- Dr. Abribat has a proven track record of developing innovative treatments for rare diseases, achieving three successful biotech/company exits
- He is a seasoned scientific and operational leader, having held senior roles in drug development and business development
- He joins the Azafaros Board as the company advances two Phase 3 pivotal studies with its lead asset nizubaglustat in Niemann-Pick Type C disease (NPC) and GM1/GM2 gangliosidoses
Azafaros, a company building a portfolio to become a leader in lysosomal storage disorders with the goal of addressing neurological symptoms, today announced the appointment of experienced biotech executive and entrepreneur, Thierry Abribat, to the company's Board of Directors.
Dr. Abribat is a seasoned biotech entrepreneur and executive with more than 25 years' experience in advancing innovative therapies, from early research through late-stage clinical development and commercialization. He was founder and CEO of global biopharmaceutical company, Amolyt Pharma, which advanced multiple programs for rare endocrine diseases through clinical and preclinical development before being acquired by AstraZeneca in 2024 for more than a billion dollars. Prior to that, he founded and led Alizé Pharma and Alizé Pharma 2, successfully building and exiting both rare disease companies.
"Thierry brings a combination of scientific depth, strategic vision, and a proven track record of value creation in biotech," said Carlo Incerti, Chairman of the Board of Directors at Azafaros. "Azafaros is enrolling two registrational Phase 3 studies with our lead product nizubaglustat. Thierry will bring valuable insight as we move into our next phase of the company, with a potential commercialization stage in sight following regulatory approval."
Dr. Abribat added: "I am delighted to join Azafaros at such an exciting stage of development. The company's science is compelling, and with my experience in company building and advancing innovative therapeutics for rare diseases, I look forward to contributing so that we can deliver on our mission/strategy to bring important new therapies to patients."
Dr. Abribat earned a doctorate in veterinary medicine and a PhD in neuroendocrinology from the National Polytechnic Institute of Toulouse and began his career as a scientist at Sanofi and at the University of Montreal.
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 Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC, Orphan Drug Designations (ODD) for GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC, as well as Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC from the US Food and Drug Administration (FDA). Additionally, nizubaglustat has been awarded Orphan Medicinal Product Designation (OMPD) for the treatment of GM1 and GM2 gangliosidoses by the European Medicines Agency (EMA) and Innovation Passport for the treatment of GM1 and GM2 gangliosidoses from the UK Medicines and Healthcare Products Regulatory Agency (MHRA).
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). This results in progressive and severe neurological impairment and premature 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 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 using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is 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 leading healthcare investors including Forbion, Jeito Capital, Seroba, Pictet Group, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.
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