In the 35-month study period, 300 patients from the U.S. (89 percent) and
The study met its primary endpoint of Verified HRS Reversal (VHRSR), which is defined as renal function improvement, avoidance of dialysis and short-term survival. 29.1 percent (58/199) of patients administered terlipressin plus albumin achieved Verified HRS Reversal versus 15.8 percent (16/101) on placebo plus albumin (p=0.012). In order to achieve Verified HRS Reversal, patients had to have two consecutive SCr values ≤1.5 mg/dL, at least two hours apart by day 14 or hospital discharge, and be alive without intervening renal replacement therapy (RRT) for at least 10 days following discharge or treatment.2
"HRS-1 is a rapidly progressing and often fatal disease that is extremely difficult to diagnose and treat, and many patients don't live beyond a few weeks without treatment. The results from the CONFIRM trial are very encouraging, and show terlipressin, if approved, has the potential to reverse the course of HRS-1 as measured by renal function improvement, avoidance of dialysis and short-term survival," said presenting author
HRS-1 has a median survival time of less than two weeks and greater than 80 percent mortality within three months if left untreated.3,4 At present, there are no approved drug therapies for HRS-1 in the U.S. or Canada.5 HRS-1 is estimated to affect between 30,000 and 40,000 patients in the U.S. annually.6,7 Terlipressin is an investigational product and its safety and effectiveness have not yet been established by the U.S.
"We are grateful to all the patients and investigators who participated in the CONFIRM trial and greatly encouraged by the positive results, which demonstrated that terlipressin may have a potential impact on the progressively worsening kidney function that is the hallmark of HRS-1," said
CONFIRM Study Key Findings2
About the Pivotal Phase 3 CONFIRM Study (multi-center, randomized, placebo-controlled, double-blind trial in the U.S. and
Find out more information about the CONFIRM trial here on the ClinicalTrials.gov website.
Terlipressin is a potent, vasopressin analogue selective for V1 receptors being investigated for the treatment of HRS-1 in the U.S. and Canada. It 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.
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.
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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 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.
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1 National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/. Accessed April 9, 2019
2 Data on file.
3 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.
4 Gines P, Sola E, Angeli P, et al. Hepatorenal syndrome. Nature Reviews. (2018) 4:23.
5 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.
6 C Pant, B S Jani, M Desai, A Deshpande,
7 US Census 2018 https://www.census.gov/search-results.html?searchType=web&cssp=SERP&q=US population 2018, accessed on
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