Reversing mitochondrial dysfunction to arrest decline in muscular dystrophy and neurodegeneration.
MitoRx Therapeutics' first-in-class first-in-target platform has potential to arrest the progression of some of the most challenging diseases, focusing internally on 3 rare indications and partnering on broader indications.
Based in Oxford and Exeter, UK, MitoRx is developing a pipeline of pre-clinical programs to progress to the clinic. Built around the pioneer of mitochondrial-targeted sulfide donor technology, Prof Matt Whiteman PhD, the team is specialized in rare diseases acceleration and IP strategy.
MitoRx Therapeutics' MTRX technology is based on over a decade’s work, and over twenty publications demonstrating pharmacological utility in multiple in vivo models.
Multiple peer-reviewed publications since 2014 have proven beyond a doubt that trans-sulfuration dysfunction is a feature of several progressive rare diseases and broader indications as part of a shared pathophysiology preventing escape from mitochondrial dysfunction.
MitoRx Therapeutics MTRX approach reverses such mitochondrial dysfunction, enabling us to overcome mitochondrial-driven disease states which lead to muscle weakness, muscle wasting, cognitive deficit and neurodegeneration.
- Professor Matt Whiteman PhD, CSO
The ideal mitochondrial medicine mechanism of action should:
• Restore mitochondrial function by modulating key mitochondrial metabolic transactivators which are under-activated in the target disease
• Simultaneously act at epigenetic, transcriptional and post-translational levels
• Be subject to safe enzymatic release within the mitochondria, avoiding rapid, abiotic or systemic release
MitoRx Therapeutics' proprietary technology exploits an endogenous mechanism which mediates sulfide-signaling, having the effect of substrate replenishment in states of trans-sulfuration deficiency omnipresent in several diseases and certain medical conditions, resulting in the restoration of adaptive metabolism.
While sulfide is rapidly metabolised, our products are potent and highly targeted, resulting in small and safe doses.
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MitoRx's pioneering new modality is the first potential medicine to restore sulfide-signaling, which is mediated by persulfidation. Our approach offers the opportunity to reverse mitochondrial dysfunction and halt progression in multiple diseases and medical conditions in which mitochondrial dysfunction drives pathology.
This includes our core internal programs for therapeutic use in DMD, CBS-deficiency, Huntington's disease, but also Alzheimer's disease, Parkinson's disease, sarcopenia, cancer cachexia, skin photoaging, COPD, and IPF. MitoRx is actively evaluating ex vivo out-licensing opportunities and collaborations in organ transplant.
MitoRx Therapeutics' mission is to arrest or profoundly delay progressive diseases featuring secondary mitochondrial dysfunction as the mediator of morbidity and mortality via restoration of sulfide-signaling dependent adaptive metabolism.
Our core team is expert in sulfide-biology, IP strategy, entrepreneurship, rare diseases, pharmacology, pre-clinical and clinical development, as well as partnering and business development.
Glyn was appointed Chairman of MitoRx Therapeutics in February 2022. He is also non-executive director at OxSonics & Orthoson. Glyn previously served as CEO of Summit Therapeutics plc 2012-2020 where he restructured the business, took the company through a NASDAQ listing and took its Duchenne muscular dystrophy (DMD) clinical development program through late-stage trials. Prior to joining Summit, he served as interim CEO of the BioIndustry Association (BIA), and as CEO at Antisoma plc, a biotech company specialising in the development of novel drugs for the treatment of cancer from 1998 to 2011. Glyn was awarded an MBE for his services to the biotechnology industry in 2006.
16 years in industry including business development for Takeda Pharma Rare Diseases, VC deal sourcing for LSP.vc, successfully advising Ducentis BioTherapeutics on fundraising. Tranformative decade as spin-out CEO of leading biotech network OBN/BioTrinity. PKD patient charity trustee. Researched metabolic engineering at Oxford.
Experienced leader in biotech IP, skilled in patent law, technology evaluation and competitive intelligence. Previously Head of IP at Adaptimmune ($ADAP). Pharmacologist with 28 years industry experience who has taken two drugs from bench to market at TopoTarget (now Onxeo), research training at Yale and Cornell.
24 years' experience in accelerating transformational therapies as Senior Global Program Lead for Takeda Rare Diseases, as well as Takeda respiratory, immunology and neuroscience programs. Recent experience as CDO at Izana Bioscience and Program Lead supporting UK Vaccine Task Force and UK Antiviral Task Forces. Comprehensive development experience at Novartis in Respiratory and Immunology from preclinical research leadership to Senior Global Programme Director leading the creation, approval and execution of integrated project plans for both Seebri Breezhaler® and Ultibro Breezhaler® through development to launch in COPD.
David brings over 30 years finance experience to his new role as Finance Director. He has held board positions at a number of venture-backed technology companies and his experience in the industry includes senior finance roles within both AstraZeneca and Evotec.
Professor of Experimental Therapeutics, University of Exeter Medical School. Previous positions include Associate Professor and Assistant Dean, Yong Loo Lin School of Medicine, National University of Singapore (2000 - 2007) and visiting professor (Physiology, 2018). Other visiting professorships / scholar include the University of Christchurch, (New Zealand; 2006) and Siriraj Hospital / Mahidol University (Thailand, 2018). Key to elucidating the role of H2S in vivo and invented the first mitochondrial-targeted sulfide delivery molecules. Grant awards totally £6 m for mitochondrial H2S rescue of disease. > 20 years’ experience of redox biochemistry and pharmacology. 180 + publications, H-index = 80.
Experienced and highly skilled medicinal chemist from within the Whiteman and Wood Labs, specialized in the synthesis and in vitro testing of new mitochondrial-targeted hydrogen sulfide-releasing compounds with over 21 publications already including conference presentations.
Senior Lecturer in Organic Chemistry, University of Exeter. 25 years experience in sulfur heterocyclic chemistry in academia and industry, including GlaxoWellcome Fellow and Tutor in Organic Chemistry, University of Oxford.
Harwell, Oxford, UK