Mission Therapeutics starts landmark trial of Parkinson's treatment


Mission Therapeutics, which has just raised £25.2 million growth capital, reveals that it has started trials of a key treatment for Parkinson’s disease (PD). Based at Babraham Research Campus, the clinical-stage biotech is developing first-in-class therapeutics targeting mitophagy.

It today reveals the start of a Phase I first-in-human clinical trial of MTX325, its potential disease-modifying treatment for PD.

Around 10 million people suffer from Parkinson's worldwide, including almost one million in the US and around 1.2 million in Europe – numbers that are set to increase as populations age.

Mission has now completed dosing the first cohort of healthy volunteers in a multi-part, adaptive Phase I study evaluating safety, tolerability, pharmacokinetics and brain penetration of MTX325.

Single ascending, multiple dose ascending and elderly healthy volunteer cohorts are planned in 2024, whereas Parkinson's Disease patients will be the focus of the trial in 2025.

Chief Scientific Officer Dr Paul Thompson, said: “The launch of this first-in-human trial is a significant step forward for Mission Therapeutics as we look to assess the potential of MTX325 as a disease-modifying treatment for Parkinson's disease.

“While existing treatments for Parkinson's can help control symptoms such as tremors, slowness of movement, and cognitive problems, none address the underlying neuronal loss which causes this devastating condition.”

Chief Medical Officer Dr Suhail Nurbhai said Mission was keen to rapidly progress the compound through initial clinical testing and aimed to demonstrate its potentially beneficial clinical profile later this year.

The start of the trial follows the publication of a key academic paper in the journal Nature Communications last December by scientists at Cambridge University, Harvard University and Mission itself.

The paper outlined their preclinical research in mouse models which provided strong experimental evidence supporting the thesis that MTX325 can modify the course of PD by targeting USP30.

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Published: 21.03.24