Gentium Reports Top Line Phase III Defibrotide Trial Results

Gentium has reported top-line results from a historically controlled, multicenter, open label, phase III trial, designed to evaluate the safety and efficacy of 25mg/kg/day of Defibrotide. The study is aimed at 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.

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.

However, 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.

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.

Gary Gemignani, executive vice-president and CFO, said: “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. 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.”