Ocugen Receives Fourth FDA Orphan Drug Designation for the Same Product, OCU400 (AAV-NR2E3) Gene Therapy, for the Treatment of Another Key Inherited Retinal Disease (IRD), PDE6B Gene Mutation-Associated Retinal Diseases

Fourth orphan drug designation for the same product, OCU400, is distinct in Ophthalmology gene therapy and demonstrates its potential to treat many Inherited Retinal Diseases

MALVERN, Pa., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the fourth Orphan Drug Designation (ODD) for OCU400 in the treatment of PDE6B gene mutation-associated retinal diseases. Retinitis Pigmentosa (RP) caused by PDE6B mutation is an inherited retinal dystrophy that leads to blindness by midlife and is characterized by the progressive loss of photoreceptors, with or without the loss of retinal pigment epithelium cells. At least one mutation in the PDE6B gene has been found to cause autosomal dominant congenital stationary night blindness, which is characterized by the inability to see in low light.

Ocugen’s Modifier Gene Therapy Platform offers a unique approach in ophthalmology by addressing multiple diseases with a single product. A novel gene therapy product candidate, OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (NHR) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector.  As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis and potentially offer longer benefit, stabilizing cells and rescuing photoreceptor degeneration and vision loss. In pre-clinical studies, OCU400 has demonstrated improved vision signals in the retina where Electroretinogram response reveals rescue under both Scotopic (dim-lit) as well as Photopic (well-lit) conditions. The Company believes targeting multiple diseases with one product could also offer a smoother regulatory pathway and the ability to recover development costs over multiple therapeutic indications. Ocugen is planning to initiate two parallel Phase I/II clinical trials next year targeting two unique IRDs.