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
Perma-Pipe Accelerates Growth with New U.S. Northeast Facility Investment to serve Artificial Intelligence Data Center customers, Provides Middle East Operations’ Update and Concludes the Board’s Review of Strategic Alternatives19.3.2026 14:00:00 CET | Press release
Perma-Pipe International Holdings, Inc. (the “Company”) today announced a strategic expansion initiative focused on accelerating growth through entry into the high-demand U.S. Northeast region. The Company is positioning itself to capitalize on the rapidly expanding Artificial Intelligence (“AI”)-driven data center market in both the United States and international markets while continuing to reinforce its leadership in critical infrastructure solutions. As part of this growth strategy, Perma-Pipe will prioritize investments aimed at expanding its presence in the rapidly evolving AI data center sector. The initiative reflects the Company’s long-term commitment to supporting next-generation technology infrastructure and strengthening its position in the global energy, industrial, and infrastructure markets. Expansion in the U.S. Northeast President & Chief Executive Officer, Saleh Sagr said, “We are excited to announce the expansion of our operations with a new facility in the Northeast
Armis Launches First-of-Its-Kind Benchmark Report Warning of Critical Security Gaps in AI-Native Development19.3.2026 13:00:00 CET | Press release
Research reveals 100% of leading generative AI models fail to generate secure code for critical development scenarios Armis, the cyber exposure management & security company, is warning that the rapid enterprise adoption of AI-native development is outpacing critical security safeguards, leaving organizations exposed to systemic vulnerabilities. New research from Armis Labs’ Trusted Vibing Benchmark Report, which evaluates 18 leading generative AI models across 31 test scenarios, reveals a 100% failure rate in generating secure code. These vulnerabilities are most prevalent in high-risk areas like memory buffer overflows, design file uploads and authentication systems. Therefore, organizations should immediately implement AI-native application security controls to reduce risk. “The era of vibe coding is here, but speed should not come at the cost of security,” said Nadir Izrael, CTO and Co-Founder of Armis. “Our research finds that the worst offenders are the same ones selling security
Morocco’s FRMF Welcomes CAF Appeal Board as Upholding Rules, Stability of International Competitions19.3.2026 12:53:00 CET | Press release
Following the announcement by the CAF Appeal Board, the Royal Moroccan Football Federation (FRMF) welcomes the decision, which reaffirms the primacy of competition regulations and reinforces the conditions necessary for the proper conduct of international tournaments. From the outset, following the incidents that led to the interruption of the match, the FRMF maintained a clear and consistent position: the strict application of the governing regulations. The Federation’s approach was solely guided by this principle. Following its appeal, CAF has now confirmed that the applicable regulations were not properly enforced. Throughout the process, the FRMF acted in full compliance with all relevant legal and procedural frameworks, with a constant focus on upholding its rights and preserving the integrity of the competition. This decision provides clarity on the applicable framework and strengthens the consistency and credibility of international competitions, particularly within African foot
Hilton Announces Exclusive Agreement with YOTEL to Expand Global Footprint in Lifestyle Segment19.3.2026 12:00:00 CET | Press release
Independent, design-led YOTEL brand gains reach through Hilton’s global distribution platformHilton Honors members gain access to a sleek, contemporary new way to stayYOTEL will be the first independent brand as part of newly established Select by Hilton brand Hilton (NYSE: HLT) today announced an exclusive agreement with YOTEL that will provide guests yet another way to stay within the hospitality leader’s growing global portfolio. With highly efficient hotels in urban markets, YOTEL has pioneered ways to meet changing guest needs through stays that feature smart room design, and clever tech-enabled features. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260318705963/en/ YOTEL Boston The franchise agreement with YOTEL expands Hilton’s network, filling a distinct customer need in the growing lifestyle segment in a manner consistent with its proven asset-light model. YOTEL will continue to independently manage and license it
Keenfolks Launches KEENFOLKS X_19.3.2026 12:00:00 CET | Press release
A Global AI-Native Marketing Infrastructure Network for Enterprise GrowthKeenfolks, the AI transformation partner behind marketing initiatives for brands including Coca-Cola, Diageo, Mars, Reckitt, Nestlé and Merck-MSD, today announced the launch of KEENFOLKS X_, a new global marketing network designed to operate on shared AI infrastructure The traditional holding company model that has shaped global marketing for decades is facing increasing pressure as capital markets, consolidation activity, and client demands point to a need for structural change. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260319751842/en/ Miguel Machado, CEO and Co-Founder, Keenfolks The marketing industry is searching for a new model. The question is no longer whether agencies need to change, but which ones were built for this shift from the start. Keenfolks, the Integrative AI® partner built natively for the Age of Intelligence, today announces th
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