Promising Friedrich’s Ataxia Drug Candidate Gains Support of NIH Scientists

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Chondrial Therapeutics recently announced that further development of its lead drug candidate for the treatment of Friedreich’s ataxia, TAT-Frataxin (TAT-FXN), will be aided by researchers with the Therapeutics for Rare and Neglected Diseases (TRND) program, run through the National Institutes of Health (NIH), with the aim of filing an Investigational New Drug (IND) application for the therapy and moving it into clinical testing.

The program is part of the NIH’s National Center for Advancing Translational Sciences (NCATS).

“This milestone event validates the exciting potential of our lead candidate, TAT-Frataxin, for the treatment of Friedreich’s Ataxia. In addition, it opens the door to leverage our proprietary platform for treatment of other mitochondrial Orphan diseases,” Mark Payne, MD, chief scientific officer at Chondrial, said in a news release.

“We are excited about being selected into this prestigious program,” added Chondrial’s CEO, Steven Plump. “It will enable Chondrial to draw upon the diverse drug development talent of the NCATS TRND team and their collaborative network. In addition, it will significantly reduce our development costs and financial risk.”

Chondrial is a clinical candidate stage biotech company established to commercialize a proprietary replacement platform for mitochondrial disease. The company focuses on orphan, or rare, diseases and Friedreich’s ataxia (FA) is its lead target.

Chondrial will be able to rely on NCATS research expertise and resources to enable an IND application to be filed with the U.S. Food and Drug Administration, with the goal of advancing TAT-Frataxin into Phase I clinical trials.

“NCATS seeks to develop new technologies and more efficient paradigms for translation, in the context of important unmet medical needs,” said Anton Simeonov, PhD, the director of NCATS’ Division of Preclinical Innovation. “Through our TRND program, we look forward to collaborating on this project for its potential to produce a treatment to patients in need.”

Ron Bartek, co-founder and founding president of the Friedreich’s Ataxia Research Alliance, also acknowledged the importance of TAT-Frataxin’s development for FA patients. “FARA is thrilled with this exciting new step in the development of TAT-Frataxin as a therapy for Friedreich’s Ataxia,” he said. “FARA has been strongly supportive of this approach since Dr. Payne first brought his work to our attention; we worked closely with him and the National Institutes of Health as he advanced this project through its early pre-clinical stages.

“We were greatly encouraged by the creation of Chondrial and, with this terrific new collaboration with the first-rate team at NCATS/TRND, we are now all the more confident that, together, we will drive TAT-Frataxin forward into the clinic and toward a treatment for our patients,” Bartek added.

FA is a debilitating neurologic disease, often striking in childhood, that leads to loss of the ability to walk or use the arms, and can result in heart failure. TAT-Frataxin, still in the preclinical development stages, aims to eventually treat the disease by replacing the missing frataxin protein that is its cause.