Mallinckrodt's terlipressin meets main goal in Phase III study of hepatorenal syndrome type 1

Shares in Mallinckrodt jumped 11% Thursday after the company announced that the Phase III CONFIRM trial evaluating terlipressin in adults with hepatorenal syndrome type 1 (HRS-1) met its primary endpoint, as well as nearly all the pre-specified secondary objectives. The company plans to submit a marketing application to the FDA for the treatment, a vasopressin analogue selective for V1 receptors, early next year.

The trial included 300 adult HRS-1 patients with liver cirrhosis and ascites with rapidly worsening renal function and no response to diuretics or albumin. Participants were randomised to receive terlipressin administered intravenously or placebo. The main goal was verified HRS reversal within 15 days, defined as the percentage of participants with two consecutive qualified serum creatinine values of 1.5 mg/dL or less, at least two hours apart. Secondary endpoints included the incidence of patients with HRS reversal within a 14-day timeframe, as well as the percentage of those maintaining HRS reversal without renal replacement therapy to day 30.

"We are encouraged by the positive top-line results of the CONFIRM trial, the largest-ever prospective trial in HRS-1," remarked Steven Romano, chief scientific officer at Mallinckrodt, adding the findings "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."

Mallinckrodt noted that there are currently no approved drug therapies for HRS-1 in Canada or in the US, where the condition affects an estimated 30,000 to 40,000 patients annually. The company added that it reached an agreement with the FDA in 2016 on the CONFIRM protocol design and data analysis under the agency's special protocol assessment process.

In 2015, Mallinckrodt gained terlipressin via the $2.3-billion purchase of Ikaria. The drug is currently approved as the standard treatment for HRS-1 in a number of countries, including several in Europe.

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