MCO-010 Gene Therapy for Retinitis Pigmentosa Receives Regulatory Feedback


Both the FDA and EU regulators indicated visual acuity may serve as primary endpoint to evaluate MCO-010 in the treatment of low-vision retinitis pigmentosa patients.

Credit: V2osk/Unsplash

Credit: V2osk/Unsplash

Nanoscope Therapeutics, Inc. has provided an update on the regulatory path of MCO-010, an ambient-light activatable Multi-Characteristic Opsin optogenetic therapy designed for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation.1

Announced on January 18, 2024, the biotechnology company received feedback on its MCO-010 optogenetic gene therapy program for patients with severe vision loss from advanced retinitis pigmentosa in connection with a productive discussion with the US Food and Drug Administration (FDA).

The Type B End-of-Phase 2 discussion led the federal health agency to declare the change from baseline in visual acuity among low vision patients could be an appropriate primary efficacy endpoint in an adequate and well-controlled study to provide substantial evidence of benefit to support approval of the drug application.

Nanoscope also received consistent advice from a Scientific Advice meeting with the Icelandic Medicines Agency (IMA) as part of the approval pathway for MCO-010 in Europe. The European agency endorsed visual acuity as the appropriate primary endpoint to evaluate the effectiveness of MCO-010 in the treatment of low-vision retinitis pigmentosa patients.

Owing to these regulatory discussions, Nanoscope has determined visual acuity will serve as the primary endpoint in the ongoing randomized, double-masked, multicenter Phase 2b RESTORE study. End-of-study data at week 100 is expected to be reported in the first half of 2024.

“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 Europe,” Sulagna Bhattacharya, co-founder, and chief executive officer of Nanoscope, said in a statement.1

Retinitis pigmentosa has been linked to over 100 different gene mutations and affects approximately 100,000 people in the US and an estimated 2 million people globally. Current gene therapies target specific gene mutations and are limited in advanced diseases with degenerated outer retina cells.

Nanoscope has indicated MCO-010 is the only broadband, fast, and most light-sensitive opsin currently in clinical trials. Given its bipolar cell targeting via MGluR6 promoter-enhancer, the MCO-010 expression cassette is designed to restore high-quality vision in real-world environments. The proprietary AAV2 vector allows robust transduction of MCO-010 in bipolar cells after intravitreal injection.

Positive topline results reported from the RESTORE trial showed the optogenetic therapy led to nominally significant visual acuity improvements in MCO-010 treated patients were observed in comparison to the control cohort.2 Results also showed nominally significant functional vision improvements assessed by multi-luminance vision-guided mobility and shape discrimination, and a favorable safety profile, with MCO-010 treatment.

MCO-010 has received both orphan drug and fast track designations from the FDA.

  1. BioSpace. Nanoscope therapeutics provides regulatory update on MCO-010 for the treatment of retinitis pigmentosa. BioSpace. January 18, 2024. Accessed January 18, 2024.
  2. Therapeutics N. Nanoscope therapeutics announces positive topline results from phase 2B restore trial of MCO-010 for the treatment of retinitis pigmentosa - nanoscope therapeutics. Nanoscope Therapeutics -. March 30, 2023. Accessed January 18, 2024.
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