STAINES-UPON-THAMES, United
Kingdom, June 14,
2017 /PRNewswire/ -- Mallinckrodt
plc (NYSE: MNK), a leading global specialty
pharmaceutical company, today confirmed enrollment of the first
patient in the company's Phase 2B study designed to assess the
efficacy and safety of H.P. Acthar® Gel (repository
corticotropin injection) as a treatment for amyotrophic lateral
sclerosis (ALS).
The U.S. Food and Drug Administration (FDA) previously
granted the company's request for a Fast Track designation and
orphan status for its H.P. Acthar Gel Investigational New Drug
application in patients with ALS. The drug is not FDA-approved for
the ALS indication.
"ALS is a rare and incurable disorder that impacts
patients from all walks of life," said Todd Levine, M.D., founder and director of the
Phoenix Neurological ALS Clinic and adjunct Professor of Neurology
at Kansas University. "The
community welcomes new research aimed at further understanding the
disease and potential new treatments for ALS."
"We are pleased to announce the first patient in this
important study of Acthar in ALS patients," said Steven Romano, M.D., Chief Scientific Officer
and Executive Vice President at Mallinckrodt. "This multi-center, double blind,
placebo-controlled trial will evaluate the effects of the drug on
established measures of disease symptoms and progression, enabling
us to assess the potential value Acthar may bring to patients with
this devastating disease."
About the PENNANT Trial
The Phase
2B clinical study is titled "A Multicenter, Double Blind,
Placebo-Controlled Study to Assess the Efficacy and Safety of H.P.
Acthar Gel in the Treatment of Subjects with Amyotrophic Lateral
Sclerosis." The study will enroll patients ages 18 to 75 with ALS
and symptom onset (defined as first muscle weakness or dysarthria)
≤ two years prior to the screening visit. Subjects will be
randomized on a 2:1 basis to receive subcutaneous (SC) H.P. Acthar
Gel 0.2 mL (16 units) daily or SC matching placebo 0.2 mL daily for
36 weeks.
The efficacy of H.P. Acthar Gel will be assessed using
standard measures of functional decline, including change from
baseline in the ALS Functional Rating Scale-Revised, assessed after
36 weeks of therapy. Approximately 195 patients will be enrolled
across multiple sites.
Find more information about the Pennant trial
here on the ClinicalTrials.gov
website.
About ALS
ALS is a progressive
neurodegenerative disease that affects motor neuron cells in the
brain and the spinal cord. Motor neurons reach from the brain and
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, voluntary and involuntary
muscle movement is lost. With the progressive loss of motor
neurons, people with ALS 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
inflammatory events that appear to accompany disease progression in
ALS might be amenable to pharmacologic interventions as a component
of disease management, and research in the field is investigating
new approaches to implement an anti-inflammatory strategy for
treating
ALS1.
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
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
psoriatic arthritis; rheumatoid arthritis, juvenile rheumatoid
arthritis and ankylosing spondylitis 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. Please click to see
full Prescribing Information and
Medication Guide.
Important Safety Information
Contraindications
- 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.
Warnings and Precautions
- 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.
Adverse Reactions
- 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
Coleman N. Lannum,
CFA
Senior Vice President, Investor Strategy and
IRO
314-654-6649
cole.lannum@mallinckrodt.com
Daniel J. Speciale,
CPA
Director, Investor
Relations
314-654-3638
daniel.speciale@mallinckrodt.com
Media
Rhonda
Sciarra
Senior Communications
Manager
908-238-6765
rhonda.sciarra@mallinckrodt.com
Meredith
Fischer
Chief Public Affairs
Officer
314-654-3318
meredith.fischer@mallinckrodt.com
1
The ALS Association, Our Research, Focus Areas, Disease
Mechanisms.
http://www.alsa.org/research/focus-areas/disease-mechanisms/
Accessed May 22,
2017.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/mallinckrodt-enrolls-first-patient-in-phase-2b-trial-of-hp-acthar-gel-repository-corticotropin-injection-for-amyotrophic-lateral-sclerosis-als-300474152.html
SOURCE Mallinckrodt plc