Mission Therapeutics authorised to initiate first clinical trial for lead DUB program, MTX652, in kidney disease

CAMBRIDGE, UK – 31 March 2022 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), today 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.

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Mission Therapeutics Ltd
Anker Lundemose MD PhD
Chief Executive Officer
Tel: +44 (0)1223 607 340
Instinctif Partners
Melanie Toyne-Sewell / Katie Duffell
Tel: +44 (0) 20 7457 2013


About Mission Therapeutics

Mission Therapeutics is an early-stage drug development company targeting the ubiquitin pathway for the treatment of kidney disease, neurodegenerative disease, rare mitochondrial diseases and fibrosis. The Company has built a leading platform for the discovery and development of first-in-class, small molecule drugs that selectively target deubiquitylating enzymes (DUBs) – an emerging drug class that is attracting significant commercial interest in the area of protein homeostasis.

Mission has strong links with key academic and research centers, including Prof. Steve Jackson’s Cancer Research UK Laboratories at the University of Cambridge Gurdon Institute, and leading UK centres in neurodegenerative diseases. The Company also has secured major industry partnerships, including its collaboration with AbbVie in November 2018, for the research and preclinical development of specified DUB inhibitors for the treatment of Alzheimer’s Disease and Parkinson’s Disease. The Company is managed by a team with broad international, commercial and clinical-science experience.

To date the Company has received £73 million / $101 million in funding and its investors comprise blue chip institutional and corporate investors including: Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital. Mission Therapeutics was founded in 2011 and is based at the Babraham Research Campus, Cambridge, UK.

For more information, please visit our website, www.missiontherapeutics.com, or follow us on Twitter or LinkedIn.