What is the drug thought to do in the body?
Bravyl is an oral formulation of a re-purposed drug called Fasudil which is a Rho kinase (ROCK) inhibitor. Rho kinase is an enzyme that is normally increased in people with MND causing an increase in inflammation and cell death. Treatment with Bravyl can inhibit this enzyme, reducing its activity and possibly the stress and damage it causes to cells. Early studies suggest Bravyl may be effective at reducing levels of a disease marker called neurofilament light chain and the clumping of toxic TDP-43 protein in cells treated in a lab.
Phase 2a
Current status: Collecting data
Primary objective: To investigate if Bravyl (oral Fasudil) is safe and effective, and to monitor the effects on neurofilament and ALSFRS-R scores at different doses.
The phase 2a trial, called REAL, was divided into two parts. Part A enrolled 31 people across the US and Australia to be treated with a 180mg dose per day for 24 weeks. The results of part A indicate treatment with the ROCK inhibitor is safe and effective. The results show a reduction in neurofilament levels and ALSFRS-R score. To read more about the results, please see here. Part B aims to measure safety and tolerability of Bravyl at a higher dose of 300mg per day in people with MND for 24 weeks. Participants on both parts will be offered the opportunity to continue into an open label extension for a further 30 months. You can find out more about the trial here.
The trial is active in the USA and Australia only.
Latest News
2024
December 2024– The drug Bravyl was tested in pre-clinical animal studies to see if the drug can reduce the toxic effects of TDP-43. The study released the results at the 35th International Symposium on ALS/MND showing that treatment with Bravyl for 6 months reduces TDP-43 forming clumps by over half. Read more about this early study here.
July 2024 – The first person in part B of the trial received the higher dose of Bravyl.
Last updated: 18/04/2024