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Nanoscope Therapeutics Inc., provides a commercial update on  MCO-010 treatment of retinitis pigmentosa (RP) using optogenetic therapy. 

Nanoscope Therapeutics Inc., a company based in Bedford, Texas, has 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. “MCO-010” (sonpiretigene isteparvovec) is an ambient-light activatable optogenetic therapy to treat advanced retinitis pigmentosa (RP). In their recent trial, with a single intravitreal injection of MCO-010, previously results showed vision function improvements after treatment were “consistent with previous studies as well as a favorable safety profile”. According to the company on a recent update, “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”.

According to the clinical trial study, the experimental MCO-010 treatment is an adeno-associated virus (AAV) serotype 2-based vector carried an opsin gene expression cassette, aimed to deliver and express genetically encoded light-sensitive proteins (opsins) to remaining light-insensitive neurons in the inner retina of patients with RP, RP patients for which the window of therapeutic opportunity for ocular genetic therapies is surpassed. Optogenetic monotherapy targets abundant inner retinal neurons has the potential to restore vision lost due to advanced RP. Optogenetic therapy can theoretically be applied to all patients with end-stage RP.  In addition to their previous efficacy test results reported, the company stated that there were no serious or severe ocular or systemic adverse events and the results were consistent with those observed in an earlier Phase 1/2 trial (NCT04919473) in which 9 of 11 (82%) of subjects demonstrated 2 luminance level improvements in vision-guided mobility or 0.3 logMAR in visual acuity.

On the recent commercial and regulatory update,  Nanoscope also received positive feedback from a Scientific Advice meeting with the IMA as part of the approval path for MCO-010 in Europe including:

  • 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.

In follow up to their announcement, Sulagna Bhattacharya, co-founder, and CEO of Nanoscope commented that, “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”.