Results From Gentium's Treatment IND for Defibrotide Presented at the American Society of Hematology Annual Meeting

Gentium S.p.A.

today announced that an oral presentation on Defibrotide was presented today at the 53nd Annual Meeting and Exposition of the American Society of Hematology held at the San Diego Convention Center in California. Dr. Paul Richardson, Clinical Director of the Dana-Farber Cancer Institute, Boston, MA (

), reported in an oral presentation the updated results from the ongoing Treatment IND Expanded Access Protocol for Defibrotide (abstract #487). The analysis was based on 269 patients with severe veno-occlusive disease (sVOD) and multi-organ failure, enrolled between December 2007 and March 2011 at 67 centers across the United States. 251 patients had undergone stem cell transplant and the remaining 18 had developed VOD after chemotherapy alone. Of 269 patients, 32% achieved a complete response and 50% survived to day 100 (D+100). Additional findings were as follows: In the subgroup of SCT patients, 31% (78/251) achieved a CR and 50% survived to D+100. In the 18 chemotherapy-only patients, CR was 39% and D+100 survival was 50%. Of 200 patients with sVOD, CR was 28% and D+100 survival was 44%. Early initiation of Defibrotide treatment (< 2 days, versus a delay of >2 days) following VOD diagnosis resulted in increased CR (35% versus 23%, p=0.03) and survival (56% versus 37%, p=0.01). CR rate and D+100 survival for the 69 patients with non-severe VOD were 42% and 62%, respectively. 134 patients met entry criteria for the original Phase 3 trial of Defibrotide and comparison to the Phase 3 historical control showed a statistically improved outcome in CR (30% vs 9%, p=<0.01) and D+100 survival (46% vs 25%; p=<0.01). The incidence of all grade GvHD in the allogeneic SCT patients (225/251) was 8%, similar to the observation of decreased GvHD in other studies of Defibrotide. These results confirm the findings of previous trials and support early intervention with Defibrotide following diagnosis of VOD. Additionally, there were low incidences of GvHD in allogeneic-SCT patients treated with Defibrotide and of treatment-associated toxicities, both consistent with prior studies of Defibrotide.