Edison Pharmaceuticals announces initiation of EPI-743 Phase 2B Leigh Syndrome Clinical Trial

November 13, 2012

Mountain View, California; November 13, 2012. 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.
-end-
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

Edison Information

Edison Pharmaceuticals, Inc.
350 North Bernardo Avenue
Mountain View, CA 94043
info@edisonpharma.com
edisonpharma.com

Edison Pharmaceuticals, Inc.

Related Clinical Trial Articles from Brightsurf:

Clinical trial investigates gabapentin for alcohol use disorder
This randomized clinical trial investigated if gabapentin, a drug often used to treat nerve pain, would be useful in the treatment of patients with alcohol use disorder (problem drinking that becomes severe) and a history of alcohol withdrawal symptoms.

Electrical stimulation helps treat constipation in clinical trial
Electrical stimulation benefited women with constipation in a recent clinical trial published in Alimentary Pharmacology & Therapeutics.

Treatment of migraine pain in randomized clinical trial
Adults experiencing a migraine of moderate or severe severity took the drug ubrogepant or placebo and reported if after two hours they were free of pain and of their most bothersome migraine-associated symptom in this randomized clinical trial.

First entirely digital clinical trial encourages physical activity
As little as a daily ping on your phone can boost physical activity, researchers from the Stanford University School of Medicine and their collaborators report in a new study.

HIV vaccine nears clinical trial following new findings
A promising vaccine that clears an HIV-like virus from monkeys is closer to human testing after a new, weakened version of the vaccine has been shown to provide similar protection as its original version.

Sickle cell drug showing promise in clinical trial
An investigational drug for the treatment of sickle cell disease is showing early promise in clinical trials for impacting biomarkers of the disease in patients, reported UConn School of Medicine researchers.

Meditation goes digital in new clinical trial
Scientists at UC San Francisco have developed a personalized digital meditation training program that significantly improved attention and memory in healthy young adults -- a group already at the peak of brain health -- in just six weeks.

Could blockchain ensure integrity of clinical trial data?
UC San Francisco researchers have created a proof-of-concept method for ensuring the integrity of clinical trials data with blockchain.

Eating crickets can be good for your gut, according to new clinical trial
A new clinical trial shows that consuming crickets can help support the growth of beneficial gut bacteria and that eating crickets is not only safe at high doses but may also reduce inflammation in the body.

Idera Pharmaceuticals presents clinical data from the ILLUMINATE-204 trial of the combination of tilsotolimod and ipilimumab for anti-PD-1 refractory metastatic melanoma at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting
Idera Pharmaceuticals Inc., a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with rare cancers and rare diseases, announced results from the ongoing ILLUMINATE-204 trial investigating tilsotolimod, Idera's intratumorally-delivered Toll-like Receptor 9 agonist, in combination with ipilimumab (Yervoy®).

Read More: Clinical Trial News and Clinical Trial Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.