Subjects: TRI, FDA
Mission Therapeutics announces US FDA approval to initiate Phase II clinical trial of its lead asset MTX652 in Acute Kidney Injury
- Mission gains clearance from US FDA for Phase II trial of MTX562 after it receives official approval of its Investigational New Drug (IND) application
- Phase II trial expected to begin Q1 2024 in up to 160 patients with acute kidney injury (AKI) following cardiac surgery
CAMBRIDGE, England, Dec. 14, 2023 /PRNewswire/ -- Mission Therapeutics ("Mission" or the "Company"), a clinical-stage biotech developing first-in-class therapeutics targeting mitophagy, today announces that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for its lead asset MTX652, allowing Mission to proceed with its planned Phase II clinical trial in the US.
Up to 160 adults with increased risk for Acute Kidney Injury (AKI) following cardiac surgery are planned to be recruited for the trial, which will take place at 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, Chief Executive Officer 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."
Dr Suhail Nurbhai, Chief Medical Officer of Mission Therapeutics, said: "Recent reports suggest that up to 50% of high-risk patients suffer acute kidney injury following heart surgery[i] [ii]. There are no approved drug treatments and the immediate and longer-term consequences can be very serious, including continued decline of renal function and/or the requirement for renal replacement therapy. We believe MTX562 has the potential to alleviate these outcomes and to meet this serious and important unmet medical need. We are delighted to have received this approval and look forward to starting this trial in 2024."
Dr Paul Thompson, Chief Scientific Officer of Mission Therapeutics, commented: "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. We are very pleased with this strong preclinical foundation, which supports our progress into clinical efficacy trials."
The FDA's decision follows completion of a Phase I First Time in Human (FTIH) study of MTX652 earlier in 2023, in which over 80 healthy volunteers received MTX652. The trial successfully demonstrated MTX652 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.
About MTX652 and USP30
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. It thereby promotes cellular health by enhancing this key mechanism, which cells use to clear dysfunctional mitochondria. MTX652 has the potential to reduce the impact of impaired mitochondria associated with Ischaemic Reperfusion Injury (IRI) in the heart and kidneys in patients who have undergone heart surgery.
About Mission Therapeutics
Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson's Disease (PD), heart failure, Duchenne's Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer's.
USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria ? consequently improving overall cellular health. Mission's USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction.
Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.
[ii] https://pubmed.ncbi.nlm.nih.gov/34474590/
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