This excerpt taken from the GENT 6-K filed Nov 16, 2005.
Clinical Highlights and Outlook
Commenting on Gentiums clinical progress during the quarter, Laura Ferro, M.D., Chairman and Chief Executive Officer, said, We achieved a number of important clinical milestones. We had a very positive meeting with the FDA, which provided constructive comments regarding our proposed U.S. Phase III trial of Defibrotide to treat Severe VOD. Following these discussions, and with input from our expert consultants, we finalized the trial protocol. We expect to initiate the Phase III study for this indication before the end of the year. We note that Defibrotide addresses a life-threatening disease for which there are absolutely no current treatment options.
In addition to using the same 10 clinical sites and the investigators that conducted the Phase II trial, our plans call for expanding the Phase III study to include as many as six additional clinical sites, Dr. Ferro added.
We are also studying Defibrotide for the prevention of VOD. In conjunction with the European Group for Blood and Marrow Transplantation, we are co-sponsoring a Phase II/III pediatric clinical trial in Europe for the use of Defibrotide to prevent VOD. We expect to begin treating children later this year in this 270-patient study to be conducted at several leading centers in Europe. We also expect to start a second Phase II/III clinical trial in Europe early in 2006 for prevention of VOD in adults. This trial will include approximately 300 patients in several centers in Europe.
Finally, very encouraging pre-clinical data using Defibrotide to treat refractory multiple myeloma led to planning an independent Phase I/II study at ten clinical centers in Italy. This trial is also expected to begin before the end of the year. According to the American Cancer Society, almost 16,000 people will develop multiple myeloma in the U.S. this year, and the five-year survival rate is approximately 30%. This is a potentially significant opportunity for Gentium.
This excerpt taken from the GENT 6-K filed Aug 18, 2005.
Clinical Highlights and Outlook
Commenting on Gentiums clinical progress, Dr. Ferro reported, During the second quarter we achieved a number of important clinical milestones. We were granted Fast Track designation by the FDA for our initial product candidate, Defibrotide for the treatment of Severe VOD, which has previously been granted Orphan Drug status. We have a meeting scheduled with FDA officials later in the third quarter with the goal of finalizing the protocol for our U.S. Phase III clinical trial. The upgrades made to our
manufacturing facility in 2004 positioned the Company for its CMC submission to the FDA. The acceptance by the FDA of this submission was a major milestone necessary for the initiation of this Phase III trial. Upon securing approval of our Phase III protocol, we expect to begin treating patients immediately, as we will be using the same centers and clinical investigators from the Phase II trial.
Dr. Ferro continued, We have expanded the terms of our licensing agreement for Defibrotide for the treatment of Severe VOD with our marketing partner, Sigma Tau, to include Canada, South America and Central America, in addition to the previously negotiated marketing rights to the U.S. Sigma Tau also has a right of first refusal to market Defibrotide for the prevention of VODin the Americas.
The Consorzio Mario Negri Sud, a private, non-profit institute in Italy that carries out basic research in cell biology related to pharmacology and biomedicine, is conducting a multi-center Phase II/III clinical trial in Europe and Israel for the treatment of VOD after stem cell transplant. We expect this trial to include approximately 340 patients. We also have studies and plans for studies with Defibrotide for the prevention of VOD underway. We are co-sponsoring with the European Group for Blood and Marrow Transplantation a Phase II/III clinical trial in Europe for the use of Defibrotide to prevent VOD in children. We expect this study to include 270 patients enrolled by several centers in Europe beginning later this year. We also expect to start a second Phase II clinical trial in Europe later this year for use of Defibrotide to prevent VOD in adults. We expect this trial to include approximately 300 patients enrolled by several centers in Europe. Finally, we are planning to begin an exploratory clinical trial of Defibrotide for the treatment of refractory multiple myeloma by the first quarter of 2006.