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BIOTECH: VC-Funded Company Launched With MIT-Spawned Technology

By HEATHER CHAMBERS | San Diego Business Journal

Afraxis, a fledgling San Diego drug developer with technology spun out of the Massachusetts Institute of Technology, has quietly been raising money the last couple of years for testing a new treatment for Fragile X syndrome.

The genetic disorder, named after a gene mutation on the X chromosome, has been identified as a leading cause of autism and mental retardation. Approximately one-third of all children diagnosed with Fragile X syndrome also have some degree of autism, according to The National Fragile X Foundation, a research advocate based in Walnut Creek. The Centers for Disease Control and Prevention also identifies the disorder as the most common cause of inherited mental retardation.

In recent weeks, the four-person biotech has emerged from stealth mode and announced some major hires, namely that of its chief scientific officer and vice president of business development, both former employees with the local biotech Phenomix Corp. And it’s quickly pushing its program into the clinic.

The young company, led by venture capitalist Jay Lichter, began in 2007 by leveraging discoveries made in the MIT lab of Nobel laureate Susumu Tonegawa. His findings — that Fragile X could be reversed in mice by targeting a certain enzyme known as PAK — led Afraxis on a path toward developing a pill that might reduce or even reverse brain abnormalities in humans.

No Approved Drugs to Treat Fragile X

Lichter, who also serves as managing director of Avalon Ventures of La Jolla, said the company started from scratch “with no compounds and no assays and no predetermined clinical path.”

It quickly paired up with Ambit Biosciences Inc., an expert in screening small molecule compounds against an array of enzymes, and identified some drug candidates to support its patent filing.

“That is really what gave birth to the company,” Lichter said.

Today, he said, Avalon has invested $6 million altogether to advance the company to the point of human trials. The young company could have its first drug candidate in human trials as early as next year, according to Chief Scientist David Campbell.

“Our goal is to identify a drug candidate by the middle of this year,” he said.

A drug to treat the disorder, one that affects approximately one in 4,000 males and fewer females, could potentially emerge as a big seller since the FDA has not approved any drugs to treat it, and there’s no known cure.

“It’s a very expensive burden on society,” said Lichter, who further explained that many people living with the disorder can’t hold jobs, have trouble paying attention and often require supervised care.

Source: http://www.sdbj.com/industry_article.asp?aID=145683

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The Autism News English, English Afraxis, autism, autistic, drug, Fragile X, Genetic Disorder, treatment