12 MND clinical trial updates to keep an eye out for in 2022 – Part 2

12 MND clinical trial updates to keep an eye out for in 2022 – Part 2

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This year is set to be an exciting time for MND research, and we are especially hopeful that MND clinical trials will take centre stage in the coming months. During 2022 we are expecting new trials to begin recruitment in the UK, other trials to readout their results and decisions announced from authorising bodies for potential treatments that have completed trials for MND.

We know how important being up to date on the latest trials is to the MND community so we will always endeavour to keep you updated with all the latest news and updates. For the latest information you can follow us on twitter, sign up to our research monthly newsletter or check out our latest news page.

In part two of this blog series, we delve into the trials that are expected to release topline results and look into the drugs that are awaiting approval decisions from authorising bodies, such as the FDA and EMA. Haven’t caught up on part 1 of this blog yet? You can read it here.

Topline Results Expected in 2022


Inflammation in the brain and spinal cord is thought to contribute to MND progression. Our bodies can help to control inflammation through a type of immune system cell called a Regulatory T Cell (Treg). Research has shown that Interleukin-2 (IL-2) can help to control Tregs and low doses have been shown to increase the number and function of Treg cells in the blood. A previous clinical trial, called IMODALS, investigated the use of low doses of IL-2 in people living with MND. The trial showed that the number of circulating Tregs was increased in people living with MND. You can read more about the results of this trial in a previous blog here. Building on this trial and looking further into the impact on disease progression and survival is MIROCALS (Modifying Immune Response and OutComes in ALS), a phase 2 trial, partly funded by the MND Association. The data of this trial is currently being analysed and topline results are expected in 2022. You can find out more about the trial here.

Blog | 16 March 2022 | Eleanor Green
Symposium Spotlight: A closer look at IMODALS clinical trial outcomes

HEALEY Platform Trial

The HEALEY platform trial aims to accelerate the process of finding an effective treatment for ALS by testing multiple drugs simultaneously and adaptively. The trial allows the participant to have a greater chance of being on the treatment rather than placebo. Since the trial started, 5 drugs have been added: zilucoplan, verdiperstat, CNM-Au8, Pridopidine and Trehalose. Showing the adaptive nature of the trial, the zilucoplan arm was stopped early as it showed no benefit in people living with ALS.

Early results from the verdiperstat, CNM-Au8 and pridopidine trials are expected in 2022. The results of the CNM-Au8 arm are eagerly anticipated after the read-out of the CNM-Au8 trial (RESCUE-ALS) in 2021. Although RESCUE-ALS didn’t meet its primary or secondary endpoints, there was some evidence of potential long-term survival benefit and reduction in disease progression. The hope is that this encouraging data will be further validated in the larger arm in the HEALEY platform trial. You can read more about the trial here.

AT-1505 (Tegoprubart)

This trial investigates AT-1501, which has recently been re-named as Tegoprubart. Tegoprubart is an antibody against a protein called CD40 ligand (CD40L), which is present on the surface of some immune cells. CD40L plays a role in regulating the immune response, which can trigger inflammation in the spinal cord. The CD40L pathway has been found to be overactive in people living with MND. It is hoped that inhibiting CD40L could block or delay the activation of the damaging inflammatory immune response. This phase 2a trial looks to investigate the safety and tolerability of tegoprubart at 4 different doses in people living with MND. This trial is an open label trial, meaning that everyone in the trial takes the treatment and there is no placebo control arm. Due to this, any efficacy results that may be drawn from the analysis of the trial will have to be treated with caution. The trial finished recruitment in early 2022 and is expected to read out early results later in the year. You can find out more about the trial here.

WVE-004 (FOCUS-C9)

The FOCUS-C9 trial investigates the use of a drug called WVE-004. This drug is specifically designed for MND caused by a mutation in the C9orf72 gene. It is an antisense oligonulecotide (ASO), which targets and interferes with the faulty genetic instructions for the C9orf72 protein. This results in the faulty instructions being destroyed, leaving the remainder of the instructions functional to allow for a healthy version of the C9orf72 protein to be made.

The phase 1/2 FOCUS-C9 trial main aim is to determine the safety and tolerability of WVE-004 at a range of different doses. Additional endpoints will also be investigated, including the levels of a key biomarker called Poly(GP). Poly(GP) is known to have consistent elevated levels in people living with C9orf72 MND, so a reduction in levels can indicate target engagement. Wave Life Sciences recently announced some interim data from the trial that showed a reduction in Poly(GP) with low single doses of WVE-004. You can read more about this data here. Further data is expected from the trial throughout 2022, including the effect of multiple doses of WVE-004 and the safety/tolerability results. The trial is still currently recruiting in the UK, you can find out more information here.

Approval Decisions Expected in 2022

AMX0035 – FDA and EMA

The FDA initially decided that a further trial was required before it could consider AMX0035 for approval. However, this was overturned in September 2021 and in December 2021 they announced that AMX0035 had been accepted for priority review for approval. This means that they will aim to review the drug in 6 months, compared to the normal 10 months. The FDA held an Advisory Committee meeting on 31 March 2022. Overall, the panel voted against the approval of AMX0035 when asked whether the data from the Phase 2 CENTAUR trial established that the drug was effective in the treatment of people with MND. Although the FDA considers the recommendations of its advisory committees, the recommendations of the panel are non-binding. The final decision regarding approval of AMX0035 will be made by the FDA by 29 June 2022.

Additionally, a market authorisation application (MMA) has been submitted to the European Medical Agency (EMA) for AMX0035. This is currently under review and a decision is expected this year. However, approval by the EMA, or any other authorising body, does not mean that the Medicines and Healthcare products Regulatory Agency (MHRA) – the authorising body in the UK – will approve licensing in the UK. The implications of Brexit have complicated the approvals process further in the UK and the arrangement currently in place is due to finish at the end of 2022, meaning that the procedure to approvals may change. We are in conversations with Amylyx about this and will update with further information when we have it.

You can read more about AMX0035 in the first part of this blog series here.

Blog | 25 Nov 2021 Mandy Spencer
Update on clinical trials – part 1

Blog | 26 Nov 2021 Mandy Spencer
Update on clinical trials – part 2

Oral Edaravone (MT-1186) – FDA

It was announced in January 2022 that a new drug application (NDA) for a formulation of oral edaravone, known as MT-1186, had been accepted for priority review by the FDA. This formulation of oral edaravone had recently completed a phase 3 clinical trial which showed that MT-1186 had a similar clinical profile, meaning that it behaved in a similar way, as the already approved intravenous form of edaravone, known as Radicava. You can read more about MT-1186 here. The target date for the FDA to complete its review, and decide if MT-1186 will be approved, is 12 May 2022. You can read about a clinical trial of another oral formulation of edaravone, known as FAB-122, in the first part of this blog series here.      

We know how important being up to date on the latest trials is to the MND community so as soon as we have any further information on these trial results and approval decisions, we will continue to communicate updates to you all. For the latest information you can follow us on twitter, sign up to our research monthly newsletter or check out our latest news page and treatment trials page.

I work in the Research Development team as a Senior Research Information Co-ordinator. I graduated from the University of Nottingham with a PhD in Chemistry in 2020. After finishing my PhD, I joined an R&D company based in Nottingham where I spent my time synthesising potential new drug treatments for a wide variety of diseases. As part of my role I will help the team communicate the latest MND research on this blog, as well as on our twitter page (@MNDResearch).

2 thoughts on “12 MND clinical trial updates to keep an eye out for in 2022 – Part 2

    1. Hi Damian,

      Thanks for your comment and we are sorry to hear of your diagnosis. You can find out more about clinical trials and their current recruiting status on our website here. Many of these trials will start recruiting in the UK in the coming months, we will make sure to update the community as soon as we know when and where these trials will be recruiting. If you do have any more questions please don’t hesitate to get in touch with us at

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