Mission raises $13.3M for Parkinson’s treatment Phase 1b trial
Phase 1a study showed MTX325 can reach brain tissue
Mission Therapeutics has raised $13.3 million to advance MTX325, its treatment candidate for Parkinson’s disease, into a Phase 1b clinical trial in Parkinson’s patients.
The funding comes after the company successfully completed a Phase 1a study demonstrating the treatment can enter functional brain tissue in healthy volunteers.
The company said the Phase 1b trial will start in the first half of next year, and has been approved by the UK’s Medicines and Healthcare products Regulatory Agency. Initial proof-of-mechanism biomarker and imaging data are expected to be available in 2027.
“Thanks to this additional $13.3m from our investors, we can now make the next vital steps progressing MTX325 into [Parkinson’s disease] patients with this essential Phase 1b clinical trial,” Anker Lundemose, MD, PhD, executive director at Mission, said in a company press release. “This will enable us to build upon the compelling preclinical data package for MTX325 … and the results from the Phase 1a studies that we have obtained.”
Parkinson’s is caused by the death of dopaminergic neurons, nerve cells that are responsible for making dopamine, a brain chemical messenger involved in motor control. Damage to mitochondria, the cells’ powerhouses, is believed to contribute to the death of dopaminergic neurons.
Mouse trial shows promise
MTX325 is an oral molecule designed to increase the number of functional mitochondria by enhancing mitophagy, a quality control process that allows cells to clear damaged mitochondria. It works by inhibiting USP30, a protein that blocks mitophagy, and has the ability to enter the central nervous system (CNS, consisting of the brain and spinal cord), where it exerts its neuroprotective effects.
In a preclinical study, MTX325 was shown to reduce mitochondrial dysfunction, alpha synuclein accumulation — a hallmark of the disease — and loss of dopaminergic neurons in a Parkinson’s mouse model.
The Phase 1a trial, which tested the therapy in healthy volunteers at single or multiple doses, demonstrated MTX325’s ability to reach the CNS. This was confirmed by analyzing samples of participants’ cerebrospinal fluid — the fluid surrounding the brain and spinal cord — and PET scans showing its distribution within the brain.
“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,” said Sarah J Fritchley, PhD, Mission’s chief development officer.
James B. Summers, PhD, Mission’s chairman said the financing 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 [Parkinson’s disease].”
The funding was led by current investors, the company said.
The clinical development of MTX325 was supported by a $5.2 million grant from the Michael J. Fox Foundation and Parkinson’s UK.
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