Advertisement
"We are pleased to have received FDA approval to start the SEPET pivotaltrial and to have fully satisfied the points addressed in the FDA's previouslyissued conditional approval," commented CEO and President Shawn Cain. "We werepleased with the compelling results from the SEPET feasibility trial, and weare excited to initiate the pivotal clinical phase of SEPET's development. Thepivotal trial design includes adaptive measures to optimize our ability toachieve the trial's primary and secondary endpoints. We believe that thepivotal trial, if successful, should support our filing for approval of SEPETin the United States and marketing efforts in the United States and theEuropean Union. Further, we believe that the design of this trial willenhance physician acceptance of SEPET as a much needed tool in sustainingpatients through acute life threatening episodes of liver failure, a marketwhich we believe exceeds a billion dollars annually."
Advertisement
Trial Design
There are three segments to the pivotal trial design. During the firstsegment of the trial, 5 non-randomized patients will be treated with SEPET toallow us to validate the patient selection criteria, clinical protocol, casereport forms, and other trial related documents. During the second segment ofthe trial, we expect to enroll 116 patients in this randomized, controlledphase of the trial. This segment is targeted to achieve the co-primaryendpoints, which are 1) the percentage of patients achieving improvement inhepatic encephalopathy ("HE") grade by a minimum of two grades by the end ofDay 7 in the SEPET treatment group versus the standard medical care group,using a 1:1 randomization between the two groups; and 2) the 30-day transplantfree survival rate in all patients (i.e. control and treatment groups) who doreach a two grade HE improvement versus all patients who do not reach a twograde HE improvement. Pending review and approval by the Data SafetyMonitoring Board, the third segment would permit the size of the trial to beincreased by an additional 52 patients, if the co-primary efficacy endpointsare reached or have not reached statistical significance but have shown apositive trend. If the co-primary endpoints of the trial are reached uponcompletion of segment two, extension of the trial into segment three mayresult in the achievement of statistical significance of one or more secondaryendpoints of the trial relating to clinical, functional, and reimbursementadvantages for SEPET treatment over standard medical care.
Patient Inclusion/Exclusion Criteria for the Trial
To be a candidate for the pivotal trial, a patient must have chronic liverdisease and be experiencing an acute episode that results in hospitalizationwith an HE grade of between II and IV. In addition, the patient must not beresponding satisfactorily to standard medical care (e.g. fluid replacement,antibiotics, lactulose) for 20 to 26 hours prior to randomization. Patientscontraindicated for a liver transplant (e.g. advanced liver cancer patientsand drinking alcoholics) are excluded from the trial.
"We hope to shortly receive permission from the German regulatoryauthority to begin segment one of the pivotal trial at one or two clinicalsites in Germany," commented Mr. Cain. "Over the next several months we willalso seek IRB approvals for up to 24 clinical sites in the United States andEurope. While we currently have very limited financial resources, we hope thatthe FDA's approval to initiate th
