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Nanoscope Therapeutics Inc., have announced key results for Phase 2b (“RESTORE”) for the treatment of retinitis pigmentosa (RP) using optogenetic therapy. 

Nanoscope Therapeutics Inc., a company based in Bedford, Texas, has reported key data from a Phase 2b multi-centre RCT (NCT04945772) of “MCO-010” (sonpiretigene isteparvovec), an ambient-light activatable optogenetic therapy to treat advanced retinitis pigmentosa (RP). In the trial, 18 patients with vision impairment due to RP received a single intravitreal injection of MCO-010, while 9 received a sham intravitreal injection. Results showed vision function improvements after treatment with MCO-010 “consistent with previous studies as well as a favorable safety profile”. Key efficacy measures, including mobility tests and BCVA scores, reported that 100% of patients (18/18) experienced a clinically meaningful improvement in vision-guided mobility (MLYMT), near-field object recognition (MLSDT) or visual acuity (BCVA) (p=0.007 vs sham).


According to the trial study, the experimental MCO-010 treatment is an adeno-associated virus (AAV) serotype 2-based vector carried an opsin gene expression cassette, aimed to deliver and express genetically encoded light-sensitive proteins (opsins) to remaining light-insensitive neurons in the inner retina of patients with RP, RP patients for which the window of therapeutic opportunity for ocular genetic therapies is surpassed. Optogenetic monotherapy targets abundant inner retinal neurons has the potential to restore vision lost due to advanced RP. Optogenetic therapy can theoretically be applied to all patients with end-stage RP.  In addition to the efficacy test results reported, the company stated that there were no serious or severe ocular or systemic adverse events and the results were consistent with those observed in an earlier Phase 1/2 trial (NCT04919473) in which 9 of 11 (82%) of subjects demonstrated 2 luminance level improvements in vision-guided mobility or 0.3 logMAR in visual acuity.


Commenting on the study, David Boyer, MD, Retina-Vitreous Associates Medical Group in Beverly Hills, CA and Nanoscope Clinical Advisory Board member stated that, “I am honored to present the RESTORE trial results at ARVO. I have had the privilege of treating severely blind patients, enrolled in MCO-010 clinical trials, and have observed some improvement in their visual function. The fact that we see any gains in vision after a single intravitreal injection is remarkable. Some participants who were living with severe vision impairment due to RP have noticed improvement in visual function. In addition, MCO-010 had a favorable safety profile. It is an honor to have been a part of the first randomized controlled trial to show a visual improvement in a profoundly visually impaired population”.