Mission Therapeutics, a clinical-stage biotechnology company developing first-in-class therapeutics that enhance mitophagy to protect cell and organ health and NeuraLight, a leader in precision brain function measures, today announced a partnership to deploy NeuraLight’s brain function biomarker assessments across Mission’s multinational trial of MTX325 in Parkinson’s disease.
MTX325 is a first-in-class, orally bioavailable and brain-penetrant USP30 inhibitor designed to restore mitophagy, the essential process by which cells clear dysfunctional mitochondria to maintain cellular health. Impaired mitophagy and mitochondrial dysfunction are central drivers of Parkinson’s disease pathology. By restoring this crucial clearing mechanism, MTX325 has the potential to protect vulnerable neurons. In preclinical studies published in Nature, USP30 inhibition was shown to protect against alpha-synuclein-induced loss of dopaminergic neurons.
In this trial, NeuraLight’s platform will provide precise, objective biomarkers of brain function derived from measurements of eye movements. The biomarkers will quantify function across neural pathways affected in Parkinson’s disease, enabling Mission to assess changes in the brain function among the patients throughout the study. For neuroprotective programs such as Mission’s, this offers a sensitive, repeatable way to test whether a therapy’s biological activity translates into measurable clinical benefit, including subtle changes that conventional clinical scales may miss.
“Mission is excited to be part of this pioneering approach to assessing oculometric changes, and we are delighted to be working with the NeuraLight team,” said Sarah J. Fritchley, PhD, Chief Development Officer at Mission Therapeutics.
The partnership reflects the broader ambition behind Mission’s program: to move Parkinson’s treatment beyond symptom management and toward intervening in the biological processes that drive neuronal loss. MTX325 represents a differentiated approach to that challenge, while NeuraLight provides the functional measurement layer needed to explore whether restoring mitochondrial quality control translates into clinical benefit.
“Mission engineered MTX325 to protect neurons by restoring a fundamental process in the brain, and our platform is built to measure whether that protection shows up as preserved brain function,” said Edmund Ben-Ami, Co-Founder and Chief Executive Officer of NeuraLight. “That requires measurement sensitive enough to catch a signal in a small early-phase trial, and robust enough to hold up across a large, multisite one. Our goal is to bring that same precise readout to every patient at every site, however distributed the trial — turning brain function into a reliable, quantitative endpoint.”
About NeuraLight
NeuraLight is using precision measures of brain function to improve the development of therapies for neurological and neurodegenerative disease. Using AI and computer vision to derive quantitative biomarkers of brain function, NeuraLight’s platform is designed to bring objective, scalable measurement to CNS clinical trials across indications including Parkinson’s, Alzheimer’s, ALS, Huntington’s disease, and other disorders. For more information, visit www.neuralight.ai.
About Mission Therapeutics
Mission Therapeutics is a clinical-stage biotechnology company discovering and developing novel therapeutics that promote the removal of dysfunctional mitochondria, supporting cell health and function. Mitochondria require lifetime quality control through a clearance mechanism known as mitophagy; when this process fails, dysfunctional mitochondria accumulate and contribute to disease across multiple serious conditions, including Parkinson’s disease, heart failure, and others. Through inhibition of the mitochondrial deubiquitylating enzyme USP30, Mission’s small-molecule candidate — MTX325 (CNS) — is designed to restore mitophagy and improve overall cellular health. Headquartered in Cambridge, U.K., Mission is backed by investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group, and Rosetta Capital. For more information, visit www.missiontherapeutics.com.
About MTX325
MTX325 is a first-in-class, highly potent and selective, orally bioavailable and brain-penetrant USP30 inhibitor targeting improved mitochondrial quality. Mitochondrial dysfunction is a leading hypothesis in Parkinson’s disease pathophysiology, and inhibition of USP30 has been demonstrated to restore mitophagy and achieve neuroprotection. In preclinical assessments, MTX325 demonstrated impact on multiple aspects of Parkinson’s disease — mitochondrial dysfunction, alpha-synuclein accumulation, and loss of nigrostriatal dopaminergic neurons. Phase Ia SAD/MAD studies were completed in healthy volunteers, with CSF sampling and PET imaging confirming penetration into the brain. The compound is advancing into a Phase Ib proof-of-mechanism study in patients with Parkinson’s disease.
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