NRG Therapeutics has dosed the first participants in its Phase I clinical trial of NRG5051, being developed as a disease-modifying treatment for amyotrophic lateral sclerosis / motor neuron disease (ALS/MND) and Parkinson’s.
The ongoing, first-in-human, double-blind, randomised study combines single and multiple ascending dose cohorts to assess the pharmacokinetic properties, safety and tolerability in healthy volunteers.
NRG5051 is an orally bioavailable, central nervous system (CNS)-penetrant inhibitor of the mitochondrial permeability transition pore (mPTP), acting via an undisclosed mitochondrially-localised protein regulator.
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The trial is being conducted at the Centre for Human Drug Research (CHDR) in Leiden, the Netherlands.
Readout from this clinical trial is anticipated by 2026-end and will inform dose selection for subsequent studies involving ALS/MND and Parkinson’s patients.
The mPTP is recognised as a contributor to mitochondrial dysfunction, inflammation and neuronal death in neurodegenerative disorders.
Mitochondria play an essential role in energy production, particularly in substantia nigra neurons affected by Parkinson’s and motor neurons impacted by ALS/MND.
NRG co-founder and CEO Neil Miller said: “The start of our first clinical trial marks a proud moment for NRG as we transition into a clinical stage company.
“Our ultimate objective is to establish the therapeutic efficacy of our novel mPTP inhibitors as disease-modifying medicines that are designed to slow or prevent the progression of neurodegenerative diseases, and this first-in-human trial is a significant step toward that goal.”
ALS/MND remains a rapidly progressing neurodegenerative condition with significant unmet medical need.
The US Food and Drug Administration (FDA) granted approval for Qalsody (tofersen) in 2023, for use in individuals with rare genetic forms of ALS/MND.
The number of Parkinson’s disease cases worldwide is expected to double by 2050.
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