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Azafaros strengthens its medical and scientific leadership team reflecting clinical advances


LEIDEN, Netherlands–(BUSINESS WIRE)–Azafaros BV today announced two key appointments, further strengthening its management team. Dr. Christian Freitag joins the company as Medical Director and Dr. Kyle Landskroner has been promoted to Scientific Director. With their comprehensive expertise in early-stage and clinical drug development, they will support the transition of Azafaros’ lead program, AZ-3102, into late-stage clinical evaluation and expansion of the Azafaros pipeline.

Christian Freitag, MD, brings over 20 years of experience in the pharmaceutical industry with roles at companies including 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). Dr. Freitag replaces Dr. Ruben Giorgino, who is starting a new career.

Christian Freitag, Chief Medical Officer of Azafaros, said: “Joining Azafaros at this time is very exciting. The company has significant plans, including collecting highly relevant data on the natural history of GM1 and GM2 gangliosidosis and completing the AZ-3102 Phase 2 study for the treatment of GM2 and Niemann’s disease. Type C pick. We are also very encouraged to move forward with our planning for the Phase 3 trial, 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, Dr. Kyle Landskroner, Scientific Director, was promoted from his previous position as head of preclinical drug development. Dr. Landskroner will be responsible for advancing Azafaros’ products into clinical development.

Stefano Portolano, General Manager of Azafaros, said: “I am very pleased to announce the reinforcement of the management team with new and established skills. With recent IND clearance and Fast Track designation from the FDA, we are on the verge of moving our lead product AZ-3102 into Phase 2. Both Christian and Kyle will bring valuable insight and experience as Azafaros progresses towards the next level of clinical development on 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 in bringing the company to where it is today.

More information about Dr. Christian Freitag and Dr. Kyle Landskroner can be found here.

About AZ-3102

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 by 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 Designations (ODD) for GM2 gangliosidosis (Sandhoff’s diseases and Tay-Sachs) and FDA NP-C.

About Lysosomal Storage Disorders

Lysosomal storage disorders are a group of over 70 diseases that are 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 metabolizing and transporting the body’s building blocks such as sugars, proteins and lipids.

These dysfunctions can either impair the assembly of crucial metabolic end products necessary for the normal functioning of the organism, 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 the accumulation of GM1 or GM2 gangliosides, respectively, in the central nervous system (CNS), resulting in progressive and severe neurological damage and death early. These diseases primarily affect infants and children, and no disease-modifying therapy is 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 management.

About Azafaros

Azafaros is a clinical-stage company founded in 2018 with an in-depth understanding of the mechanisms of rare genetic diseases, a compound library from 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 lead clinical-stage 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). By applying their know-how, network and courage, the Azafaros team challenges traditional development pathways 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.



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