CHESTERFIELD, United Kingdom, Nov. 21, 2016 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today confirmed that it will initiate a company-sponsored, Phase 2 proof-of-concept clinical trial to assess the efficacy of H.P. Acthar® Gel (repository corticotropin injection) in patients with Amyotropic Lateral Sclerosis (ALS, also known as Lou Gehrig's disease). The study is expected to begin in the first half of 2017.
Mallinckrodt also announced the U.S. Food and Drug Administration (FDA) has granted the company's request for a Fast Track designation for its Acthar Investigational New Drug (IND) application in patients with ALS. The company is working closely with the FDA as it establishes the Phase 2 study design.
"We recently completed our pilot study of Acthar in ALS patients, and are pleased to report that the primary objectives of the study were met. The trial explored the safety and tolerability of four potential Acthar dosing regimens in ALS patients," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. "Although the pilot study was not designed to generate controlled evidence regarding efficacy, we conducted a number of exploratory analyses, including a comparison of the course of disease observed in a small set of patients continuing on Acthar for 36 weeks versus a historical, placebo-treated population. Differences observed suggested further study was appropriate. The study data have also allowed us to select a dose regimen to study the drug further in a rigorous controlled trial. This next proof-of-concept study will evaluate the effects of Acthar on established measures of disease symptoms and progression, enabling us to assess the potential value Acthar may bring to patients with this devastating disease."
The FDA's Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. The Fast Track program addresses a broad range of serious conditions.
Mallinckrodt's investment into this clinical trial illustrates a tenet of its Acquire to Invest strategy, applying substantial resources on building a robust, organic pipeline.
About the Pilot Study
The 8-week, randomized, open-label evaluation examined the acute safety and tolerability of four different dosing regimens for Acthar in patients with ALS. The study included an optional 28-week, open-label extension phase to gather additional longer-term safety and tolerability data. For more information about the completed trial, click here to visit the ClinicalTrials.gov website.
About Amyotrophic Lateral Sclerosis
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their demise and when the motor neurons die, the ability of the brain to initiate voluntary movement and muscle control muscle movement is lost. With voluntary muscle action progressively affected, people may lose the ability to speak, eat, move and breathe.
There is increasing evidence that neuro-inflammation accompanies the death of motor neurons in ALS. Several places in the inflammatory events that appear to accompany ALS might be amenable to drug action that could help in the disease. Research in the field is continuing to find new approaches to implement an anti-inflammatory strategy in treating ALS1.
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:
- 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 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.
- Treatment of symptomatic sarcoidosis.
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.
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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1 The ALS Association, Our Research, Focus Areas, Disease Mechanisms. http://www.alsa.org/research/focus-areas/disease-mechanisms/ Accessed November 18, 2016.
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