Source: BUSINESS WIRE
Thursday, November 20, 2008
Results from Data Safety Monitoring Board Review
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Assessment of Historical Control Arm by Independent Medical Review
Committee
VILLA GUARDIA, Italy--(BUSINESS WIRE)--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.
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.”