San Diego’s Afraxis Gets NIH Support to Develop Compound for Rare Fragile X Syndrome

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.

Author: Bruce V. Bigelow

In Memoriam: Our dear friend Bruce V. Bigelow passed away on June 29, 2018. He was the editor of Xconomy San Diego from 2008 to 2018. Read more about his life and work here. Bruce Bigelow joined Xconomy from the business desk of the San Diego Union-Tribune. He was a member of the team of reporters who were awarded the 2006 Pulitzer Prize in National Reporting for uncovering bribes paid to San Diego Republican Rep. Randy “Duke” Cunningham in exchange for special legislation earmarks. He also shared a 2006 award for enterprise reporting from the Society of Business Editors and Writers for “In Harm’s Way,” an article about the extraordinary casualty rate among employees working in Iraq for San Diego’s Titan Corp. He has written extensively about the 2002 corporate accounting scandal at software goliath Peregrine Systems. He also was a Gerald Loeb Award finalist and National Headline Award winner for “The Toymaker,” a 14-part chronicle of a San Diego start-up company. He takes special satisfaction, though, that the series was included in the library for nonfiction narrative journalism at the Nieman Foundation for Journalism at Harvard University. Bigelow graduated from U.C. Berkeley in 1977 with a degree in English Literature and from the Columbia University Graduate School of Journalism in 1979. Before joining the Union-Tribune in 1990, he worked for the Associated Press in Los Angeles and The Kansas City Times.