STAINES-UPON-THAMES, United
Kingdom, Oct. 21, 2019
/PRNewswire/ -- Mallinckrodt
plc (NYSE: MNK), a global biopharmaceutical company, today
announced it will present results from its pivotal Phase 3 CONFIRM
study in a late-breaker session on Monday,
Nov. 11 at 3:30 pm ET during
The Liver Meeting® 2019, the annual meeting of the American
Association for the Study of Liver Diseases (AASLD) in Boston.
"We are honored to be presenting our CONFIRM abstract at AASLD,"
said Steven Romano, M.D.,
Executive Vice President and Chief Scientific Officer
at Mallinckrodt. "We are dedicated to developing potential
treatments for patients with severe and critical conditions,
including complications of advanced liver disease, such as
hepatorenal syndrome type 1."
The CONFIRM clinical trial assessed the efficacy and safety of
terlipressin in 300 adults with hepatorenal syndrome type 1
(HRS-1), an acute and life-threatening syndrome involving acute
kidney failure in people with cirrhosis.1 CONFIRM is the
largest prospective trial ever conducted in this patient
population. Mallinckrodt announced in August that the study
met its primary endpoint of verified HRS-1 reversal (p=0.012).
"Verified HRS-1 reversal" is a term denoting three components:
renal function improvement, avoidance of dialysis and short-term
survival.
The company plans to submit a New Drug Application to the U.S.
Food and Drug Administration (FDA) in early 2020. Terlipressin is
an investigational product and its safety and effectiveness have
not yet been established by the FDA or Health Canada.
"HRS-1 is estimated to affect between 30,000 and 40,000 patients
in the U.S.2,3 annually. It is a rapidly
progressing and devastating condition, and many patients don't live
beyond a few weeks if left untreated,"4,5 said
presenting author Florence Wong, MBBS, MD, FRACP, FRCPC,
hepatologist at Toronto General Hospital, and professor of Medicine
at the University of Toronto. "I am encouraged by the
results of the CONFIRM trial of terlipressin, which, if approved,
may make a difference in this difficult-to-treat patient population
and I look forward to sharing these results for the first time at
AASLD."
HRS-1 has a very poor prognosis, with a median survival of less
than two weeks and greater than 80 percent mortality within three
months if left untreated.4,5 At present, there are
no approved drug therapies for HRS-1 in the U.S.
or Canada.6
About the Pivotal Phase 3 CONFIRM Study (multi-center,
randomized, placebo-controlled, double-blind trial in the U.S. and
Canada):7
- The trial was designed to confirm efficacy and safety of
terlipressin for the treatment of HRS-1
- CONFIRM is the largest prospective trial ever conducted (n=300)
in HRS-1 patients
- Eligibility criteria included adults with liver cirrhosis and
ascites with rapidly worsening renal function and no response to
diuretics or albumin
- Subjects were randomized in a 2:1 ratio to receive terlipressin
plus albumin (n=199) or placebo plus albumin (n=101)
- The primary endpoint of Verified HRS reversal was intended to
evaluate renal function improvement, avoidance of dialysis and
short-term survival
Find out more information about the CONFIRM
trial here on the ClinicalTrials.gov website.
About Terlipressin
Terlipressin is a potent,
vasopressin analogue selective for V1 receptors being investigated
for the treatment of HRS-1 in the U.S. and Canada.
Terlipressin is an investigational product in these countries as
the safety and efficacy have not been established with, nor has
approval been granted by regulatory authorities in either country.
Terlipressin is approved for use outside the U.S. and Canada.
ABOUT MALLINCKRODT
Mallinckrodt is a
global business consisting of multiple wholly owned subsidiaries
that develop, manufacture, market and distribute specialty
pharmaceutical products and therapies. The company's Specialty
Brands reportable segment's 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; analgesics and
gastrointestinal products. Its Specialty Generics reportable
segment includes specialty generic drugs and active pharmaceutical
ingredients. 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.
CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING
STATEMENTS
This release includes forward-looking statements
with regard to terlipressin and the study described in this
release, including expectations with regard to future regulatory
filings and its potential impact on patients. The statements are
based on assumptions about many important factors, including the
following, which could cause actual results to differ materially
from those in the forward-looking statements: satisfaction of
regulatory and other requirements; actions of regulatory bodies and
other governmental authorities; changes in laws and regulations;
issues with product quality, manufacturing or supply, or patient
safety issues; and other risks identified and described in more
detail in the "Risk Factors" section of Mallinckrodt's most recent Annual Report on Form
10-K and other filings with the SEC, all of which are available on
its website. The forward-looking statements made herein speak only
as of the date hereof and Mallinckrodt
does not assume any obligation to update or revise any
forward-looking statement, whether as a result of new information,
future events and developments or otherwise, except as required by
law.
CONTACT
For Trade Media
Inquiries
Caren Begun
Green Room Communications
201-396-8551
caren@greenroompr.com
For Financial/Dailies Media Inquiries
Daniel Yunger
Kekst CNC
212-521-4879
mallinckrodt@kekstcnc.com
Investor Relations
Daniel J.
Speciale, CPA
Vice President, Investor Relations and IRO
314-654-3638
daniel.speciale@mnk.com
Mallinckrodt, the "M" brand mark and
the Mallinckrodt Pharmaceuticals logo are trademarks of
a Mallinckrodt company. Other brands are trademarks of
a Mallinckrodt company or their respective owners.
© 2019 Mallinckrodt. US-1901869 10/19
References
1 National Organization for Rare Disorders.
Hepatorenal Syndrome. Available
at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/.
Accessed April 9, 2019
2 C Pant, B S Jani, M Desai, A Deshpande,
Prashant Pandya, Ryan Taylor, R Gilroy, M Olyaee. Hepatorenal
syndrome in hospitalized patients with chronic liver disease:
results from the Nationwide Inpatient Sample 2002–2012. J Investig
Med 2016;64:33–38.
3 US Census 2018
https://www.census.gov/search-results.html?searchType=web&cssp=SERP&q=US
population 2018, accessed on 06 August
2019.
4 Colle I and Laterre PF. Hepatorenal syndrome: the
clinical impact of vasoactive therapy, Expert Review of
Gastroenterology & Hepatology. (2018) 12:2, 173-188, DOI:
10.1080/17474124.2018.1417034.
5 Gines P, Sola E, Angeli P, et al. Hepatorenal
syndrome. Nature Reviews. (2018) 4:23.
6 Boyer TD, Medicis JJ, Pappas SC, et al. A
randomized, placebo-controlled, double-blind study to confirm the
reversal of hepatorenal syndrome type 1 with terlipressin: the
REVERSE trial design. Open Access Journal of Clinical
Trials 2012:4. https://www.dovepress.com/a-randomized-placebo-controlled-double-blind-study-to-confirm-the-reve-peer-reviewed-article-OAJCT.
7 Data on file. Mallinckrodt ARD, Inc.
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SOURCE Mallinckrodt plc