NEW YORK, NY--(Marketwire - Jul 16, 2012) - Phoenix Nest announces the launch of its virtual biotech, with plans to take advantage of the new legislation enacted by Congress and signed into law on July 9, 2012, The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187. The law will empower the FDA to use all the science available for allowing surrogate endpoints in clinical trials for rare diseases to determine whether a drug is working, significantly decreasing the development time and cost.
Phoenix Nest was created to find treatments for the ultra rare pediatric disease Sanfilippo Syndrome, with emphasis on subtype C. Sanfilippo Syndrome is caused by a genetic defect, resulting in the body's inability to break down a complex sugar molecule. After a period of normal development, the child will start to decline. As the disease progresses, children become unable to eat or walk on their own and die before adulthood.
With the new legislation just signed into law, biotechs focused on rare and ultra rare diseases have a much better chance of getting treatments to clinical trial in a timely manner. Currently, fewer than 400 FDA-approved treatments exist for nearly 7,000 rare diseases, half of which affect children. With access to the Accelerated Approval pathway, interest and development will surge for rare disease treatments.
"The previous law required the FDA to ask for clinical data -- data that isn't readily available for ultra rare diseases. Now we can use surrogate endpoints, data that is taken from our animal models," says CEO and Sanfilippo parent, Jill Wood.
Phoenix Nest was built through collaboration with leading academics and experienced researchers -- a formidable team focused on creating a treatment for Sanfilippo Syndrome. "All of our resources are spent on research and development with minimal overhead," states Chief Scientific Officer, Sean Ekins, Ph.D.
Phoenix Nest is a privately held company, created to find treatments for the ultra rare pediatric disease Sanfilippo Syndrome. Phoenix Nest was founded by these Sanfilippo medical research foundations: Jonah's Just Begun, JLK-Sanfilippo Research Foundation, Sanfilippo Sud and Sanfilippo Barcelona. The name Phoenix Nest comes from the mythological bird that made its nest from frankincense, myrrh and spices. In essence, the bird's nest is a pharmacy; and the virtual biotech, Phoenix Nest, wants to provide a place where parents can find treatments for their sick children.