First patient dosed in clinical trial of oral treatment for Parkinson’s
Study testing Mission Therapeutics' therapy recruiting in several locations
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- The first patient was dosed in a Phase 1b clinical trial testing MTX325, an oral treatment for Parkinson’s.
- The trial is still recruiting participants at several locations.
- MTX325 aims to be a disease-modifying therapy by improving mitochondrial quality control through the blocking of the USP30 enzyme.
The first patient has been dosed in Mission Therapeutics’ Phase 1b clinical trial of MTX325, an oral treatment for Parkinson’s disease.
With several locations open in the U.K., France, Germany, and Australia, the Phase 1b clinical trial (NCT07630545) is still recruiting up to 36 adults aged 40-75 with mild to moderate idiopathic (of unknown cause) Parkinson’s.
The first patient was dosed at the Royal Liverpool University Hospital in the U.K. Initial data are expected in 2027.
The start of this part of the trial follows the successful completion of an earlier part conducted in healthy volunteers, which showed that the treatment was generally safe and could reach functional brain tissue.
“Dosing the first Parkinson’s disease patient is a significant milestone in the development of MTX325, a potential disease-modifying treatment for this serious neurodegenerative disease,” Sarah Fritchley, PhD, Mission’s chief development officer, said in a company press release.
Treatment aims for ‘mitochondrial quality control’
Parkinson’s is caused by the progressive loss of nerve cells that produce dopamine, a brain chemical messenger involved in motor control. While the exact factors that trigger the loss of these neurons are not fully known, damage to mitochondria, the cells’ powerhouses, is believed to play a role.
MTX325 aims to promote mitophagy, a quality-control process that allows cells to clear damaged or dysfunctional mitochondria. It does so by blocking USP30 activity, an enzyme that suppresses mitophagy.
“By improving mitochondrial quality control, MTX325 has the potential to change the course of treatment for patients with Parkinson’s disease – moving beyond management of symptoms offered by current therapies,” said Suhail Nurbhai, Mission’s chief medical officer.
In preclinical studies, MTX325 was shown to enter the central nervous system (CNS, the brain and spinal cord) and to boost mitophagy. It has also been shown to increase the number of dopaminergic neurons and dopamine levels, and to reduce a form of the alpha-synuclein protein that is more prone to clumping. Toxic clumping of alpha-synuclein is a hallmark of Parkinson’s, and is also thought to contribute to nerve cell death.
The Phase 1 trial is assessing MTX325’s safety, tolerability, pharmacokinetics (how it moves through the body), and ability to penetrate the CNS in both healthy volunteers and people with Parkinson’s.
In Phase 1b, people with Parkinson’s will receive 20 mg capsules of MTX325, or a placebo, for 28 days. The main goals are to assess MTX325’s safety, tolerability, pharmacokinetic profile, and CNS penetration in people with Parkinson’s, as well as effects on relevant mechanisms and disease biomarkers.
The trial is supported by $13.3 million from current investors and a $5.2 million grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and Parkinson’s UK.
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