Fasudil mesylate

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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

MONTREAL, June 17, 2024 /PRNewswire/ - Raya Therapeutic Inc., ("Raya") a mission-driven company focused on the treatment of ALS and other neurodegenerative diseases, is pleased to announce supportive clinical data for its lead compound, fasudil (RT1968, in the treatment of Amyotrophic Lateral Sclerosis (ALS)), as disclosed in a conference abstract for the ENCALS meeting by the investigators of the ROCK-ALS Phase 2 clinical trial. As shared in the conference abstract, the ROCK-ALS Phase 2 study was a randomized, double-blind, placebo-controlled trial that enrolled 120 patients across sites in Germany, France, and Switzerland. Subjects were randomly assigned (1:1:1) to receive 30 mg of fasudil, 60 mg of fasudil, or placebo intravenously (iv) over 20 treatment days. Assessments were performed at 45, 90 and 180 days after treatment initiation. Primary endpoints were tolerability and safety, and secondary endpoints included, among others, motor unit number index (MUNIX), which estimates how many motor neurons innervate a muscle, and slow vital capacity (SVC), which is a measure of respiratory function. In the intention-to-treat population (118 participants), safety and tolerability were not significantly different between groups. In the secondary outcomes, MUNIX showed a significantly reduced decline with Fasudil 60 mg at 26 and 90 days, and for Fasudil 30 mg at 90 days after treatment initiation. In addition, there was a directional difference in a slower loss of SVC favoring the 60 mg of fasudil treatment at day 26, which was statistically significant in a post-hoc subgroup analysis of females at all time points, as stated in the conference abstract. The authors, who include the principal investigators of this investigator-initiated trial, conclude the following regarding the results of this trial in the abstract: "Fasudil treatment was well tolerated and safe in ALS patients. MUNIX evaluation suggests a slower loss of motor units in fasudil-treated patients compared to placebo suggesting a benefit of fasudil to be explored in further clinical trials." These preliminary results were shared in the conference abstract and have not yet been peer-reviewed. "We are excited by the encouraging data from the ROCK-ALS trial of fasudil and remain steadfast in our mission to develop breakthrough therapies for ALS and other neurodegenerative disorders," said Anjan Aralihalli, President and founder of Raya. Based on these encouraging findings, Raya will now test fasudil in more extensive studies with a larger patient cohort to further assess the compound's efficacy in ALS. In addition, Raya has reformulated fasudil into a novel formulation to enhance ease of use, especially for the approximately 80% of ALS patients that suffer from dysphagia. Anjan Aralihalli continues: "With a comprehensive combination strategy on the horizon, along with plans to initiate clinical trials next year for both mono and combination therapies, we are optimistic about the future prospects of fasudil and our entire pipeline. We are especially grateful to our licensor, University Medical Center Göttingen (UMG), for their pivotal support and collaboration as the principal sponsor of this Phase 2 study. Their commitment and support have been instrumental in advancing fasudil for ALS patients." For more information about Raya and its innovative pipeline, visit or LinkedIn Members of the Raya management team will be attending the ENCALS Annual Meeting in Stockholm, Sweden from June 17-20. About Raya Raya is a mission-driven company focused on the treatment of ALS, leveraging the latest techniques for the selection and development of disease-modifying therapies. The company has a robust pipeline of five distinct clinical stage compounds that each target different pathways involved in motor neuron degeneration seen in ALS patients. The compounds were in-licensed following a rigorous selection process based on biological plausibility, clinical target engagement and functional clinical effects reflective of efficacy. This diversified approach may enable the development of combination therapies which may have a significant impact on disease progression. To develop further combination therapies, Raya entered into a strategic research collaboration with argenx last summer ( ). This partnership explores synergistic effects of their combined drug candidates in the hope of developing more effective treatment options for ALS patients. Raya is further supported by global experts and will leverage a unique patient-friendly platform trial design offering new hope in the fight against ALS. About Fasudil Fasudil is a small molecule inhibitor of the rho-associated kinase (ROCK) and is approved for the treatment of subarachnoid hemorrhage in a few Asian countries. In preclinical studies, fasudil attenuates neurodegeneration, modulates neuroinflammation, and fosters axonal regeneration. About UMG (University Medical Center Göttingen) The UMG located in Göttingen, Lower Saxony, Germany is one of the leading university medical facilities in Germany. It is comprised of a Medical Faculty engaged in research and teaching, a University Hospital providing patient care and Research allowing for the swift transfer of innovative therapy options into patient care and education.