Nanoscope Therapeutics’ gene therapy MCO-010 (sonpiretigene isteparvovec) is in the spotlight with encouraging Phase IIb results from its RESTORE trial (NCT04945772) in the retinitis pigmentosa (retinitis) market. Offering a new mechanism of action, it presents a looming challenge to Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), the current frontrunner in gene therapy for retinitis. With promising safety profiles and ongoing trials, MCO-010’s efficacy has the potential to redefine the treatment landscape for retinitis patients, says GlobalData, a leading data and analytics company.

MCO-010, which has FDA fast track designation and FDA orphan drug designation, is of paramount interest due to being a gene-agnostic optogenetic therapy. RESTORE trial results in patients with retinitis showed that at 52 weeks, high-dose and low-dose groups both exhibited statistically significant improvements in best-corrected visual acuity (BCVA).

According to Nanoscope, the RESTORE trial is the first pivotal randomized controlled trial of a mutation-agnostic RP gene therapy that demonstrates a statistically significant improvement exceeding the clinically important BCVA >0.3 LogMAR threshold. The trial also met its key secondary endpoint, confirming that this improvement was continued in the high-dose group for 76 weeks; the high dose (1.2 x 1011 gc/eye) is the intended commercial dose for MCO-010. MCO-010 also demonstrated improvements in the Y mobility test and the shape discrimination test at 52 weeks, in both high and low dose groups.

As for the safety profile of MCO-010, in general, the treatment was tolerated well and results were consistent with preceding trials. Most adverse events were mild to moderate in severity, relating to anterior chamber cell and ocular hypertension.

Further data from the RESTORE trial is set to be unveiled at the Annual Scientific Meeting of the Association for Research in Vision and Ophthalmology, which will take place in Seattle, Washington, on 06 May 2024.

Sara Reci, MSc, Senior Pharma Analyst at GlobalData, comments: “MCO-010, like Luxturna, is a gene therapy. Despite this, it stands out by offering an alternative mechanism of action. MCO-010 is an optogenetic therapy that enables light-based vision restoration and reduces light-induced chronic retinal cell damage through delivering white opsin to target cells, which generates substantial photocurrent at white light intensity levels that resemble daylight conditions. This has positive implications for patients, which include potentially introducing a second gene therapy into the RP space, as well as providing a treatment with a new mechanism of action for RP patients.”

The key opinion leaders (KOLs) interviewed by GlobalData noted that Nanoscope, in developing MCO-010, has shown some improvements, and is moving towards pivotal trials, and that the therapy itself is interesting. However, KOLs also pointed out that in general, a shared issue among optogenetic therapies is that it is unclear how high of a resolution patients can reach. Nonetheless, Nanoscope plans to apply for FDA approval for MCO-010 in H2 2024 based on these Phase IIb results.

Reci concludes: “Should Nanoscope receive approval for MCO-010, it may have tremendous potential to be popular among retinitis patients and clinicians alike, given its promising RESTORE trial results, and additionally the need for more therapies within the RP space.”