Gentium Spa (AMEX:GNT)
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Gentium S.p.A. (AMEX:GNT) (the "Company") today reported
that an independent study of Defibrotide was the subject of a
published paper titled "Successful Treatment with Defibrotide for
Sinusoidal Obstruction Syndrome (also known as Veno-Occlusive Disease
or "VOD") after Hematopoietic Stem Cell Transplantation (SCT)," which
appeared in the December 2005 issue of Kobe Journal of Medical
Science. The lead author of the paper was Kimikazu Yakushijin,
Division of Endocrinology/Metabolism, Neurology and
Hematology/Oncology, Department of Clinical Molecular Medicine, Kobe
University Graduate School of Medicine, Japan.
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 which can
lead to liver failure and the failure of the kidneys and other organs.
SCT is a frequently used treatment following high dose chemotherapy
and radiation therapy. Based on the Company's review of more than 200
published papers, approximately 20% of patients who undergo SCT
develop VOD, approximately one-third of those who develop VOD progress
to multiple organ failure (Severe VOD), and approximately 80% of
Severe VOD patients die within 100 days of the SCT. There are
currently no approved therapies to treat or prevent VOD in the U.S. or
the E.U.
According to the published study, all patients showed evidence of
multiple organ failure at the start of treatment, four patients were
treated with Defibrotide for 14 to 27 days, three patients (75%)
responded to the therapy, two patients survived at 100 days post SCT
(50%), and none of the patients suffered from significant adverse
effects.
The paper concluded that this is the first report dealing with the
use of Defibrotide to treat Japanese patients with VOD and because
Defibrotide is considered to be promising for the treatment of VOD,
it is important that further studies be initiated as soon as possible
in Japan.
Commenting on the publication, Dr. Laura Ferro, chairman and chief
executive officer of Gentium, stated, "We were pleased with Dr.
Yakushijin's independent study as it builds on our body of clinical
evidence that supports the use of Defibrotide to treat Severe VOD.
Data from Harvard University's Dana Farber Cancer Institute's U.S.
Phase II study showed an increase in survival rate at 100 days post
SCT of approximately 39%, compared with the historical 100-day
survival rate of approximately 20%."
Defibrotide to Treat Severe VOD
The Company is awaiting Institutional Review Board approval for
its pivotal U.S. Phase III trial of Defibrotide to treat Severe VOD
and expects to initiate that trial in the coming weeks.
Defibrotide to Prevent VOD
The Company recently initiated a Phase II/III trial with
Defibrotide to prevent VOD 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.
The Company plans to initiate a Phase II/III trial with
Defibrotide to prevent VOD in adult patients in Europe during the
second quarter of 2006 and in the U.S. by early 2007.
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.
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. 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 which 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. Based on the Company's review of more than 200
published papers it is estimated that approximately 20% of patients
who receive SCT contract VOD. Approximately 80% of patients who
contract Severe VOD die within 100 days of SCT. VOD is considered one
of the most important and challenging complications of SCT. There
currently are no approved therapies to treat or prevent VOD in the
U.S. or the E.U.
About Gentium
Gentium S.p.A. is a biopharmaceutical company located in Villa
Guardia (Como) 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 VOD, Fast Track designation by
the U.S. FDA for the treatment of Severe VOD in recipients of stem
cell transplants, and Orphan Drug designation by European Union
regulators to treat and prevent VOD.
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."