|By Marketwired .||
|May 22, 2014 01:00 AM EDT||
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.
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.
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.
Chief Executive Officer
+41 61 906 89 64
Bert Loomis was a visionary. This general session will highlight how Bert Loomis and people like him inspire us to build great things with small inventions. In their general session at 19th Cloud Expo, Harold Hannon, Architect at IBM Bluemix, and Michael O'Neill, Strategic Business Development at Nvidia, discussed the accelerating pace of AI development and how IBM Cloud and NVIDIA are partnering to bring AI capabilities to "every day," on-demand. They also reviewed two "free infrastructure" pr...
Feb. 26, 2017 01:15 PM EST Reads: 1,759
Cloud Expo, Inc. has announced today that Aruna Ravichandran, vice president of DevOps Product and Solutions Marketing at CA Technologies, has been named co-conference chair of DevOps at Cloud Expo 2017. The @DevOpsSummit at Cloud Expo New York will take place on June 6-8, 2017, at the Javits Center in New York City, New York, and @DevOpsSummit at Cloud Expo Silicon Valley will take place Oct. 31-Nov. 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
Feb. 26, 2017 01:15 PM EST Reads: 2,164
In his general session at 18th Cloud Expo, Lee Atchison, Principal Cloud Architect and Advocate at New Relic, discussed cloud as a ‘better data center’ and how it adds new capacity (faster) and improves application availability (redundancy). The cloud is a ‘Dynamic Tool for Dynamic Apps’ and resource allocation is an integral part of your application architecture, so use only the resources you need and allocate /de-allocate resources on the fly.
Feb. 26, 2017 01:00 PM EST Reads: 1,774
Have you ever noticed how some IT people seem to lead successful, rewarding, and satisfying lives and careers, while others struggle? IT author and speaker Don Crawley uncovered the five principles that successful IT people use to build satisfying lives and careers and he shares them in this fast-paced, thought-provoking webinar. You'll learn the importance of striking a balance with technical skills and people skills, challenge your pre-existing ideas about IT customer service, and gain new in...
Feb. 26, 2017 01:00 PM EST Reads: 2,984
The Internet of Things will challenge the status quo of how IT and development organizations operate. Or will it? Certainly the fog layer of IoT requires special insights about data ontology, security and transactional integrity. But the developmental challenges are the same: People, Process and Platform. In his session at @ThingsExpo, Craig Sproule, CEO of Metavine, demonstrated how to move beyond today's coding paradigm and shared the must-have mindsets for removing complexity from the develop...
Feb. 26, 2017 12:45 PM EST Reads: 6,369
SYS-CON Events announced today that Hitrons Solutions will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Hitrons Solutions Inc. is distributor in the North American market for unique products and services of small and medium-size businesses, including cloud services and solutions, SEO marketing platforms, and mobile applications.
Feb. 26, 2017 12:45 PM EST Reads: 522
@DevOpsSummit at Cloud taking place June 6-8, 2017, at Javits Center, New York City, is co-located with the 20th International 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 wait for long developm...
Feb. 26, 2017 12:30 PM EST Reads: 1,993
For basic one-to-one voice or video calling solutions, WebRTC has proven to be a very powerful technology. Although WebRTC’s core functionality is to provide secure, real-time p2p media streaming, leveraging native platform features and server-side components brings up new communication capabilities for web and native mobile applications, allowing for advanced multi-user use cases such as video broadcasting, conferencing, and media recording.
Feb. 26, 2017 11:30 AM EST Reads: 6,676
In his session at @ThingsExpo, Steve Wilkes, CTO and founder of Striim, will delve into four enterprise-scale, business-critical case studies where streaming analytics serves as the key to enabling real-time data integration and right-time insights in hybrid cloud, IoT, and fog computing environments. As part of this discussion, he will also present a demo based on its partnership with Fujitsu, highlighting their technologies in a healthcare IoT use-case. The demo showcases the tracking of patie...
Feb. 26, 2017 11:00 AM EST Reads: 1,307
DevOps and microservices are permeating software engineering teams broadly, whether these teams are in pure software shops but happen to run a business, such Uber and Airbnb, or in companies that rely heavily on software to run more traditional business, such as financial firms or high-end manufacturers. Microservices and DevOps have created software development and therefore business speed and agility benefits, but they have also created problems; specifically, they have created software securi...
Feb. 26, 2017 11:00 AM EST Reads: 4,084
Almost two-thirds of companies either have or soon will have IoT as the backbone of their business. Though, IoT is far more complex than most firms expected with a majority of IoT projects having failed. How can you not get trapped in the pitfalls? In his session at @ThingsExpo, Tony Shan, Chief IoTologist at Wipro, will introduce a holistic method of IoTification, which is the process of IoTifying the existing technology portfolios and business models to adopt and leverage IoT. He will delve in...
Feb. 26, 2017 11:00 AM EST Reads: 2,754
In today's enterprise, digital transformation represents organizational change even more so than technology change, as customer preferences and behavior drive end-to-end transformation across lines of business as well as IT. To capitalize on the ubiquitous disruption driving this transformation, companies must be able to innovate at an increasingly rapid pace. Traditional approaches for driving innovation are now woefully inadequate for keeping up with the breadth of disruption and change facing...
Feb. 26, 2017 10:45 AM EST Reads: 8,108
SYS-CON Events announced today that Outlyer, a monitoring service for DevOps and operations teams, has been named “Bronze Sponsor” of SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. Outlyer is a monitoring service for DevOps and Operations teams running Cloud, SaaS, Microservices and IoT deployments. Designed for today's dynamic environments that need beyond cloud-scale monitoring, we make monitoring effortless so you...
Feb. 26, 2017 10:30 AM EST Reads: 2,395
Cloud Expo, Inc. has announced today that Andi Mann and Aruna Ravichandran have been named Co-Chairs of @DevOpsSummit at Cloud Expo 2017. The @DevOpsSummit at Cloud Expo New York will take place on June 6-8, 2017, at the Javits Center in New York City, New York, and @DevOpsSummit at Cloud Expo Silicon Valley will take place Oct. 31-Nov. 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
Feb. 26, 2017 10:15 AM EST Reads: 2,417
Stratoscale, the software company developing the next generation data center operating system, exhibited at SYS-CON's 18th International Cloud Expo®, which took place at the Javits Center in New York City, NY, in June 2016.Stratoscale is revolutionizing the data center with a zero-to-cloud-in-minutes solution. With Stratoscale’s hardware-agnostic, Software Defined Data Center (SDDC) solution to store everything, run anything and scale everywhere, IT is empowered to take control of their data ce...
Feb. 26, 2017 10:15 AM EST Reads: 1,464