Gentium Announces the Data Safety Monitoring Board Review of the Phase III Trial of Defibrotide to Treat Patients with Severe Veno-Occlusive Disease
VILLA GUARDIA, Italy--(BUSINESS WIRE)--Gentium S.p.A (the â€œCompanyâ€) announced today that the Data Safety Monitoring Board (DSMB) has initiated the planned interim analysis for the Company's Phase III trial of Defibrotide to treat patients with severe veno-occlusive disease.
The DSMB has conducted a safety analysis of Defibrotide as part of the interim analysis and concluded that there are no safety concerns at this time. Additionally, the DSMB evaluated stratification between the prospective treatment and historical control arms and indicated that the groups appear to be balanced. The DSMB has asked the Company to clarify and supplement certain trial data in order to complete the remainder of the interim analysis. After the DSMB indicates that it has concluded the interim analysis, the Company will announce further results of the DSMBâ€™s review.
â€œWe are pleased with the results of the safety and stratification analyses,â€ said Dr. Laura Ferro, Chairman and Chief Executive Officer of Gentium. â€œPatient accrual in the trial continues, and 76 patients have been enrolled to date in the prospective treatment arm.â€
The DSMB consists of independent oncology and statistical experts whose primary responsibility is to monitor, on a periodic basis, the data from the trial and to provide recommendations to Gentium on whether the study should proceed as originally planned, be modified, or be discontinued due to futility or safety concerns. Members of the DSMB are independent from Gentium and do not participate as clinical investigators in the Phase III trial of Defibrotide.
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 Company believes that there are no approved therapies to treat or prevent VOD in the U.S. or the EU.
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.
Cautionary Note Regarding Forward-Looking Statements
This press release contains â€œforward-looking statements.â€ In some cases, you can identify these statements by forward-looking words such as â€œmay,â€ â€œmight,â€ â€œwill,â€ â€œshould,â€ â€œexpect,â€ â€œplan,â€ â€œanticipate,â€ â€œbelieve,â€ â€œestimate,â€ â€œpredict,â€ â€œpotentialâ€ or â€œcontinue,â€ 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 20F filed with the Securities and Exchange Commission under the caption â€œRisk Factors.â€