Gentium Spa (AMEX:GNT)
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Gentium S.p.A. (AMEX:GNT) (the "Company") today
announced the initiation of a Phase II/III trial with Defibrotide to
prevent Veno-Occlusive Disease ("VOD"), a complication of bone marrow
and stem cell transplantation (SCT), in pediatric patients. The
randomized study will include 270 pediatric patients undergoing SCT at
30 clinical sites in Europe and Israel and will evaluate the ability
of Defibrotide to prevent VOD. Secondary endpoints are measuring the
severity of VOD and the occurrence of transplant-associated
microangiopathy in each group. The European Group for Blood and Marrow
Transplantation (EBMT) is co-sponsoring the study with additional
support from the Deutsche Krebshilfe (German Cancer Aid). The
principal investigator is Selim Corbacioglu, M.D., Dept of Pediatrics
- University of Ulm, Germany.
Certain chemotherapy and radiation therapies such as those used in
stem cell transplantation can damage cells of the blood vessels and
produce VOD, a blockage of the small veins of the liver which leads to
damage of the liver cells. Approximately 20% of patients who undergo
SCT develop VOD and approximately one-third of these patients progress
to multiple organ failure (Severe VOD). Nearly 80% of Severe VOD
patients die within three months. There are currently no approved
therapies to treat or prevent VOD.
Laura Ferro, M.D., president and chief executive officer of
Gentium, said, "Preventing VOD in stem cell transplant patients is
critically important, especially for pediatric patients who are
particularly susceptible to developing VOD due to the nature of their
treatment regimen. We look forward to also beginning our European
trial for the prevention of VOD and transplant-associated
microangiopathy in adults in the second quarter of this year.
"Earlier clinical trials of Defibrotide to treat and prevent VOD
have shown encouraging results. We are pleased to begin this
prevention trial in children and hope it will provide evidence of
Defibrotide's potential efficacy to prevent VOD.
"We are delighted that the EBMT and the Deutsche Krebshilfe have
agreed to support our efforts to develop a preventative drug for
patients at risk of developing VOD, a potentially fatal disease, for
which there are no currently approved therapies," concluded Dr. Ferro.
About Defibrotide
Defibrotide is a single-stranded DNA that protects the vascular
endothelial cells, particularly those of small vessels, from damage
and activation. After binding to endothelial cells, Defibrotide
decreases cell adhesion and pro-coagulant activity of activated
endothelial cells, and increases the fibrinolytic potential of
endothelial cells. Defibrotide's effects are predominately local
within the vascular bed, and there is no significant effect on
systemic coagulation. Its beneficial pharmacological effects are due
to its anti-thrombotic, anti-inflammatory and anti-ischemic
properties.
Defibrotide is expected to be the subject of a U.S. Phase III
study as a treatment for Severe VOD in the coming weeks. The Company
also intends to initiate studies of Defibrotide to prevent VOD in the
U.S. by early 2007.
About VOD
VOD is a potentially life-threatening condition. The International
Bone Marrow Transplant Registry estimated that approximately 45,000
people received blood and bone marrow transplants in 2002. Based on
the Company's review of more than 200 published papers it is estimated
that up to 20% of patients who receive blood and bone marrow
transplants contract VOD. When Severe VOD occurs, blood vessels in the
liver become blocked, liver failure follows and kidneys cease to
function as a result of the toxic effects of cancer treatments such as
high dose chemotherapy and radiation, used during SCT. Approximately
80% of patients who contract Severe VOD die within 100 days of SCT
without treatment. VOD is considered one of the most important and
challenging complications of SCT. There currently are no approved
therapies to treat or prevent VOD.
About Gentium
Gentium S.p.A. is a biopharmaceutical company located in Villa
Guardia (Como), Italy that is 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. FDA to treat Severe 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 Prospectus filed with
the Securities and Exchange Commission under Rule 424(b)(4) under the
caption "Risk Factors."
GNT-G