NRG Therapeutics Launches First-in-Human Trial for Mitochondrial-Targeting ALS/Parkinson’s Drug

A clinical trial targeting mitochondrial failure could redefine the treatment landscape for neurodegenerative diseases—and aging itself.

NRG Therapeutics has initiated a Phase 1 clinical trial of NRG5051, an experimental drug designed to stabilize mitochondrial function in amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) and Parkinson’s. The trial, conducted at the Centre for Human Drug Research in Leiden, evaluates safety, tolerability, and pharmacokinetics in healthy volunteers using a randomized, double-blind design.

The drug works by inhibiting dysregulated processes within mitochondria, which are central to cellular energy production and a shared biological mechanism in neurodegeneration and aging.

This approach represents a shift from symptom-focused treatments to addressing the root causes of disease progression.

ALS currently has few disease-modifying therapies, while Parkinson’s prevalence is expected to double by 2050 due to aging populations. Mitochondrial dysfunction is a hallmark of both conditions, making NRG5051 a potential candidate in broader longevity science efforts.

Neil Miller, CEO of NRG Therapeutics, said:

"The commencement of our first clinical trial represented a significant achievement and signaled our transition into a clinical-stage organization."

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