Latest Research News
Here you can find the most up to date research news. Stay tuned to find out about clinical trial outcomes, breakthroughs in the lab, interesting research papers and more.
FDA announces the approval of AMX0035
29 September 2022
The Food and Drug Administration (FDA) have announced that they have approved AMX0035 for the treatment of MND in the United States, as RELYVRIO™. This approval is based off the data from the Phase 2 CENTAUR trial. Two countries have now approved AMX0035, with Canada conditionally approving the drug in June 2022. The FDA approval in the US is a welcome step forward in the fight against MND and further highlights the commitment and dedication of the research community in finding effective treatments. You can find out more about the approval in a press release. You can also find out more about what this approval means to people living with MND in the UK on our blog and also read some FAQs.
The Phase 3 clinical trial, called PHOENIX, is still recruiting in the UK. To find out where it is recruiting head to our AMX0035 webpage.
Top-line results from the HEALEY Platform trial for Verdiperstat announced
29 September 2022
The HEALEY center announced the first top-line results from the HEALEY Platform trial for the Verdiperstat arm. The primary endpoint of the regime was not met. This meant that participants randomised to Verdiperstat did not have a statistically significant slowing of disease progression as measured by ALSFRS-R over 24-weeks compared to participants randomised to placebo. There were also no statistically significant benefits for other measures including muscle strength, respiratory function and survival. Full study results are expected to be released later in the year. You can read more in a press release.
The HEALEY Platform trial is designed to accelerate the process of drug development and the path for effective treatment for MND by testing multiple drugs simultaneously and adaptively.
New study suggest changes are needed in genetic testing guidelines for MND
27 September 2022
Current guidelines in the UK state that only those diagnosed with MND who have a family history or have a young onset (under 40 years old) should be offered genetic testing. New research, published in Brain, found that under these current guidelines many people who have a genetic link to MND are not being offered genetic testing and their genetic link remains undiscovered. This means that these people would also not have access to gene therapies that are starting to emerge for MND. The researchers say that genetic testing and counselling should be offered to everyone who is diagnosed (or may be) with MND, regardless of age and family history. You can read more about this research over on our blog.
CNM-Au8 continues to show potential survival benefit in RESCUE-ALS open-label extension
25 September 2022
Clene Nanomedicine have announced additional data from the open-label extension of the RESCUE-ALS trial which is testing CNM-Au8 in people living with MND. Whilst the trial failed to reach its primary endpoint of reducing disease progression, the trial has continued to show a potential survival benefit over 144 weeks. This additional data is consistent with the findings which were announced in June and suggest that participants who received CNM-Au8 at the start of the trial had a 70% lower risk of death compared to those who received CNM-Au8 36-weeks later in the open-label extension. You can read more about these results in a press release from Clene or in a poster Clene presented at the 2022 AANEM Scientific Conference.
Published results from extended clinical trial of Tofersen shows promise in slowing MND progression
21 September 2022
Results from the Phase 3 clinical of Tofersen have been published in the New England Journal of Medicine. Tofersen is an experimental drug which has been designed specifically for people living with MND with an SOD1 mutation. The results showed that Tofersen was found to slow the progression for these people over a period of 12 months. Additionally, the drug was found to hit its therapeutic target by reducing the levels of toxic SOD1 protein. Researchers also saw a lowering in a protein called neurofilament light chain, which is a potential marker of nerve damage and could be an indicator of decline in disease progression. You can read more about the results of the trial over on our research blog.
Phase 2 clinical trial results of RT001 announced
15 September 2022
The results have the Phase 2 clinical trial for RT001 has been announced by BioJiva. RT001 is a investigational fatty acid and is expected to help make cells less vulnerable to damage and protect from degeneration. The 6-month placebo-controlled trial enrolled 43 people living with MND and was followed by a 6-month open label phase where everyone received the drug. The results showed a small slowing in disease progression as measured by ALSFRS-R. The results also suggested participants with more severe disease (defined as those who had an ALSFRS-R score of less than 41) had a larger slowing in disease progression. You can find out more about the results in a press release from BioJiva.
Whilst these results are promising it is important to note that this was a small trial with a small number of participants and the results will be need to be supported by a larger clinical trial.
Amylyx releases additional preclinical data for AMX0035
13 September 2022
Amylyx Pharmaceuticals announced a new publication showing preclinical data comparing AMX0035, which is the combination of Sodium Phenylbutyrate and TUDCA, to the individual components. In this preclinical data, they tested the combination of AMX0035 versus the individual components in ALS-derived cells. They found that AMX0035 changed the activity of many more genes involved in ALS-relevant pathways compared to either of the individual components alone.
AMX0035 has been tested in a Phase 2 clinical trial and is currently under review by the FDA in the US and a decision is expected by 29 September 2022. A Phase 3 clinical trial is also underway and is recruiting in the UK. Top-line results are expected in 2024.
TUDCA is currently being evaluated in a Phase 3 clinical trial. The trial is expected to read out top-line results in late 2023.
FDA Advisory Committee Vote for the approval of AMX0035 in the US
8 September 2022
Amylyx Pharmaceuticals have announced the outcome of the second FDA Advisory Committee meeting on AMX0035 as a treatment for MND. Overall, the panel voted for the approval of AMX0035 when asked whether the data from the Phase 2 Centaur trial and the additional information that has been published by Amylyx since March 2022 establishes that the drug was an effective treatment for people with MND. This is the second time that the panel has come together to vote about the approval of AMX0035, with the panel voting against the approval in the first meeting in March 2022. Since this first meeting Amylyx has released additional data from the Centaur trial and open label extension. You can read more about the Advisory Committee vote here.
Although the FDA considers the recommendations of its advisory committees, the recommendations of the panel are non-binding. The final decision regarding the approval of the New Drug Application for AMX0035 will be made by the FDA by 29 September 2022. You can read more about AMX0035 here.
Coya Therapeutics announces publication of phase 2a clinical trial results for COYA 101
01 September 2022
Coya Therapeutics have announced the results of a phase 2a trial of COYA 101 which show that the treatment is safe and well tolerated in people with ALS. This trial and open label extension also suggested that most participants given COYA 101 had a reduced decline in ALSFRS-R score and an increase in regulatory T-cell activity, which helps to reduce excessive inflammation. You can read more about this here.
Whilst these are promising results, the phase 2a trial only enrolled a very small number of participants and a larger trial is needed to fully assess the efficacy and long-term safety of COYA 101.
AB Science announces it has filed an application for conditional Marketing Authorisation to the EMA for Masitinib
25 August 2022
AB Science have filed an application to the European Medicines Agency (EMA) for a conditional Marketing Authorisation for Masitinib in the treatment of ALS. The application will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP) with a decision to be made whether to approve or not is aimed to be in 210 days. The application is based on the Phase 2/3 data from the AB10015 study. A conditional Marketing Authorisation means if the drug is approved, additional data will be needed from the ongoing Phase 3 trial to maintain the approval of the drug. You can read more here and find out more about Masitinib here.
New publication finds a new key deficit in MND
22 August 2022
MND Association funded Lady Edith Wolfson Fellow Dr Andrew Tosolini recently published a new paper which found selective deficits in a key transport system known as axonal transport. The paper highlighted that this transport system behaves differently in motor neurones in MND compared to healthy motor neurones. This work adds another crucial piece to the puzzle of what goes wrong in neurones in MND and will help in the development of therapies to slow MND in the future. You can read more here.
We sat down with Dr Tosolini to discuss this new research in more detail and you can read our chat and find our more about this research in a blog post here.
Real-world data analysis of survival with RADICAVA (edaravone) announced
15 August 2022
Mitsubishi Tanabe Pharma America have announced the publication of a paper which discusses the analysis of people living with MND taking Radicava (edaravone) in a real-world setting rather than that of controlled clinical trial. The data suggests that continued treatment with Radicava in people living with MND was associated with prolonged survival of an average of 6 months compared to those not treated with the drug. You can read more here.
Whilst real-world data is useful to continue to support approval of a new drug and bridge the gap between clinical trials and everyday practice, it is important to note that the data may be subject to bias since it is no longer under the randomised control of a clinical trial.
BrainStorm have announced they will submit a BLA for NurOwn
15 August 2022
BrainStorm have announced today that they will submit a Biologic License Application (BLA) seeking approval from the U.S. Food and Drug Agency (FDA) for NurOwn. You can read more here.
BrainStorm also announced a correction has been made to the Muscle and Nerve publication from December 2021. Analysis in the original publication used an incorrect model. This correction had an impact on one of the secondary endpoints, average change from baseline in ALSFRS-R, in the pre-specified subgroup of participants with a baseline score of at least 35. The data showed a statistically significant treatment difference of more than 2 points of those on NurOwn compared to those on placebo for that subgroup. You can read the correction here.
Encouraging laboratory results lead to feasibility trial to test Terazosin in MND
11 August 2022
Encouraging results from the laboratory have led to a small feasibility trial to test Terazosin in MND. The study, which has already opened for recruitment, will recruit 50 participants from the Oxford MND Care Centre. Terazosin is already approved to treat high blood pressure and benign prostatic hyperplasia, making this another example of how existing drugs could be repurposed for MND. Early research in the laboratory has shown that Terazosin protected motor neurones in models of MND by increasing energy levels. You can read more here and find out more about the early laboratory research in a publication here.
UK sees more MND clinical trials open for recruitment
05 August 2022
Over the past couple of months an additional four clinical trials have opened for recruitment in the UK. There is now a range of MND clinical trials that are open for recruitment including trials for people living with MND who have a specific gene mutation (such as FUS, C9orf72 and SOD1), as well as trials for those who don't or do not know. You can find out more about which trials are now open for recruitment and which sites are recruiting here.
MND Association holds inaugural MND EnCouRage UK event
28 July 2022
The MND Association recently held the inaugural MND EnCouRage UK event aimed at recognising, retaining and encouraging the important work of early career researchers and providing them with an opportunity to spend time with people living with MND. You can find out all the highlights in a blog post here.
FDA grants priority review for Tofersen for SOD1 MND
26 July 2022
Biogen have announced the Food and Drug Administration (FDA) have accepted the New Drug Application for Tofersen. They have also granted a priority review with a decision expected by January 25 2023. Tofersen has been designed for people living with MND with an SOD1 mutation which is known to affect about 2% of people living with MND. You can find out more here or read more about Tofersen here.
CNM-Au8 receives positive EMA opinion on Orphan Drug Status
22 July 2022
Clene have announced that CNM-Au8 has received a positive opinion from the European Marketing Agency (EMA) for their Orphan Drug Status submission. You can read more here or find out more about Orphan Drug Status here.
You can also find out more about CNM-Au8 here.
Viral protein HERV-K ENV is implicated in MND
21 July 2022
Ancient viruses (retroviruses), such as HERV-K, have left genetic material in our DNA and it is thought that the reactivation of this genetic material could cause MND. HERV-K contains the blueprint (genetic code) to make the HERV-K ENV protein which may be activated in people with MND.
Researchers have now found, in cell models, that treating neurones with HERV-K ENV led to a reduction in overall neurone count and the surviving neurones were damaged. The researchers also found that treatment with GN-K01, which is designed to bind to the protein, helped to prevent the toxic effects in cell models. You can read more here.
Further data reported from the RESCUE-ALS Open Label Extension Trial
14 July 2022
Clene Nanomedicine has announced additional survival data from the open label extension trial of CNM-Au8, known as RESCUE-ALS. The additional results suggested participants who were originally randomised to CNM-Au8 lived longer than those who received CNM-Au8 9 months later. Analysis from the trial has suggested a 70% decreased risk of death for participants originally randomised to CNM-Au8, but further studies are needed to investigate this further. You can read more here or find out more about CNM-Au8 here.
CNM-Au8 is also being investigated in the HEALEY Platform Trial, which investigates the treatment for a larger cohort of participants. Results from this trial are expected in the next couple of months.
FDA plans to reconvene Advisory Committee for approval of AMX0035
5 July 2022
Amylyx announced the Food and Drug Agency (FDA) have decided to reconvene the Advisory Committee to discuss the approval of AMX0035. The Advisory Committee originally met in March 2022 but voted against the approval. This new meeting, which is planned for 7 September 2022, will focus on the additional results recently published by Amylyx. You can read more here and find out more about AMX0035 and the additional results here.
MND Association funded researcher Tatyana Shelkovnikova receives prestigious fellowship from UKRI
22 June 2022
MND Association fellow Tatyana Shelkovnikova has received a prestigious fellowship from UK Research and Innovation (UKRI). The fellowship is known as the Future Leaders Fellowship scheme and rewards outstanding researchers and innovators. You can read more here.
MND Association helps fund new £4.24 million grant to kick-start UK-wide collaborative research effort
21 June 2022
A group of charities and government research organisations have awarded £4.25 million to MND experts at six UK universities to kick start efforts to end MND. This new ‘MND Collaborative Partnership’ brings together people living with MND, charities MND Association, LifeArc, MND Scotland and My Name’5 Doddie Foundation, government bodies Medical Research Council (MRC) and National Institute for Health and Care Research (NIHR), and researchers from King’s College London, University of Sheffield, University of Liverpool, University College London, University of Oxford and University of Edinburgh. The partnership team will work together to find solutions to address problems currently hindering MND research and aims to discover meaningful MND treatments within years rather than decades. You can read more about this here or on blog post here.
MND Association Research Team give their highlights from the 2022 ENCALS meeting
17 June 2022
Members of the research recently attended the 20th European Network for the Cure of Amyotrophic Lateral Sclerosis (ENCALS) meeting in Edinburgh, Scotland, on the 1-3rd of June 2022. Here you can listen to them chat with Head of Research Dr Nick Cole about the meeting, being back in-person and their research highlights.
Health Canada approves ALBRIOZA (AMX0035) for the treatment of ALS
13 June 2022
Health Canada has approved ALBRIOZA (AMX0035), with conditions, for the treatment of ALS. This approval is subject to conditions, including being dependent on the safety and efficacy data from the ongoing Phase 3 PHOENIX trial. Topline results from the PHOENIX trial are expected in 2024. You can read more about the approval here.
The Food and Drug Administration (FDA) in the US and the European Medicines Agency (EMA) in the EU are both still reviewing AMX0035 for approval. The FDA approval decision is expected by 29 September 2022 and the EMA approval decision is expected in early 2023. You can find out more about AMX0035 here.
The Phase 3 trial, called PHOENIX, is currently underway in the US and some of Europe. The trial is currently 'in preparation' in the UK and we will update the community with more information when we have it.
Registration for the 33rd International Symposium on ALS/MND opens
6 June 2022
Registration for the 33rd International Symposium has opened. The event will be held online from Tuesday 6 December 2022 to Friday 9 December 2022. Registration costs £60. You can find out more and register here.
Are you a researcher and want to present your work? Abstract submission is also now open, so make sure you submit your abstract before 12 July. You can find out more information here.
The FDA has extended the approval decision date for AMX0035 to the end of September
3 June 2022
The FDA has extended the approval decision date, otherwise known as the Prescription Drug User Fee Act (PDUFA) goal date from 29 June to September 29 2022. The FDA has extended the date to allow more time to review additional analyses of data. You can read more here.
Biogen announces 12 month data from VALOR Phase 3 trial
3 June 2022
Biogen have announced 12 month data from the open label extension of the Phase 3 trial of Tofersen (VALOR) at the 2022 ENCALS meeting. The additional results from the open label extension suggested participants who were originally randomised to Tofersen had a reduced decline in ALSFRS-R compared to those who received Tofersen 6 months later. You can read more here or access the presentation from ENCALS here. You can also find out more about Tofersen here.
Eledon announces topline results from Phase 2a trial of Tegoprubart
31 May 2022
Topline results from the Phase 2a trial of Tegoprubart have been announced, showing the drug to be well-tolerated. The trial also found that the drug demonstrated dose dependent target engagement and inflammatory biomarkers associated with ALS were observed and reduced. You can read more here and find out more about Tegoprubart here.
Find out about this weeks MND/ALS research publications
25 May 2022
Each week we will be adding a list of the latest research papers published relating to MND/ALS. This week includes papers on new potential treatments such as WVE-004, clinical trial designs, biomarkers, TDP-43, SOD1 and much more. You can read the full list here.
Gene therapy reduces toxic RNA in preclinical studies
23 May 2022
A gene therapy known as CRISPR, which can be used to alter the genetic sequence within a cell, has been shown to reduce the buildup of toxic RNA (RNA is the photocopy of DNA which is used to make proteins) molecules found in C9orf72 MND. The researchers suggested that the therapy only affected the toxic C9orf72 RNA, meaning that the C9orf72 RNA needed for the function of motor neurones could still be produced. You can read more here. You can also find out more about CRISPR on our research blog here.
Find out about this weeks MND/ALS research publications
20 May 2022
Each week we will be adding a list of the latest research papers published relating to MND/ALS. This week includes papers on gene therapies, biomarkers, TDP-43, SOD1 and much more. You can read the full list here.
Further results from the AMX0035 CENTAUR trial reported
16 May 2022
Further results from the AMX0035 CENTAUR trial have been reported, including data up to 35 months after initial randomisation. The results suggested that the risk of events such as, trachestomy, permanent ventilation and hospitalisation was reduced by 47% for those on the trial who were originally randomised to the AMX0035 group. You can read more here.
FDA approves oral form of edaravone for treatment of ALS
13 May 2022
The Food and Drug Administration (FDA) has approved an oral form of edaravone known as Radicava ORS in the US. Radicava ORS is an orally administered version of Radicava, which was approved by the FDA in 2017 as an intravenous infusion. Radicava ORS can be self-administered at home. You can read more about the approval here.
Radicava (edaravone) is currently not approved for use in the UK. However, a new trial, called ADORE, which is investigating an oral form of Edaravone (FAB122) recently starting recruiting in Europe. Trial sites in the UK are currently 'in preparation'. You can find out more here.
Find out about this weeks MND/ALS research publications
13 May 2022
Each week we will be adding a list of the latest research papers published relating to MND/ALS. This week includes papers on respiratory onset MND, glucose metabolism in MND, TDP-43, SOD1 and much more. You can read the full list here.
Further survival data reported from AMX0035 Centaur trial
6 May 2022
New statistical analysis of the data from the Centaur trial has suggested a survival benefit of 10.6 months for those who were originally randomised to AMX0035 and continued treatment in the open label extension compared to those who never received AMX0035. This analysis, know an “rank-preserving structural failure time model”, estimates what the survival difference would have been if those who had initially been given placebo had stayed on placebo throughout the study rather than switching to AMX0035 in the open-label phase. You can read more here and find out more about AMX0035 here.
WVE-004 demonstrates proof-of-concept in preclinical studies
5 May 2022
WVE-004, an experimental therapy for people living with ALS who have mutations in the C9orf72 gene has shown proof-of-concept in preclinical studies. Researchers found that WVE-004 reaches the tissues affected in C9orf72 ALS in mouse models. They also found that the therapy reduced levels of biomarkers, such as poly-GP. You can read more here. You can read more about the FOCUS-C9 trial of WVE-004 in humans here.
Treg therapy ALS001 may ease oxidative stress
27 April 2022
A phase 1 trial investigating Treg cell therapy, ALS001, has shown promising results. The trial evaluated critical biomarkers of neuroinflammation and oxidative stress, showing some suppression of oxidative stress. The researchers also discussed the potential future ability to select ALS/MND patients who exhibit a greater chance of therapeutic response to Treg cell therapy. You can read more here.
Researchers identify that mitochondrial dysfunction is one of the first things to go wrong in SOD1-ALS
6 April 2022
Researchers in Germany have suggested that dysfunction of the powerhouse of the cell, the mitochondria, is one of the first steps in a cascade that leads to SOD1-ALS. They found that this dysfunction occurred before elevated levels of the aggregated SOD1 protein, the known hallmark of SOD1-ALS, was observed. You can read more about the research here.
Testing for both drugs in MND-SMART to be continued after stage 1 review
5 April 2022
The independent trial committee for the MND-SMART clinical trial recently reviewed stage 1 data for the drugs, memantine and trazodone, currently being tested in the trial. They decided that testing should continue for both drugs to determine if either drug slows the progression of MND. You can read more about the review here and find out more about the MND-SMART trial here.
Wave Life Sciences announces interim results for the FOCUS-C9 clinical trial
4 April 2022
Wave Life Sciences announced interim results for the FOCUS-C9 trial which is investigating the use of WVE-004 for people living with MND. WVE-004 is a drug specifically designed for MND caused by a mutation in the C9orf72 gene. The results showed a reduction in a biomarker known as Poly(GP) with low single doses of WVE-004. Further data is expected from the trial throughout 2022. You can read more about the results here and find out more about the trial in a recent blog post here.
FDA Advisory Committee vote against the approval of AMX0035 in the United States
31 March 2022
Amylyx Pharmaceuticals have announced in a press release the outcome of the FDA Advisory Committee meeting on AMX0035 as a treatment for MND. Overall, the panel voted against the approval of AMX0035 when asked whether the data from the Phase 2 CENTAUR trial of AMX0035 establishes 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 the New Drug Application for AMX0035 will be made by the FDA by June 29 2022. You can read more about AMX0035 and both the Phase 2 and Phase 3 trial here.
Biogen and Ionis announce the discontinuation of clinical programs of BIIB078,a gene therapy for C9orf72 ALS
28 March 2022
Biogen and Ionis Pharmaceuticals have announced that they are discontinuing the clinical program for their investigational gene therapy for ALS associated with a C9orf72 mutation. Top-line results from the phase 1 study of the antisense oligonucleotide (ASO) revealed no significant differences between those on the ASO and those on the placebo, meaning that it failed to show any clinical benefit for those with ALS. You can read more about this in the press release.
More data from RESCUE-ALS presented at Muscular Dystrophy Association (MDA) Annual Meeting
17 March 2022
Additional results from the RESCUE-ALS trial, which investigated CNM-Au8 as a potential treatment for ALS, were announced at the MDA annual meeting. These results included evidence of significant survival benefit when compared to the validated ENCALS (European Network for the Cure of ALS) survival prediction model. Additional analysis of the MUNIX scores found that the score decreased less for those with limb-onset ALS when taking CNM-Au8 compared to placebo. You can read more in the press release and find out more about the trial.
TRICALS announces that the MAGNET platform trial has started recruitment
15 March 2022
TRICALS, the largest European research initiative to find a cure for ALS, has announced that the first participants have been recruited for the MAGNET platform trial. You can read more in the press release and find out more about the platform trial.
Zilucoplan arm in the HEALEY Platform trial ends early
1 March 2022
UCB have announced that the Zilucoplan arm in the HEALEY Platform trial has ended early after showing a lack of benefit. The platform trial is adaptive meaning it has early stopping rules and other features to accelerate the testing of investigational treatments for MND. You can read more on Massachusetts General Hospital website and find out more about the trial.