The progressive neurodegenerative condition affects around 153,000 people in the UK

Mission Therapeutics has been awarded $5.2m in funding from Parkinson’s UK’s Virtual Biotech programme and the Michael J. Fox Foundation to advance a potential treatment for early-stage Parkinson’s disease (PD).

The funding will help support the company’s ongoing phase 1 programme, which consists of a 28-day dosing of its small drug molecule, MTX325, to assess its safety, tolerability, pharmacokinetic profile and central nervous system penetration in patients living with the neurodegenerative condition.

Affecting around 153,000 people in the UK, PD is a progressive neurological condition that damages parts of the brain over time, causing tremors, slow movement and stiff and inflexible muscles.

Mission’s MTX325 is a potent, selective, small-molecule brain-penetrant USP30 inhibitor that works to protect dopamine-producing neurons by improving mitochondrial quality and function.

Previous research has already shown evidence that a build-up of dysfunctional mitochondria in cells is linked to a variety of diseases, including PD, kidney disease, heart failure, idiopathic pulmonary fibrosis and Duchenne’s muscular dystrophy.

In December, researchers from Cambridge and Harvard University, along with Mission, showed strong experimental evidence that supported MTX325 in PD by targeting USP30 in a study published in Nature Communications.

The phase 1 in-human clinical trial of MTX325 began in March and has already produced positive results. Multiple ascending doses are set to begin in early 2025 to evaluate MTX325 as well as observe its effects on relevant mechanisms and disease biology biomarkers.

Anker Lundemose, chief executive officer, Mission, commented: “This significant grant… underlines the huge potential of MTX325 as a disease-modifying treatment for this terrible neurodegenerative illness [and] represents a major endorsement of our mitophagy strategy in human diseases, including PD.”

The announcement comes less than a week after Herantis Pharma announced €3.6m in funding from Parkinson’s UK’s Virtual Biotech programme and the Michael J. Fox Foundation to finance its early-stage clinical trial to evaluate the tolerability and safety of HER-096 in PD and its ongoing biomarker project.