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Interim Results From Gentium's Treatment IND for Defibrotide and Additional Data on Defibrotide in the Prevention of Acute Graft Versus Host Disease (aGvHD) Presented at the American Society of Hematology Annual Meeting

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VILLA GUARDIA (COMO), Italy, Dec. 11, 2013 (GLOBE NEWSWIRE) -- Gentium S.p.A. (Nasdaq:GENT) (the "Company") today announced that additional data on Defibrotide were presented this week at the 55th Annual Meeting and Exposition of the American Society of Hematology at the Ernst N. Morial Convention Center in New Orleans, LA, December 7-10, 2013.

Professor Paul G. Richardson, RJ Corman Professor of Medicine, Clinical Director Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute Harvard Medical School, Boston, MA (USA) reported, in an oral presentation, the updated results from the ongoing Treatment IND Expanded Access Protocol, a large prospective study on the use of Defibrotide in the treatment of Hepatic Veno-Occlusive Disease ("VOD") in hematopoietic stem cell transplant patients, (abstract # 700). This updated interim analysis was based on 470 patients with VOD, enrolled between December 2007 and December 2012 at 75 centers across the United States. 425 patients had undergone hematopoietic stem cell transplant ("HSCT") and of those patients, 35% of patients achieved a complete response (CR) and 55% survived to day 100 (D+100). Additional findings were as follows:

  • In the subgroup of 284 HSCT patients with severe VOD, 29% achieved a CR and 48% survived to day 100.
  • In the sub-group of 141 HSCT patients with non-severe VOD, 47% achieved a CR and 69 % survived to D+100.
  • In the subgroup of 45 patients who had VOD following chemotherapy but no HSCT, 40% achieved a CR and 62% survived to D+100.
  • Delayed initiation of Defibrotide treatment (more than 2 days after VOD diagnosis) resulted in reduced CR (25% versus 39%, p=0.0052) and survival (38% versus 61%, p=0.0001) compared to early administration (within 2 days from VOD diagnosis).

To access Professor Richardson's abstract click on this link: https://ash.confex.com/ash/2013/webprogram/Paper59460.html.

In a poster (#4591), Professor Selim Corbacioglu, Chair Dept. of Paediatric Haematology and Oncology, Children's Hospital University of Regensburg, Germany, presented additional information on the impact of prophylaxis with Defibrotide on the occurrence of acute Graft versus Host Disease (aGvHD) in allogeneic HSCT. A secondary objective of a prospective, randomized, open-label controlled study of in 356 children at high-risk for hepatic VOD post-HSCT [Corbacioglu, Lancet 2012] was to analyze the impact of Defibrotide prophylaxis on the incidence and severity of aGvHD. At day+100 post-HSCT the incidence of aGvHD was 47% in the DF arm vs. 65% in the control group (p=0.0046). Defibrotide showed a consistent reduction of the more severe grade II–IV aGvHD from 37% to 22% (p=0.0130). In addition the use of corticosteroids (which are used to treat aGvHD) was significantly lower in patients receiving Defibrotide prophylaxis (37% vs. 48% in control arm, p=0.0363). Professor Corbacioglu points out that the reduction in aGvHD observed with Defibrotide "is additional to standard GvHD prophylaxis that was fully implemented in these patients."

To access Professor Corbacioglu's presentation click on this link: https://ash.confex.com/ash/2013/webprogram/Paper57466.html.

About Gentium

Gentium S.p.A. (Nasdaq:GENT), 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, has been granted Orphan Drug status by the U.S. FDA, by the EMA, by the Korean Federal & Drug Administration (KFDA), both to treat and to prevent Veno-Occlusive Disease ("VOD"), by the Commonwealth of Australia-Department of Health for the treatment of VOD and Fast Track Designation by the U.S. FDA to treat VOD. In October 2013, the European Commission ("EU") granted Marketing Authorization for Defitelio® (defibrotide) for the treatment of severe VOD in adults and children undergoing hematopoietic stem cell transplantation therapy. In November 2013, the EU has granted Orphan Drug Designation for Defibrotide for the prevention of Graft versus Host Disease (GvHD).

For additional info and to sign up for email alerts, please visit www.gentium.com.

About Defibrotide

Defibrotide is an investigational drug for the prevention and treatment of hepatic veno-occlusive disease (VOD), a serious and potentially fatal complication of hematopoietic stem-cell transplantation (HSCT). The efficacy of Defibrotide to treat hepatic VOD in HSCT patients is supported by data from a multi-center Phase 3 historically controlled trial, evaluating Defibrotide for the treatment of severe VOD (patients with VOD and multi-organ failure), a Phase 2 dose finding study, and interim data reported from the ongoing Phase 3 expanded access U.S. Treatment IND program in patients with severe hepatic VOD. Additional data include a Phase 3 randomized controlled study of Defibrotide in the prevention of hepatic VOD in pediatric HSCT patients. Defibrotide has generally been well-tolerated in the clinical setting, and results in more than 1,300 patients to date have shown that generally Defibrotide does not appear to increase the risk of complications in HSCT patients. In the European Union, Defibrotide, marketed as Defitelio®, has obtained a Marketing Authorization for the treatment of severe VOD.

About VOD

Veno-occlusive disease (VOD) is a potentially life-threatening condition, which typically occurs as a significant complication of stem cell transplantation. Certain high-dose conditioning regimens used as part of stem cell transplantation can damage the lining cells of hepatic blood vessels and result in VOD, a blockage of the small veins in 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). Stem cell transplantation is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children.

About GvHD

Graft versus Host Disease (GvHD) is one of the most important and potentially fatal complication of hematopoietic stem cell transplantation (HCT). GvHD is seen most often in cases where the blood marrow donor is unrelated to the patient or when the donor is related to the patient but not a perfect match. It can occur in 30-50% of patients transplanted with either an HLA-matched sibling donor or matched unrelated donor.

Cautionary Note Regarding Forward-Looking Statements

This release contains "forward-looking statements" that involve a number of risks and uncertainties the outcome of which could materially and/or adversely affect actual future results and the market price of Gentium's securities. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "potential" or "continue," the negative of these terms and other comparable terminology. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. It is possible that actual results, including with respect to the possibility of any future regulatory approval, may differ materially from those anticipated in these forward-looking statements. For a discussion of some of the risks and important factors that could affect future results, see the discussion in our Form 20-F filed with the Securities and Exchange Commission under the caption "Risk Factors."

CONTACT: Gentium S.p.A. Salvatore Calabrese, +39 031-5373-260 SVP Finance, COO/CFO scalabrese@gentium.it or The Trout Group Chelsea Wheeler +1 646 378 2941 cwheeler@troutgroup.com

Source:Gentium S.p.A.

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