5 October 2020 - - The US Food and Drug Administration has granted an orphan-drug designation for EYS611 for the treatment of retinitis pigmentosa, France- and US-based biotechnology company Eyevensys said.

Eyevensys is developing EYS611, a DNA plasmid that encodes for the human transferrin protein, to benefit patients diagnosed with RP, as well as other degenerative retinal diseases, including late stage, dry age-related macular degeneration and glaucoma.

Transferrin is an endogenous protein that helps manage iron levels in the eye.

While iron is essential for retinal metabolism and the visual cycle, excessive iron can induce oxidative stress and is extremely toxic to the retina.

Iron overload has been associated with photoreceptor death in several retinal degenerative diseases.

By acting as an iron chelating and neuroprotective agent, EYS611 helps slow the progression of diseases like RP regardless of the specific genetic mutation causing the condition.

Eyevensys just reported data from preclinical testing in the September 2020 issue of the journal Pharmaceutics.

The paper, entitled "Transferrin non-viral gene therapy for treatment of retinal degeneration" (Bigot, et al., Pharmaceutics), shows that EYS611 is safe and effective for preserving photoreceptors and retina functionality in acute toxicity and inherited rat models of retinal degeneration.

The FDA's Orphan Drug Designation Program provides orphan status to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

Eyevensys is a privately held, clinical-stage biotechnology company developing its innovative technology to enable the sustained intraocular production of therapeutic proteins to treat a range of ophthalmic diseases.

The Eyevensys technology, developed by Pr. Francine Behar-Cohen, uses electroporation to deliver proprietary DNA plasmids encoding therapeutic proteins into the ciliary muscle of the eye.

This approach induces the sustained intraocular production of therapeutic proteins.

Eyevensys' lead product EYS606 is a potential new treatment for patients with chronic non-infectious uveitis.

EYS606 combines Eyevensys' proprietary Electrotransfection System with plasmids encoding for the production of a potent fusion protein which neutralizes the activity of TNFα, a cytokine that has been shown to play a pivotal role in mediating intraocular inflammation in NIU.

EYS606 is currently in a phase I/II clinical trial in the EU and has been granted an Orphan drug designation by the European Medicines Agency for the treatment of NIU.

The therapeutic potential of EYS606 in patients with active, chronic NIU is being further investigated in Part 2 of the ongoing EYS606-CT1 study in the EU and in a second phase 2 trial, the Electro Study (EYS606-CT2) being conducted in the US.

Additionally, Eyevensys is developing EYS611, a treatment for Retinitis Pigmentosa and potentially other retinal degenerative conditions including the later stages of Dry AMD and glaucoma.

The treatment encodes for a potent iron chelator with antioxidant and endogenous neuroprotective properties.

In animal models, the treatment has been shown to be safe and effective at preserving and slowing the degeneration of retinal structure and function.

Eyevensys is also advancing a dual gene plasmid, EYS809, expressing two therapeutic proteins for wet AMD, diabetic macular edema, and central retinal vein occlusion, and it is exploring further compounds for undisclosed indications.

Eyevensys was founded in 2008. The company has offices in Paris, France and the US. The company is funded by the Boehringer Ingelheim Venture Fund, Pureos Bioventures, Bpifrance through the Innobio Fund, CapDecisif, Inserm Transfert Initiative, Pontifax and the Global Health Sciences Fund.