Woolsey Pharmaceuticals Completes Patient Recruitment for High-Dose Cohort of ALS Study

NEW YORK, Nov. 12, 2024 /PRNewswire/ -- Woolsey Pharmaceuticals today announced the successful completion of patient recruitment (n=31) in the high-dose (300 mg/day) cohort of the REAL study, which is assessing BRAVYL® (oral fasudil) for the treatment of Amyotrophic Lateral Sclerosis (ALS). This follows promising biomarker and clinical findings from the study's standard-dose cohort.

At the 180 mg/day dose (n=31), the biomarker Neurofilament Light (NfL), a sensitive indicator of neuronal damage and rate of ALS progression, decreased by 15% from baseline to 6 months (p=0.0006); of note, increased levels of NfL are associated with more rapid decline in function. Additionally, there were directionally positive clinical improvements compared to a matched historical control — a 17% lower decline in ALSFRS-R, a 37% lower decline in slow vital capacity (SVC), and a 56% lower decline in muscle strength. Moreover, greater decreases in NfL were correlated with less deterioration on the ALSFRS-R (p=0.028).

Given these encouraging biomarker and clinical findings at the 180 mg/day dose, along with an excellent safety profile, Woolsey Pharmaceuticals made the decision to re-open the REAL study to explore 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. "Furthermore, recently published results from a university-led, three-arm, double-blind study of fasudil in ALS patients found a relationship between dose and improved motor neuron function, which is very encouraging for our ongoing research," Jacobson stated, referencing "Safety, tolerability, and efficacy of fasudil in amyotrophic lateral sclerosis [ROCK-ALS]: a phase 2, randomised, double-blind, placebo-controlled trial, Jan C Koch et at, The Lancet Neurology, November 2024)."

Study results from the 300 mg/day dosing cohort are expected to be released by mid-2025.

About ALS

ALS is a fatal neurodegenerative disease characterized by inevitable, and often rapid, decline in patients as the disorder advances. Mean survival time is only two to five years. Accordingly, the ability to impede worsening represents a meaningful advancement in efforts to enhance the prognosis and quality of life of individuals impacted by the devastating condition.

About Woolsey Pharmaceuticals

Woolsey Pharmaceuticals' mission is to help usher in a new era of neurodegenerative disease treatment, saving and improving the lives of patients in need.

SOURCE Woolsey Pharmaceuticals, Inc.