Gentium: Defibrotide - Orphan Drug mit Blockbusterpotential - 500 Beiträge pro Seite

[Neoe]

Ich bin hier seit einigen Monaten dabei und möchte den Wert nun vorstellen.

Alleine heute 50 Prozent beim 5-fachen des normalen Volumens.

Marketcap: 18 Mio. USD
Ticker: GENT (NASDAQ)
Produkt: Defibrotide in Phase III gegen Venenverschlüsse bei Knochenmarkstransplantation. Kein anderes Medikament zugelassen und keines in klinischen Tests.
Marktpotential: Aktuelle Indikation rund 200-300 Mio. Euro weltweit
Weitere Indikationen, u.a. Hautkrebs, eröffnen weiteres, großes Potential.

Das Unternehmen ist nach wie vor äußerst risikoreich, aber mittlerweile Lichten sich die Wolken. Eine Finanzierung steht bevor und im Juli gibt es abschließende Ergebnisse zur Phase III von Defibrotide.

Die Situation bei Gentium ist durchaus komplex, wer die Zeit hat sich einzulesen, findet hier aber auch nach den Anstiegen der letzten Monate immer noch eine super Möglichkeit (meine Meinung).
Das Unternehmen ist auch alles andere als ein Start-Up. Eigene cGMP Einrichtungen zur Produktion bestehen. Defibrotide ist durch Compassionate Use bereits jahrelang erprobt in der angestrebten Indikation.

Die Opinion Leader in der Branche befinden Defibrotide für wirksam. Die European Bone Marrow and Transplant Organisation (EBMT, www.ebmt.org) hat die gerade laufende Phase III zu Teilen gesponsert. Die größte Studie die je von dieser Organisation unterstützt wurde.


Corporate Website: www.gentium.com

[donnerpower]

Ich bin hier seit einigen Monaten dabei und möchte den Wert nun vorstellen.

Alleine heute 50 Prozent beim 5-fachen des normalen Volumens.

..schön das jetzt damit kommst

[Woddy01]

Also nur mal so zur Falschinformation im Threadtitel:

Da steht was von Blockbusterpotential und in der Erläuterung steht was von geschätzen MarktUmsatzpotential 200-300 Millionen pro Jahr.

So das paßr ja schon mal vorne und hinten nicht.

Vom sogenannten Blockbusterpotential spricht man ab Umsätze von größer 1 Milliarde pro Jahr und nicht weniger.

Soll keinerlei basherei sein aber wenn schon sollte der Threadtitel nur wahres preisgeben oder??

Trotzdem viel Glück mit der Aktie.

[Neoe]

@woody:
Beachte doch bitte, dass ich weitere Indikationen mit einbeziehe, für die bereits Daten vorliegen. Insgesamt sind dann 1 Mrd. realistisch.

Gentium und Defibrotide bringen folgende Eigenheiten mit sich:

- Defibrotide über ein Jahrzent am Markt für Thrombosebehandlung (nur in Italien zugelassen)
- Medikament wurde illegal importiert von Apotheken in der ganzen Welt, da es Wirksamkeit bei Venenverschlüssen zeigte und anderen Indikationen, daher BLOCKBUSTERPOTENTIAL)
- Gentium wurde von der internationalen Ärzte-Community und Investoren aufgefordert, Studien durchzuführen
- Wichtig: Die Produktionsanlage ist die einzige ihrer Art und zudem ist das Medikament kaum durch Generika zu ersetzen ohne erneut alle Studienphasen I bis III zu durchlaufen. Das liegt an den besonderen Charakteristika von Defibrotide


@donnerpower:
In der Vergangenheit habe ich oft genug zu früh über einzelne Werte geschrieben. Das waren mir persönlich zu viele Totalausfälle. Gentium scheint gerade einige hohe Risiken aus dem Weg zu räumen, aber es gibt genug bestehende um hier weiterhin überdurchschnittliche Gewinne einzufahren. Wir werden sehen, wie es weitergeht.

[Neoe]

Antwort auf Beitrag Nr.: 37.306.746 von Neoe am 02.06.09 22:43:41

Gerade wieder 16 Prozent im Plus.

Scheint keinen zu interessieren.

[Neoe]

Antwort auf Beitrag Nr.: 37.306.746 von Neoe am 02.06.09 22:43:41

@donnerpower:

Gerade +40%. Habs gestern gesagt. Immer noch Grund zur Klage?

[Neoe]

Antwort auf Beitrag Nr.: 37.306.746 von Neoe am 02.06.09 22:43:41

Ich muss mich nochmals korrigieren hinischtlich des Marktpotentials.
Gentium schätzt den Markt für die Venenverschlussindikation bereits auf 1 Mrd:

Treatment of severe VOD represents an estimated global market opportunity of $600 million. Prevention
(prophylaxis) of VOD could replace treatment and become a $1 billion opportunity. Additional chemoprotective
indications could represent larger market opportunities.

(Auszug aus einer Corporate-Präsentation in 2006)

Ein Vermarktungsallianz mit Sigma-Tau besteht für Nordamerika. Allerdings bisher nur für die kleinere Indiaktion "schwerwiegender Venenverschluss" nicht die Prophylaxeindikation.

[Neoe]

Antwort auf Beitrag Nr.: 37.306.746 von Neoe am 02.06.09 22:43:41

Gentium Reports First Quarter Financial Results; Provides Financial and Clinical Update

* On Thursday June 4, 2009, 4:00 pm EDT

*
Buzz up!
* Print

Related:

* Gentium S.p.A

VILLA GUARDIA (COMO), Italy--(BUSINESS WIRE)--Gentium S.p.A. (NASDAQ: GENT - News) (the “Company”) today reported financial results for the quarter ended March 31, 2009.
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Financial Highlights

The Company reports its financial condition and operating results using U.S. Generally Accepted Accounting Principles (GAAP). The Company’s financial statements are prepared using the Euro as its functional currency. On March 31, 2009, €1.00 = $1.3261.

For the first quarter ended March 31, 2009 compared with the prior year’s first quarter:

* Total revenues were €1.01 million, compared with €2.69 million resulting from the discontinuation of the sale of Prociclide and Noravid (both forms of Defibrotide) through Crinos S.p.A. in the Italian market.
* Operating costs and expenses were €4.16 million, compared with €7.53 million
* Research and development expenses, which are included in operating costs and expenses, were €1.45 million, compared with €3.61 million
* Operating loss was €3.14 million, compared with €4.84 million
* Net loss was €2.96 million, compared with €6.08 million
* Basic and diluted net loss per share was €0.20 compared with €0.41 per share

Cash and cash equivalents were €4.62 million compared with €11.49 million as of December 31, 2008. During the first quarter ended March 31, 2009, the Company made a final installment payment of €4.0 million to Crinos S.p.A. related to the acquisition of marketing authorizations and trademarks for Prociclide and Noravid.

Clinical Update

At the end of the first quarter, Gentium announced preliminary top-line results from a Phase 2/3 European pediatric prevention clinical trial of Defibrotide at the Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT) in Göteborg, Sweden. The results from this clinical trial demonstrated a 40% reduction in incidence of VOD within 30 days after stem cell transplantation (SCT) with a statistical P-value of 0.0539. In addition, the analysis of data pursuant to the protocol (patients who completed 30 days in the study), which included 164 patients in the prophylaxis arm and 169 patients in the control arm, showed a 43% reduction rate of the incidence of VOD within 30 days and achieved a statistical P-value of 0.0366. The data also demonstrated the excellent safety profile of Defibrotide showing no difference in adverse events between the prophylaxis and control arms.

Subsequent to March 31, 2009, Defibrotide has become available on a named-patient basis throughout the European and Asia-Pacific markets. Defibrotide will be supplied by IDIS, a recognized leader in the management of named-patient programs. The product is being distributed to healthcare professionals for the treatment and prevention of veno-occlusive disease (VOD) post SCT. Through the named-patient program, Gentium is charging for the use of Defibrotide, but will continue to supply the product free-of-charge for use in clinical trials.

“The preliminary pediatric prevention data presented at the EBMT support the continued development of Defibrotide to treat and prevent veno-occlusive disease,” stated Dr. Laura Ferro, CEO of Gentium S.p.A. “With no other treatment options available for these patients, we have already seen strong numbers in requests for Defibrotide through our named-patient program in Europe and Asia and our compassionate use program in the United States. We strongly believe this underscores and validates the unmet need and market potential for Defibrotide and remain hopeful that we will obtain regulatory approval for Defibrotide in Europe and the U.S.”

“In a short five-week-period since the first sale of Defibrotide through the named-patient program in Europe and Asia-Pacific, we have generated over €676,000 in revenue,” stated Gary Gemignani, CFO of Gentium S.p.A. “We expect this program to generate cash flow that will allow us to operate beyond the end of this year.” “We are hopeful that this program along with the cost reduction measures already implemented will allow us greater flexibility as we continue to evaluate our financial and strategic options.”

About VOD

Veno-occlusive disease is a potentially life-threatening condition, which typically occurs as an important complication of stem cell transplantation. Certain high-dose chemo-radiation therapy regimens used as part of SCT can damage the lining cells of hepatic blood vessels and so result in VOD, a blockage of the small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so-called severe VOD). SCT is a frequently used treatment modality following chemotherapy or radiation treatments for hematologic cancers and other conditions in both adults and children. There is currently no approved agent for the treatment or prevention of 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, is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration and EMEA to prevent and to treat VOD and Fast Track designation by the U.S. FDA for the treatment of severe VOD in recipients of stem cell transplants.

[Neoe]

Ich führe weiter Selbstgespräche:

Kurs zieht gerade wieder an auf 1,94 $.

Ende Juni ist die HV, im Juli gibt´s Studienergebnisse für Defibrotide Phase III.

[EvertonFC]

Antwort auf Beitrag Nr.: 37.373.929 von Neoe am 11.06.09 17:21:56

Hallo Neoe

Keine Selbstgespräche mehr. Ich finde diese Aktie auch interessant und hoffe, dass sie nochmal zurückkommt.

Kapitalbedarf scheint man jedoch nach Aussagen des CEO aufgrund der Einnahmen aus Europa in diesem Jahr nicht mehr zu haben.

Wenn im Juli die Phase III Daten kommen sollen, wann kann mit einer Einreichung bei der FDA und späteren Zulassung gerechnet werden?

Ich sehe knapp 15 Mio Aktien aber weiss noch nicht wieviel es fully diluted sind. Hast du hierzu Informationen? Das Unternehmen hat keine Share Structure auf seiner Website.

Gruss
EFC

[Neoe]

Zur Share Structure kann ich dir sagen, dass sie aktuell rund 18,5 Mio. Shares ausgeben dürfen. Optionen sind wenn, dann zu Kursen weit über den aktuellen, auszuüben. Ansonsten gibt es wohl einige Shareholder Options, das übliche Verfahren.

In den USA wird zunächst wohl keine Zulassung beantragt. Man wird dort ebenfalls eine Treatment IND beantragen, wie bereits hier in Europa erfolgrecih durchgeführt mit IDIS.
Aber:
Bei der EMEA soll der Antrag Anfang nächsten Jahres eingerecht werden, sollten die endgültigen Ergebnisse Im Juli keine bösen Überraschungen mit sich bringen. Man weiß natürlich nie, aber ich hoffe das Beste.

[EvertonFC]

Die Aktie kommt bei kleinen Umsätzen wieder etwas zurück und hat glaube ich noch Luft nach unten. Das sagt jedoch nichts über die langfristige Chance von Gentium aus.

Die Phase III Studienergebnisse für Defibrotide stehen noch für diesen Monat an.

[Neoe]

Ende diesen Monats wird es spannend:

- Verkaufszahlen Defibotide -> sieht sehr gut aus
- Und natürlich: Defibrotide Phase III Daten

[Neoe]

@all:

Die vorher von mir gegebene Guideline, könnte sich nochmal verändern.

Wer genauere Informationen möchte:
http://files.shareholder.com/downloads/GNT/634104660x0x3075…

[Neoe]

Antwort auf Beitrag Nr.: 37.625.997 von Neoe am 22.07.09 20:14:01

Sehr positiv aufgenomme Nachricht (s.u.) und demnächst kommen (vermutlich sehr gute) Zahlen:


Gentium Announces Director Resignations and Other Governance Changes

VILLA GUARDIA (COMO), Italy, Aug 06, 2009 (BUSINESS WIRE) -- Gentium S.p.A. (Nasdaq: GENT) today announced that five of the eight members of its Board of Directors have resigned, triggering the automatic termination of the entire Board of Directors under Italian law. The Company's Board of Statutory Auditors will promptly call an ordinary shareholders' meeting to appoint the new Board of Directors following determination of the proposed slate of candidates. This meeting is expected to be held in October 2009. On the basis of Italian law, the Board of Statutory Auditors has temporarily assumed responsibility for the ordinary administration of the Company.

The director resignations followed the communication to the Board of Directors by Dr. Laura Ferro, the Chairperson of the Board, President and CEO of Gentium, of her intention to step down as CEO of the Company. The resigning directors, consisting of Gigliola Bertoglio, Luca Breveglieri, Marco Codella, Laura Ferro and Andrea Zambon, expressed differing reasons for their decisions. The reasons included dissatisfaction with the corporate governance and management of the Company and a sentiment that, once other directors were resigning, the shareholders should again be given the opportunity to elect a new Board of Directors to make the important strategic decisions facing the Company at this time. The Board of Directors had been recently elected at the annual ordinary shareholders' meeting held on June 30, 2009, prior to Dr. Ferro's communication of her intention to step down as CEO.

Addressing other governance matters, the Company also held an extraordinary shareholders' meeting on June 30, 2009 at which, among other actions taken, the elimination of the par value of the Company's Ordinary Shares was approved. In addition, the Company recently submitted a notice to Nasdaq of its decision to avail itself of the full exemption from Nasdaq's corporate governance rules available to foreign issuers, including with respect to Nasdaq's requirements for shareholder approval of securities issuances and the existence of Board committees. The par value change and Nasdaq exemption are intended to provide the Company with increased flexibility to pursue strategic and cost-saving initiatives.

Dr. Ferro stated "We have asked Gary Gemignani, our CFO, to play a more active role in the management of the Company in light of his strategic, financial and operational abilities and achievements and look forward to his continued leadership contributions to the Company through this transitional phase for our Board and Management. It is with mixed emotions that I have announced my intention to step down as CEO, but believe that it is the right decision for personal reasons. As to the reconstitution of the Board of Directors, I believe that only a full Board elected by the shareholders should make the important decisions required at this time. I am very proud of Gentium's accomplishments to date and remain confident in the tremendous potential of Defibrotide."

Mr. Gemignani commented "Laura Ferro has served as a founder and strong advocate for Gentium for over 15 years. I look forward to building upon her valued efforts towards the development of Defibrotide and to explore strategic options for the Company. I also look forward to working with Dr. Ferro and the Board of Statutory Auditors to help identify director candidates that can bring additional expertise and perspective to the Company. As to clinical trials, the Company plans to report final data from its U.S. pivotal and European pediatric trials in the coming months, as well as communicate our longer term development and regulatory strategy."

About Gentium

Gentium, S.p.A., located in Como, Italy, is a biopharmaceutical company focused on the research, discovery and development of drugs to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate, is an investigational drug that has been granted Orphan Drug status and Fast Track Designation by the U.S. FDA to treat Severe VOD and Orphan Medicinal Product Designation by the European Commission both to treat and to prevent VOD.

[Neoe]

Selten schlechte News gesehen, die so verdammt gut waren. Wer die Hintergründe kennt, weiß was das zu bedeuten hat. Gentium wird eine absolute Bombe werden:


Gentium Reports Top Line Results from the Phase III Treatment Trial of Defibrotide for Severe Veno-Occlusive Disease

VILLA GUARDIA (COMO), Italy--(BUSINESS WIRE)--Gentium S.p.A. (NASDAQ: GENT - News) today announced top-line results from a historically controlled, multicenter, open label, Phase III trial designed to evaluate the safety and efficacy of 25 mg/kg/day of Defibrotide for the treatment of severe veno-occlusive disease (sVOD) in hematopoietic stem cell transplant (SCT) patients. The results demonstrate strong trends in favor of the Defibrotide-treated patients for complete response and survival, but did not reach the protocol-specified levels of significance for the primary and secondary endpoints at 100 days. With regard to safety, adverse events were well balanced between the historical control and treatment arms. The Company plans to present full results from this trial at the American Society of Hematology Conference in New Orleans, LA, December 5-8, 2009.
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The primary endpoint of the trial was complete response at 100 days following SCT and utilized historical controls (patients who in the past received the best therapy and supportive care available at the time, but not Defibrotide) as a comparator. Secondary endpoints included survival rate at 100 days and six months post SCT. The historical control database was generated through a sequential, retrospective medical chart review, with final selection of the control group performed by an independent medical review committee (MRC). The MRC remained blinded to patient outcome data throughout the duration of the trial. Per the study protocol, data in the primary efficacy analysis were adjusted by quintiles of propensity score based on four stratification variables (allogeneic/autologous SCT, adult/pediatric, one/two+ SCTs, and ventilator/dialysis dependence) to aid in obtaining balance between the treatment and historical control arms in a non-randomized trial. As a stand-alone trial utilizing a historical control arm, the study protocol specified a p-value of less than or equal to 0.01 for the primary endpoint to achieve statistical significance, while the secondary endpoints required a p-value of less than or equal to 0.05, the more common threshold for statistical significance.

123 patients with symptoms consistent with VOD were identified and then reviewed for eligibility in the historical control arm by the MRC, with 32 cases selected as having an unequivocal diagnosis of sVOD (graft versus host disease was ruled out) and met all protocol-required entry criteria. 102 patients were enrolled in the Defibrotide treatment group and baseline characteristics were balanced between the two arms.

For the primary efficacy analysis on an intent to treat basis, 24% of patients in the Defibrotide arm compared to 9% of patients in the historical control arm achieved complete response at 100 days (p-value = 0.015). For the secondary efficacy analysis on an intent to treat basis, 38% of patients in the Defibrotide arm compared to 25% of patients in the historical control arm demonstrated survival at 100 days (p-value = 0.051). Thus, while the primary endpoint achieved a p-value less than 0.05 and the secondary endpoint showed a strong trend towards statistical significance, neither reached the level of significance required in the protocol for proof of efficacy with a single study.

“I am encouraged by the results of this trial, especially given the extremely sick patient population that was enrolled,” said Dr. Paul Richardson, Clinical Director of the Dana-Farber Cancer Institute’s Jerome Lipper Multiple Myeloma Center and principal investigator of the trial. “The data generated from this trial confirms the activity of Defibrotide seen in earlier studies, and supports the benefit of Defibrotide for the treatment of sVOD in improving complete response rates and survival, as well as its potential in less advanced stages of the disease.”

“Given the outcome of the data safety monitoring board’s interim review announced in November of last year, we expected that reaching the required statistical threshold for a single trial would be difficult,” said Gary Gemignani, Executive Vice-President and Chief Financial Officer. “We are pleased that the data are compelling and believe the results place us in a strong position to continue discussions with the FDA and others regarding next steps toward a regulatory filing. Additionally, we plan on announcing final results from our randomized, pediatric prevention study in the upcoming weeks.”

[EvertonFC]

Ich habe mir nochmal die Präsentation angeschaut.

Wichtig für eine Beurteilung ist, dass 40% des Marktpotentials für dieses Medikament in Nord- und Südamerika liegt und hier erhält Gentium nur 38% der Erträge. Insgesamt ist das Ertragspotential verglichen mit der Mcap trotzdem gross.

Investoren brauchen aber noch einen langen Atem. Die Confirmatory Studies, die zu einer Zulassung in EU und USA führen sollen werden in 2011 beginnen mit einer möglichen Zulassung durch die FDA in 2013.

Die Studienergebnisse der letzten Jahre sehen gut aus, wobei es mir auch zunächst schwer gefallen ist das letzte Ergebnis richtig zu bewerten.

Als Near Term Catalyst werden die Ergebnisse der Pediatric Prevention Study dienen. Aufgrund der Vorergebnisse ist ein positiver Ausgang wahrscheinlich.

[Neoe]

Antwort auf Beitrag Nr.: 37.820.836 von EvertonFC am 20.08.09 19:31:36

@everton:

Du hast insofern recht, dass Gentium in der Indikation Treatment of severe VOD "nur" 38% bekommen würde.

Die Indikation "Prevention of VOD" ist aber nicht auslizenziert. Das ist sehr wichtig, da Prevention der deutlich größere Markt ist und diese Indikation zukünftig verfolgt wird.

Das Filing im EU-Raum wird Anfanf 2010 kommen. Davon gehe ich aufgrund der Daten und der Notwendigkeit des Mediakmentes aus. Dann kommt sicher die Confirmatoy Study.

[EvertonFC]

Antwort auf Beitrag Nr.: 37.830.620 von Neoe am 21.08.09 23:14:41

Danke für deine Ergänzung

Wenn ich die Präsentation richtig gelesen habe wird aber auch für eine offizielle Zulassung in der EU die Confirmatoy Study notwendig sein.

Etwas ungewöhnlich ist, dass Gentium als ein italienisches Unternehmen an der Nasdaq und nicht an der Heimatbörse gelistet ist. Ist bekannt, warum sich für diese Konstellation entschieden hat?

[Neoe]

Gentium Reports Second Quarter Financial Results; Provides Financial and Clinical Update

VILLA GUARDIA (COMO), Italy, Sep 17, 2009 (BUSINESS WIRE) -- Gentium S.p.A. (NASDAQ: GENT) today reported financial results for the second quarter ended June 30, 2009.

Financial Highlights

Gentium S.p.A., or the Company, reports its financial condition and operating results using U.S. Generally Accepted Accounting Principles (GAAP). The Company's financial statements are prepared using the Euro as its functional currency. On June 30, 2009, EUR 1.00 = $1.4134.

For the second quarter ended June 30, 2009 compared with the prior year's second quarter:

* Total revenues were EUR 2.61 million, compared with EUR 1.86 million. The increase was primarily attributable to the launch of the named-patient program for Defibrotide throughout the European and Asia-Pacific markets by IDIS Limited.
* Operating costs and expenses were EUR 3.02 million, compared with EUR 6.50 million.
* Research and development expenses, which are included in operating costs and expenses, were EUR 0.36 million, compared with EUR 1.76 million. Research and development expenses for second quarter 2009 and 2008 are net of EUR 0.71 million and EUR 1.14 million, respectively, of government grants in the form of a tax credit.
* Operating loss was EUR 0.41 million, compared with EUR 4.63 million.
* Net loss was EUR 0.49 million, compared with EUR 4.53 million.
* Basic and diluted net loss per share was EUR 0.03 compared with EUR 0.30 per share.

For the six months ended June 30, 2009 compared with the comparable prior-year period:

* Total revenues were EUR 3.62 million, compared with EUR 4.55 million.
* Operating costs and expenses were EUR 7.17 million, compared with EUR 14.03 million. Research and development expenses, which are included in operating costs and expenses, were EUR 1.81 million, compared with EUR 5.37 million. Research and development expenses for second quarter 2009 and 2008 are net of EUR 0.71 million and EUR 1.14 million, respectively, of government grants in the form of a tax credit.
* Operating loss was EUR 3.55 million, compared with EUR 9.48 million.
* Interest income (expense), net, was (EUR 0.07) million, compared with EUR 0.16 million.
* Net loss was EUR 3.45 million, compared with EUR 10.61 million.
* Basic and diluted net loss per share was EUR 0.23 compared with EUR 0.71 per share.

Cash and cash equivalents were EUR 1.36 million compared with EUR 11.49 million as of December 31, 2008. In March 2009, the Company made a final installment payment of EUR 4.0 million to Crinos S.p.A related to the acquisition of marketing authorizations and trademarks for Prociclide and Noravid. Excluding the payment to Crinos, net cash used in operating activities for the six-month-period ended June 30, 2009, would have amounted to EUR 5.31 compared to EUR 8.65 million for the same period of 2008. The reduction in net cash used in operating activities between periods reflects a decrease in spending in R&D activities coupled with increased cash flows from the named-patient program. The Company also utilized Cassa Integrazione, a mechanism available to companies in Italy to temporarily lay off employees with the Italian government funding a portion of the costs of the employees during the layoff-period.

The Company anticipates that its current cash will meet its operating requirements through January of 2010. However, in order for the Company to continue as a going concern beyond this point, the Company will likely need to obtain capital from external sources.

Clinical Update

The Company has submitted abstracts for the American Society of Hematology Conference in New Orleans, LA, December 5-8, 2009. Data was submitted for the Phase III historically controlled trial for the treatment of severe VOD and for the Phase II/III European prevention of VOD trial in pediatric stem cell patients. The results from both studies are encouraging and demonstrate strong trends in favor of the Defibrotide-treated patients in both the treatment of severe VOD and the prevention of VOD. In the treatment study, 24% of patients in the Defibrotide arm compared to 9% of patients in the historical control arm achieved complete response at 100 days (p-value = 0.015) while 38% of patients in the Defibrotide arm compared to 25% of patients in the historical control arm demonstrated survival at 100 days (p-value = 0.051). In the prevention study, a 40% reduction in incidence of VOD within 30 days after stem cell transplantation (SCT) was observed in the prophylaxis arm (Intent to Treat p-value = 0.054, Per Protocol p-value = 0.037). The Company plans to announce further data from the pediatric prevention study in the upcoming weeks.

"Gentium continues to move forward with the development of Defibrotide to treat and prevent VOD, a disease for which there is currently no other viable treatment option," stated Gary Gemignani, Executive Vice President and Chief Financial Officer of Gentium S.p.A. "We believe that the results from our Phase II/III European pediatric prevention trial and the Phase III historically controlled treatment trial in the U.S. demonstrate the activity of Defibrotide that has consistently been seen in numerous investigator-sponsored studies. We have been able to extend our cash reserves through the margins generated from the named-patient program and our cost reduction measures. We look forward to working collaboratively with the Company's incoming Board of Directors as we pursue strategic financing options to provide the capital necessary to further the development of Defibrotide."

Operating Results

Total Product sales, net for the six-month-period ended June 30, 2009 were EUR 3.53 million compared to EUR 2.91 million for the same period in 2008, an increase of EUR 0.62 million. The increase was primarily due to the launch in April 2009 of Defibrotide via a named-patient program administered by IDIS Limited throughout the European and Asia-Pacific markets. Named-patient program gross sales for the period from April 21, 2009 through June 30, 2009 amounted to EUR 1.21 million or EUR 1.04 million net of service payments to IDIS. Through August 31, 2009, the Company has generated EUR 2.3 million in gross revenue, or EUR 2.0 million in net revenue, through the named-patient program.

Sales to a related party, Sirton, for the six-month-period ended June 30, 2009 and 2008 represented 6% and 12% of the total product sales, respectively. The decrease in sales to Sirton is primarily due to the fact that the Company entered into direct sales agreements with Sirton's customers in order to mitigate the risk associated with Sirton's poor financial condition.

Sales to third parties decreased to EUR 2.30 million for the six-month-period ended June 30, 2009 compared to EUR 2.36 million for the same period in 2008. The six-month-period ended June 30, 2009 did not include sales of Prociclide and Noravid (both forms of Defibrotide) due to the discontinuation of the sale of these products in the Italian market, which in the prior year period accounted for revenues of EUR 1.13 million.

Other revenues, primarily cost-sharing revenues from Sigma-Tau, were EUR 0.01 million for the six-month-period ended June 30, 2009, compared to EUR 1.64 million for the same period in 2008. Fluctuation versus the prior period is primarily due to timing on the recognition of reimbursement of certain costs incurred for the Company's Phase III clinical trial of Defibrotide to treat Severe VOD.

Cost of goods sold was EUR 2.00 million for the six-month-period ended June 30, 2009 compared to EUR 2.95 million for the same period in 2008. Cost of goods sold as a percentage of product sales, net was 57% for the six-month-period ended June 30, 2009 compared to 102% for the same period in 2008. The percentage decrease is primarily due to (i) higher margin on Defibrotide sold through the named-patient program, (ii) price increases in the active pharmaceutical ingredient business, and (iii) discontinuation of negative margins associated with Prociclide and Noravid. The Company has fully expensed, during the prior six-month-period, costs associated with the production of Defibrotide; therefore, costs of goods sold do not reflect the full costs of production because a portion of the active pharmaceuticals ingredients, labor and overhead costs incurred to produce Defibrotide sold through the named-patient program were previously expensed. Additionally, the higher percentage for the six-month-period ended in 2008 was primarily due to the fact that product sales to Sirton were not recognized after March 2008, due to Sirton's poor financial condition and concerns over the collectability of such receivables.

The Company incurred research and development expenses of EUR 1.81 million for the six-month-period ended June 30, 2009 compared to EUR 5.37 million for the same period in 2008, which are net of EUR 0.71 million and EUR 1.14 million, respectively, of government grants in the form of a tax credit. Research and development expenses were primarily for the development of Defibrotide to treat and prevent VOD. The decrease from the comparable period in 2008 is primarily due to lower development costs related to the treatment trial as this trial has been completed.

General and administrative expenses were EUR 2.76 million for the six-month-period ended June 30, 2009 compared to EUR 4.80 million for the same period in 2008. General and administrative expenses from the prior six-month-period reflect the establishment of an allowance for doubtful accounts of EUR 1.50 million which was partially released for EUR 0.34 million in 2009. The decrease in general and administrative expense is also attributable to lower payroll costs due to the temporary layoffs under the Cassa Integrazione program during the six-month period ended June 30, 2009.

Foreign currency exchange gain (loss) is primarily due to re-measurement of U.S. dollar cash balances. The positive result between 2009 and 2008 is due to a more favorable exchange rate in 2009 and a lower cash balance in 2009 versus 2008.

Net loss was EUR 3.45 million for the six-month-period ended June 30, 2009 compared to EUR 10.61 million for the same period in 2008. The difference was primarily due to lower research and development expenses, offset by an increase in higher margin from product sales through the named-patient program.

In addition, the Company has moved forward on two key strategic initiatives. In August 2009, the Company received formal notification from the Italian Health Authority (AIFA) that the marketing authorizations for Prociclide and Noravid had been withdrawn. This move was important because it allows the Company to re-position and re-launch the VOD formulation of Defibrotide in Europe. Also, the Company has received approval from the U.S. FDA to move forward with Cost Recovery for the Treatment IND program. Under current regulations, companies are allowed to recover certain costs related to the implementation of a Treatment IND. The Company plans to launch the Cost Recovery program in the fourth quarter 2009.

About VOD

Veno-occlusive disease is a potentially life-threatening condition, which typically occurs as an important complication of stem cell transplantation. Certain high-dose chemo-radiation therapy regimens used as part of SCT can damage the lining cells of hepatic blood vessels and so result in VOD, a blockage of the small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so-called severe VOD). SCT is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children. There is currently no approved agent for the treatment or prevention of VOD in the U.S. or the EU.

About Gentium

Gentium, S.p.A., located in Como, Italy, is a biopharmaceutical company focused on the research, discovery and development of drugs to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate, is an investigational drug that has been granted Orphan Drug status and Fast Track Designation by the U.S. FDA to treat Severe VOD and Orphan Medicinal Product Designation by the European Commission both to treat and to prevent VOD.

[Neoe]

Antwort auf Beitrag Nr.: 37.832.513 von EvertonFC am 22.08.09 19:42:24

@Everton:

Zu deiner Frage. Gentium war ein kleines Familienunternehmen, das meinem Verständnis nach von Investoren überzeugt wurde, Defibrotide zu entwickeln. Diese Investoren kamen wohl überwiegend aus den USA. Daher das NASDAQ-Listing.

[Neoe]

Ich sehe hier goldene Zeiten auf Gentium (und hoffentlich auch mich) zukommen:

Gentium Reports Third Quarter Financial Results; Provides Financial Update

VILLA GUARDIA (COMO), Italy, Nov 30, 2009 (BUSINESS WIRE) -- Gentium S.p.A. (NASDAQ: GENT) (the "Company") today reported financial results for the quarter ended September 30, 2009.

Financial Highlights

Gentium S.p.A., or the Company, reports its financial condition and operating results using U.S. Generally Accepted Accounting Principles (GAAP). The Company's financial statements are prepared using the Euro (EUR ) as its functional currency. On September 30, 2009, EUR 1.00 = $1.4643.

For the third quarter ended September 30, 2009 compared with the prior year's third quarter:

* Total revenues were EUR 2.50 million, compared with EUR 1.85 million. The increase was primarily attributable to named-patient sales of Defibrotide throughout the European and Asia-Pacific markets, offset by a decrease in other revenues, mainly cost sharing revenues from Sigma-Tau.
* Operating costs and expenses were EUR 3.49 million, compared with EUR 8.86 million. Operating costs and expense for the three-month period ended September 30, 2008 included a write down of acquired assets of EUR 3.05 million.
* Research and development expenses, which are included in operating costs and expenses, were EUR 0.85 million, compared with EUR 2.51 million. Lower costs were due to the completion in enrollment in both the pediatric prevention and treatment studies as well as cost-reduction initiatives.
* Operating loss was EUR 0.99 million, compared with EUR 7.02 million. Operating losses in 2008 included a write down of acquired assets of EUR 3.05 million. The lower operating loss for the nine months ended September 30, 2009 was due to the higher revenues and gross margins attributable to named-patient sales and lower general and administrative and research and development expenses.
* Net loss was EUR 1.02 million, compared with EUR 5.85 million.
* Basic and diluted net loss per share was EUR 0.07 compared with EUR 0.39 per share.

For the nine-month period ended September 30, 2009 compared with the comparable prior-year period:

* Total revenues were EUR 6.12 million, compared with EUR 6.40 million.
* Operating costs and expenses were EUR 10.66 million, compared with EUR 22.89 million.
* Research and development expenses, which are included in operating costs and expenses, were EUR 2.66 million, compared with EUR 7.87 million. Research and development expenses for the nine-month periods ended September 30, 2009 and 2008 are net of EUR 0.76 million and EUR 0.79 million, respectively, of government grants in the form of a tax credit, accrued as a reduction of research and development expenses.
* Operating loss was EUR 4.54 million, compared with EUR 16.49 million.
* Interest income (expense), net, was (EUR 0.09) million, compared with EUR 0.17 million.
* Net loss was EUR 4.48 million, compared with EUR 16.46 million.
* Basic and diluted net loss per share was EUR 0.30, compared with EUR 1.10 per share.

Cash and cash equivalents and short term available for sale securities as of September 30, 2009 were EUR 0.97 million compared with EUR 11.49 million as of December 31, 2008. In March 2009, the Company made a final installment payment of EUR 4.0 million related to the acquisition of marketing authorizations and trademarks for Prociclide and Noravid (previous forms of Defibrotide sold only in Italy). Net cash used in operating activities for the nine-month period ended September 30, 2009 was EUR 5.68million compared with EUR 11.09million for the same period in 2008. The reduction in net cash used in operating activities between the two periods reflects a decrease in spending on development activities coupled with increased cash flows from the named-patient program. The Company also utilized Cassa Integrazione, a mechanism available to companies in Italy to temporarily lay-off employees, with the Italian government funding a portion of the costs of the employees during the lay-off period.

In light of increased revenues from the named-patient program and cost-reduction initiatives, among other factors, the Company currently anticipates that its cash will meet its operating requirements through June 2010. However, in order for the Company to continue as a going concern beyond this point, the Company will likely need to obtain capital from external sources.

"We are pleased with the revenue that continues to be generated through the named-patient program," said Gary Gemignani, Executive Vice-President and Chief Financial Officer. "We have also initiated a cost recovery program, which is being administered by US Oncology, for distribution of Defibrotide through our expanded access program in the U.S., which we believe, along with the named-patient program, continues to demonstrate the demand that exists for Defibrotide. We continue to evaluate our strategic options and look forward to continuing our efforts toward obtaining regulatory approval to market Defibrotide."

Company Update

Gentium reported top-line results from a historically controlled, multicenter, open label, Phase III trial designed to evaluate the safety and efficacy of 25 mg/kg/day of Defibrotide for the treatment of severe veno-occlusive disease (sVOD) in hematopoietic stem cell transplant (SCT) patients. The results demonstrated strong trends in favor of the Defibrotide-treated patients for complete response and survival, but did not reach the protocol-specified levels of significance for the primary and secondary endpoints at 100 days. Gentium will announce final results from this trial and the Company's Phase II/III Pediatric Prevention trial in two oral presentations at the American Society of Hematology Conference (ASH) on December 7, 2009 in New Orleans.

In the third quarter, Gentium also announced that five of the eight members of its Board of Directors resigned, thereby triggering automatic termination of its entire Board of Directors under Italian law. Following an ordinary meeting of shareholders, the Company recently announced the election of a new Board of Directors and the appointment of Dr. Khalid Islam as Interim CEO.

Operating Results

Net product sales for the nine-month period ended September 30, 2009 were EUR 5.99 million compared with EUR 4.12 million for the same period in 2008, an increase of EUR 1.87 million or 45%. The increase was primarily due to the initiation of a named-patient program in April 2009 to distribute Defibrotide throughout the European and Asia-Pacific markets. Named-patient program gross sales for the period from April 21, 2009 through September 30, 2009 amounted to EUR 2.82 million or EUR 2.43 million net of related service payments. Through October 30, 2009, the Company has generated EUR 3.54 million in gross revenue, or EUR 3.05 million in net revenue, through the named-patient program.

Sales to a related party, Sirton, for the nine-month periods ended September 30, 2009 and 2008 represented 3% and 13% of the total product sales, net, respectively. The decrease in sales to Sirton is primarily due to the fact that the Company entered into direct agreements with Sirton's customers in order to mitigate the risk associated with Sirton's poor financial condition.

Sales to third parties decreased to EUR 3.37 million for the nine-month period ended September 30, 2009 compared with EUR 3.57 million for the same period in 2008. The nine-month period ended September 30, 2009 does not includes sales of Prociclide and Noravid (due to discontinuation of such sale), which in the prior period amounted to EUR 1.72 million. The discontinuation of sales of Prociclide and Noravid was offset for the current nine-month period due to higher volume of sales to third parties and price increase.

Other revenues, primarily cost-sharing revenues from Sigma-Tau, were EUR 0.13 million for the nine-month period ended September 30, 2009, compared with EUR 2.28 million for the same period in 2008. Fluctuation versus the prior period is primarily due to timing on the recognition of reimbursement of certain costs incurred for the Company's Phase III clinical trial of Defibrotide to treat Severe VOD.

Cost of goods sold was EUR 3.13 million for the nine-month period ended September 30, 2009 compared with EUR 4.32 million for the same period in 2008. Cost of goods sold as a percentage of product sales was 52% for the nine-month period ended September 30, 2009 compared with 105% for the same period in 2008. The percentage decrease is primarily due to (i) higher margins on Defibrotide sold through the named-patient program, (ii) price increases in the active pharmaceutical ingredient business, and (iii) discontinuation of negative margins associated with Prociclide and Noravid. The Company has fully expensed, during the prior nine-month period, costs associated with the production of Defibrotide; therefore, costs of goods sold do not reflect the full costs of production, because a portion of the active pharmaceuticals ingredients, conversion and labor and overhead costs incurred to produce Defibrotide sold through the named-patient program were previously expensed. Additionally, the higher percentage for the nine-month period ended in 2008 was primarily due to the fact that product sales to Sirton were not recognized after March 2008, due to Sirton's poor financial condition and concerns over the collectability of such receivables. Cost of goods sold as of September 30, 2009 and 2008 include an allowance on inventory of EUR 0.1 million and EUR 0.06 million, respectively.

The Company incurred research and development expenses of EUR 2.66 million for the nine- month period ended September 30, 2009 compared with EUR 7.87 million for the same period in 2008. Research and development expenses for the nine-month periods ended September 30, 2009 and 2008 are net of EUR 0.76 million and EUR 0.79 million, respectively, of government grants, in the form of a tax credit, which have been accrued as a reduction of expense. Research and development expenses were primarily for the development of Defibrotide to treat and prevent VOD. The decrease from the comparable period in 2008 is primarily due to lower stock based compensation costs and development expenses (including contract research organization expenses and regulatory activities) following the completion of enrollment of the treatment trial, as well as cost-reduction initiatives.

General and administrative expenses were EUR 3.96 million for the nine-month period ended September 30, 2009 compared with EUR 6.36 million for the same period 2008. General and administrative expenses from the prior nine-month-period reflect the establishment of an allowance for doubtful accounts of EUR 1.77 million, which was partially released for EUR 0.41 million in 2009. The decrease in general and administrative expense is also attributable to lower stock based compensation costs and a decrease in payroll costs due to the temporary layoffs under the Cassa Integrazione program during the nine-month period ended September 30, 2009. For the nine-month period ended September 30, 2009, general and administrative expenses include recognition of service fees for named-patient program of EUR 0.39 million.

Foreign currency exchange gain (loss) is primarily due to remeasurement of U.S. dollar cash balances. The positive result between 2009 and 2008 is due to a more favorable exchange rate in 2009 between the Euro and U.S. dollar and a lower cash balance in 2009 versus 2008.

Interest income (expense), net amounted to (EUR 0.09) million and EUR 0.17 million for the nine- month periods ended September 30, 2009 and 2008, respectively. Gross interest income amounted to EUR 0.03 million and EUR 0.43 million for the nine-month periods ended September 30, 2009 and 2008, respectively, a decrease of EUR 0.40 million. The decrease is a result of a lower amount of invested funds in the nine-month period ended September 30, 2009 and a decrease in interest rates. Interest expense totalled EUR 0.12 million and EUR 0.26 million for the nine-month periods ended September 30, 2009 and 2008, respectively, a decrease of EUR 0.13 million attributable to a fluctuation in interest rate and decrease of principal debt outstanding.

Net loss was EUR 4.48 million for the nine-month period ended September 30, 2009 compared with EUR 16.46 million for the same period in 2008. The difference was primarily due to lower research and development expenses, general and administrative expenses, and other income and revenues, as well as no write-down of acquired assets, which in 2008 amounted to EUR 3.5 million, offset by an increase in margin from product sales through the named-patient program.

About VOD

Veno-occlusive disease is a potentially life-threatening condition, which typically occurs as an important complication of stem cell transplantation (SCT). Certain high-dose chemo-radiation therapy regimens used as part of SCT can damage the lining cells of hepatic blood vessels and so result in VOD, a blockage of the small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so-called severe VOD). SCT is a frequently used treatment modality following chemotherapy or radiation treatments for hematologic cancers and other conditions in both adults and children. There is currently no approved agent for the treatment or prevention of 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, is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration and EMEA to prevent and to treat VOD and Fast Track designation by the U.S. FDA for the treatment of severe VOD in recipients of stem cell transplants.

[Neoe]

Aber das Ding interessiert euch nach wie vor nicht.

Hier noch die Abstracts für das ASH-Meeting. Der Teufel steckt im Detail...statistisch signifikante Reduzierung der All-cause-mortality (Secondary Endpoint), darüber hinaus ist vor allem die signifikante Reduktion von Graft-versus-Host-Disease hervorzuheben. Gentium wird in den kommenden Jahren richtig abgehen, solange nur das Management keinen Mist macht:

ABSTRACT 1:
653 Defibrotide (DF) for the Prevention of Hepatic Veno-Occlusive Disease (VOD) in Pediatric Stem Cell Transplantion: Results of a Prospective Phase II/III Randomized, Multicenter Study
Oral and Poster Abstracts
Oral Session: Clinical Care - Transplantation Regimen Toxicities and Engraftment: Novel Approaches to Predict, Assess and Reduce Transplant Related Organ Toxicities
Monday, December 7, 2009: 5:30 PM
353-355 (Ernest N. Morial Convention Center)

Background: Hepatic VOD is a life-threatening complication following SCT with a particularly high incidence in children. Development of VOD is one of the most common causes of early death after SCT. DF (Gentium SpA), a polydisperse oligonucleotide, demonstrates a protective effect on vascular endothelial cells in vitro. Small non-randomized trials to assess DF for the prophylaxis of VOD were promising without significant anticoagulant effects.
Methods: Eligibility criteria included pts <18 years with myeloablative SCT and at least 1 of the following high risk criteria for VOD: conditioning with busulfan and melphalan, pre-existing liver disease, 2nd myeloablative transplant, allo-SCT for leukemia in 2nd relapse, macrophage activating syndromes, prior abdominal irradiation, prior gemtuzumab, osteopetrosis, and adrenoleukodystrophy. Pts were prospectively randomized to the control arm (no prophylactic DF) or to receive DF 25mg/kg/day IV from the start of conditioning until D+30 post SCT. All pts diagnosed with VOD received DF for treatment. Primary endpoint: incidence of hepatic VOD by D+30 using modified Seattle criteria (2 or more of the following: bilirubin > 2 mg/dL, hepatomegaly, ascites and/or unexplained weight gain > 5%). VOD was assessed by physical exam; hepatomegaly and ascites were confirmed by abdominal ultrasound. A blinded independent review committee of 3 expert hematologists confirmed the diagnosis of VOD. Although the study was not powered to assess mortality, a composite score was assessed as a secondary endpoint that incorporated VOD-associated toxicity (respiratory failure, renal failure, encephalopathy) and mortality. Incidence and severity of graft versus host disease (GvHD) was assessed. As the true incidence of VOD in this population was unknown, the trial incorporated a planned adaptive interim analysis to be reviewed by an independent DSMB.
Results: Based on the recommendations of the DSMB, 360 pts were enrolled between January 2006 and January 2009 by 28 centers in the EU and Israel. An Intent-to-Treat (ITT) analysis was performed on all randomized pts who signed informed consent (DF: 180; control: 176). Median age was 4.8 years; 24% infants, 52% children (ages 2-11 years) and 23% adolescents. 41% were female, 59% male. 68% were allo-, 31% auto-SCT. There were no significant differences between the two arms in disease types or risk factors. Ninety-three percent (93%) of the patients completed the primary endpoint at day +30. In the ITT analysis, 12% (22/180) of the pts of the DF arm and 20% (35/176) of the control group developed VOD by D+30 (P=0.054); in the PP analysis, the VOD incidence was 12% (20/164) vs 21% (35/169) (P=0.037). VOD was experienced by 23% of the infants, 14% of the children and 13% of the adolescents. The composite score (assessing VOD morbidity and mortality) was significantly in favor of the DF arm (P=0.034). Significantly less acute GvHD by D+100 was reported in the DF pts (32% (57/180) vs 43% (75/176); P=0.023 by Wilcoxon test). Observation of VOD in either arm led to a higher mortality: mortality of pts with VOD equaled 24.6% (14/57) compared to 7% in pts without VOD (21/299). Renal failure was observed in 1% (2/180 pts) of DF pts vs 6% (10/176) of the control (P=0.017); respiratory failure was observed in 7% vs 9% (NS); and encephalopathy in 1% vs 2% (NS). SAEs were experienced by 58% of the DF pts vs 59% of the control, including infections (24% vs 27%) and respiratory disorders (12% vs 9%); 9 hemorrhagic events were seen in the DF arm compared to 21 in the control.
Conclusions: This Phase II/III randomized study demonstrates the efficacy and safety of DF in preventing VOD in pediatric pts at high risk of VOD. Use of prophylactic DF results in a 40% reduction in the incidence of VOD. Consistent with the role of DF in endothelial protection, both renal failure and acute GvHD were significantly lower in the DF arm. Safety of DF was confirmed by lack of significant toxicity (including hemorrhage). DF can be recommended for the prevention of VOD in this high risk population.


ABSTRACT 2:
654 Defibrotide (DF) in the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF) Following Stem Cell Transplantation (SCT): Results of a Phase 3 Study Utilizing a Historical Control
Oral and Poster Abstracts
Oral Session: Clinical Care - Transplantation Regimen Toxicities and Engraftment: Novel Approaches to Predict, Assess and Reduce Transplant Related Organ Toxicities
Monday, December 7, 2009: 5:45 PM
353-355 (Ernest N. Morial Convention Center)

Background: Phase 2 clinical trials of DF in the treatment of severe VOD/MOF have demonstrated a complete response (CR) in 36-46% of patients (pts) with encouraging overall survival and tolerability (Richardson Blood 2002; Richardson ASH 2006). Given the life-threatening nature of VOD/MOF, a trial randomizing pts to placebo or best supportive care was considered but rejected. A phase 3 trial, comparing DF in the treatment of VOD/MOF post-SCT to a contemporaneous historical control (HC) was therefore performed. The HC was created using a sequential review of medical charts starting 6 months prior to use of DF at each center.
Methods: Eligible pts met Baltimore VOD criteria by D+21 (total bilirubin ≥ 2.0 mg/dL with ≥ 2 of the following: hepatomegaly, ascites or 5% weight gain) and either renal and/or pulmonary failure by D+28. Exclusion criteria: severe GvHD involving liver or gut; clinically significant bleeding; or need for >1 pressors to maintain BP. As this is a non-randomized study, the primary efficacy analysis compared CR by D+100, adjusted by quintiles of propensity score based on 4 stratification variables, at an overall two-sided 0.01 significance level (Koch et al,1989). CR was defined as bilirubin < 2 mg/dL + resolution of MOF; stratification variables were allogeneic/autologous SCT, adult/pediatric, 1 or 2+ SCTs, and ventilator/dialysis dependence. A secondary endpoint was mortality at D+100. DF was given at 6.25 mg/kg IV q6h; treatment duration was recommended for at least 21d. To create the HC, 35 centers sequentially reviewed up to 266 cases. To determine HC eligibility, the Medical Review Committee (MRC, composed of 2 independent expert hematologists) assessed all pts who met VOD criteria with MOF. The MRC were provided data for each pt (a redacted medical chart or pt narrative, depending on the privacy laws for each center) only up to the date on which the pt met inclusion criteria. The MRC remained blinded to outcome data at all times. One interim analysis was planned.
Results: For the HC, 6821 medical charts were screened, identifying 123 pts with features consistent with VOD in a setting of renal and/or pulmonary dysfunction that were reviewed for eligibility by the MRC. The MRC selected 32 cases as having an unequivocal diagnosis of VOD whose MOF was secondary to VOD, who met all protocol entry criteria; for all eligible pts, a confounding diagnosis of GvHD was ruled out. In the DF-treated group, 102 pts were enrolled. Following the interim analysis (comparing 61 DF pts to 32 HC pts), the DMC recommended an increase in HC sample size to 51 pts; given the large number of medical charts already reviewed, this was not considered feasible. In the final analysis (comparing 102 DF pts to 32 HC pts), the 2 groups were balanced regarding stratification variables. Baseline demographics (DF vs HC pts): median age 21 vs 18 yrs (43% and 44% pediatric); 63% vs 53% male, 88% vs 84% allogeneic SCT; 13% vs 3% with prior SCT; and 38% vs 38% ventilator/dialysis dependent. Median time post-SCT to VOD diagnosis was 13 and 11 days. Acute leukemia was the underlying diagnosis in 44% and 47%. Median duration of DF therapy was 22 days (range 1-60 days), with a median daily dose of 19 mg/kg/d. For the primary efficacy analysis, D+100 CR rate equaled 24% vs 9% (99%CI difference in CR rate: -1 – 35%; 95%CI difference in CR rate: 3 – 30%); p=0.015. D+100 mortality rate equaled 62% vs 75% (95%CI difference in rate -32 – 3%); p=0.051 by stratified log-rank. Consistent with prior studies, DF in children resulted in higher CR compared with HC (CR 36% vs 7%; p=0.04). Use of DF was associated with improved outcome in less sick pts (D+100 CR for pts without ventilator dependence equaled 40% vs 9%; p=0.051 and for pts without dialysis dependence equaled 34% vs 9 %; p=0.027). For pts receiving autologous SCT (n=12 and 5 pts in DF and HC arms), DF was associated with markedly improved CR (75% vs 0%, p=0.005). Hemorrhagic adverse events (any grade) were similar between the two groups (65% vs 69%); 18% of DF pts experienced a drug-related toxicity that led to discontinuation. D+100 CR strongly correlated with D+100 survival in both DF and HC groups (p<0.0001, p=0.0016).

Conclusions: DF improves CR by D+100 in pts with severe VOD/MOF post-SCT with a p value < 0.05. In addition, there was a trend towards improved D+100 survival in this critically ill population. DF-associated toxicities are consistent with prior studies, supporting the observation that DF is generally well tolerated.



Das dazugehörige Press Release:
Gentium Announces Presentation of Defibrotide Clinical Data at the American Society of Hematology Annual Meeting

VILLA GUARDIA (COMO), Italy, Nov 16, 2009 (BUSINESS WIRE) -- Gentium S.p.A. (Nasdaq: GENT) announced today that results from the Company's Phase II/III Pediatric Prevention trial and Phase III Treatment trial for Defibrotide of Hepatic Veno-Occlusive Disease will be presented at American Society of Hematology Conference (ASH), December 5-8 in New Orleans.

The results of these trials will be highlighted in two separate oral presentations on Monday, December 7, 2009 during the session, "Clinical Care - Transplantation Regimen Toxicities and Engraftment: Novel Approaches to Predict, Assess and Reduce Transplant Related Organ Toxicities" in the Ernest N. Morial Convention Center, 353-355. Details are as follows:

* "Defibrotide (DF) for the Prevention of Hepatic Veno-Occlusive Disease (VOD) in Pediatric Stem Cell Transplantation: Results of a Prospective Phase II/III Randomized, Multicenter Study" to be presented at 5:30 PM CT by Selim Corbacioglu, M.D., Pediatrics, University of Ulm, Germany

Abstract: http://ash.confex.com/ash/2009/webprogram/Paper22303.html

* "Defibrotide (DF) in the Treatment of Severe Hepatic Veno-Occlusive Disease (VOD) with Multi-Organ Failure (MOF) Following Stem Cell Transplantation (SCT): Results of a Phase 3 Study Utilizing a Historical Control" to be presented at 5:45 PM CT by Paul G. Richardson, M.D., Dana-Farber Cancer Institute, Boston, MA

Abstract: http://ash.confex.com/ash/2009/webprogram/Paper22224.html

Defibrotide will also be featured in the press conference entitled, "Transplantation: Preventing Complications and Improving Outcomes" on Saturday, December 5th from 2:00 PM - 3:00 PM CT in the Ernest N. Morial Convention Center, Room 350.

[EvertonFC]

Antwort auf Beitrag Nr.: 38.497.327 von Neoe am 02.12.09 19:37:43

Mich interessiert die Aktie nach wie vor und sie bleibt auf der Watchlist. Ich werde mich demnächst nochmal auf den neuesten Stand bringen. Financial Results habe ich bei Stockhouse gesehen. Danke für die Updates.

[greenhorn1998]

http://investor.shareholder.com/gentium/releasedetail.cfm?Re…

Würde mich über Meinungen sehr freuen, bin noch nicht so drin in der Thematik...

Gruß, greenhorn

[Neoe]

Nochmal zur Erinnerung.

Aktuell ist Gentium wieder relativ billig zu haben. Ich kann Sie nur jedem wärmstens empfehlen.

Gentium wird dieses Jahr einen Gewinn erzielen, ohne das Defibrotide überhaupt zugelassen ist. Ich erwarte hier Euro 10 Mio. Umsatz in 2010 allein durch die Treatment NDA (in den USA) und das Named Patient Program in Europa.

Ich muss sagen, ich habe so ein erfolgreiches Named Patient Program noch nie erlebt. Mal sehen was unter der Treatment NDA läuft, ist erst kürzlich angelaufen.

Sigma-Tau ist übrigens wichtiger Shareholder und entwickelt Defibrotide mit Gentium in den USA. Royalty Rate: 38%. In Europa wird Gentium voraussichtlich alleine vermarkten.

Die aktuelle Marketcap von rund USD 25 Mio. ist nur als Witz zu beschreiben. Letztes Jahr war Gentium äusserst riskant, aber letztendlich trotz leichter Fehlerquote erfolgreich. Sollte Defibrotide zugelassen werde, hat es eine unendliche Anzahl von Anwendungsgebieten.


Zu Sigma Tau:

Gentium Announces Extension of License and Supply and Cost Sharing Agreements with Sigma-Tau
New Agreement Further Strengthens Existing Relationship; Up to $15M in Payments to Gentium

VILLA GUARDIA (Como), Italy, Jan 11, 2010 (BUSINESS WIRE) -- Gentium S.p.A. (Nasdaq: GENT) today announced that it has amended its existing License and Supply and Cost Sharing Agreements with Sigma-Tau Pharmaceuticals, Inc. for the development and commercialization of Defibrotide in North America, Central America and South America. Gentium will retain exclusive rights to Defibrotide in Europe and the rest of the world.

The License and Supply Agreement has been amended to include a license to Sigma-Tau for the intravenous formulation of Defibrotide for the prevention of veno-occlusive disease (VOD) in the Americas and to transfer the New Drug Application (NDA) post approval in the United States (US). In addition, Gentium and Sigma-Tau have agreed to establish a joint steering committee to discuss in good faith, inter alia, the development, filing and relevant funding of Defibrotide for any therapeutic indication in the territory licensed to Sigma-Tau.

Under the amended terms of the Cost Sharing Agreement, the two companies will continue to share development costs for studies currently required for the filing of an NDA for Defibrotide. In addition, the parties have agreed to negotiate in good faith the funding of certain additional costs that may be required to obtain regulatory approval in the US, and that $1,000,000 of costs reimbursed by Sigma-Tau will be deductible from its future royalty payments due to Gentium under the License and Supply Agreement.

In return for the amended terms, Gentium will receive an initial payment of $7,000,000 from Sigma-Tau in connection with the execution of the amended agreements. An additional payment of $6,000,000 will be due to Gentium following approval from the FDA to market Defibrotide in the US and a further $2,000,000 will be payable following the transfer of the approved NDA to Sigma-Tau. Gentium will continue to receive a 7% royalty on net sales of Defibrotide and a supply margin equal to the greater of 31% of net sales of Defibrotide or EUR 50.00 per unit in the Americas.

"We have been collaborating with Sigma-Tau on the development of Defibrotide since 2001 and are delighted to expand this relationship as we move toward completing the studies required for filing an NDA," said Dr. Khalid Islam, Gentium's Chairman and Interim CEO. "We believe that Sigma-Tau is a valuable strategic partner whose aligned interest and marketing know-how in the Americas adds significant value. Moreover, this agreement with Sigma-Tau will strengthen our balance sheet without the issuance of additional shares."

"We believe Defibrotide has the potential to not only treat, but also prevent VOD," said Gregg Lapointe, Chief Executive Officer of Sigma-Tau Pharmaceuticals, Inc. "We are focused on developing therapies for rare, orphan diseases and remain committed to working closely with Gentium to make Defibrotide, a potentially lifesaving treatment, available to patients as quickly as possible."

[EvertonFC]

Nun hat Gentium die Indikation "Prevention of VOD" zusätzlich an Sigma auslizenziert. Der Deal spült Geld in die Kasse und wird zusätzliche Dilution zunächst verhindern. Ich bin mir nicht sicher, ob der Deal zu diesen Konditionen gut für Gentium ist, wenn man bedenkt, dass die Prävention der deutlich größere Markt sein soll.

Die Aktie ist weiter spannend. Ich glaube sie leidet vor allem unter Nichtbeachtung, dem noch langen Zeithorizont der Entwicklung, der Verunsicherung nach den Rücktritten des CEO und des Board Chairman und der Beurteilung der Nicht-Signifikanz in den letzten Studienergebnissen. Diese Beurteilung fällt mir auch schwer.

[Neoe]

Antwort auf Beitrag Nr.: 38.993.915 von EvertonFC am 23.02.10 15:09:08

Ich danke dir für diesen Beitrag Everton.

Er bildet wahrscheinlich tatsächlich sehr realistisch dei Wahrnehmung von Gentium im Markt ab.

Übrigens: Filing mit EMEA für Q2 angestrebt.

Zum Sigma Deal: Upfront ist niedrig, Royalties sind bzw. Manufacturing Fees sind perfekt für Gentium.

Dilution wird auf absehbare Zeit nicht notwendig sein und Gentium wird dieses Jahr profitabel.

[EvertonFC]

Das Unternehmen scheint noch den richtigen Weg oder die richtige Führung zu suchen. Ob die heutigen Entscheidungen auf Druck von Aktionären oder nach grossen operativen Problem zustande gekommen sind? Es sieht fast so aus, denn es gibt eine Reihe von Neubesetzungen und Umstrukturierungen. Die kurzfristig schwache Kursentwicklung hat vielleicht interne Probleme angedeutet, die nun behoben werden sollen. Auf in eine gute Zukunft Gentium!

Gentium Announces Management Changes and Corporate Restructuring

VILLA GUARDIA, Italy, March 1, 2010 /PRNewswire via COMTEX News Network/ --
Gentium S.p.A. (Nasdaq: GENT) today announced management and corporate restructuring changes resulting from a strategic decision to consolidate the Company's resources and operations. Following the expansion of the license agreement with Sigma-Tau, which will provide Gentium with up to $15 million in non-dilutive funding, Gentium will be closing the Company's New York office and consolidating corporate activities and the executive management team within its headquarters in Italy. Mr. Gary Gemignani, Executive Vice-President and Chief Financial Officer since 2006, will be leaving the Company effective March 31, 2010, but will provide transitional services through a consulting agreement. Dr. Khalid Islam, currently Gentium's Chairman and Interim CEO, will assume the role of Chief Executive Officer on a full-time basis. In addition, Mr. Salvatore Calabrese, Gentium's current Vice-President of Finance, has been promoted to Senior Vice-President of Finance.

Gentium's New York office currently houses operational, financial and strategic and business development personnel. The closure of this office and consolidation of corporate operations into the Como, Italy headquarters of Gentium are expected to result in one-time payments of approximately $1.5 million and are anticipated to yield annual cost savings of approximately $1.3 million.

"The Board and I would like to thank Gary for his dedication and service to Gentium," said Dr. Islam. "As Executive Vice President and CFO, he has helped lead the Company through some challenging periods, including transitions at the CEO and Board level, has initiated the named-patient and cost recovery programs, and has laid important groundwork to ensure that Defibrotide is positioned to be commercial-ready upon approval. We wish him well in his future endeavors."

Dr. Islam continued, "In the near future, Gentium's key efforts will be dedicated to the preparation of Defibrotide dossiers for filing with regulatory authorities and seeking marketing approval. Phase III clinical data has shown strong trends toward statistical significance and a meaningful impact on severe Veno-Occlusive Disease (VOD), a life threatening disease with no approved therapies. This is also reflected in the substantial interest already being generated by our named-patient program, which together with cost recovery and our expense reduction efforts has significantly reduced our overall cash burn."

[Neoe]

Antwort auf Beitrag Nr.: 39.033.332 von EvertonFC am 01.03.10 15:28:57

Hat sich bereits über Monate abgezeichnet und ist ein Ergebnis der Sigma-Tau Transaktion. Gewicht wird nach Europa verlagert. Gentium wird hier Defibrotide selbst vertreiben. Insgesamt überwiegt aktuell das Gewicht der europäsichen Aktionäre in den aktuellen Entscheidungen. Das ist das Ergebnis. Keine wirkliche News und wenn keine schlechten. Obwohl du richtigerweise sagst, dass die Dinge jetzt mal eine klare Struktur annehmen müssen. Ich galube, dass ist jetzt passiert.

[Neoe]

Gestern aus dem Nichts um 30 Prozent gestiegen beim 40-fachen des üblichen Handelsvolumens.
Nächste Woche werden Daten von Defibrotide auf einer Konferenz vorgestellt, außerdem kommen zahlen und sie werden meines Erachtens die Botschaft entalten, dass Gentium der erste Biotech wird, der aufgrund eines Named Patient Programs profitabel wird.

Sobald die Konjunktur anzieht und risikoreichere Werte wie Gentium wieder gesucht werden, wird es kein Halten mehr geben.

[HuntingParty]

Antwort auf Beitrag Nr.: 39.182.486 von Neoe am 20.03.10 08:09:01

Na also - geht doch; hab mal einen Teil zu 2,90 verkauft


http://www.streetinsider.com/Special+Reports/Morning+Movers+…

[Neoe]

Nochmal die komplette News:

VILLA GUARDIA, Italy, March 23 /PRNewswire-FirstCall/ -- Gentium S.p.A. (Nasdaq:GENT - News) today announced that the abstract titled, "Defibrotide prevents hepatic VOD and reduces significantly VOD-associated complications in children at high risk: final results of a prospective phase II/III multicenter study," was presented on Sunday, March 21, 2010, at the opening session of the European Blood and Marrow Transplantation (EBMT) 36th Annual Meeting in Vienna. The abstract received the esteemed Van Bekkum Award for the best abstract submitted to the physician's program. Dr. Selim Corbacioglu, Professor of Pediatrics, University of Regensburg (Germany) and Principal Investigator of the Pediatric Prevention trial, accepted the award and presented the final results of the study.

This Phase II/III, randomized, controlled study evaluated the prophylactic use of Defibrotide in pediatric stem cell transplant (SCT) patients at high risk for veno-occulsive disease (VOD). In the intent to treat analysis (ITT), which included 356 patients (180 patients in the prophylaxis arm and 176 in the control arm), Defibrotide demonstrated a 40% reduction in the incidence of VOD within 30 days after SCT, the primary endpoint of the study. VOD incidence was 20% in the control arm and 12% in the prophylaxis arm (p=0.0488 Competing Risk; p=0.0507 Kaplan-Meier). In the per-protocol analysis, the VOD incidence was 20% in the control arm and 11% in the prophylaxis arm (p=0.0225 Competing Risk; p=0.0234 Kaplan-Meier). A p-value equal to or less than 0.05 is needed to achieve statistical significance.

In addition, a pre-specified analysis showed that the incidence and severity of acute graft versus host disease (GvHD) by day 100 in allogeneic SCT recipients was significantly reduced from 63% for the control arm to 45% for the prophylaxis arm (p=0.0046 for incidence of GvHD and p=0.0034 for severity). Renal failure was reduced from 6% in the control group to 1% in the Defibrotide group (p=0.0169). With regard to safety, Defibrotide was well tolerated and no difference in adverse events was observed between the two study arms.

"The results from the study demonstrated that Defibrotide reduced the incidence of VOD in high risk pediatric patients," stated Dr. Selim Corbacioglu. "SCT patients who develop VOD, regardless of its severity and despite prompt treatment, have a mortality rate four times greater than patients without VOD. We are also very enthusiastic that Defibrotide has the potential to reduce the incidence and severity of acute GvHD, a life threatening complication, which is frequent in SCT and has limited treatment options. We believe that Defibrotide could have a future role for the prevention of GvHD."

"The Defibrotide VOD prevention study was the largest study performed in the pediatric SCT setting and its results have been included in the press conference of the EBMT," said Professor Dietger Niederwieser of University of Leipzig, Chairman of the opening session, President of the EBMT, and newly elected president of the Worldwide Network for Blood & Marrow Transplantation (WBMT). "The results of this study, which demonstrate a reduction in VOD as well as the potential beneficial effects on GvHD and renal failure, are consistent with the effects of endothelial protection and confirm the potential of Defibrotide in preventing transplant related toxicities."

"We are very encouraged by the recognition of this study by the leaders of the European hematology community," said Dr. Massimo Iacobelli, Scientific Director of Gentium. "We are fully engaged in preparing the regulatory submissions for the prevention and treatment of VOD."

Defibrotide's biological activity and mechanism of action were highlighted in a separate poster-presentation, "Defibrotide protects human microvascular endothelial cells from fludarabine-mediated pro-inflammatory and pro-apoptotic signals: a microarray (Affimetrix) analysis." This study provides new biological insights on how Defibrotide might protect endothelial cells against chemotherapy-induced activation and cell death, without affecting the antileukemic effect of fludarabine and without affecting normal endothelium.

The satellite symposium "Defibrotide for the Prevention and Treatment of Hepatic VOD following stem cell transplant," was very well attended, featuring presentations by key opinion leaders Dr. Ernst Holler (University of Regensburg); Dr. Selim Corbacioglu (University of Regensburg); Dr. Paul Richardson (Dana Farber Cancer Institute, Boston, US), and Dr. Enric Carreras (University of Barcelona). The symposium was chaired by Dr. Rob Soiffer (Dana Farber Cancer Institute, Boston, US).

About the EBMT and the Van Bekkum Award

The European Group for Blood and Marrow Transplantation (EBMT) is a European non-profit organization that was established in 1974 to allow scientists and physicians involved in clinical bone marrow transplantation to share their experience and develop cooperative studies. Representing 527 transplant centers across 57 countries in and outside of Europe, the EBMT promotes all activity aiming to improve stem cell transplantation or cellular therapy.

The Van Bekkum award is named for Dr. Dirk Van Bekkum, who is internationally recognized for his work in bone marrow transplantation; among other achievements, his laboratory was the first to identify what is now known to be graft-versus-host disease.

About the Phase II/III European Pediatric Prevention Trial

The EBMT phase II/III European pediatric prevention trial was a prospective, multi-center, open label, randomized clinical trial to evaluate the prophylactic use of Defibrotide in patients under 18 years of age who were undergoing stem cell transplantation (SCT) and were at high risk for developing hepatic Veno-Occlusive Disease (VOD). Patients randomized in the prophylaxis arm received 25 mg/kg/day of Defibrotide in four divided doses beginning at the time of conditioning and until 30 days post transplant. Patients randomized to the control arm received no VOD prophylactic therapy. Patients were permitted to receive Defibrotide as therapy if they developed VOD. The primary endpoint of the study was development of VOD within 30 days post SCT, based on the modified Seattle criteria. A blinded independent review committee of three expert hematologists confirmed the diagnosis of VOD.

About VOD

Veno-occlusive disease is a potentially life-threatening condition, which typically occurs as an important complication of stem cell transplantation. Certain high-dose conditioning regimens used as part of SCT can damage the lining cells of hepatic blood vessels and so result in VOD, a blockage of the small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so-called severe VOD). SCT is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children. There is currently no approved agent for the treatment or prevention of VOD in the US or the EU.

About GvHD

Graft-versus-host disease (GVHD) is a complication that occurs frequently after allogeneic stem cell transplantation, where with to the blood forming cells a completely new immune system is transplanted from the donor. The differences between the donor and recipient often cause T cells (a subtype of white blood cells) from the donor to recognize the recipient's body tissues as foreign. When this happens, the newly transplanted immune system attacks the transplant recipient's body. Risk of GvHD increases with the degree of mismatch between donor and recipient.

About Gentium

Gentium S.p.A., located in Como, Italy, is a biopharmaceutical company focused on the development and manufacture of drugs to treat and prevent a variety of diseases and conditions, including vascular diseases related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate, is an investigational drug that has been granted Orphan Drug status by the U.S. FDA and Orphan Medicinal Product Designation by the European Commission both to treat and to prevent VOD and Fast Track Designation by the U.S. FDA to treat VOD.

[EvertonFC]

Starke Performance der Aktie aber sie ist hierzulande nach wie vor unbekannt, obwohl diese stattliche Studie in Deutschland abgehalten wurde.

Neoe, du kennst dich bei der Auswertung besser aus, aber 40% Reduktion der Venenverschlüsse im Vergleich zur Kontrollgruppe nenne ich eine signifikante Wirkung.

Als Zusatznutzen kann sich die Reduktion der Graft versus Host Disease (GvHD) von 63% auf 45% sehen lassen.

[erdo1]

hab mir das mal auf die schnelle durchgezogen, war das jetzt der ablschluss der phase 3 studie?
Auf der Nasdaq Homepage ist ein Kursziel von 5$ angegeben, ist da schon eine positive Phase 3 Studie mit einkalkuliert?

Danke, und LG

[Neoe]

@ everton:
@ erdo1:
Leider wurde für VOD die statisitsche Signifikanz in der Intent to Treat Population knapp verpasst. Ich denke aber, dass aufgrund der klinischen Relevanz in VOD, der Reduktion dvon GvHD (absoluter Knüller, wen man bedenkt wie schwer GvHD zu behandeln ist und ein Riesengeschenk für alle Patienten), aufgrund der Reduktion der Sterblichkeit und aufgrund der ebenfalls positiven US-Studienergebnisse prognostiziere ich, dass Gentium am Ende des Tages die Zulassung erhalten wird.
Bis dahin werden wir bereits wieder bei 20 Dollar stehen.

Ich denke, der beste Beweis für die Qualität von Defibrotide, ist der permanente Einsatz von absoluten Kapazitäten auf ihrem Gebiet:
Professor Dietger Niederwieser, Dr. Selim Corbacioglu und ihre Gegenüber auf amerikanischer Seite. Mir klingt die Aussage noch in den Ohren, das Defibrotide zur Behandlung von VOD nur der Anfang ist, diese Leute wissen bereits, dass Defibrotide für einiges mehr eingestzt werden kann. Man muss bedenken, dass Gentium Defibrotide gar nicht entwicklen wollte, sondern von wichtigen Ärzten auf dem Gebiet der Onkologie dazu bewegt wurde, diese Studien anzupacken. Das sollte jeder bei der Bewertung von Gentium und ihres Produktes im Hinterkopf behalten.

[Neoe]

All time record trading volume und über USD 3,20 geschlossen. Bei mir herrscht Bombenstimmung.

[erdo1]

@neoe
super das du erfolg damit hast.

Wie ist deine einschhätzung bzgl. des kurses, geht momentan noch was nach oben, oder soll man jetzt mit einem einstieg warten.

[MrRipley]

Antwort auf Beitrag Nr.: 39.208.118 von EvertonFC am 24.03.10 18:06:41

Danke Everton und Neone, ich habe die Aktie seit einer Woche auf der WL, weiss aber nicht mehr warum. Wahrscheinlich wegen der Tatsache, wo Neone draufsteht ist auch etwas drin

[Sebi-K]

lohnt sich hier heute noch ein einstieg?

[Neoe]

Ein Einstieg lohnt sich dann, wenn man an die Story glaubt. Aufgrund kurzfristiger Trading-Profits würde ich hier nicht einsteigen.

Ich persönlich halte Gentium für eine der kommenden Biotech-Stories.
Das Produkt ist zu gut.

[EvertonFC]

Diese Aktie schlägt immer wieder zurück. Wer in die Korrektur verkauft hat wird bestraft.

Quartalszahlen und Ausblick für das Jahr 2010:

http://finance.yahoo.com/news/Gentium-Announces-Fourth-prnew…

Im letzten Quartal aufgrund des Named Patient Programm bereits Break-Even. Cash Flow Postive in 2010.

Aktuell 75% im Plus.

[MrRipley]

Antwort auf Beitrag Nr.: 39.264.189 von EvertonFC am 01.04.10 16:34:35

Glückwunsch alter Schwede, Du hast es ja echt drauf!

[EvertonFC]

Antwort auf Beitrag Nr.: 39.264.849 von MrRipley am 01.04.10 17:39:14

Leider partizipiere ich daran nicht, da das Kapital knapp wird. Du weisst ja, ich bin gross bei ZNN.V und EXAS engagiert.

Was nicht ist kann noch werden. Kauf 3000 St. GENT zu 3.98 USD.

[Neoe]

Ist eigentlich jemand mit dabei?

[EvertonFC]

Die Aktie strebt nach der News auch ohne neues Coverage weiter nach oben.

Ich frage mich noch wie man innerhalb überschaubarer Zeit die Timeline für den FDA-Antrag von 2013 auf 2011 vorverlegen konnte?

[Neoe]

Antwort auf Beitrag Nr.: 39.308.232 von EvertonFC am 10.04.10 15:03:18

Die Timeline war meines Wissens nie 2013, sondern eigentlich Q2 2010.

Einen neue Phase III wird es nicht geben. In der durchgeführten Größe (380 Patienten) wäre das nahezu unmöglich. Ich denke, die Behörden werden bei der Zulassung die notwendige Toleranz zeigen. Einige Endpoints wurden statistisch knapp verfehlt, andere getroffen. In jedem Fall waren sie immer klinisch relevant.

Was jetzt gemacht wurde, ist eine GAP-Analyse. Ausstehende Daten, die für ein Filing benötigt werden, wurden identifiziert. D.h. notwendige klinische Daten in kleinerem Umfang, die den Behörden - FDA und EMEA - noch fehlen, werden bis Anfang nächsten Jahres generiert und dann in Q2 2011 eingereicht. M.E. ein sehr sinnvolles Vorgehen.

Zusätzlich wird man dann Daten von mehr als 1000 Personen aus dem Named Patient Program einreichen können.

Mit etwas Fantasie kann man sich ausmalen, was alles möglich ist in den nächsten 2 Jahren.

[HuntingParty]

Antwort auf Beitrag Nr.: 39.306.464 von Neoe am 09.04.10 22:08:50

Ich bin nachwie vor mit reduziertem Bestand dabei. Vielen Dank für die sorgfältige und gute Recherche.

[MaatMod]

Für eine aktuelle Diskussion enthistorisiert.

MfG MaatMOD

[EvertonFC]

Gentium sollte nich in Vergessenheit geraten....

Die Umsätze an der Nasdaq sind zwar recht dünn aber die Aktie entwickelt sich stetig nach oben. Sobald kleiner Verkaufsdruck aufkommt sammeln einige Hände stetig ein.

Die Tagesvola ist mit der geringen Liquidität noch etwas hoch. Ohne grosse Beachtung ist die Aktie mittlerweile über 7 USD gestiegen.

Bis auf eine Konferenzteilnahme hat es seit März keine News mehr gegeben.

[Aurum2010]

5.94 -2.85 (-32.42%)
http://www.google.com/finance?q=NASDAQ:GENT

Gentium S.p.A. Withdraws New Drug Application for Defibrotide

Gentium S.p.A. announced that it has voluntarily withdrawn the New Drug Application (NDA) for Defibrotide following recent correspondence from the United States Food and Drug Administration (FDA) identifying numerous 'Refuse to File' issues regarding the Company's NDA submission. In their initial review, the FDA raised concerns regarding the completeness of the datasets for both the treatment and prevention studies. The FDA requested that the Company conduct additional quality reviews of the original datasets and databases. The FDA also requested additional details regarding the conduct and monitoring of the trials by the independent review committee.

http://www.reuters.com/finance/stocks/GENT.O/key-development…

Chance für nen Einstieg?