Eyevensys S.A.S., a biotech company, founded by Professor Francine Behar-Cohen and colleagues, has announced the receipt of Orphan Drug Designation from the European Medicines Agency for its lead product, EYS606, developed for the treatment of non-infectious uveitis. The experimental product consists of an engineered gene transcript coding for soluble TNFα a receptor loaded into an electrotransfer delivery device for direct elctroporation into the cilary muscle. The company claims that their non-viral strategy can deliver steady, low level therapeutic protein to the eye for up to a year following a single injection. The company aims to initaite a Phase I clinical trial before June, FY2016.
The platform developed by Eyesensys employs electroporation technology to deliver engineered plasmids devoid of bacterial sequence and encoding specific therapeutic proteins. While the immediate target indication is non-infectious uveitis the company plans to develop similar long-lasting approaches for AMD and diabetic retinopathy in order to provide an alternative to monthly injections of anti-VEGF antibodies. Eyevensys’ TNFα product provides for selective expression and quenching of cell signaling in the cilary muscle to treat the uveitis. The cilary muscle expresses the plasmid encoding TNFα and secretes the protein into the vitreous humor where it can bind its target and thereby interrupt angiogenesis caused by VEGF signaling or inflammation. According to the company, non-infectious intermediate and posterior uveitis represents between 10%-15% of uveitis cases, and is the 4th cause of blindness, affecting an estimated 250,000 patients in the US and Europe.
Orphan Drug Designation is available in both the US and the European Union for drugs or biologics that treat a life-threatening or chronically debilitating rare disease affecting fewer than 200,000 patients in the US or a prevalence not exceeding five in 10,000 individuals in the EU. Many companies seek the designation to add value to a potential product over its commercial lifespan. Commenting on the regulatory milestone Raffy Kazandjian, CEO of Eyevensys stated, “We’re pleased to announce that EMA’s Committee for Orphan Medicinal Products has issued on the 21st of January 2016 a positive opinion on orphan designation for EYS606 in the treatment of non-infectious uveitis. This confirms the potential of the Ciliary Electro-Transfection technology to offer patients suffering from severe ophthalmic diseases an enduring therapeutic solution”.