Nanoscope's phase 2 win for gene therapy shows potential of light-sensitive approach to eye disease

Nanoscope has scored a phase 2 win for its gene therapy using light-sensitive molecules to treat retinal disease, keeping alive the hope of a first treatment to improve vision in these patients.

The therapy, dubbed MCO-010, hit the primary endpoint in a trial of 27 patients with advanced retinitis pigmentosa (RP) and severe vision impairment, irrespective of gene mutation. Of the 18 patients injected with MCO-010, 16 (89%) demonstrated an improvement of two or more levels across two visual tests at 12 months, compared to four of the nine (44%) patients who received placebo. The company said the results show that MCO-010 led to vision function improvements.

Specifically, 12 of the MCO-010 patients saw an improvement by two or more luminesce levels when measured by the multi-luminance mobility test, in which participants navigate obstacles in varying degrees of darkness. Only three of the placebo patients achieved this.

Meanwhile, 10 of the MCO-010 patients saw an improvement of two or more luminance levels in the multi-luminance shape discrimination test, compared to two of the placebo cohort.

MCO-010 was well tolerated with no serious or severe ocular or systemic adverse events reported, the biotech pointed out in the release. There was a similar incidence of treatment-emergent adverse events across both arms of the study, with the most common events being blood cells appearing in the anterior chamber of the eye, high eye pressure and conjunctival hemorrhage.

MCO-010, which has already secured fast-track and orphan tags from the FDA, is an ambient-light activatable multi-characteristic opsin (MCO) optogenetic therapy. Unlike other gene therapies, which are aimed at a specific gene mutation in the outer retinal cells, Nanoscope’s pitch is that its therapy targets “abundant” inner retinal neurons to restore vision lost as RP progresses.

The therapy is also undergoing a phase 2 trial in another vision loss condition called Stargardt disease, with a six month readout expected in this half of the year. In preclinical development, Nanoscope has MCO-020, a non-viral laser-delivered gene therapy for geographic atrophy.