Drug Name: CAT-4001
Company: Catabasis Pharmaceuticals
Location: US-Massachusetts
Drug Type: Small Molecule
Conditions: ALS, Friedriech’s ataxia
Mechanism Type: Anitoxidant
Mechanism: CAT-4001, an activator of Nrf2 and an inhibitor of NF-κB, aims to protect neurons by reducing oxidative stress and inflammation. The drug candidate, initially developed for Friedrich’s ataxia, is a CNS-penetrant linked conjugate of monomethyl fumarate and docosahexaenoic acid which can be enzymatically cleaved upon cellular uptake, enabling release of both small molecules simultaneously. Studies in cellular and animal models have shown that the conjugate is more effective than either compound alone.
U.S. Status for ALS: Preclinical

[1] CAT-4001 improves mitochondrial function in a Friedreich’s ataxia model Reilly, JF et al., 2017 International Ataxia Research Conference.
[2] Catabasis. Pipeline – CAT-4001. Catabasis, 2016. Accessed 9 Mar 2016 from

Last updated February 8th, 2018

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