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Company of the Month: MitoRx

MitoRx Therapeutics are doing ground-breaking work to create the ideal mitochondrial medicine. After forming their company in March 2021, the co-founders Dr Jon Rees (CEO and Co-Founder), Dr Norman Law (CTO, Head of IP and Co-Founder) and Prof Matt Whiteman (Inventor, Co-Founder and CSO) recently secured exclusive rights to the intellectual property (IP) for their innovation, as well as closing an oversubscribed seed investment round. We spoke to Jon about the discovery.

Panning for gold

It’s fair to say that Jon was well-qualified to spot a golden opportunity for improvement in the treatment of rare diseases. Having spent most of his career in and around investment in life sciences, he spent the last five years as a consultant looking for investable rare disease IP for both large investors and pharma companies. When he came across the idea that Prof Matt Whiteman and Dr Roberta Torregrossa were working on at Exeter University, it was at too early a stage for any of his clients to be interested, but it did pique Jon’s curiosity.

“In the words of Prof Hideo Kimura – who discovered the importance of hydrogen sulfide as a signalling molecule in the body – ‘if you are fortunate enough to come across a secret of nature, you should pick it up’. In other words, if you come across a previously unknown phenomenon, you are obligated to take up the torch. When I realised what Matt and Roberta had discovered, it was a bit of an epiphany for me.”

The secret they had found was a potential new method of treatment, with promise across a whole range of degenerative diseases driven by mitochondria not working properly. Mitochondria – found in cells throughout the body – produce 90% of the energy our body needs to function. Medical disorders that cause the mitochondria to malfunction can lead to long-term degenerative diseases, affecting any part of the body, especially the nervous system, muscles, eyes and bone.

A new approach to treatment

The potential medicines they are developing at MitoRx – MTRX-Myo and MTRX-Neuro – aim to reset the mitochondria’s energy state, stopping it from malfunctioning, and thus, halting the progression of disease. In particular, they’re focused on degenerative diseases of the muscle and central nervous system – muscular dystrophies and dementia.

“If it works as well in patients as it does in the lab, it has the potential to completely disrupt the treatment of degenerative disease,” says Jon.

Although rare, diseases such as Duchenne muscular dystrophy – the first focus for the team – have a significant impact on patients. MitoRx believes that a lack of dystrophin – a protein that stabilises muscle cell membranes – leads to mitochondrial dysfunction, which drives the progression of the disease. Progressive muscle degeneration eventually results in the loss of the ability to walk, heart problems and diminished lung function, some patients experience cognitive impairment, learning or behavioural difficulties, and, ultimately, have a much-reduced life expectancy. MitoRx’s proposed treatment will be a daily oral medication, taken from diagnosis (usually around four or five years old), for the rest of the patient’s life. The hope is that this would effectively allow the patient to live their life largely unaffected by the disease. It would be transformative, compared to patients’ current outlook.

“For the last three decades the standard of care for pharmaceutical treatment of Duchenne has been steroids, which have concerning side effects with long term use. The ‘Great Hope’ was that Duchenne would be treatable using gene replacement therapy, but clinical trial results have been disappointing. The results might suggest the key gene in muscular dystrophy is too big and too poorly understood for this strategy. It’s the second longest gene expressed in humans, just small enough to fit inside the capacity of the viral vectors currently used in gene therapy, so it is heavily edited to lop out huge sections in order to make dystrophin. We think that addressing the consequences of the loss of dystrophin is going to be a better strategy than replacing the gene, and that means reversing mitochondrial dysfunction.”

MitoRx’s ‘golden pill’. Because the compounds they are using are so potent, the treatment they are developing requires very little material – which they predict will ultimately lower production costs.


Within the next month, the team will begin testing that promise in preclinical trials, with a view to moving to in-human trials for muscular dystrophy in 2026.

They have recently received promising results from testing in mammalian muscle cells, so the team is feeling buoyant about the potential for success.

Investors see potential

Jon believes the scale of the potential impact of their treatment is the chief reason behind their success in fundraising so far. Earlier this year, they took part in a MedCity Investment Hub pitching event, and received a number of investments through the event. When they closed the investment round, Jon says they turned down as much capital as they accepted.

“I think investors were struck by the potential. Something like an Alzheimer’s diagnosis has a massive impact on patients and their families. If we can halt the degeneration, so that patients and their families don’t have to experience the loss of personal identity and the associated grief of that loss, it would mean such diagnoses would no longer need to be the sentence that they are now. In the case of diseases like Huntington’s and Duchenne, our therapeutic target is not only to halt progression, but to completely prevent onset of the disease.”

Expanding team

Others that have seen potential in the idea include impressive new members of their growing team. These include Glyn Edwards MBE – who joined as Chairman – and Dr Christine Charman – who joined as consultant Chief Development Officer. Both have extensive experience in taking treatments to market, with Glyn having headed up Summit Therapeutics, which took another promising treatment for Duchenne to late-stage clinical trials.

Aside from their impressive in-house team, they also call on a global outsourced team of scientists and advisers. Committed to using the best-in-class, wherever they may be, they are working with people across the UK, North America and Australia.

They are also keen to share material with interested academics working in the field, and are already working with some that have helped make connections with potential patients for future trials.

For an early-stage company, this biotech certainly seems to have caught the eye of investors and pharma companies alike. There’s a hint of more announcements later this year – as they field requests for collaborations – so we’ll just say ‘watch this space’.

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