Mission Therapeutics wins $5.2m for potential Parkinson’s treatment
Cambridge-based Mission is a clinical-stage biotech company developing first-in-class therapeutics targeting mitophagy. MTX325 is described as a potent, selective, small molecule brain-penetrant USP30 inhibitor, which is designed to protect dopamine-producing neurons by improving mitochondrial quality and function.
The funding will support a 28-day dosing of MTX325 in patients with early-stage Parkinson's disease (PD), as part of Mission's ongoing MTX325 Phase I program.
Patient dosing is expected to start early in 2025. The aims are to understand MTX325's safety, tolerability, pharmacokinetic profile and CNS penetration in PD patients, as well as observing effects on relevant mechanisms and disease biology biomarkers.
Mission started its multi-part, adaptive Phase I first-in-human clinical trial of MTX325 in March, beginning with a single ascending dose stage in healthy volunteers. Initial investigations of MTX325's CNS penetration ability in these healthy volunteers have yielded positive results. The multiple ascending dose stage of the study was initiated last month.
Anker Lundemose, Chief Executive Officer, Mission Therapeutics, said: “This significant grant, from two of the world's leading Parkinson's disease organisations, underlines the huge potential of MTX325 as a disease-modifying treatment for this terrible neurodegenerative illness.
“It also represents a major endorsement of our mitophagy strategy in human diseases including PD."
Chief Scientific Officer Dr Paul Thompson, added: “We have already made excellent progress in healthy volunteers with preliminary data from the ongoing clinical trial showing that MTX325 has a good single dose safety profile, pharmacokinetics and CNS penetration.
“We look forward to starting the PD patient part of the trial in the new year, which this generous funding from MJFF and Parkinson's UK is helping to support."
Last December, scientists at Cambridge University, Harvard University and Mission Therapeutics published a key academic paper outlining preclinical research on USP30 and MTX325 in the journal Nature Communications.
The paper detailed knockout mouse model data which, the authors said, provided strong experimental evidence supporting the thesis that MTX325 can modify the course of PD by targeting USP30.
A growing body of scientific evidence has linked a build-up of dysfunctional mitochondria in cells to a range of diseases including Parkinson's, kidney disease, heart failure, idiopathic pulmonary fibrosis (IPF) and Duchenne's muscular dystrophy (DMD).