Mission Therapeutics Successfully Completes First Clinical Assessment for Lead DUB Program, MTX652

  • Safety, tolerability and pharmacokinetic endpoints successfully met
  • Follow-on clinical trials to be initiated in 2023

CAMBRIDGE, UK – 5 January 2023 – Mission Therapeutics (“Mission”), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), today announced the successful completion  of its first Phase I clinical assessment for lead USP30 DUB inhibitor, MTX652.

The Phase I First Time In Human (FTIH) study commenced in May 2022, following clinical trial approval in March. Involving over 80 healthy volunteers, the study was led by Principal Investigator Dr Annelize Koch, Senior Medical Director at Simbec-Orion.

The trial successfully achieved its key goals of demonstrating the safety, tolerability and pharmacokinetics of MTX652, given as single- and multiple-ascending doses of oral solution or suspension to the healthy subjects. MTX652 was shown to be well tolerated and safe up to a single dose of 200mg and multiple doses of 100mg once daily for 14 days. It also demonstrated an excellent pharmacokinetic profile with dose proportionality, and time independent exposure.

Following the successful completion of the single and multiple ascending dose cohorts, Mission is moving forward with further assessment of MTX652 in elderly subjects and using other dose forms. The Company is also preparing plans to initiate further clinical trials later this year to demonstrate the beneficial effects of MTX652 in patients with muscular, cardiac and kidney pathologies.

Dr Suhail Nurbhai, CMO of Mission Therapeutics commented:

“We are delighted with the successful completion of our first Phase I clinical assessment. These MTX652 results represent a major milestone for Mission and, coupled with the results from our broad translational pharmacology program, give us a great deal of optimism that we may eventually be able to help patients with a range of poorly-treated diseases. Importantly, it also helps to validate our unique discovery platform.

“We are now in the process of planning for our next stage of clinical development and are looking forward to MTX652 entering further clinical trials later this year.”

About Mitochondria and USP30

Mitochondria are essential for energy production and cellular health, and when they become dysfunctional or damaged, they are labelled with ubiquitin, marking them for degradation. USP30, a specific mitochondrial-associated DUB, removes this ubiquitin, thereby inhibiting the degradation of damaged mitochondria, which could affect cellular health and eventually lead to the development of various medical conditions.

Mission’s MTX652 potently and specifically inhibits USP30 with the aim of enabling appropriate degradation of dysfunctional mitochondria to preserve overall mitochondrial quality and improve cellular health.
Mitochondrial dysfunction is increasingly implicated in many poorly treated pathologic conditions including muscular dystrophy, heart failure, cardiomyopathy, kidney diseases, idiopathic pulmonary fibrosis, rare mitochondrial diseases, and neurodegenerative diseases such as Parkinson’s Disease. Inhibition of USP30 is therefore a promising therapeutic approach for treatment of these conditions and Mission is investigating its USP30 inhibitor compounds as potential therapeutic interventions for a breadth of diseases.

– ENDS –


Mission Therapeutics Ltd
Anker Lundemose MD PhD
Chief Executive Officer
Tel: +44 (0)1223 607 340
Instinctif Partners
Melanie Toyne-Sewell / Agnes Stephens / Jonjo Cordey

Email: missiontherapeutics@instinctif.com
Tel: +44 (0) 207 457 2002


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 $103 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.