Nanoscope Therapeutics releases positive long-term safety results from EXTEND study

Nanoscope Therapeutics has released positive long-term safety results from its EXTEND study, a 5-year follow-up of participants who received a single intravitreal injection of MCO-010 in a previously conducted phase 1/2a trial.

The company noted that the EXTEND trial followed 10 patients with advanced retinitis pigmentosa (RP) who had previously received MCO-010. According to the company, the results showed that a single intravitreal injection of MCO-010 is safe and well-tolerated over 5 years, with no serious adverse effects or new safety signals. The safety profile of MCO-010 remained consistent between the phase 1/2a trial and the long-term follow-up period.¹

Patients who received a higher MCO-010 dose during the initial trial demonstrated statistically significant improvements in visual acuity after 1 year. Additionally, over the same 5 years, patients reported stable or improved vision-related quality of life.

The company defines the MCO platform as a “one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration.” MCO-010 has been granted Fast Track and Orphan Drug designation by the US FDA, and a rolling Biologics License Application (BLA) is currently underway.

Samarendra Mohanty, PhD, president and chief scientific officer of Nanoscope, commented on the results in a press release from the company.¹

“These results demonstrate the excellent long-term safety and tolerability of MCO-010, even five years after a single injection. The absence of serious safety signals and strong participant retention underscore the potential of MCO-010 as a durable, non-invasive therapy for severe vision loss RP patients,” stated Mohanty.

Recently, Nanoscope Therapeutics released positive 3-year follow-up data from its REMAIN study, the long-term extension of the phase 2b/3 RESTORE trial (NCT04945772) evaluating MCO-010 in retinitis pigmentosa (RP).²

According to the company, results from the REMAIN study showed patients maintained an average best corrected visual acuity (BCVA) gain from baseline of approximately 0.3 LogMAR through week 152. Additionally, BCVA-Area Under the Curve (AUC) profiles across both the RESTORE and REMAIN trials show five times greater vision gains when compared to sham treatment.²

References:

1. Nanoscope Therapeutics reports five-year safety results from phase 1/2a follow-up study of MCO-010 optogenetic therapy in retinitis pigmentosa. Published November 4, 2025. Accessed November 4, 2025. https://nanostherapeutics.com/2025/11/04/nanoscope-therapeutics-reports-five-year-safety-results-from-phase-1-2a-follow-up-study-of-mco-010-optogenetic-therapy-in-retinitis-pigmentosa/

2. Nanoscope Therapeutics releases positive 3-year follow-up data from REMAIN study. Published October 24, 2025. Accessed November 4, 2025. https://www.ophthalmologytimes.com/view/nanoscope-therapeutics-releases-positive-3-year-follow-up-data-from-remain-study