First Patient Enrolled in Mallinckrodt's Phase 2 Duchenne Muscular Dystrophy Trial

-- Trial to Assess Efficacy and Safety of Investigational Drug MNK-1411 in Patients with DMD --

-- European Medicines Agency Grants MNK-1411 Orphan Medicinal Product Designation --

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Aug 02, 2018, 06:45 ET

STAINES-UPON-THAMES, United Kingdom, Aug. 2, 2018 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today confirmed enrollment of the first patient in the company's Phase 2 study assessing the efficacy and safety of investigational drug MNK-1411 (cosyntropin, also known as tetracosactide) in patients ages four to eight with Duchenne Muscular Dystrophy (DMD).

"We are very excited Mallinckrodt has chosen to study how this investigational drug, MNK-1411, might help in treating Duchenne Muscular Dystrophy," said Pat Furlong, President and CEO, Parent Project Muscular Dystrophy. "In the fight to end Duchenne Muscular Dystrophy, our community welcomes partners dedicated to this goal."

The company also announced that MNK-1411 recently received the European Medicines Agency (EMA) Orphan Medicinal Product designation for the potential treatment of Duchenne Muscular Dystrophy.

"Mallinckrodt is focused on meeting the unmet medical needs of patients, particularly those with rare diseases like DMD," said Steven Romano, MD, Executive Vice President and Chief Scientific Officer at Mallinckrodt. "We are pleased to enroll the first patient in our Phase 2 clinical trial of investigational drug MNK-1411 in boys with DMD. We are also excited by the recent EMA designation of Orphan Drug status as confirmation of the potential benefit of this investigational therapy for European patients."

About the Clinical Trial
The Phase 2 clinical study is entitled: A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy (the BRAVE study). The study will include patients who have a diagnosis of DMD confirmed by complete dystrophin deficiency, or an identifiable mutation in the DMD gene, or complete dystrophin gene sequencing consistent with DMD; patients must also have a typical clinical profile consistent with DMD to be included.

Approximately 130 patients who meet eligibility criteria will be randomly assigned in a 2:1 ratio to receive one of two weight-based doses of MNK-1411, or they will receive volume-matched placebo, administered subcutaneously 2 times/week for 24 weeks. Study subjects who complete the 24-week randomized, double-blind, placebo-controlled treatment period will be eligible to receive active treatment for an additional 24 weeks during an optional open-label extension. Key efficacy outcome measures for the study will include timed function tests, such as a 10-meter walk/run, 4-stair climb and rise from floor, and the NorthStar Ambulatory Assessment.

Find more information about the U.S. trial here for the U.S. Food and Drug Administration's ClinicalTrials.gov website. Details can be found here for Belgium and here for Spain on the European Union Clinical Trial register website.

About MNK-1411
MNK-1411 (cosyntropin), an investigational drug, is a depot formulation of cosyntropin acetate, a synthetic 24 amino acid melanocortin receptor agonist formulated for prolonged release.

About Duchenne Muscular Dystrophy (DMD)
DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5. The disease primarily affects boys. Symptoms include muscle weakness, which can begin as early as age 3, first affecting the muscles of the hips, pelvic area, thighs and shoulders, and later the skeletal (voluntary) muscles in the arms, legs and trunk.¹

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 the investigational drug MNK-1411 including expectations with regard to the study described in this release as well as 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.

CONTACTS
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Daniel J. Speciale, CPA
Investor Relations and Strategy Officer
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[email protected]

Media
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Senior Communications Manager
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Meredith Fischer
Chief Public Affairs Officer
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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 Mallinckrodt. US-1800213 08/18.