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Replies to #22401 on Biotech Values
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DewDiligence

01/22/06 3:50 AM

#22409 RE: rkrw #22401

Re: GNT

>…if it contributes to an undervalued situation, I'm all for ceo's with thick accents, stuttering, tourette :-) <

I suppose you’re right, but I may have to ask you what the heck Dr Ferro is talking about when she does the next webcast.

(Perhaps I’ve been spoiled by listening to Speedel’s Alice Huxley, whose English is impeccable.)
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terry hallinan

01/22/06 6:49 AM

#22413 RE: rkrw #22401

Language and accent:

I'm all for ceo's with thick accents, stuttering, tourette :-) If they have the goods, eventually it won't matter.

Yes it does. It matters in the short term and the long run.

Little struck me as a greater illustration than some side comments by a reporter visiting a refusenik couple under house arrest (officially or de facto) in the bad old days of the Soviet Union. The couple had so many visits from the high and mighty of the U.S. that one might have wondered how they could possibly handle the ordeal but they nonetheless remained isolated from news of outside doings.

The couple asked the reporter what sort of position their previous visitor, Ted Sorenson, held. In short, the refuseniks couldn't understand how somebody that dumb could be important.

Ted Sorenson, of course, wrote the empty slogans that propelled another dullard to the very peak of world renown that could only be topped by an assassin's bullet.

Try to imagine the current dunce saying the same words and you should get the picture.

"They just hate him because he's an Israeli," my informant told me about David Platt (CEO of PRW).

I don't think many care.

The double whammy, however, of a glycobiologist stumbling through a second language lecturing on an arcane science that even few biologists know much about is a terrific challenge for someone like myself without a growing hearing impairment. Mix in an intolerance for fools and you have a problem.

The handicap can be overcome a lot more easily than Ted Sorenson's problem but it is a grave handicap nevertheless.

Nothing at all has been more costly to me over time than underrating the ability of snake oil salesmen to push their potions and fixes, genuine or fraudulent. I took a quick triple in IMCL long ago and found a loser to stash the cash in when I thought Sam Waksal was selling too much snow.

Dr. Ferro has an easy out. Any Italian here in Rome excels at hand signals.

Best, Terry
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DewDiligence

06/16/06 1:28 AM

#30199 RE: rkrw #22401

GENT GTCB
Hepatic veno-occlusive disease in pediatric
stem cell transplantation: impact of pre-emptive
antithrombin replacement and combined
antithrombin/defibrotide therapy


[The conclusion is that combo treatment with defibrotide and antithrombin was effective but preemptive antithrombin monotherapy was not. Defibrotide is being developed by GENT for the treatment of VOD.]

http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list_...

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Haematologica. 2006 Jun;91(6):795-800.

Haussmann U, Fischer J, Eber S, Scherer F, Seger R, Gungor T.

Division of Immunology/Hematology/BMT, University Children's Hospital, Steinwiesstrasse 75, CH-8032 Zurich, Switzerland.

Background and Objectives. Hepatic veno-occlusive disease (VOD) remains a serious complication after hematopoietic stem cell transplantation (HSCT). Based on a protective effect of antithrombin III (ATIII) on endothelial cells, we assessed the incidence of VOD after pre-emptive ATIII replacement and the outcome of VOD after combined high dose defibrotide (DF) and ATIII therapy.

Design and Methods. This prospective case series comprised two phases. In the first phase 71 children did not receive any specific VOD prophylaxis or therapy (controls). In the second phase 91 children were given pre-emptive ATIII replacement in case of decreased ATIII activity (<=70%). If VOD was diagnosed clinically (according to modified Seattle criteria), high dose defibrotide (60 mg/day) and ATIII replacement therapy were combined. The severity of VOD was determined according to the degree of multiple organ dysfunction.

Results. The incidence of VOD was similar in both groups (13/71, 18% vs. 14/91, 15%). All 14 patients in the second group who developed VOD showed decreased ATIII activity not more than 1 day prior to the clinical diagnosis of VOD. The resulting short duration of pre-emptive ATIII therapy failed to prevent VOD (OR 0.96). None of the patients (n=72) maintaining normal ATIII levels developed VOD. All 14 patients with VOD who received combined therapy achieved complete remission and 93 % (13/14) survived until day +100, compared to six survivors (46%) in the first [control] group.

Interpretation and Conclusions. Pre-emptive ATIII administration did not alter the incidence of VOD. Combination treatment with ATIII and defibrotide was safe and yielded excellent remission and survival rates.
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DewDiligence

11/05/07 1:17 PM

#54317 RE: rkrw #22401

GENT Changes Primary Endpoint in Phase-3 VOD Trial

[GENT is off about 5% today. Comments?]

http://biz.yahoo.com/bw/071105/20071105005842.html?.v=1

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Monday November 5, 7:22 am ET

VILLA GUARDIA, Italy--(BUSINESS WIRE)--Gentium S.p.A. (NASDAQ: GENT ) announced today that it plans to submit to the U.S. Food and Drug Administration (the “FDA”) an amendment to the protocol for its ongoing Phase III trial of Defibrotide for the treatment of hepatic veno-occlusive disease ("VOD") with multi-organ failure. The decision to submit the amendment was prompted by recent discussions between the Company and the FDA.

Under the amended protocol, the primary endpoint for the 160 patient, historically controlled, multi-center Phase 3 study would change from survival at 100 days to complete response as defined by bilirubin < 2 mg/dL and the resolution of multiple organ failure. Survival at 100 days will be evaluated as a secondary endpoint. Under the original protocol, complete response was a secondary endpoint which also included the return of hepatomegaly to baseline and the resolution of right upper quadrant pain. However, these two additional criteria will now be eliminated due to their more subjective nature. Because data on both endpoints have been collected during the course of the trial, no additional changes to the size or design of the study are required.

Dr. Laura Ferro, Chairman and Chief Executive Officer of Gentium said, "We have spoken with various scientific and regulatory advisors, and we are comfortable with complete response as our primary endpoint. The impact of Defibrotide on complete response seen in our Phase II trial was highly correlated to survival at 100 days in severe VOD patients. To date, the trial has enrolled 65 of the expected 80 patients in its prospective arm and remains on track to report results during the first half of 2008.”

About VOD

Veno-occlusive disease (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 in 2002 approximately 45,000 people received blood and bone marrow transplants, which are types of SCT. 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 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 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. Food and Drug Administration to prevent and to treat VOD and Fast Track designation for the treatment of severe VOD in recipients of stem cell transplants.
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