Optogenetic therapy shows potential for vision restoration in phase 2b trial
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Key takeaways:
- Study participants with advanced retinitis pigmentosa who received MCO-010 experienced improvements in vision.
- MCO-010 was well tolerated, with no serious or severe adverse events reported.
MCO-010 demonstrated positive topline results for vision restoration in patients with advanced retinitis pigmentosa, according to a press release from Nanoscope Therapeutics.
The multicenter, randomized, double-masked, sham-controlled phase 2b RESTORE clinical trial included 18 participants with severe vision impairment due to retinitis pigmentosa who received one intravitreal injection of the multi-characteristic opsin optogenetic therapy compared with nine participants who received a sham intravitreal injection.
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Of the 18 participants treated with MCO-010, 88.9% experienced a clinically significant improvement in luminance level of two or more in vision-guided mobility or near object recognition at 12 months compared with 44.4% of patients treated with placebo (P < .05).
Additionally, seven participants treated with MCO-010 improved by –0.3 logMAR or more in best corrected visual acuity vs. one participant treated with placebo.
The most common treatment-emergent adverse events reported across both groups were anterior chamber cells, ocular hypertension and conjunctival hemorrhage. Overall, the optogenetic therapy was well tolerated, and no serious or severe ocular or systemic adverse events were reported.
“These results suggest that MCO-010 provides substantial benefit to patients with severe vision loss due to advanced RP, a condition for which there are currently no available treatments,” Sulagna Bhattacharya, co-founder and CEO of Nanoscope, said in the release. “We are looking forward to engaging with the FDA and other regulatory agencies on the future of MCO-010, with the goal of expeditiously getting this novel therapy to patients.”
The FDA previously granted MCO-010 orphan drug and fast track designations for retinitis pigmentosa and Stargardt disease.
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