Mallinckrodt has reported positive data from the Phase III CONFIRM trial of terlipressin conducted in hepatorenal syndrome type 1 (HRS-1) patients.
Top-line results showed that the trial met the primary endpoint of verified HRS-1 reversal, which comprises an improvement in renal function, dialysis avoidance, and short-term survival.
HRS-1 is a rare and acute condition that causes complications of liver disease resulting in kidney failure. The condition is associated with a median survival period of fewer than two weeks.
Terlipressin is a potent analogue of vasopressin. The investigational drug is designed to selectively target V1 receptors.
Performed in the US and Canada, the multi-centre, randomised, placebo-controlled, double-blind CONFIRM study has been designed to assess the safety and efficacy of the drug candidate.
Mallinckrodt noted that the company signed an agreement with the US Food and Drug Administration (FDA) in 2016 regarding the clinical design, endpoints, and statistical data analyses of the Phase III trial.
The trial enrolled a total of 300 adult HRS-1 patients having liver cirrhosis and ascites. Participants had rapidly deteriorating renal function and did not experience a response to diuretics or albumin.
During the study, the drug candidate was compared to placebo. The primary outcome was verified HRS reversal, while secondary measures included durability and incidence of HRS reversal.
Mallinckrodt executive vice-president and chief scientific officer Steven Romano said: “We are pleased and encouraged by the positive top-line results of the CONFIRM trial, the largest-ever prospective trial in HRS-1.
“The CONFIRM trial will support regulatory submissions of terlipressin as a treatment for HRS-1 in the US and Canada, and we look forward to sharing full results soon.”
The company intends to file a new drug application (NDA) with the FDA early next year to seek approval for terlipressin to treat patients suffering from HRS-1.