A Phase 2a Open-Label Preliminary Safety, Tolerability, and Biomarker Study of Oral Fasudil in Patients With the 4-Repeat Tauopathies of Progressive Supranuclear Palsy-Richardson Syndrome or Corticobasal Syndrome.

A Phase 2a Open-Label Preliminary Safety, Tolerability, and Biomarker Study of Oral Fasudil in Patients with the 4-Repeat Tauopathies of Progressive Supranuclear Palsy-Richardson Syndrome or Corticobasal Syndrome

Start Date22 Jan 2021

Sponsor / Collaborator

Woolsey Pharmaceuticals, Inc.Startup

100 Clinical Results associated with Fasudil mesylate

100 Translational Medicine associated with Fasudil mesylate

100 Patents (Medical) associated with Fasudil mesylate

1,191

Literatures (Medical) associated with Fasudil mesylate

01 Mar 2024·Alcohol (Fayetteville, N.Y.)

Inhibition of the Rho/ROCK pathway promotes the expression of developmental and migration-related genes in astrocytes exposed to alcohol

Article

Author: Zuoyi, Huang ; Xiaofeng, Zhu ; Shunjie, Bai ; Chengji, Wu ; Xinyan, Huang ; Xiaogang, Zhong ; Chenglong, Huang ; Huiying, Zhao ; Xunzhong, Qi ; Miao, Guo ; Guangtao, Sun

Astrocytes are an important regulator of alcohol dependence. Furthermore, the downregulation of Rho-associated coiled coil-containing protein kinase 2 (ROCK2) attenuates alcohol-induced inflammation and oxidative stress in astrocytes. On the basis of these findings, we examined the effects of alcohol and a Rho/RACK kinases inhibitor on astrocyte function and investigated their effects on mRNA expression to further explore the protective mechanisms of a Rho/RACK kinases inhibitor in astrocytes after alcohol exposure. CTX TNA2 astrocytes were cultured with alcohol and Rho/RACK kinases inhibitor intervention before undergoing transcriptome sequencing, quantitative reverse transcription polymerase chain reaction (qRT-PCR), and wound healing assays. Alcohol exposure modulated cell morphology and inhibited astrocyte migration, whereas Fasudil improved cell morphology and promoted astrocyte migration after alcohol exposure. Transcriptome sequencing results indicated that alcohol exposure modulates the expression of genes involved in astrocyte development. Fasudil reversed the effects of alcohol exposure on the astrocyte developmental process. Four genes related to the developmental process and migration - Ccl2, Postn, Itga8, and Serpine1 - with the highest protein-protein interaction correlations (node degree >7) were selected for verification by qRT-PCR, and the results were consistent with those of the sequencing and wound healing assays. Our results suggest that the Rho/ROCK pathway is essential for alcohol to be able to interfere with astrocyte development and migration gene expression. The Rho/ROCK pathway inhibitor Fasudil reversed the adverse effects of alcohol exposure on astrocytes and may have clinical applications.

01 Mar 2024·Microvascular research

Role of protein kinase D1 in vasoconstriction and haemodynamics in rats

Article

Author: Mizuno, Yusuke ; Goto, Takahisa ; Sugawara, Yoh ; Sawada, Yuri ; Oku, Shinya

AIMS:

Protein kinase D (PKD), once considered an effector of protein kinase C (PKC), now plays many pathophysiological roles in various tissues. However, little is known about role of PKD in vascular function. We investigated the role of PKD in contraction of rat aorta and human aortic smooth muscle cells (HASMCs) and in haemodynamics in rats.

METHODS AND RESULTS:

Isometric tension of rat aortic was measured to examine norepinephrine-induced contraction in the presence of PKD, PKC and Rho-kinase inhibitors. Phosphorylation of PKD1, myosin targeting subunit-1 (MYPT1), myosin light chain (MLC), CPI-17 and heat-shock protein 27 (HSP27), and actin polymerization were measured in the aorta. Phosphorylation of MYPT1 and MLC was also measured in HASMCs knocked down with specific siRNAs of PKD 1, 2 and 3. Intracellular calcium concentrations and cell shortening were measured in HASMCs. Norepinephrine-induced aortic contraction was accompanied by increased phosphorylation of PKD1, MYPT1 and MLC and actin polymerization, all of which were attenuated with PKD inhibitor CRT0066101. PKD1 phosphorylation was not inhibited by PKC inhibitor, chelerythrine or Rho kinase inhibitor, fasudil. In HASMCs, the phosphorylation of MYPT1 and MLC was attenuated by PKD1, but not PKD2, 3 knockdown. In HASMCs, CRT0066101 inhibited norepinephrine-induced cell shortening without affecting calcium concentration. Administration of CRT0066101 decreased systemic vascular resistance and blood pressure without affecting cardiac output in rats.

CONCLUSIONS:

PKD1 may play roles in aorta contraction and haemodynamics via phosphorylation of MYPT1 and actin polymerization in a calcium-independent manner.

01 Mar 2024·Neural regeneration research

Fasudil-modified macrophages reduce inflammation and regulate the immune response in experimental autoimmune encephalomyelitis.

Article

Author: Ma, Cungen ; Liu, Chunyun ; Chai, Zhi ; Guo, Minfang ; Wang, Qing ; Liu, Rong ; Guo, Shangde ; Xiao, Baoguo

Multiple sclerosis is characterized by demyelination and neuronal loss caused by inflammatory cell activation and infiltration into the central nervous system. Macrophage polarization plays an important role in the pathogenesis of experimental autoimmune encephalomyelitis, a traditional experimental model of multiple sclerosis. This study investigated the effect of Fasudil on macrophages and examined the therapeutic potential of Fasudil-modified macrophages in experimental autoimmune encephalomyelitis. We found that Fasudil induced the conversion of macrophages from the pro-inflammatory M1 type to the anti-inflammatory M2 type, as shown by reduced expression of inducible nitric oxide synthase/nitric oxide, interleukin-12, and CD16/32 and increased expression of arginase-1, interleukin-10, CD14, and CD206, which was linked to inhibition of Rho kinase activity, decreased expression of toll-like receptors, nuclear factor-κB, and components of the mitogen-activated protein kinase signaling pathway, and generation of the pro-inflammatory cytokines tumor necrosis factor-α, interleukin-1β, and interleukin-6. Crucially, Fasudil-modified macrophages effectively decreased the impact of experimental autoimmune encephalomyelitis, resulting in later onset of disease, lower symptom scores, less weight loss, and reduced demyelination compared with unmodified macrophages. In addition, Fasudil-modified macrophages decreased interleukin-17 expression on CD4⁺ T cells and CD16/32, inducible nitric oxide synthase, and interleukin-12 expression on F4/80⁺ macrophages, as well as increasing interleukin-10 expression on CD4⁺ T cells and arginase-1, CD206, and interleukin-10 expression on F4/80⁺ macrophages, which improved immune regulation and reduced inflammation. These findings suggest that Fasudil-modified macrophages may help treat experimental autoimmune encephalomyelitis by inducing M2 macrophage polarization and inhibiting the inflammatory response, thereby providing new insight into cell immunotherapy for multiple sclerosis.

News (Medical) associated with Fasudil mesylate

17 Jun 2024

·www.biospace.com

Raya Therapeutic Announces Supportive Clinical Results of Fasudil (RT1968) in ALS Patients

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.

Phase 2Clinical Result

21 Feb 2023

·www.prnewswire.com

Amyotrophic Lateral Sclerosis Pipeline Booms as 100+ Therapies Likely to Enter in the Treatment Domain | DelveInsight

The prevalence of amyotrophic lateral sclerosis has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of amyotrophic lateral sclerosis and the growing research and development activities to develop novel therapies to treat amyotrophic lateral sclerosis to drive the market. The companies developing the potential therapies in the last stage of development include Biogen, AB Sciences, Helixmith, and several others. LAS VEGAS, Feb. 21, 2023 /PRNewswire/ -- DelveInsight's ' Amyotrophic Lateral Sclerosis Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline amyotrophic lateral sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the amyotrophic lateral sclerosis pipeline domain. Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report DelveInsight's amyotrophic lateral sclerosis pipeline report depicts a robust space with 100+ active players working to develop 100+ pipeline therapies for amyotrophic lateral sclerosis treatment. Key amyotrophic lateral sclerosis companies such as Biogen, Ionis Pharmaceuticals, Ferrer Internacional S.A., AbbVie, Calico Life Sciences LLC, Genuv Inc., Kadimastem, Corcept Therapeutics, AL-S Pharma, Rapa Therapeutics LLC, Cytokinetics, MediciNova, Retrotope, Inc. Woolsey Pharmaceuticals, Sanofi, PTC Therapeutics, Helixmith Co., Ltd., Annexon, Inc., Denali Therapeutics Inc., Revalesio Corporation, Clene Nanomedicine, Ashvattha Therapeutics, Inc., Apellis Pharmaceuticals, Inc., Procypra Therapeutics, Knopp Biosciences, InFlectis BioScience, AI Therapeutics, Inc., Cellenkos, ZZ Biotech, LLC, QurAlis Corporation, Alector Inc., NeuroSense Therapeutics Ltd., Novartis Pharmaceuticals, Eledon Pharmaceuticals, and others are evaluating new amyotrophic lateral sclerosis drugs to improve the treatment landscape. Promising amyotrophic lateral sclerosis pipeline therapies in various stages of development include BIIB067, FAB122, ABBV-CLS-7262, Trametinib, BIIB105, AstroRx, Dazucorilant, AP-101, RAPA-501, ION-363, Reldesemtiv, MN-166, RT001, Fasudil, SAR443820, PTC857, ANX005, DNL343, RNS60, BIIB100, CNMAu8, 18F-OP-801, Pegcetacoplan (APL-2), Cu(II)ATSM, KNS-760704, IFB-088, LAM-002A, CK 0803, 3K3A-APC, QRL-201, Dexpramipexole, AL001, PrimeC, BLZ945, AT-1501, and others. In February 2023, NeuroSense Therapeutics Ltd. and QuantalX Neuroscience Ltd; the developer of Delphi-MD, a clinically objective neurodiagnostic medical device , announced a collaboration to improve early diagnosis and treatment of neurodegenerative diseases.In addition, the companies agreed that QuantalX's Delphi-MD would be used for early diagnosis and ongoing monitoring of trial participants in NeuroSense's planned future pivotal Phase III efficacy trial of PrimeC in people living with amyotrophic lateral sclerosis (ALS), pending the successful conclusion of its ALS Phase IIb trial. In February 2023, Amylyx Pharmaceuticals, Inc. announced the completion of enrollment in PHOENIX, a global, 48-week, randomized, placebo-controlled Phase III clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) in people living with amyotrophic lateral sclerosis (ALS). Amylyx anticipates topline results in 2024. The study enrolled 664 participants living with ALS. In February 2023, UMass Chan Medical School signed a three-year sponsored research agreement with NeuShen Therapeutics Inc. to investigate a gene therapy treatment for amyotrophic lateral sclerosis, a rare neurological disease. Based in Shanghai and Boston, NeuShen is focused on developing innovative treatments for central nervous system disorders using adeno-associated virus-based gene therapy and small molecule delivery. In January 2023, Inflamed Pharma, part of the incubator and accelerator company Xlife Sciences, partnered with researchers at the University Medical Center Gottingen in Germany to develop its investigational therapy ProcCluster for the treatment of amyotrophic lateral sclerosis (ALS).The collaboration, led by Jan C. Koch, MD, a principal investigator at the university's Department of Neurology, will be focused particularly on investigating ProcCluster in cellular models of ALS. In January 2023, Amylyx Pharmaceuticals and Neopharm signed a license and distribution agreement to commercialize AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine) to treat amyotrophic lateral sclerosis (ALS).Neopharm will commercialize the oral, fixed-dose medication in Israel, West Bank, Gaza, and the Palestinian Authority, subject to regulatory review and clearance.According to the agreement, Amylyx will grant exclusive rights to Neopharm for commercializing the therapy in the covered territory. In January 2023, uniQure N.V. and Apic Bio announced that they have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders. Request a sample and discover the recent advances in amyotrophic lateral sclerosis drug treatment @ Amyotrophic Lateral Sclerosis Pipeline Report The amyotrophic lateral sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage amyotrophic lateral sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the amyotrophic lateral sclerosis clinical trial landscape. Amyotrophic Lateral Sclerosis Overview Amyotrophic Lateral Sclerosis (ALS) is a rare neurological disease that affects the nerve cells (neurons) that control voluntary muscle movement (those muscles we choose to move). Voluntary muscles are responsible for movements such as chewing, walking, and talking. The disease is progressive, which means that the symptoms worsen over time. There is currently no cure for ALS and no effective treatment to slow or reverse the disease's progression. Muscle weakness or stiffness are common early ALS symptoms. Individuals gradually lose strength and the ability to speak, eat, move, and even breathe as all voluntary muscles are affected. Most people with ALS die from respiratory failure, usually within 3 to 5 years of the onset of symptoms. However, approximately 10% of people with ALS live for ten years or more. There is no single test that can definitively diagnose ALS. A detailed history of the symptoms observed by a physician during physical examination, as well as a review of the individual's full medical history and a series of tests to rule out other diseases, are used to make the ALS diagnosis. Find out more about drugs for amyotrophic lateral sclerosis @ New Amyotrophic Lateral Sclerosis Drugs A snapshot of the Amyotrophic Lateral Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging amyotrophic lateral sclerosis pipeline therapies @ Amyotrophic Lateral Sclerosis Clinical Trials Amyotrophic Lateral Sclerosis Therapeutics Assessment The amyotrophic lateral sclerosis pipeline report proffers an integral view of amyotrophic lateral sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Amyotrophic Lateral Sclerosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: RNA interference, Superoxide dismutase 1 expression inhibitors, Angiogenesis inhibitors, Colony stimulating factor inhibitors, Coronavirus-3C-like-proteinase inhibitors, Endopeptidase Clp inhibitors, Focal adhesion protein-tyrosine kinase inhibitors, Lyn protein-tyrosine kinase inhibitors, Macrophage colony-stimulating factor receptor modulators, Mast cell inhibitors, Platelet-derived growth factor receptor antagonists, Protein tyrosine kinase inhibitors, Proto oncogene protein c-kit inhibitors, Proto-oncogene protein c-fyn modulators, Type 3 fibroblast growth factor receptor antagonists, Angiogenesis inducing agents, Gene transference, Hepatocyte growth factor expression stimulants, G protein-coupled receptor modulators, HSP90 heat-shock protein modulators, Ion channel modulators, Antioxidants, NAD(P)H dehydrogenase (quinone) modulators, CD40 ligand inhibitors, Complement C1 inhibitors, Cyclo-oxygenase 2 inhibitors, DNA gyrase inhibitors Key Amyotrophic Lateral Sclerosis Companies: Biogen, Ionis Pharmaceuticals, Ferrer Internacional S.A., AbbVie, Calico Life Sciences LLC, Genuv Inc., Kadimastem, Corcept Therapeutics, AL-S Pharma, Rapa Therapeutics LLC, Cytokinetics, MediciNova, Retrotope, Inc. Woolsey Pharmaceuticals, Sanofi, PTC Therapeutics, Helixmith Co., Ltd., Annexon, Inc., Denali Therapeutics Inc., Revalesio Corporation, Clene Nanomedicine, Ashvattha Therapeutics, Inc., Apellis Pharmaceuticals, Inc., Procypra Therapeutics, Knopp Biosciences, InFlectis BioScience, AI Therapeutics, Inc., Cellenkos, ZZ Biotech, LLC, QurAlis Corporation, Alector Inc., NeuroSense Therapeutics Ltd., Novartis Pharmaceuticals, Eledon Pharmaceuticals, and others. Key Amyotrophic Lateral Sclerosis Pipeline Therapies: BIIB067, FAB122, ABBV-CLS-7262, Trametinib, BIIB105, AstroRx, Dazucorilant, AP-101, RAPA-501, ION-363, Reldesemtiv, MN-166, RT001, Fasudil, SAR443820, PTC857, ANX005, DNL343, RNS60, BIIB100, CNMAu8, 18F-OP-801, Pegcetacoplan (APL-2), Cu(II)ATSM, KNS-760704, IFB-088, LAM-002A, CK 0803, 3K3A-APC, QRL-201, Dexpramipexole, AL001, PrimeC, BLZ945, AT-1501, and others. Dive deep into rich insights for new drugs for amyotrophic lateral sclerosis treatment; visit @ Amyotrophic Lateral Sclerosis Medications Table of Contents For further information on the amyotrophic lateral sclerosis pipeline therapeutics, reach out @ Amyotrophic Lateral Sclerosis Drug Treatment Related Reports Amyotrophic Lateral Sclerosis Epidemiology Amyotrophic Lateral Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted amyotrophic lateral sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Amyotrophic Lateral Sclerosis Market Amyotrophic Lateral Sclerosis Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key amyotrophic lateral sclerosis companies including Helixmith, Sanofi, Denali Therapeutics, Alector, GSK, NeuroSense Therapeutics, Biogen, among others. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Duchenne muscular dystrophy companies including Edgewise Therapeutics, Pfizer, Daiichi Sankyo, among others. Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonsense mutation Duchenne muscular dystrophy companies, including Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, among others. Becker Muscular Dystrophy Pipeline Becker Muscular Dystrophy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key becker muscular dystrophy companies, including Epirium Bio, Ultragenyx, Strykagen, among others. Facioscapulohumeral Muscular Dystrophy PIpeline Facioscapulohumeral Muscular Dystrophy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key facioscapulohumeral muscular dystrophy companies, including miRecule, Arrowhead Pharmaceuticals, Fulcrum Therapeutics, among others. 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It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Additionally, get in touch with our business executive to explore @ Healthcare Due Diligence Services Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3License out/inPhase 2

18 Feb 2021

·www.biospace.com

Woolsey Pharmaceuticals Emerges from Stealth Mode to Announce Patients Enrolled in Two New CNS Studies

NEW YORK--(BUSINESS WIRE)-- Woolsey Pharmaceuticals, a clinical-stage company developing treatments for neurodegenerative diseases, today announced dosing of the first patient with BRAVYL in its FOUND (Fasudil fOr Uncontrolled waNDering) study for Alzheimer’s and Vascular Dementia patients who have wandering behaviors of elopement and/or getting lost. The Company also announced the first patient in its ROCKIT-1 (ROCK Inhibitor in Tauopathies – 1) trial of BRAVYL in Progressive Supranuclear Palsy (PSP) and Corticobasal Syndrome (CBS) patients has commenced treatment. ABOUT WANDERING IN DEMENTIA PATIENTS Dementia is the leading cause of dependency and disability amongst older adults. Wandering is one of the most dangerous dementia-related behaviors. Earlier this month, in the midst of a massive winter storm that slammed the Northeast, a 67-year-old Allentown, PA woman who had Alzheimer’s disease, wandered away from her home. After a frantic search, she was found four blocks away, lying in a snowbank. She was pronounced dead from hypothermia a few hours later. The stress experienced by families and caregivers when a person living with dementia wanders and becomes lost is significant. Wandering represents the “tipping point” for many caregivers, as they are more likely to report significant depression and burden — which, in turn, can lead to the gut-wrenching decision to place a loved one in a nursing home or locked memory care unit. BRAVYL (oral fasudil), is a potent inhibitor of Rho-kinase (ROCK) and is believed to reduce wandering behaviors by improving blood flow in the hippocampus, a part of the brain that plays a major role in navigation, learning, and memory. BRAVYL is non-sedating. BRAVYL has been tested in a small study of dementia patients who had symptoms of wandering. The disappearance of wandering symptoms in these patients was observed shortly after the start of treatment. Not long after discontinuation of treatment, patients returned to their pretreatment (wandering) state. This on-off effect was replicated more than once in these subjects. THE FOUND STUDY FOUND is a phase 2a study that will evaluate whether BRAVYL improves wandering behaviors of excess movement and pacing, cognition, memory, neuropsychiatric symptomatology, and caregiver/nursing staff burden, among other endpoints. Participation will be for up to 26 weeks. FOUND is currently recruiting at multiple sites in the United States and Australia. “Wandering away from home or a care facility is dangerous for people with dementia and stressful for families and caregivers who must continually monitor the person’s movement 24 hours a day,” said Dr. David Thomas, professor at Eastern Michigan University and noted expert on dementia-related wandering. “If results from an earlier study could be replicated here, it would certainly represent a historic and momentous step forward in the quality of life and care of people who wander.” “We are pleased to enroll the first patient in the FOUND trial, which will further our understanding of the potential role of BRAVYL for the treatment of dementia patients who wander,” said David Geliebter, Co-Founder and Executive Chairman of Woolsey Pharmaceuticals. “Today’s announcement is an important milestone in bringing more than just hope to these patients and their loved ones.” Data readout in the study is anticipated in 2022. ABOUT PSP and CBS Progressive supranuclear palsy (PSP) and corticobasal syndrome (CBS) are rare neurodegenerative disorders associated with the accumulation of insoluble deposits of predominantly 4 microtubule binding domain repeat (4R) tau protein in specific brain regions. PSP affects movement, gait and balance, speech, swallowing, vision, mood and behavior, and thinking. One of the classic signs of the disease is an inability to aim and move the eyes properly, which individuals may experience as blurring of vision. CBS patients present with bizarre rigidity and dystonia (a condition in which a person's muscles contract uncontrollably). There are currently no effective therapies for either disorder. From the onset of PSP and CBS, the lifespan of patients averages 7 years. Levels of Rho-kinase (ROCK) are elevated in both PSP and CBS patients, and is thought to be involved in the accumulation of tau aggregates. It is hypothesized that BRAVYL, a ROCK inhibitor, will reduce levels of ROCK activity in the brains of PSP and CBS patients, reducing 4R tau phosphorylation and aggregation, as well as inducing clearance of existing 4R tau aggregates that are the hallmark of the two diseases. THE ROCKIT-1 STUDY ROCKIT-1 is a phase 2a open-label study to assess preliminary safety, tolerability, and effect on neurodegeneration biomarkers of BRAVYL in patients with 4-Repeat (4R) Tauopathies to assess whether the drug improves clinical features of the disease. The study will enroll approximately 15 patients, all of whom will be treated with BRAVYL for 48 weeks. ROCKIT-1 is being conducted at the University of California, San Francisco (UCSF), under the direction of Dr. Adam Boxer. Dr. Boxer is the Endowed Professor in Memory and Aging in the Department of Neurology. He directs UCSF’s Neurosciences Clinical Research Unit and the Alzheimer’s Disease and Frontotemporal Degeneration Clinical Trials Program at the UCSF Memory and Aging Center. “Studies of progressive supranuclear palsy and corticobasal syndrome suggest that mitigating pathogenic tau levels is a rational strategy for tauopathy treatment,” notes Dr. Boxer. “BRAVYL, a ROCK 1 and ROCK 2 inhibitor, mitigated pathogenic tau levels in animal models. ROCK 1 and ROCK 2 have been shown to be upregulated in brain tissue of PSP and CBS patients. Any therapeutic that can demonstrate biological proof of concept in this patient population would be a major advancement in this field.” “Dosing the first patient in our ROCKIT-1 clinical trial marks a critical turning point in our efforts to advance a potentially disease-modifying treatment for patients with PSP and CBS,” said Sven Jacobson, Co-Founder and Chief Executive Officer of Woolsey. “We are excited to progress clinical development of BRAVYL and to bring forward a much-needed therapy for these rapidly progressing neurodegenerative diseases.” BRAVYL has been granted “orphan drug” designation (ODD) by the Food and Drug Administration (FDA) for both PSP and CBS. The FDA’s Office of Orphan Drug Products awards orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. ODD provides certain benefits, including seven years market exclusivity upon regulatory approval. Data readout in the study is anticipated in 2022. ABOUT WOOLSEY Woolsey Pharmaceuticals does not discover new drugs. We are an indications discovery company. We discover new ways to use existing drugs for other diseases and conditions for which patients in need have no therapeutic solutions. In late 2019, Woolsey licensed exclusive rights to BRAVYL from a leading Japanese pharmaceutical company. The drug’s active ingredient, fasudil, is approved in Japan and China in an IV formulation for the treatment of cerebral vasospasm. Woolsey’s drug, BRAVYL, is an oral version of fasudil. Woolsey Pharmaceuticals is a portfolio company of Embark Healthcare (embarkhc.com).

Orphan Drug

100 Deals associated with Fasudil mesylate

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	C04AX32	DB08162

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Cerebral Infarction	CN	Asahi Kasei Pharma America Corp.	22 Aug 2001
Vasospasm, Intracranial	CN	Asahi Kasei Pharma America Corp.	22 Aug 2001

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	Phase 2	-	Raya Therapeutic Inc.	-

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03792490 (ROCK-ALS, PRNewswire) Manual	Phase 2	Amyotrophic Lateral Sclerosis	120	fasudil 30 mg	xfvjwwjjqy(lqipjypeug) = Fasudil treatment was well tolerated and safe in ALS patients. fvzebhjykj (sralblcrou ) View more	Positive	17 Jun 2024
				fasudil 60 mg
NCT00498615 (CTgov)	Phase 3	Scleroderma, Systemic \| Raynaud Disease	17	Fasudil (40 mg Fasudil)	aqbrfliirx(dwhoxakjjz) = thrzsxhsey bkdtpybqbc (kctgqjgbnx, roiudwxusc - fsajjkntns) View more	-	04 Nov 2014
				Fasudil (Placebo)	aqbrfliirx(dwhoxakjjz) = zmsyqfaeuu bkdtpybqbc (kctgqjgbnx, kqegearjui - xxdpkhdbgk) View more

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