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.

Contacts:
Santhera Pharmaceuticals
Thomas Meier
Chief Executive Officer
+41 61 906 89 64
[email protected]

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
SYS-CON Events announced today that DXWorldExpo has been named “Global Sponsor” of SYS-CON's 21st International Cloud Expo, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. Digital Transformation is the key issue driving the global enterprise IT business. Digital Transformation is most prominent among Global 2000 enterprises and government institutions.
SYS-CON Events announced today that NetApp has been named “Bronze Sponsor” of SYS-CON's 21st International Cloud Expo®, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. NetApp is the data authority for hybrid cloud. NetApp provides a full range of hybrid cloud data services that simplify management of applications and data across cloud and on-premises environments to accelerate digital transformation. Together with their partners, NetApp em...
One of the biggest challenges with adopting a DevOps mentality is: new applications are easily adapted to cloud-native, microservice-based, or containerized architectures - they can be built for them - but old applications need complex refactoring. On the other hand, these new technologies can require relearning or adapting new, oftentimes more complex, methodologies and tools to be ready for production. In his general session at @DevOpsSummit at 20th Cloud Expo, Chris Brown, Solutions Marketi...
SYS-CON Events announced today that SIGMA Corporation will exhibit at the Japan External Trade Organization (JETRO) Pavilion at SYS-CON's 21st International Cloud Expo®, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. uLaser flow inspection device from the Japanese top share to Global Standard! Then, make the best use of data to flip to next page. For more information, visit http://www.sigma-k.co.jp/en/.
Most of the time there is a lot of work involved to move to the cloud, and most of that isn't really related to AWS or Azure or Google Cloud. Before we talk about public cloud vendors and DevOps tools, there are usually several technical and non-technical challenges that are connected to it and that every company needs to solve to move to the cloud. In his session at 21st Cloud Expo, Stefano Bellasio, CEO and founder of Cloud Academy Inc., will discuss what the tools, disciplines, and cultural...
Why Federal cloud? What is in Federal Clouds and integrations? This session will identify the process and the FedRAMP initiative. But is it sufficient? What is the remedy for keeping abreast of cutting-edge technology? In his session at 21st Cloud Expo, Rasananda Behera will examine the proposed solutions: Private or public or hybrid cloud Responsible governing bodies How can we accomplish?
SYS-CON Events announced today that N3N will exhibit at SYS-CON's @ThingsExpo, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. N3N’s solutions increase the effectiveness of operations and control centers, increase the value of IoT investments, and facilitate real-time operational decision making. N3N enables operations teams with a four dimensional digital “big board” that consolidates real-time live video feeds alongside IoT sensor data a...
DevOps at Cloud Expo, taking place October 31 - November 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 21st Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. The widespread success of cloud computing is driving the DevOps revolution in enterprise IT. Now as never before, development teams must communicate and collaborate in a dynamic, 24/7/365 environment. There is no time to w...
Real IoT production deployments running at scale are collecting sensor data from hundreds / thousands / millions of devices. The goal is to take business-critical actions on the real-time data and find insights from stored datasets. In his session at @ThingsExpo, John Walicki, Watson IoT Developer Advocate at IBM Cloud, will provide a fast-paced developer journey that follows the IoT sensor data from generation, to edge gateway, to edge analytics, to encryption, to the IBM Bluemix cloud, to Wa...
With the rise of DevOps, containers are at the brink of becoming a pervasive technology in Enterprise IT to accelerate application delivery for the business. When it comes to adopting containers in the enterprise, security is the highest adoption barrier. Is your organization ready to address the security risks with containers for your DevOps environment? In his session at @DevOpsSummit at 21st Cloud Expo, Chris Van Tuin, Chief Technologist, NA West at Red Hat, will discuss: The top security r...
There is huge complexity in implementing a successful digital business that requires efficient on-premise and cloud back-end infrastructure, IT and Internet of Things (IoT) data, analytics, Machine Learning, Artificial Intelligence (AI) and Digital Applications. In the data center alone, there are physical and virtual infrastructures, multiple operating systems, multiple applications and new and emerging business and technological paradigms such as cloud computing and XaaS. And then there are pe...
SYS-CON Events announced today that B2Cloud will exhibit at SYS-CON's 21st International Cloud Expo®, which will take place on Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA. B2Cloud specializes in IoT devices for preventive and predictive maintenance in any kind of equipment retrieving data like Energy consumption, working time, temperature, humidity, pressure, etc.
DevOps at Cloud Expo – being held October 31 - November 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA – announces that its Call for Papers is open. Born out of proven success in agile development, cloud computing, and process automation, DevOps is a macro trend you cannot afford to miss. From showcase success stories from early adopters and web-scale businesses, DevOps is expanding to organizations of all sizes, including the world's largest enterprises – and delivering real r...
With major technology companies and startups seriously embracing Cloud strategies, now is the perfect time to attend 21st Cloud Expo October 31 - November 2, 2017, at the Santa Clara Convention Center, CA, and June 12-14, 2018, at the Javits Center in New York City, NY, and learn what is going on, contribute to the discussions, and ensure that your enterprise is on the right path to Digital Transformation.
Your clients expect transactions to never fail, cloud access to be fast and always on, and their data to be protected - no exceptions. Hear about how Secure Service Container (SSC), an IBM-exclusive open technology, enables secure building and hosting of next-generation applications, both cloud and on-premises. SSC protects the full stack from external and insider threats, allows automatic encryption of data in-flight and at-rest, and is tamper-resistant during installation and runtime – with no...