Drug startup’s treatment for genetic disorder may help with teen liver disease

NOVATO – A compound undergoing tests by a Marin pharmaceutical developer for treating a rare genetic disease in children shows promise in combating an ominous trend: teenagers with obesity-caused liver disease.

Raptor Pharmaceuticals Corp. announced late last week that the Phase 2a – proof of concept – clinical test has enrolled 12 teenagers. The trial should be complete by the middle of 2009, according to Ted Daley, president of Raptor’s clinical division.

“It’s a very sobering, even tragic trend. Liver disease has traditionally been an adult affliction,” said Mr. Daley. Raptor has been working with clinicians at the University of California at San Diego to test DR Cysteamine, a new formulation of a drug to treat the genetic disease Nephronpathic Cystinosis.

“One of our collaborators, Ranjan Dohil, was aware of the fact that cysteamine also has indications for both Huntington’s Disease and non-alcoholic steatohepalitis (NASH). Another of our collaborators, Jerry Schneider, has a NASH clinic for teens. They offered us the opportunity to broaden the range of our compound and we took it,” said Mr. Daley.

If the compound proves successful, it will be used to treat NASH patients of all ages, but Dr. Schneider’s work with teens made initially focusing on young people most practical, he said.

According to a surge of recent medical studies, 2 percent to 5 percent of American children older than 5, nearly all them obese or overweight, have non-alcoholic fatty liver disease. Some experts believe as many as 10 percent of all children and 50 percent of obese children may have it.

Some require liver transplants as teens. Experts worry that many more will need a new liver by their 30s or 40s. Currently there is no treatment except, in early stages, weight loss.

The dozen teens undergoing tests in the Raptor study all have confirmed diagnoses of liver disease. The trial will use measurement of liver enzymes to determine if its cysteamine compound is causing improvement.

Raptor was founded in Novato in 2006 by former BioMarin executives. It employs seven full-time and uses 12 others on a consultation basis.