First Patient Enrolled in Mallinckrodt Phase 3 Terlipressin Trial

CHESTERFIELD, United Kingdom, July 28, 2016 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today confirmed enrollment of the first patient in the company's Phase 3 clinical study to evaluate the efficacy and safety of terlipressin (for injection) in subjects with Hepatorenal Syndrome (HRS) type 1.

"Patients diagnosed with HRS type 1 typically have a very poor prognosis, and there is a significant unmet need for an approved treatment," said lead investigator Thomas D. Boyer, M.D., Director, Liver Research Institute, University of Arizona College of Medicine – Tucson. "I am excited to work with my colleagues in the hepatology community and with Mallinckrodt on this study of a potentially important therapy for U.S. patients."

Terlipressin is being investigated for the treatment of HRS type1, an acute, rare and life-threatening condition requiring hospitalization, with no currently approved therapy in the U.S. or Canada. Terlipressin is approved for use in countries outside the U.S., including several in Europe^1,2,3,4.

"Mallinckrodt is committed to advancing therapies for patients with rare diseases and critical conditions who have limited treatment options," said Steven Romano, M.D., Chief Scientific Officer and Senior Vice President at Mallinckrodt. "Terlipressin is approved in many other countries for patients afflicted with this devastating condition. We are pleased to enroll the first patient in the CONFIRM trial, which will support the regulatory submissions for the use of terlipressin as HRS type 1 therapy in the U.S and Canada."

About the CONFIRM Trial
The Phase 3 study is titled, "A Multi-Center, Randomized, Placebo Controlled, Double-Blind Study to Confirm Efficacy and Safety of Terlipressin in Subjects With Hepatorenal Syndrome Type 1 (The CONFIRM Study)." The primary endpoint of the study is verified HRS reversal in a period of up to 14 days. As is typical with sizeable rare disease clinical trials, the study is expected to take a number of years.

Find more information about the CONFIRM trial here on the ClinicalTrials.gov website.

About Hepatorenal Syndrome
HRS type 1 is characterized by a rapid, progressive renal impairment and has a very poor prognosis, with >80% mortality within three months. HRS is a rare syndrome of marked renal dysfunction in patients with cirrhosis, decompensated liver disease and portal hypertension. At present, there are no approved drug therapies for HRS type 1 in the U.S. or Canada. The only curative treatment for HRS type 1 and the underlying end-stage cirrhosis is liver transplantation. However, many patients will not survive long enough to receive a liver transplant. Increased understanding of the pathophysiology of HRS type 1 has demonstrated that vasoconstrictive drug therapy may reverse HRS type 1.

About Terlipressin
Terlipressin is a synthetic vasopressin analogue in development for the treatment of HRS type 1 in the U.S. and Canada.

ABOUT MALLINCKRODT
Mallinckrodt is a global business that develops, manufactures, markets and distributes specialty pharmaceutical and biopharmaceutical products and therapies, as well as nuclear imaging products. Areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, nephrology and pulmonology; immunotherapy and neonatal respiratory critical care therapies; analgesics and hemostasis products; and central nervous system drugs. The company's core strengths include the acquisition and management of highly regulated raw materials and specialized chemistry, formulation and manufacturing capabilities. The company's Specialty Brands segment includes branded medicines; its Specialty Generics segment includes specialty generic drugs, active pharmaceutical ingredients and external manufacturing; and the Nuclear Imaging segment includes nuclear imaging agents. To learn more about Mallinckrodt, visit www.mallinckrodt.com.

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CONTACTS

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314-654-8618
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Meredith Fischer
Senior Vice President, Communications and Public Affairs
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Investor Relations
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¹ Nadim, M., et al. Management of the critically ill patient with cirrhosis: A multidisciplinary perspective. Journal of Hepatology 2016, vol. 64, 717–735.
² Angeli, P., et al. Diagnosis and management of acute kidney injury in patients with cirrhosis: Revised consensus recommendations of the International Club of Ascites. Journal of Hepatology 2015, vol. 62, 968–974.
³ Nadim, M., et al. Hepatorenal syndrome: the 8th international consensus conference of the Acute Dialysis Quality Initiative (ADQI) Group. Critical Care 2012 16:R23.
⁴ European Association for the Study of the Liver clinical practice guidelines on the management of ascites, spontaneous bacterial peritonitis, and hepatorenal syndrome in cirrhosis. Journal of Hepatology 2010, vol. 53, 397–417.

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