Extended clinical trial for drug Tofersen shows promise in slowing MND progression
A Phase 3 clinical trial of the investigational drug tofersen in patients with MND caused by the faulty SOD1 gene, has shown it can slow and reduce progression of the disease if taken over a period of 12 months. Results of the clinical trial indicated a notable reduction in the rate of decline in patient mobility and lung function after a year of taking the drug. More information here.
A new study reports that abnormalities in the SOD1 protein are also a common factor in sporadic MND.
Interesting findings from Kay Double and Benjamin Trist at the University of Sydney – the SOD1 protein appears to be mislocalised and accumulates in sporadic forms of MND as well as those carrying mutated forms of SOD1. Click here to read Australians 9 News report. The journal article can be read here.
FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for SOD1 MND
Biogen is seeking approval of tofersen under the FDA’s accelerated approval pathway, based on the use of neurofilament as a surrogate biomarker. Decision expected 25 Jan 2023. Results from their phase 1 up to most recent phase 3 VALOR and OLE studies is included. Tofersen targets subtype of MND caused by SOD1 mutation. Click link to read news release.
AMX0035 approved by Health Canada
Albrioza, also known as AMX0035, has been approved by Health Canada subject to conditions, including being dependent on the safety and efficacy data from the ongoing Amylyx Pharma Phase 3 PHOENIX trial.
The previous phase 2/3 clinical trial called CENTAUR achieved its primary endpoint of slowing decline in function as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) relative to placebo. AMX0035 is the first investigational therapy to demonstrate statistically significant benefit on this prespecified primary outcome in people with ALS since edaravone.
This decision is an advancement in available treatment options for people with MND. If results from the larger phase 3 PHOENIX trial are positive, hopefully more countries will follow Canada’s lead on granting approval . Click here to read more.
VALOR Biogen’s Tofersen trial: a look at the open label extension data
Tofersen, has been designed to work by stopping the production of abnormal SOD1 protein that is toxic to motor neurons. A previous clinical trial called VALOR showed some hints that Tofersen was having some effect in reducing the amount of SOD1 protein in people with MND however, no significant difference between those taking the medicine and those taking the placebo was shown on the primary endpoint (ALSFRS-R). Following this, an open label extension phase commenced to see if taking the treatment for a longer time led to more of an effect. This new data was presented at ENCALS and showed results leading to cautious optimism that the treatment may be effective if given for a longer time. Click here to read more.
Eledon Announces Positive Topline Results from Phase 2a Trial of Tegoprubart
Topline results from the Phase 2a trial of Tegoprubart have been announced, showing the drug to be well-tolerated, with no drug-related serious adverse events. This 12 week trial was an open label, dose escalating, safety, and biomarker study. The trial also demonstrated tegoprubart dose-dependent target engagement and pro-inflammatory biomarkers associated with ALS were dose-dependently reduced. Pro-inflammatory biomarker reduction was associated with a non-significant trend toward slowing disease progression as measured by ALSFRS-R. Click here to read the news release. More information about the trial is available here.
FDA approves Oral Form of Radicava for the treatment of adults with MND
On May 12, the US Food and Drug Administration (FDA) granted approval to oral edaravone (RADICAVA ORS) for the treatment of ALS/MND. This antioxidant drug was approved in 2017 but required IV administration. The long-term safety and tolerability of RADICAVA ORS up to 96 weeks is currently being evaluated in an ongoing phase 3 study. Although not available in New Zealand, it is hoped this drug may be considered by Medsafe as more studies show the impact it has on people with MND/ALS. Click here to read more about this drug.
Rapid Progress in ALS/MND: A Reflection of Advances in Neuromuscular Disorder Care
With a burst of activity in recent months, the pipeline of development for ALS/MND has proven to be a miniature likeness of the wider progress being made in the care and management of neuromuscular disorders. This article summarises some of the advancements made in the past year. Read more here.
CRISPR- the magic behind gene editing in MND
If you are interested in learning more about CRISPR read this blog by MND Association which discusses Professor Jennifer Doudna’s work on developing the gene-editing technology CRISPPR- Cas9, used to re-write the DNA in cells.
Wave Life Sciences announces interim results for the FOCUS-C9 clinical trial
Wave Life Sciences announces a positive update to the ongoing phase 1b/2a FOCUS-C9 Study which is investigating the use of WVE-004 for people living with C9orf72-associated MND or Frontotemporal Dementia (FTD).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. Click here to read the latest news release.
Earlier dementia detection could change lives. Can this NZ study unlock the secret?
Signs of dementia can be treated if they’re found early enough, but early detection is not often the reality for dementia patients. Dr Brigid Ryan, chair of the Younger Onset Dementia Aotearoa Trust and leader of the New Zealand Genetic Frontotemporal Dementia Study is trying to change that with her world-first study. Click here to read more.
Silence is golden: A new clinical trial for FUS-ALS
Researchers at King’s College London led by New Zealander Professor Christopher Shaw, are the first in Europe to embark on a clinical trial targeting a specific genetic form of ALS caused by mutations in the fused in sarcoma (FUS) gene. Click here to read more.
ALS-TDI & Google develop new ALS/MND symptom severity measurement tool
A new machine learning algorithm to measure ALS/MND symptom severity has been developed by Google researchers and scientists at ALS TDI. The authors’ hope that these objective measures could spur the further development of reliable and objective digital measures for MND progression. Click here to read more.
TDP-43 pathology: From noxious assembly to therapeutic removal
Identifying disease progression in MND
Early Access Program for Tofersen in SOD1-ALS
Biogen has expanded eligibility for its early access program (EAP) for the investigational therapy tofersen. The EAP is now open to all people with SOD1-MND, in countries where such programs are permitted by local regulations and future access may be secured. More information is available on Biogen’s website.
Detailed images of molecule associated with ALS could open door to therapies
Scientists at the Medical Research Council Laboratory for Molecular Biology in Cambridge, UK have determined the structure of the molecule associated with amyotrophic lateral sclerosis (ALS) and multiple other neurodegenerative diseases. Read the news release here.
Trinity College researchers make breakthrough in understanding MND
Researchers at Trinity College Dublin, have found that MND has 4 distinct patterns of changes in electrical signals that can be identified using EEG (electroencephalography). This new research has identified the progression of motor neuron disease (MND) in the nervous system and may pave the way for targeted treatments. The study has been published online in the journal Brain. Click here to read the news release.
Positive Phase 1 Results, Regulatory Progress Announced for 2 Therapeutics in ALS
Two investigational small molecule therapeutics have reported positive phase 1 clinical results, as well as regulatory progress, in the treatment of ALS/MND. Clinical data on the investigational therapeutics, DNL343 and SAR443820/DNL788, were presented at the 2021 Annual Northeast ALS (NEALS) Meeting, held virtually October 6-7. Click here to read more.
Study identifies protein important for motor coordination and exercise performance
Researchers at Karolinska Institutet in Sweden have identified a protein that improves muscular metabolism, motor coordination and exercise performance in mice. The findings, published in Cell Metabolism, could be of therapeutic value for patients with muscle and neurological diseases, such as ALS. Click here for the full article.