Articles from Azafaros B.V.

Azafaros, a company building a portfolio to become a leader in lysosomal storage disorders with the goal of addressing neurological symptoms, today announced that its lead asset, nizubaglustat, will feature in five scientific presentations accepted at the WORLDSymposium™ 2026, the leading global conference on lysosomal diseases, taking place in San Diego, California, USA between February 2-6.

Azafaros B.V. today announced that its lead asset, nizubaglustat, has been granted orphan drug designation from regulatory authorities in both the United States and the European Union for the treatment of GM1 gangliosidosis. Additionally, the company’s Clinical Trial Application (CTA) for two global Phase 3 studies investigating the drug’s efficacy and safety in GM1/GM2 gangliosidoses and Niemann-Pick Type C (NPC) was approved by multiple European countries. Azafaros expects to initiate the two global trials in Q2, 2025.

Azafaros B.V., a clinical-stage biotechnology company focused on rare diseases and lysosomal storage disorders, today announced that advancements with its lead asset, nizubaglustat, will feature prominently on the agenda at the upcoming WORLDSymposium™ 2025, the pre-eminent conference on lysosomal diseases.

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).

Azafaros B.V. today announced two key appointments, further strengthening its senior management team. Dr. Christian Freitag joins the company as Chief Medical Officer and Dr. Kyle Landskroner has been promoted to Chief Scientific Officer. With their comprehensive expertise in clinical and early-stage drug development, they will support the transition of Azafaros’ lead program, AZ-3102, into late-stage clinical evaluation and the expansion of Azafaros’ pipeline.

Azafaros B.V. today announced its participation at the 18th Annual WORLDSymposium™, a research conference dedicated to lysosomal storage diseases, taking place from February 7 – 11, 2022, in San Diego, CA. The conference is also offering virtual participation. Azafaros will share first-in-human data on its lead candidate AZ-3102, a novel orally available azasugar molecule with a unique dual mode of action, as well as the design for a natural history study in GM1 and GM2 gangliosidosis and preclinical in vivo data on AZ-3102 in Niemann-Pick and Sandhoff disease mouse models.

Azafaros B.V. today announced that Stefano Portolano, M.D., has joined Azafaros as Chief Executive Officer (CEO) and has concurrently been appointed to the Board of Directors of the company. Dr. Portolano is an experienced industry executive with a track record of successfully implementing commercialization and market access strategies across a broad spectrum of indications. Within his 25 years in the biotech and pharmaceutical industries, he has focused on leadership roles in organizations that develop orphan specialty medicines for patients with rare diseases.