Mallinckrodt Completes Enrollment of Phase 3 Terlipressin CONFIRM Trial

-- Mallinckrodt's CONFIRM study is evaluating the efficacy and safety of terlipressin in hepatorenal syndrome type 1 --

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May 14, 2019, 06:55 ET

STAINES-UPON-THAMES, United Kingdom, May 14, 2019 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced that the company has achieved target enrollment of 300 participants in its Phase 3 CONFIRM clinical study to evaluate the efficacy and safety of terlipressin, an investigational agent, in adult patients with Hepatorenal Syndrome Type 1 (HRS-1).

"Currently, there are no treatments approved in the U.S. and Canada for the treatment of HRS-1, which is a serious disease that has a high mortality rate," said lead investigator Arun Sanyal, M.D., Z. Reno Vlahcevic Professor of Medicine, Physiology and Molecular Pathology at Virginia Commonwealth University. "Reaching this critical milestone of full enrollment in the CONFIRM study is an important step forward for both Mallinckrodt and the hepatology community as there is a significant unmet medical need for these patients in the U.S. and Canada."

About the Phase 3 CONFIRM Study (multi-center, randomized, placebo controlled, double-blind trial):

Designed to confirm efficacy and safety of terlipressin to treat HRS-1
Largest prospective trial ever conducted in HRS-1 patients
Inclusion criteria include adults with liver cirrhosis and ascites with rapidly worsening renal function and no response to diuretics or albumin
The primary endpoint (Verified HRS Reversal) is designed to evaluate renal function improvement, avoidance of dialysis, and short-term survival

In 2016, Mallinckrodt and the U.S. Food and Drug Administration (FDA) reached agreement on the CONFIRM protocol design and data analysis under the agency's special protocol assessment (SPA) process. A SPA is an advanced declaration from the FDA that an uncompleted Phase 3 trial's design, clinical endpoints, and statistical data analyses are acceptable for FDA approval.

Top-line data of terlipressin, an investigational agent, is expected to be available by the end of 2019. Mallinckrodt anticipates submitting the New Drug Application in early 2020 to the FDA if data from the Phase 3 trial are supportive.

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

"We are proud to have reached full patient enrollment in the CONFIRM trial, which will support the regulatory submissions for the potential approval of terlipressin as a treatment for HRS-1 in the U.S and Canada," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. "The ongoing CONFIRM trial and development of terlipressin are testaments to Mallinckrodt's commitment to advancing therapies for patients with rare and severe illnesses."

About Terlipressin
Terlipressin is a potent, vasopressin analogue selective for V1a receptors being investigated for the treatment of HRS-1 in the U.S. and Canada. Terlipressin is an investigational product in these countries and 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 Hepatorenal Syndrome Type 1 (HRS-1)
HRS-1 is a life-threatening, rare, and acute disease characterized by complications of liver disease that leads to kidney failure.[1] HRS-1 has a very poor prognosis, with a median survival time of less than two weeks and greater than 80 percent mortality within three months.[2]^,[3] At present, there are no approved drug therapies for HRS-1 treatment in the U.S. or Canada.[4]

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; and analgesics. Its Specialty Generics and Amitiza reportable segment includes specialty generic drugs, active pharmaceutical ingredients and AMITIZA^® (lubiprostone). 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, including with regard related clinical data and regulatory filings, its potential impact on patients and anticipated benefits associated with its use. 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: clinical trial results, 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

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Caren Begun	Daniel Yunger	Daniel J. Speciale, CPA
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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-1900637 5/19

References

¹ National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/. Accessed April 10, 2019.
² 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.
³ Gines P, Sola E, Angeli P, et al. Hepatorenal syndrome. Nature Reviews. (2018) 4:23.
⁴ 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.

SOURCE Mallinckrodt plc