MCO-010 Sustains Vision Gains in Retinitis Pigmentosa at 4 Years
- Patients with retinitis pigmentosa face progressive vision loss, but a disease-agnostic gene therapy called MCO-010 sustained visual acuity gains through long-term follow-up data presented from the REMAIN study.
- The long-term results stem from the REMAIN study, which tracked patients out to week 204 following an initial trial phase.
- The four-year findings build directly on the initial RESTORE trial, which randomly assigned 27 patients to a single injection of MCO-010 at one of two doses or a...
Patients with retinitis pigmentosa face progressive vision loss, but a disease-agnostic gene therapy called MCO-010 sustained visual acuity gains through long-term follow-up data presented from the REMAIN study.
Four-Year Vision Gains in Retinitis Pigmentosa
The long-term results stem from the REMAIN study, which tracked patients out to week 204 following an initial trial phase. At week 204 of the REMAIN study, mean best-corrected visual acuity gains were 0.261 logMAR in the high-dose group and 0.244 logMAR in the low-dose group. Overall, I think still maintaining 13, 14 letters gained in the entire study cohort,
Fan said.
Comparison With the RESTORE Trial Baseline
The four-year findings build directly on the initial RESTORE trial, which randomly assigned 27 patients to a single injection of MCO-010 at one of two doses or a sham procedure. The dosing levels tested were 1.2 × 10¹¹ or 0.9 × 10¹¹ gene copies per eye. At week 52 of that initial study, mean best-corrected visual acuity improved from baseline by 0.337 logMAR in the high-dose group and 0.382 logMAR in the low-dose group.
Those earlier improvements reached statistical significance with P values of .02 for the high-dose cohort and .03 for the low-dose cohort. While patients with retinitis pigmentosa face a very tragic, progressive vision loss, no Food and Drug Administration-approved gene-agnostic therapy currently exists for them.
How MCO-010 Targets Retinal Bipolar Cells
MCO-010 operates as a disease-agnostic therapy by delivering a light-sensitive opsin protein directly to retinal bipolar cells. Once delivered, these bipolar cells serve as a proxy for lost photoreceptors in the damaged eye. When light is present, the modified cells send signals directly to the brain.