Gentium's Defibrotide Subject of Independent Study in Japan; First Japanese Study Reports That Defibrotide Shows Success Treatin
January 30 2006 - 8:00AM
Business Wire
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."
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