Mallinckrodt Initiates Proof-of-Concept Study of Nitric Oxide Gas in Ex-Vivo System of Human Lung Transplants

-- Study Begins Evaluation on First Set of Lungs, Will Assess Organs Using Proprietary Grading System --

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Aug 01, 2018, 16:45 ET

STAINES-UPON-THAMES, United Kingdom, Aug. 1, 2018 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced it has initiated a company-sponsored, experimental, proof-of-concept study of nitric oxide gas with ex-vivo lung perfusion compared to lung perfusion alone in human lung transplants. The study has enrolled its first set of lungs, and the actual lungs to be evaluated are being assigned for research use, not for human lung transplantation.

The study's primary objectives are to assess the effects of nitric oxide in perfused ex-vivo lungs using a proprietary grading system and to evaluate the total ex-vivo perfusion time of the lungs through periodic assessments during Ex-Vivo Lung Perfusion (EVLP).

EVLP describes a process of evaluating and preparing donor lungs outside the body; nutrients are perfused, or circulated, through the organ, and the organ is ventilated to mimic physiologic function prior to transplant surgery. EVLP has the potential to increase the available donor pool through restoring and repairing donor lungs.¹

"Mallinckrodt is focused on providing treatment options for the unmet needs of and to improve outcomes for patients with severe and critical conditions. The company has more than 20 years of experience with and investment in nitric oxide as a signaling molecule," said Steven Romano, MD, Executive Vice President and Chief Scientific Officer at Mallinckrodt. "We are pleased to begin and learn from this important research evaluating nitric oxide gas for potential use in lung transplantation as the company seeks to establish and grow its presence in this therapeutic area."

About the Study
This proof-of-concept randomized, multicenter, blinded study will assess the effects of nitric oxide in modified criteria human lung transplants via EVLP. It is planned to be conducted at four major U.S. lung transplant centers through the fourth quarter of 2018. The study will evaluate extended criteria bilateral donor lungs, not suitable for lung transplantation. Upon a successful proof-of-concept study, the company expects to initiate a safety study to assess use of nitric oxide in human lung transplantation in 2019.

About Lung Transplantation in Humans
In the U.S. each year, about 2,000 people receive a lung transplant²– the surgical replacement of either a single or two (double) badly diseased lungs with healthy lungs from a human organ donor. A variety of diseases and conditions can damage a person's lungs and hinder their ability to function effectively, such as chronic obstructive pulmonary disease (COPD) including emphysema; scarring of the lungs; pulmonary fibrosis; cystic fibrosis; sarcoidosis; and pulmonary hypertension.³ A major limiting factor to the number of transplants performed is donor lung shortage.⁴ According to Organ Procurement Transplant Network data, approximately 1,400 patients with severe lung disease are on the waiting list for a transplant.⁵

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; analgesics and gastrointestinal products. 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 concerning Mallinckrodt's study of nitric oxide in modified criteria human lung transplants via Ex-Vivo Lung Perfusion including expectations with regard to the study, future research plans and the 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; future commercialization efforts; 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.

CONTACTS
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Rhonda Sciarra
Senior Communications Manager
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Meredith Fischer
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Investor Relations
Daniel J. Speciale, CPA
Investor Relations and Strategy Officer
314-654-3638
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Mallinckrodt, the "M" brandmark and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners. © 2018 08/18


¹Columbia University Department of Surgery Center for Advanced Lung Disease and Transplantation. http://columbiasurgery.org/conditions-and-treatments/ex-vivo-lung-perfusion Accessed April 4, 2018.
² Valapour M, Skeans MA, Smith JM, et al. OPTN/SRTR 2015 Annual Data Report: Lung. American Journal of Transplantation. 2017;17:357-424. doi:10.1111/ajt.14129.
³ Mayo Clinic Patient Care and Health Information, Lung Transplant. https://www.mayoclinic.org/tests-procedures/lung-transplant/about/pac-20384754 Accessed April 9, 2018.
⁴ Van Raemdonck D; Neyrinck A; Verleden GM, et al. Lung Donor Selection and Management. Annals of the American Thoracic Society. 2009; 6:28-38.
⁵ Organ Procurement and Transplant Network Data. https://optn.transplant.hrsa.gov/data/ Accessed April 26, 2018.