First Patient Enrolled in Mallinckrodt Phase 4 Trial of H.P. Acthar® Gel for Lupus

-- Mallinckrodt's Trial Designed to Measure Improvement in Disease Activity --

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Nov 14, 2016, 04:30 ET

CHESTERFIELD, United Kingdom, Nov. 14, 2016 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty biopharmaceutical company, today confirmed enrollment of the first patients in the company's Phase 4 clinical study assessing the efficacy of H.P. Acthar^® Gel (repository corticotropin injection) in Systemic Lupus Erythematosus (SLE) patients with persistently active disease. This randomized, double-blind, placebo-controlled trial builds on data from a pilot study of H.P. Acthar Gel in patients with active SLE, recently published online in the journal Lupus Science & Medicine in a manuscript titled "Efficacy and tolerability of repository corticotropin injection in patients with persistently active SLE: results of a phase 4, randomized, controlled pilot study."

H.P. Acthar^® Gel is approved by the U.S. Food and Drug Administration (FDA) for use during an exacerbation or as a maintenance therapy in select patients with SLE.¹

"The data generated in our pilot clinical study support the use of Acthar to treat lupus patients – for which it is already indicated - who have clinically significant disease activity despite receiving standard of care therapies," said Steven Romano, M.D., Chief Scientific Officer and Executive Vice President at Mallinckrodt. "We are pleased to enroll the first patient in this follow-on trial, which will generate additional data to better understand how clinicians may utilize Acthar in the management of these more difficult to manage patients with SLE."

About the Trial
The Phase 4 trial is titled, "A multicenter, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of H.P. Acthar Gel in subjects with persistently active SLE despite moderate dose corticosteroids." The primary endpoint of the study is to measure reduction in disease activity as reflected by the SLE Responder Index (SRI) at week 16. The SRI is a composite endpoint that includes three different measures of disease activity to reflect response of SLE to therapy. The target patient enrollment is 160. As is typical with sizeable clinical trials in segments of a disease population, the study is expected to take several years.

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

About Systemic Lupus Erythematosus
SLE is an autoimmune disease in which the immune system produces antibodies to cells within the body leading to widespread inflammation and tissue damage.² It is the most common form of lupus, a condition that impacts an estimated 1.5 million Americans.³ Ninety percent of those diagnosed with lupus are women, often between the ages of 15-44.³ Lupus is characterized by periods of illness "flares" and remissions and the disease can affect the joints, skin, brain, lungs, kidneys, and blood vessels. Symptoms may include fatigue, pain or swelling in joints, skin rashes, and fevers.²

About H.P. Acthar Gel (repository corticotropin injection)
H.P. Acthar Gel (repository corticotropin injection), is an injectable drug approved by the FDA for the treatment of 19 indications. Of these, today the majority of Acthar use is in these indications:

Orphan monotherapy for treatment of infantile spasms (IS) in infants and children under 2 years of age.
Inducing a diuresis or a remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus.
Treatment of acute exacerbations of multiple sclerosis in adults.
Use during an exacerbation or as maintenance therapy in selected cases of SLE.
Use during an exacerbation or as maintenance therapy in selected cases of systemic dermatomyositis (polymyositis).
Use as adjunct therapy for short-term administration in select cases of rheumatoid arthritis.
Treatment of symptomatic sarcoidosis.

For more information about Acthar, please visit www.acthar.com. Full Prescribing Information may be accessed here.

Important Safety Information

Acthar should never be administered intravenously.
Administration of live or live attenuated vaccines is contraindicated in patients receiving immunosuppressive doses of Acthar.
Acthar is contraindicated where congenital infections are suspected in infants.
Acthar is contraindicated in patients with scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of a peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, adrenocortical hyperfunction or sensitivity to proteins of porcine origins.
The adverse effects of Acthar are related primarily to its steroidogenic effects.
Acthar may increase susceptibility to new infection or reactivation of latent infections.
Suppression of the hypothalamic–pituitary–adrenal (HPA) axis may occur following prolonged therapy with the potential for adrenal insufficiency after withdrawal of the medication. Cushing's Syndrome may occur during therapy but generally resolves after therapy is stopped. Monitor patients for signs and symptoms.
Monitor patients for elevation of blood pressure, salt and water retention, and hypokalemia.
Acthar often acts by masking symptoms of other diseases/disorders. Monitor patients carefully during and following discontinuation.
Acthar can cause gastrointestinal (GI) bleeding and gastric ulcer with an increased risk for perforation with certain GI disorders. Monitor for signs of bleeding.
Acthar may be associated with central nervous system effects ranging from euphoria, insomnia, irritability, mood swings, personality changes, depression, and psychosis. Existing conditions may be aggravated.
Patients with comorbid disease may have that disease worsened. Caution should be used in patients with diabetes and myasthenia gravis.
Prolonged use of Acthar may produce cataracts, glaucoma and secondary ocular infections.
Acthar is immunogenic and prolonged use may increase the risk of hypersensitivity reactions.
There is an enhanced effect in patients with hypothyroidism and those with cirrhosis of liver.
Long-term use may have negative effects on growth and physical development in children. Monitor pediatric patients.
Decrease in bone density may occur. Monitor during long-term therapy.
Pregnancy Class C: Acthar has been shown to have an embryocidal effect and should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus.
Common adverse reactions include fluid retention, alteration in glucose tolerance, elevation in blood pressure, behavioral and mood changes, increased appetite and weight gain.
Specific adverse reactions reported in IS clinical trials in infants and children under 2 years of age included: infection, hypertension, irritability, Cushingoid symptoms, constipation, diarrhea, vomiting, pyrexia, weight gain, increased appetite, decreased appetite, nasal congestion, acne, rash, and cardiac hypertrophy. Convulsions were also reported, but these may actually be occurring because some IS patients progress to other forms of seizures and IS sometimes masks other seizures, which become visible once the clinical spasms from IS resolve.

Please see full Prescribing Information here for additional Important Safety Information.

ABOUT MALLINCKRODT
Mallinckrodt is a global business that develops, manufactures, markets and distributes specialty pharmaceutical and biopharmaceutical products and therapies, as well as nuclear imaging products. 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 hemostasis products; and central nervous system drugs. The company's core strengths include the acquisition and management of highly regulated raw materials and specialized chemistry, formulation and manufacturing capabilities. The company's Specialty Brands segment includes branded medicines; its Specialty Generics segment includes specialty generic drugs, active pharmaceutical ingredients and external manufacturing; and the Nuclear Imaging segment includes nuclear imaging agents. To learn more about Mallinckrodt, visit www.mallinckrodt.com.

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CONTACTS

Media
Rhonda Sciarra
Senior Communications Manager
908-238-6765
[email protected]

Meredith Fischer
Chief Public Affairs Officer
314-654-3318
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Investor Relations
Coleman N. Lannum, CFA
Senior Vice President, Investor Strategy and IRO
314-654-6649
[email protected]

Daniel J. Speciale, CPA
Director, Investor Relations
314-654-3638
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¹ H.P. Acthar^® Gel (repository corticotropin injection) [prescribing information]. Mallinckrodt ARD, Inc.
² Systemic lupus erythematosus (SLE or lupus), The Centers for Disease Control and Prevention, Available at: http://www.cdc.gov/arthritis/basics/lupus.htm. Accessed October 19, 2016.
³ Lupus Foundation of America Press Kit, About Us. Available at: http://www.lupus.org/about/statistics-on-lupus. Accessed October 19, 2016.

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