Gentium Spa (AMEX:GNT)
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From Jun 2019 to Jun 2024
Dana-Farber/Harvard Cancer Center, Massachusetts General Hospital,
Beth Israel Deaconess Medical Center and The Children's Hospital
Receive IRB Approval to Commence Study
Gentium S.p.A. (AMEX:GNT) (the Company) has received its first
Institutional Review Board (IRB) approval to initiate a U.S. Phase III
clinical trial with Defibrotide for the treatment of Veno-Occlusive
Disease (VOD) with Multiple Organ Failure (Severe VOD) as a
complication of stem cell transplantation (SCT). Data from this
80-patient, multi-center trial will be compared to an historical
control group of 80 patients, with survival at day 100 as the primary
endpoint. The Company believes that approximately 80% of patients with
Severe VOD die within 100 days of SCT without treatment. The Company
believes that there are no approved treatments for Severe VOD.
The IRB of the Dana-Farber/Harvard Cancer Center of Boston, Mass.,
which is also the IRB for Dana-Farber Cancer Institute, Massachusetts
General Hospital, Beth Israel Deaconess Medical Center and The
Children's Hospital, is the first to give its approval to commence the
study. All four of these institutions are expected to participate in
the trial. Work to compile historical control data will begin
immediately, and the first patients are expected to be treated by
early May 2006.
The multi-center trial will be conducted at approximately 20 U.S.
cancer centers pending the IRB approval at each institution. In
addition to the institutions mentioned above, the Company expects the
trial to include M.D. Anderson Cancer Center, Fred Hutchinson Cancer
Research Center, Memorial Sloan-Kettering Cancer Center, Johns Hopkins
Hospital and Health System, Duke University Hospital, City of Hope
Cancer Center, The Children's Hospital of Philadelphia, University of
Minnesota Medical Center and The St. Jude Children's Research
Hospital, among others.
Paul Richardson, M.D., Clinical Director of Dana-Farber Cancer
Institute's Jerome Lipper Multiple Myeloma Center and Assistant
Professor of Medicine at Harvard Medical School, is the principal
investigator of this Phase III trial. He recently presented Phase II
data of Defibrotide as a treatment for Severe VOD at the 2005 American
Society of Hematology (ASH) Annual Scientific Meeting. Commenting on
the140-patient, multi-center, randomized, dose-finding Phase II study,
Dr. Richardson said, "Treatment of Severe VOD with Defibrotide
resulted in a survival rate at 100 days post SCT of approximately 40%,
which compares favorably with the 100-day survival rate of
approximately 20% or less reported in published studies of Severe VOD.
These results are encouraging, with response and long-term survival
seen even in patients who either were on dialysis or were
ventilator-dependent at the start of Defibrotide therapy. In addition,
the side effect profile of Defibrotide was favorable in this extremely
sick patient population."
"As we continue to advance the development of Defibrotide, we are
increasingly impressed by the consistency of the clinical findings and
I am optimistic that we will soon be able to offer an effective
treatment option for patients suffering from this otherwise often
fatal disease," commented Laura Ferro, M.D., president and chief
executive officer of Gentium. "We are pleased to initiate this U.S.
Phase III study and hope to see compelling clinical results."
The U.S. Food and Drug Administration (FDA) has granted
Defibrotide Orphan Drug Status and Fast Track designation for the
treatment of Severe VOD. In addition, previous clinical trials with
Defibrotide for the treatment of Severe VOD have been supported by
grants from FDA's Office of Orphan Products Development.
About VOD
VOD is a potentially life-threatening condition. 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 that 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. The International Bone Marrow Transplant Registry
estimated that approximately 45,000 people received blood and bone
marrow transplants, which are types of SCT, in 2002. Based on the
Company's review of more than 200 published papers, it believes that
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. The Company believes that
there are no approved therapies to treat or prevent VOD in the U.S or
the EU.
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)(5) under the
caption "Risk Factors."