Mission Therapeutics has achieved a significant milestone as the US Food and Drug Administration (FDA) has granted approval for its Investigational New Drug (IND) application for MTX652, the company's primary asset. This regulatory clearance allows Mission to progress with its planned Phase II clinical trial in the United States.

The upcoming trial aims to enroll up to 160 adults at an increased risk of Acute Kidney Injury (AKI) following cardiac surgery. Multiple sites in North America and Europe will host this double-blind, placebo-controlled study, scheduled to commence early next year. The trial will assess MTX652's efficacy in protecting high-risk patients from AKI by evaluating standard markers of renal function and injury over time.

Dr. Anker Lundemose, CEO of Mission Therapeutics, highlighted the significance of the FDA's approval, emphasizing the progress of two USP30 inhibitors in clinical trials—MTX652 for acute kidney injury and MTX325 for Parkinson's Disease. This validation underscores the uniqueness of Mission's approach and the breadth of its therapeutic assets.

Dr. Suhail Nurbhai, Chief Medical Officer of Mission Therapeutics, stressed the critical unmet medical need for effective treatments for AKI following heart surgery. With no approved drug treatments currently available, MTX652 holds promise in addressing this serious medical challenge.

Dr. Paul Thompson, Chief Scientific Officer of Mission Therapeutics, emphasized the encouraging results from preclinical experiments, where MTX652 demonstrated significant protective effects in various kidney injury models. The broad scope of these effects indicates potential clinical benefits, supporting the transition to clinical efficacy trials.

The FDA's decision follows the successful completion of a Phase I First Time in Human (FTIH) study for MTX652 earlier in 2023. The study involving over 80 healthy volunteers demonstrated the safety and tolerability of MTX652, paving the way for the upcoming Phase II clinical trial.

MTX652 is a potent and selective compound designed to enhance mitochondrial quality and function by inhibiting USP30, a deubiquitylating enzyme localized to mitochondria. By targeting USP30, MTX652 aims to improve mitophagy, a key cellular mechanism for clearing dysfunctional mitochondria. The therapeutic potential of MTX652 extends to reducing the impact of impaired mitochondria associated with Ischaemic Reperfusion Injury (IRI) in the heart and kidneys of patients who have undergone heart surgery.

 

Source: prnewswire.com

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