Leiden, Netherlands–(business wire)– Azafaros BV today received Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) to conduct a Phase 2 clinical trial of AZ-3102, a lead asset for the treatment of GM2 announced that Gangliosidosis (GM2) and Niemann-Pick disease type C (NP-C). In addition to IND approval, the FDA granted AZ-3102 Fast Track designation for GM1 and GM2 gangliosidosis and NP-C investigations. Fast Track is a process designed to expedite the development and expedite review of medicines that treat serious conditions and meet unmet medical needs.1
Azafaros’ RAINBOW Phase 2 Study is a Multinational, Double-Blind Study Investigating the Safety, Tolerability, and Pharmacokinetics of Once-Daily Oral AZ-3102 for 12 Weeks in GM2 and NP-C Patients , placebo-controlled, parallel-group trials. From 12 to 20 years old. This research is being conducted in other countries as well as the United States. The pharmacodynamics and effects of AZ-3102 on potential disease biomarkers will also be measured during the study. Professor Mark C. Patterson, MD, Professor of Neurology, Pediatrics, and Medical Genetics, Pediatric Neurology, Mayo Clinic, Rochester, Minnesota, is the principal investigator for the US portion of the study.
“GM2 gangliosidosis and Niemann-Pick disease type C are debilitating and life-threatening diseases that have a devastating impact on patients and their families, and new treatment options are urgently needed.” AZ-3102 has unique characteristics that make it potentially an effective and innovative treatment for these patients.” Professor Patterson.
Stefano Portolano, MD, CEO of Azafaros, added: We look forward to furthering our Phase 2 study and initiating a pivotal Phase 3 study, marking two important milestones in providing a new treatment option for patients with severe and rare genetic diseases. looking forward to ”
AZ-3102 is a therapeutic candidate developed for people with lysosomal storage diseases (LSD) with neurological involvement. AZ-3102 is an orally available, brain-penetrating azasaccharide designed with a unique dual mechanism of action by inhibiting two key enzymes that regulate glycosphingolipid metabolism.
In 2022, the compound received Fast Track Designation for GM1 and GM2 gangliosidosis, Niemann-Pick disease type C (NP-C), GM2 gangliosidosis (Sandhoff and Tay-Sachs disease) and C. from NP- FDA
About lysosomal storage diseases
Lysosomal storage diseases are a group of over 70 diseases characterized by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These disorders are individually rare, but collectively affect her 1 in 5,000 live births. They usually begin in infancy and childhood, but adult-onset forms also occur.
They are caused by genetic mutations that affect the function of certain enzymes, transporters, receptors, or hormones involved in the metabolism and transport of body components such as sugars, proteins, and lipids.
These malfunctions can impair the assembly of important metabolic end products required for normal functioning of the body, or lead to harmful accumulation of intermediate metabolites.
GM1 and GM2 gangliosidosis (Tay-Sachs and Sandhoff disease), Niemann-Pick, Krabbe, Farber, Fabry, and Gaucher disease are examples of lysosomal lipid storage disorders.
GM1 and GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) are caused by accumulation of GM1 or GM2 gangliosides in the central nervous system (CNS), respectively, and are associated with progressive and severe neurological disability and early death. Brings These diseases primarily affect infants and children, and no disease-modifying treatments are currently available.
Niemann-Pick disease type C (NP-C) is a progressive, life-limiting neurological condition caused by mutations in the NPC1 or NPC2 genes and aberrant endosomal-lysosomal trafficking, and is associated with a variety of ganglioside-containing disorders in the CNS. Causes lipid accumulation. Disease onset occurs throughout the life of the affected individual, from prenatal to adulthood. The focus of treatment is symptom management.
Azafaros is a clinical-stage company founded in 2018, led by a team of experienced industry experts with a deep understanding of the mechanisms of rare genetic diseases, the compound library of Leiden University. Azafaros aims to build a pipeline of disease-modifying therapies to provide new treatment options for patients and their families. The Company’s lead clinical-stage programs are GM1 and GM2 gangliosidosis (Tay-Sachs and Sandhoff disease) and Niemann-Pick disease type C (NP-C). Azafaros’ team leverages its know-how, network and courage to challenge traditional development paths and bring new medicines to patients with rare diseases in need faster. Azafaros is backed by a syndicate of leading Dutch and Swiss investors including Forbion, BioGeneration Ventures, BioMedPartners and Schroder Capital.