This site is part of the Informa Connect Division of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC's registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 3099067.

BIO-Europe Spring
Save the dateMarch 17–19, 2025 | Milan, Italy

MitoRx Therapeutics Limited


MitoRx Therapeutics is an Oxford-based first-in-class first-in-mechanism preclinical stage platform biotech developing highly potent small molecule therapeutics that halt progression in degenerative diseases whose pathology is driven by mitochondrial dysfunction. We achieved platform proof-of-concept Q4 2023 in mammalian disease models. Our internal foci are neurodegenerative and neuromuscular disease (NMD). $5m seed financing has been raised since 2022 facilitating platform validation in mammalia and generating derisked front-running leads with the potential to become clinical candidates. MitoRx seeks investors to lead or co-lead an aggressive Series A to take forward up to two programs to early efficacy readouts in the clinic. Pre-clinical results in mammalian disease models demonstrate that our leads are functionally protective in neuromuscular (DMD) and inflammatory disease (COPD, anti-fibrotic, for partnering), and we have an active pre-clinical program in neurodegenerative disease (Huntington’s disease) reporting Q2 2024 pending expansion to broader neurodegenerative disease indications. Previous in vivo results demonstrate our approach is neuroprotective. DMD patient-derived cell-based work demonstrates restoration of mitochondrial ROS to healthy patient levels. MitoRx is the sole biotech globally which has developed technology that restores the functional S-persulfidation post-translational signalling system regulating mitochondrial metabolism whose disruption has been demonstrated in Huntington’s disease, Parkinson’s disease, Alzheimer’s disease, Duchenne muscular dystrophy and other progressive degenerative conditions. MitoRx expects to be the only mitochondrial platform biotech globally to demonstrate utility in neuromuscular, neurodegenerative and inflammatory disease models. Multiple asset classes allow MitoRx to take a flexible approach in interactions with pharma.Summary of Products, Services or TechnologiesMitoRx Therapeutics has developed a number of severable classes of safe, efficacious and highly potent small molecules targeted to the mitochondria where they achieve restoration-of-function of the sulfide-signaling system mediated by S-persulfidation required to reverse pathological mitochondrial dysfunction, required for protective regulation of mitochondrial metabolism. Useful pharmacological activity in neuromuscular, neurodegenerative, lung inflammatory and metabolic disease.