Gentium Provides An Update On The Phase 3 Treatment Trial Of Defibrotide For Severe Veno-Occlusive Disease

Main Category: Cancer / Oncology
Also Included In: Cardiovascular / Cardiology;  Liver Disease / Hepatitis;  Urology / Nephrology
Article Date: 20 Nov 2008 - 6:00 PDT

email to a friend   printer friendly   view / write opinions   rate article

Gentium S.p.A. (NASDAQ: GENT) announced today interim results from an independent Data Safety Monitoring Board (DSMB) review of the Company's Phase 3 treatment trial of Defibrotide for Severe Veno-Occlusive Disease (VOD). The DSMB reported that in order for the study to be 80% powered to detect a p-value of .01, the necessary statistical hurdle under the current protocol for FDA approval, the sample size should be increased to 160 patients in the treatment arm and 80 patients in the historical control arm. The DSMB also noted that a sample size of 102 patients in the treatment arm and 51 patients in the historical control arm would be needed to achieve a p-value of .05. Furthermore, the DSMB indicated that the data presented thus far do not raise any safety concerns and did not recommend that the trial be stopped for futility.

Gentium also announced today the preliminary results of an independent Medical Review Committee's (MRC) selection of historical control patients in the trial. Following the results of a prior DSMB meeting announced on June 5, 2008 in which the DSMB expressed concerns regarding the practical application of the criteria used to enroll historical control patients, the MRC met to re-review the criteria and eligibility of all historical control cases. After reviewing the available information, the MRC was only able to conclude that 32 out of the 86 patients initially included in the historical control arm definitively met the eligibility criteria and had a confirmed diagnosis of severe VOD. There are currently 102 patients enrolled in the treatment arm of the study.

"While we were hoping to be in a better position following the reviews of the DSMB and MRC concerning our Phase 3 trial, we remain committed to the development of Defibrotide," said Dr. Laura Ferro, CEO of Gentium. "We do not intend to enroll the additional number of patients required to achieve a p-value of .01; however, we are evaluating the possible enrollment of additional patients in the historical control arm, which would allow us greater potential to achieve a p-value that could be used as supportive data in favor of an approval of Defibrotide."

"We are currently in discussions with our corporate partner regarding the future development of Defibrotide and are also evaluating our strategic options for the Company," said Gary Gemignani, CFO of Gentium. "We continue to make progress in our European Pediatric Phase 2/3 prevention trial. To date, 345 out of 360 patients have been enrolled and we look forward to reporting results in 2009."

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. http://www.gentium.com

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."

http://www.gentium.com