BioSpace Collaborative

Academic/Biomedical Research
News & Jobs
Biotechnology and Pharmaceutical Channel Medical Device and Diagnostics Channel Clinical Research Channel BioSpace Collaborative    Job Seekers:  Register | Login          Employers:  Register | Login  

NEWSLETTERS
Free Newsletters
Archive
My Subscriptions

NEWS
News by Subject
News by Disease
News by Date
PLoS
Search News
Post Your News
JoVE

CAREER NETWORK
Job Seeker Login
Most Recent Jobs
Search Jobs
Post Resume
Career Fairs
Career Resources
For Employers

HOTBEDS
Regional News
US & Canada
  Biotech Bay
  Biotech Beach
  Genetown
  Pharm Country
  BioCapital
  BioMidwest
  Bio NC
  BioForest
  Southern Pharm
  BioCanada East
  US Device
Europe
Asia

DIVERSITY

PROFILES
Company Profiles

INTELLIGENCE
Research Store

INDUSTRY EVENTS
Research Events
Post an Event
RESOURCES
Real Estate
Business Opportunities

 News | News By Subject | News by Disease News By Date | Search News
Get Our Industry eNewsletter FREE email:    
   

Edison Pharmaceuticals, Inc. Announces Initiation of EPI-743 Phase 2B Leigh Syndrome Clinical Trial


11/13/2012 9:39:23 AM

MOUNTAIN VIEW, Calif., Nov. 13, 2012 /PRNewswire/ -- Edison Pharmaceuticals today announced the initiation of a phase 2B study entitled, "A Phase 2B Randomized, Placebo-Controlled, Double-Blind Clinical Trial of EPI-743 in Children with Leigh Syndrome." Four clinical trial sites have been selected in the United States: Lucile Packard Children's Hospital, Stanford University Medical Center Palo Alto, California; Akron Children's Hospital Akron, Ohio; Seattle Children's Hospital Seattle, Washington; and Texas Children's Hospital, Baylor University Houston, Texas.

The trial is a placebo-controlled study lasting six months, and then extending an additional six months, during which all subjects will receive EPI-743. Subjects must be between one and twelve years old, possess genetic confirmation of Leigh syndrome, and meet certain disease severity criteria. More information on the study specifics is available on ClinicalTrials.gov.

The primary endpoint of the clinical trial is the Newcastle Paediatric Mitochondrial Disease Scale. Secondary endpoints include neurologic, muscular, imaging, and biomarker indices. The study design was based on positive phase 2A data recently reported in the journal Molecular Genetics and Metabolism, "EPI-743 reverses the progression of the pediatric mitochondrial disease- genetically defined Leigh Syndrome" 107 (2012) 383-388.

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 death of central nervous system regions responsible for the control of breathing and coordination of movement. Leigh syndrome is a member of a larger family of diseases called mitochondrial disease, which share as a common biochemical mechanism 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. It serves as a cofactor for the novel drug target NADPH quinone oxidase 1 (NQO1). Through a redox-based mechanism, EPI-743 augments endogenous glutathione biosynthesis essential for the control of oxidative stress.

Edison Pharmaceuticals

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

Clinical Study Site Information

Akron Children's Hospital
htonni@chmca.org

Lucile Packard Children's Hospital, Stanford University Medical Center
kconnors@stanford,edu

Seattle Children's Hospital
laurie.guidry@seattlechildrens.org

Texas Children's Hospital, Baylor University
catherine.loffredo@bcm.edu

SOURCE Edison Pharmaceuticals



Read at BioSpace.com

   

ADD TO DEL.ICIO.US    ADD TO DIGG    ADD TO FURL    ADD TO STUMBLEUPON    ADD TO TECHNORATI FAVORITES
 

//-->