Azafaros B.V. today announced two appointments to their executive management team. Silvia Ragno, PhD, joins the company as Chief Operating Officer and Henrik Torp Nielsen as Head of Finance. With 35 years of combined experience in the pharmaceutical industry, they join as Azafaros prepares to start a clinical Phase 2 trial, the RAINBOW study (NCT05758922), with its lead asset AZ-3102, treating, for the first time, patients with GM2 gangliosidosis and Niemann-Pick disease type C.

“We are excited to welcome Silvia and Henrik to our team. Silvia brings a wealth of experience in the start-up environment, drug development, business development, and rare disease areas. Her primary responsibility as COO will be strategic planning and execution of our investor relations and business development efforts,” said Stefano Portolano, Chief Executive Officer of Azafaros. “Henrik has previously occupied diverse finance roles in numerous countries and is a formidable addition to our team as Head of Finance. His international experience in business development and innovation on the medical market will be a considerable asset to the company.”

Dr. Ragno worked in the global pharmaceutical industry for 20 years before joining Azafaros. She co-founded Stargazer Pharmaceuticals Inc. and acted as Chief Operating Officer for the company leading the preclinical and clinical program strategy for the development of a new treatment for the rare genetic Stargardt’s disease. She successfully raised the first tranche of Series A funding from Novo Holdings, the founding investor of Stargazer, while working as Senior Director, External Asset Leader, for Takeda Development Centre Europe's TAKcelerator™, an incubator-like group accelerating transformative therapies for people with rare diseases. Prior to this, she worked at Novartis Pharmaceutical UK as Global Program Team Director, leading various programs in several therapeutic areas.

With more than 15 years of experience in the international pharmaceutical industry, Mr. Nielsen brings a particularly diversified knowledge of business development, innovation, and medical market access to Azafaros. He previously held several senior roles at Novartis, notably European Head of Business Development Finance, Oncology, as well as Nordic Chief Financial Officer, Oncology and Head of Commercial and Medical Finance, successfully supporting and closing several major business development deals.

Further information on Dr. Silvia Ragno and Henrik Torp Nielsen can be found here.

About AZ-3102

AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).

In the beginning of 2023, AZ-3102 received two Rare Pediatric Disease Designations (RPDD) by the United States Food and Drug Administration (FDA) for the treatment of GM1 and GM2 gangliosidoses, Orphan Medicinal Product Designation (OMPD) by the European Medicines Agency (EMA) for the treatment of GM2 gangliosidosis as well as an Innovation Passport by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of GM1 and GM2 gangliosidoses.

In 2022, the compound received Fast Track Designation for GM1 and GM2 gangliosidoses as well as Niemann-Pick disease Type C (NP-C), and Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NP-C from the FDA.

About GM1 and GM2 Gangliosidoses

GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides, respectively, in the central nervous system (CNS), resulting in progressive and severe neurological impairment and early death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available.

About Niemann-Pick Disease Type C (NP-C)

Niemann-Pick disease type C (NP-C) is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of disease happens throughout the lifespan of an affected individual, from prenatal life through adulthood. The mainstay of therapy is symptom management.

About Azafaros

Azafaros is a clinical stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms, 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 therapeutics to offer patients and their families new treatment options. The company’s lead clinical-stage program is AZ-3102, a small molecule azasugar, orally available and brain penetrant, with the potential to treat GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) and Niemann Pick disease type C (NP-C). By applying its know-how, network, and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by a syndicate of leading Dutch and Swiss investors including Forbion, BioGeneration Ventures, BioMedPartners and Schroders Capital.

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