Gentium S.p.A.'s Stem Cell Complication Drug Fails Trial

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* Defibrotide fails to meet trial goals

* Sees defibrotide best positioned in a prevention setting

* Plans to present full trial results Dec 5-8

* Shares fall as much as 22 pct
(Adds company comment, updates share movement)

BANGALORE, Aug 19 Italian biopharmaceutical
firm Gentium S.p.A. GENT.O said its experimental drug to
treat the blockage of small veins in the liver missed the main
goal of a late-stage study, sending its shares down as much as
22 percent.

Gentium shares recouped most of the losses to trade down 2
percent at $3.33 Wednesday afternoon on Nasdaq.

“We missed statistical significance but not by much,” Chief
Financial Officer Gary Gemignani said by phone.

“We thought it was going to be much worse than it turned
out and in fact this is very encouraging.”

Given the outcome of the data safety monitoring board’s
interim review announced in November, the company expected that
reaching the required statistical threshold for a single trial
would be difficult, Gemignani said in a statement.

The company was testing the drug, defibrotide, for severe
veno-occlusive disease (VOD), a potentially life-threatening
condition that typically occurs as a complication of stem-cell

Gemignani believes that the drug, which is also being
developed as a preventive treatment for VOD in pediatric
patients in Europe, will ultimately be best positioned in a
prevention setting.

“When you look at many of the studies that have been
conducted so far, there is a clear signal that the earlier the
patient gets the drug, the better the likelihood of an
outcome,” he said.


The main goal of the trial was to measure the complete
response at 100 days following a stem-cell transplant (SCT),
compared with patients receiving the best therapy and
supportive care available at the time.

While the standard measure for significance, called p-value
for the primary endpoint, should be 0.01 or less in a clinical
trial, defibrotide yielded a p-value of 0.015.

Defibrotide also failed to meet its secondary endpoint of
survival rate at 100 days and six months post SCT.

Gentium plans to present full results from the trial from
Dec. 5 to 8 at the American Society of Hematology Conference in
New Orleans.

Gemignani also said the company plans to report final
results from the pediatric prevention study in the upcoming

Shares of the company were down 6 percent at $3.20
Wednesday afternoon on Nasdaq. They had touched a low of $2.65
earlier in the session.

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(Reporting by Anand Basu in Bangalore; Editing by Deepak
Kannan and Vinu Pilakkott)

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