Further update on ellorarxine

There has been ongoing coverage in the UK media of an early-stage compound, ellorarxine, that targets the retinoic acid receptor (RAR). We provided information on this compound in January 2025.

RAR has been proposed as a potential target for intervention in motor neuron disease (MND) and frontotemporal dementia (FTD) and the company Nevrargenics has produced a series of novel compounds that modulate this system.

Understanding the mechanism(s) underlying MND is essential if we are to find ways of slowing, or stopping, the disease. Investigating how RARs might fit into these biological processes is important, and identification of new targets, which provide possible points of intervention, is crucial for the development of potential new treatments for MND. However, based on the currently published research, we believe that ellorarxine is still at an early stage of development and that the necessary approvals are not in place to facilitate the fast-tracked timescales currently being shared in the media.

Taking a new compound into clinical trials requires a substantial body of research that provides a strong evidence base indicating that it does interfere with MND relevant systems. This is usually undertaken in cell-based models of the disease (rodent and human cells) as well as animal models of the disease. It also requires evidence that the compound is likely to be safe for people to receive.

Once there is enough evidence to suggest the compound convincingly interferes with MND disease progression in research models, an application can be made to undertake a clinical trial in people. This would usually start with a safety trial in healthy people (phase 1), but sometimes this may include people with the disease. Either route requires approval from the Medicines and Healthcare products Regulatory Agency (MHRA).

It has been established that, despite the statements made by Nevrargenics, there is currently no MHRA approval for a clinical trial of ellorarxine in the UK. At the end of January, the company updated their website to remove mention of trial approval, but this assertion is still present in the many media articles and coverage published last month.

As with all MND charities, we remain committed to funding research in a manner which is safe and appropriate for the community. Every application we receive goes through robust review processes, involving a diverse panel of multidisciplinary experts, to ensure we are spending the money our supporters have generously raised to fund the very best and most promising research.

Over the last five years, the three main MND charities have been proactively campaigning for UK Government investment in MND research. Three years ago, we secured £50 million of funding through our collaborative campaign, alongside people with MND. This investment is now starting to generate new research, including the recent launch of a new rapid drug prioritisation trial for MND in the UK, EXPERTS-ALS.

The charities are also committed to directly funding research ourselves, including the MND-SMART clinical trial, which is now the largest MND trial in UK history. This is increasing the pace of research in the UK, and it has only been achieved by us working together, with the UK research community, with people with MND, and the relevant organisations and regulators.

If you would like more information about the process by which research becomes a treatment, MND Scotland, the MND Association and My Name’5 Doddie Foundation recently produced a series of infographics to explain the pathway a new drug needs to navigate before it can become a treatment. This can be found here.