Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today provided an update following recent regulatory discussions with the U.S. Food and Drug Administration (“FDA”) and from the Scientific Advice meeting with the Icelandic Medicines Agency (“IMA”), regarding the regulatory path of MCO-010 for the treatment of RP.
In connection with the productive Type B End-of-Phase 2 discussion with the FDA, Nanoscope received feedback on its MCO-010 optogenetic gene therapy program in patients with severe vision loss from advanced RP. The FDA stated that change from baseline in a measure of visual acuity in low vision patients could be an appropriate primary efficacy assessment in an adequate and well-controlled study to provide substantial evidence of benefit to support BLA approval. MCO-010 has received both orphan drug and fast track designations from the FDA.
Nanoscope also received positive feedback from a Scientific Advice meeting with the IMA as part of the approval path for MCO-010 in Europe.
The IMA endorsed visual acuity as the appropriate primary endpoint to evaluate low-vision RP patients after MCO-010 treatment.
The IMA also endorsed 0.3 logMAR change as being clinically meaningful for the severe vision loss RP patients.
The IMA suggested Nanoscope explore conditional approval for MCO-010 in Europe based on the existing data.
Based on regulatory discussions, Nanoscope has reverted to visual acuity as the primary endpoint in the ongoing randomized, double-masked, multicenter phase 2b study (RESTORE). The end-of-study, 100-week data are expected to be reported in H1 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 in Europe,” said Sulagna Bhattacharya, co-founder, and CEO of Nanoscope.