The international study will examine whether MTX325 affects processes linked to Parkinson’s disease, as well as assess its safety and tolerability.
As reported in a recent press release, Mission Therapeutics says it has given the first participant with Parkinson’s disease a dose of MTX325, an experimental medicine being studied as a possible way to affect the underlying disease—not just manage symptoms.
Parkinson’s is a progressive neurological condition. It affects brain cells involved in producing dopamine, a chemical important for controlling movement. Symptoms vary, but may include tremor, stiffness, slowed movement and balance problems. Some people also experience non-movement symptoms, such as sleep, mood or thinking changes. Available treatments can help manage symptoms, but the effects and needs differ from person to person.
What is MTX325 designed to do?
MTX325 is an oral medicine designed to reach the brain and block an enzyme called USP30. The company is investigating whether blocking USP30 can help cells clear damaged mitochondria—the structures that supply energy to cells.
This process, known as mitophagy, is one way cells remove worn-out mitochondria. Researchers believe problems with cell energy and waste-clearance systems may be involved in Parkinson’s, but the role of these processes in the disease is still being studied.
In laboratory and other preclinical research, MTX325 affected mitophagy and measures related to alpha-synuclein, a protein associated with Parkinson’s, as well as dopamine-producing brain cells. These findings helped motivate testing in people, but they do not show that MTX325 will slow or stop Parkinson’s in patients.
What will the study examine?
The Phase 1b MTX325-101 study plans to enroll 36 people with mild to moderate Parkinson’s in the United Kingdom, other parts of Europe and Australia. Researchers will assess safety and tolerability and use biological tests and imaging to look for signs that the drug is affecting its intended processes—a goal called demonstrating “proof of mechanism.”
The study follows a completed Phase 1a trial in healthy volunteers. Mission reports that the earlier study found MTX325 reached the central nervous system, and a PET imaging study showed it distributed into brain tissue. These results do not establish whether the medicine benefits people with Parkinson’s.
Recruitment is underway, and the company expects to report data in late 2027.
Dr. Sarah Fritchley, Mission Therapeutics’ chief development officer, called the first participant’s treatment an important milestone. The company’s chief medical officer, Dr. Suhail Nurbhai, said MTX325 could potentially offer an approach that goes beyond symptom management if future studies show it can change the course of the disease.
For now, MTX325 remains experimental. This Phase 1b study is designed to learn more about its effects and safety—not to establish that it is an effective treatment. Larger and later-stage trials would be needed to determine whether it can help people with Parkinson’s.
