Washington, D.C. — The Friedreich's Ataxia Research Alliance (FARA) has awarded a $3 million grant to Edison Pharmaceuticals to advance the development of EPI-A0001 for the treatment of Friedreich's ataxia. While continuing to support Friedreich's ataxia research intended to discover potential therapeutic approaches, FARA, with this new grant, demonstrates its additional focus on translating such discoveries into therapeutic initiatives — moving from drug discovery to drug development. FARA's board sees this new step as part of developing a balanced portfolio of both research and development initiatives, and will seek to fund other promising therapeutic programs through a variety of mechanisms.
"The Edison program is compelling in terms of the quality of science, the experience of the team and the promise it shows for treatment in the near term," stated Mr. Ronald Bartek, President of FARA. "FARA's mission is to slow, stop and reverse the damage done by Friedreich's ataxia. This first FARA grant in drug development reflects the fact that we believe scientific research is on the threshold of treatments that will stop this disease in its tracks — even beginning the process of reversal. FARA is proud to have played a role in Edison's kickoff and we are eager to help move its program promptly through the required preclinical stages."
Earlier this year, FARA awarded a $300,000 grant to Edison Pharmaceuticals in the first step of an ongoing collaboration with the Muscular Dystrophy Association (MDA) and Seek A Miracle / MDA, who provided an additional $100,000 to the effort. That initial $400,000 joint grant was intended to initiate the pre-clinical toxicology and pharmacology studies needed to move the most promising Edison drug candidates into clinical trials.
Friedreich's ataxia is an inherited disease. It is caused by a defect in the gene encoding for a protein — frataxin — whose function is essential for biological energy production. In the last 5 years, medical and research communities have shown growing interest in Friedreich's ataxia. This attention is based on a renewed interest in the role that mitochondria play in disease, and an emerging body of literature suggesting that Friedreich's ataxia may share a common mechanism with other neurodegenerative diseases and movement disorders, as well as the aging process.
Today, there is no effective treatment or cure available for Friedreich's ataxia. It often first becomes noticeable as a balance and coordination problem in children of elementary school age. Further loss of strength and coordination in all four extremities usually forces those with the disease into wheelchairs by their teens. It is progressive and can lead to impaired vision, speech and hearing. Friedreich's ataxia is also commonly associated with severe heart disease, spinal abnormalities and diabetes. In the later stages of the disorder, patients are frequently incapacitated with cardiac failure leading to death. Friedreich's ataxia, although in relative terms rare, is the most prevalent inherited ataxia, affecting about 1 in every 50,000 people in the United States.
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's Ataxia. Since its founding in 1998 by Friedreich's ataxia-affected families and scientists, FARA has provided almost $7 million in grants to scientists around the world who are pursuing treatments. http://www.FAResearchAlliance.org
President, Friedreich’s Ataxia Research Alliance