About

About Rogue MitoTherapeutics Friedreich’s Ataxia and ALS, once considered intractable, are becoming treatable diseases. Mitochondrial failure is a common denominator. Rogue MitoTherapeutics is developing a first-in-class small molecule that delivers glutathione directly into the mitochondrial compartment of these patients, restoring iron-sulfur cluster assembly and bioenergetic function. Mitochondrial glutathione is an essential cofactor for building and maintaining iron-sulfur clusters — metal centers critical for energy production, metabolism, protein synthesis, and genomic stability. When mitochondrial glutathione is depleted, iron-sulfur clusters fail and respiratory chain function collapses. This is documented as an early event in both Friedreich’s Ataxia and ALS, and Rogue’s compound is designed to work on top of existing treatments — including omaveloxolone — by acting at the substrate level inside mitochondria where current drugs do not penetrate. Preclinical characterization includes: • Preservation of mitochondrial ultrastructure and respiratory function in a severe transgenic mouse model, consistent with a disease-modifying mechanism at the level of mitochondrial integrity • Rapid BBB penetration confirmed in mice • Clean preclinical safety across zebrafish developmental toxicology (22 endpoints, nontoxic to 100 µM) and mouse exposure studies • Relevant mechanistic proprietary biomarkers newly discovered in patient tissue By building mitochondrial resilience at its source — restoring the glutathione that mitochondria require to maintain their own machinery — we believe we can do substantially better. Mitochondrial glutathione depletion is a convergent mechanism across neurodegenerative disease. Rogue holds issued patents internationally with additional patents being prosecuted. Rogue is seeking investors and strategic partners for its programs in Friedreich’s Ataxia and ALS. Partnering Objectives Co-development, and investment.