Welcome!

News Feed Item

Edison Announces Initiation of EPI-743 Phase 2B/3 Leigh Syndrome Clinical Trial With Dainippon Sumitomo Pharma Co. Ltd. in Japan

First subject enrolled in pivotal clinical trial

MOUNTAIN VIEW, Calif., March 10, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced the initiation of a phase 2B/3 study entitled, "A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome" to be conducted in conjunction with Dainippon Sumitomo Pharma Co, Ltd. 

The trial is an open-label study lasting six months. To be eligible for the trial, children must have clinical and radiographic evidence of Leigh syndrome with or without genetic confirmation, and meet certain disease severity criteria. At least five subjects will be enrolled. The clinical trial primary endpoint is the Newcastle Paediatric Mitochondrial Disease Scale. Secondary endpoints include neurologic, muscular, and biomarker indices.

The study design agreed to by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and Dainippon Sumitomo Pharma is based upon positive phase 2A data previously reported in Molecular Genetics and Metabolism EPI-743 reverses the progression of the pediatric mitochondrial disease—Genetically defined Leigh Syndrome 107 (2012) 383-388, and on the ongoing phase 2B clinical trial of EPI-743 in Leigh syndrome patients being conducted in the United States.

Leigh Syndrome

Leigh syndrome is an inherited lethal, progressive, predominately pediatric, neuromuscular disorder for which there are no approved treatments. Initially described in 1951, the hallmarks of the disease include bilateral necrosis (death) of central nervous system regions responsible for the control of breathing and other neurologic functions. Leigh syndrome belongs to a large family of disorders identified as "mitochondrial disease." The disorders share as a common biochemical feature defects in cellular energy metabolism.

EPI-743

EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of Leigh syndrome and other inherited mitochondrial diseases. EPI-743 is a member of the para-benzoquinone class of drugs. Through a redox-based mechanism, EPI-743 augments endogenous glutathione biosynthesis – essential for the control of oxidative stress. EPI-743 is in phase 2 clinical development for the treatment of inherited respiratory chain disorders. Double-blind, placebo-controlled trials are ongoing for the following indications: Friedreich's ataxia, Leigh syndrome, Cobalamin C defect, and Undiagnosed Disorders of Oxidation-Reduction.

Edison Pharmaceuticals
Edison Pharmaceuticals is a specialty pharmaceutical company dedicated to developing treatments for children and adults with orphan mitochondrial diseases.

Dainippon Sumitomo Pharma Co., Ltd.
Dainippon Sumitomo Pharma Co., Ltd. (DSP) is a multi-billion dollar, top-ten listed pharmaceutical company in Japan with a diverse portfolio of pharmaceutical products. DSP aims to produce innovative pharmaceutical products in the psychiatry and neurology, which is designated as a key therapeutic area, as well as oncology. DSP has about 7,000 employees worldwide. Additional information about DSP is available through its corporate website at www.ds-pharma.com.

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
In his session at Cloud Expo, Robert Cohen, an economist and senior fellow at the Economic Strategy Institute, provideed economic scenarios that describe how the rapid adoption of software-defined everything including cloud services, SDDC and open networking will change GDP, industry growth, productivity and jobs. This session also included a drill down for several industries such as finance, social media, cloud service providers and pharmaceuticals.
"Dice has been around for the last 20 years. We have been helping tech professionals find new jobs and career opportunities," explained Manish Dixit, VP of Product and Engineering at Dice, in this SYS-CON.tv interview at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
Extracting business value from Internet of Things (IoT) data doesn’t happen overnight. There are several requirements that must be satisfied, including IoT device enablement, data analysis, real-time detection of complex events and automated orchestration of actions. Unfortunately, too many companies fall short in achieving their business goals by implementing incomplete solutions or not focusing on tangible use cases. In his general session at @ThingsExpo, Dave McCarthy, Director of Products...
"ReadyTalk is an audio and web video conferencing provider. We've really come to embrace WebRTC as the platform for our future of technology," explained Dan Cunningham, CTO of ReadyTalk, in this SYS-CON.tv interview at WebRTC Summit at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
@DevOpsSummit 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. @DevOpsSummit at Cloud Expo New York Call for Papers is now open.
Infrastructure is widely available, but who’s managing inbound/outbound traffic? Data is created, stored, and managed online – who is protecting it and how? In his session at 19th Cloud Expo, Jaeson Yoo, SVP of Business Development at Penta Security Systems Inc., discussed how to keep any and all infrastructure clean, safe, and efficient by monitoring and filtering all malicious HTTP/HTTPS traffic at the OSI Layer 7. Stop attacks and web intruders before they can enter your network.
The many IoT deployments around the world are busy integrating smart devices and sensors into their enterprise IT infrastructures. Yet all of this technology – and there are an amazing number of choices – is of no use without the software to gather, communicate, and analyze the new data flows. Without software, there is no IT. In this power panel at @ThingsExpo, moderated by Conference Chair Roger Strukhoff, Dave McCarthy, Director of Products at Bsquare Corporation; Alan Williamson, Principal...
Businesses and business units of all sizes can benefit from cloud computing, but many don't want the cost, performance and security concerns of public cloud nor the complexity of building their own private clouds. Today, some cloud vendors are using artificial intelligence (AI) to simplify cloud deployment and management. In his session at 20th Cloud Expo, Ajay Gulati, Co-founder and CEO of ZeroStack, will discuss how AI can simplify cloud operations. He will cover the following topics: why clou...
Video experiences should be unique and exciting! But that doesn’t mean you need to patch all the pieces yourself. Users demand rich and engaging experiences and new ways to connect with you. But creating robust video applications at scale can be complicated, time-consuming and expensive. In his session at @ThingsExpo, Zohar Babin, Vice President of Platform, Ecosystem and Community at Kaltura, discussed how VPaaS enables you to move fast, creating scalable video experiences that reach your aud...
Rapid innovation, changing business landscapes, and new IT demands force businesses to make changes quickly. In the eyes of many, containers are at the brink of becoming a pervasive technology in enterprise IT to accelerate application delivery. In this presentation, attendees learned about the: The transformation of IT to a DevOps, microservices, and container-based architecture What are containers and how DevOps practices can operate in a container-based environment A demonstration of how ...
Cloud Expo, Inc. has announced today that Andi Mann returns to 'DevOps at Cloud Expo 2017' as Conference Chair The @DevOpsSummit at Cloud Expo will take place on June 6-8, 2017, at the Javits Center in New York City, NY. "DevOps is set to be one of the most profound disruptions to hit IT in decades," said Andi Mann. "It is a natural extension of cloud computing, and I have seen both firsthand and in independent research the fantastic results DevOps delivers. So I am excited to help the great t...
Without lifecycle traceability and visibility across the tool chain, stakeholders from Planning-to-Ops have limited insight and answers to who, what, when, why and how across the DevOps lifecycle. This impacts the ability to deliver high quality software at the needed velocity to drive positive business outcomes. In his general session at @DevOpsSummit at 19th Cloud Expo, Phil Hombledal, Solution Architect at CollabNet, discussed how customers are able to achieve a level of transparency that e...
WebRTC is the future of browser-to-browser communications, and continues to make inroads into the traditional, difficult, plug-in web communications world. The 6th WebRTC Summit continues our tradition of delivering the latest and greatest presentations within the world of WebRTC. Topics include voice calling, video chat, P2P file sharing, and use cases that have already leveraged the power and convenience of WebRTC.
"At ROHA we develop an app called Catcha. It was developed after we spent a year meeting with, talking to, interacting with senior citizens watching them use their smartphones and talking to them about how they use their smartphones so we could get to know their smartphone behavior," explained Dave Woods, Chief Innovation Officer at ROHA, in this SYS-CON.tv interview at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
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...