Amarantus Biosciences, Inc., a development-stage biotechnology company, announced its intentions of pursuing an orphan drug designation with the FDA. The company’s lead therapeutic drug, MANF, facilitates proper protein folding and processing in the endoplasmic reticulum. With a focus on developing technologies to treat brain disorders such as Parkinson’s disease and Traumatic Brain Injuries, Amarantus seeks to exploit MANF’s unique abilities and possible role in improving patient outcomes.
In order for a product to receive the orphan drug designation, it must treat a rare condition at the request of a sponsor and meet specific criteria. The sponsor qualifies for certain benefits, such as reduced taxes and marketing incentives.
Recently, Amarantus proved its superior effectiveness over its competition, GDNF, in a study with a rat model of Parkinson’s disease. Researchers injected 6-OHDA into the rats’ brains, causing Parkinson’s-like behavioral symptoms. Two weeks later, one group of rats received 10 micrograms of MANF and the other received the same amount of GDNF. In the MANF group, rats reduced behavioral deficits by 43 percent after four weeks and by an additional 10 percent after six weeks. GDNF reduced behavioral deficits by 16 percent and 20 percent, respectively.
The company expressed their satisfaction with the positive results after years of research. President and CEO of Amarantus, Gerald Comissiong, stated that the company believes the data will allow them “to attract the interest of investment firms and potential partners who will be able to now characterize the substantial opportunity our technology represents.”
MANF-based products as treatments for brain disorders could be crucial to patient improvement. Protein misfolding plays a significant role in a variety of human diseases, such as Parkinson’s and Alzheimer’s. Although Amarantus has been focusing on MANF’s potential to treat Parkinson’s disease, the company will explore the protein’s utility in treating diseases that have a much smaller population.
The Orphan Drug Strategy could accelerate commercialization by identifying therapeutic applications for MANF that would require a few clinical trials. But MANF’s recent positive data from the Parkinson’s study is validating Amarantus’ approach. Commissiong notes the wide range of MANF’s abilities “to mediate protein folding in an extracellular fashion, making it potentially an ideal biologic drug candidate for a wide range of human conditions.”