$5.2M grant to help test MTX325, protecting neurons, in patients

Mission Therapeutics' potential disease-modifying therapy now in Phase 1 trial

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

Share this article:

Share article via email
A clipboard holds a checklist that reads

Mission Therapeutics has received $5.2 million from The Michael J. Fox Foundation (MJFF) and Parkinson’s UK to start clinical testing MTX325, its experimental and neuron-protective oral therapy, in people in the early stages of Parkinson’s disease.

“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,” Anker Lundemose, Mission’s CEO, said in a company press release.

Specifically, the funds will help to support moving MTX325 into patient testing within the company’s Phase 1 clinical trial program, a multipart U.K. study investigating the therapy’s safety and its properties at single and multiple ascending doses in healthy adults and those with mild to moderate Parkinson’s.

Enrolled patients, ages 18 to 75, will be treated with MTX325 for 28 days, with dosing expected to begin in 2025.

Recommended Reading
A collection of neurons, or nerve cells, is shown.

Proteins pair to help alpha-synuclein spread in brain: Study

Oral MTX325 showing safety, good properties in healthy adults

A first group of healthy adults have finished taking single doses of MTX325, and Mission reports that the therapy showed a good safety and pharmacokinetics (how compounds move through the body) profile in these people, and confirmed its ability to reach the central nervous system (CNS, brain and spinal cord).

The administration of multiple ascending doses in another healthy adult group is underway, the company stated.

“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 [Parkinson’s disease] patient part of the trial in the new year, which this generous funding from MJFF and Parkinson’s UK is helping to support,” said Paul Thompson, PhD, Mission’s chief scientific officer.

Parkinson’s is caused by the death of dopaminergic neurons, specialized nerve cells that are responsible for making dopamine, a major brain chemical messenger involved in motor control. Damage to mitochondria, the cells’ powerhouses, is believed to contribute to the death of dopaminergic neurons in Parkinson’s patients.

Therapy aims to protect neurons by promoting mitochondrial health

MTX325 is designed to boost a cellular quality-control process called mitophagy, which allows cells to tag and clear damaged mitochondria. The therapy works by blocking the activity of USP30, a protein that impedes mitophagy. MTX325 has shown an ability to penetrate the CNS and reach dopamine-producing nerve cells, where it exerts its neuroprotective effects by allowing for more and better functioning mitochondria.

“We now know that mitochondria play a crucial role in the development of Parkinson’s, so addressing mitochondrial problems could have far-reaching benefits for those living with the condition,” said Arthur Roach, director of the Parkinson’s Virtual Biotech at Parkinson’s UK.

“We are delighted to be working with Mission Therapeutics to help fund this vital, UK-based clinical trial. Disease-modifying treatments are one of the great hopes of people with Parkinson’s,” Roach added.

Divided in five parts, the Phase 1 trial expects to enroll up to 158 adults both with and without Parkinson’s. Its main aims are to assess MTX325’s safety, tolerability, and pharmacokinetics, as well as its ability to penetrate the CNS. Therapy effects on disease-related mechanisms and biomarkers also will be evaluated.

“Mission Therapeutics has made great advances in the understanding of how mitochondrial health can play a pivotal role in the development of Parkinson’s disease in recent years. We look forward to seeing the results of the MTX325 trial,” said Katharina Klapper, director of clinical research at MJJF.