Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. ALS is part of a wider group of disorders known as motor neuron diseases. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body, governing voluntary movements and muscle control. ALS causes progressive degeneration of these motor neurons leading to their death. When the motor neurons die, the brain can no longer initiate and control muscle movement. With voluntary muscle action progressively affected, people may lose the ability to speak, eat, move, and breathe.
There is a large and convincing body of preclinical evidence of BRAVYL’s effect on ALS in animal models. Additionally, there is persuasive human data from a compassionate use study in Europe, where three ALS patients were dosed with an intravenous version of BRAVYL for a month. In all three patients, decline in function was attenuated during and one month after fasudil infusions, in one patient breathing capacity increased during treatment, and in one muscle strength increased during treatment.
The REAL Study
(Rho kinase inhibition in Amyotrophic Lateral Sclerosis)
REAL is a phase 2a open-label study to assess preliminary safety, tolerability, and effect on clinical outcomes as well as neurodegeneration biomarkers of BRAVYL in patients with ALS.
The study will enroll approximately 20 patients, all of whom will be treated with BRAVYL for 24 weeks.