Mission Therapeutics receives $5.2m from Michael J. Fox Foundation and Parkinson's UK for MTX325 development

Mission Therapeutics (Mission), a clinical-stage biotech firm specializing in therapeutics targeting mitophagy, has received $5.2 million in funding from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and Parkinson's UK. This investment will propel the development of Mission's potential disease-modifying treatment for Parkinson's disease (PD), MTX325.

MTX325 is a selective, small molecule brain-penetrant inhibitor of USP30, an enzyme that hinders mitophagy, the cellular process of clearing dysfunctional mitochondria. By inhibiting USP30, MTX325 aims to protect dopamine-producing neurons, thereby improving mitochondrial quality and function. The funding will support a 28-day dosing trial of MTX325 in patients with early-stage Parkinson's disease, expected to commence in early 2025. This trial will assess MTX325's safety, tolerability, pharmacokinetic profile, and central nervous system (CNS) penetration in PD patients, along with its effects on disease biology biomarkers.

Mission initiated its adaptive Phase I clinical trial of MTX325 in March, starting with a single ascending dose stage in healthy volunteers. The initial results have shown that MTX325 possesses a commendable safety profile, pharmacokinetics, and CNS penetration. The multiple ascending dose stage of the study commenced in June.

Anker Lundemose, CEO of Mission Therapeutics, emphasized the significance of the grant from MJFF and Parkinson's UK, highlighting the potential of MTX325 as a transformative treatment for Parkinson's disease. Dr. Paul Thompson, Chief Scientific Officer, echoed this sentiment, pointing to the promising early data and expressing optimism about the upcoming trials in PD patients, facilitated by the generous funding.

Katharina Klapper, Director of Clinical Research at MJFF, praised Mission Therapeutics for its advancements in understanding mitochondrial health's role in Parkinson's disease development. Dr. Arthur Roach, Director of the Parkinson's Virtual Biotech at Parkinson's UK, expressed enthusiasm about collaborating with Mission Therapeutics, underscoring the importance of developing disease-modifying treatments for Parkinson's. He noted the critical role mitochondria play in the disease's progression and the potential benefits of addressing mitochondrial dysfunction.

Parkinson's disease is a neurodegenerative disorder affecting around 10 million people globally, characterized by low dopamine levels. Currently, there are no approved treatments that modify the disease's progression. MTX325 aims to address this gap by enhancing mitophagy through USP30 inhibition, thereby protecting neurons and potentially altering the disease's course.

Previous research by scientists at Cambridge University, Harvard University, and Mission Therapeutics has provided strong evidence supporting MTX325's potential to modify Parkinson's disease. Their study, published in Nature Communications, demonstrated that knockout of USP30 in a mouse model produced similar positive effects as MTX325, reinforcing the therapeutic approach.

Mounting scientific evidence has linked mitochondrial dysfunction to various diseases, including Parkinson's, kidney disease, heart failure, and muscular dystrophies. Mission Therapeutics is at the forefront of developing novel treatments that promote the removal of dysfunctional mitochondria, enhancing cell health and function. The company is supported by notable investors such as Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, and others.

About Mission Therapeutics: The company is a leader in discovering and developing therapeutics that target mitochondrial dysfunction through USP30 inhibition. Their work focuses on improving cellular health and function, with potential applications in numerous diseases driven by mitochondrial dysfunction.

About MTX325: This compound is designed to improve mitochondrial quality and function by enhancing mitophagy, offering a promising approach to treating Parkinson's disease by targeting mitochondrial dysfunction.

About Parkinson's UK and the Parkinson's Virtual Biotech: Parkinson's UK funds research into promising treatments and aims to deliver new therapies swiftly through the Parkinson's Virtual Biotech program, a collaborative initiative working towards finding a cure.