Mallinckrodt Phase 3 Terlipressin Trial Achieves its Enrollment Target Ahead of Schedule

STAINES-UPON-THAMES, United Kingdom, July 13, 2017 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced it has enrolled the 75^th subject in the company's ongoing Phase 3 pivotal trial CONFIRM to evaluate the efficacy and safety of terlipressin (for injection) in subjects with Hepatorenal Syndrome (HRS) type 1.

Phase 3 trial enrollment has been robust and the company has achieved one quarter of its targeted enrollment for this trial aligned with the company's expectations. Additionally, an important interim analysis of trial data to assess the safety and efficacy of terlipressin in study subjects will be completed when 150 subjects are enrolled and this also marks the halfway point toward that milestone.

Terlipressin is being investigated for the treatment of HRS type 1, an acute, rare and life-threatening condition requiring hospitalization, with currently no approved therapy in the U.S. or Canada. The safety and efficacy of terlipressin for the treatment of HRS type 1 has not been established with the U.S. Food and Drug Administration or Health Canada.  Terlipressin is approved for use in countries outside the U.S., including Europe, Latin America, Asia and Australia^1,2,3,4.

The interim analysis has been approved under the Special Protocol Assessment agreed upon with the U.S. Food and Drug Administration. Though it sets a high hurdle for determining safety and efficacy of the study drug, it also allows for potential early termination of the study. Given the known profile of terlipressin in many countries for this patient population, and the unmet need in HRS type 1 in the U.S. and Canada, Mallinckrodt looks forward to the results of the interim analysis.

"Mallinckrodt is committed to advancing therapies for patients with rare diseases and critical conditions who have limited treatment options such as those suffering from HRS type 1," said Steven Romano, M.D., Chief Scientific Officer and Executive Vice President at Mallinckrodt. "We are pleased to reach this important milestone in the terlipressin CONFIRM trial, and look forward to learning more about how the drug can help patients with this serious condition."

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 type 1 reversal in a period of up to 14 days.

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

About Hepatorenal Syndrome
HRS is characterized by rapid, progressive functional renal failure and has a very poor prognosis, with >80 percent 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.

About Terlipressin
Terlipressin is a synthetic vasopressin analogue being investigated for the treatment of HRS type 1 in the U.S. and Canada. Safety and efficacy has not been established with nor has approval been granted by regulatory authorities in either country.

ABOUT MALLINCKRODT
Mallinckrodt is a global business that develops, manufactures, markets and distributes specialty pharmaceutical products and therapies. Areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, nephrology, pulmonology and ophthalmology; immunotherapy and neonatal respiratory critical care therapies; and analgesics and hemostasis products. 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 and its Specialty Generics segment includes specialty generic drugs, active pharmaceutical ingredients and external manufacturing. To learn more about Mallinckrodt, visit www.mallinckrodt.com.

Mallinckrodt uses its website as a channel of distribution of important company information, such as press releases, investor presentations and other financial information. It also uses its website to expedite public access to time-critical information regarding the company in advance of or in lieu of distributing a press release or a filing with the U.S. Securities and Exchange Commission (SEC) disclosing the same information. Therefore, investors should look to the Investor Relations page of the website for important and time-critical information. Visitors to the website can also register to receive automatic e-mail and other notifications alerting them when new information is made available on the Investor Relations page of the website.

CONTACTS
Investor Relations
Coleman N. Lannum, CFA
Senior Vice President, Investor Strategy and IRO
314-654-6649
cole.lannum@mallinckrodt.com

Daniel J. Speciale, CPA
Director, Investor Relations
314-654-3638
daniel.speciale@mallinckrodt.com

Media
Rhonda Sciarra
Senior Communications Manager
908-238-6765
rhonda.sciarra@mallinckrodt.com

Meredith Fischer
Chief Public Affairs Officer
314-654-3318
meredith.fischer@mallinckrodt.com

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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SOURCE Mallinckrodt plc