The Associated Press
Italian biopharmaceutical company Gentium SpA said Wednesday results from a late-stage study of its drug Defibrotide failed to show a level of statistical significance required for proof of efficacy.
But the results did show "strong trends in favor of the Defibrotide-treated patients for complete response and survival," the company said in a statement.
The company is studying the drug as a treatment for veno-occlusive disease in stem-cell transplant patients, a common complication occurring soon after bone marrow transplants.The study's primary endpoint was complete response at 100 days after a stem cell transplant, and its secondary endpoints included survival rate at 100 days and six months after stem-cell transplants. Gentium Chief Financial Officer Gary Gemignani said in the statement the company expected that reaching "the required statistical threshold for a single trial would be difficult." "We are pleased that the data are compelling and believe the results place us in a strong position to continue discussions with the FDA and others regarding next steps toward a regulatory filing," he said. The study's clinical director, Dr. Paul Richardson, said in the statement he was encouraged by the results, "especially given the extremely sick patient population that was enrolled." Gentium's shares were off 17 cents, or 5 percent, at $3.23 in early Nasdaq trading Wednesday.