CAT-4001 is a treatment for Friedreich’s ataxia and amyotrophic lateral sclerosis that is under development at Catabasis Pharmaceuticals.

Friedreich’s ataxia is a rare genetic disease caused by FXN gene mutations. The abnormalities lead to reduced production of the frataxin protein, which is crucial to the functioning of mitochondria, cell components that generate energy.

Symptoms of FA include impaired muscle coordination, loss of muscle strength and sensation, slurred speech, and impaired vision and hearing. The disease worsens over time.

There are no effective treatments for FA, although a lot of potential therapies are in the pipeline. CAT-4001 is still in early stages of development.

How CAT-4001 works

CAT-4001 activates a protein called Nrf2 that reduces inflammation and oxidative stress. It also inhibits the NFkB protein, which plays a role in inflammation.

CAT-4001 consists of monomethyl fumarate and docosahexaenoic acid, or DHA. Catabasis designed it not only to reduce inflammation and oxidative stress but also to restore mitochondrial function.

Studies in cell cultures and animals have shown CAT-4001 to be more effective than either of its components in addressing underlying problems of FA. This is true whether each component is used by itself or in combinations with other therapies.

CAT-4001 in clinical trials

CAT-4001 has yet to be tested in humans, but the results of animal studies have been encouraging.

In 2015, Catabasis received funding from the Friedreich’s Ataxia Research Alliance to test CAT-4001 in cell cultures and animal models of FA.

The grant, the Kyle Bryant Translational Research Award, was named for a man who was diagnosed with FA in his early teens. It goes to researchers whose work can lead to new treatments.


Note: Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Friedreich’s Ataxia News, or its parent company, Bionews Services, and are intended to spark discussion about issues pertaining to Friedreich’s Ataxia.