Mission Therapeutics raises $13.3 million to progress first-in-class Parkinson's disease candidate MTX325 through clinical trials

• Financing will support the full execution of Phase Ib proof-of-mechanism study of MTX325 in patients with Parkinson's disease (PD)
• Comes after successful completion of Phase 1a studies of MTX325, a first-in-class and potentially disease-modifying treatment for PD, in healthy volunteers
• Phase Ib study due to start in H1 2026

CAMBRIDGE, England, Oct. 15, 2025 /PRNewswire/ -- Mission Therapeutics ("Mission" or the "Company"), a clinical-stage biotech developing first-in-class therapeutics that promote cell and organ health by enhancing mitophagy, today announces it has raised $13.3 million to progress the clinical development of its lead candidate MTX325 through a Phase Ib proof-of-mechanism study in patients with Parkinson's disease. The financing was led by current investors.

It comes after Mission successfully completed Phase Ia studies – including recent PET scans confirming MTX325 adequately penetrates functional brain tissues in healthy volunteers. The clinical development of MTX325 is also supported by a $5.2 million grant from the Michael J. Fox Foundation and Parkinson's UK. Mission has regulatory approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) for the Phase Ib clinical trial of MTX325 to begin.

Dr Anker Lundemose, Executive Director at Mission Therapeutics, said: "Thanks to this additional $13.3m from our investors, we can now make the next vital steps progressing MTX325 into PD patients with this essential Phase 1b clinical trial. This will enable us to build upon the compelling preclinical data package for MTX325, published in Nature Communications in 2023, and the results from the Phase 1a studies that we have obtained."

Dr Sarah J Fritchley, Chief Development Officer, Mission Therapeutics, said: "The overall objectives of this Phase Ib trial are to demonstrate robust clinical proof-of-mechanism (PoM) in patients with Parkinson's disease, and to gather further information on safety and tolerability. We look forward to progressing MTX325 rapidly through clinical testing and anticipate we will have PoM data in H2 2027."

Dr James B. Summers, Chairman of Mission Therapeutics, said: "This latest financing round is a sign of our investors' confidence in the Company and the enormous potential of MTX325 as a first-in-class, disease-modifying treatment for PD."

MTX325 works by inhibiting USP30, a mitochondrial de-ubiquitylating enzyme (DUB), thereby increasing mitochondrial ubiquitylation and promoting appropriate mitophagy – the essential process cells use to rid themselves of dysfunctional mitochondria. If cells such as neurons cannot rid themselves of dysfunctional mitochondria, they themselves start to malfunction and die. A growing body of scientific evidence has linked a build-up of dysfunctional mitochondria in cells to a range of diseases, including Parkinson's disease (PD), kidney disease and heart failure.

A paper published in Nature Communications in November 2023, written by scientists at Cambridge University, Harvard University, University of Dundee, and Mission Therapeutics, provided key experimental evidence to support the thesis that MTX325 can modify the course of Parkinson's by targeting USP30. By first using a USP30 knockout mouse model, and then a pharmacological strategy deploying MTX325, they found USP30 inhibition led to protection against loss of dopamine and dopaminergic neurons induced by alpha-synuclein in vivo. USP30 inhibition also reduced biomarkers of PD including phosphorylated alpha-synuclein and glial cell activation.

In December 2023, the sister publication Nature Reviews Drug Discovery commented that restoring mitophagy to accelerate the removal of damaged mitochondria was "an appealing disease-modifying therapeutic strategy" for Parkinson's disease.

About MTX325

MTX325 is a first-in-class, highly potent and selective, orally bioavailable and brain-penetrant USP30 inhibitor targeting improved mitochondrial quality. Mitochondrial dysfunction is a leading hypothesis in Parkinson's disease pathophysiology. Inhibition of USP30 is a well validated approach for restoring mitophagy to achieve neuroprotection in Parkinson's disease.

In pre-clinical assessments, MTX325 demonstrated impact on multiple aspects of Parkinson's disease – mitochondrial dysfunction, alpha-synuclein accumulation and loss of nigrostriatal dopaminergic neurons. It is currently in clinical development for disease modification in Parkinson's disease. Phase 1a SAD/MAD was successfully completed in healthy volunteers and CSF sampling confirmed a high level of penetration into CNS.  PET study further confirmed distribution into brain parenchyma.

Proof-of-mechanism biomarker and imaging data in PD patients will be available in 2027.  Composition of matter IP exclusivity extends through 2041.

About Mission Therapeutics

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in multiple serious conditions including Parkinson's disease (PD), acute kidney injury (AKI), heart failure, Duchenne's Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer's.

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX325 (targeting the CNS) and MTX652 (peripheral) which, through inhibition of the mitochondrial DUB enzyme USP30, promote clearance of dysfunctional mitochondria – consequently improving overall cellular health.

Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.  To execute the MTX325 Phase 1b study, Dr Sarah Fritchley was promoted to Chief Development Officer on April 1^st 2025.