Mallinckrodt's Phase 3 trial of terlipresin in HRS-1 meets primary endpoint
Mallinckrodt announced positive top-line results from its pivotal Phase 3 CONFIRM clinical study evaluating the efficacy and safety of terlipressin in 300 adults with hepatorenal syndrome type 1. The study met its primary endpoint of verified HRS-1 reversal. 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% 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.