– Clinical findings from historical manuscript published in Clinical Drug Investigation reinforce the efficacy, safety profile, and use of Acthar Gel in appropriate patients across 12 autoimmune and inflammatory indications1 –
– Economic findings from manuscript published in ClinicoEconomics and Outcomes Research support Acthar Gel's clinical and economic benefits for patients across nine autoimmune and inflammatory indications2 –
DUBLIN, Oct. 10, 2023 /PRNewswire/ -- Mallinckrodt plc (OTCMKTS: MNKTQ), a global specialty pharmaceutical company, today announced the publication of two journal manuscripts reinforcing the clinical and economic evidence supporting the efficacy, safety profile, continued use, and cost-effectiveness of Acthar Gel for appropriate patients across a range of FDA-approved autoimmune and inflammatory disease indications.1,2
Mallinckrodt's clinical manuscript titled "Acthar Gel Treatment for Patients with Autoimmune and Inflammatory Diseases: A Historical Perspective and Characterization of Clinical Evidence" was published on October 4, 2023 in the journal, Clinical Drug Investigation. Mallinckrodt's health economics manuscript titled "Acthar Gel (RCI): A Narrative Literature Review of Clinical and Economic Evidence" was published on June 24, 2023 in ClinicoEconomics and Outcomes Research.
Acthar is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides. Acthar Gel is approved by the U.S. Food and Drug Administration (FDA) for the treatment of several autoimmune disorders and medical conditions known to cause inflammation.3
Please see indications and Important Safety Information below.
Authored by Jeffrey Kaplan, M.D., Kansas City Multiple Sclerosis & Headache Center, and colleagues, "Acthar Gel Treatment for Patients with Autoimmune and Inflammatory Diseases: A Historical Perspective and Characterization of Clinical Evidence," sought to review the history of Acthar and the findings of existing literature on preclinical and clinical studies that have investigated the mechanism of action, safety profile, efficacy, and real-world effectiveness of Acthar. The indications addressed in the study include the treatment of inflammatory diseases such as rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), dermatomyositis and polymyositis (DM/PM), multiple sclerosis (MS) relapse, inflammatory ocular diseases, sarcoidosis, and nephrotic syndrome (NS).1
"Acthar® Gel has an established efficacy and safety profile supported by robust clinical research and experience," said Dr. Kaplan. "For patients and providers managing the challenges associated with chronic inflammatory and autoimmune conditions, the findings of this study further elucidate Acthar's proposed mechanism of action and reinforce the extensive history of clinical evidence that has shown its potential to improve health and treatment outcomes across a range of FDA-approved indications."1
This publication is accompanied by an Audiocast.
All six authors across specialties representing key Acthar Gel indications researched in this study discuss a brief overview of the manuscript, including the history of Acthar, its mechanism of action (MOA), and clinical characteristics, in addition to introductions on each author and reflections on their own personal experiences with Acthar in clinical practice.
Authored by George J. Wan, Ph.D., M.P.H., Vice President, Evidence Generation and Data Sciences at Mallinckrodt, and colleagues, "Acthar Gel (RCI): A Narrative Literature Review of Clinical and Economic Evidence" reviewed key studies of clinical efficacy and healthcare resource utilization and cost from 1956 to 2022 to summarize the key clinical and economic findings among nine FDA-approved Acthar Gel indications: infantile spasms (IS), MS relapse, RA, SLE, DM/PM, ocular inflammatory diseases (primary uveitis and severe keratitis), symptomatic sarcoidosis, and proteinuria in NS.2
"The findings of this health-economics analysis deepen our understanding of Acthar's ability to serve as a cost-effective, value-based treatment option across multiple FDA-approved indications studied – including how appropriate use of Acthar may be associated with reduction of healthcare utilization costs such as total hospitalizations, patient length of stay, and concurrent treatment burden, such as the use of corticosteroids,"2 said Wan.
Data collected in a retrospective analysis may have errors or omissions. Outcomes may be influenced by therapies not evaluated in the study and the clinical outcomes may not be solely attributable to Acthar.
These studies were funded by Mallinckrodt Pharmaceuticals.
ABOUT DERMATOMYOSITIS/POLYMYOSITIS (DM/PM)
DM/PM are rare inflammatory diseases that cause progressive muscle weakness,4 usually in the muscles closest to the trunk of the body.5 For instance, muscle weakness associated with PM involves those in the hips, thighs, shoulders, upper arms, and neck.6 DM also causes skin rashes.7 People of all ages can be affected, though it usually occurs between the ages of 40-60 and is more common in women.7,8
ABOUT INFANTILE SPASMS (IS)
IS is a rare seizure disorder that affects approximately 2,500 children in the U.S. every year.9 It most commonly occurs between three and seven months of age.9 Sometimes called West syndrome, IS demands early identification, diagnosis, and treatment to help limit lasting effects.9 Children with IS generally have one or more of the following symptoms: a certain type of seizure (called "spasms"), a disorganized and chaotic brain-wave pattern called hypsarrhythmia as recorded on an EEG (electroencephalogram).10
ABOUT MULTIPLE SCLEROSIS (MS)
MS is a chronic neurologic disorder that affects the central nervous system (i.e., the brain and spinal cord).11 Symptoms can include fatigue, mobility issues, numbness or tingling, vision problems, muscle spasticity, tremors, and cognitive changes.12,13 More than eight in 10 people with MS will experience a relapse, or flare-up, that brings new or worsening symptoms.12
ABOUT PROTEINURIA IN NEPHROTIC SYNDROME (NS)
NS is a collection of symptoms that occur when the blood vessels in the kidney begin to leak excess protein in the urine, a condition called proteinuria.14 A variety of diseases and underlying disorders damage the kidneys and cause proteinuria in people with NS.14 These etiologies can include glomerular diseases such as: idiopathic membranous nephropathy, focal segmental glomerulosclerosis, minimal change disease, membranoproliferative glomerulonephritis, lupus nephritis, and IgA nephropathy.15,16,17,18 In these and other related disorders, the glomeruli, or small blood vessels that work as the kidney's filtering system, are damaged.14
Proteinuria is one of the most important adverse prognostic factors for progression to end stage renal failure in patients with glomerular disease. One of the goals of treating NS includes reducing or eliminating proteinuria.19
ABOUT RHEUMATOID ARTHRITIS (RA)
RA is an autoimmune disease.20 It is a chronic condition that causes pain, stiffness, and swelling of the joints – all symptoms caused by inflammation.20 An estimated 1.5 million U.S. adults are living with RA.20
ABOUT SYSTEMIC LUPUS ERYTHEMATOSUS (SLE)
SLE is an autoimmune disease in which the immune system produces antibodies to cells within the body leading to possible inflammation and tissue damage.21 It is the most common form of lupus,21 a condition that impacts an estimated 1.5 million Americans.22 Far more of those diagnosed with lupus are women, often between the ages of 15-44.21 Lupus is characterized by periods of illness "flares" and remissions and the disease can affect the joints, skin, brain, lungs, kidneys, and blood vessels.21 Symptoms may include fatigue, pain or swelling in joints, skin rashes, and fevers.21
ABOUT SYMPTOMATIC SARCOIDOSIS
Sarcoidosis is a challenging and rare multisystem disease.23 In some cases, the symptoms may come and go throughout a lifetime.23 This is referred to as symptomatic sarcoidosis.23 In people with sarcoidosis the immune system overreacts, forming clumps of cells called granulomas that result in inflammation to the body's tissues.24 The disease can impact any organ, but it most often impacts the lungs, lymph nodes, eyes, and skin.25 Nearly 90 percent of people with sarcoidosis will suffer lung problems.25 Concomitant involvement of organs outside of the lungs is common, occurring in more than half of all sarcoidosis cases, according to one study.26
Uveitis is a mix of acute or chronic inflammatory eye disorders that affects the uveal tract (a layer of tissue just behind the outer layer of the eye) and adjacent structures, including the sclera, cornea, vitreous humor, retina, and optic nerve head.27,28,29,30 Symptoms vary based on the site of inflammation, but can include decreased vision, eye pain, tearing, redness, light sensitivity, and floaters.27,28 Uveitis has numerous etiologies, associated conditions, and underlying immune responses that complicate its diagnosis and treatment.28,31 Uveitis accounts for up to 10% of cases of legal blindness in the United States. In the United States, the annual incidence (occurrence of new cases) of uveitis ranges between 17.4 and 52.4 per 100,000 person-years.32,33,34 It most affects individuals of working age.28
ABOUT SEVERE KERATITIS
Keratitis is a painful inflammation of the cornea which can result in partial or total loss of vision if left untreated and is a significant cause of ocular morbidity around the world.35 It can result from infectious agents (e.g., microbes including bacteria, fungi, amebae, and viruses) or from non-infectious causes (e.g., eye trauma, chemical exposure, and ultraviolet exposure).36 Conditions that affect the integrity of the ocular surface epithelium (exposure keratitis, neurotrophic keratitis, keratomalacia, recurrent corneal erosions) may also lead to development of sterile corneal ulcers.37 Non-infectious corneal ulcers may be associated with various collagen vascular or other autoimmune diseases, sometimes as the presenting sign of the disease.37
Acthar Gel is indicated for:
IMPORTANT SAFETY INFORMATION
Acthar is contraindicated:
Warnings and Precautions
Please see full Prescribing Information for additional Important Safety Information.
Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company's Specialty Brands reportable segment areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, hepatology, nephrology, pulmonology, ophthalmology, and oncology; immunotherapy and neonatal respiratory critical care therapies; analgesics; cultured skin substitutes and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. 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 contains forward-looking statements, including with regard to Acthar® Gel, its potential to improve health and treatment outcomes, its ability to reduce healthcare utilization costs, and 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 or adverse side effects or adverse reactions associated with Acthar; pricing pressure on Mallinckrodt's products due to legal changes or changes in insurers' reimbursement practices from recent increased public scrutiny of healthcare and pharmaceutical costs; the reimbursement practices of governmental health administration authorities, private health coverage insurers and other third-party payers; 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.
Green Room Communications
Daniel J. Speciale
Senior Vice President, Finance and Chief Financial Officer, Specialty Generics
Vice President, Investor Relations
Mallinckrodt, the "M" brand mark and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners.
©2023 Mallinckrodt. US-2300691 9/23
1 Data on File. Ref-06841. Mallinckrodt Pharmaceuticals.
2 Wan GJ., Niewoehner J., Hayes K. Acthar Gel (RCI): A Narrative Literature Review of Clinical and Economic Evidence. ClinicoEconomics and Outcomes Research. 2023; 15:499-512. https://doi.org/10.2147/CEOR.S410082
3 Acthar® Gel (repository corticotropin injection) [prescribing information]. Mallinckrodt ARD LLC.
4 Types of Myositis. The Myositis Association. Available at: http://www.myositis.org./learn-about-myositis/types-of-myositis. Accessed August 2023.
5 Medline Plus. Myositis. Available at: https://medlineplus.gov/myositis.html. Accessed August 2023.
6 Mayo Clinic. Polymyositis. Available at: http://www.mayoclinic.org/diseases-conditions/polymyositis/basics/symptoms/con-20020710. Accessed August 2023.
7 Rheumatology Center. About Dermatomyositis and Polymyositis. Available at: https://www.actharrheumatology.com/dermatomyositis-and-polymyositis/about-dermatomyositis-and-polymyositis. Accessed August 2023.
8 Bernatsky S, et al. Estimating the prevalence of polymyositis and dermatomyositis from administrative data: age, sex and regional differences. Ann Rheum Dis. 2008;68:1192-1196.
9 Wheless J, Gibson P, Rosbeck K, et al. Infantile spasms (West syndrome): update and resources for pediatricians and providers to share with parents. BMC Pediatrics. 2012;12(1):108. doi:10.1186/1471-2431-12-108.
10 Go CY, Mackay MT, Weiss SK, et al. Evidence-based guideline update: medical treatment of infantile spasms. Neurology. 2012; 78:1974-1980.
11 Multiple Sclerosis Foundation. Get Educated-Common Questions-Multiple Sclerosis. Available at: https://www.acthar.com/Static/pdf/IS_Caregiver_Brochure_English.pdf. Accessed August 2023.
12 National Multiple Sclerosis Society. Relapsing-remitting MS (RRMS). Available at: http://www.nationalmssociety.org/What-is-MS/Types-of-MS/Relapsing-remitting-MS. Accessed August 2023.
13 National Multiple Sclerosis Society. MS Symptoms & Signs of MS. Available at: https://www.nationalmssociety.org/Symptoms-Diagnosis/MS-Symptoms. Accessed September 2023.
14 National Institute of Health. National Institute of Diabetes and Digestive Kidney Diseases. Nephrotic Syndrome in Adults. 2020. Available at: https://www.niddk.nih.gov/health-information/kidney-disease/nephrotic-syndrome-adults. Accessed September 2023.
15 NephCure Kidney International. Nephrotic Syndrome. Available at https://nephcure.org/livingwithkidneydisease/understanding-golmerular-disease/understanding-nephrotic-syndrome/. Accessed September 2023
16 NephCure Kidney International. Focal Segmentation Glomerulosclerosis. Available at https://nephcure.app.box.com/s/rc98yosh958s00ea0g44d1pul8iesz9a/. Accessed September 2023.
17 NIDDK. IgA Nephropathy. Available at: https://www.niddk.nih.gov/health-information/kidney-disease/iga-nephropathy/. Accessed September 2023.
18 Satpathy HK. IgA Nephropathy. In: Ferri FF, ed. Ferri's Clinical Advisor 2013. 1st ed. St Louis: Mosby; 2012; 570-571.
19 Troyanov et al. J Am Soc Nephrol. 2005;16(4):1061–1068.
20 What is Rheumatoid Arthritis? Arthritis Foundation. Available at: http://www.arthritis.org/about-arthritis/types/rheumatoid-arthritis/what-is-rheumatoid-arthritis.php. Accessed August 2023.
21 Lupus Detailed Fact Sheet. 2018. The Centers for Disease Control and Prevention. Available at: https://www.cdc.gov/lupus/facts/detailed.html#previnc. Accessed September 2023.
22 Lupus Foundation of America. National Resource Center on Lupus. Lupus Fact and Statistics. 2018. Available at: https://resources.lupus.org/entry/facts-and-statistics. Accessed September 2023.
23 Sarcoidosis. American College of Chest Physicians. Available at: http://chestnet.org/sarcoid. Accessed September 2023.
24 Sarcoidosis Overview. 2019. Cleveland Clinic. Available at: https://my.clevelandclinic.org/health/diseaes/11863-sarcoidosis-overview. Accessed September 2023.
25 Judson MA, Boan AD, Lackland DT. The Clinical Course of Sarcoidosis: Presentation, Diagnosis, and Treatment in a Large White and Black Cohort in the United States. Sarcoidosis Vasc Diffuse Lung Dis. 2012. 29(2);119-127.
26 Chopra I, et al. Repository Corticotropin Injection in Patients with Advanced Symptomatic Sarcoidosis: Retrospective Analysis of Medical Records. Ther Adv Res Dis. 2019(13);1-11. DOI: 10.1177/1753466619888127.
27 Barisani-Asenbauer T, Maca SM, Mejdoubi L, Emminger W, Machold K, Auer H. Uveitis—a rare disease often associated with systemic diseases and infections—a systematic review of 2619 patients. Orphanet J Rare Dis. 2012;7:57.
28 Mustafa M, Muthusamy P, Hussain SS, Shimmi SC, Sein MM. Uveitis: pathogenesis, clinical presentations and treatment. IOSR J Pharm. 2014;4:42-47.
29 Guly CM, Forrester JV. Investigation and management of uveitis. BMJ. 2010;341:c4976.
30 Chan CC, Li Q. Immunopathology of uveitis. Br J Ophthalmol. 1998;82:91-99.
31 Lee RWJ, Dick AD. Current concepts and future directions in the pathogenesis and treatment of non-infectious intraocular inflammation. Eye. 2012;26(1):17-28. doi:10.1038/eye.2011.255.
32 Acharya, NR, Tham VM, Esterberg E, et al. Incidence and prevalence of uveitis: results from the Pacific Ocular Inflammation Study. JAMA Ophthalmol. 2013;131:1405-1412.
33 Gritz DC, Wong IG. Incidence and prevalence of uveitis in Northern California; the Northern California Epidemiology of Uveitis Study. Ophthalmology. 2004;111:491-500; discussion 500.
34 Suhler EB, Lloyd MJ, Choi D, Rosenbaum JT, Austin DF Incidence and prevalence of uveitis in Veterans Affairs Medical Centers of the Pacific Northwest. Am. J. Ophthalmol. 2008;146:890.e8–896.e8
35 Sharma S. Keratitis. Bioscience Reports. 2001;21:419-444.
36 Collier SA, Gronostaj MP, MacGurn, AK, Cope JR, Awsumb KL, Yoder JS, et al. Estimated burden of keratitis--United States, 2010. MMWR Morb Mortal Wkly Rep. 2014;63(45):1027–1030.
37 Donzis PB, Mondino BJ. Management of noninfectious corneal ulcers. Surv Ophthalmol. 1987;32:94–110.
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