Mission Therapeutics receives $5.2M to advance novel small-molecule Parkinson’s therapy

Mission Therapeutics has been awarded $5.2 million from The Michael J. Fox Foundation for Parkinson’s Research and Parkinson’s UK to advance a selective, small molecule, brain penetrant USP30 inhibitor for Parkinson’s disease.

MTX325 is designed to protect dopamine-producing neurons by improving mitochondrial quality and function, Mission said in a press release. The funding will assist a pending phase 1 clinical trial featuring a 28-day dosing of the novel therapeutic in those in the early stages of the disease and will assess safety, tolerability, pharmacokinetic profile as well as central nervous system penetration in those with PD. Patient dosing is expected to start early in 2025.

The company initiated a first-in-human clinical trial of MTX325 in March, beginning with a single ascending dose stage in healthy volunteers. Interim data showed encouraging CNS penetration ability, according to the release. The multiple ascending dose stage of the study was initiated in June.

“This significant grant, from two of the world’s leading Parkinson’s disease organizations, underlines the huge potential of MTX325 as a disease-modifying treatment for this terrible neurodegenerative illness,” Mission Therapeutics CEO Anker Lundemose said in the release. “It also represents a major endorsement of our mitophagy strategy in human diseases including [Parkinson’s disease].”