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

-- Mallinckrodt's Trial Designed to Measure Ability to Achieve Low Disease Activity --

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Nov 14, 2016, 07:00 ET

CHESTERFIELD, United Kingdom, Nov. 14, 2016 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty biopharmaceutical company, today confirmed enrollment of the first patient in the company's Phase 4 clinical study assessing the efficacy and safety of H.P. Acthar^® Gel (repository corticotropin injection) in rheumatoid arthritis (RA) patients with persistently active disease.

H.P. Acthar Gel is approved by the U.S. Food and Drug Administration (FDA) for use as adjunct therapy for short-term administration in RA to tide the patient over an acute episode or exacerbation.¹

"A significant number of patients with rheumatoid arthritis will have active symptoms and signs of joint inflammation despite the use of non-biologic and/or biologic disease modifying therapies. Mallinckrodt is committed to providing treatment options for appropriate difficult-to-treat patients like these who have limited alternatives," said Steven Romano, M.D., Chief Scientific Officer and Executive Vice President at Mallinckrodt. "Clinical experience supports the use of Acthar as therapy for patients with RA, for which it is already indicated. We are pleased to enroll the first patient in this clinical trial designed to better understand the impact of Acthar on persistent disease activity in these patients."

About the Trial
The Phase 4 trial is titled, "A multicenter, 2-part study to assess the efficacy and safety of H.P. Acthar Gel in subjects with rheumatoid arthritis with persistently active disease." The primary endpoint of the study is to evaluate H.P. Acthar Gel treatment response, as measured by the DAS28-ESR (Disease Activity Score with 28 joint count and erythrocyte sedimentation rate) at week 12 (Part 1). Subjects who achieve low disease activity will enter a 12-week, double-blind randomized maintenance period (Part 2).

The target patient enrollment is 230. As is typical with sizeable clinical trials involving a small subset of a disease population, the company expects to take a number of years to complete the study.

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

About Rheumatoid Arthritis
RA is an autoimmune disease. It is a chronic condition that causes pain, stiffness, and swelling of the joints—all symptoms caused by inflammation. An estimated 1.5 million U.S. adults are living with RA.² Treatment is aimed at stopping inflammation to put the disease in remission and relieve symptoms.³ Nonsteroidal anti-inflammatory drugs are used to ease symptoms whereas corticosteroids, disease-modifying anti-rheumatic drugs and biologics are used to slow down the disease activity.³

About H.P. Acthar Gel (repository corticotropin injection)
H.P. Acthar Gel 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 systemic lupus erythematosus.
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 RA.
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
[email protected]

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
[email protected]

¹ H.P. Acthar^® Gel (repository corticotropin injection) [prescribing information]. Mallinckrodt ARD, Inc.
² What is Rheumatoid Arthritis? Arthritis Foundation. Available at: http://www.arthritis.org/about-arthritis/types/rheumatoid-arthritis/what-is-rheumatoid-arthritis.php. Accessed October 19, 2016.
³ Arthritis Foundation. Rheumatoid Arthritis Treatment. http://www.arthritis.org/about-arthritis/types/rheumatoid-arthritis/treatment.php. Accessed October 19, 2016.

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