Expanded Access Policy

Azafaros’ lead asset AZ-3102 is currently being investigated in clinical trials for the treatment of GM2 gangliosidosis and Niemann Pick type C disease. If successful, data generated during the pivotal clinical trials may provide the basis for drug approval submissions to regulatory authorities, including the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). Some patients may be interested in seeking access to investigational drugs before the regulatory approval of a drug via compassionate use or expanded access. At the current stage of drug development, it is Azafaros’ policy to provide access to AZ-3102 only as part of one of our clinical trials. This Policy may be reviewed and amended in the future.