Gentium Announces Successful Completion of Defibrotide Phase 1 Study in Japan

VILLA GUARDIA (COMO), Italy, Nov. 12, 2013 (GLOBE NEWSWIRE) -- Gentium S.p.A. GENT (the "Company") today announced that the medical team from the Phase 1 Trial Unit of the Translational Research Center at the National University Corporation, Hamamatsu University School of Medicine ("HUSM"), Fukushima Medical University ("FMU") has successfully completed the phase 1 clinical study to evaluate the Safety and Pharmacokinetics of Defibrotide (DF) in Healthy Adults.

This Investigator led clinical trial was adopted as a Health and Labour Sciences Research Grant in 2012 for Practical Use of Medical Technology (categorized as a study project to support practical drug/medical device use during the reconstruction of disaster-hit areas in eastern Japan).

The primary objective of the study was to evaluate the safety and pharmacokinetics of defibrotide in healthy Japanese adult subjects. The secondary objective was to evaluate the effect of defibrotide on the coagulation and fibrinolytic systems in vivo. Two dose levels (3 mg/kg and 6.25 mg/kg, termed Cohort 1 and Cohort 2, respectively) were evaluated, each cohort consisted of 10 subjects (8 subjects were given DF, 2 were given placebo).

All 20 subjects completed the protocol defined treatment and there were no premature discontinuations. No serious adverse events were noted and only one mild, transient and reversible adverse event observed. Moreover, no clinically significant changes were observed in laboratory test results in any of the dosing groups.

The Study Director, Professor Atsushi Kikuta, Division of Pediatric Oncology, Clinical Oncology Center, Fukushima Medical University commented, "The results indicate not only the appropriateness but also the necessity of clinical investigations targeting VOD patients for the purposes of further assessment of efficacy and confirmation of safety of DF based on the pharmacokinetic and pharmacodynamic profiles of defibrotide obtained from the present study."

Gentium provided free drug and technical support to HSUM.

About Gentium

Gentium S.p.A. 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 and Orphan Medicinal Product Designation by the EMA both to treat and to prevent veno-occlusive disease ("VOD") and Fast Track Designation by the U.S. FDA to treat VOD. The European Medicines Agency's Committee for Orphan Medicinal Products ("COMP") has issued a positive opinion on the Company's application for orphan medicinal product status for Defibrotide for the prevention of Graft versus Host Disease ("GvHD"). The positive opinion of the COMP has now been forwarded to the European Union ("EU") commission for final approval and publication in the EU community register. In October 2013, the European Commission granted marketing authorization for Defitelio® (defibrotide) for the treatment of severe hepatic VOD in adults and children undergoing hematopoietic stem cell transplantation therapy.

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About Fukushima Medical University

Fukushima Medical University promotes, through the highly advanced level of medical and nursing research of the faculty members, the welfare of the population of the prefecture and educate doctors and nurses to work for medical and welfare organizations in the prefecture after graduation. The University, its affiliated institute and hospital endeavour not only to carry out pioneering research that has gained worldwide recognition, but also to practice patient-oriented medicine with the highest ethical standards. Its ultimate goal is to fulfill its function of research and education for the local as well as international community.

About the Health and Labour Sciences Research Grant

The aim of Health and Labour Sciences Research Grant is to promote the Health and Labour Sciences Research. By the promotion of Health and Labour Sciences Research, the Ministry of Health, Labour and Welfare secures the scientific promotion of the administrative purposes about the national healthcare, the welfare, Environmental Health, occupational safety and health and plans the improvement of the level of technology. The Ministry of Health, Labour and Welfare forms competitive study environment regarding some original or pioneer researches with strong social requests and advertises for research projects every year.