Mallinckrodt announces positive top-line results from its pivotal phase III CONFIRM trial of terlipressin in patients with hepatorenal syndrome Type 1

Mallinckrodt Plc announced positive top-line results from its pivotal Phase III CONFIRM clinical study evaluating the efficacy and safety of terlipressin in 300 adults with hepatorenal syndrome type 1 (HRS-1). The study met its primary endpoint of verified HRS-1 reversal (p=0.012). Verified HRS-1 reversal includes three components: renal function improvement, avoidance of dialysis and short-term survival. The company plans to present the data at an upcoming medical meeting.

HRS-1 is a life-threatening, rare and acute disease characterized by complications of liver disease that lead to kidney failure. HRS-1 has a very poor prognosis, with a median survival time of less than two weeks and greater than 80 percent mortality within three months. At present, there are no approved drug therapies for HRS-1 in the U.S. or Canada.

HRS-1 is estimated to affect between 30,000 and 40,000 patients in the U.S annually. The company plans to submit a New Drug Application to the FDA in early 2020. Terlipressin is an investigational product and its safety and effectiveness have not yet been established by the FDA or Health Canada.

"The initial results from the Phase III CONFIRM study are very encouraging in that they demonstrate terlipressin reversed the course of HRS-1 as measured by improvement in renal function, avoidance of dialysis and short-term survival. The study met nearly all of the pre-specified secondary endpoints," said lead investigator Arun Sanyal, M.D., Z. Reno Vlahcevic Professor of Medicine, Physiology and Molecular Pathology at Virginia Commonwealth University. "HRS-1 is a life-threatening disease that is extremely difficult to treat. We anticipate the complete results will continue to help inform the effectiveness and safety profile of terlipressin in this patient population with urgent unmet medical needs."