NRG Therapeutics Presents Data on Lead Clinical Asset NRG5051 Confirming Novel Disease-Modifying Approach to ALS Intrado

Poster presentation at the ALS Association’s ALS Nexus conference, August 23-26
Disclosed that NRG5051 acts by inhibiting the mPTP through a novel NLRX1-mediated mechanism of action
Data showed that NRG5051 displays profound anti-inflammatory and neuroprotective effects in ALS mouse models

STEVENAGE, United Kingdom, Aug. 24, 2026 (GLOBE NEWSWIRE) -- NRG Therapeutics Ltd. (“NRG”), an innovative clinical stage neuroscience company targeting a novel mechanism to restore mitochondrial dysfunction in neurodegenerative diseases, today presented a poster on preclinical and mechanistic data of its lead clinical asset NRG5051 which is being developed as a treatment for amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), and Parkinson’s.

In the poster NRG has disclosed that NRG5051, a first-in-class, orally bioavailable and CNS-penetrant next-generation inhibitor of the mitochondrial permeability transition pore (mPTP), acts through a novel NLRX1-mediated mechanism of action.

The scientists at NRG and their collaborators at WEHI are among the first to demonstrate that NLRX1 is essential for mPTP opening, establishing it as a key component or regulator of pore function. NLRX1 belongs to the NOD-like receptor family, a group of proteins that sense cellular stress and regulate immune responses. NLRX1 is unique within this family in being localized to the mitochondria.

The mPTP is a key inner mitochondrial membrane channel implicated in the toxic effects of misfolded TDP43, which accumulates in mitochondria and triggers energetic failure and a damaging interferon response. Data presented, from research part funded by the ALS Association and Innovate UK, confirmed that NRG5051 displays profound anti-inflammatory and neuroprotective effects in acute NLS-TDP43 ALS mouse models, including reduced plasma neurofilament light (NfL) chain, a translatable fluid biomarker of neuronal damage.

NRG5051 is currently in a Phase 1 randomized, double-blind clinical trial to assess safety, tolerability and pharmacokinetic parameters in healthy volunteers. Dosing in ALS patients is scheduled for later this year which will inform dose selection for future Phase 2 proof-of-concept trials in ALS patients planned for 2027.

NRG Therapeutics’ co-founder and CEO Neil Miller said, “Alongside advancing NRG5051 through preclinical studies and now into human clinical trials, we are pleased to have unravelled its novel mode of action in modulating the mPTP. Prevention of mPTP opening, via NLRX1, restores mitochondrial integrity and protects the function of vulnerable neurons. NRG5051 has the potential to be a truly disease-modifying medicine, offering a major step forward in our goal to slow or prevent the progression of ALS and other neurodegenerative diseases.”

ALS is a rare, rapidly progressing neurodegenerative disease with high unmet medical need. Despite recent success in treating patients with the very rare SOD-1 genetic form of ALS, the majority (90%) of patients with sporadic disease remain poorly treated by existing medicines.

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About NRG Therapeutics – https://www.nrgtherapeutics.com

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.

The Company’s pipeline of first-in-class small molecules is based on inhibiting the mitochondrial permeability transition pore (mPTP) through a novel mechanism of action. Inhibition of the mPTP has been shown to protect neurons, reduce neuroinflammation and improve motor function in preclinical disease models. Its lead clinical asset, NRG5051, is currently in a Phase 1 clinical trial.

Based at the Stevenage Bioscience Catalyst (SBC), UK, NRG Therapeutics is a private company with equity investment from Brandon Capital, British Business Bank, Criteria Bio Ventures, Dementia Discovery Fund, M Ventures, Novartis Venture Fund, Omega Funds and Parkinson’s UK. The company has also received awards from Innovate UK (Biomedical Catalyst Award), The Michael J. Fox Foundation, Target ALS and The ALS Association to support its innovative R&D programmes.