Amarantus Submits FDA Orphan Drug Application For MANF In Retinitis Pigmentosa

Amarantus Bioscience has applied to the Food and Drug Administration (FDA) for Orphan Drug designation for MANF (mesencephalic-astrocyte-derived neurotrophic factor) in retinitis pigmentosa (RP). Amarantus announced the application in a press release, stating that MANF was discovered with its proprietary PhenoGuard Protein Discovery Engine. RP is receiving special interest of late, as Astellas and Harvard have recently partnered to research treatments for RP, a disease that causes a loss of vision resulting in blindness. If approved, Orphan Drug status confers seven-year marketing exclusivity and other benefits.

MANF in Retinitis Pigmentosa

Mesencephalic-Astrocyte-derived Neurotrophic Factor (MANF) is a naturally-occurring protein. The body produces MANF to reduce or prevent programmed cell death (apoptosis) in response to injury or disease. Amarantus and the University of Miami's Bascom Palmer Eye Institute and the Buck Institute for Research on Aging have proven MANF’s efficacy in animal models of retinitis pigmentosa. Toxicology studies show that intravitreal administration is well-tolerated.

PhenoGuard Protein Discovery Engine

MANF was developed with Amarantus’ PhenoGuard Protein Discovery Engine, a regenerative medicine approach. The therapeutic platform identifies novel secreted human proteins with biological activity for specific indications. MANF was the first discovery, made from astrocytes, and Amarantus now has over 88 candidates in its PhenoGuard Cell Line Library. Amarantus seeks to expand its capabilities beyond RP to other indications, including glaucoma, Parkinson's disease, Alzheimer's disease, and Wolfram's Syndrome. The company is also evaluating traumatic brain injury (TBI), myocardial infarction, diabetes, antibiotic-induced ototoxicity, and other areas with high unmet need.

Astellas, Harvard Partnership

Astellas and Harvard are taking a different approach in searching for treatments for RP. The two have partnered to use adeno-associated virus vectors (AAV) to identify genes that support vision. AAV vectors show promise due to their ability to infect non-dividing and dividing cells. Constance L. Cepko, Ph.D., a professor of Genetics and Ophthalmology at Harvard, said, “These treatments may also be effective in people that have other types of ocular disease, such as age-related macular degeneration, or glaucoma."