Mission Therapeutics moves kidney disease drug to phase 2 after FDA approval

Up to 160 adults with increased risk for acute kidney injury (AKI) following cardiac surgery are planned to be recruited for the study from multiple sites in North America and Europe.

The double-blind, placebo-controlled trial is expected to begin early next year and is intended to show that MTX652 protects this high-risk group of patients from AKI by assessing standard markers of renal function and renal injury over time.

Dr Anker Lundemose, CEO of Mission Therapeutics, said: “The FDA’s approval of our Phase II clinical study for our lead asset MTX652 marks a major milestone for Mission. We now have two USP30 inhibitors advancing through clinical trials, MTX652 for acute kidney injury and MTX325 for Parkinson’s Disease, validating our unique approach and the breadth of our assets.”

The FDA’s decision comes after the completion of Mission’s phase 1 study of MTX652 earlier in 2023, in which over 80 healthy volunteers received the drug.

The trial demonstrated that the treatment was safe and well tolerated up to a single dose of 200mg a day and multiple doses of 100mg daily for 14 days, with an ‘excellent’ pharmacokinetic profile.

Mission chief scientific officer Paul Thompson explained that MTX652 had shown significant protective effectives in multiple models of kidney injury and the company was ‘very pleased’ to be progressing with this strong preclinical foundation.

MTX652 is a potent and selective compound designed to improve mitochondrial quality and function by enhancing mitophagy through inhibition of USP30, a deubiquitylating enzyme localised to mitochondria which is a negative regulator of mitophagy.

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