$5.2M to advance trial of MTX325, disease-modifying therapy for… – Parkinson’s News Today

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

This significant grant, from two of the worlds leading Parkinsons disease organisations, underlines the huge potential of MTX325 as a disease-modifying treatment for this terrible neurodegenerative illness, Anker Lundemose, Missions CEO, said in a company press release.

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

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

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

Parkinsons 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 Parkinsons patients.

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 Parkinsons, so addressing mitochondrial problems could have far-reaching benefits for those living with the condition, said Arthur Roach, director of the Parkinsons Virtual Biotech at Parkinsons 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 Parkinsons, Roach added.

Divided in five parts, the Phase 1 trial expects to enroll up to 158 adults both with and without Parkinsons. Its main aims are to assess MTX325s 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 Parkinsons disease in recent years.We look forward to seeing the results of the MTX325 trial, said Katharina Klapper, director of clinical research at MJJF.

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$5.2M to advance trial of MTX325, disease-modifying therapy for... - Parkinson's News Today