Welcome!

News Feed Item

Edison Pharmaceuticals Announces Phase 2 Positive Clinical Results for EPI-743 in Rett Syndrome

Head growth significantly improved in placebo-controlled trial

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
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
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
Edison Pharmaceuticals is focused on developing treatments for children and adults with orphan mitochondrial diseases.

SOURCE Edison Pharmaceuticals, Inc.

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
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...
The 20th International Cloud Expo has announced that its Call for Papers is open. Cloud Expo, to be held June 6-8, 2017, at the Javits Center in New York City, brings together Cloud Computing, Big Data, Internet of Things, DevOps, Containers, Microservices and WebRTC to one location. With cloud computing driving a higher percentage of enterprise IT budgets every year, it becomes increasingly important to plant your flag in this fast-expanding business opportunity. Submit your speaking proposal ...
Cloud promises the agility required by today’s digital businesses. As organizations adopt cloud based infrastructures and services, their IT resources become increasingly dynamic and hybrid in nature. Managing these require modern IT operations and tools. In his session at 20th Cloud Expo, Raj Sundaram, Senior Principal Product Manager at CA Technologies, will discuss how to modernize your IT operations in order to proactively manage your hybrid cloud and IT environments. He will be sharing be...
New competitors, disruptive technologies, and growing expectations are pushing every business to both adopt and deliver new digital services. This ‘Digital Transformation’ demands rapid delivery and continuous iteration of new competitive services via multiple channels, which in turn demands new service delivery techniques – including DevOps. In this power panel at @DevOpsSummit 20th Cloud Expo, moderated by DevOps Conference Co-Chair Andi Mann, panelists will examine how DevOps helps to meet th...
NHK, Japan Broadcasting, will feature the upcoming @ThingsExpo Silicon Valley in a special 'Internet of Things' and smart technology documentary that will be filmed on the expo floor between November 3 to 5, 2015, in Santa Clara. NHK is the sole public TV network in Japan equivalent to the BBC in the UK and the largest in Asia with many award-winning science and technology programs. Japanese TV is producing a documentary about IoT and Smart technology and will be covering @ThingsExpo Silicon Val...
SYS-CON Events announced today that CA Technologies has been named "Platinum 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, New York, and 21st International Cloud Expo, which will take place in November in Silicon Valley, California.
Five years ago development was seen as a dead-end career, now it’s anything but – with an explosion in mobile and IoT initiatives increasing the demand for skilled engineers. But apart from having a ready supply of great coders, what constitutes true ‘DevOps Royalty’? It’ll be the ability to craft resilient architectures, supportability, security everywhere across the software lifecycle. In his keynote at @DevOpsSummit at 20th Cloud Expo, Jeffrey Scheaffer, GM and SVP, Continuous Delivery Busine...
In his keynote at 19th Cloud Expo, Sheng Liang, co-founder and CEO of Rancher Labs, discussed the technological advances and new business opportunities created by the rapid adoption of containers. With the success of Amazon Web Services (AWS) and various open source technologies used to build private clouds, cloud computing has become an essential component of IT strategy. However, users continue to face challenges in implementing clouds, as older technologies evolve and newer ones like Docker c...
SYS-CON Events announced today that Hitachi, the leading provider the Internet of Things and Digital Transformation, will exhibit at 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. Hitachi Data Systems, a wholly owned subsidiary of Hitachi, Ltd., offers an integrated portfolio of services and solutions that enable digital transformation through enhanced data management, governance, mobility and analytics. We help globa...
While some vendors scramble to create and sell you a fancy solution for monitoring your spanking new Amazon Lambdas, hear how you can do it on the cheap using just built-in Java APIs yourself. By exploiting a little-known fact that Lambdas aren’t exactly single threaded, you can effectively identify hot spots in your serverless code. In his session at 20th Cloud Expo, David Martin, Principal Product Owner at CA Technologies, will give a live demonstration and code walkthrough, showing how to o...
Developers want to create better apps faster. Static clouds are giving way to scalable systems, with dynamic resource allocation and application monitoring. You won't hear that chant from users on any picket line, but helping developers to create better apps faster is the mission of Lee Atchison, principal cloud architect and advocate at New Relic Inc., based in San Francisco. His singular job is to understand and drive the industry in the areas of cloud architecture, microservices, scalability ...
SYS-CON Events announced today that T-Mobile will exhibit at 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. As America's Un-carrier, T-Mobile US, Inc., is redefining the way consumers and businesses buy wireless services through leading product and service innovation. The Company's advanced nationwide 4G LTE network delivers outstanding wireless experiences to 67.4 million customers who are unwilling to compromise on ...
SYS-CON Events announced today that Juniper Networks (NYSE: JNPR), an industry leader in automated, scalable and secure networks, will exhibit at 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. Juniper Networks challenges the status quo with products, solutions and services that transform the economics of networking. The company co-innovates with customers and partners to deliver automated, scalable and secure network...
DevOps is often described as a combination of technology and culture. Without both, DevOps isn't complete. However, applying the culture to outdated technology is a recipe for disaster; as response times grow and connections between teams are delayed by technology, the culture will die. A Nutanix Enterprise Cloud has many benefits that provide the needed base for a true DevOps paradigm. In his Day 3 Keynote at 20th Cloud Expo, Chris Brown, a Solutions Marketing Manager at Nutanix, will explore t...
DevOps is being widely accepted (if not fully adopted) as essential in enterprise IT. But as Enterprise DevOps gains maturity, expands scope, and increases velocity, the need for data-driven decisions across teams becomes more acute. DevOps teams in any modern business must wrangle the ‘digital exhaust’ from the delivery toolchain, "pervasive" and "cognitive" computing, APIs and services, mobile devices and applications, the Internet of Things, and now even blockchain.