NIH buddies with Afraxis to escort Fragile X program through "valley of death"

Tools

San Diego-based Afraxis is getting some key support from the National Institutes of Health as it journeys through the dreaded "valley of death" with an experimental program for Fragile X syndrome--a rare genetic disease linked with autism.  

The NIH's Therapeutics for Rare and Neglected Disease Program announced on Monday that Afraxis was one of a handful of biotech companies that had made the cut, putting it in line for some of the millions it has on hand to back treatments for rare diseases. As a result, Afraxis will gain the support of NIH scientists and some of the state-of-the-art lab space that can help out a fledgling biotech.

Afraxis's lead program targets p21-activated kinase, a strategy that essentially looks to bolster the dendritic spine of neurons. The inspiration for the company came from Nobel Prize-winning scientist Susumu Tonegawa, who noted that correcting the abnormalities in dendritic spines also corrected behavioral abnormalities in mice, according to an article in BioWorld. And that in turn inspired Avalon Ventures's Jay Lichter to launch the biotech back in 2007.

Xconomy reported last year that Avalon had invested some $6 million in the biotech.

"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, 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."

- here's the release

Special Report: Rare diseases: All the rage in Big Pharma

Related Articles:
A wide open market for drug developers? - Autism drug report
Novartis drug shows early promise fragile X syndrome
Roche begins Ph II Fragile X studies