PARIS--(BUSINESS WIRE)--Regulatory News:
Ipsen (Euronext: IPN; ADR: IPSEY) today announced topline results for two double-blind phase III studies of Dysport® (abobotulinumtoxinA) in Pediatric Lower Limb (PLL) spasticity in children with cerebral palsy and in Adult Lower Limb (ALL) spasticity in patients who had experienced a stroke or traumatic brain injury.
In the PLL phase III study, conducted in children with hemiparetic or diplegic cerebral palsy, treatment with Dysport® showed a statistically significant response versus placebo in the improvement of muscle tone, as measured by the Modified Ashworth Scale (MAS; primary endpoint), and a statistically significant overall benefit versus placebo, as measured by the Physician Global Assessment (PGA; first secondary endpoint).
In the ALL phase III study, conducted in hemiparetic patients who had experienced a stroke or traumatic brain injury, treatment with Dysport® at the dose of 1500U showed a statistically significant response versus placebo in the improvement of muscle tone, as measured by the Modified Ashworth Scale (MAS; primary endpoint). An overall benefit (measured by the Physician Global Assessment (PGA); first secondary endpoint) versus placebo was observed but did not reach statistical significance according to the pre-specified statistical analysis.
Other spasticity and functional outcome results are currently being analyzed. The safety profile observed in the studies was consistent with the known safety profile of Dysport® in these indications.
Comprehensive results from these double-blind studies will be disclosed in the next few months at major international congresses. Ipsen will share these results with key regulatory agencies this year.
Claude Bertrand, Executive Vice-President Research & Development and Chief Scientific Officer of Ipsen commented: “We believe these results should meet the expectations of physicians by potentially providing a new alternative for treating adults and children suffering from lower limb spasticity. This unique data is a testimony to Ipsen’s growing scientific leadership in the field of toxins. We are grateful to the clinicians, caregivers, patients and their families who were involved in this study.”
Pr. Mauricio Delgado, Principal Investigator of the PLL study stated: “This is the largest pediatric double-blind placebo controlled study demonstrating that Dysport® is an effective and safe treatment for spasticity in children with Cerebral Palsy. Unlike previous studies, this study was designed to demonstrate efficacy through a variety of outcome measures that are of direct relevance to the patient and their family. The study brought together an international and multidisciplinary group of investigators including pediatric neurologists, physiatrists, orthopedic surgeons and physical therapists who got the benefit of using standardized clinical assessments having a positive impact on their clinical practice.”
Pr. Jean Michel Gracies, Principal Investigator of the ALL study stated: “This global double-blind phase III study provides evidence that Dysport® demonstrates high benefit in adults with stroke or traumatic brain injury causing lower limb spasticity. This study also reveals that it was possible to achieve highly productive collaboration with a very large number of investigators from several countries and health systems. In addition, it must be stressed that this study involved multidisciplinary teams including physical and occupational therapists, particularly involved in patient assessment. These important results reinforce the positioning of Dysport® as an excellent treatment for patients with spastic paresis.”
About the studies
The Phase III study conducted in children with cerebral palsy included 235 patients and was multicentric, prospective, double blind, randomized, and placebo-controlled. It was conducted in the USA, France, Turkey, Poland, Mexico and Chile. The purpose of this study in children was to assess the efficacy and safety of Dysport® compared to placebo in improving lower limb spasticity in hemiparetic or diplegic cerebral palsy patients.
The phase III study in adults suffering from lower limb spasticity included 388 patients and was international, multicentric, prospective, double blind, randomized 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 and safety of Dysport® compared to placebo in improving lower limb spasticity in hemiparetic adult patients who had experienced a stroke or a traumatic brain injury.
The primary endpoint for both studies was the improvement of muscle tone in the treated lower limb measured by the Modified Ashworth Scale (MAS). 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 an outcome measure used to assess the overall clinical benefit for the patient. 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 82 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 and pediatric lower limb spasticity in many international markets, but not in the United States (US). Dysport® is also approved for the treatment of adult lower limb spasticity in some European countries, 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 studies in adults and children with lower limb spasticity are of an investigational use of Dysport® in the USA.
Ipsen is a global specialty-driven pharmaceutical company with total sales exceeding €1.2 billion in 2013. 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 2013, R&D expenditure totaled close to €260 million, representing more than 21% of Group sales. Moreover, Ipsen also has a significant presence in primary care. The Group has close to 4,600 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.
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