Azafaros Secures € 132M in Oversubscribed Series B Financing to Advance Phase 3 Clinical Programs of Innovative Therapies in Lysosomal Storage Disorders
Azafaros, a clinical-stage company focused on developing disease-modifying therapeutics to offer new treatment options to patients with rare lysosomal storage disorders, announces the completion of an oversubscribed €132M Series B financing led by Jeito Capital, co-led by Forbion Growth and with additional participation from Seroba, Pictet Group and existing investors Forbion Ventures, Schroders Capital and BioGeneration Ventures (BGV).
Positive Niemann-Pick disease type C (NPC) and GM2 gangliosidosis data from nizubaglustat Phase 2 RAINBOW study conducted by Azafaros presented at major metabolic disease conference
Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients with Niemann-Pick disease type C (NPC) or GM2 gangliosidosis, were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal.
Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients
Azafaros B.V. today announced that the first patient has been enrolled into its Phase 2 RAINBOW study (NCT05758922). The clinical trial is being conducted in Brazil and the US and will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of its lead asset, AZ-3102, in patients with GM2 gangliosidosis and Niemann-Pick disease type C (NP-C).
Azafaros Appoints Chief Operating Officer and Head of Finance
Azafaros B.V. today announced two appointments to their executive management team. Silvia Ragno, PhD, joins the company as Chief Operating Officer and Henrik Torp Nielsen as Head of Finance. With 35 years of combined experience in the pharmaceutical industry, they join as Azafaros prepares to start a clinical Phase 2 trial, the RAINBOW study (NCT05758922), with its lead asset AZ-3102, treating, for the first time, patients with GM2 gangliosidosis and Niemann-Pick disease type C.
Azafaros Receives Additional Regulatory Designations for AZ-3102 from FDA, EMA and MHRA
Azafaros B.V. today announced its progress in ongoing interactions with Health Authorities regarding its lead asset, AZ-3102.
Azafaros Receives FDA's IND Clearance and Fast Track Designation for Lead Asset AZ-3102
Azafaros B.V. today announced that it has received Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) to conduct a clinical Phase 2 trial for its lead asset, AZ-3102, for the treatment of GM2 gangliosidosis (GM2) and Niemann-Pick disease type C (NP-C). In addition to the IND clearance, the FDA has granted Fast Track Designation for the investigation of AZ-3102 for GM1 and GM2 gangliosidoses and NP-C. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.1
Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease
Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in Niemann-Pick disease type C (NP-C). The designation was based on promising preclinical data of AZ-3120 in a NP-C mouse model, recently presented at the 18th Annual WORLDSymposium™. AZ-3102, Azafaros’ lead program, is currently in clinical development as a potential treatment for the rare lysosomal storage diseases GM1 and GM2 Gangliosidoses and has completed a successful first-in-human clinical study in healthy subjects showing positive safety, tolerability, and pharmacodynamics data. The compound already received ODD from the FDA for GM2 Gangliosidosis including both Sandhoff and Tay-Sachs diseases. Based on its mode of action, AZ-3102 has broad applicability in addressing these inherited metabolic disorders.
Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ Conference
Azafaros B.V. announced positive clinical data from its first-in-human Phase 1 study with AZ-3102, the company’s lead program in development as a potential treatment for pediatric neurogenetic lysosomal storage disorders (LSDs). AZ-3102 is an azasugar, orally available, small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism by modulating the metabolism of glycosphingolipids. Azafaros also presented the design of PRONTO, a prospective longitudinal global study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 Gangliosidoses.
Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis
Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel oral small molecule, in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases. The ODD for GM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survival. These promising preclinical findings support Azafaros’ strategy to develop AZ-3102 as a potential disease-modifying treatment for GM1 and GM2 gangliosidoses and other neurogenetic, pediatric, metabolic lysosomal disorders. The company has successfully completed a Phase 1 clinical trial with AZ-3102 in healthy volunteers and will be presenting first-in-human clinical data from the study at the 18th Annual WORLDSymposium™, being held from February 7 – 11, 2022, in San Diego, CA.
