March 31, 2022 – “Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs) announced it has been granted approval to take its lead USP30 DUB inhibitor, MTX652, into clinical study.
Mitochondria are essential for energy production and cellular health. When they become damaged or stop working properly, they are labelled with ubiquitin, marking them for degradation.
The mitochondrial-associated DUB, USP30, removes this ubiquitin and thereby inhibits the degradation, which can affect cell health.
Mission has developed MTX652 to inhibit USP30 with the aim to enable appropriate degradation of dysfunctional mitochondria to preserve and improve cellular health.
Specific kidney cells are rich in mitochondria and highly vulnerable to injury if those mitochondria are not working correctly. Mitochondrial dysfunction is strongly implicated in kidney injury and chronic kidney disease (CKD). In addition to kidney diseases, mitochondrial quality is implicated in a range of other poorly treated conditions such as idiopathic pulmonary fibrosis, muscular dystrophy and primary mitochondrial disease. Mission is also investigating the potential of USP30 inhibitors to treat these diseases.
Following candidate nomination last year, MTX652 has successfully advanced through preclinical regulatory toxicology studies and is ready to be progressed into the clinic.
The planned initial clinical trial will evaluate the safety, tolerability and pharmacokinetics of MTX652 in over 60 participants given either a single or multiple doses.
The trial is due to start in April 2022.
Dr Suhail Nurbhai, CMO of Mission Therapeutics, commented: “We are delighted that our lead programme is entering the clinic.
Mission has built a groundbreaking platform for the discovery and development of first-in-class small molecule drugs that selectively target DUBs.
MTX652 entering the clinic is a major milestone for the Company and a tremendous achievement. We are excited to be taking this important next step.”
Kidney diseases are a major global health problem and are reported by the US CDC as a leading cause of death in the US.
CKD affects an estimated 780 million people around the world.
The attributable UK NHS costs in 2009/10 were around £1.5 billion and the estimated US Medicare costs for CKD are greater than US$87 billion.
Acute kidney injury (AKI) and CKD are increasingly viewed as a continuum of kidney disease and increase risk to patients of damage to non-renal organs, cardiovascular disease, and early death. Preventing these conditions is therefore an important unmet medical need.”
https://missiontherapeutics.com/mission-therapeutics-authorised-to-initiate-first-clinical-trial-for-lead-dub-program-mtx652-in-kidney-disease/
