Here you can find the latest MND research news from around the world. Stay tuned to find out about clinical trial outcomes, breakthroughs in the lab, interesting research papers and more.
New imaging technique helps researchers explore why motor neurones die
9 August 2024
The new technique, developed at the University of Birmingham, enables proteins to be studied in greater detail than ever before. Proteins are vital for all the tasks our cells undertake and some proteins can become faulty in MND. Researchers at the Sheffield Institute for Translational Neuroscience (SITraN) have used this new technique to study the SOD1 protein. The detailed imaging identified the loss of metal ions, which play a crucial in how a protein functions, and clumping of the SOD1 protein in models of MND. The researchers now aim to investigate whether these changes can be treated in the models, which is the first step to developing a potential new treatment for people with MND. You can read more here.
Researchers investigate rare MND reversals
5 August 2024
Researchers at Duke University have identified a very small group people who have undergone what they describe as a MND (also known as ALS) reversal. These reversals are described as ‘a person who has regained significant or complete motor function having previously been diagnosed with MND’ and are very rare. The researchers are now trying to understand more about these reversals and whether genetic, environment or lifestyle factors could play a role.
The genetic makeup of 22 people with reversals was compared to people with more ‘typical’ MND. A change in the IGFBP7 gene was found to be more common in those with MND reversals. The researchers have theorised that this change leads to less IGFBP7 being made, which may make motor neurones more resistant to some of the mechanisms that lead to motor neurone death. You can read more about the research into MND reversals on our blog or in a recent publication.
Results of phase 2 trial of TPN-101 announced
24 July 2024
Transposon Therapeutics have announced the results of a phase 2 trial of TPN-101 in people with C9orf72 MND. The trial investigated TPN-101 against a placebo (dummy drug) for 24 weeks, in 42 people with MND. TPN-101 is designed to reduce inflammation and neurodegeneration. It is thought that ancient viruses have left their genetic material in our DNA during human evolution. Researchers have theorised that the build-up of proteins in MND activates this viral genetic material. This activation leads to an immune response, which can lead to nerve cell death. TPN-101 aims to reduce the activation of this genetic material.
TPN-101 was found to be safe and well tolerated. After 24-weeks, participants who received TPN-101 experienced a 50% less decline in vital capacity, a measure of respiratory function. However, no change in the ALSFRS-R was observed between those receiving TPN-101 and those receiving placebo, suggesting there was no change to disease progression. The study also looked at markers of inflammation and motor neuron damage (neurofilament). At the end of the study, these markers were found to be lower for those who received TPN-101 compared to those who received placebo. This small study hints at some potential benefit for people with MND, but further research is still needed. You can read more about the results here.
Results from trial investigating gene therapy BIIB078 published
23 July 2024
Back in March 2022, Biogen and Ionis Pharmaceuticals announced they were discontinuing the development of a gene therapy (BIIB078) for people with C9orf72 MND. The therapy was designed to reduce levels of toxic proteins observed in people with the C9orf72 gene change.
The full results from the trial have now been published. The trial was designed to find the best dose to take forward into larger trials. The researchers found that 60 mg and 90 mg decreased levels of toxic proteins – suggesting that the treatment was doing what it was designed to do. However, no significant difference in functional measures were found between the treatment and placebo. In fact, those receiving 90 mg of BIIB078 showed a potential increase in disease progression, as measured by ALSFRS-R. In both doses, the levels of a marker of motor neuron damage (neurofilament light chain) were found to increase, suggesting that more damage was happening to motor neurons.
While the results from this trial are disappointing, researchers will now try to understand why the treatment didn’t benefit people with MND, which will help in the development of the next generation of potential treatments. You can access the publication here.
Immune cell therapy shows promise in early studies
10 July 2024
Researchers at Massachusetts General Hospital have published results of early testing of an immune cell therapy which could be a potential treatment for ALS. It is thought that increased inflammation and changes in activity of the immune system might be involved in the development and progression of ALS. The therapy involved regular infusions of a type of immune cell, called a B cell. B cells are found within our bodies and are known to produce antibodies, help to reduce inflammation and support the repair of damaged cells. This immune therapy aims to increase the number and activity of B cells within the body.
Mice were given 10 infusions of B cells from healthy donor mice over 10 weeks. Results showed that repeated infusions of B cells were able to delay the onset, extend survival and reduce cell death in mouse models of the disease. The researchers then went on to test the potential immune therapy in one person with the disease, giving them two infusions of B cells from a healthy donor over 60 days. They found that the therapy was safe, led to an improvement of 5 points on the ALSFRS-R scale and decreased the levels of different markers of inflammation in the body. These results support plans for the potential immune cell therapy to be tested further in a phase 1 clinical trial. You can read more in a press release or read the publication.
Gene therapy improves signalling between motor neurones in early laboratory testing
05 July 2024
Researchers at University College London have shown that targeting the UNC13A gene may help to send signals between motor neurones, a process which is normally faulty in MND. TDP-43 is an essential protein which helps to keep cells healthy. However, in 97% of MND cases, this protein can become faulty and move to the wrong place in the cell, impacting the way the rest of the cell can function. Previous research has suggested that when TDP-43 is misplaced, it can affect how the UNC13A gene functions. The UNC13A gene forms the instructions for the UNC13A protein, which plays a key role in passing signals between neurons. When the gene is faulty, it results in a faulty UNC13A protein, which can lead to a reduction in signalling between neurons and neuronal death.
Researchers tested an experimental gene therapy in models of MND with misplaced TDP-43. The study showed the treatment increased signalling between neurons allowing them to continue functioning. This treatment could have the potential to protect muscle strength and functioning in people with TDP-43 associated MND. Future laboratory studies are now needed to continue to develop this new potential treatment. You can read more about the study here.
Additional results announced for the PARADIGM trial of PrimeC
01 July 2024
NeuroSense Therapeutics, the company behind potential treatment PrimeC, have announced additional data from the open label stage of the Phase 2b PARADIGM trial. The PARADIGM trial initial results were released in December 2023 and found that PrimeC was safe and well tolerated by people with ALS. PrimeC was shown to slow disease progression by 29% on the ALSFRS-R and slow decline in lung function by 13% for those who received the potential treatment compared to placebo. After 6 months on the PARADIGM trial, participants were given the opportunity to join the open label stage where all participants received the treatment (even those who were previously given the placebo).
After 12 months of the open label stage, there was found to be a 36% slowing in disease progression on the ALSFRS-R for those who received PrimeC throughout compared to those who were initially on placebo. Those who were on PrimeC from the start were also found to have a 43% improvement in survival when compared to those who were on placebo in the trial. These results help to show that PrimeC may be more beneficial for people with ALS when it is taken earlier and for longer. NeuroSense Therapeutics are planning a phase 3 trial of the potential treatment in the USA and Europe to fully understand whether it could be a beneficial treatment for people with ALS. You can read more in the press release.
Masitinib receives negative opinion from European Medicines Agency Committee
28 June 2024
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has announced a negative opinion for the conditional approval of Masitinib for the treatment of MND. The CHMP panel reviews the scientific evidence and forms an opinion on whether new drugs should be approved in the EU. The committee based their review on data from the phase 2 trial. AB Science, the pharmaceutical company behind Masitinib, have announced that they will request a re-examination. This process will take approximately 4 months. You can read more in a press release.
The EMA determines approvals for the EU. After Brexit, a new process (known as the International Recognition Procedure) has been put into place so that the regulator for Great Britain (England, Wales and Scotland), known as the Medicines Healthcare products Regulatory Agency (MHRA) may rely on a decision taken by other international regulators, such as the EMA, on the approval of new treatments.
A phase 3 trial of Masitinib is currently underway. You can find out more about Masitinib on our website.
Top line results of Phase 2a trial of Bravyl for MND announced
24 June 2024
Woolsey Pharmaceuticals have announced the initial results of a small phase 2a trial of Bravyl. People with MND have been shown to have higher levels of Rho kinase (ROCK) in their blood. Rho kinase plays an important role in regulating the shape, movement and growth of cells, as well as contributing to cell death and inflammatory processes. Bravyl is a Rho kinase inhibitor, which means it aims to reduce the levels of ROCK and could potentially slow neurodegeneration. The trial was open label, where all 31 participants received the possible treatment over 24 weeks.
The drug was found to be safe and well tolerated in people with MND. The study monitored levels of a marker of motor neuron damage, known as neurofilament light chain, and found after 6 months, levels were 15% lower compared to levels before the start of the study. Lower levels suggest that the disease is less active and hints at a slowing in disease progression. This potential slowing in disease progression was also shown in other measures, such as measures of respiratory function and muscle strength. You can find out more about the results here.
This study shows promising results, but the potential treatment was only tested in a small group of participants and there was no placebo group to compare results. Further testing is needed in a larger, placebo-controlled trial to find out if Bravyl is an effective treatment for MND.
Sano Genetics launch a new MND genetic testing and research initiative in the UK
12 June 2024
Sano Genetics have launched a new initiative called ‘Light The Way’ which is an online platform offering support for people diagnosed with or at risk of developing MND. It has interactive educational resources to help people to better understand the genetic links to MND, as well as offering genetic testing and counselling to a small number of people affected. Genetic counselling can help people to make a decision on whether they want to undergo genetic testing and explain the meaning of results if testing is done.
The platform also includes peer-to-peer support groups so that people can talk to others who are or have been in a similar situation to themselves. This peer and counselling support is available during and after the testing process. People will also be given the opportunity to take part in research studies and the ‘Light The Way’ team are conducting a study as part of this programme. The study, called Beacon, will collect information on the wellbeing of people over 9 months. This will help to improve understanding of the psychological impact of genetic testing and risk. You can find out more about it here.
Researchers uncover more about the role of FUS protein in MND and Frontotemporal dementia (FTD)
11 June 2024
Researchers at the VIB-KU Leuven Center for Brain & Disease Research have shed more light on the role that a protein called FUS might play in some cases of MND. The FUS protein can become faulty in some forms of MND and FTD and forms toxic clumps in the neurons. This new study has revealed more information on how these clumps of FUS behave in the diseases. The researchers injected clumps of FUS into mice who had been engineered to make the human form of the FUS protein.
They found that the injected toxic clumps of FUS caused further clumping of FUS within the neurons and that this spread to other areas of the brain. The spread of FUS clumps contributed to cell damage and cognitive and behavioural changes in the mice. This suggests that clumps of human FUS protein can cause the formation of other clumps in cells, like a domino effect, and these can spread throughout the brain to cause damage to neurons. Further research is needed to understand more about how and why FUS spreads throughout areas of the brain to discover whether the spread, and damage to the cells, can be stopped or delayed. You can read more about this here.
New top line results of a phase 1 trial of CTLA4-Ig and interleukin-2 for MND announced
10 June 2024
Coya Therapeutics have announced the initial results of a small phase 1 trial of a combination of two compounds called CTLA4-Ig and interleukin-2. The potential treatment is thought to help reduce inflammation in the brain by promoting anti-inflammatory behaviour of immune cells called T-regs. It was tested in an open label study, where everyone received the possible treatment, in 4 people with MND. Participants were given the drugs every 2 weeks for a total of 48 weeks.
The drugs were found to be safe and well tolerated by people with MND. The anti-inflammatory functions of the T-regs were found to increase throughout the trial, before dropping to normal levels 6 weeks after the last treatment. During the 48 weeks of treatment, the change in ALSFRS-R score was on average -0.13 points/month which suggests that the potential treatment might help to slow disease progression. The study also looked at biomarkers of oxidative stress, inflammation and motor neuron damage (neurofilament light chain). These were all found to decrease in most participants during the study. This provides further evidence to suggest that the combination therapy might be beneficial for people with MND. You can find out more about the results here.
This study shows promising results but it only tested the possible treatment in 4 people with MND and there was no placebo group in this trial to compare results. It suggests that further testing is needed in a larger, placebo controlled trial to find out whether it may be an effective treatment for MND.
Research finds the MIF protein might slow disease progression in MND
06 June 2024
Researchers at Ben-Gurion University of the Negev in Israel have shown that increasing levels of a protein called MIF (macrophage migration inhibitory factor) might be able to slow disease progression. Previous research has suggested that MIF blocks clumps of toxic SOD1 protein forming in SOD1 MND. In this study, the team tested the effects of increasing the MIF protein in mice with SOD1 MND. MIF levels were increased by injecting the gene into the blood of the mice so that the gene could be taken up by cells of the nervous system. This gene was used to make more MIF protein in neurons.
The study found that mice treated with the gene showed preserved muscle strength and motor function, slower disease progression, and extended survival compared to untreated mice. The scientists then also looked at MIF levels in motor neurons made from stem cells of people with different gene changes linked to MND. They found that MIF levels were decreased in the different types of MND and not just SOD1 MND. This suggests that lower levels of MIF might play a role in the disease for most people with MND. Further research is needed to fully uncover how increased MIF slows disease progression and how the findings might be used to develop new potential treatments for MND. You can read more about the study here.
Biogen announces the approval of Tofersen by the European Commission
30 May 2024
The European commission has granted a marketing authorisation for Tofersen for the treatment of SOD1 MND. Following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in February, the European Commission have made the final decision to approve the treatment. This approval was based on data collected from the Phase 3 VALOR study and open label extension.
Tofersen, also known as QALSODY, has been approved under exceptional circumstances. This route is used when the benefit of a treatment is found to be positive but the data may not be enough for full approval, due to the rare nature of the disease.
This means that people with SOD1 MND in the EU will soon be able to access Tofersen. Biogen, the company behind the drug, are working with clinicians and local authorities to bring the treatment to people in the EU as quickly as possible. You can read more about this in a press release.
Tofersen is not currently available in the UK and it is still being assessed by NICE, who advise whether a new treatment should be made available on the NHS.
Online platform to support carers of MND moves closer to testing
20 May 2024
Researchers from the University of East Anglia, and supported by the MND Association, have completed a feasibility study of an online platform which aims to support carers of people with MND who have behavioural symptoms. Changes in behaviour can happen in around 50 - 75% of people with MND and negatively impact the wellbeing of their carers. The MiNDToolkit is an online platform aimed at supporting carers in managing behavioural symptoms in MND.
There have not been any other research studies investigating a platform in managing behavioural symptoms for carers of people with MND. Therefore, researchers conducted a feasibility study to help check how feasible it would be to undertake a randomised control trial, which is needed to determine how beneficial the platform is. The study had high retention, with over 80% completing the study. Additionally, the platform had high engagement and was well accepted by carers. The researchers stated that based off this data, the MiNDToolkit should be tested in a randomised clinical trial to determine how effective it is for carers. This study will also help to design the trial to hopefully increase its chance of success. You can read more here.
Results of Phase 1/2 trial for BIIB105 announced
16 May 2024
Ionis Pharmaceuticals and Biogen have announced that the topline results of the phase 1/2 trial of BIIB105. Based on the data from the trial, Ionis and Biogen have decided to stop the development of BIIB105.
BIIB105 was designed to reduce levels of ataxin-2. Previous research has shown ataxin-2 helps to drive the formation of toxic clumps of TDP-43, which are found in 97% of all MND cases. BIIB105 was found to reduce the levels of ataxin-2, however it was not found to have any clinical benefits to people with MND. Levels of a marker of nerve damage (neurofilament light chain) were also not reduced, suggesting that BIIB105 did not reduce damage to motor neurones. You can read more here.
Researchers identify new potential target for MND therapy
15 May 2024
Researchers at Western University, in Canada, have found that a protein called NF242 can interact with another important protein in MND called TDP-43. When these proteins interact with each other, in models of MND, they were shown to reduce damage to motor neurons.
TDP-43 is found to be abnormal in around 97% of people with MND. It forms toxic clumps which leads to motor neuron damage and death. Researchers have theorised that reducing the toxicity of TDP-43 could slow disease progression. This research shows that NF242 could be useful in reducing TDP-43 toxicity and potentially be beneficial for people with MND. Further research is now needed to continue to test NF242 and its potential benefit. You can read more here.
New psychological therapy shows promise in improving quality of life for people with MND
10 May 2024
Researchers from the University of Sheffield and University College London have found a new psychological intervention improves quality of life for people with MND when used alongside existing care. The new intervention, called Acceptance and Commitment Therapy (ACT), combines aspects of cognitive behavioural therapy (CBT) with acceptance and mindfulness-based strategies. ACT helps people to learn new ways of handling distressing thoughts and feelings.
191 participants were enrolled into the study and were randomly assigned to receive either ACT plus usual care or just usual care. The trial found ACT and usual care together maintained or improved the quality of life of people with MND after six and nine months compared to those who only received usual care. You can read more about the results in a press release.
The MND Association is now committing to facilitate ACT training for psychologists who work with people with MND. You can read more here.
Wearable sensors could help monitor MND disease progression
6 April 2024
Researchers from the University Medical Centre Utrecht have found that data from wearable sensors could be used to predict disease progression and survival. In the study, people with MND wore a sensor on their hips, for three to seven days. This was then repeated every two to three months for up to 18 - 24 months. The researchers suggest this study provides evidence to support the use of wearable sensors in clinical trials, to help monitor any treatment effects. However, they suggest that wearable sensors could not be used on their own, and could only be used to help support other measures of disease progression.
Using wearable sensors allows for additional data to be collected at home, rather than in the clinic, which may enhance the detection of treatment effects. You can read more here.
Amylyx to voluntarily remove AMX0035 from the market
4 April 2024
Based on the negative results from the Phase 3 clinical trial (PHOENIX), Amylyx have decided to voluntarily discontinue the marketing authorisations for AMX0035 (also known as RELYVRIO and ALBRIOZA) in the US and Canada. AMX0035 is no longer available for new patients. Amylyx also announced that the open label extension from PHOENIX will continue, to allow for further data to be collected and analysed. You can read more in a press release.
AMX0035 was approved for use in the US and Canada in 2022, based on the data from the Phase 2 CENTAUR clinical trial. Earlier this year the European Medicines Agency recommended against the approval of AMX0035 in Europe. You can read more about AMX0035 here.
Phase 3 clinical trial of TUDCA does not meet primary endpoint
27 March 2024
The TUDCA consortium have announced that the phase 3 clinical trial of TUDCA did not meet its primary endpoint. The primary endpoint of the trial was to observe a greater than 20% reduction in the slope of the ALSFRS-R score after 18 months. This would indicate a reduction in disease progression. However, this was not achieved by the end of the study. A statistically significant difference between treatment and placebo groups was also not observed for survival and a marker of nerve damage, called neurofilament light chain. Reductions in this marker can indicate a reduction in damage to motor neurones. Read more in a press release and read our statement with MND Scotland and My Name’5 Doddie here.
Trehalose arm of the HEALEY Platform trial does not meet primary or secondary endpoints
19 March 2024
Top-line results from the trehalose regiment of the HEALEY Platform trial show that the trial did not meet its primary or secondary endpoints. This suggests that there was no significant change in ALSFRS-R scores (from baseline to 24 weeks) for those who received the treatment compared to those who received placebo (dummy drug). There were also no differences found in secondary endpoints, such as muscle function and respiratory measures. You can read more in a press release.
The HEALEY Platform trial is designed to accelerate the process of drug development and identifying effective treatments for MND by testing multiple drugs at the same time and being able to easily remove drugs that are not found to be beneficial and to quickly add new drugs for investigation.
Phase 3 clinical trial of AMX0035 does not meet primary or secondary endpoints
8 March 2024
Amylyx Pharmaceuticals have announced that the phase 3 clinical trial of AMX0035 (PHOENIX) did not meet its primary or secondary endpoints. This suggests that there was no significant change in ALSFRS-R scores (from baseline to 48 weeks) for those who received the treatment compared to those who received the placebo (dummy drug). There were also no differences found in secondary endpoints, such as quality of life and muscle function measures, between those who were given AMX0035 compared to those on placebo. The full results will be published later this year.
AMX0035, also known as Relyvrio and Albrioza, was approved for MND in the USA and Canada in 2022. The initial results of this phase 3 trial may mean that the drug is withdrawn from the market in these countries. Amylyx will discuss the results with regulatory authorities and MND communities before making a decision on the future of the drug in the next few weeks. You can read more about this in a press release here and find out more about AMX0035 here.
New tool could detect signs of MND before symptoms begin
7 March 2024
Researchers from the University of Aberdeen, University of Edinburgh and other international partners have started to develop a new tool which could identify signs of MND earlier. The test has currently only been used with tissue samples and further development of the test would be needed before it could be used in clinic.
The test works by tagging toxic clumps of TDP-43, which are known to form in ~97% of people with MND. This new approach improved the accuracy of detecting these clumps compared to previous traditional approaches. Using this method the researchers were able to detect toxic clumps before symptoms of MND began. It is hoped that this new tool could, in the future, help diagnose the disease earlier and allow people earlier access to new treatments as they emerge. You can read more here.
Tofersen receives positive opinion from European Medicines Agency Committee
23 February 2024
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has announced a positive opinion for the approval of tofersen (Qalsody) for the treatment of SOD1 MND. The recommendation is for a marketing authorisation under exceptional circumstances, which means tofersen will be subject to monitoring once approved. The CHMP panel reviews the scientific evidence and forms an opinion on whether new drugs should be approved in Europe. The committee based their review on data from the phase 3 VALOR trial and its open label extension. This positive recommendation will now be sent to the European Commission who will make the final decision on approval in the EU within 67 days. You can read more about this recommendation here or in a press release.
The EMA determines approvals for the EU. It is thought that Biogen are currently pursuing the International Recognition Procedure (IRP) for approval in Great Britain (England, Wales and Scotland). This procedure allows the regulator for Great Britain, known as the Medicines Healthcare products Regulatory Agency (MHRA), to take into account the decisions from trusted regulatory partners, such as the EMA, when making the decision to approve new treatments.
Regulatory approval in Great Britain does not automatically mean the treatment will be available on the NHS. First, it must be assessed by NICE, who advise the NHS whether a new treatment is cost effective. During their evaluation NICE review evidence from clinical trials, assess the benefits and quality of life the treatment would provide and weigh these against the cost of the treatment. Tofersen is in the early stages of this process with NICE.
A phase 3 trial of tofersen, called ATLAS, is currently underway. This trial is investigating whether treatment before physical symptoms appear can delay the onset of symptoms and slow disease progression when symptoms do develop. You can find out more about tofersen and ATLAS on our website.
SAR443820 clinical trial does not meet primary endpoint
20 February 2024
Denali Therapeutics reported that the phase 2 clinical trial of SAR443820 (HIMALAYA) did not meet its primary endpoint. This suggests that there was no significant change in ALSFRS-R (from baseline to 24-weeks) for those who received the treatment compared to those who received the placebo (dummy drug). Further details on the trial outcome are expected in the coming months. You can read more about SAR443820 here.
Single-dose gene therapy stops MND progression in early laboratory research
15 February 2024
Researchers from Macquarie University have developed a single-dose gene therapy which has been found to clear clumps of a toxic protein, called TDP-43, in early laboratory testing. Toxic clumps of TDP-43 are found in 97% of cases of MND. The researchers found that when TDP-43 is faulty, levels of another protein, called 14-3-3θ, are increased. These two proteins then interact, and this leads to build up in cells. The new potential gene therapy (CTx1000) can help to control the interaction between the two proteins.
In early laboratory testing, in models of MND, a single dose of CTx1000 only targeted faulty TDP-43. This meant that the healthy version of TDP-43 could still be produced and work as it should, while the faulty version was prevented from forming. The researchers now hope to continue the development of CTx1000 which will hopefully lead to testing in clinical trials. You can read more about CTx1000 here.
MND Association announces new £5 million investment to give more people with MND access to clinical trials
6 February 2024
The MND Association are developing a new network of MND Association Research Nurses across England, Wales and Northern Ireland. The new Network aims to give everyone diagnosed with MND a chance to take part in research. The specialist nurses will help people with MND navigate the research trial process – providing information, identifying trials and research opportunities patients are eligible for, explaining what’s involved, and supporting their ongoing involvement. MND Association Research Nurses will work alongside multidisciplinary teams in Care Centres and Networks, with the first nurses expected to be in post by the end of the year. Find out more in a news story.
Function of the cell's powerhouse linked to MND severity
29 January 2024
Researchers from Sheffield Institute for Translational Neuroscience (SITraN) have discovered that the function of the powerhouse of the cell (mitochondria) can affect how long people with MND live. Mitochondria are essential for cell survival and previous research has suggested that the mitochondria may not produce enough energy in people with MND.
The researchers found that the function of the mitochondria does not affect the risk of developing MND. However, there was a link between function of the mitochondria and survival. This suggests that new therapies could be developed to change the function of the mitochondria and potentially slow disease progression and increase survival time. You can read more here.
AB Science provide update on European approval process for Masitinib
26 January 2024
AB Science have provided an update on the ongoing marketing authorisation application of masitinib for the treatment of MND by the European Medicines Agency (EMA). The Committee for Medicinal Products for Human Use (CHMP), part of the EMA, have asked AB Science to submit a written response to the List of Outstanding Issues. This has pushed back the timeline to a decision. A decision whether to approve masitinib is now expected before mid-2024.
Masitinib is a potential treatment for MND which is currently being tested in a phase 3 clinical trial in the US and Europe. A previous phase 2/3 trial showed potentially promising results for a specific group of people with MND. You can read more about Masitinib here.
New research investigates family members risk of MND
22 January 2024
A new research study has suggested that family members of someone with C9orf72 MND may have an increased risk of developing MND, regardless of whether they carry the gene change or not. Researchers looked at 131 families from Ireland who had been affected by C9orf72 MND, the most common genetic form of MND. They found that some family members were also diagnosed with MND but did not have the C9orf72 gene change. This suggests that family members may have a higher risk of developing MND, even if they do not have the gene change. The researchers emphasised that each family is different and that this research highlights the importance of individualised support. Further research is now needed to understand more about why this increased risk may occur and if this is observed in other populations.
Top-line results from trial testing oral edaravone suggests no benefit
10 January 2024
Ferrer have announced the top-line results from the Phase III ADORE clinical trial testing oral edaravone (FNP122) in people with MND. The trial did not meet primary or secondary endpoints. There was no significant different in change in ALSFRS-R (from baseline to 48-weeks) for those who received the treatment compared to those who received placebo (dummy drug). This suggests that oral edaravone is not beneficial for people with MND. The data also showed no improvement in long-term survival when comparing oral edaravone to placebo over 72-weeks. This formulation of oral edaravone (FNP122) was found to be safe and well-tolerated. You can find out more about the results here or read more about the ADORE trial here.
Ferrer have also announced that the open-label extension of the ADORE study, where everyone on the trial has an option to receive the treatment, will now end due to the lack of benefit observed.