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
The Bolivarian Republic of Venezuela Announces the Initiation of a Comprehensive Public Debt Restructuring Process13.5.2026 22:08:00 CEST | Press release
The Bolivarian Republic of Venezuela announces today its decision to initiate a comprehensive restructuring of its external public debt obligations. CONTEXT & RATIONALE Over the past decade, Venezuela's economy has been severely impacted by external shocks – from the end of the commodity super-cycle to foreign sanctions and COVID-19 – resulting in a sharp economic contraction, a protracted default since 2017, and a ballooning of public sector liabilities. The country nonetheless possesses a strong economic potential, including unique hydrocarbon and mineral endowments. A recovery is underway, and Venezuela is committed to accelerating it through a comprehensive reform agenda in support of growth, fiscal sustainability, monetary stabilization, and enhanced governance. Restoring public debt sustainability is central to this agenda: the current debt overhang constrains external financing, limits public investment capacity, and prevents full re-engagement with the international financial s
Boomi Announces Intent to Acquire Lunar.dev to Deliver Governed Agent Connectivity Across the Enterprise13.5.2026 21:00:00 CEST | Press release
Proposed acquisition extends Boomi’s AI gateway capabilities, enabling enterprises to govern massive volumes of traffic for production-ready AI Boomi, the data activation company for AI, today announced it has signed a letter of intent to acquire Lunar.dev, an innovator in AI and MCP gateway. The proposed acquisition is expected to enrich the Boomi Enterprise Platform and Boomi Connect with advanced capabilities to govern and scale AI usage across enterprise systems. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260513850557/en/ Boomi Announces Intent to Acquire Lunar.dev to Deliver Governed Agent Connectivity Across the Enterprise As organizations move from AI experimentation to production, controlling how agents and AI applications interact with LLMs at scale has become critical. Lunar.dev addresses this need with an AI gateway that delivers granular, policy-driven control over AI interactions, with the visibility, securi
BeOne Medicines’ BEQALZI™ (sonrotoclax) Approved by U.S. FDA as First and Only BCL2 Inhibitor for R/R Mantle Cell Lymphoma13.5.2026 19:27:00 CEST | Press release
BEQALZI is a foundational BCL2 inhibitor designed for greater potency and selectivity, with potential to improve efficacy, tolerability, and convenience versus others in the classApproval of BEQALZI marks the first new BCL2 inhibitor approved in a decade in the U.S. and the only BCL2 inhibitor approved in MCL, aiming to set a new standard of innovation BeOne Medicines Ltd. (“BeOne”) (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to BEQALZI™(bee-KAHL-zee; sonrotoclax), a foundational, next-generation BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. BEQALZI was designed to enhance BCL2 inhibition—with greater potency, selectivity, and a pharmacologic profile with potential to improve efficacy, tolerability, a
Boomi Unveils Innovations That Power the Agentic Enterprise13.5.2026 16:30:00 CEST | Press release
Orchestration capabilities simplify workflows, while advanced agent connectivity and an enhanced context layer activate data and scale AI Boomi, the data activation company for AI, today announced a major expansion of the Boomi Enterprise Platform at Boomi World 2026, introducing new capabilities across orchestrated agentic workflows, agentic engineering, governed agent connectivity, grounded agent context, and localized agent infrastructure. Together, these innovations are designed to power the agentic enterprise — where agents and humans work together to drive action and operationalize AI at scale. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260513996223/en/ Boomi Unveils Innovations That Power the Agentic Enterprise The enterprise has reached a defining moment as AI becomes the primary interface for work and MCP emerges as the new standard. While the shift toward a headless, agentic enterprise is inevitable, this visio
Boomi and Red Hat Collaborate on Production-Ready Agentic AI13.5.2026 16:30:00 CEST | Press release
Companies team to deliver a unified, enterprise-scale agentic stack to help organizations with managing data sovereignty and optimizing AI costs Boomi, the data activation company for AI, and Red Hat, the world’s leading provider of open source solutions, today announced a strategic collaboration to deliver a single, integrated stack for deploying agentic AI at scale. For many organizations, building production AI today means assembling numerous disconnected vendor choices spanning agent builders, orchestration tools, governance platforms, model providers, integration middleware, and security infrastructure, which can lead to data leaks and unpredictable costs. Boomi and Red Hat are working together to simplify AI innovation for customers by bringing together Boomi’s Agentstudio with the enterprise-grade power of Red Hat AI. This makes it easier for organizations to build agents that solve real business problems while supporting corporate standards for sovereignty, infrastructure flexi
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