GENENTECH DNase CYSTIC FIBROSIS TREATMENT CLINICAL TRIALS

Executive Summary

GENENTECH DNase CYSTIC FIBROSIS TREATMENT CLINICAL TRIALS will likely begin "within the next few months," the South San Francisco, California-based biotech company announced May 29. Genentech filed an IND application for the recombinant enzyme product with FDA during the week of May 21. Initial safety and dosing trials will involve the administration of aerosolized DNase in young adults with cystic fibrosis. Trials in children will follow after the safety of the drug is established. No decision has been made as to the number of centers or patients that will be involved, according to the firm. The scientific team developing DNase is headed by Genentech Immunobiology Director Steven Shak, MD. DNase contains a naturally occurring enzyme that breaks down DNA molecules that accumulate in the lungs as the mucus associated with cystic fibrosis. Genentech expects that patients will have "few allergic responses" to human-derived DNase because, unlike an earlier version based on bovine sources, the recombinant product "mimics the natural human enzyme normally present in blood and saliva." In addition, DNase "cannot penetrate the membranes of normal cells and therefore cannot damage DNA within live cells," the firm said. Preclinical laboratory testing so far has shown that DNase is "effective in liquefying secretions obtained from the lungs of patients with cystic fibrosis," Genentech said in its May 29 release. Genentech says it plans to file for orphan drug status. Approximately 30,000 children and young adults in the U.S. have the disease.