AZAFAROS-B.V.
Azafaros B.V. today announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic diagnosis of either GM2 gangliosidosis or Niemann-Pick disease type C (NPC).
About the Trial
The trial was conducted across three sites in Brazil, involving 13 patients older than 12 years of age, with the aim of determining the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat. The results will guide the identification of the target dose for Azafaros’ planned Phase 3 pivotal studies.
With the 12-week, double-blind and placebo-controlled part of the study complete, patients have now entered the extension phase, where all participants receive the study drug.
The data from the RAINBOW study will be presented at the SSIEM Annual Symposium 2024, which will take place on 3 - 6 September in Porto, Portugal.
Stefano Portolano, CEO at Azafaros, said: “The results of the Phase 2 RAINBOW study mark a pivotal moment in the development of nizubaglustat. The dual mode of action sets nizubaglustat apart as a leap forward from other agents. We are excited by the results of the study and more than confident that nizubaglustat has the potential to become a convenient, life-changing therapy for patients affected by GM1 and GM2 gangliosidoses and NPC. Azafaros is thankful to the patients and their families for their important contribution to the RAINBOW study. Azafaros is now poised to embark on a global Phase 3 trial next year.”
Lead RAINBOW investigator, Roberto Giugliani, added: “We are pleased to report the completion of this important step in the development of a new therapy for GM2 gangliosidosis and Niemann-Pick disease type C, which delivers data to determine the best dosing of nizubaglustat and allows the planning of the next phase.”
In a statement, a representative from the Associação Niemann-Pick & Batten Brasil (ANPB), a patient organisation in Brazil, said there is an urgent need for new treatments for NPC.
"Current therapeutic options are limited and do not offer a definitive cure. Clinical research plays a crucial role in this process, allowing the development and evaluation of new therapies that can improve patients' quality of life and slow the progression of the disease. Therefore, we are excited about developing innovative and convenient therapies.”
About nizubaglustat
Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).
Nizubaglustat has received the following designations and support:
United States Food and Drug Administration (FDA)
Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC.
Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC.
IND Clearance and Fast Track Designation for GM1/GM2 gangliosidoses and NPC
European Medicines Agency (EMA)
Orphan Medicinal Product Designation (OMPD) for the treatment of GM2 gangliosidosis.
UK Medicines and Healthcare Products Regulatory Agency (MHRA)
Innovation Passport for the treatment of GM1 and GM2 gangliosidoses.
About GM1 and GM2 Gangliosidoses
GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides respectively, in the central nervous system (CNS), resulting in progressive and severe neurological impairment and early death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available.
About Niemann-Pick Disease Type C (NPC)
Niemann-Pick disease type C (NPC) is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of disease can happen throughout the lifespan of an affected individual, from prenatal life through adulthood.
About Azafaros
Azafaros is a clinical-stage company founded in 2018 by scientists with a deep understanding of rare genetic disease mechanisms, using discoveries made by scientists at Leiden University and Amsterdam UMC. Azafaros is led by a team of highly experienced industry experts and aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. The Azafaros team is dedicated to rapidly bring new drugs to the rare disease patients who need them. The company is supported by a syndicate of leading Dutch and Swiss investors including Forbion, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240619007436/en/
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Europastry and Blue Yonder Team Up To Strategically Drive Efficiency, Sustainability and Customer Satisfaction15.1.2025 10:00:00 CET | Press release
Global bakery leader implements Blue Yonder transportation management solutions supported by BlueGistics to optimize supply chain operations across Europe Delivering frozen bakery products presents several challenges that can impact the efficiency, profitability and sustainability of the supply chain. To address these challenges, Europastry, a world leader in the frozen bakery industry, has selected Blue Yonder’s transportation management solutions to evolve its supply processes and optimize its logistics operations. The transformational project was deployed by BlueGistics, a Blue Yonder partner and worldwide consulting company specializing in the implementation of Transportation Management Systems. With an ever-growing distribution network spanning more than 80 countries, Europastry sought to increase efficiency in its transportation processes, reduce costs, and continue delivering its wide range of bakery products efficiently. Blue Yonder’s transportation management solutions provide
Tax Systems Launches AI-powered, SaaS Solution for Global Pillar Two Compliance and Reporting15.1.2025 09:00:00 CET | Press release
Secure, scalable software that ensures organisations are prepared for the challenges of OECD Pillar Two rules now and in the future Tax Systems, the global multi-tax compliance SaaS provider, has today announced the availability of Pillar2, an intuitive solution to de-risk and simplify compliance with the OECD’s global Pillar Two tax rules. Designed for organisations operating across multiple jurisdictions - Pillar2 will save businesses valuable time and reduce the risk of errors, so they can cost effectively operationalise Pillar Two reporting and comply with confidence. Pillar2 integrates seamlessly with the provisioning and country-by-country reporting applications on TaxSuite - Tax Systems’ modular platform for international tax compliance. This means organisations can upload information once and re-use it as a single source of truth for their tax compliance data - helping to ensure consistency and accuracy across the tax reporting cycle and strengthen tax control frameworks. The s
Regula Launches Full Support for Digital Travel Credentials in Latest Software Update15.1.2025 08:00:00 CET | Press release
Regula, a global developer of forensic devices and identity verification solutions, has updated its Regula Document Reader SDK. Now, the software fully supports the new Digital Travel Credential (DTC) format, aligned with the International Civil Aviation Organization (ICAO) standards. This enhancement enables governments, airlines, and border control authorities worldwide to process travel documents with unmatched security, efficiency, and ease—whether on-site or remotely. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250115990355/en/ With Regula Document Reader SDK, travelers can create their own digital credentials by extracting a Virtual Component (DTC-VC) from a physical electronic identity document like ePassport. (Graphic: Regula) The DTC is a secure digital solution that streamlines travel experiences. It consolidates key personal information into a single virtual document that travelers can store on their mobile dev
Cargill Puts Bold Innovation at Center of Effort to Decarbonize Shipping15.1.2025 08:00:00 CET | Press release
Continued investment in wind assisted propulsion, next-generation fuels and digital solutions key to moving the world’s goods more sustainably Cargill is advancing a new era in sustainable shipping, putting innovation and collaboration at the core of its mission to move goods responsibly across the globe. Through wind-assisted propulsion technologies, dual-fuel methanol-powered vessels, and digital solutions, Cargill is setting the standard for helping the industry advance progress toward the International Maritime Organization’s (IMO) 2050 net-zero target. Combining technical expertise with a commitment to industry-wide partnerships, Cargill is helping to future-proof the maritime sector while enabling customers to meet their sustainability goals. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250114753261/en/ “Decarbonizing shipping is a monumental task that requires courage, innovation, and collaboration,” said Jan Dielem
Bureau Veritas: Statement15.1.2025 07:34:00 CET | Press release
Following recent media commentary, Bureau Veritas indicates that it is in discussions with SGS regarding a potential business combination. There can be no assurance that these discussions will result in a transaction or other agreement. Bureau Veritas will not comment further and will only communicate in due course, if and when applicable. About Bureau Veritas Bureau Veritas is a world leader in inspection, certification, and laboratory testing services with a powerful purpose: to shape a world of trust by ensuring responsible progress. With a vision to be the preferred partner for customers’ excellence and sustainability, the company innovates to help them navigate change. Created in 1828, Bureau Veritas’ 83,000 employees deliver services in 140 countries. The company’s technical experts support customers to address challenges in quality, health and safety, environmental protection, and sustainability. Bureau Veritas is listed on Euronext Paris and belongs to the CAC 40, CAC 40 ESG, S
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom