Wednesday, September 21, 2005

Study of Defibrotide to treat Multiple Myeloma

Gentium S.p.A. Announces Initiation of Independent Phase I/II Study of Defibrotide to treat Multiple Myeloma
Gentium S.p.A. (AMEX:GNT) (the "Company") announced today that its lead product, Defibrotide, will be the subject
of an independent Phase I/II study to treat Multiple Myeloma (MM) in combination with Melphalan, Prednisone, and
Thalidomide (MPT) at approximately 10 cancer centers in
Italy. The principal investigator for this study is Dr. Mario
Boccadoro, M.D., Division of Hematology,
University of Turin, Italy.
The study is a Phase I/II dose-escalating, multi-center, non-comparative, open label study designed to assess the safety
and the efficacy of Defibrotide with MPT regimen as a salvage treatment in advanced refractory MM patients.
Dr. Laura Ferro, Chairman and CEO of the Company, said, "We are encouraged by the continued interest from world leading
clinicians to independently test Defibrotide to treat Multiple Myeloma. Preclinical studies conducted at the
Jerome Lipper
Multiple
Myeloma Center
at Harvard University's Dana Farber Cancer Institute were very promising and suggest that
Defibrotide sensitizes MM cells to anti-MM agents in the bone marrow by preventing the molecular cascade of events triggered
by MM-bone marrow stromal cells contact. This pre-clinical study provided the framework for this Phase I/II clinical trial of
oral MPT in combination with Defibrotide in the treatment of Multiple Myeloma."
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 extensive beneficial pharmacological effects owe to its
anti-thrombotic, anti-inflammatory and anti-ischemic properties.

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