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Ellorarxine (NVG645) is a synthetic small molecule retinoic acid receptor (RAR) modulator, specifically targeting the RARβ isoform, developed by Nevrargenics. It is designed to treat a range of neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS), motor neuron disease (MND), frontotemporal dementia (FTD), Parkinson's disease, and Alzheimer's disease. The compound exhibits neuroprotective, neuroplastic, and neuroregenerative properties by promoting neurite outgrowth, increasing MAP2 expression, and improving mitochondrial viability. It has demonstrated the ability to cross the blood-brain barrier and concentrate in the spinal cord. As of early 2024, the drug is in the IND-enabling stage and has received approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) to proceed with Phase 1 clinical trials using a 3+3 plus extension design.
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