AZAFAROS-B.V.
16.7.2024 10:01:28 CEST | Business Wire | Press release
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
Cognite Positioned as a Front Runner in LNS Research Solution Selection Matrix for Industrial AI Platforms23.4.2026 20:00:00 CEST | Press release
Cognite, the global leader in Industrial AI, has been named a Front Runner in the LNS Research Solution Selection Matrix for Industrial AI Platforms (SSM). The LNS Research SSM evaluates vendors using a rigorous methodology that leverages its “3P” framework—Product, Potential, and Presence—to assess both current capabilities and future readiness. Within the report, Cognite is recognized in three key areas: depth in user-centric industrial AI applications, its knowledge graph-based data model, and its strong partner ecosystem. Together, all three give Cognite the ability to unify and contextualize complex industrial data, enabling organizations to operationalize AI at scale. “Cognite brings a sophisticated data model built for industrial complexities, along with advanced analytics and AI-driven insights, which is crucial for industrial organizations to achieve meaningful productivity gains and enterprise-wide ROI,” said Vivek Murugesan, Research Analyst at LNS Research. “These capabilit
Venture Global Announces Closing of $750,000,000 Senior Secured Notes by Venture Global Calcasieu Pass, LLC23.4.2026 19:47:00 CEST | Press release
Venture Global, Inc. (“Venture Global”, NYSE: VG) announced today that its subsidiary, Venture Global Calcasieu Pass, LLC (“VGCP”) has closed an offering of $750,000,000 aggregate principal amount of 6.000% senior secured notes due 2036 (the “Notes”). The Notes will mature on May 1, 2036. VGCP used the net proceeds from the offering, together with cash on hand and proceeds received from certain hedge terminations, for the prepayment, in full, of VGCP’s outstanding term loans, and to pay fees and expenses in connection with the offering. The Notes are guaranteed by TransCameron Pipeline, LLC (VGCP’s affiliate). The Notes are secured on a pari passu basis by a first-priority security interest in the assets that secure VGCP’s existing senior secured first lien credit facilities and VGCP’s existing senior secured notes. The Notes were not registered under the Securities Act of 1933, as amended (the “Securities Act”) or the securities laws of any state or other jurisdictions, and the Notes
Does Muscle Composition Identify High-Risk Individuals in Chronic Kidney Disease? New AMRA Medical Study Says Yes, Reveals Strong Link to Mortality23.4.2026 17:10:00 CEST | Press release
AMRA Medical researchers recently set out to answer the question: what is the relationship between adverse muscle composition (AMC) and mortality in kidney disease? Recent findings demonstrate that AMC, characterized by low muscle volume z-score and high muscle fat infiltration, is an independent and strong predictor of all-cause mortality in individuals with chronic kidney disease (CKD). The findings, published in Clinical Journal of the American Society of Nephrology, were based on data from the UK Biobank imaging study and further reinforce the clinical relevance of MRI-derived muscle biomarkers in identifying high-risk populations across chronic diseases. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260423198497/en/ AMRA's MRI-based muscle biomarkers in clinical trials Researchers from AMRA, alongside our collaborators at Linköping University (LiU), analyzed 894 participants with CKD using AMRA’s MRI-based body composi
Diligent Unveils AI Board Member and Agentic GRC Workforce at Elevate 202623.4.2026 15:00:00 CEST | Press release
Diligent’s agentic AI strengthens board oversight and transforms how GRC teams work — reducing manual effort and outside-advisor dependency, tightening control over sensitive workflows and enabling leaders to make faster, more defensible decisions Diligent, the AI leader in governance, risk and compliance (GRC) SaaS solutions, today unveiled a new generation of autonomous AI agents, including AI Board Member — a secure AI assistant for directors — and a coordinated network of agents embedded across the Diligent One Platform. Accessible through a single command center, agents automate multi-step workflows across governance, risk, compliance and third-party management, giving organizations the impact of a “GRC manager” without adding headcount. “AI supercharges teams that embrace it, and boards are no exception,” said Brian Stafford, President and CEO of Diligent. “With AI Board Member and a network of autonomous agents woven through the Diligent One Platform, we’re giving the C-suite an
Safe Software Bolsters Leadership Team with CFO and CPO Appointments to Drive Next Phase of Growth23.4.2026 15:00:00 CEST | Press release
CFO and CPO executive hires to enhance operational and financial performance on the company’s path to $250M by 2028 Safe Software (Safe), creator of FME, the only All-Data, Any-AI enterprise integration platform, today welcomes two executives to its leadership team as it moves into the next phase of growth. Judd Lee has been appointed as the company’s Chief Financial Officer (CFO), and Vanessa Ribreau has been appointed as Chief People Officer (CPO). Having recently crossed $100M in revenue, the expanded leadership team will further enable the company to scale as it accelerates toward its goal of reaching $250M in revenue by 2028. Judd Lee is a seasoned CFO with over 15 years of experience partnering closely with CEOs to align financial strategy and accelerate sustainable growth. He is known for his strong commitment to mentorship and for building high-performing global teams rooted in trust, morale and a shared sense of purpose. With more than two decades of financial leadership acros
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