LEIDEN, Netherlands–(BUSINESS WIRE)–Azafaros BV today announced two key appointments, further strengthening its management team. The Doctor. Christian Freitag joins the company as Chief Medical Officer and Dr. Kyle Landskroner has been promoted to Scientific Director. With their extensive experience in early-stage and clinical drug development, they will support the transition of Azafaros’ flagship program, AZ-3102, to late-stage clinical evaluation and expansion of Azafaros’ pipeline.
Christian Freitag, MD, brings over 20 years of experience in the pharmaceutical industry with positions at companies such as Hoffmann La Roche, Shire and BTG, where he led global clinical development projects. Prior to joining Azafaros, Dr. Freitag was Medical Director at Dynacure, where he was responsible for medical and regulatory strategy, including clinical development of the lead compound in myotubular and centronuclear myopathies (CNM). The Doctor. Freitag replaces Dr. Ruben Giorgino, who is starting a new career.
Christian Freitag, Medical Director at Azafaros, said: “Joining Azafaros at this time is very exciting. The company has significant plans, including the collection of highly relevant data on the natural history of GM1 and GM2 gangliosidosis and the completion of the AZ-3102 Phase 2 study for the treatment of GM2 and Niemann’s disease. Type C selection. We are also very encouraged to move forward with our design for the Phase 3 study in light of the promising data from the preclinical and Phase 1 studies.”
To further strengthen the management team as the company moves into the next stage of clinical development, doctor Kyle Landskroner, Scientific Director, was promoted from his previous role as head of preclinical drug development. The Doctor. Landskroner will be responsible for advancing Azafaros products into clinical development.
Stefano Portolano, General Manager of Azafaros, said: “It is with great satisfaction that I announce the reinforcement of the management team with new and consolidated skills. With the recent IND clearance and Fast Track designation from the FDA, we are on the verge of moving our flagship product AZ-3102 into Phase 2. Christian and Kyle will bring valuable information and experience as Azafaros progresses to the next level of clinical development in our mission to bring disease-modifying treatment options to patients in need. I would like to take this opportunity to thank Ruben for the significant contribution he has made to bringing the company to where it is today.
More information about Dr. Christian Freitag and Dr. Kyle Landskroner can be found here.
AZ-3102 is a therapeutic candidate developed for people with Lysosomal Storage Disorder (LSD) with neurological impairment. AZ-3102 is an orally available brain penetrating azasugar designed to have a unique dual mode of action, inhibiting two key enzymes that modulate glycosphingolipid metabolism.
In 2022, the compound received Fast Track designation for GM1 and GM2 gangliosidosis, as well as Niemann-Pick disease type C (NP-C) and orphan drug (ODD) designation for GM2 gangliosidosis (Sandhoff and Tay-Sachs diseases) and FDA NP-C.
About Lysosomal Storage Disorders
Lysosomal storage disorders are a group of more than 70 diseases characterized by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These disorders are individually rare, but collectively affect 1 in 5,000 live births. They usually present in infancy and childhood, although adult-onset forms also occur.
They are caused by genetic mutations that affect the function of specific enzymes, transporters, receptors or hormones involved in the metabolism and transport of the body’s building blocks such as sugars, proteins and lipids.
These dysfunctions can impair the assembly of crucial metabolic end products needed for normal body functioning or lead to harmful accumulations of intermediate metabolites.
GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases), Niemann-Pick, Krabbe, Farber, Fabry and Gaucher diseases are examples of lysosomal lipid storage disorders.
GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are caused by accumulation of GM1 or GM2 gangliosides, respectively, in the central nervous system (CNS), resulting in progressive and severe neurological damage and early death. These diseases primarily affect infants and children, and no disease-modifying therapies 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, resulting in the accumulation of various lipids, including gangliosides, in the CNS. The onset of the disease occurs throughout life, from prenatal life to adulthood. The mainstay of therapy is symptom control.
Azafaros is a clinical stage company founded in 2018 with a deep understanding of the mechanisms of rare genetic diseases, a library comprised of Leiden University and led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapies to provide patients and their families with new treatment options. The company’s flagship clinical internship program is AZ-3102, an orally available, brain-penetrating small molecule azasugar with the potential to treat GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff) and Niemann Pick disease type C ( NP-C). Applying their know-how, network and courage, the Azafaros team challenges traditional development paths to rapidly bring new medicines to rare disease patients who need them. Azafaros is backed by a syndicate of leading Dutch and Swiss investors, including Forbion, BioGeneration Ventures, BioMedPartners and Schroder Capital.