell that was noted, but those all resolved by the end of the study. The difficult thing with seeing patients with Stargardt disease is that there's no approved therapy for Stargardt disease, and so we're happy to see people be able to get any sort of treatment to stabilize or potentially improve the vision. As I mentioned before the macular phenotype, which is more typical Stargardt disease. Those patients in the STARLIGHT study saw meaningful improvements in visual acuity, and so I think that should be a great guidance for the registrational Phase 3 study in the future to select those patients so that we can maximize visual improvement in patients.

OTE: How do these results inform patient selection, timing of intervention, or potential combination therapies for optogenetic treatment in inherited retinal diseases?

Fan: I'm just hopeful for the future of treatment for patients with inherited retinal disease, not just Stargardt disease, but retinitis pigmentosa and other more rare conditions. There's just really been a significant growth of interest in IRDs and drug development for IRDs in the past couple of years. So I'm really hopeful for my patients and looking forward to the future.