Mission Therapeutics raises £25.2 million to progress clinical candidates in the area of mitophagy
Summary
A growing body of scientific evidence has linked a build-up of dysfunctional mitochondria in cells to a range of diseases, including Parkinsons Disease (PD), Kidney Disease, Heart Failure, and Duchennes Muscular Dystrophy (DMD).MTX325, a CNS penetrant which is a potential disease-modifying treatment for Parkinsons 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 Parkinsons candidate MTX325 - published in Nature Communications - followed by regulatory approval for MTX325 clinical trials in the UK. 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, 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 Parkinsons Disease.The same month, Mission gained clearance from the US FDA to commence a Phase II trial of MTX652, after the Company received official approval of its Investigational New Drug (IND) application for the candidate drug.About Mission TherapeuticsMission 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. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria consequently improving overall cellular health.