RWC 2024: Longitudinal BCVA and safety analysis of mutation-agnostic MCO-010 optogenetic therapy for retinitis pigmentosa

Christine Kay, MD, spoke with Modern Retina at the 2024 Retina World Congress in Fort Lauderdale, Florida. She shared insights from her presentation titled, "Longitudinal BCVA and Safety Analysis Of Mutation-Agnostic MCO-010 Optogenetic Therapy For Retinitis Pigmentosa: Patient Case From A Phase 2b Randomized, Sham-Controlled, Patient- And Assessor-Masked Clinical Trial."

Video Transcript:

Editor's note: The below transcript has been lightly edited for clarity.

Christine Kay, MD:

Hello, my name is Christine Kay, I'm a vitreoretinal surgeon and inherited retinal disease specialist at Vitreoretinal Associates located in Gainesville, Florida. I'm excited to be here at Retina World Congress 2024 in Fort Lauderdale, presenting the longitudinal safety and BCVA analysis in patients with MCO-010, a gene agnostic optogenetic therapy for patients with advanced RP, looking at the RESTORE trial data. The RESTORE trial was an optogenetics randomized control gene therapy trial for patients with advanced RP. Patients were randomized into 3 arms. There was a low dose arm, again of this intravitreally delivered optogenetics therapy, a high dose arm, and a sham arm. Each arm wwith 9 patients, so a total of 27 patients were treated. This is looking at patients with RP. Retinitis pigmentosa is one of the most common inherited retinal dystrophies with a prevalence of 1 out of 4000, and that affects approximately 1.5 million patients globally. It is the first efficacy results we're seeing in patients with advanced RP, and we have a currently high unmet need for patients with advanced RP and vision loss. The inclusion criteria for the trial involved looking at patients with a visual acuity no better than 1.9 logMAR. The primary endpoint of the trial was looking at BCVA improvement from baseline to sham at week 52. And importantly, in this trial, the primary endpoint was met. There was a statistically increased visual acuity BCVA at week 52 as compared to baseline in the treated patients, and actually both dose cohorts, both the high and the low dose, as compared to the sham treated patients. In regards to the safety data. The safety looked excellent. The profile showed no SAEs, drug-related SAEs, and patients treated with this therapy. There was mild to moderate ocular inflammation as would be expected for any individual delivered AAV gene therapy. And this was readily treatable with reintroduction typically of topical steroid, only 1 patient remained on any topical steroid out of their week 52 visit. So, that is an excellent safety profile. There was 1 patient, as far as efficacy, and this is what I talked about in my podium presentation, with an excellent visual acuity improvement. That was a 1.83 logMAR visual acuity improvement, and a lot of us don't think in terms of logMAR. So, in terms of a Snellen visual acuity, that patient was approximately 20/3000, or hand motion vision and a typical clinical visit, and by the week 52 visit, had improved to between 20/40 and 20/50 visual acuity, which is a profound improvement in visual acuity and not possibly explained by intervisit variability in this patient population. So it was exciting to be able to deliver, in conclusion, the data of efficacy and safety again, this was MCO-010 and intravitreally delivered optogenetic gene therapy for the treatment of advanced RP with an excellent safety profile. And again I'm here at Retina World Congress 2024 in Fort Lauderdale, and I am a consultant and advisor for Nanoscope Therapeutics. Thank you very much.