Gentium shares sink on Phase III vein drug failure

Italy's Gentium ($GENT) saw its shares sag on the news that its experimental therapy to treat the blockage of small veins in the liver failed the primary and secondary endpoints of a late-stage trial.

The veno-occlusive disease treatment Defibrotide demonstrated "strong trends" in its favor, but failed to deliver the necessary data for complete response following 100 days after a stem cell transplant compared to patients receiving a best-care drug as well as an improved survival rate after 100 days and six months following treatment. Still, researchers claimed that they were left optimistic about the drug.  

"I am encouraged by the results of this trial, especially given the extremely sick patient population that was enrolled," said Dr. Paul Richardson, clinical director of the Dana-Farber Cancer Institute's Jerome Lipper Multiple Myeloma Center and principal investigator of the trial. "The data generated from this trial confirms the activity of Defibrotide seen in earlier studies, and supports the benefit of Defibrotide for the treatment of sVOD in improving complete response rates and survival, as well as its potential in less advanced stages of the disease."

"Given the outcome of the data safety monitoring board's interim review announced in November of last year, we expected that reaching the required statistical threshold for a single trial would be difficult," Chief Financial Officer Gary Gemignani said. "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. Additionally, we plan on announcing final results from our randomized, pediatric prevention study in the upcoming weeks."

- read the Gentium release
- here's the Reuter's story