|By PR Newswire||
|September 3, 2014 12:01 AM EDT||
MOUNTAIN VIEW, Calif., Sept. 3, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced results of a recently completed phase 2 placebo-controlled trial in pediatric patients. Rett syndrome patients treated with EPI-743 demonstrated a statistically significant increase in head circumference. Decreased brain growth and head circumference are hallmarks of the disease.
Rett syndrome is a genetic neurodevelopmental disorder predominately affecting girls, and is usually diagnosed by 24 months of age. It is characterized by a constellation of neurological, behavioral, functional, and structural features. These include delayed development, characteristic hand wringing, seizures, impaired respiratory function, slowing of brain growth, and resultant diminished head size, as well as other neurological disorders.
The double-blind, placebo-controlled clinical trial was conducted at the General University Hospital (Azienda Ospedaliera Universitaria Senese) of Siena, Italy, a leading international Rett syndrome clinical care and research center. A total of 24 subjects, 2.5 to 8 years of age, were enrolled in the six-month trial. While the study did not meet the primary endpoint of improving the subjective Rett syndrome disease severity score, subjects treated with EPI-743 demonstrated an objective improvement in head growth. Specifically, head growth in the treatment group was 0.75 cm from baseline, compared to 0.29 cm in the placebo group (p=0.05). In a subgroup analysis of the children with the largest head growth, improvements in oxygenation, hand function, and disease-relevant biomarkers were also recorded. These data will be submitted for publication in the near future.
The natural history of decreased head growth in girls with Rett syndrome has been quantified and is an established metric of disease. Improvement in head growth was recorded in nine of the 12 subjects in the EPI-743 treatment arm.
In this study, EPI-743 was well-tolerated with no drug-related serious adverse events or dose-limiting toxicities reported.
The rationale for the trial was to determine if EPI-743 could address putative mitochondrial/oxidative stress components of Rett syndrome.
"Given the established role of oxidative stress in the pathology of Rett syndrome, we were interested in exploring whether a mitochondrial-targeted therapeutic would result in treatment benefit," said Joussef Hayek, MD, Professor of Child Neuropsychiatry and Principal Investigator of the study. "This study represents an important step in building a bridge between understanding the underlying pathology of Rett syndrome and discovering new treatments."
"With the encouraging results on such an objective and meaningful endpoint, we plan to engage with health authorities, patient foundations, and the clinical/research community in the next several months to define the next steps that may ultimately lead to new options for children with Rett syndrome and other mitochondrial diseases," said Matthew Klein, MD MS FACS, Chief Medical Officer at Edison Pharmaceuticals, Inc.
Rett syndrome is a severe rare neurodevelopmental disorder affecting females almost exclusively. The disease results in slowing of brain growth, seizures, loss of motor function, loss of speech, and impaired respiratory function, as well as a characteristic hand stereotypy with progressive loss of purposeful hand function. Children with Rett syndrome also demonstrate autistic-like features, including diminished eye contact; for this reason Rett syndrome has been considered an autism spectrum disorder. Recent studies have demonstrated that Rett syndrome is characterized by high levels of systemic oxidative stress as well as alterations in mitochondrial morphology. Plasma levels of oxidative stress biomarkers have been shown to correlate with disease severity and progression. A recently published study, led by Claudio De Felice, MD, Professor of Neonatology and a co-investigator in the clinical trial, has documented the relationship between levels of oxidative stress markers in the brain and the disease in an in vivo disease model.
Rett syndrome affects one in every 10,000 to 15,000 live female births. The majority of cases result from a mutation in the MeCP2 gene that is carried on the X chromosome. Rett syndrome is typically diagnosed between six months and 18 months of age. Children then progress through the four stages of Rett syndrome, which are characterized by worsening neurological status and seizures. Death typically occurs prior to age 40. There is no cure for Rett syndrome and therapy is directed at management of disease symptoms.
EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of disorders of energy metabolism. Currently EPI-743 is in phase 2 clinical development.
Edison Pharmaceuticals is focused on developing treatments for children and adults with orphan mitochondrial diseases.
SOURCE Edison Pharmaceuticals, Inc.
Docker is hot. However, as Docker container use spreads into more mature production pipelines, there can be issues about control of Docker images to ensure they are production-ready. Is a promotion-based model appropriate to control and track the flow of Docker images from development to production? In his session at DevOps Summit, Fred Simon, Co-founder and Chief Architect of JFrog, will demonstrate how to implement a promotion model for Docker images using a binary repository, and then show h...
Oct. 9, 2015 06:15 AM EDT
Containers have changed the mind of IT in DevOps. They enable developers to work with dev, test, stage and production environments identically. Containers provide the right abstraction for microservices and many cloud platforms have integrated them into deployment pipelines. DevOps and Containers together help companies to achieve their business goals faster and more effectively.
Oct. 9, 2015 06:15 AM EDT
The buzz continues for cloud, data analytics and the Internet of Things (IoT) and their collective impact across all industries. But a new conversation is emerging - how do companies use industry disruption and technology enablers to lead in markets undergoing change, uncertainty and ambiguity? Organizations of all sizes need to evolve and transform, often under massive pressure, as industry lines blur and merge and traditional business models are assaulted and turned upside down. In this new da...
Oct. 9, 2015 06:00 AM EDT Reads: 283
WebRTC converts the entire network into a ubiquitous communications cloud thereby connecting anytime, anywhere through any point. In his session at WebRTC Summit,, Mark Castleman, EIR at Bell Labs and Head of Future X Labs, will discuss how the transformational nature of communications is achieved through the democratizing force of WebRTC. WebRTC is doing for voice what HTML did for web content.
Oct. 9, 2015 06:00 AM EDT Reads: 1,393
Too often with compelling new technologies market participants become overly enamored with that attractiveness of the technology and neglect underlying business drivers. This tendency, what some call the “newest shiny object syndrome,” is understandable given that virtually all of us are heavily engaged in technology. But it is also mistaken. Without concrete business cases driving its deployment, IoT, like many other technologies before it, will fade into obscurity.
Oct. 9, 2015 05:45 AM EDT
Achim Weiss is Chief Executive Officer and co-founder of ProfitBricks. In 1995, he broke off his studies to co-found the web hosting company "Schlund+Partner." The company "Schlund+Partner" later became the 1&1 web hosting product line. From 1995 to 2008, he was the technical director for several important projects: the largest web hosting platform in the world, the second largest DSL platform, a video on-demand delivery network, the largest eMail backend in Europe, and a universal billing syste...
Oct. 9, 2015 05:45 AM EDT
There are many considerations when moving applications from on-premise to cloud. It is critical to understand the benefits and also challenges of this migration. A successful migration will result in lower Total Cost of Ownership, yet offer the same or higher level of robustness. Migration to cloud shifts computing resources from your data center, which can yield significant advantages provided that the cloud vendor an offer enterprise-grade quality for your application.
Oct. 9, 2015 05:30 AM EDT Reads: 247
The IoT is upon us, but today’s databases, built on 30-year-old math, require multiple platforms to create a single solution. Data demands of the IoT require Big Data systems that can handle ingest, transactions and analytics concurrently adapting to varied situations as they occur, with speed at scale. In his session at @ThingsExpo, Chad Jones, chief strategy officer at Deep Information Sciences, will look differently at IoT data so enterprises can fully leverage their IoT potential. He’ll sha...
Oct. 9, 2015 05:15 AM EDT Reads: 503
Today air travel is a minefield of delays, hassles and customer disappointment. Airlines struggle to revitalize the experience. GE and M2Mi will demonstrate practical examples of how IoT solutions are helping airlines bring back personalization, reduce trip time and improve reliability. In their session at @ThingsExpo, Shyam Varan Nath, Principal Architect with GE, and Dr. Sarah Cooper, M2Mi's VP Business Development and Engineering, will explore the IoT cloud-based platform technologies driv...
Oct. 9, 2015 05:15 AM EDT
In today's digital world, change is the one constant. Disruptive innovations like cloud, mobility, social media, and the Internet of Things have reshaped the market and set new standards in customer expectations. To remain competitive, businesses must tap the potential of emerging technologies and markets through the rapid release of new products and services. However, the rigid and siloed structures of traditional IT platforms and processes are slowing them down – resulting in lengthy delivery ...
Oct. 9, 2015 05:00 AM EDT Reads: 1,008
The modern software development landscape consists of best practices and tools that allow teams to deliver software in a near-continuous manner. By adopting a culture of automation, measurement and sharing, the time to ship code has been greatly reduced, allowing for shorter release cycles and quicker feedback from customers and users. Still, with all of these tools and methods, how can teams stay on top of what is taking place across their infrastructure and codebase? Hopping between services a...
Oct. 9, 2015 05:00 AM EDT Reads: 489
As a CIO, are your direct reports IT managers or are they IT leaders? The hard truth is that many IT managers have risen through the ranks based on their technical skills, not their leadership ability. Many are unable to effectively engage and inspire, creating forward momentum in the direction of desired change. Renowned for its approach to leadership and emphasis on their people, organizations increasingly look to our military for insight into these challenges.
Oct. 9, 2015 04:45 AM EDT
DevOps and Continuous Delivery software provider XebiaLabs has announced it has been selected to join the Amazon Web Services (AWS) DevOps Competency partner program. The program is designed to highlight software vendors like XebiaLabs who have demonstrated technical expertise and proven customer success in DevOps and specialized solution areas like Continuous Delivery. DevOps Competency Partners provide solutions to, or have deep experience working with AWS users and other businesses to help t...
Oct. 9, 2015 04:00 AM EDT Reads: 218
The broad selection of hardware, the rapid evolution of operating systems and the time-to-market for mobile apps has been so rapid that new challenges for developers and engineers arise every day. Security, testing, hosting, and other metrics have to be considered through the process. In his session at Big Data Expo, Walter Maguire, Chief Field Technologist, HP Big Data Group, at Hewlett-Packard, will discuss the challenges faced by developers and a composite Big Data applications builder, foc...
Oct. 9, 2015 04:00 AM EDT Reads: 500
Nowadays, a large number of sensors and devices are connected to the network. Leading-edge IoT technologies integrate various types of sensor data to create a new value for several business decision scenarios. The transparent cloud is a model of a new IoT emergence service platform. Many service providers store and access various types of sensor data in order to create and find out new business values by integrating such data.
Oct. 9, 2015 04:00 AM EDT Reads: 566