By enhancing mitochondrial quality control, company aims to improve neuronal health and modify the course of neurodegeneration.
British biotech Mission Therapeutics has secured $13.3 million in new funding to advance its mitophagy-targeting Parkinson’s disease candidate, MTX325, through clinical development. The financing, led by the company’s existing investors, will enable Mission to complete a Phase 1b proof-of-mechanism study in patients with Parkinson’s disease, set to begin in the first half of 2026.
Headquartered at the Babraham Research Campus in Cambridge, Mission specializes in developing therapeutics designed to improve cell and organ health by enhancing mitophagy, the cellular process responsible for clearing damaged mitochondria. Often described as the powerhouses of our cells, mitochondria generate around 90 percent of the energy required for normal cellular function. Each cell contains thousands of these tiny organelles, which also play crucial roles in regulating metabolism, stress responses, and cell survival.
Mitophagy and Parkinson’s
When mitochondria become damaged or dysfunctional, they can no longer produce energy efficiently and begin releasing harmful reactive oxygen species that drive inflammation and cell death. To prevent this, cells rely on the mitophagy process to identify and remove impaired mitochondria before they cause harm. In Parkinson’s disease, evidence suggests that this quality-control process becomes disrupted, leading to the accumulation of defective mitochondria within neurons. Over time, this contributes to the loss of dopamine signaling and the progressive motor and cognitive symptoms that characterize the disease. Restoring or enhancing mitophagy is regarded as a promising strategy for slowing or potentially modifying the course of Parkinson’s disease.
Mission’s research focuses on targeting deubiquitylating enzymes (DUBs), a family of around 100 human proteins that regulate the ubiquitin pathway – a process controlling protein stability, location and turnover within cells. By selectively inhibiting these enzymes, Mission’s compounds seek to restore cellular homeostasis and energy balance, addressing underlying mechanisms common to multiple diseases.
MTX325 is an inhibitor of USP30, a DUB that plays a central role in regulating mitophagy. Mission is betting that inhibiting USP30 removes a molecular brake on mitophagy, enabling cells to eliminate dysfunctional mitochondria more effectively. By enhancing mitochondrial quality control, MTX325 aims to improve neuronal health and potentially modify the course of Parkinson’s disease, where mitochondrial dysfunction is thought to be a major driver of neurodegeneration.
Trial data expected in 2027
The funding follows the successful completion of Phase 1a studies of MTX325 in healthy volunteers, including data that confirmed the compound adequately penetrates functional brain tissue. These early results, combined with cerebrospinal fluid sampling showing high central nervous system penetration, supports the compound’s potential to achieve its intended biological activity in the brain.
Mission’s exec director Dr Anker Lundemose said the new funding “will enable us to build upon the compelling preclinical data package for MTX325, published in Nature Communications in 2023, and the results from the Phase 1a studies that we have obtained.”
Mission’s preclinical studies have shown that MTX325 impacts several key mechanisms implicated in Parkinson’s disease, including mitochondrial dysfunction, alpha-synuclein accumulation, and loss of nigrostriatal dopaminergic neurons. The company says it has already obtained approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to initiate the Phase 1b study.
“The overall objectives of this Phase 1b trial are to demonstrate robust clinical proof-of-mechanism (PoM) in patients with Parkinson’s disease, and to gather further information on safety and tolerability,” said Mission’s Chief Development Officer, Dr Sarah J Fritchley. “We look forward to progressing MTX325 rapidly through clinical testing and anticipate we will have PoM data in H2 2027.”
Mission’s investors include Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital. The development of MTX325 was also supported by a $5.2 million grant from the Michael J. Fox Foundation and Parkinson’s UK.
Alongside MTX325, which is being developed for central nervous system disorders, the company is advancing MTX652, a peripheral USP30 inhibitor aimed at diseases outside the brain, such as kidney and heart conditions. Both candidates are part of Mission’s broader pipeline targeting mitochondrial dysfunction in serious illnesses including acute kidney injury, heart failure, Duchenne muscular dystrophy, idiopathic pulmonary fibrosis and Alzheimer’s disease.
