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British biotech Mission Therapeutics has raised $13.3 million from existing investors to advance its Parkinson’s disease drug candidate MTX325, which targets mitophagy—the process that clears damaged mitochondria. The funding will support a Phase 1b proof-of-mechanism trial in Parkinson’s patients, set to begin in early 2026 at the company’s headquarters on the Babraham Research Campus in Cambridge. This follows promising Phase 1a results in healthy volunteers showing that MTX325 successfully crosses the blood-brain barrier and reaches target tissues.
Mitophagy plays a critical role in maintaining mitochondrial health, essential for energy production and cell survival. In Parkinson’s disease, this process becomes impaired, leading to a buildup of defective mitochondria that drive neuronal damage and loss of dopamine signaling. Mission aims to restore this cellular quality control system by inhibiting USP30, a deubiquitylating enzyme that normally acts as a molecular brake on mitophagy. By removing this brake, MTX325 could help neurons eliminate dysfunctional mitochondria more effectively, potentially slowing or modifying disease progression.
Mission’s preclinical research, published in Nature Communications in 2023, showed that MTX325 improved mitochondrial function, reduced alpha-synuclein buildup, and protected dopamine-producing neurons in Parkinson’s models. The company has already secured approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to start the next trial phase, with proof-of-mechanism data expected by late 2027.
The new financing round was backed by major investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group, and Rosetta Capital, alongside grants from the Michael J. Fox Foundation and Parkinson’s UK. Beyond Parkinson’s, Mission is also developing MTX652, a peripheral USP30 inhibitor for heart and kidney diseases, as part of its broader pipeline tackling mitochondrial dysfunction in conditions such as heart failure, acute kidney injury, Duchenne muscular dystrophy, idiopathic pulmonary fibrosis, and Alzheimer’s disease.
