I only see failed product. Defibrotide has been a total failure for adult and pediatric VOD.
GENT even does not know how to do a randomized, well controlled clinical trial.
I don't think GENT is a biotech company. I treat it as a natural product company. Its defibrotide is RNA extract from pig. If pig RNA works, I guess RNA from my backyard tomato may work, too.
I group it with BNVI as natural product companies, not biotech. By the way, BNVI uses Chinese herb extract to treat woman's hot flashes. You know how much a woman has to take each time? "Please take 20g or more each time", The company said. Could you swallow bitter herb medicine like a chocolate bar?
Those two "biotech" will fail and close their doors within a year or so.
Defibrotide is a pro-drug from bovine lung (not pigs), that is it inspires the release of the endothelial component (plasminogen activator) which reacts with plasminogen in the blood to form plasmin, a clot digestor that your body has made (autologous). It works. Many publications on its effectivness but the business strategies have not addressed the best targets IMHO.
Defibrotide is derived from porcine mucosal and not from bovine lung.
Gentium web site: Defibrotide Is the sodium salt of a complex mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA. It has been shown that defibrotide has protective effects on vascular endothelial cells, particularly those of small vessels. It has extensive beneficial pharmacological effects owing to its antithrombotic, anti-inflammatory and antiischemic properties.
No one is responding to you since it's obvious you haven't done the least amount of research on Gentium and it's VOD product. I suggest you take a closer look at exactly what the results of the trials were. For a fast track approval by FDA the standards are very high, GENTIUM didn't miss the mark by much and the future looks very bright as a result of those trials. I have been in this one since .82 cents and I'm still holding, of course I sell on a high and buy back on a dip like we've just seen and in the end it's all about making money.
A failed trial is a failed trial. You need to do extra trails to make it a success if you can. But in this case, the "extract" failed for both adult and pediatric use. Unless you can find an underlining factor that cause the failure, and you can design a trial in better way, you will get failure again even you try another trial. So far, GENT has not find its underlining factor, yet. The management is just excusing themselves by saying "we may be out of luck." Could you be out of luck twice? how about a third time?
The cause of failure is not the luck. The RNA extract does not work. In addition, GENT has no money to do more trials.
I also see that the manufacturing and quality control will be a very tough part for this RNA extract from pig. See DSCO as a good sample.