|[December 17, 2013]
Ipsen Announces Positive Initial Results from the International Double-Blind Clinical Phase III Study of Dysport® in the Treatment of Adults Suffering from Upper Limb Spasticity
PARIS --(Business Wire)--
Ipsen (Paris:IPN)(Euronext: IPN; ADR: IPSEY) today announced positive
initial results from the double-blind phase III study of Dysport®
(abobotulinumtoxinA) in Adult Upper Limb spasticity. Regarding the
primary endpoints, treatment with Dysport® showed
statistically significant response versus placebo in the improvement of
muscle tone, as measured by the Modified Ashworth Scale (MAS). In
addition, a statistically significant clinical benefit for the patients
treated with Dysport® was demonstrated versus placebo, as
measured by the Physician Global Assessment (PGA). The safety profile
observed in the study was consistent with the known safety profile of
Dysport® in this indication. Comprehensive results from this
double-blind study will be disclosed in the next few months at major
Marc de Garidel, Chairman and CEO of Ipsen stated: "These
clinical results are an important step in our ambition to become a
global leader in the treatment of targeted debilitating diseases.
Expanding our scope of indications for Dysport®
would help us further develop the neurology franchise, notably in the
Claude Bertrand, Executive Vice-President Research & Development and
Chief Scientific Officer of Ipsen commented: "Ipsen is very
pleased with the initial results of the Dysport® study
in Adult Upper Limb spasticity. We believe it should meet the
expectations of physicians by potentially providing a new alternative
for treating patients suffering from this debilitating condition. We are
grateful to the clinicians, caregivers, patients and their families who
were involved in this study."
About the study
This phase III research study included 243 patients and was
multicentric, prospective, double blind, randomised, and
placebo-controlled. It was conducted in the USA, France, Italy, Belgium,
Czech Republic, Poland, Slovakia, Russia and Hungary.
The purpose of this study was to assess the efficacy of Dysport®
compared to placebo in improving upper limb spasticity in hemiparetic
patients following a stroke or a brain trauma. The study co-primary
endpoints were the improvement of muscle tone in the treated upper limb
measured by the Modified Ashworth Scale (MAS) and the clinical benefit
for the patients assessed by the Physician Global Assessment (PGA).
Patients were offered to continue in an open label long-term study
wherein they will be receiving additional Dysport® treatment
cycles within a total of 15 months.
About the Modified Ashworth Scale (MAS)
The MAS is the reference clinical scale to assess muscle tone in
clinical trials in patients with spasticity. It allows categorizing the
severity of spasticity by evaluating resistance to passive movement. It
ranges from 0 (=no increase in tone) to 4 (=affected limb rigid in
flexion or extension).
About the Physician Global Assessment (PGA)
The PGA is a well-known tool used by clinicians to assess the overall
clinical benefit for their patients. It is a 9-point scale that ranges
from -4 (markedly worse) to +4 (markedly better).
Dysport® is an injectable form of botulinum toxin type A
(BTX-A), which is isolated and purified from Clostridium BTX-A bacteria.
It is supplied as a lyophilized powder.
Dysport® was first registered for the treatment of
blepharospasm and hemifacial spasm in the United Kingdom in 1990, and is
licensed in more than 75 countries for various indications including:
blepharospasm, adult upper and lower limb spasticity, hemifacial spasm,
spasmodic torticollis (ST) (previously referred to as cervical
dystonia), pediatric lower limb spasticity due to cerebral palsy (CP),
axillary hyperhidrosis, and glabellar lines.
Dysport® is approved for the treatment of upper limb
spasticity in many international markets, but not in the United States
(US). Dysport®'s only approved therapeutic indication in the
United States (US) is for the treatment of adults with cervical dystonia
(referred to spasmodic torticollis in other markets). As such, data from
this study in adults with upper limb spasticity are with respect to an
investigational use of Dysport® in the USA.
Important Safety Information about Dysport®
Postmarketing reports indicate that the effects of Dysport®
and all botulinum toxin products may spread from the area of injection
to produce symptoms consistent with botulinum toxin effects. These may
include asthenia, generalized muscle weakness, diplopia, blurred vision,
ptosis, dysphagia, dysphonia, dysarthria, urinary incontinence, and
breathing difficulties. These symptoms have been reported hours to weeks
after injection. Swallowing and breathing difficulties can be life
threatening, and there have been reports of death. The risk of symptoms
is probably greatest in children treated for spasticity, but symptoms
can also occur in adults treated for spasticity and other conditions,
particularly in those patients who have underlying conditions that would
predispose them to these symptoms. In unapproved uses, including
spasticity in children and adults, and in approved indications, cases of
spread of effect have been reported at doses comparable to those used to
treat cervical dystonia and at lower doses.
Dysport® is contraindicated in patients with hypersensitivity
to any botulinum toxin product or its excipients, including human
albumin, lactose, and cow's milk protein, or who have an infection at
the proposed injection site.
Lack of interchangeability between botulinum toxin products
The potency Units of Dysport® are not interchangeable with
other preparations of botulinum toxin products and, therefore, Units of
biological activity of Dysport® cannot be compared to or
converted into Units of any other botulinum toxin products. Recommended
dose and frequency of administration should not be exceeded.
Dysphagia and breathing difficulties
Immediate medical attention may be required in cases of respiratory,
speech, or swallowing difficulties. Dysphagia may persist for several
weeks, and require use of a feeding tube to maintain adequate nutrition
and hydration. Aspiration may result from severe dysphagia and is a
particular risk when treating patients in whom swallowing or respiratory
function is already compromised. Concomitant neuromuscular disorder may
exacerbate clinical effects of treatment.
Pre-existing neuromuscular disorders
Patients with neuromuscular disorders should be monitored particularly
closely when given botulinum toxin as they may be at increased risk of
clinically significant effects, including severe dysphagia and
respiratory compromise from typical doses.
Dysport® contains human albumin. Based on effective donor
screening and product manufacturing processes, it carries an extremely
remote risk for transmission of viral diseases or Creutzfeldt-Jakob
disease (CJD). No cases of transmission of viral diseases or CJD have
ever been identified for albumin.
The possibility of an immune reaction when injected intradermally is
unknown. The safety of Dysport® for the treatment of
hyperhidrosis has not been established.
Patients receiving concomitant treatment of Dysport® and
aminoglycosides or other agents interfering with neuromuscular
transmission (e.g., curare-like agents), or muscle relaxants, should be
observed closely because the effect of botulinum toxin may be
potentiated. Use of anticholinergic drugs may potentiate systemic
anticholinergic effects. The effect of administering different botulinum
neurotoxins during the course of treatment with Dysport® is
Based on animal data, may cause fetal harm. Dysport® should
be used during pregnancy only if the potential benefit justifies the
potential risk to the fetus. Care should be exercised when administering
Dysport® to elderly patients, reflecting the greater
frequency of concomitant disease and other drug therapy.
The most commonly observed adverse reactions (>5% of patients) with
Dysport® for the treatment of cervical dystonia are muscular
weakness, dysphagia, dry mouth, injection site discomfort, fatigue,
headache, neck pain, musculoskeletal pain, dysphonia, injection site
pain, and eye disorders.
Ipsen is a global specialty-driven pharmaceutical company with total
sales exceeding €1.2 billion in 2012. Ipsen's ambition is to become a
leader in specialty healthcare solutions for targeted debilitating
diseases. Its development strategy is supported by 3 franchises:
neurology, endocrinology and uro-oncology. Moreover, the Group has an
active policy of partnerships. Ipsen's R&D is focused on its innovative
and differentiated technological platforms, peptides and toxins. In
2012, R&D expenditure totalled close to €250 million, representing more
than 20% of Group sales. The Group has close to 4,900 employees
worldwide. Ipsen's shares are traded on segment A of Euronext Paris
(stock code: IPN, ISIN code: FR0010259150) and eligible to the "Service
de Règlement Différé" ("SRD"). The Group is part of the SBF 120 index.
Ipsen has implemented a Sponsored Level I American Depositary Receipt
(ADR) program, which trade on the over-the-counter market in the United
States under the symbol IPSEY. For more information on Ipsen, visit www.ipsen.com.
Forward Looking Statement
The forward-looking statements, objectives and targets contained herein
are based on the Group's management strategy, current views and
assumptions. Such statements involve known and unknown risks and
uncertainties that may cause actual results, performance or events to
differ materially from those anticipated herein. All of the above risks
could affect the Group's future ability to achieve its financial
targets, which were set assuming reasonable macroeconomic conditions
based on the information available today.
Moreover, the targets described in this document were prepared without
taking into account external growth assumptions and potential future
acquisitions, which may alter these parameters. These objectives are
based on data and assumptions regarded as reasonable by the Group. These
targets depend on conditions or facts likely to happen in the future,
and not exclusively on historical data. Actual results may depart
significantly from these targets given the occurrence of certain risks
and uncertainties, notably the fact that a promising product in early
development phase or clinical trial may end up never being launched on
the market or reaching its commercial targets, notably for regulatory or
competition reasons. The Group must face or might face competition from
Generics that might translate into a loss of market share.
Furthermore, the Research and Development process involves several
stages each of which involves the substantial risk that the Group may
fail to achieve its objectives and be forced to abandon its efforts with
regards to a product in which it has invested significant sums.
Therefore, the Group cannot be certain that favourable results obtained
during pre-clinical trials will be confirmed subsequently during
clinical trials, or that the results of clinical trials will be
sufficient to demonstrate the safe and effective nature of the product
concerned. The Group also depends on third parties to develop and market
some of its products which could potentially generate substantial
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revise any forward looking statements, targets or estimates contained in
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