|By PR Newswire||
|March 17, 2014 12:01 AM EDT||
MOUNTAIN VIEW, Calif., March 17, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced the initiation of a phase 2 study entitled "A Phase 2 Safety and Efficacy Study of EPI-743 (Vincerinone™) in Children with Pearson Syndrome." The Investigative New Drug application (IND) was approved by the Food and Drug Administration, Office of Hematology and Oncology Products.
The trial is a subject-controlled study design lasting 12 months in which all subjects will receive EPI-743. The primary endpoint of the study is the incidence of episodes of sepsis, metabolic crisis, and hepatic failure. Secondary endpoints include transfusion avoidance and other disease-relevant endocrine and neurological outcome assessments. Given the rarity of Pearson syndrome, the study is a single arm design, and is being conducted at multiple sites worldwide.
Historically, mitochondrial disease has been described as a group of neuromuscular diseases. This description connotes the energy derangements associated with brain and muscle function. However, today our knowledge of both the molecular and genetic basis of the diseases has significantly grown. It is now established that genetic defects in either nuclear or mitochondrial derived DNA are responsible for a myriad of clinical "mitochondrial" diseases that can affect virtually every organ system. Pearson syndrome is one such example.
Pearson syndrome has an estimated prevalence of less than 1:1,000,000. It is typically diagnosed in infancy, though it can be diagnosed in neonates. Its clinical hallmarks are transfusion-dependent anemia, neutropenia, and pancreatic dysfunction. Given the notable hematologic derangements associated with the syndrome, Edison has submitted the Pearson syndrome IND to the Office of Hematology and Oncology Products. This allows Edison to utilize the division's expertise in the development of drugs for diseases with significant hematological manifestations.
"Pearson syndrome is a very rare mitochondrial disease with a devastating clinical outcome," stated Mathew Klein, MD, FACS, Chief Medical Officer, Edison Pharmaceuticals. "We are working with our clinical investigator team worldwide to accelerate enrollment in this trial and to systematically explore whether EPI-743 can offer benefit for this patient population."
The FDA has previously granted orphan status to EPI-743 for the treatment of inherited respiratory chain diseases, as well as for Friedreich's ataxia. Edison is currently conducting a number of phase 2A and 2B trials in various mitochondrial disease indications. A complete list of ongoing trials can be found on www.clinicaltrials.gov.
Pearson syndrome is an ultra-rare, fatal mitochondrial disease characterized by sideroblastic anemia, pancytopenia and pancreatic dysfunction. Pearson syndrome results from a mitochondrial deletion, spanning a range of mitochondrial genes. Hematologic features are often present at birth and include severe, transfusion-dependent macrocytic anemia as well as neutropenia and thrombocytopenia. There is no treatment for Pearson syndrome and most patients die in infancy or early childhood secondary to metabolic disorders or infections. A subset of patients survive past age three and develop a neurological syndrome– resembling Kearn-Sayre syndrome– characterized by ophthalmologic and neuromuscular impairments.
EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of children and adults with 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, which is 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 is a specialty pharmaceutical company dedicated to developing treatments for children and adults with mitochondrial diseases.
SOURCE Edison Pharmaceuticals
Traditional on-premises data centers have long been the domain of modern data platforms like Apache Hadoop, meaning companies who build their business on public cloud were challenged to run Big Data processing and analytics at scale. But recent advancements in Hadoop performance, security, and most importantly cloud-native integrations, are giving organizations the ability to truly gain value from all their data. In his session at 19th Cloud Expo, David Tishgart, Director of Product Marketing ...
Aug. 30, 2016 05:00 PM EDT Reads: 889
Fact: storage performance problems have only gotten more complicated, as applications not only have become largely virtualized, but also have moved to cloud-based infrastructures. Storage performance in virtualized environments isn’t just about IOPS anymore. Instead, you need to guarantee performance for individual VMs, helping applications maintain performance as the number of VMs continues to go up in real time. In his session at Cloud Expo, Dhiraj Sehgal, Product and Marketing at Tintri, wil...
Aug. 30, 2016 04:15 PM EDT Reads: 954
Internet of @ThingsExpo, taking place November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 19th Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. The Internet of Things (IoT) is the most profound change in personal and enterprise IT since the creation of the Worldwide Web more than 20 years ago. All major researchers estimate there will be tens of billions devices - comp...
Aug. 30, 2016 03:30 PM EDT Reads: 3,766
StarNet Communications Corp has announced the addition of three Secure Remote Desktop modules to its flagship X-Win32 PC X server. The new modules enable X-Win32 to safely tunnel the remote desktops from Linux and Unix servers to the user’s PC over encrypted SSH. Traditionally, users of PC X servers deploy the XDMCP protocol to display remote desktop environments such as the Gnome and KDE desktops on Linux servers and the CDE environment on Solaris Unix machines. XDMCP is used primarily on comp...
Aug. 30, 2016 03:00 PM EDT Reads: 846
SYS-CON Events announced today that StarNet Communications will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. StarNet Communications’ FastX is the industry first cloud-based remote X Windows emulator. Using standard Web browsers (FireFox, Chrome, Safari, etc.) users from around the world gain highly secure access to applications and data hosted on Linux-based servers in a central data center. ...
Aug. 30, 2016 02:30 PM EDT Reads: 948
SYS-CON Events announced today Telecom Reseller has been named “Media Sponsor” of SYS-CON's 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Telecom Reseller reports on Unified Communications, UCaaS, BPaaS for enterprise and SMBs. They report extensively on both customer premises based solutions such as IP-PBX as well as cloud based and hosted platforms.
Aug. 30, 2016 02:30 PM EDT Reads: 1,033
SYS-CON Events announced today that Adobe has been named “Bronze Sponsor” of SYS-CON's 18th Cloud Expo, which will take place on June 7-9, 2016, at the Javits Center in New York, New York. Adobe is changing the world though digital experiences. Adobe helps customers develop and deliver high-impact experiences that differentiate brands, build loyalty, and drive revenue across every screen, including smartphones, computers, tablets and TVs. Adobe content solutions are used daily by millions of co...
Aug. 30, 2016 02:00 PM EDT Reads: 3,759
Why do your mobile transformations need to happen today? Mobile is the strategy that enterprise transformation centers on to drive customer engagement. In his general session at @ThingsExpo, Roger Woods, Director, Mobile Product & Strategy – Adobe Marketing Cloud, covered key IoT and mobile trends that are forcing mobile transformation, key components of a solid mobile strategy and explored how brands are effectively driving mobile change throughout the enterprise.
Aug. 30, 2016 01:45 PM EDT Reads: 646
Pulzze Systems was happy to participate in such a premier event and thankful to be receiving the winning investment and global network support from G-Startup Worldwide. It is an exciting time for Pulzze to showcase the effectiveness of innovative technologies and enable them to make the world smarter and better. The reputable contest is held to identify promising startups around the globe that are assured to change the world through their innovative products and disruptive technologies. There w...
Aug. 30, 2016 01:30 PM EDT Reads: 866
SYS-CON Events announced today that Pulzze Systems will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Pulzze Systems, Inc. provides infrastructure products for the Internet of Things to enable any connected device and system to carry out matched operations without programming. For more information, visit http://www.pulzzesystems.com.
Aug. 30, 2016 01:20 PM EDT Reads: 246
Data is the fuel that drives the machine learning algorithmic engines and ultimately provides the business value. In his session at Cloud Expo, Ed Featherston, a director and senior enterprise architect at Collaborative Consulting, will discuss the key considerations around quality, volume, timeliness, and pedigree that must be dealt with in order to properly fuel that engine.
Aug. 30, 2016 01:15 PM EDT Reads: 2,065
19th Cloud Expo, taking place November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA, will feature technical sessions from a rock star conference faculty and the leading industry players in the world. Cloud computing is now being embraced by a majority of enterprises of all sizes. Yesterday's debate about public vs. private has transformed into the reality of hybrid cloud: a recent survey shows that 74% of enterprises have a hybrid cloud strategy. Meanwhile, 94% of enterpri...
Aug. 30, 2016 01:00 PM EDT Reads: 3,230
SYS-CON Events announced today that Hitrons Solutions will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Hitrons Solutions Inc. is distributor in the North American market for unique products and services of small and medium-size businesses, including cloud services and solutions, SEO marketing platforms, and mobile applications.
Aug. 30, 2016 11:45 AM EDT Reads: 799
Almost two-thirds of companies either have or soon will have IoT as the backbone of their business in 2016. However, IoT is far more complex than most firms expected. How can you not get trapped in the pitfalls? In his session at @ThingsExpo, Tony Shan, a renowned visionary and thought leader, will introduce a holistic method of IoTification, which is the process of IoTifying the existing technology and business models to adopt and leverage IoT. He will drill down to the components in this fra...
Aug. 30, 2016 10:30 AM EDT Reads: 412
SYS-CON Events announced today that eCube Systems, a leading provider of middleware modernization, integration, and management solutions, will exhibit at @DevOpsSummit at 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. eCube Systems offers a family of middleware evolution products and services that maximize return on technology investment by leveraging existing technical equity to meet evolving business needs. ...
Aug. 30, 2016 09:45 AM EDT Reads: 887