NRG Therapeutics presented a poster at the ALS Association’s ALS Nexus conference (23–26 August 2026) detailing preclinical and mechanistic data for NRG5051, its orally bioavailable, CNS-penetrant inhibitor of the mitochondrial permeability transition pore.
According to the company, NRG5051 acts through a novel NLRX1-mediated mechanism and showed profound anti-inflammatory and neuroprotective effects in acute NLS-TDP43 ALS mouse models, including reduced plasma neurofilament light, a biomarker of neuronal damage.
NRG Therapeutics disclosed that collaborators at WEHI helped demonstrate NLRX1’s role in mPTP opening and noted that the mPTP is implicated in the toxic effects of misfolded TDP43.
Research supporting the data was partly funded by the ALS Association and Innovate UK. NRG5051 is currently in a randomized, double-blind Phase 1 trial in healthy volunteers, with dosing in ALS patients scheduled later in 2026 to inform dose selection for Phase 2 trials planned for 2027.
The company claims NRG5051 has the potential to be a disease-modifying therapy for ALS.
