Mission Therapeutics, a biotech company in the early stages of clinical development, focuses on improving cellular and organ health by enhancing mitophagy. The company recently raised $13.3 million to support the clinical development of its main drug candidate, MTX325, which is being tested in a Phase Ib trial to prove its mechanism of action for Parkinson’s disease. This funding was led by existing investors and follows positive results from early human studies.

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Earlier, Mission completed Phase Ia trials that included PET scans, which confirmed that MTX325 reaches active brain tissue in healthy volunteers. The company also received a $5.2 million grant from the Michael J. Fox Foundation and Parkinson’s UK, and has received approval from the UK’s Medicines and Healthcare Products Regulatory Agency to begin the Phase Ib trial.

Dr. Anker Lundemose, Executive Director at Mission Therapeutics, said the new funding allows important steps in testing MTX325 in Parkinson’s patients. He mentioned that this will build on promising preclinical results published in Nature Communications in 2023 and the Phase 1a data. Dr. Sarah Fritchley, Chief Development Officer, added that the Phase Ib trial aims to show clear proof of the drug’s mechanism in patients and further evaluate its safety and how well the body tolerates it. She expects to report proof-of-mechanism data by the second half of 2027.

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Dr. James Summers, Chairman of Mission Therapeutics, is confident about MTX325’s potential as a treatment that may slow or stop the progression of Parkinson’s disease. He said the funding reflects strong support from investors. MTX325 works by blocking USP30, an enzyme involved in mitochondria, which are the powerhouses of cells. By inhibiting USP30, MTX325 increases the ubiquitylation of mitochondria, which helps the body remove unhealthy mitochondria through mitophagy. This process is essential for keeping cells healthy and functioning. Scientists believe that problems with mitochondria, like faulty or accumulating mitochondria, play a role in diseases such as Parkinson’s, heart failure, and kidney disease.

In a study published in Nature Communications, researchers from Cambridge, Harvard, Dundee, and Mission Therapeutics showed that MTX325 helped protect dopamine neurons in animal models exposed to alpha-synuclein. The study also found that it reduced biomarkers like phosphorylated alpha-synuclein and glial activation. A commentary in Nature Reviews Drug Discovery in late 2023 said restoring mitophagy is a promising way to treat Parkinson’s. MTX325 is designed to be a first-in-class, strong, selective, orally available, and brain-penetrant inhibitor of USP30 aimed at improving mitochondrial quality. In preclinical tests, it improved mitochondrial health, reduced alpha-synuclein buildup, and helped neurons survive. Human trials with single and multiple doses have already shown that the drug reaches the central nervous system, and PET scans confirmed it spreads into brain tissue.

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