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 75th 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 Australia1,2,3,4.
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