VILLA GUARDIA, Italy--(BUSINESS WIRE)--Gentium S.p.A (NASDAQ: GENT - News) announced today that an independent Data Safety Monitoring Board (DSMB) has provided an update on the planned interim analysis for the Company’s Phase III, historically controlled, multi-center study of Defibrotide for the treatment of severe veno-occlusive disease in hematopoietic stem cell transplant patients. The DSMB meeting was conducted after data relating to the primary and secondary efficacy endpoints (achievement of complete response and survival by day 100, respectively) had been collected on 46 patients in the Defibrotide treatment arm and 86 patients in the historical control group.
In January, the Company previously announced the initiation of the DSMB’s interim analysis. At that stage, the DSMB concluded that there were no safety concerns and that the prospective treatment and historical control arms appeared to be well-balanced as to the stratification factors in the protocol. In order to complete the interim analysis, the DSMB requested that the Company provide supplemental trial data. The Company has since provided the DSMB with additional information.
In its latest report, the DSMB reconfirmed its findings regarding safety and balance of the trial arms based on the data presented to date. However, the DSMB did have concerns and questions about the data assembled and, accordingly, the DSMB “did not regard it proper to make a recommendation concerning the further conduct of the study at this time.” The DSMB did make one recommendation that the Company obtains confirmation of “the criteria used to select historical controls and the practical application of those criteria to guarantee that the historical patients match the patients in the prospective study arm.” The Company is evaluating the comments made by the DSMB and will respond accordingly.
At present, the Company has already enrolled more patients than required under the protocol, with 86 patients in the historical control group and 101 patients in the treatment arm, and does not anticipate enrolling further patients in the trial.
“Due to the life threatening nature of severe VOD and the absence of any effective alternative therapy, it was necessary to use a historical control arm in the trial rather than a placebo control arm in order to meet ethical standards and attract the participation from key clinical transplant centers. Collecting high quality data from historical patient records is by its nature a challenging process,” said Dr. Laura Ferro, Chairman and Chief Executive Officer of Gentium. “We will continue treatment and follow up of the last patients enrolled in our treatment arm and expect to have top-line data from this trial during Q4 2008.”
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.”
Contact:
Gentium S.p.A. Gary Gemignani, +1-212-332-1666 Chief Financial Officer ggemignani@gentium.com or
The Trout Group Marcy Strickler, +1-646-378-2927 mstrickler@troutgroup.com or
Lifonti & Company Luca Ricci Maccarini, +39-02-7788871 luca.maccarini@lifonti.it
Source: Gentium S.p.A
