Potential Friedreich’s Ataxia Therapy Advances with FARA’s Support

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Catabasis Pharmaceuticals, Inc., recently announced that it will receive the Kyle Bryant Translational Research Award, given by the Friedreich’s Ataxia Research Alliance (FARA), to further evaluate CAT-4001, a potential Friedreich’s ataxia (FA) drug.

CAT-4001 is being developed by Catabasis as a potential treatment for neurodegenerative diseases, such as FA and amyotrophic lateral sclerosis (ALS). CAT-4001 is a small molecule that activates Nrf2 and inhibits NF-kB, two pathways that have been implicated in FA and ALS. Previous studies have shown that CAT-4001 effectively modulates both pathways in animal models and cellular assays.

The two-year award will be used for the “Evaluation of CAT-4001 in Frataxin-deficient mouse models and dorsal root ganglia neurons to enable its therapeutic development for Friedreich’s ataxia,” an animal model study to be led by Dr. Andrew Nichols of Catabasis, together with colleagues Dr. Mark Payne of Indiana University, and Dr. Jordi Magrane of Weill Cornell College of Medicine.

“We are excited to support this research as it brings a potential new candidate to the FA treatment pipeline, and the research development team advancing the work is an example of FARA’s private public partnership strategy. We believe that we make the most meaningful research advancements when FARA, our academic investigators and our pharmaceutical partners take a collaborative approach to answering research questions,” Jennifer Farmer, FARA’s executive director, said in a news release.

The Kyle Bryant Translational Research Award — named to honor the initiative of a young man diagnosed with FA in his early teens who started a cycling event, rideATAXIA, in 2007 to promote and support research — specifically focuses on preclinical and clinical studies into potential FA treatments.

“We greatly appreciate the support of the Friedreich’s Ataxia Research Alliance as we advance the preclinical development of CAT-4001 as a candidate to treat FA,” said Andrew Nichols, PhD, senior vice president at Catabasis. “We are sensitive to the unmet medical need for patients affected by Friedreich’s ataxia and believe partnerships such as this could make a meaningful difference in discovering and developing therapies for them.”

The award’s terms were not announced.