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
Murata Launches Ultra-Low Power AMR Sensors to Boost Battery Life in Healthcare and Wearables Devices27.4.2026 04:00:00 CEST | Press release
Murata Manufacturing Co., Ltd. (TOKYO: 6981) (ISIN: JP3914400001) has commenced mass production of its MRMS166R and MRMS168R anisotropic magnetoresistance (AMR) sensors for healthcare, wearable, and IoT devices. The MRMS166R is the first AMR sensor to combine an average current consumption of 20 nA with operation from a 1.2 V supply, enabling extended battery life in coin cell-powered systems. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260413296947/en/ [Murata Manufacturing Co., Ltd.] AMR sensor The devices are solid-state magnetic sensors used for switching applications. They detect the presence or absence of a magnetic field and generate an output signal that system logic uses to control functions such as transitions between active and sleep modes. This enables contactless switching without mechanical components, improving reliability, and supporting sealed, miniaturized designs. Automatic switching between active and
Sun Pharma signs Definitive Agreement to Acquire Organon27.4.2026 01:51:00 CEST | Press release
Organon stockholders to receive US$ 14.00 per share in cashThe deal values Organon at EV of US$ 11.75 billionCombined Business leverages complementary portfolios and global scale for sustained long‑term value creation Sun Pharmaceutical Industries Limited(Reuters: SUN.BO, Bloomberg: SUNP IN, NSE: SUNPHARMA, BSE: 524715) (together with its subsidiaries and/or associated companies, “Sun Pharma”) and Organon & Co. (NYSE: OGN) (“Organon”) today announced that they have entered into a definitive agreement under which Sun Pharma will acquire all outstanding shares of Organon for US$ 14.00 per share in an all‑cash transaction with an enterprise valuation of US$ 11.75 billion. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260426881370/en/ Organon is a global healthcare company formed through a spinoff from Merck, known as MSD outside of the United States and Canada, in 2021. Organon has a legacy of deep trust and strong brand equit
Andersen Global udvider sin tilstedeværelse i Afrika med lanceringen af medlemsfirma i Cameroun26.4.2026 20:56:00 CEST | Pressemeddelelse
Andersen Global træder ind på det camerounske marked, idet Phoenix Advisory bliver medlemsfirma og overgår til Andersen-brandet. Andersen in Cameroun er et specialiseret rådgivningsfirma, der leverer strategiske, erhvervsmæssige, finansielle og operationelle rådgivningsydelser til organisationer i forskellige vækstfaser. Firmaet er grundlagt af fagfolk med erfaring fra store internationale organisationer og kombinerer et stærkt lokalt og centralafrikansk funderet kendskab til forretningsmæssige, juridiske og skattemæssige regler med en skræddersyet tilgang, der har til formål at skabe konkrete og målbare resultater for klienterne. "Overgangen til Andersen-brandet afspejler vores engagement i at vokse sammen med vores kunder og imødekomme stadig mere komplekse behov," udtalte administrerende partner Albert Désiré Zang. "Vi tror på, at langsigtet succes skabes gennem tillid, kvalitet og vedvarende support. At blive et medlemsfirma styrker vores evne til at levere gennemtænkte, bæredygtig
PMCOrganometallix Announces Price Increase on All Products24.4.2026 22:55:00 CEST | Press release
Due to significant changes in market conditions, PMC Organometallix, Inc. announces that effective May 1, 2026, or as contracts permit, prices across all product lines globally will increase by 10-25%. This adjustment is driven by sustained cost pressures from key inputs including rising raw material costs and escalating freight and logistics expenses. While the company has been absorbing these increases, the current economic environment brought on by the geopolitical crisis of the Iran conflict requires this adjustment to continue providing the high-quality, consistent materials and supply reliability that customers expect. PMC Organometallix will implement these changes in a transparent, collaborative manner and values your partnership while navigating these economic challenges. Customers with questions or to discuss a specific situation should contact their account representative. About PMC Group PMC Group is a growth-oriented, diversified, global chemicals and plastics company deli
Frankfurt Higher Regional Court upholds BESREMi® arbitral award in favor of AOP Health24.4.2026 18:52:00 CEST | Press release
Today, the Higher Regional Court of Frankfurt upheld the February 20251 partial final ICC arbitral award in favor of AOP Orphan Pharmaceuticals GmbH (“AOP Health”) in its dispute with PharmaEssentia Corp. (“PharmaEssentia”). The ruling confirms the award which found the Taiwanese company to be liable for certain damages. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260424005227/en/ Portrait Dr. Rudolf Widmann, Founder AOP Health Credit: AOP Health/Daniel Ospelt Dr. Rudolf Widmann, one of the two founders of AOP Health, explains: “We very much welcome the Frankfurt Higher Regional Court’s decision that confirms our position. In the interest of our patients, we are dedicated to maintaining stable and sustainable access to BESREMi® and to responsibly navigating future challenges.” The Product in Dispute The conflict centers around BESREMi® (ropeginterferon alfa-2b), a product launched in 2019 and developed by AOP Health into
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