- FDA and EU Regulators provide consistent advice that visual acuity may serve as primary endpoint to evaluate the effectiveness of MCO-010 in the treatment of low-vision retinitis pigmentosa (RP) patients
- Regulatory feedback provides clarity for potential path to approval in the US and in Europe for MCO-010 in RP; Nanoscope preparing for immediate execution
DALLAS, Jan. 18, 2024 /PRNewswire/ -- 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. About Retinitis Pigmentosa
Retinitis Pigmentosa (RP) encompasses a group of rare genetic disorders in which the retina’s photoreceptor cells degrade over time, leading to impaired vision and eventual blindness. These disorders are believed to be linked to over 100 different gene mutations. Approximately 100,000 people in the U.S. and an estimated 2 million people worldwide suffer from RP, making it the leading cause of inheritable blindness. About MCO-010
Current gene therapies aim to treat patients with specific gene mutations, and are further limited in advanced disease with degenerated outer retina cells. Ambient-light activatable MCO optogenetic monotherapy targets abundant inner retinal neurons and has the potential to restore vision permanently lost due to advanced RP. MCO-010 (sonpiretigene isteparvovec, suspension for intravitreal injection) is the only broadband, fast, and most-light sensitive opsin currently in clinical trials. With bipolar cell targeting via mGluR6 promoter-enhancer, the MCO-010 expression cassette is designed for restoring high quality vision in real-world environments. The proprietary AAV2 vector allows robust transduction of MCO-010 in bipolar cells upon intravitreal injection. The Phase 1/2 study of MCO-010 in advanced RP patients demonstrated favorable safety and dose-dependent improvement in visual acuity. Nominally significant visual acuity improvements in MCO-010 treated patients were observed in comparison to control in Phase 2b RESTORE trial. There were also nominally significant functional vision improvements as assessed by multi-luminance vision-guided mobility and shape discrimination, along with a favorable safety profile. About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126 ). MCO-010 has received FDA fast track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy. Investor Contact:
Argot Partners
212-600-1902
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