Woolsey Pharmaceuticals announced the completion of patient recruitment for the high-dose cohort of the REAL study, evaluating BRAVYL (oral fasudil) for the treatment of Amyotrophic Lateral Sclerosis (ALS). The study has enrolled 31 patients in the 300 mg/day arm, building on earlier promising results from the standard-dose cohort.
Encouraging Biomarker and Clinical Findings
In the standard-dose cohort (180 mg/day, n=31), treatment with BRAVYL led to a 15% decrease in Neurofilament Light (NfL) levels from baseline to 6 months (p=0.0006). NfL is a recognized biomarker for neuronal damage and the rate of ALS progression; elevated levels are associated with a more rapid functional decline. Furthermore, compared to a matched historical control, the standard-dose cohort exhibited directionally positive clinical improvements, including a 17% reduction in the decline of ALSFRS-R scores, a 37% reduction in the decline of slow vital capacity (SVC), and a 56% reduction in the decline of muscle strength. Notably, greater reductions in NfL correlated with less deterioration on the ALSFRS-R (p=0.028).
Rationale for High-Dose Cohort
Based on the encouraging biomarker and clinical findings observed at the 180 mg/day dose, coupled with a favorable safety profile, Woolsey Pharmaceuticals decided to reopen the REAL study to investigate the effects of BRAVYL at an increased dose of 300 mg/day. "Experience with this higher dose in ALS patients will be invaluable as we progress to a larger study," said Sven Jacobson, CEO of Woolsey Pharmaceuticals. Jacobson also referenced a recent university-led study that found a relationship between fasudil dose and improved motor neuron function, further supporting the ongoing research.
Anticipated Results and Future Directions
Woolsey Pharmaceuticals anticipates releasing the results from the 300 mg/day dosing cohort by mid-2025. These findings will be crucial in determining the future development path of BRAVYL for ALS.
About ALS
ALS is a progressive neurodegenerative disease characterized by the degeneration of motor neurons in the brain and spinal cord. This leads to muscle weakness, paralysis, and ultimately, respiratory failure. The average survival time for individuals with ALS is only two to five years, highlighting the urgent need for effective treatments that can slow disease progression and improve quality of life.
