Eyevensys Receives FDA Orphan Drug Designation for EYS611 for Treatment of Retinitis Pigmentosa

Eyevensys announced that the FDA has granted an orphan-drug designation for EYS611 for the treatment of retinitis pigmentosa (RP).

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 (AMD) 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.

“We are delighted to have received orphan-drug designation from the FDA as it is an important regulatory milestone. We look forward to translating our unique non-viral gene therapy program to patients with RP, to slow the progression of this degenerative retinal disease with no currently approved treatment that compromises patients’ vision and eventually lead to blindness,” Thierry Bordet, PhD, Chief Scientific Officer, said in a company news release.

“This orphan-drug designation acknowledges the unmet needs of individuals suffering from RP, and this opportunity to move our therapy through the development process with this designation is an encouraging milestone. With EYS611, we are optimistic we will advance a therapeutic option for all patients with RP independent of the underlying genetic mutation that is much less invasive and can be used to treat patients at an earlier stage of disease than traditional viral vector gene replacement therapies that target only the macula and are being developed only for a select handful of RP patients with specific mutations,” said Ronald Buggage, MD, Eyevensys’ Chief Medical Officer.

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 U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.