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
FDA Authorizes ZYN ULTRA Nicotine Pouches Following Scientific Review21.8.2026 21:11:00 CEST | Press release
Authorizations add selection of ZYN ULTRA products to PMI U.S.’s growing portfolio of better alternatives for legal-age adults who smoke or use traditional oral tobacco products Philip Morris International Inc. (NYSE: PM) today announces that the U.S. Food and Drug Administration (FDA) issued Marketing Granted Orders to PMI’s U.S. affiliate, Swedish Match USA, Inc., authorizing the marketing of 11 ZYN ULTRA moist oral nicotine pouch products, including all 9mg variants and one 11mg variant. Additional 11mg variants remain under scientific review. Today’s action further enhances PMI’s leadership role in the smoke-free category. “We are delighted with the FDA’s decision to authorize a range of ZYN ULTRA products, which will build on ZYN’s position as America’s leading smoke-free product brand,” said Stacey Kennedy, PMI U.S. CEO. “We look forward to expanding our portfolio of better choices for the 45 million Americans who consume nicotine products.” ZYN ULTRA positions the brand to furth
58% of Consumers Say They Don't Care Whether a Product is a National Brand or Private Label. They Just Buy What They Need21.8.2026 12:00:00 CEST | Press release
New NIQ & World Data Lab report reveals how consumer polarization is redefining value, accelerating private label adoption, and reshaping competition on the shelf As fast-moving consumer goods (FMCG) prices rose 26% globally between 2021 and 2025, consumers have become more deliberate about where they save and where they spend. NielsenIQ (NYSE: NIQ) has released new findings showing that private label is no longer viewed simply as a lower-cost substitute, but as a credible competitor across value, mainstream, and premium segments. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260819950224/en/ Private label is no longer just a value play. The findings, published in NIQ’s latest report, A Tale of Two Consumers: The Polarized Mindsets Reshaping Global Consumption, created in collaboration with World Data Lab, show how consumer polarization is redefining value and reshaping competition on the shelf. The report also draws on ins
Tanium Reappoints Co-Founder Orion Hindawi as CEO to Drive Next Chapter of Growth20.8.2026 21:10:00 CEST | Press release
Company to focus on deepening its Autonomous IT capabilities, further expanding AI offerings across its portfolio, and strengthening customer and partner engagement Tanium, a leader in Autonomous IT, today announced that Co-Founder and Executive Chairman Orion Hindawi has been appointed Chief Executive Officer, effective immediately. Dan Streetman is stepping down as CEO and as a member of the Board after leading Tanium for the last three years, during which time Tanium scaled its go-to-market operations and secured strong industry analyst recognition for innovation within the Tanium platform. Streetman will continue to advise Tanium as part of the transition and co-founder David Hindawi will return to the role of Chairman of the Board. With this foundation in place, this transition positions Tanium to deepen its Autonomous IT capabilities and further expand AI offerings across its portfolio and strengthen customer and partner engagement. The Hindawis co-founded Tanium in 2007 to build
PCI Energy Solutions to Join Mitsubishi Electric, Reinforcing Long-Term Commitment to Customers, Employees, and the Energy Industry20.8.2026 19:46:00 CEST | Press release
Mitsubishi Electric to retain PCI’s core management team and support continuity of operations, customer service, and product innovation PCI Energy Solutions ("PCI"), a leading U.S.-based provider of enterprise software for energy management and optimization, announced today that it has entered into a definitive agreement to be acquired by Mitsubishi Electric Corporation. The agreement was executed on August 20, 2026 (Japan Standard Time). The Transaction represents a strong endorsement of PCI's business, technology, employees, customer relationships and position in the energy industry. Mitsubishi Electric intends to retain PCI's core management team following completion of the Transaction, enabling PCI to preserve leadership continuity, industry expertise and its customer-focused operating model while benefiting from Mitsubishi Electric's global scale, complementary capabilities and long-term investment capacity. The agreement has been signed, but the Transaction has not yet closed. PC
Perma-Pipe Secures More Than $67 Million in New Orders in the Second Quarter of 202620.8.2026 19:02:00 CEST | Press release
Awards include significant Oil & Gas projects in MENA and Canada, as well as the Company's largest single leak detection project to date and continued growth at its new Ohio facility. Perma-Pipe International Holdings, Inc. (Nasdaq: PPIH), a global leader in engineered piping and corrosion protection solutions, today announced that the Company secured more than $67 million in new orders during the second quarter of fiscal 2026, representing strong demand across its strategically important end-markets and geographies. The orders further strengthen Perma-Pipe's backlog and provide increased visibility into future revenue growth. The Company's backlog remains well diversified across geographies, customers and end-markets. The Company continued to see strong demand across its core Oil & Gas and infrastructure markets, while also securing important new business in emerging applications, including advanced leak detection and monitoring solutions. During the quarter, Perma-Pipe secured sizabl
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