Acute kidney injury is a sudden and rapid decline in kidney function and can occur after cardiac surgery. Credit: mi-viri via Getty Images.

The US Food and Drug Administration (FDA) has cleared UK-based Mission Therapeutics’s investigational new drug (IND) application for its acute kidney injury (AKI) drug MTX652. 

Following this announcement, Mission Therapeutics will proceed with a Phase II trial to investigate the drug. The double-blind, placebo-controlled trial will enrol up to 160 adult patients who have an increased risk for AKI following cardiac surgery and will take place at multiple sites across North America and Europe. 

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MTX652 is a small molecule deubiquitylating enzyme (DUB) inhibitor that inhibits ubiquitin-specific protease 30 (USP30), a mitochondrial deubiquitinase enzyme. The drug is developed based on a DUB screening platform. 

According to GlobalData’s Pharma Intelligence Centre, Mission Therapeutics has 12 other pipeline candidates, including MTX325, a DUB inhibitor indicated for the treatment of Parkinson’s disease. This month, the company obtained clinical trial authorisation from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to assess MTX325 in a Phase I study for Parkinson’s disease.  

GlobalData is the parent company of Pharmaceutical Technology. 

The FDA clearance of MTX652 is based on data from the randomised, placebo-controlled, double-blind Phase I study that concluded in January 2023. MTX652 was administered in seven single-dose cohorts that tested doses between 0.25 and 200mg and four multiple-dose cohorts between 3.5mg to 100mg, once a day for 14 days. The primary endpoint of bioavailability, pharmacokinetics, and safety was achieved. 

In the announcement accompanying the clearance, chief scientific officer at Mission Paul Thompson said: “In preclinical experiments, MTX652 demonstrated significant protective effects in multiple models of kidney injury, and this breadth of effect is very encouraging in indicating potential clinical benefit.” 

Mission Therapeutics is involved in partnerships with several companies, including pharma giant Pfizer, since 2013, raising $15m in 2020 in an equity investment fundraising round. The company paired up with AbbVie in 2018 for the research and preclinical development of DUB therapies to treat Alzheimer’s disease and Parkinson’s disease.