Mallinckrodt Reports Top-Line Results from Proof-of-Concept Study of Nitric Oxide Gas in Ex-Vivo System of Human Lung Transplants

STAINES-UPON-THAMES, United Kingdom, April 18, 2019 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced top-line results from its company-sponsored, proof-of-concept study of nitric oxide gas (gNO) with ex-vivo lung perfusion compared to lung perfusion alone in human lung transplants.

The study's primary objectives were to assess the effect of nitric oxide in perfused ex-vivo lungs using an experimental, proprietary grading system to evaluate improvements in lung function including oxygenation and transplantability, as well as the total ex-vivo perfusion time of the lungs through periodic assessments during Ex-Vivo Lung Perfusion (EVLP) as compared to standard of care alone.

Top-line results from the randomized and blinded portion of this proof of concept study showed improvement in lung function and oxygenation of lungs perfused with gNO, and a higher percentage of lungs were able to be preserved for a longer period in EVLP (up to 12 hours). The EVLP time period prior to lung transplantation in this study is longer than the current standard of four to six hours.

Matthew Hartwig, M.D., a lung transplant surgeon at Duke University and the study's Principal Investigator, said: "We are very excited about the preliminary results from this proof-of-concept study. We still have a lot of work to do, but in human lungs not suitable for clinical transplantation, the study showed an improvement in oxygenation and a longer duration of out-of-body perfusion time in lungs perfused with gNO, which are both important parts of a successful ex-vivo lung perfusion."

EVLP describes a process of evaluating, rehabilitating, and improving possible donor lungs outside the body. At normal body temperature, the lungs are perfused and 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. 

"Mallinckrodt is focused on providing treatment options for underserved patients' needs, improving outcomes for those 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, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. "We are pleased to see these encouraging preliminary, top-line data, and plan to publish the full data set at a future research meeting. This is important research in evaluating the therapy's potential use in lung transplantation as the company seeks to establish and grow its presence in a key therapeutic area."  

About the Study
This proof-of-concept, randomized, multicenter, blinded study assessed the effects of gNO in modified criteria human lung transplants via EVLP. It was conducted at three major U.S. lung transplant centers. The study evaluated extended (modified) criteria bilateral donor lungs, not eligible for lung transplantation. The study is ongoing with a small open-label sub-study in a single center to be completed in the second quarter of 2019.

About Lung Transplantation in Humans
In the U.S. each year, over 2,200 people receive a lung transplant¹ – the surgical replacement of either a single or two (double) severely 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.² The major limiting factor to the number of transplants performed is donor lung shortage.³ According to Organ Procurement Transplant Network data, approximately 130.5 deaths per 100,000 persons with severe lung disease occurred in 2014, with about 1400 patients with severe lung disease on the waiting list in the U.S.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 concerning the study described in this release, 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; 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
Media
Daniel Yunger
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Investor Relations
Daniel J. Speciale, CPA
Vice President, Investor Relations and IRO
314-654-3638
[email protected]

Mallinckrodt, the "M" brand mark and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company.
© 2019 Mallinckrodt. US1900533 4/19

¹ 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.
² Mayo Clinic Patient Care and Health Information, Lung Transplant.  https://www.mayoclinic.org/tests-procedures/lung-transplant/about/pac-20384754 Accessed April 8, 2019.
³ Van Raemdonck D; Neyrinck A; Verleden GM, et al. Lung Donor Selection and Management. Annals of the American Thoracic Society. 2009; 6:28-38.
⁴ Organ Procurement and Transplant Network Data. https://optn.transplant.hrsa.gov/data/ Accessed April 8, 2019.

SOURCE Mallinckrodt Pharmaceuticals

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