By Mike Hollan

MTX325 is in Phase I trials and is believed to be able to modify the course of the disease.
The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and Parkinson’s UK award a multi-million dollar grant to Mission Therapeutics.1 The $5.2 million grant will be used to advance Mission Therapeutics MTX325, a potentially disease modifying treatment for Parkinson’s.
In a press release, Mission Therapeutics’ CEO Anker Lundermose said, “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. It also represents a major endorsement of our mitophagy strategy in human diseases including PD."
The company’s chief scientific officer, Dr. Paul Thompson, PhD,, added, “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."
Mission Therapeutics announced the start of in-human Phase I clinical trials for MTX325 in March of this year.2 The first phase of the trial will focus on multiple dose ascending and elderly patients for the current year. In 2025, the trial will shift its focus to patients with Parkinson’s Disease.
Researchers believe that MTX325 can be used to target USP30, a deubiquitylating enzyme that inhibits mitophagy, which has been linked to diseases like Parkinson’s. It is believed by targeting this enzyme, MTX325 can alter the course of the disease.
In a press release issued at the time, Dr. 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."
In the same press release, the company’s chief medical officer Dr. Suhail Nurbhai, said, “We are delighted to have brought this second USP30 inhibitor into clinical development. The overall objectives of this Phase I trial are to confirm the safety, tolerability, and CNS penetration of MTX325, in both healthy volunteers and patients with Parkinson's Disease, and help us determine appropriate doses for future efficacy testing. We look forward to progressing this compound rapidly through initial clinical testing and aim to demonstrate its potentially beneficial clinical profile later this year."
In regard to the latest news about the grant from MJFF, the organization’s director of clinical research Katharina Klapper 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."
Dr Arthur Roach, director of the Parkinson's virtual biotech at Parkinson's UK, added, “We are delighted to be working with Mission Therapeutics to help fund this vital, UK-based clinical trial. Disease-modifying treatments are one of the great hopes of people with Parkinson's. We now know that mitochondria play a crucial role in the development of Parkinson's, so addressing mitochondrial problems could have far-reaching benefits for those living with the condition."