The progressive neurodegenerative disease affects around 153,000 people in the UK.
Mission Therapeutics has received $5.2 million in funding from Parkinson’s UK’s Virtual Biotechnology Programme and the Michael J. Fox Foundation to advance the development of potential treatments for the early stages of Parkinson’s disease (PD).
The funding will be used to support the company’s ongoing Phase 1 program, which involves evaluating the safety, tolerability, pharmacokinetic profile and central nervous system penetration of the small molecule drug MTX325 over a 28-day treatment period in patients with neurodegenerative diseases.
Parkinson’s, which affects around 153,000 people in the UK, is a progressive neurological disease that damages parts of the brain over time, causing tremors, slowed movements and stiff, inflexible muscles.
Mission’s MTX325 is a potent, selective, small molecule, brain-penetrant USP30 inhibitor that protects dopamine-producing neurons by improving mitochondrial quality and function.
Previous studies have already provided evidence that the accumulation of dysfunctional mitochondria within cells is linked to a variety of diseases, including Parkinson’s disease, kidney disease, heart failure, idiopathic pulmonary fibrosis and Duchenne muscular dystrophy.
In December, researchers from Cambridge and Harvard University, together with Mission, published strong experimental evidence supporting the efficacy of MTX325 in PD by targeting USP30. Nature Communications.
Phase 1 human clinical trials of MTX325 began in March and have already shown positive results. Multiple ascending dose studies are expected to begin in early 2025 to evaluate MTX325 and observe the effects on relevant mechanisms and disease biology biomarkers.
Anker Lundemose, CEO of Mission, commented: “This important grant underscores the great potential of MTX325 as a disease-modifying treatment for this devastating neurodegenerative disease and represents a major endorsement of our mitophagy strategy in human diseases, including Parkinson’s.”
The announcement was made by Herantis Pharma Announced The €3.6 million funding from Parkinson’s UK’s Virtual Biotech Programme and the Michael J. Fox Foundation will fund early-stage clinical trials and ongoing biomarker projects to evaluate the tolerability and safety of HER-096 in Parkinson’s disease.
