Fasudil Hydrochloride Hydrate

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 These preliminary results were shared in the conference abstract and have not yet been peer-reviewed.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.

Peer-reviewed research finds the company’s novel technology enables faster dataset construction, further shortening Avicenna’s timelines to develop life-saving medicines. DURHAM, N.C.--(BUSINESS WIRE)-- Avicenna Biosciences today introduced an extension to its machine learning (ML) technology platform to enhance medicinal chemistry and expedite clinical-stage drug discovery. The company has raised $14.5 million in funding to date, with DCVC Bio leading its 2022 seed round, and this month published a paper in the peer-reviewed Journal of Chemical Information and Modeling. Co-authored with researchers from Schrödinger and Microsoft Research AI4Science, the paper outlines how combining Schrödinger’s physics-based methods with Avicenna’s novel ML methods can make the lead-to-drug optimization phase of small molecule drug discovery faster, less expensive and more successful – particularly when it comes to engineering potency and selectivity against a potential biological target. “We’re accustomed to hearing scientific success stories, but the countless failures that happen along the way often get overlooked. In medicinal chemistry especially, failure is prevalent. It can require hundreds of millions of dollars across many clinical attempts to bridge the complex gap from chemistry and biology to medicine, and successfully develop an approved drug,” said Dr. Thomas Kaiser, co-founder and Chief Scientific Officer at Avicenna. “Avicenna is applying novel ML methods to navigate the unknowns in medicinal chemistry and design around them. Our methods allow a drug design team to learn from years of failure and optimize against validated targets more efficiently. We’re making the most critical phase of drug design — the last mile of drug discovery — substantially faster and more cost effective.” Technology Applications and Results Avicenna is leveraging its technology to develop its own therapeutic programs, with an initial focus on neurodegenerative diseases. For example, Rho kinase (ROCK) inhibitors demonstrate potential in neurodegeneration and metabolic diseases. However, designing an orally dosed, central nervous system-penetrant ROCK inhibitor has proved extremely difficult. Fasudil, a promising ROCK inhibitor currently in Phase 2 clinical trials, must be dosed intravenously twice daily, precluding its use in illnesses like chronic kidney disease or neurodegeneration. To overcome these challenges, Avicenna has initiated its own ROCK Inhibitor Program. Using its novel ML technology to identify drug-like compounds with desired pharmacokinetic properties, the company has already achieved: Faster Timelines: 9 months from idea to in vivo proof of concept Reduced Costs: $220,000 from concept to initiation of Investigational New Drug (IND)-enabling studies Better Therapeutics: Two development candidates discovered while only synthesizing 11 total compounds Research Paper Implications “Creating new medicines and safely delivering them to the people who need them is incredibly difficult and full of risk. Avicenna is working to make drug discovery straightforward and fast by creating new algorithms to identify molecules with ideal drug-like properties,” said Dr. John Hamer, Managing Partner at DCVC Bio. “Avicenna’s collaborative research with Schrödinger and Microsoft demonstrates how a physics-based augmentation of ML requires just tens of molecules to optimize small molecules against a new drug target, as opposed to thousands. We couldn’t be more excited to support this potentially game-changing biotechnology company as it scales to its next stage of growth.” Titled “FEP-Augmentation as a Means to Solve Data Paucity Problems for Machine Learning in Chemical Biology,” Avicenna, Schrödinger, and Microsoft’s newly published paper describes how to use physics-based methods such as Schrödinger’s free energy perturbation technology, FEP+, to generate virtual data, which can be used to augment the sparse data sets commonly found in early medicinal chemistry optimization. These augmented datasets can be used for ML training where it was previously impossible to train without the disclosed approach. The augmentation is comparably informative for ML training as going through the effort and expense of making and testing the needed compounds. Ultimately, this allows an early-stage discovery team to access ML which can then quickly query millions of lead-like compounds and identify promising leads for drug development. The paper outlines the key mechanistic considerations for augmenting such data sets and lays the groundwork for implementation, demonstrating that an initial series of 10-20 related compounds, accompanied by 3D structures co-resolved with a small set of ligands, can serve as an accelerated foundation for lead optimization. The research shows that combining FEP+ with ML can accelerate the hit-to-lead phase of drug discovery, which can generate profound implications, including: Shorten the time spent in hit-to-lead optimization Significant reduction in synthetic effort Team and Partnership Opportunities The Avicenna team’s collective background in mathematics, chemistry and medicine brings a uniquely comprehensive perspective to their core discipline of medicinal chemistry. Co-founders Drs. Kaiser and Pieter Burger met while working within the Liotta Research Group at Emory University. The group is well known for its successes in drug development across virology, oncology and neurology, creating more than 20 FDA-approved therapeutics. As a synthetic organic chemist, Dr. Kaiser led the Liotta antivirals group, and as a structural bioinformaticist, Dr. Burger led its computational group. In addition to developing its own therapeutic programs, Avicenna partners with clinical-stage biotech startups and major pharmaceutical organizations to optimize their drug discovery campaigns and maximize risk-return pro easily fitting into partners’ existing chemistry workflows. For more information on Avicenna and its technology platform or to inquire about partnership opportunities, visit . About Avicenna Biosciences Founded in 2020, Avicenna is on a mission to solve the intractable drug design challenges that previously stopped drug candidates in their tracks. The company’s machine learning-driven medicinal chemistry platform makes lead-to-drug optimization faster, cheaper and more successful – transforming sub-optimal clinical candidates into life-saving drugs. Avicenna is backed by DCVC Bio, with Entrepreneur-in-Residence Christopher S. Meldrum having joined the Company as President and CEO. For more information, visit and follow the company on LinkedIn. View source version on businesswire.com: Contacts Source: Avicenna Biosciences View this news release online at: