The Friedreich’s Ataxia Research Alliance (FARA) joins its public and private partners in announcing that a phase I clinical trial of a promising new drug, designated A0001, began dosing on July 11th. This milestone achievement illustrates the power of a new model for advancing therapies, especially in rare diseases.

“FARA believes in the essential nature of public-private partnerships that involve government agencies such as the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), academic investigators, the pharmaceutical industry, and multiple non-profits like FARA and the Muscular Dystrophy Association (MDA),” said FARA President Ron Bartek. “FARA is grateful to its partners that span the spectrum from discovery through clinical development as we work together to advance promising compounds like A0001 toward the goal of approved treatments and cures for Friedreich’s Ataxia (FA) and other neurodegenerative disorders.”


A0001 is a small molecule that has the potential for treating defects in the mitochondrial respiratory chain like those believed to cause significant damage in FA and a number of other neurological and neuromuscular disorders. In April 2006, the FDA granted orphan drug designation status to A0001 for treatment of inherited mitochondrial respiratory chain diseases.

The opening of this clinical trial demonstrates the power of the public-private partnerships FARA has been fortunate to build and bring to bear on the quest for treatments and a cure for FA. FARA began its support of A0001 in 2005 with substantial research grants to and investment in Edison Pharmaceuticals. The MDA, via Seek A Miracle/MDA, joined FARA in co-funding the initial research grant to Edison.

"We all know that drug development is expensive, but this is a wonderful example of how investments can be leveraged by multiple groups to get more therapies into the clinic," says Dr. Sharon Hesterlee, MDA VP for Translational Research.

Later in 2005, Dr. Robert Wilson of the University of Pennsylvania, FARA and Edison joined forces as co-applicants to the National Institutes of Health (NIH) program called RAID (Rapid Access to Intervention Development), designed to help move drugs from the discovery bench to the clinical trial bedside. The application was successful and contract services provided by NIH RAID played an important role in completing the preclinical preparations of A0001 so the molecule could be made ready for the clinical trial.

“This is a great example of how the various stakeholders joined forces to help transform a promising laboratory discovery into a therapeutic that is now in clinical trial,” says Dr. Story Landis, NINDS Director. The NINDS manages the NIH RAID program and is also responsible for Friedreich’s Ataxia research as part of its mission to reduce the burden of neurological disorders. NINDS’ support for the A0001 pre-clinical project consisted of formulation, a genotoxicity study, a dose escalation study, synthesis of radiolabeled drug for ADME and radioautography studies, dose ranging studies in two species, a 90 day GLP toxicity study, and safety pharmacology studies.

The phase I clinical trial just initiated by Penwest Pharmaceuticals will be conducted in healthy volunteers and is designed to evaluate the safety and tolerability of A0001 at various doses, as well as to collect pharmacokinetic data. In addition, the trial is designed to determine if there is a maximum tolerated dose of the drug. This data will be used in subsequent phases of clinical trials in patients with FA and other diseases that involve mitochondrial dysfunction.

For futher information, contact FARA at (703) 426 1576 or This email address is being protected from spambots. You need JavaScript enabled to view it..

About FARA
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.