EPI743

Learn about Edison's new drug emergency treatment protocol for mitochondrial disease

June 2011: Exciting news has been shared with our Mito community about EPI-743, a new drug that is being investigated for use in mitochondrial diseases. While research has been growing over the last five years in the field of mitochondrial medicine, EPI-743 is the first drug of its kind to be used as part of an FDA-approved emergency treatment protocol.

What is EPI-743, and how does it work?
Is EPI-743 FDA approved, and what does that mean?
Who has EPI-743 been used in already?
• What do the preliminary data from the treatment protocol suggest?
Who can take EPI-743 and how?
What will happen over the next year with this drug trial?

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ClinicalTrials.Gov Inclusion Criteria: EPI-743 for Mitochondrial Respiratory Chain Diseases

About Dr. Greg Enns:

Dr. Enns is an Associate Professor of Pediatrics and has been Director of the Biochemical Genetics Program at Stanford University since 1998.  He was trained in clinical genetics and clinical biochemical genetics at the University of California, San Francisco, graduating from the program in 1998.  Dr. Enns has also directed the California Department of Health Services Newborn Screening Area Service Center for Northern California since 2003.  He is on the Board of Directors of the Society for Inherited Metabolic Disease and the Scientific and Medical Advisory Board of the United Mitochondrial Disease Foundation.  As a clinician, he cares for patients who have a broad range of metabolic disorders and focuses on diagnosing and managing those with mitochondrial disorders, urea cycle defects, aminoacidemias, organic acidemias, and lysosomal storage disorders.  His current research involves the development of a panel of sensitive blood biomarkers of redox imbalance, using tandem mass spectrometry and Hi-D FACS, so that patients who have mitochondrial dysfunction can be detected and monitored non-invasively.  He is most interested in developing clinical trials for patients with metabolic disorders, and has a particular interest in novel therapeutics for mitochondrial disease.   

About Dr. Guy Miller:

Guy Miller, MD, PhD is the founder of Edison and has served as Chairman and Chief Executive Officer of its Board since the company’s inception in 2005. Prior to this, he founded Galileo Pharmaceuticals, a biopharmaceutical company, and was its Chairman and CEO from 1995-2005.

Dr. Miller holds an MD from the Medical College of Pennsylvania and a PhD in chemistry from the University of Virginia. He completed his surgical internship at the University of Chicago, and a residency in anesthesiology and critical care medicine at Johns Hopkins. Dr. Miller completed a fellowship in multidisciplinary critical care medicine at Johns Hopkins, where he was on the faculty as an Assistant Professor until 1996. He is currently an attending physician in medical-surgical critical care medicine at Stanford University-PAVAMC.

He also serves as Executive Chairman of Ampere Life Sciences, a spinout of Edison focused on antioxidants and consumer products.

About Edison Pharma:

Edison Pharmaceuticals, Inc. was founded with one goal: to develop a clinically meaningful treatment for inherited mitochondrial disease. Mitochondrial disease is most often diagnosed in childhood, is highly debilitating and often results in early death. When mitochondrial disease progresses to adulthood, it is significantly disabling and also life-shortening.

The term inherited mitochondrial disease actually represents a family of diseases. While they are known by a variety of names, they share a common feature—a defect in how energy is made and regulated. Today there are over 2,000 known genetic defects that cause mitochondrial disease. There are no approved drugs to treat mitochondrial disease.

In 2005 patient families, physicians, scientists, and foundations joined together around a single mission: to build a company that would translate promising laboratory findings into clinically meaningful drugs. Since the beginning of operations in late 2006 Edison has placed three new drugs into clinical development. EPI-743 is now in phase 2B/3 pivotal clinical trials for inherited mitochondrial disease. EPI-A0001 has now recently completed a phase 2A study in the mitochondrial disease Friedreich's ataxia where it has improved neurological outcome measures.

To accomplish its mission, Edison built a discovery and translational research platform that centers on the regulation of energy metabolism, and its clinical measurement. The company holds over 100 patents that span cell biology, chemistry, and methods for drug discovery.

Edison Pharmaceuticals, Inc. is headquartered in Mountain View, California and has European offices in The Netherlands. In Mountain View, the company occupies 15,000 square feet of state-of-the-art research and development space.

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EPI-743 Investigator Team to Report on Edison Mitochondrial Drug

 FDA allows broadened diagnostic criteria for expanded access trial enrollment

 Boston, MA.  June 8, 2011 - Edison Pharmaceuticals announced today that enrollment criteria for the EPI-743 Expanded Access Protocol EPI-2009-1 has been amended and will now include patients with genetic diagnosis and those that meet specific clinical criteria, absent genetic confirmation, are eligible. Details on inclusion criteria can be found at clinicaltrials.gov.

EPI-743 is an experimental drug being developed by Edison for inherited mitochondrial disease. Since June 2010, the Unites States Food and Drug Administration (FDA) has allowed investigators to treat patients under Edison’s Expanded Access Investigational New Drug (IND) application 107,401.  Enrollment had been restricted to use in subjects with genetically confirmed inherited respiratory chain diseases of the mitochondria. The modified expanded access protocol now includes patients that meet predetermined clinical criteria for mitochondria disease and where a discrete genetic defect is not identifiable.

Edison CEO Dr. Guy Miller says, “This is a first step in providing patients clinically diagnosed with mitochondrial disease, absent genetically confirmed etiology, broader access to EPI-743. We are grateful to the US and European EPI-743 Investigator Team for their hard work, dedication and commitment.”

A first report on EPI-743 data obtained under the Expanded Access IND will be presented at the United Mitochondrial Disease Foundation’s scientific meeting this month in Chicago in a session chaired by principal investigator Gregory M. Enns, MB, ChB (Lucile Packard Children’s Hospital, Stanford University Medical Center) and Bruce H. Cohen, MD, FAAN (Akron Children’s Hospital).  A separate abstract was recently presented on EPI-743 and Leber’s hereditary optic neuropathy at the American Ophthalmology Society Meeting in May of this year.

MitoAction would like to applaud the worldwide EPI-743 Investigator Team and the vision of Edison Pharmaceuticals on behalf of our children, their families, and the adult mitochondrial patient community.  We offer our support and appreciation of their work to develop life-changing treatments for mitochondrial disease.

ADDITIONAL LINKS & READING:

ClinicalTrials.Gov Study Details: EPI-743 for Mitochondrial Respiratory Chain Diseases

Edison Pharmaceuticals Announces Results of EPI-A0001 Phase 2A Double Blind Placebo Controlled 28-day Clinical Trial in the Mitochondrial Disease– Friedreich’s Ataxia

Preliminary Report on Initial Subjects Diagnosed With Genetically Confirmed
Mitochondrial Disease at End-Of-Life Treated With EPI-743 Under FDA-Approved
Expanded Access Protocol EPI-2009-1 (UMDF 2011 Symposium Abstract)

Edison Pharmaceuticals to Provide Expanded Access to EPI-743 for Mitochondrial Disease