Phase 3 Studies for Azafaros Receive Key Regulatory Approvals, Set to Begin in 2025
Azafaros B.V. today revealed that its leading asset, nizubaglustat, has received Orphan Drug Designation (ODD) from both U.S. and European regulatory authorities for the treatment of GM1 gangliosidosis. Additionally, the company’s Clinical Trial Application (CTA) for two global Phase 3 studies examining the efficacy and safety of the drug in GM1/GM2 gangliosidoses and Niemann-Pick Type C (NPC) has been approved across several European countries. Azafaros anticipates launching these global Phase 3 trials in the second quarter of 2025.
Azafaros B.V., a clinical-stage company focused on developing treatments for rare lysosomal storage disorders, has made significant progress with its lead product, nizubaglustat. This small molecule drug shows great promise as a potential treatment for diseases involving neurological impairment, including GM1/GM2 gangliosidoses and NPC. Azafaros recently received both Orphan Drug Designation (ODD) and Orphan Medicinal Product Designation (OMPD) from U.S. and European regulatory authorities for GM1 gangliosidosis, a rare and severe condition with no approved treatments.
These designations are pivotal for the continued development of nizubaglustat, positioning it as a potential first-in-class therapy for GM1 and GM2 gangliosidoses and a best-in-class therapy for NPC. Earlier this year, the company reported positive topline data from a successful Phase 2 study. The study demonstrated that nizubaglustat had a favorable safety profile and showed early signs of efficacy, with clinical endpoints improving or stabilizing in the majority of patients. These promising findings have paved the way for further Phase 3 trials, reinforcing the potential of nizubaglustat to address these debilitating diseases.
Commenting on the orphan drug designations, Stefano Portolano, Chief Executive Officer of Azafaros, expressed the company’s excitement about these approvals. He emphasized their strategic importance as Azafaros prepares to initiate its Phase 3 trials in Q2 2025. Portolano also highlighted the dual mode of action of nizubaglustat, which differentiates it from existing therapies and underscores its potential to be a groundbreaking treatment for these rare neurological disorders. He extended his gratitude to the patients and their families who contributed to the clinical progress of the drug.
The orphan drug designations are a major milestone for Azafaros, particularly in light of the recent promotion of Anke Arnold-Tugulu to the role of Chief Regulatory Officer. Dr. Arnold-Tugulu, who continues to lead Azafaros’ regulatory strategy, remarked on the importance of these designations in advancing the development of nizubaglustat, especially for GM1 gangliosidosis, for which there are currently no approved treatments. She also acknowledged the potential for nizubaglustat to provide new hope for patients suffering from these diseases.
Orphan drug designations are highly valuable for companies focusing on rare diseases. They provide essential incentives such as market exclusivity and reduced regulatory fees, which help foster innovation and are critical for developing therapies for conditions like GM1 gangliosidosis, GM2 gangliosidosis, and NPC, where treatment options are extremely limited. With orphan drug status secured in both the U.S. and the European Union, Azafaros is well-positioned to accelerate the development of nizubaglustat and bring it closer to patients in need.
Nizubaglustat itself is an orally available, brain-penetrant azasugar with a unique dual mechanism of action. It is being developed for the treatment of rare lysosomal storage disorders, including GM1 and GM2 gangliosidoses and NPC. In addition to the ODD and OMPD designations, nizubaglustat has received multiple forms of regulatory support from key health authorities. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designations (RPDD) for GM1, GM2 gangliosidoses, and NPC, as well as Orphan Drug Designations (ODD) for GM2 gangliosidosis and NPC. Furthermore, nizubaglustat has received Investigational New Drug (IND) clearance and Fast Track Designation for GM1/GM2 gangliosidoses and NPC from the FDA.
In Europe, nizubaglustat has received Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA) for GM2 gangliosidosis. In the UK, the Medicines and Healthcare Products Regulatory Agency (MHRA) has granted an Innovation Passport for the treatment of GM1 and GM2 gangliosidoses.
GM1 and GM2 gangliosidoses are life-threatening lysosomal storage disorders caused by the accumulation of specific gangliosides in the central nervous system, resulting in severe neurological impairment and premature death, particularly in infants and children. These diseases are progressive, with no disease-modifying therapies currently available. Similarly, Niemann-Pick disease Type C (NPC) is a progressive neurological disorder caused by mutations in the NPC1 or NPC2 gene, leading to abnormal lipid accumulation in the CNS. Like GM1 and GM2 gangliosidoses, NPC has limited treatment options, and patients typically experience life-limiting effects of the disease.
Azafaros, founded in 2018, has established a strong scientific foundation, with groundbreaking discoveries made by researchers at Leiden University and Amsterdam UMC. The company’s leadership team comprises highly experienced professionals who are dedicated to addressing rare genetic diseases and advancing new treatment options for patients. Azafaros is actively working on advancing its pipeline of disease-modifying therapeutics, including progressing its Phase 3 clinical studies for nizubaglustat in GM1, GM2 gangliosidoses, and NPC.
Azafaros is supported by a syndicate of leading Dutch and Swiss investors, including Forbion, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital. The company is committed to swiftly advancing its Phase 3 clinical trials, focusing on addressing the needs of patients affected by rare, often neglected diseases. Through its Phase 3 studies, Azafaros aims to provide promising, disease-modifying therapies that could offer new hope for these life-threatening conditions.
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