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$5.2m to aid Mission Therapeutics from The Michael J Fox Foundation for Parkinson’s Research





Mission Therapeutics has been awarded $5.2m from The Michael J Fox Foundation and Parkinson’s UK to advance its potential disease-modifying treatment MTX325.

MTX325 is a potent, selective, small molecule brain-penetrant USP30 inhibitor, which is designed to protect dopamine-producing neurons by improving mitochondrial quality and function. The funding will support a 28-day dosing of MTX325 in patients with early-stage Parkinson’s disease (PD), as part of Mission’s ongoing MTX325 Phase I programme.

Mission Therapeutics' work in the lab
Mission Therapeutics' work in the lab

Dosing for patients with early-stage Parkinson’s disease is expected to start early in 2025.

Dr Paul Thompson, chief scientific officer, Mission Therapeutics, said: “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 PD patient part of the trial in the new year, which this generous funding from MJFF and Parkinson’s UK is helping to support.”

Anker Lundemose, CEO, Mission Therapeutics NOTE: LO RES 53k!!!! ONLINE ONLY
Anker Lundemose, CEO, Mission Therapeutics NOTE: LO RES 53k!!!! ONLINE ONLY

Mission started its multi-part, adaptive Phase I first-in-human clinical trial of MTX325 in March, beginning with a single ascending dose stage in healthy volunteers. Initial investigations of MTX325’s CNS penetration ability in these healthy volunteers have yielded positive results. The multiple ascending dose stage of the study was initiated last month (June).

Anker Lundemose, CEO, Mission Therapeutics, said: “This significant grant, from two of the world’s leading Parkinson’s disease organisations, underlines the huge potential of MTX325 as a disease-modifying treatment for this terrible neurodegenerative illness.

Mission Therapeutics is based at The Glenn Berge Building, Babraham Research Campus
Mission Therapeutics is based at The Glenn Berge Building, Babraham Research Campus

“It also represents a major endorsement of our mitophagy strategy in human diseases including PD.”

Katharina Klapper, director of clinical research at The Michael J Fox Foundation, said: “Mission Therapeutics has made great advances in the understanding of how mitochondrial health can play a pivotal role in the development of Parkinson’s disease in recent years. We look forward to seeing the results of the MTX325 trial.”



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