Afraxis will partner with the NIH to advance work on the San Diego company’s disease-modifying treatments for Fragile X syndrome, a rare genetic disorder that is the most common inherited form of mental retardation, according to a statement from the company today. The biotech’s lead program targets p21-activated kinase (PAK), a protein that regulates the development and activity of neural structures called dendritic spines. Recent scientific discoveries have linked Fragile X, Alzheimers, and other disorders to underlying defects in the development and function of dendritic spines.
Afraxis, which has been fully funded by San Diego’s Avalon Ventures, says potential therapies arising from the work could actually modify the disease rather than just treat symptoms.
The NIH Therapeutics for Rare and Neglected Disease (TRND) program is a Congressionally mandated program intended to encourage and speed the development of new drugs for rare and neglected diseases that might otherwise be ignored by industry.
TRND collaborations help early stage companies overcome one of the biggest obstacles facing drug development, which is crossing the “valley of death” that determines whether a promising compound also holds promise as a clinical therapeutic. “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,” says Jay Lichter, an Avalon partner and Afraxis CEO, in a statement released by the company.