Prophylactic Treatment with Defibrotide in Post Transplant Patients May Reduce Incidence of Death in VOD

Publication Supports Minimal Morbidity and Mortality from Veno-occlusive Disease in Post-Transplant Patients

NEW YORK, NY, USA | June 01, 2007 |
Gentium S.p.A. (Nasdaq: GENT) announced the publication of the results of an independent clinical study supporting the prophylactic treatment of patients post allogeneic stem cell transplant with Defibrotide as a preventative therapy for veno-occlusive disease, a deadly complication frequent in patients who receive stem cell transplants. Results of the study demonstrated the effectiveness of treatment with Defibrotide in reducing both the incidence of VOD, as well as potentially severe hemorrhagic complications. The data from the Royal Marsden Hospital in the UK were published in the May 14th issue of the journal Bone Marrow Transplantation.

In this study, patients received Defibrotide twice a day for twenty-one days. Patients did not receive concurrent heparin therapy. Fifty-eight patients, all of whom had received allogeneic stem cell transplant, were treated with Defibrotide. No patients met the Baltimore criteria for VOD and no patients died of suspected VOD within 100 days of transplantation. The transplant related mortality (TRM) within 100 days was 5/58 (8.6%) of patients. VOD was not felt to have contributed to the deaths of any of these patients. Patients tolerated Defibrotide well and the drug did not have to be discontinued in any patient. There were no cases of hemorrhagic complications attributable to Defibrotide.

"We are pleased with the results of this study and believe that they are supportive of the ongoing Phase 2/3 pivotal trial evaluating the incidence and outcome of VOD with the prophylactic use of Defibrotide in pediatric stem cell transplantation patients," commented Laura Ferro, M.D., President and CEO of Gentium. "We hope that the results published will translate into a positive outcome for patients in our ongoing trial."

For review of the abstract of this publication please see: http://www.nature.com/bmt/journal/vaop/ncurrent/abs/1705696a.html

Defibrotide is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration to prevent and to treat severe veno-occlusive disease (VOD) and Fast Track designation for the treatment of severe VOD in recipients of stem cell transplants. Defibrotide is currently in a U.S. Phase III trial to treat severe VOD and a European Phase II/III trial to prevent VOD in pediatric patients.

About VOD
Veno-occlusive disease (VOD) is a potentially life-threatening condition. Certain high-dose chemotherapy and radiation therapies and stem cell transplantation (SCT) can damage cells of the blood vessels and result in VOD, a blockage of the small veins of the liver that can lead to liver failure and the failure of other organs (severe VOD). SCT is a frequently used treatment following high-dose chemotherapy and radiation therapy. The International Bone Marrow Transplant Registry estimated that in 2002 approximately 45,000 people received blood and bone marrow transplants, which are types of SCT. Based on the Company's review of more than 200 published papers, it believes that approximately 20% of patients who undergo SCT develop VOD, approximately one-third of those who develop VOD progress to severe VOD and approximately 80% of severe VOD patients die within 100 days of the SCT. The Company believes that there are no approved therapies to treat or prevent VOD in the U.S. or the EU.

About Gentium
Gentium S.p.A. is a biopharmaceutical company focused on the research, discovery and development of drugs derived from DNA extracted from natural sources, and drugs that are synthetic derivatives, to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate in the U.S., is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration to prevent and to treat VOD and Fast Track designation for the treatment of severe VOD in recipients of stem cell transplants.

SOURCE: Gentium S.p.A.

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