Click here to close now.


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 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
The Internet of Things (IoT) is growing rapidly by extending current technologies, products and networks. By 2020, Cisco estimates there will be 50 billion connected devices. Gartner has forecast revenues of over $300 billion, just to IoT suppliers. Now is the time to figure out how you’ll make money – not just create innovative products. With hundreds of new products and companies jumping into the IoT fray every month, there’s no shortage of innovation. Despite this, McKinsey/VisionMobile data...
With major technology companies and startups seriously embracing IoT strategies, now is the perfect time to attend @ThingsExpo 2016 in New York and Silicon Valley. Learn what is going on, contribute to the discussions, and ensure that your enterprise is as "IoT-Ready" as it can be! Internet of @ThingsExpo, taking place Nov 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 17th Cloud Expo and will feature technical sessions from a rock star conference faculty ...
In his General Session at DevOps Summit, Asaf Yigal, Co-Founder & VP of Product at, explored the value of Kibana 4 for log analysis and provided a hands-on tutorial on how to set up Kibana 4 and get the most out of Apache log files. He examined three use cases: IT operations, business intelligence, and security and compliance. Asaf Yigal is co-founder and VP of Product at log analytics software company In the past, he was co-founder of social-trading platform Currensee, which...
Just over a week ago I received a long and loud sustained applause for a presentation I delivered at this year’s Cloud Expo in Santa Clara. I was extremely pleased with the turnout and had some very good conversations with many of the attendees. Over the next few days I had many more meaningful conversations and was not only happy with the results but also learned a few new things. Here is everything I learned in those three days distilled into three short points.
DevOps is about increasing efficiency, but nothing is more inefficient than building the same application twice. However, this is a routine occurrence with enterprise applications that need both a rich desktop web interface and strong mobile support. With recent technological advances from Isomorphic Software and others, rich desktop and tuned mobile experiences can now be created with a single codebase – without compromising functionality, performance or usability. In his session at DevOps Su...
As organizations realize the scope of the Internet of Things, gaining key insights from Big Data, through the use of advanced analytics, becomes crucial. However, IoT also creates the need for petabyte scale storage of data from millions of devices. A new type of Storage is required which seamlessly integrates robust data analytics with massive scale. These storage systems will act as “smart systems” provide in-place analytics that speed discovery and enable businesses to quickly derive meaningf...
In his keynote at @ThingsExpo, Chris Matthieu, Director of IoT Engineering at Citrix and co-founder and CTO of Octoblu, focused on building an IoT platform and company. He provided a behind-the-scenes look at Octoblu’s platform, business, and pivots along the way (including the Citrix acquisition of Octoblu).
In his General Session at 17th Cloud Expo, Bruce Swann, Senior Product Marketing Manager for Adobe Campaign, explored the key ingredients of cross-channel marketing in a digital world. Learn how the Adobe Marketing Cloud can help marketers embrace opportunities for personalized, relevant and real-time customer engagement across offline (direct mail, point of sale, call center) and digital (email, website, SMS, mobile apps, social networks, connected objects).
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...
We all know that data growth is exploding and storage budgets are shrinking. Instead of showing you charts on about how much data there is, in his General Session at 17th Cloud Expo, Scott Cleland, Senior Director of Product Marketing at HGST, showed how to capture all of your data in one place. After you have your data under control, you can then analyze it in one place, saving time and resources.
Culture is the most important ingredient of DevOps. The challenge for most organizations is defining and communicating a vision of beneficial DevOps culture for their organizations, and then facilitating the changes needed to achieve that. Often this comes down to an ability to provide true leadership. 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 ab...
In recent years, at least 40% of companies using cloud applications have experienced data loss. One of the best prevention against cloud data loss is backing up your cloud data. In his General Session at 17th Cloud Expo, Sam McIntyre, Partner Enablement Specialist at eFolder, presented how organizations can use eFolder Cloudfinder to automate backups of cloud application data. He also demonstrated how easy it is to search and restore cloud application data using Cloudfinder.
Two weeks ago (November 3-5), I attended the Cloud Expo Silicon Valley as a speaker, where I presented on the security and privacy due diligence requirements for cloud solutions. Cloud security is a topical issue for every CIO, CISO, and technology buyer. Decision-makers are always looking for insights on how to mitigate the security risks of implementing and using cloud solutions. Based on the presentation topics covered at the conference, as well as the general discussions heard between sessi...
The Internet of Everything is re-shaping technology trends–moving away from “request/response” architecture to an “always-on” Streaming Web where data is in constant motion and secure, reliable communication is an absolute necessity. As more and more THINGS go online, the challenges that developers will need to address will only increase exponentially. In his session at @ThingsExpo, Todd Greene, Founder & CEO of PubNub, exploreed the current state of IoT connectivity and review key trends and t...
As organizations shift towards IT-as-a-service models, the need for managing & protecting data residing across physical, virtual, and now cloud environments grows with it. CommVault can ensure protection & E-Discovery of your data - whether in a private cloud, a Service Provider delivered public cloud, or a hybrid cloud environment – across the heterogeneous enterprise.