Patients with RP face “a very tragic, progressive vision loss,” Fan said, and no FDA-approved gene-agnostic therapy exists for them. MCO-010 is a disease-agnostic therapy that delivers a light-sensitive opsin protein to retinal bipolar cells. The bipolar cells can then serve as a proxy for lost photoreceptors, sending signals to the brain when light is present.
What did RESTORE and REMAIN show?
RESTORE randomly assigned 27 patients to a single injection of MCO-010 at 1 of 2 doses (1.2 × 10¹¹ or 0.9 × 10¹¹ gene copies per eye) or sham.¹ At week 52, mean best-corrected visual acuity (BCVA) improved from baseline by 0.337 logMAR in the high-dose group and 0.382 logMAR in the low-dose group (P = .02 and P = .03, respectively).¹ At week 204 of REMAIN, the long-term follow-up study, mean BCVA gains were 0.261 logMAR and 0.244 logMAR, respectively. That is equivalent to an approximately 3-line Early Treatment Diabetic Retinopathy Study improvement.¹ “Overall, I think still maintaining 13, 14 letters gained in the entire study cohort,” Fan said. No serious adverse events were reported, and the mild to moderate intraocular inflammation that occurred was treatable.¹