Mission Therapeutics raises $13.3m for Parkinson’s trials
The financing was led by current investors. While the backers for this round were not named Mission is supported by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.
Mission is a clinical-stage biotech developing first-in-class therapeutics that promote cell and organ health by enhancing mitophagy.
The financing 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, added: “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.”
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.
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.
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. To execute the MTX325 Phase 1b study, Dr Fritchley was promoted to Chief Development Officer in April.
