Mission awarded $5.2m for disease-modifying Parkinson’s treatment

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Mission Therapeutics has been awarded $5.2 million from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and Parkinson’s UK.

The funding will help advance Mission’s potential disease-modifying treatment for Parkinson’s disease (PD), MTX325.

MTX325 is a potent, selective, small molecule brain-penetrant USP30 inhibitor, which is designed to protect dopamine-producing neurons by improving mitochondrial quality and function.

The money will support a 28-day dosing of MTX325 in patients with early-stage PD, as part of an ongoing MTX325 Phase I programme. Patient dosing is expected to start early in 2025.

The company previously raised £25.2 ($32.1) million to accelerate clinical trials for MTX325 and MTX652.

Dr Paul Thompson, Chief Scientific Officer, Mission Therapeutics, said: “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.”

Mitochondrial health and Parkinson’s

MTX325 protects dopamine-producing neurons by enhancing a cellular quality control process called mitophagy, in which faulty mitochondria are tagged and then removed. It does this by inhibiting USP30, a deubiquitylating enzyme (DUB) which impedes normal mitophagy.

Katharina Klapper, Director of Clinical Research at MJJF, added: “Mission Therapeutics has made great advances in the understanding of how mitochondrial health can play a pivotal role in the development of PD in recent years. We look forward to seeing the results of the MTX325 trial.”

Last December scientists at Cambridge University, Harvard University and Mission Therapeutics published a key academic paper outlining preclinical research on USP30 and MTX325. 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 PD, kidney disease, heart failure, idiopathic pulmonary fibrosis (IPF) and Duchenne’s muscular dystrophy (DMD).

Diana Spencer, Senior Digital Content Editor, DDW

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