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
VELOCIFERO Takes Action Against Counterfeiting in China13.11.2026 17:32:00 CET | Press release
White s.r.o., exclusive owner of the VELOCIFERO brand, takes action in China to protect its intellectual property rights. Initial measures in Chongqing and Taizhou have already resulted in the seizure of vehicles alleged to be counterfeit. VELOCIFERO is taking action against counterfeiting in China. White s.r.o., the exclusive owner of the VELOCIFERO brand and its related intellectual property rights, has launched decisive measures to protect its creative and industrial assets. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261113797570/en/ Photo courtesy of White s.r.o. “Vehicle sealed in Chongqing as part of actions undertaken to protect VELOCIFERO’s intellectual property rights.” The matter originated in June 2026 when, according to the company’s complaint, computers were removed from the technical office of Taizhou Siweiluo, the company appointed to manufacture VELOCIFERO products. White s.r.o. subsequently became aware
LangChain Raises $300M to Enable Every Company to Own Their Intelligence13.11.2026 16:53:00 CET | Press release
CapitalG leads Series C funding round at a $3B valuation to advance LangChain's agent engineering platform LangChain today announced it has raised $300 million in new funding at a $3 billion valuation, bringing total funding to date to $460 million. The round was led by existing investor CapitalG, with significant participation from new investors GIC and DFJ Growth and existing investors IVP and Sapphire Ventures. LangChain is advancing its mission to help every company own its intelligence. As agents transform the economy, companies are building domain-specific agents differentiated by their knowledge, data, and user experience. These agents will power new products and transform internal company operations, creating lasting competitive advantages for the companies that control them. "Agents are going to transform how companies operate and the products they deliver. The companies that control these systems will build lasting competitive advantages," said Harrison Chase, Co-Founder and
Kinaxis Named a Leader in Five 2026 IDC MarketScapes for Worldwide Supply Chain Planning13.11.2026 13:00:00 CET | Press release
Maestro delivers concurrent planning on a single platform across discrete, process, consumer packaged goods and life sciences Kinaxis® Inc. (TSX:KXS), a global leader in end-to-end supply chain planning and orchestration, today announced it has been named a Leader in five 2026 IDC MarketScape vendor assessments for worldwide supply chain planning. The recognition spans the overall market and four industries - discrete industries, process industries, consumer packaged goods and life sciences industries - all served by a single platform, Kinaxis Maestro®, the company's AI-powered supply chain orchestration platform. "Supply chain leaders don't need a better forecast once a month. They need to know what changed this morning, what it means across the business, and what to do about it," said Andrew Bell, Chief Product Officer of Kinaxis. "I believe being named a Leader across five IDC MarketScape vendor assessments, in industries that plan in very different ways, shows that a single concurr
SBC Medical Announces Capital and Business Alliance with NeuroBrain Dynamics to Advance Clinic Profitability Through Medical AI13.11.2026 12:30:00 CET | Press release
Companies to co-develop six medical AI solutions targeting revenue expansion and cost reduction; SBC Medical secures priority sales rights in Japan and South Korea and targets B2B expansion across Asia SBC Medical Group Holdings Incorporated (Nasdaq: SBC) (“The Company” or “SBC Medical”), a Medical Services Organization (MSO) providing management support across a wide range of healthcare fields, today announced a strategic capital and business alliance agreement with NeuroBrain Dynamics Inc. (“NBD”), a Taipei-based company dedicated to enterprise generative AI solutions. As part of the agreement executed on November 13, 2026, SBC Medical has made a minority equity investment in NBD. Through this alliance, SBC Medical plans to introduce two complementary suites of generative AI solutions across its network of 287 clinics in Japan and overseas*: “revenue-growth AI,” to elevate average revenue per visit and customer retention through personalized customer guidance, and “cost-reduction AI,
SBC Medical Group Holdings Reports Third Quarter 2026 Financial Results13.11.2026 12:30:00 CET | Press release
Revenue Up 35%, Net Income Attributable to SBC Medical Up 22%, and Adjusted EBITDA1 Up 66% Year-over-Year; Net Income Margin of 27% and Adjusted EBITDA Margin of 47% — Previously Announced Service Fee Revisions and New Clinic Openings Contribute to Revenue Growth and Operating Margin Improvement SBC Medical Group Holdings Incorporated (Nasdaq: SBC) (“SBC Medical” or the “Company”), a Medical Services Organization (MSO) providing management support across a wide range of healthcare fields to medical institutions in Japan and abroad, today announced its consolidated financial results for the third quarter of fiscal year 2026 (the three months ended September 30, 2026) and the three cumulative quarters of fiscal year 2026 (the nine months ended September 30, 2026). Third Quarter 2026 Financial Highlights Total revenues were $59 million, up 35% year-over-year. Income from operations was $26 million, up 66% year-over-year, with an operating margin of 45%. Net income attributable to SBC Medi
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