Afraxis to participate in NIH's Therapeutics for Rare and Neglected Disease Program

Sep 27 2011

Afraxis, a San Diego-based biotechnology company developing drugs to treat rare genetic diseases through the modulation of p21-activated kinase (PAK), announced today that it has been selected to participate in the National Institutes of Health's (NIH) Therapeutics for Rare and Neglected Disease (TRND) Program. As part of the program, Afraxis will partner with the NIH to pursue the ongoing development of the company's disease-modifying treatments for Fragile X syndrome. Fragile X syndrome is a rare genetic disorder that is the most common inherited form of mental retardation.

"TRND collaborations help companies focusing on rare and neglected diseases overcome one of the biggest obstacles facing all drug development companies: advancing programs through that 'valley of death' between identification of a promising compound, and clinical stage development when a program becomes more attractive to a big pharma partner," said Jay Lichter, Ph.D., president and CEO of Afraxis. "This is a tremendous opportunity for Afraxis as the NIH is providing access to state-of-the-art laboratory facilities, the expertise of collaborating NIH scientists, and the financial resources to continue our research and development efforts through the initiation of human clinical trials."

Afraxis is developing disease-modifying treatments for Fragile X syndrome and other central nervous system disorders based on a leadership position in the understanding of PAK biology. Fragile X symptoms can range from learning disabilities to more severe cognitive or intellectual disabilities, such as mental retardation. Fragile X is the most common known cause of autism or "autistic-like" behaviors. The disease is caused by a mutation in the FMR gene on the X chromosome and effects approximately one in 3,600 males and one in 4,000 to 6,000 females.

The TRND program is designed to encourage the development of drugs for rare and neglected diseases. The program aims to speed development of new drugs that might otherwise be ignored by industry because they are too early in the development process where the failure rates are high. To make a potential compound attractive to private partners in order to get a treatment to patients, TRND advances a compound through the drug development process to the point where it can be tested in humans. TRND provides all aspects of preclinical development needed to turn promising compounds into potential medicines ready for clinical evaluation. It is anticipated that the average TRND project will take approximately three years.

"TRND collaborates with researchers and companies with an aim of translating promising molecules into potential new drugs for rare and neglected diseases," said NHGRI's John McKew PhD, Chief of TRND's Therapeutic Development Branch. "This is a difficult phase of drug development associated with high failure rates for any molecule. The outcome can be uncertain."