Unravel Biosciences, Inc.

Study site selection and finalization of clinical trial agreement final step toward initiation of RVL-001 proof-of-concept clinical studies early next year BOSTON, Oct. 24, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company established to advance drugs for complex diseases, and the clinical research unit of the PECET of the University of Antioquia, certified in Good Clinical Practices by the Colombian Health Regulatory Authority ("INVIMA") and located in in Medellín, Colombia, today announced the finalization of a clinical trial agreement enabling PECET to be the designated study site for Unravel's RVL-001 proof of concept clinical trials for Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS). Unravel's proprietary BioNAV™ drug discovery platform identified RVL-001 as a potentially promising therapeutic drug for Rett Syndrome and PTHS. Building on previously announced initiatives by Unravel to initiate a proof-of-concept clinical trials of RVL-001 in Colombia for RTT as well as a collaboration by the Pitt Hopkins Research Foundation (PTHF) to conduct an extension study of RVL-001 in PTHS, today's agreement with PECET in Colombia formally establishes the clinical infrastructure for Unravel to rapidly evaluate BioNAV™ identified drugs in patients. "We are excited to collaborate with the highly skilled PECET team to accelerate therapeutics development for rare disease patients," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "By conducting multiple clinical trials in parallel at a single clinical trial site enables faster and less costly drug development to support more patients with unmet needs." RTT and PTHS are both rare neurogenetic disorders starting in early childhood that lead to debilitating cognitive, motor and autonomic disability. Despite one approved treatment for RTT, there remains a significant unmet need for novel treatments having meaningful efficacy and acceptable safety and tolerability. There are no known treatments for PTHS. In addition to Unravel's RVL-001 program for RTT and PTHS, the company has also initiated development work on RVL-002, a first-in-class novel molecule for Rett syndrome. Initiation of patient dosing for the RVL-001 trial in RTT and extension trial in PTHS are both anticipated in early 2025, with study startup activities including clinical trial material manufacturing and patient recruitment initiated earlier this year as previously announced. "We are pleased to be the designated clinical trial center for Unravel, it is a great challenge that we will take on with great enthusiasm to contribute to the search for solutions for this type of disease," said Iván Darío Vélez, Professor Emeritus of the University of Antioquia and founder of PECET. "PECET has extensive expertise conducting clinical studies with international biopharma sponsors including US to support worldwide registration and approval and we look forward to supporting Unravel in their development efforts". About Unravel Biosciences Unravel Biosciences revolutionizes drug development, uncovering disease-modifying targets from patient data powered by AI systems biology computation, rapid in vivo screening, and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ foundational model of human health combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel's platform has led to four clinical programs. Unravel's platform developed RVL001, a novel formulation to treat neurodevelopmental disorders, RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027, a molecule targeting a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform access to foundation and biotech partners to accelerate and clinically derisk therapeutics using existing drugs as a key stepping stone to validating novel targets and mechanisms in real patient populations. and SOURCE Unravel Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The collaboration will allow for a more rapid and efficient approach to the identification and pre-clinical evaluation of potential drug candidates across multiple diseases to advance into clinical testing. BOSTON and BAR HARBOR, Maine, Sept. 17, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company spun out of Harvard University to advance drugs for complex diseases and The Jackson Laboratory (JAX), a pioneer in the discovery of precise genomic solutions for disease, today announced the initiation of a collaboration to expedite and efficiently identify promising drug candidates for clinical development. Unravel will utilize their proprietary BioNAV™ drug discovery platform and JAX's extensive expertise in pre-clinical disease modeling to identify potential drugs and novel drug targets across multiple diseases. The collaboration will prioritize already approved drugs that have the potential to be repurposed for other diseases, facilitating rapid clinical translation and near-term beneficial impact to patients. "We are thrilled to collaborate with JAX on this key multi-program effort to efficiently tackle multiple rare disorders," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "By seamlessly iterating between preclinical models and advanced computational analyses of multimodal datasets, we are revolutionizing the time, cost, and risk of drug discovery to enable rapid clinical translation and derisking for patients in need." The first disease to be studied in the collaboration will be Alternating Hemiplegia of Childhood (AHC), a rare neurodevelopmental disorder characterized by repeated episodes of weakness or paralysis leading to hemiplegia or quadriplegia. AHC is estimated to occur in approximately 1 in 1,000,000 births and is caused by mutations in the ATP1A3 gene. Unravel will generate RNASeq data from JAX's ATP1A3 disease relevant mouse models and analyze the data using Unravel's BioNAV™ platform to generate in silico hits of promising therapeutic candidates. These drug candidates will then be evaluated in JAX's pre-clinical models for efficacy as well as to inform dose selection for clinical testing. "Our collaboration with Unravel Biosciences is deeply meaningful for the patients and foundations we serve," said Cat Lutz, Ph.D., MBA, vice president of the Rare Disease Translational Center at JAX. "Validating Unravel's leads in our patient-specific mouse models has the potential to provide immediate benefits to patients and pave the way for future targeted genetic therapies. We also believe linking our scientific resources creates more seamless navigation for patients and families." While it is expected that rare disease foundations will drive much of the initial demand for research under the collaboration, this innovative drug discovery work is also anticipated to generate interest by biotechnology and pharmaceutical companies to support their drug development programs, as well as for use by Unravel and JAX to accelerate development of their own proprietary assets. About Unravel Biosciences Unravel Biosciences is the first rapid prototyping therapeutics company, integrating AI systems biology computation with rapid in vivo screening and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel's platform has led to four clinical trials starting in 2024. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027, a molecule targeting a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform access to foundation and biotech partners to accelerate and clinically derisk therapeutics. and About The Jackson Laboratory The Jackson Laboratory is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center and more than 3,000 employees in locations across the United States, Japan and China. Its mission is to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health. For more information, please visit . SOURCE Unravel Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Unravel Biosciences是一家人工智能驱使治疗公司，致力于开发治疗复杂疾病的药物。该公司前不久宣布，其治疗雷特综合征的药物vorinostat (RVL-001) 已获得美国食品药品管理局（FDA）的孤儿药资格认定。 Unravel 公司专有的 BioNAV™ 药物发现平台发现 RVL-001为治疗雷特综合征的有效药物。在过去的 18 个月中，Unravel 公司高效、快速地开展了 RVL-001 在雷特综合征患者中的临床试验。去年，Unravel 宣布其拟议的雷特综合征概念验证研究获得了 FDA 在 IND 前会议上的积极反馈。今年 3 月，Unravel 宣布开始生产 RVL-001 临床试验材料，用于即将在美国和哥伦比亚进行的雷特综合征临床试验。上个月，Unravel 宣布与Vanderbilt大学合作开展一项具有里程碑意义的美国多中心临床研究，该研究将评估 RVL-001在雷特综合征患者中的应用。 Unravel 联合创始人兼首席执行官 Richard Novak 博士说：“获得 FDA 的孤儿药资格认定是对我们团队致力于开发新的治疗方案、造福罕见病患者生活的肯定，此次的里程碑，加上我们最近宣布的与Vanderbilt的雷特综合征临床试验合作，以及我们即将在哥伦比亚开展的 POC Rett 研究，都说明了 Unravel 公司能够以颠覆性的速度快速开发原型药物并评估其在患者身上的疗效。” 雷特综合征是一种罕见的神经遗传性疾病，起病于儿童早期，会导致认知、运动和自主神经残疾。雷特综合征在全球的发病率在每1万名女婴中就有一人，是一种影响广泛的罕见病，只有一种治疗方法获得了美国食品及药物管理局（FDA）的批准，对具有显著疗效、可接受安全性和耐受性的新型治疗方法的需求仍未得到满足。除了 Unravel 的 RVL-001 项目外，公司还启动了 RVL-002 的开发工作，这是治疗雷特综合征的创新新型分子。 原文链接： www.prnewswire.com 译：李天予