A mutation-agnostic gene therapy for retinitis pigmentosa has achieved “clinically meaningful vision improvement in legally blind individuals with progressive and permanent neurodegeneration of the retina”.

Announcing the positive top-line results in the RESTORE trial of MCO-010, Nanoscope Therapeutics said it expected to submit an application to the United States Food and Drug Administration this year, as the first step in executing its global regulatory strategy.

Nanoscope Therapeutics said MCO-010 achieved its primary and key secondary endpoints with statistical significance and no serious adverse events. The results represent the first evidence of effectiveness of a mutationagnostic gene therapy for a genetic disease.

Nanoscope Therapeutics said there was a statistically significant improvement of bestcorrected visual acuity (BCVA) at week 52 in both the high-dose (0.337 LogMAR; p=0.021) and low-dose (0.382 LogMAR; p=0.029) treatment groups compared with the sham control group (0.050 LogMAR ).

PERSISTENT IMPROVEMENT

The Phase 2b RESTORE trial represents the only randomised controlled trial in retinal degenerative disease to demonstrate improvement beyond the clinically important BCVA > 0.3 LogMAR threshold in a statistically significant manner.

Improvements in visual function persisted or increased following week 52 in the study, demonstrating the durable effect of a single intravitreal injection of MCO-010.

BCVA improvement at week 76, a key secondary endpoint, was statistically significant in the highdose treatment group compared to the control group (0.539 LogMAR; p=0.001). At week 76, the improvement in BCVA in the low-dose treatment group was not statistically significant compared to control (0.374 LogMAR; p=0.065).

These results are consistent with what has been previously observed in the earlier Phase 1/2a open-label study, the company said. The highdose MCO-010 (1.2E11gc/eye) is planned to be the commercial dose.

Additional data from this clinical trial will be presented in a series of scientific meetings in the United States in coming months.

NO ADVERSE EVENTS

Nanoscope Therapeutics said MCO-010 was generally well tolerated with no treatment-related serious or severe adverse events reported, consistent with prior studies. The most common adverse events were mild or moderate anterior chamber cell and ocular hypertension. No adverse events of special interest related to intraocular inflammation, such as endophthalmitis, retinitis, retinal vasculitis, retinal occlusive vasculitis, or hypotony, were reported in the treatment groups.

“We observed significant vision restoration in many patients with severe vision loss, including those who were completely blind,” said Dr David Boyer an investigator in the trial and Adjunct Clinical Professor of Ophthalmology at the University of Southern California Keck School of Medicine.

“Many patients treated with MCO-010 derived a clinically meaningful benefit measurable on the primary visual function test, and this effect was confirmed by a parallel improvement in functional vision assessments.

“If approved, MCO-010 is poised to make a positive, meaningful impact on the lives of patients affected by this debilitating condition.”

Retinitis pigmentosa encompasses a group of rare genetic disorders in which the retina’s photoreceptor cells degrade over time, leading to impaired vision and eventual blindness. An estimated two million people worldwide suffer from retinitis pigmentosa, making it the leading cause of inheritable blindness.

Nanoscope Therapeutics is a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degeneration.

The company’s co-founder, President and Chief Scientific Officer, Dr Samarendra Mohanty said the results were the “culmination of more than a decade of work”.