Welcome!

News Feed Item

Santhera Reports Positive Outcome for Catena(R)/Raxone(R) in Phase III DMD Trial Supported by Additional Respiratory Function Data

LIESTAL, SWITZERLAND -- (Marketwired) -- 05/22/14 -- Santhera Pharmaceuticals (SIX:SANN) today reported that the results of secondary respiratory function endpoints from the on-going analysis of the DELOS trial in Duchenne Muscular Dystrophy (DMD) corroborate the positive outcome for the primary endpoint. These data provide further supportive evidence of a treatment benefit for Catena®/Raxone® in DMD.

As previously announced, the DELOS trial met the primary endpoint, the difference between Catena®/Raxone® and placebo in the change from baseline to week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p). Hospital-based spirometry assessments demonstrated that Catena®/Raxone® significantly reduced the annual decline in PEF%p by 66% compared to patients taking placebo. The average annual decline in PEF%p was 9.0% for placebo (Baseline: 54.3%; Week 52: 45.3% (n=27), pless than 0.001) versus 3.1% for Catena®/Raxone® (Baseline PEF%P: 53.1%; Week 52: 50.1% (n=30); p=0.13) for a treatment group difference in change from Baseline to Week 52 of 5.96% (p=0.04).

Santhera today announced that this finding has been corroborated by the results of secondary endpoints assessing respiratory function in all randomized and treated subjects. When measured weekly by the patient at home using the hand-held ASMA-1 device (secondary endpoint), Catena®/Raxone® significantly reduced the annual decline in PEF%p by 80% compared to patients taking placebo. The ASMA-1 device showed a significant 9.0% decline in PEF%p occurred between Baseline and Week 52 in the placebo group (n=31; pless than 0.001), compared to a non-significant decline of 1.8% in the Catena®/Raxone® group (n=31; p=0.44), for a treatment group difference in change from Baseline to Week 52 of 7.2% (p=0.03).

Furthermore, for Forced Expiratory Volume in 1 second (as percent predicted, FEV1%p), an additional endpoint for respiratory muscle strength, Catena®/Raxone® significantly reduced the annual decline by 78% compared to patients taking placebo. The annual decline in FEV1%p in the placebo group was 10.7% compared to 2.4% in the Catena®/Raxone® group (p=0.03).

Importantly, the outcome for Forced Vital Capacity (as percent predicted, FVC%p), a measure of restrictive lung disease predictive of morbidity and mortality in DMD, also supported a treatment benefit of Catena®/Raxone®. The annual decline in FVC%p was reduced by 37% in Catena®/Raxone®-treated patients (9.0% decline in FVC%p in the placebo group versus a 5.7% decline in the Catena/Raxone group; p=0.08). No differences were observed between treatment groups in Maximal Inspiratory or Expiratory Pressures or in Peak Cough Flow.

In summary these outcomes provide clear evidence of a clinical benefit for Catena®/Raxone® in delaying the loss of respiratory function in patients with DMD compared to placebo.

As also reported previously, treatment with Catena®/Raxone® was safe and well tolerated. A total of 93.8% of Catena®/Raxone®-treated and 94.1% of placebo-treated patients experienced at least one Adverse Event (AE). Serious AEs were reported in 6.3% of Catena®/Raxone® treated and in 14.7% of placebo-treated patients. Nasopharyngitis (25.8%) and headache (19.7%) were the most common adverse events but there were no differences in the incidence of these events between the treatment groups. Diarrhoea was slightly more commonly observed in Catena®/Raxone®-treated patients (25.0% versus 11.8%), whilst upper respiratory tract infections were more frequently observed in placebo-treated patients (17.6% versus 6.3%). Overall adverse events were mild to moderate in intensity.

"We are very excited to observe this treatment benefit for Catena®/Raxone® in delaying respiratory deterioration in DMD, as evidenced by the significant outcome for the primary PEF endpoint in DELOS. The positive results of secondary outcomes, in addition to the primary endpoint, are clearly supportive and establish the clinical meaningfulness of Catena®/Raxone® treatment. This is of critical importance for the demonstration of substantial evidence of effectiveness in the regulatory process", explained Nick Coppard, SVP Development at Santhera. "Having demonstrated a significant and clinically relevant benefit without safety concerns, we believe we are well positioned to discuss regulatory approval with the authorities".

These data are presented today at the BioEURquity Europe 2014 Conference in Amsterdam and will be available for download on the Company's website www.santhera.com under Investors/Presentations.

About DELOS

DELOS was a Phase III, double-blind, placebo-controlled trial which randomized and treated 64 European and US patients with DMD who were not receiving concomitant corticosteroids to either Catena®/Raxone® tablets (900mg: 300mg 3 times daily, N=31) or to matching placebo (N=33). Their average age was 14.3 years and 5 were ambulatory and 59 non-ambulatory at Baseline. There were no eligibility criteria for mutational status. The study was designed to assess the efficacy of Catena®/Raxone® in improving or delaying the loss of respiratory function in patients with DMD not using corticosteroids compared to placebo. The preservation of respiratory function is acknowledged by clinicians and by regulatory authorities as of major clinical importance for patients with DMD.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and results in rapidly progressive muscle weakness. It is a genetic, degenerative disease that is inherited in an X-linked recessive mode with an incidence of approximately 1 in 3,500 live born males worldwide. DMD is characterized by a loss of the protein dystrophin, leading to cell damage, impaired calcium homeostasis, elevated oxidative stress and reduced energy production in muscle cells. This results in progressive muscle weakness and wasting and early morbidity due to respiratory failure. Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport and supplementing cellular energy levels.

About Santhera

Santhera Pharmaceuticals (SIX:SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera develops Catena®/Raxone® as treatment for patients with Leber's Hereditary Optic Neuropathy (LHON), Duchenne Muscular Dystrophy (DMD) and primary progressive Multiple Sclerosis (ppMS), all areas of high unmet medical need with no current therapies. Santhera previously received temporary approval (cATU) for Raxone® in the treatment of LHON in France and has recently submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for the treatment of LHON in the European Union. Santhera recently estimated that the combined annual peak sales potential for Catena®/Raxone® in the LHON and DMD indications could reach CHF 600 million.

For further information, please visit the Company's website www.santhera.com.

Raxone® and Catena® are trademarks of Santhera Pharmaceuticals.

Disclaimer / Forward-looking statements

This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

More Stories By Marketwired .

Copyright © 2009 Marketwired. All rights reserved. All the news releases provided by Marketwired are copyrighted. Any forms of copying other than an individual user's personal reference without express written permission is prohibited. Further distribution of these materials is strictly forbidden, including but not limited to, posting, emailing, faxing, archiving in a public database, redistributing via a computer network or in a printed form.

Latest Stories
The IoT is changing the way enterprises conduct business. In his session at @ThingsExpo, Eric Hoffman, Vice President at EastBanc Technologies, discussed how businesses can gain an edge over competitors by empowering consumers to take control through IoT. He cited examples such as a Washington, D.C.-based sports club that leveraged IoT and the cloud to develop a comprehensive booking system. He also highlighted how IoT can revitalize and restore outdated business models, making them profitable ...
StackIQ has announced the release of Stacki 3.2. Stacki is an easy-to-use Linux server provisioning tool. Stacki 3.2 delivers new capabilities that simplify the automation and integration of site-specific requirements. StackIQ is the commercial entity behind this open source bare metal provisioning tool. Since the release of Stacki in June of 2015, the Stacki core team has been focused on making the Community Edition meet the needs of members of the community, adding features and value, while ...
The cloud competition for database hosts is fierce. How do you evaluate a cloud provider for your database platform? In his session at 18th Cloud Expo, Chris Presley, a Solutions Architect at Pythian, gave users a checklist of considerations when choosing a provider. Chris Presley is a Solutions Architect at Pythian. He loves order – making him a premier Microsoft SQL Server expert. Not only has he programmed and administered SQL Server, but he has also shared his expertise and passion with b...
With 15% of enterprises adopting a hybrid IT strategy, you need to set a plan to integrate hybrid cloud throughout your infrastructure. In his session at 18th Cloud Expo, Steven Dreher, Director of Solutions Architecture at Green House Data, discussed how to plan for shifting resource requirements, overcome challenges, and implement hybrid IT alongside your existing data center assets. Highlights included anticipating workload, cost and resource calculations, integrating services on both sides...
Big Data engines are powering a lot of service businesses right now. Data is collected from users from wearable technologies, web behaviors, purchase behavior as well as several arbitrary data points we’d never think of. The demand for faster and bigger engines to crunch and serve up the data to services is growing exponentially. You see a LOT of correlation between “Cloud” and “Big Data” but on Big Data and “Hybrid,” where hybrid hosting is the sanest approach to the Big Data Infrastructure pro...
"We are a well-established player in the application life cycle management market and we also have a very strong version control product," stated Flint Brenton, CEO of CollabNet,, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
We all know the latest numbers: Gartner, Inc. forecasts that 6.4 billion connected things will be in use worldwide in 2016, up 30 percent from last year, and will reach 20.8 billion by 2020. We're rapidly approaching a data production of 40 zettabytes a day – more than we can every physically store, and exabytes and yottabytes are just around the corner. For many that’s a good sign, as data has been proven to equal money – IF it’s ingested, integrated, and analyzed fast enough. Without real-ti...
In his session at 18th Cloud Expo, Sagi Brody, Chief Technology Officer at Webair Internet Development Inc., and Logan Best, Infrastructure & Network Engineer at Webair, focused on real world deployments of DDoS mitigation strategies in every layer of the network. He gave an overview of methods to prevent these attacks and best practices on how to provide protection in complex cloud platforms. He also outlined what we have found in our experience managing and running thousands of Linux and Unix ...
In his session at @DevOpsSummit at 19th Cloud Expo, Yoseph Reuveni, Director of Software Engineering at Jet.com, will discuss Jet.com's journey into containerizing Microsoft-based technologies like C# and F# into Docker. He will talk about lessons learned and challenges faced, the Mono framework tryout and how they deployed everything into Azure cloud. Yoseph Reuveni is a technology leader with unique experience developing and running high throughput (over 1M tps) distributed systems with extre...
"Operations is sort of the maturation of cloud utilization and the move to the cloud," explained Steve Anderson, Product Manager for BMC’s Cloud Lifecycle Management, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
I wanted to gather all of my Internet of Things (IOT) blogs into a single blog (that I could later use with my University of San Francisco (USF) Big Data “MBA” course). However as I started to pull these blogs together, I realized that my IOT discussion lacked a vision; it lacked an end point towards which an organization could drive their IOT envisioning, proof of value, app dev, data engineering and data science efforts. And I think that the IOT end point is really quite simple…
A critical component of any IoT project is what to do with all the data being generated. This data needs to be captured, processed, structured, and stored in a way to facilitate different kinds of queries. Traditional data warehouse and analytical systems are mature technologies that can be used to handle certain kinds of queries, but they are not always well suited to many problems, particularly when there is a need for real-time insights.
UpGuard has become a member of the Center for Internet Security (CIS), and will continue to help businesses expand visibility into their cyber risk by providing hardening benchmarks to all customers. By incorporating these benchmarks, UpGuard's CSTAR solution builds on its lead in providing the most complete assessment of both internal and external cyber risk. CIS benchmarks are a widely accepted set of hardening guidelines that have been publicly available for years. Numerous solutions exist t...
Unless your company can spend a lot of money on new technology, re-engineering your environment and hiring a comprehensive cybersecurity team, you will most likely move to the cloud or seek external service partnerships. In his session at 18th Cloud Expo, Darren Guccione, CEO of Keeper Security, revealed what you need to know when it comes to encryption in the cloud.
We're entering the post-smartphone era, where wearable gadgets from watches and fitness bands to glasses and health aids will power the next technological revolution. With mass adoption of wearable devices comes a new data ecosystem that must be protected. Wearables open new pathways that facilitate the tracking, sharing and storing of consumers’ personal health, location and daily activity data. Consumers have some idea of the data these devices capture, but most don’t realize how revealing and...