Mallinckrodt Highlights First Patients in Company-Sponsored Prospective Observational Registry on the Use of H.P. Acthar® Gel (Repository Corticotropin Injection) Following Acute Multiple Sclerosis Relapse

STAINES-UPON-THAMES, United Kingdom, May 25, 2017 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, highlights the first patients enrolled in an ongoing company-sponsored study on the use of H.P. Acthar^® Gel (repository corticotropin injection) following acute multiple sclerosis (MS) relapse. The prospective observational registry will contribute to the understanding of the type of MS patient receiving H.P. Acthar Gel for a relapse and the impact of this treatment. The study will also assess patient reported outcomes from patients who receive H.P. Acthar Gel regarding treatment patterns, MS relapse recovery and safety outcomes. Preliminary demographic data will be presented on Thursday, May 25 at the 2017 Annual Meeting of the Consortium of Multiple Sclerosis Centers (CMSC) in New Orleans, La.

"Despite advances in the management of MS, many patients still experience relapse, and an incomplete recovery from relapse may contribute to continued accrual of disability for diagnosed patients^1,2,3," said Tunde Otulana, Chief Medical Officer at Mallinckrodt. "Mallinckrodt is committed to conducting studies in MS relapse patients treated with Acthar to collect valuable information about their treatment patterns, MS relapse recovery and safety outcomes."

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

A Prospective Observational Registry of H.P. Acthar Gel for the Treatment of Multiple Sclerosis Relapse is enrolling patients and collecting demographic data to characterize the patient population who receive H.P. Acthar Gel to treat acute MS relapse. The study will describe treatment patterns, MS relapse recovery and safety outcomes. The goal is to enroll 260 patients at up to 60 study sites over 24 months. Patients who have experienced a relapse are clinically evaluated with the MS Impact Scale, version 1 (MSIS-29v1), Expanded Disability Status Scale (EDSS), Functional System Score, and Clinical Global Impression of Improvement scale at baseline, two months, and six months following their clinical episode. Patients are also evaluated with health outcomes assessments, including the healthcare resource utilization and Work Productivity and Activity Impairment questionnaires.

Preliminary data on 45 patients (enrolled as of October 27, 2016) found:

• Patients are female (69%), Caucasian (64%), and on average approximately 50 years old⁴ with a baseline of EDSS of 4.4⁵ and MSIS-29v1 physical subscale score of 65.4⁶ following the index relapse.
• At least 71% of patients are receiving a disease-modifying therapy at time of relapse.
• Median number of doses of H.P. Acthar Gel received is five (equivalent to one multi-dose vial), with a median dose strength of 80 U.
• Three serious adverse events -- MS relapse, urinary tract infection, and asthenia (weakness and energy loss) -- have been reported. These adverse events were judged by the investigators to be unlikely related or not related to H.P. Acthar Gel, and all patients recovered. Thus far, adverse events are consistent with the known adverse event profile for the drug.

Find more information about the observational registry here on the ClinicalTrials.gov website.

The poster is available on the Mallinckrodt website.

ABOUT MULTIPLE SCLEROSIS (MS)
MS is a neurologic disorder that affects the central nervous system (i.e., the brain and spinal cord).⁷ 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.⁸  

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:

• As an orphan monotherapy medication for the treatment of infantile spasms 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 MS 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. The 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. Adrenal insufficiency may be minimized by tapering of the dose when discontinuing treatment. During recovery of the adrenal gland, patients should be protected from the stress (e.g., trauma or surgery) by the use of corticosteroids. Monitor patients for effects of HPA suppression after stopping treatment.
• 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.

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.

CONTACTS

Investor Relations
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Senior Vice President, Investor Strategy and IRO 
314-654-6649 
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Director, Investor Relations 
314-654-3638 
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Senior Communications Manager 
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Meredith Fischer 
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¹ Confavreux C, Vukusic S, Moreau T, Adeleine P. Relapses and progression of disability in multiple sclerosis. New England Journal of Medicine, 2000, Nov 16;343(20):1430-8. Available at: https://www.ncbi.nlm.nih.gov/pubmed/11078767. Accessed May 23, 2017.  
² Confavreux C, Vukusic S, Adeleine P. Early clinical predictors and progression of irreversible disability in multiple sclerosis: an amnesic process. Brain. 2003 Apr; 126(Pt 4):770-82. Available at: https://www.ncbi.nlm.nih.gov/pubmed/12615637. Accessed May 23, 2017.
³ Hirst C, Ingram G, Pearson O, Pickersgill T, Scolding N, Robertson N. Contribution of relapses to disability in multiple sclerosis. Journal of Neurology. 2008 Feb;255(2):280-7.
⁴ Data available for 36 patients.
⁵ Data available for 27 patients, rated on a scale from 0 (normal neurologic exam) to 10 (death due to MS).
⁶ Data available for 35 patients, scored on a scale of 0 to 100, with 100 representing worst possible score.
⁷ Multiple Sclerosis Foundation. Symptoms of Multiple Sclerosis. Available at: http://msfocus.org/Symptoms-of-Multiple-Sclerosis.aspx. Accessed April 19, 2017.
⁸ National Multiple Sclerosis Society. Relapsing-remitting MS (RRMS). Available at: http://www.nationalmssociety.org/What-is-MS/Types-of-MS/Relapsing-remitting-MS. Accessed April 21, 2017.

SOURCE Mallinckrodt plc

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