Mission Therapeutics has dosed the first patient in its Phase 1b MTX325-101 study for Parkinson’s disease at Royal Liverpool University Hospital in the United Kingdom.
The trial is planned to enrol 36 participants with mild to moderate idiopathic Parkinson’s across the UK, Europe and Australia and is designed to establish clinical proof-of-mechanism and assess safety, tolerability, biomarkers and imaging, with a data readout anticipated in late 2027.
The company describes MTX325 as a first-in-class, orally bioavailable, brain-penetrant inhibitor of the mitochondrial deubiquitinating enzyme USP30 intended to improve clearance of dysfunctional mitochondria and slow disease processes.
Mission Therapeutics claims preclinical studies showed increased mitophagy and beneficial effects on alpha-synuclein accumulation and dopaminergic neurons, and that a completed Phase 1a study in healthy volunteers demonstrated high CNS penetration with PET confirmation of brain distribution.
Mission Therapeutics is backed by investors including Pfizer Venture Investments, Sofinnova Partners and Roche Venture Fund, and MTX325 holds composition-of-matter exclusivity through 2041.
