- Promotion of Dr Arnold-Tugulu comes after several major regulatory milestones reached for our lead asset, nizubaglustat
Leiden, The Netherlands, 2 April 2024 – Azafaros B.V. today announced the promotion of its regulatory lead, Dr Anke Arnold-Tugulu, to the position of Chief Regulatory Officer.
Since joining Azafaros in April 2022, Dr Arnold-Tugulu has led the company through numerous regulatory interactions with the EMA and FDA, building a strong relationship with these authorities. This resulted in Azafaros receiving valuable feedback on our lead asset’s development plan and achieving several valuable designations, which acknowledges the high unmet need in our target diseases, GM1 and GM2 gangliosidoses and Niemann Pick type C.
Prior to joining Azafaros, Dr Arnold-Tugulu held roles of increasing responsibility at Actelion Pharmaceuticals in Switzerland. She then became Regulatory Affairs Manager at Hoffmann La Roche in Basel, later joining CSL Behring as Director of Regulatory Affairs, leading the therapeutic areas for transplantation and respiratory treatments in Europe. More recently, she worked for smaller companies such as Agios as Head of International Regulatory Affairs, and Swedish Orphan Biovitrum (Sobi) as Global Regulatory Lead for Aspaveli®/Empaveli™, in the field of rare diseases.
“I’m humbled that I’ve been promoted to Chief Regulatory Officer at Azafaros,” commented Dr Arnold-Tugulu.
“The trust and opportunity extended to me by our board and leadership team is deeply appreciated. This promotion reflects the growing importance of the regulatory affairs function in building a company’s strategy. In the challenging environment of biotech start-ups, and even more in the rare disease field, an early dialogue with regulators is important when defining the clinical development plan of promising assets.
Stefano Portolano, CEO at Azafaros, said: “Anke has been an invaluable member of our team since joining us, and her dedication and leadership in navigating complex regulatory procedures have been exemplary. Her strategic insights and attention to detail have been instrumental in ensuring compliance and driving the success of our regulatory initiatives.
“As Chief Regulatory Officer, Anke will continue to play a pivotal role in shaping regulatory strategies at Azafaros and ensuring that we maintain the highest standards of compliance in all aspects of our operations.”
About nizubaglustat
Nizubaglustat is a small molecule, orally available and brain penetrant 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 (NPC).
Nizubaglustat has received the following designations and support:
United States Food and Drug Administration (FDA)
Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC.
Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC.
Fast Track Designation and IND clearance for GM1 and GM2 gangliosidoses.
European Medicines Agency (EMA)
Orphan Medicinal Product Designation (OMPD) for the treatment of GM2 gangliosidosis.
UK Medicines and Healthcare Products Regulatory Agency (MHRA)
Innovation Passport for the treatment of GM1 and GM2 gangliosidoses.
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 (NPC)
Niemann-Pick disease type C (NPC) 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 can happen throughout the lifespan of an affected individual, from prenatal life through adulthood.
About Azafaros
Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms, compound discoveries made by scientists at Leiden University and Amsterdam UMC, and led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. By applying its knowledge, 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, Asahi Kasei Pharma Ventures, and Schroders Capital.
For further information:
Azafaros B.V.
Email: info@azafaros.com