Azafaros Strengthens Medical and Scientific Management Team Reflecting Clinical Progress

Leiden, Netherlands–(Business Wire) — Azafaros BV has today announced two key appointments, further strengthening its senior management team. Dr. Christian Freitag joins the company as Chief Medical Officer and Ph.D. Kyle Landskroner has been promoted to Chief Scientific Officer. With their combined expertise in clinical and early drug development, they will support the transition of Azafaros’ lead program, AZ-3102, to late-stage clinical evaluation and the expansion of Azafaros’ pipeline.

Christian Freitag is a medical doctor with over 20 years of experience in the pharmaceutical industry, having led global clinical development programs at companies such as Hoffmann La Roche, Shire and BTG. Prior to joining Azafaros, Dr. Freitag was Chief Medical Officer at Dynacure, responsible for medical and regulatory strategy, including clinical development of lead compounds in myotube and centronuclear myopathy (CNM). Dr. Freitag replaces Dr. Ruben Giorgino and is embarking on a new career path.

Azafaros Chief Medical Officer Christian Freitag commented: “Joining Azafaros at this time is very exciting. The company has important programs including gathering data highly relevant to the natural history of GM1 and GM2 gangliosidosis and executing AZ-3102 for the treatment of GM2 and Niemann-Pick disease type C Phase 2 study. We also strongly encourage our plans to move forward with a Phase 3 trial given the promising data from preclinical and Phase 1 studies.”

Further strengthening the executive team as the company moves into the next phase of clinical development, Dr. Kyle Landskroner, Chief Scientific Officer, has been 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, CEO of Azafaros, said: “I am pleased to announce the strengthening of the management team with new and existing capabilities. With the recent IND approval and FDA Fast Track designation, we are ready to move our lead product, AZ-3102, into Phase 2. With Christian and Kyle will bring valuable insight and experience as Azafaros enters our next phase of clinical development. The mission is to bring disease-modifying treatments to patients in need. I would like to take this opportunity to thank Ruben for getting the company to where it is today significant contribution.”

More information on Dr. Christian Freitag and Dr. Find Kyle Landskroner here.

About AZ-3102

AZ-3102 is a therapeutic candidate being developed for patients with lysosomal storage disorders (LSD) with neurological involvement. AZ-3102 is an orally available, brain-permeable azasaccharide with a unique dual mode of action by inhibiting two key enzymes that regulate glucose and lipid metabolism.

In 2022, the compound received Fast Track designation for GM1 and GM2 hypergangliosidosis and Niemann-Pick disease type C (NP-C), as well as for GM2 hypergangliosidosis (Sandhoff and Tai Sachs disease) and NP-C from the US Food and Drug Administration.

About Lysosomal Storage Diseases

Lysosomal storage disorders are a group of more than 70 disorders characterized by lysosomal dysfunction, most of which are autosomal recessive. Individually, these disorders are rare but collectively affect 1 in 5,000 live births. They usually appear in infancy and childhood, but there are adult-onset forms as well.

They are caused by genetic mutations that affect the function of specific enzymes, transporters, receptors or hormones involved in metabolizing and transporting body components such as sugars, proteins and lipids.

These malfunctions may impair the assembly of key metabolic end products required for proper body function, or lead to the deleterious accumulation 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 in the central nervous system (CNS), respectively, leading to progressive and severe Neurological impairment and early death. These disorders mainly affect infants and children, and there are currently no disease-modifying treatments.

Niemann-Pick disease type C (NP-C) is a progressive, life-limiting neurological disorder caused by mutations in the NPC1 or NPC2 genes and abnormal endosome-lysosomal trafficking, leading to accumulation of gangliosides). Onset of the disease occurs throughout the lifespan, from prenatal through adulthood. The pillar of treatment is symptom management.

About Azafaros

Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms from a compound library at Leiden University, led by a team of experienced industry experts. Azafaros aims to establish a portfolio of disease-modifying therapies to provide new treatment options for patients and their families. The company’s lead clinical-stage program is AZ-3102, a small molecule azarose, which is orally administered and brain permeable, with the potential to treat GM1 hypergangliosidosis and GM2 hypergangliosidosis (Tay-Sachs and Sandhoff disease ) and Niemann Pick disease type C (NP-C). By applying its expertise, network and courage, the Azafaros team challenges traditional development pathways to rapidly bring new medicines to the rare disease patients who need them. Azafaros is backed by a consortium of leading Dutch and Swiss investors, including Forbion, BioGeneration Ventures, BioMedPartners and Schroder Capital.

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