£25.2 million raised to progress Parkinson’s treatment

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Mission Therapeutics has raised £25.2 ($32.1) million to accelerate development of its lead drug candidates in the area of mitophagy, MTX325 and MTX652, through clinical trials.

The financing was jointly led by existing investors Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group, and Rosetta Capital.

MTX325 and MTX652 both inhibit USP30, a mitochondrial de-ubiquitylating enzyme (DUB), increasing damage-associated mitochondrial ubiquitylation to promote mitophagy – the essential process cells use to rid themselves of dysfunctional mitochondria. 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, heart failure, and Duchenne’s muscular dystrophy (DMD).

MTX325, a CNS penetrant which is a potential disease-modifying treatment for Parkinson’s disease, is about to enter Phase I trials; while peripherally-restricted MTX652 is currently in Phase II investigating acute kidney injury (AKI) associated with cardiac surgery.

Dr Anker Lundemose, Chief Executive Officer of Mission Therapeutics, said: “Mission Therapeutics has made huge strides in developing its pipeline, first progressing MTX652 into Phase II, then obtaining robust preclinical proof-of-concept data for its Parkinson’s candidate MTX325 – published in Nature Communications – followed by regulatory approval for MTX325 clinical trials in the UK. Thanks to this additional £25.2m from our investors, we can now make the next vital steps, progressing with essential clinical trials.”

Dr James Summers, Acting Chairman of Mission Therapeutics, said: “Mission’s laser focus on mitophagy has resulted in a promising suite of drugs that tackle a range of hard-to-treat diseases in a unique and novel way. This latest financing round is a sign of our investors’ confidence in the company and the enormous potential of our clinical assets.”

Disease-modifying therapy for Parkinson’s

The Nature Communications paper, published in November 2023 by scientists at Cambridge University, Harvard University 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 potential 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.

The same month, Mission gained clearance from the US Food and Drug Administration (FDA) to commence a Phase II trial of MTX652, after the company has received official approval of its Investigational New Drug (IND) application for the candidate drug.

Megan Thomas, Multimedia Editor, DDW

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