Gentium presents evaluation results of Defibrotide for VOD at the ASH conference

Dec 8 2009

Gentium S.p.A. (NASDAQ: GENT) today announced final clinical trial results from the Company’s Phase II/III Pediatric Prevention trial and Phase III Treatment trial for Defibrotide of Hepatic Veno-Occlusive Disease (VOD), which were presented at the American Society of Hematology Conference (ASH) in New Orleans. The results of both trials strongly trended toward statistical significance. The Prevention trial demonstrated a 40% reduction in the incidence of VOD at day 30>

Dr. Selim Corbacioglu, Pediatrics, University of Regensburg (Germany) and Principal Investigator of the Pediatric Prevention trial reported in an oral presentation that on an intent to treat basis (ITT), Defibrotide demonstrated a 40% reduction>

“I believe that the results of the pediatric prevention study, which is the largest trial to date conducted in the pediatric bone marrow transplant setting, confirmed that Defibrotide is well tolerated and is effective in preventing VOD,” said Dr. Selim Corbacioglu. “Additionally, we are enthusiastic that Defibrotide was able to significantly reduce the incidence and severity of acute graft versus host disease, a life threatening complication of stem cells transplants for which there are limited, effective prophylactic and treatment options. This activity is consistent with the drug’s role in the protection of endothelial cells.”

Dr. Paul G. Richardson, Clinical Director of the Dana-Farber Cancer Institute’s Jerome Lipper Multiple Myeloma Center and Principal Investigator of the Treatment trial reported in a separate oral presentation that on an ITT basis, 24% of patients in the Defibrotide arm compared to 9% of patients in the historical control arm achieved complete response at 100 days>

“I am very encouraged by the results of this trial, especially given the extremely sick patient population enrolled,” said Dr. Paul Richardson. “The data generated from this trial confirms the activity of Defibrotide seen in earlier studies, with the clinical benefit reflected by a significant improvement in complete response rate, as well as a promising trend for survival. In addition, the results support its potential in less advanced stages of VOD.”

“To date, Defibrotide has been evaluated for the treatment and prevention of VOD in over 1,100 patients globally across multiple clinical trials, a compassionate use program and under a treatment IND protocol,” said Dr. Massimo Iacobelli, Scientific Director of Gentium. “We believe that the efficacy data generated has consistently been favorable and demonstrated an acceptable safety profile. With the final results of these two studies in hand, and other supportive data, we look forward to determining the next steps toward approval.”

Defibrotide has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency to prevent and to treat VOD and Fast Track designation by the U.S. FDA for the treatment of severe VOD in recipients of stem cell transplants.