Gentium Provides Clinical Update on Phase III Defibrotide Clinical Program
VILLA GUARDIA, Italy - Gentium announced today that it plans to submit to the U.S. Food and Drug Administration (the â€œFDAâ€) an amendment to the protocol for its ongoing Phase III trial of Defibrotide for the treatment of hepatic veno-occlusive disease ("VOD") with multi-organ failure. The decision to submit the amendment was prompted by recent discussions between the Company and the FDA.
Under the amended protocol, the primary endpoint for the 160 patient, historically controlled, multi-center Phase 3 study would change from survival at 100 days to complete response as defined by bilirubin < 2 mg/dL and the resolution of multiple organ failure. Survival at 100 days will be evaluated as a secondary endpoint. Under the original protocol, complete response was a secondary endpoint which also included the return of hepatomegaly to baseline and the resolution of right upper quadrant pain. However, these two additional criteria will now be eliminated due to their more subjective nature. Because data on both endpoints have been collected during the course of the trial, no additional changes to the size or design of the study are required.
Dr. Laura Ferro, Chairman and Chief Executive Officer of Gentium said, "We have spoken with various scientific and regulatory advisors, and we are comfortable with complete response as our primary endpoint. The impact of Defibrotide on complete response seen in our Phase II trial was highly correlated to survival at 100 days in severe VOD patients. To date, the trial has enrolled 65 of the expected 80 patients in its prospective arm and remains on track to report results during the first half of 2008.â€
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.
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 "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 20F filed with the Securities and Exchange Commission under the caption "Risk Factors."