Two year clinical trial

In this two-year clinical trial, called RESTORE, two doses of MCO-010 were compared to a sham treatment in 27 people with RP. A sham treatment is also known as a placebo or control treatment, which does not contain the active ingredient, and provides a comparison to help evaluate the efficacy of the MCO-010 in improving vision for patients with RP.

Originally, the trial was testing the treatment’s effectiveness using the Multi-Luminance Mobility Test, which is a relatively new way of assessing improvements in navigation after treatment. MLMT results were assessed at week 52, but it did not show clear results.

Change to the primary endpoint ensures success

In January 2024, Nanoscope changed the primary endpoint of this Phase IIb trial back to a more traditional test of best-corrected visual acuity (BCVA), a vision test that measures how well people can see with the best possible correction, usually glasses or contact lenses.

The company reported in late March 2024 that both doses of MCO-010 led to significant improvements in visual acuity at week 52. The low dose failed to beat the control at week 76, however the high dose did show significantly better results than the control.

It is worth noting that the people who received this therapy had very poor vision at baseline (light perception only), and the improvement in their vision would still not allow them to read letters on a visual acuity chart. However, it has restored form vision, and given them a significant improvement in sight.

Nanoscope presented the results of their trial at the recent Association for Research in Vision and Ophthalmology (ARVO) meeting in Seattle, USA, on 6 May 2024. Friend of Retina Australia, Associate Professor Lauren Ayton from the Centre for Eye Research Australia and University of Melbourne, was in the audience and was impressed with the results.

Lauren commented, “While the treatment is not yet able to restore vision to the stage where people can read letters on a chart, the results are very exciting. One of the most interesting outcomes for me is that people were able to perceive colours with the treatment. For example, one of the recipients of the therapy was then able to see and detect the colours of the handholds he used at rock-climbing, which he had not previously been able to do.”

Hope for people living with RP for a new treatment

Nanoscope is now planning to submit for approval from the Food and Drug Administration (FDA) in the USA for their treatment later this year. If approved, MCO-010 could be a groundbreaking treatment for RP, offering hope for those with this condition.