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NRX-1075

Development stage: Preclinical
Lead developer: NurrOn Pharmaceuticals
Modality: Small Molecules

Overview

NRX-1075 is a small molecule Nurr1 activator being developed by NurrOn Pharmaceuticals for the treatment of Friedreich's ataxia. Nurr1 (Nuclear receptor related 1 protein) is a transcription factor essential for the development and maintenance of neurons and the regulation of neuroinflammation. NRX-1075 works by enhancing Nurr1's dual functions: increasing the transcriptional activation of neuroprotective genes and enhancing the transrepression of neurotoxic proinflammatory gene expression. This dual mechanism is intended to provide neuroprotection and restore cellular function in patients with Friedreich's ataxia. As of early 2024, the compound is in the preclinical stage, with the company focusing on IND-enabling studies.

Targets

NR4A2 (Nuclear receptor subfamily 4 group A member 2)

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