CAMBRIDGE, England, Nov. 2, 2023 /PRNewswire/ -- Mission Therapeutics ("Mission"), a clinical-stage biotech company developing first-in-class therapeutics targeting mitophagy, today announces that it will present an oral abstract on its experimental drug candidate MTX652 at the American Society of Nephrology's Kidney Week in Philadelphia, from 2-5 November 2023.
Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria to maintain cell health and function. MTX652 targets USP30, a deubiquitylating (DUB) enzyme uniquely localized to the mitochondria that removes ubiquitin groups and is a negative regulator of the PINK1/Parkin pathway and mitophagy. Mitophagy is a quality control system that ensures dysfunctional mitochondria are removed.
Pau Aceves, MS, MSc, PharmD., Head of Clinical Pharmacology and Pharmacometrics, Mission Therapeutics, commented: We look forward to presenting our latest Phase 1 and Modelling & Simulation results on MTX652 for the treatment of acute kidney injury (AKI). The American Society of Nephrology is a major organization working toward a goal of a world without kidney diseases and we are excited to have the opportunity to share our progress at this important conference.
Details of the presentation are as follow:
Presentation Title: MTX652, a Novel Selective USP30 Inhibitor for the Treatment of AKI: Phase 1 Results in Healthy Subjects and Model-Driven Human Efficacious Dose Projections
Session Name: Onconephrology and Precision Pharmacology
Session Date: Thursday, November 2nd, 2023
Presentation Time: 4:48 PM - 4:57 PM
Presenter: Pau Aceves, MS, MSc, PharmD., Head of Clinical Pharmacology and Pharmacometrics
Location: Room 107, Pennsylvania Convention Center
Mission has completed its first Phase I clinical study with MTX652, in which it demonstrated a highly encouraging safety, tolerability and pharmacokinetic profile. A Phase II trial of MTX652 in patients at risk of acute kidney injury following cardiac surgery is planned to start in Q1 2024.
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.
A Phase I trial of MTX325 in healthy volunteers and patients with PD is planned to start in Q1 2024.
Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.
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