First Patient Enrolled in Mallinckrodt Phase 4 Trial of H.P. Acthar® Gel (Repository Corticotropin Injection) for Multiple Sclerosis Relapse

STAINES-UPON-THAMES, United Kingdom, May 23, 2017 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today confirmed enrollment of the first patient in the company's Phase 4 pilot study assessing the efficacy of H.P. Acthar^® Gel (repository corticotropin injection) for the management of exacerbations of relapsing and remitting multiple sclerosis (RRMS) in patients whose relapse symptoms have not responded to a course of high-dose corticosteroids.

"Some MS patients may have relapse symptoms that don't resolve with steroid therapy, and in my experience, H.P. Acthar Gel may provide benefit in these cases," said Aaron Miller, M.D., a program advisor and participating site investigator from the Icahn School of Medicine at Mount Sinai in New York. "This study is an important step to confirming the appropriate patient type for this product as an approved and appropriate treatment for MS exacerbations."

H.P. Acthar Gel is U.S. Food and Drug Administration (FDA)-approved for treatment of acute exacerbations of multiple sclerosis in adults.

"Mallinckrodt is committed to providing alternative treatment options to patients who need them," said Steven Romano, M.D., Chief Scientific Officer and Executive Vice President at Mallinckrodt. "Clinical experience and current labeling support the use of Acthar to speed recovery from relapses in MS. We are pleased to enroll the first patient in the OPTIONS trial to better understand the patients who will benefit from Acthar therapy, and to support its role in the management of RRMS."

About the OPTIONS Trial
The Phase 4 clinical study is titled "A Multicenter, Randomized, Double-Blind, Placebo-Controlled Parallel Group, Pilot Study to Assess the Efficacy and Safety of H.P. Acthar Gel in Subjects With Relapsing-Remitting Multiple Sclerosis". The study will enroll patients with MS relapses of moderate severity and symptoms that have not adequately improved following a typical course of high dose IV or oral corticosteroid therapy, then randomize them to receive additional treatment with H.P. Acthar Gel or placebo in blinded fashion. 

Improvement in relapse symptoms will be assessed using several standard MS disease activity measures, including the Expanded Disability Status Scale (EDSS), MS Impact Scale (MSIS-29), and Clinical Global Impressions-Improvement Scale (CGI-I). Because this trial targets only a subset of RRMS patients experiencing relapses who have not responded acutely to corticosteroid therapy, the study may take a number of years to recruit.

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

About MS
MS is a neurologic disorder that affects the central nervous system (i.e., the brain and spinal cord).¹ Symptoms can include fatigue, balance/coordination issues, numbness or tingling, vision problems, muscle spasms, tremors and emotional changes. More than eight in 10 people with MS will experience a relapse, or flare-up, that brings new or worsening symptoms.²

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 H.P. Acthar Gel use is in these indications:

• As an orphan monotherapy medication for the treatment of 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 rheumatoid arthritis, to tide patients over an acute episode or exacerbation.
• Treatment of symptomatic sarcoidosis.
• Treatment of severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa such as: keratitis; iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis; anterior segment inflammation.

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 (CNS) 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 mask 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 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; and analgesics and hemostasis products. 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 and its Specialty Generics segment includes specialty generic drugs, active pharmaceutical ingredients and external manufacturing. To learn more about Mallinckrodt, visit www.mallinckrodt.com.

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CONTACTS
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Director, Investor Relations
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Meredith Fischer
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¹ Multiple Sclerosis Foundation. Symptoms of Multiple Sclerosis. Accessed April 19, 2016 at http://msfocus.org/Symptoms-of-Multiple-Sclerosis.aspx

² National Multiple Sclerosis Society. Relapsing-remitting MS (RRMS). Accessed April 19, 2016 at http://www.nationalmssociety.org/What-is-MS/Types-of-MS/Relapsing-remitting-MS

SOURCE Mallinckrodt Pharmaceuticals

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