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Gentium Announces Completion of Data Safety Monitoring Board Review and Continuation of the Phase II/III European Pediatric Prevention Trial of Defibrotide

Posted on: Thursday, 5 June 2008, 18:00 CDT

Gentium S.p.A (NASDAQ: GENT) announced today that an independent Data Safety Monitoring Board (DSMB) has completed the planned interim analysis for the Company's Phase II/III multi-center, open label, randomized trial in Europe to evaluate prophylactic use of Defibrotide in pediatric patients undergoing stem cell transplantation at high risk for hepatic veno-occlusive disease (VOD). The interim analysis was performed subsequent to the enrollment of 240 patients.

The DSMB concluded that there are no significant safety concerns, the prophylactic treatment arm (Defibrotide) and the control arm (no drug) are well balanced, and there is no evidence of clinical futility in the trial. Further, the DSMB indicated that the results to date were satisfactory and recommended that the trial continue to accrue patients. The DSMB also recommended increasing total patient enrollment to 180 patients per arm from 135 patients per arm to achieve a more statistically significant benefit of Defibrotide over the control. There are no further planned interim analyses for this trial.

"The DSMB's recommendation is very encouraging and we await the final outcome of this key trial with great interest," said Dr. Dietger Niederwieser, Department of Hematology and Oncology, University of Leipzig and President of the European Group for Blood and Marrow Transplantation, the co-sponsor of the trial. "I am hopeful that the results will be consistent with the promising data from earlier published studies in prophylaxis and support the efficacy of Defibrotide in preventing VOD."

"We are pleased with the DSMB's review and are following their recommendation to accrue 180 patients in each arm of the study," said Dr. Laura Ferro, Chairman and Chief Executive Officer of Gentium. "There are currently 276 patients enrolled in the trial and we plan to complete enrollment around year-end and report top-line results during the first half of 2009. We have also been advised recently by EMEA that our planned filing in this indication could be eligible for an accelerated review, which could reduce the review cycle from seven months to five."

Gentium will host a conference call today, June 5, 2008 at 5:00 p.m. (ET) to discuss this press release and the other press release issued today by the Company regarding clinical trial matters for Defibrotide.

Dial-in Information: US/Canada Toll-Free callers: 877-407-8031 US/Canada Toll or International Toll callers: 201-689-8031

Live audio of the conference call will be simultaneously broadcast over the internet via a webcast. To access the live webcast, log on to the Gentium's corporate website at http://www.gentium.com

A replay of the call also will be available until 11:59 PM US Eastern Time on June 12, 2008. To access the replay, dial 1-877-660-6853 from the US or Canada (toll-free) or 1-201-612-7415 from other locations, and enter account #286 and conference ID#287992. Additionally, an archived replay of the conference web cast will be available on the Gentium website for 30 days.

About VOD

Veno-occlusive disease (VOD) is a potentially life-threatening condition, which typically occurs as an important complication of stem cell transplantation (SCT). Certain high-dose chemo-radiation therapy regimens used as part of SCT can damage the cells lining the hepatic blood vessels and so result in VOD, a blockage of the small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so called severe VOD with multiple organ failure). SCT is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children. There is currently no approved agent for the treatment or prevention of 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, is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration and EMEA 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.

Cautionary Note Regarding Forward-Looking Statements

This press release contains "forward-looking statements." In some cases, you can identify these statements by forward-looking words such as "may,""might,""will,""should,""expect,""plan,""anticipate,""believe,""estimate,""predict,""potential" or "continue," the negative of these terms and other comparable terminology. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. It is possible that actual results may differ, possibly materially, from those anticipated in these forward-looking statements. For a discussion of some of the risks and important factors that could affect future results, see the discussion in our Form 20-F filed with the Securities and Exchange Commission under the caption "Risk Factors."

Source: Business Wire

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