Mallinckrodt Confirms Positive Late-Stage Readout For Hepatorenal Syndrome Drug

Shares of Mallinckrodt PLC MNK traded higher following a clinical readout from the company.

What Happened

Mallinckrodt announced positive top-line results from the pivotal Phase 3 study dubbed CONFIRM that evaluated the efficacy and safety of terlipressin in 300 adults with hepatorenal syndrome type 1, or HRS-1.

HRS-1 is a life-threatening rare and acute disease characterized by complications of liver disease that lead to kidney failure. Terlipressin is a potent vasopressin analogue selective for VI receptors.

The study met the primary endpoint of verified HRS-1 reversal, including three components: renal function improvement, avoidance of dialysis and short-term survival.

"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," said Arun Sanyal, the lead investigator of the study.

Why It's Important

HRS-1 has no approved drug in the U.S. or Canada. It's estimated to affect about 30,000 to 40,000 patients in the U.S. annually.

The median survival time of a patient diagnosed with the disease is less than two weeks, with 80% percent mortality occurring within three months.

Mallinckrodt said it plans to submit an NDA to the FDA in early 2020. It's also looking forward to present the data from the late-stage study at an upcoming medical meeting.

The stock traded higher by 2.37% to $4.75 at time of publication.

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