NRG Therapeutics doses its first-in-human phase 1 trial for NRG5051 to treat ALS/MND and Parkinson’s disease

"NRG Therapeutics Ltd, a clinical stage neuroscience company targeting a novel mechanism to rectify mitochondrial dysfunction in neurodegenerative diseases, announced that the first participants have been dosed in its first-in-human phase 1 clinical trial of its lead candidate NRG5051 which is being developed as a treatment for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) and Parkinson’s disease.

NRG5051 is a first-in-class, orally bioavailable and CNS-penetrant next-generation inhibitor of the mitochondrial permeability transition pore (mPTP), acting through a novel undisclosed mitochondrially-localized protein regulator.

This phase 1 randomized, double-blind trial is a combined single and multiple ascending dose trial of NRG5051 designed to assess safety, tolerability and pharmacokinetic parameters in healthy volunteers. It is being conducted at the Centre for Human Drug Research (CHDR) in Leiden, The Netherlands. 

The clinical trial is expected to readout by the end of 2026 and will inform dose selection in future studies in amyotrophic lateral sclerosis/motor neuron disease and Parkinson’s patients.

The mitochondrial permeability transition pore is a well-established driver of mitochondrial dysfunction, inflammation, and neuronal death in neurodegenerative disorders. Mitochondria are crucial for energy production, especially in substantia nigra neurons (Parkinson’s) and motor neurons (ALS/MND) which have high energy demands and consequently are particularly sensitive to mitochondrial health. The pathological proteins in amyotrophic lateral sclerosis/motor neuron disease (TDP-43) and Parkinson’s (a-synuclein) are toxic to mitochondria, driving mitochondrial dysfunction which is a common underlying pathology in these diseases. NRG5051 has been shown to be neuroprotective and anti-inflammatory in in vivo preclinical models of amyotrophic lateral sclerosis/motor neuron disease and Parkinson’s disease.

NRG Therapeutics’ 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 mitochondrial permeability transition pore 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.”

Amyotrophic lateral sclerosis/motor neuron disease is a rare, rapidly progressing neurodegenerative disease with high unmet medical need. In 2023 the US Food and Drug Administration approved Qalsody (tofersen) as a disease-modifying treatment for a rare genetic form of amyotrophic lateral sclerosis/motor neuron disease based on a biomarker (reduction in neurofilament light chain) and clinical data. However, patients with sporadic disease remain poorly treated by existing medicines. Parkinson’s is one of the fastest growing neurodegenerative conditions with a global prevalence predicted to double by 2050. There is currently no treatment available to slow or halt progression of Parkinson’s, with existing medicines providing temporary symptomatic relief only.

NRG Therapeutics is a clinical stage neuroscience company building a pipeline of disease-modifying mitochondrial therapeutics to slow or halt the progression of neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), and Parkinson’s disease.

NRG Therapeutics’ pipeline of small molecule assets is based on inhibiting the mitochondrial permeability transition pore (mPTP) through a novel mechanism of action. Inhibition of the mitochondrial permeability transition pore has been shown to protect neurons, reduce neuroinflammation and improve motor function in pre-clinical disease models. Its lead candidate, NRG5051, is currently in a phase 1 clinical trial.

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