STAINES-UPON-THAMES, United
Kingdom, Oct. 1, 2019
/PRNewswire/ -- Mallinckrodt
plc (NYSE: MNK), a global biopharmaceutical company, today
announced positive top-line findings from its observational
registry to assess treatment patterns and response to
Acthar® Gel (repository corticotropin injection) in
reducing the clinical impact of disease exacerbations in patients
with multiple sclerosis (MS) relapse. The results for the main
subsets of patients are highly statistically significant for all
the efficacy endpoints. Mallinckrodt
plans to share the data at an upcoming medical meeting.
MS is a chronic, degenerative disease that can cause numerous
impairments, including fatigue, balance/coordination issues,
numbness or tingling, vision problems, muscle spasms, tremors and
emotional changes, and some people with MS experience relapses
while on standard therapies1.
Acthar Gel is U.S. Food and Drug
Administration (FDA)-approved for the treatment of acute
exacerbations of MS in adults. Controlled clinical trials have
shown Acthar Gel to be effective in speeding the resolution of
acute exacerbations of MS. However, there is no evidence that it
affects the ultimate outcome or natural history of the disease.
Please see Important Safety Information for Acthar Gel below.
The registry was designed to assess the characteristics of
patients receiving Acthar Gel for MS exacerbations (sometimes
referred to as "flares" or "relapses"), as well as the outcome at
two months and whether or not the outcome was maintained at six
months. The average age of patients was 47 years, and the mean
duration of MS was 10 years at the start of the study. Registry
data showed that patients treated with Acthar Gel reported
significant improvements in symptoms associated with MS
exacerbations at two months from baseline, as measured by the MS
Impact Scale, version 1 (MSIS 29v1), the primary measure of the
study. The response was sustained at six months. Physicians also
reported an improvement in physical symptoms in patients treated
with Acthar Gel, based on Expanded Disability Status Scale (EDSS)
scores.
"Mallinckrodt is committed to
generating data that helps clarify to the prescriber those patients
appropriate for treatment with Acthar Gel to manage MS relapses,"
said Steven Romano, M.D., Executive
Vice President and Chief Scientific Officer at Mallinckrodt. "The registry evaluated the effect of
Acthar Gel in prospectively defined patients who were treated with
Acthar Gel following an acute relapse. In addition, we look forward
to Mallinckrodt's ongoing, randomized,
double-blind, placebo-controlled OPTIONS study, which we anticipate
will provide data on some of the more difficult-to-treat MS
patients in the future."
About the Observational Registry of Acthar Gel for Multiple
Sclerosis Relapse
- The Observational Registry of Acthar Gel for Multiple
Sclerosis Relapse followed patients with recurrent MS relapse
for up to 24 months, and a minimum of six months, to characterize
the patient population with recurrent MS exacerbations (relapse)
and describe MS exacerbation recovery, treatment patterns and
safety outcomes in adult patients treated with Acthar Gel in
standard practice in the United
States.
- The registry enrolled 148 adult (≥18 years) patients at 55 U.S.
sites.
- The average age of enrolled patients was 47 years.
- 60 percent of patients reported a history of insufficient
treatment response to, intolerance of, or intravenous access
problems with high-dose corticosteroid therapy.
- 58 percent reported one or more MS relapses in the two years
before enrolling in the registry.
- The primary measure of the registry was MS relapse at month two
versus baseline as measured by MSIS-29v1 – physical subscale.
- Adverse events were consistent with those in previous trials of
Acthar Gel.
- An electronic patient-reported outcome scale was used to
describe MS exacerbation recovery (e.g., neurological functional
and symptom improvement; productivity loss) and treatment response
over a period of six months following index exacerbation and any
relapses, as well as to describe the safety profile of Acthar Gel.
Patient assessments were used to describe relapse
characteristics
(e.g., symptoms, time to relapse and relapse treatment) for
patients who experienced relapses following the index exacerbation
during the study period.
- Acute exacerbations were determined by the treating
clinician.
The MSIS-29v1 is a scientifically rigorous, disease-specific,
health-related, quality of life instrument that measures the
patient's perspective on disease impact. It consists of two
subscales assessing the physical and psychological impacts of
MS.2
The EDSS is a clinician-administered assessment scale that
measures neurological impairment. It is used to describe and
monitor disease progression and is widely used to measure disease
outcomes in clinical trials.3
A limitation of the study is that, as it was a registry, there
was no control group.
The study and analysis were conducted by Mallinckrodt. Additional information about the
study can be found here.
About Multiple Sclerosis
MS is a neurologic disorder
that affects the central nervous system (i.e., the brain and spinal
cord).4 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.1
Acthar Gel (repository corticotropin
injection) Indications
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:
- Adjunctive therapy for short-term administration (to tide the
patient over an acute episode or exacerbation) in rheumatoid
arthritis, including juvenile rheumatoid arthritis (selected cases
may require low-dose maintenance therapy)
- The treatment of symptomatic sarcoidosis
- Monotherapy for the treatment of infantile spasms in infants
and children under 2 years of age
- Treatment during an exacerbation or as maintenance therapy in
selected cases of systemic lupus erythematosus
- The treatment of acute exacerbations of multiple sclerosis in
adults. Controlled clinical trials have shown Acthar Gel to be
effective in speeding the resolution of acute exacerbations of
multiple sclerosis. However, there is no evidence that it affects
the ultimate outcome or natural history of the disease
- Inducing a diuresis or a remission of proteinuria in nephrotic
syndrome without uremia of the idiopathic type or that due to lupus
erythematosus
- Treatment during an exacerbation or as maintenance therapy in
selected cases of systemic dermatomyositis (polymyositis)
- 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
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-axis (HPA) 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
- Acthar can cause elevation of blood pressure, salt and water
retention, and hypokalemia. Blood pressure, sodium and potassium
levels may need to be monitored
- Acthar often acts by masking symptoms of other
diseases/disorders. Monitor patients carefully during and for a
period following discontinuation of therapy
- Acthar can cause GI bleeding and gastric ulcer. There is also
an increased risk for perforation in patients with certain
gastrointestinal disorders. Monitor for signs of bleeding
- Acthar may be associated with central nervous system effects
ranging from euphoria, insomnia, irritability, mood swings,
personality changes, and severe depression, and psychosis. Existing
conditions may be aggravated
- Patients with comorbid disease may have that disease worsened.
Caution should be used when prescribing Acthar in patients with
diabetes and myasthenia gravis
- Prolonged use of Acthar may produce cataracts, glaucoma and
secondary ocular infections. Monitor for signs and symptoms
- Acthar is immunogenic and prolonged administration of Acthar
may increase the risk of hypersensitivity reactions. Neutralizing
antibodies with chronic administration may lead to loss of
endogenous ACTH activity
- There is an enhanced effect in patients with hypothyroidism and
in those with cirrhosis of the liver
- Long-term use may have negative effects on growth and physical
development in children. Monitor pediatric patients
- Decrease in bone density may occur. Bone density should be
monitored for patients on 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 for Acthar are similar to those of
corticosteroids and 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
Other adverse events reported are included in the full
Prescribing Information.
Please see full Prescribing Information.
ABOUT MALLINCKRODT
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's
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 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 includes forward-looking statements
concerning Acthar Gel including expectations regarding related
clinical trials, its potential impact on patients and anticipated
benefits associated with its use. 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; 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.
CONTACTS
For Trade Media
Inquiries
Caren Begun
Green Room Communications
201-396-8551
caren@greenroompr.com
For Financial/Dailies Media Inquiries
Daniel Yunger
Kekst CNC
212-521-4879
mallinckrodt@kekstcnc.com
Investor Relations
Daniel J.
Speciale, CPA
Vice President, Investor Relations and IRO
314-654-3638
daniel.speciale@mnk.com
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.
©2019 Mallinckrodt. US-1901601 9/19
References
1 National Multiple Sclerosis Society.
Relapsing-remitting MS (RRMS). Available at:
http://www.nationalmssociety.org/What-is-MS/Types-of-MS/Relapsing-remitting-MS.
Accessed September 10, 2019.
2 Hobart J, Lamping D, Fitzpatrick R, Riazi A,
Thompson A. The multiple sclerosis impact scale (MSIS-29) -- A new
patient-based outcome measure. Brain 2001, 124: 962–973.
3 Kurtzke J: Rating neurologic impairment in
multiple sclerosis: an expanded disability status scale (EDSS).
Neurology 1983, 33: 1444–1452.
4 Willis, BMJ Best Practice Multiple Sclerosis.
October 2018. p. 4.
View original content to download
multimedia:http://www.prnewswire.com/news-releases/mallinckrodt-announces-positive-top-line-findings-from-its-observational-registry-assessing-relapse-recovery-in-multiple-sclerosis-relapse-patients-treated-with-acthar-gel-repository-corticotropin-injection-300928259.html
SOURCE Mallinckrodt plc