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
SYS-CON Events announced today that Ryobi Systems 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. Ryobi Systems Co., Ltd., as an information service company, specialized in business support for local governments and medical industry. We are challenging to achive the precision farming with AI. For more information, visit http:...
As you move to the cloud, your network should be efficient, secure, and easy to manage. An enterprise adopting a hybrid or public cloud needs systems and tools that provide: Agility: ability to deliver applications and services faster, even in complex hybrid environments Easier manageability: enable reliable connectivity with complete oversight as the data center network evolves Greater efficiency: eliminate wasted effort while reducing errors and optimize asset utilization Security: imple...
High-velocity engineering teams are applying not only continuous delivery processes, but also lessons in experimentation from established leaders like Amazon, Netflix, and Facebook. These companies have made experimentation a foundation for their release processes, allowing them to try out major feature releases and redesigns within smaller groups before making them broadly available. In his session at 21st Cloud Expo, Brian Lucas, Senior Staff Engineer at Optimizely, will discuss how by using...
The next XaaS is CICDaaS. Why? Because CICD saves developers a huge amount of time. CD is an especially great option for projects that require multiple and frequent contributions to be integrated. But… securing CICD best practices is an emerging, essential, yet little understood practice for DevOps teams and their Cloud Service Providers. The only way to get CICD to work in a highly secure environment takes collaboration, patience and persistence. Building CICD in the cloud requires rigorous ar...
Transforming cloud-based data into a reportable format can be a very expensive, time-intensive and complex operation. As a SaaS platform with more than 30 million global users, Cornerstone OnDemand’s challenge was to create a scalable solution that would improve the time it took customers to access their user data. Our Real-Time Data Warehouse (RTDW) process vastly reduced data time-to-availability from 24 hours to just 10 minutes. In his session at 21st Cloud Expo, Mark Goldin, Chief Technolo...
In this strange new world where more and more power is drawn from business technology, companies are effectively straddling two paths on the road to innovation and transformation into digital enterprises. The first path is the heritage trail – with “legacy” technology forming the background. Here, extant technologies are transformed by core IT teams to provide more API-driven approaches. Legacy systems can restrict companies that are transitioning into digital enterprises. To truly become a lead...
SYS-CON Events announced today that CAST Software 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. CAST was founded more than 25 years ago to make the invisible visible. Built around the idea that even the best analytics on the market still leave blind spots for technical teams looking to deliver better software and prevent outages, CAST provides the software intelligence that matter ...
SYS-CON Events announced today that Daiya Industry will exhibit at the Japanese 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. Ruby Development Inc. builds new services in short period of time and provides a continuous support of those services based on Ruby on Rails. For more information, please visit https://github.com/RubyDevInc.
When it comes to cloud computing, the ability to turn massive amounts of compute cores on and off on demand sounds attractive to IT staff, who need to manage peaks and valleys in user activity. With cloud bursting, the majority of the data can stay on premises while tapping into compute from public cloud providers, reducing risk and minimizing need to move large files. In his session at 18th Cloud Expo, Scott Jeschonek, Director of Product Management at Avere Systems, discussed the IT and busine...
Is advanced scheduling in Kubernetes achievable? Yes, however, how do you properly accommodate every real-life scenario that a Kubernetes user might encounter? How do you leverage advanced scheduling techniques to shape and describe each scenario in easy-to-use rules and configurations? In his session at @DevOpsSummit at 21st Cloud Expo, Oleg Chunikhin, CTO at Kublr, will answer these questions and demonstrate techniques for implementing advanced scheduling. For example, using spot instances ...
As businesses evolve, they need technology that is simple to help them succeed today and flexible enough to help them build for tomorrow. Chrome is fit for the workplace of the future — providing a secure, consistent user experience across a range of devices that can be used anywhere. In her session at 21st Cloud Expo, Vidya Nagarajan, a Senior Product Manager at Google, will take a look at various options as to how ChromeOS can be leveraged to interact with people on the devices, and formats th...
First generation hyperconverged solutions have taken the data center by storm, rapidly proliferating in pockets everywhere to provide further consolidation of floor space and workloads. These first generation solutions are not without challenges, however. In his session at 21st Cloud Expo, Wes Talbert, a Principal Architect and results-driven enterprise sales leader at NetApp, will discuss how the HCI solution of tomorrow will integrate with the public cloud to deliver a quality hybrid cloud e...
SYS-CON Events announced today that Yuasa System 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. Yuasa System is introducing a multi-purpose endurance testing system for flexible displays, OLED devices, flexible substrates, flat cables, and films in smartphones, wearables, automobiles, and healthcare.
Companies are harnessing data in ways we once associated with science fiction. Analysts have access to a plethora of visualization and reporting tools, but considering the vast amount of data businesses collect and limitations of CPUs, end users are forced to design their structures and systems with limitations. Until now. As the cloud toolkit to analyze data has evolved, GPUs have stepped in to massively parallel SQL, visualization and machine learning.
The session is centered around the tracing of systems on cloud using technologies like ebpf. The goal is to talk about what this technology is all about and what purpose it serves. In his session at 21st Cloud Expo, Shashank Jain, Development Architect at SAP, will touch upon concepts of observability in the cloud and also some of the challenges we have. Generally most cloud-based monitoring tools capture details at a very granular level. To troubleshoot problems this might not be good enough.