Friday, March 22, 2024
Mission Therapeutics ("Mission"), a biotech company in the clinical stage, has initiated the Phase I first-in-human clinical trial of MTX325, its promising treatment aimed at modifying Parkinson's Disease (PD).
With approximately 10 million individuals affected by Parkinson's Disease globally, including nearly one million in the US and about 1.2 million in Europe, these numbers are anticipated to rise with aging populations.
The Company has completed dosing the initial group of healthy volunteers in a multi-part, adaptive Phase I study, evaluating MTX325's safety, tolerability, pharmacokinetics, and its ability to penetrate the brain. The trial plan for 2024 includes single ascending doses, multiple dose escalations, and cohorts comprising elderly healthy volunteers, with a focus on Parkinson's Disease patients slated for 2025.
Dr. Paul Thompson, PhD, Chief Scientific Officer at Mission Therapeutics, stated, "Initiating this first-in-human trial marks a significant advancement for Mission Therapeutics, as we assess MTX325's potential as a disease-modifying therapy for Parkinson's Disease. While current treatments for Parkinson's address symptoms like tremors and cognitive issues, none tackle the underlying neuronal loss driving this debilitating condition."
Dr. Suhail Nurbhai, Chief Medical Officer at Mission Therapeutics, expressed his delight in advancing this second USP30 inhibitor into clinical development. He emphasized the trial's objectives to confirm MTX325's safety, tolerability, and central nervous system penetration in both healthy volunteers and Parkinson's Disease patients, aiming to determine suitable doses for future efficacy evaluations.
The trial's commencement follows the publication of a significant academic paper in Nature Communications last December by researchers from Cambridge University, Harvard University, and Mission Therapeutics. The paper detailed preclinical research in mouse models, offering compelling evidence supporting MTX325's potential to alter the course of Parkinson's Disease by targeting USP30.
USP30, a deubiquitylating enzyme (DUB) inhibiting mitophagy, the process by which cells eliminate dysfunctional mitochondria, is the focus of MTX325. Scientific evidence increasingly links the accumulation of dysfunctional mitochondria to various diseases, including Parkinson's Disease, Kidney Disease, Heart Failure, idiopathic pulmonary fibrosis (IPF), and Duchenne's Muscular Dystrophy (DMD).
MTX325, a potent and selective compound with central nervous system penetration, aims to enhance mitochondrial quality and function by boosting mitophagy. By inhibiting USP30, localized to mitochondria and acting as a negative regulator of mitophagy, MTX325 holds promise in modifying the progression of Parkinson's Disease, as evidenced by its effects in PD mouse models following USP30 deletion through gene knockout.
Source: missiontherapeutics.com
