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Nanoscope Therapeutics Advances Regulatory Path for MCO-010 in Retinitis Pigmentosa Treatment

Nanoscope Therapeutics Advances Regulatory Path for MCO-010 in Retinitis Pigmentosa Treatment

January 25, 2024

Nanoscope Therapeutics has provided update on the key developments arising from regulatory discussions with the U.S. Food and Drug Administration (FDA) and a scientific advice meeting with the Icelandic Medicines Agency (IMA) regarding the regulatory pathway for MCO-010, an optogenetic gene therapy for the treatment of retinitis pigmentosa (RP).

Following the Type B end-of-phase 2 discussion with the FDA, Nanoscope received valuable feedback on its MCO-010 optogenetic gene therapy program designed for patients experiencing severe vision loss due to advanced RP. The FDA emphasized that a change from baseline in visual acuity among low vision patients could serve as an appropriate primary efficacy assessment in an adequately controlled study, providing substantial evidence to support Biologics License Application (BLA) approval. It is noteworthy that MCO-010 has already been granted orphan drug and fast track designations by the FDA.

Simultaneously, Nanoscope also engaged in a Scientific Advice meeting with the IMA, a crucial step in the approval process for MCO-010 in Europe. The IMA endorsed visual acuity as the suitable primary endpoint for evaluating low-vision RP patients post MCO-010 treatment. Additionally, the IMA expressed support for considering a 0.3 logMAR change as clinically meaningful for RP patients with severe vision loss, and suggested Nanoscope explore conditional approval for MCO-010 in Europe based on existing data.

In response to these regulatory discussions, Nanoscope has now adjusted the primary endpoint in the ongoing randomized, double-masked, multicenter Phase 2b study named RESTORE. Visual acuity will now be the primary endpoint, and the end-of-study 100-week data is anticipated to be reported in the first half of 2024.

Sulagna Bhattacharya, Co-founder, and CEO of Nanoscope, expressed optimism about the recent regulatory developments, stating, “Consistent input from both the FDA and IMA on the potential for visual acuity to serve as the primary endpoint for the clinical program is a major milestone for Nanoscope and provides MCO-010 with a clear regulatory path in the US and in Europe.”

About MCO-010

MCO-010, also known as sonpiretigene isteparvovec, is a suspension designed for intravitreal injection as part of Nanoscope's ambient-light activatable MCO optogenetic monotherapy. This gene therapy targets inner retinal neurons and holds the promise of restoring vision permanently lost due to advanced RP.

MCO-010 is unique as it is the only broadband, fast, and most-light sensitive opsin currently undergoing clinical trials. The phase 1/2 study of MCO-010 in advanced RP patients demonstrated favorable safety and dose-dependent improvement in visual acuity. The ongoing Phase 2b RESTORE trial has shown nominally significant visual acuity improvements and functional vision enhancements, along with a favorable safety profile, offering hope for individuals suffering from RP.