DALLAS — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) has selected “sonpiretigene isteparvovec” for the nonproprietary name of the Company’s MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP) and Stargardt disease. MCO-010 has been granted orphan drug designation by the US Food and Drug Administration for both diseases and multiple mid- to late-stage clinical trials are ongoing, including the Company’s Phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial for RP. Top line data from RESTORE are expected in Q1 2023.
The name “sonpiretigene isteparvovec” for MCO-010 was selected by the WHO Expert Advisory Panel of the International Pharmacopoeia and Pharmaceutical Preparations. MCO-010 reached the status of recommended INN after a period of public consultation and was included in the INN Recommended List 128, published in the No. 4 issue of the WHO Drug Information, Volume 36 in January 2023.
Dr. Samarendra Mohanty, President of Nanoscope, said “The WHO’s INN system has facilitated the name of sonpiretigene isteparvovec for the active pharmaceutical ingredient, which is the foundation of our clinical development in several retinal degenerative diseases with high unmet need. We believe that finalizing this name is an important step towards potential future commercialization of MCO-010.”
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company’s lead asset, MCO-010, is presently in RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.