Gentium Receives First IRB Approval to Begin U.S. Phase III Trial with Defibrotide to Treat Veno-Occlusive Disease with Multipl
March 28 2006 - 8:00AM
Business Wire
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."
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