Azafaros Secures €132M in Oversubscribed Series B Financing to Advance Phase 3 Clinical programs of innovative therapies in lysosomal storage disorders
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Leiden, Netherlands
Financing round led by Jeito Capital, co-led by Forbion Growth with participation from Seroba, Pictet Group and other existing investors
Proceeds to fund two Phase 3 pivotal programs with nizubaglustat, lead asset in Niemann-Pick disease Type C (NPC) disease and GM1/GM2 gangliosidoses as well as expanding the Azafaros pipeline to other indications
Nizubaglustat has been awarded Orphan Drug Designation in both the US and Europe as well as Fast-track status in the US. The company expects to initiate both Phase 3 studies later this year
Azafaros, a clinical-stage company focused on developing disease-modifying therapeutics to offer new treatment options to patients with rare lysosomal storage disorders, announces the completion of an oversubscribed €132M Series B financing led by Jeito Capital, co-led by Forbion Growth and additional participation from Seroba, Pictet Group and existing investors Forbion Ventures, Schroders Capital and BioGeneration Ventures (BGV) .
This financing enables Azafaros to accelerate the development of its lead product nizubaglustat, scheduled to enter Phase 3 studies for Niemann-Pick Type C (NPC) disease and GM1/GM2 gangliosidoses later this year as well as expanding Azafaros pipeline to other indications.
Rachel Mears, Partner at Jeito Capital; Julien Elric, Principal at Jeito Capital; and Audrey Cacaly, Principal at Forbion Growth will join Azafaros’s Board of Directors as Board members.
Founded in 2018 by BGV, leveraging science from Leiden University and Amsterdam UMC, Azafaros is led by a team of highly experienced industry experts in rare disease drug development and commercialization. The company is developing a first-in-class dual-acting drug candidate to offer new treatment options to patients with lysosomal storage disorders, a group of severe rare genetic diseases that often cause progressive neurodegeneration and, in many cases, fatal outcomes.
This successful Series B round marks a significant milestone for Azafaros, allowing us to accelerate the development of nizubaglustat and leverage our scientific understanding and competencies to bring additional candidates into development. The fact that we have been able to attract leading life sciences investors to join our existing, strong group of specialist investors is a testament to the impressive accomplishments of the team and the large unmet medical need that currently exists for patients with these hugely debilitating neurological diseases. We look forward to bringing nizubaglustat to patients.
Stefano Portolano, CEO, Azafaros
Azafaros has been impressive in its execution with nizubaglustat poised to begin Phase 3 clinical development and the potential to significantly improve the lives of NPC and GM1/GM2 patients. We are excited to support and accelerate the Azafaros team in this important next step in the Company’s clinical development journey. Leading this round further demonstrates Jeito’s commitment to making a meaningful difference in patients’ lives by pursuing much needed benefits for those suffering from rare diseases .
Rachel Mears, Partner, Jeito Capital
Azafaros exemplifies the kind of science-driven, mission-focused company we seek to back.
Nizubaglustat has the potential to fundamentally change the treatment landscape for rare
genetic diseases, and we are proud to support their journey as they move closer to delivering
real hope and this much needed treatment option to patients and families.
Audrey Cacaly, Principal, Forbion Growth
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 Europea 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 th central nervous system (CNS), resulting 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 type C (NPC) disease
Niemann-Pick type C disease 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 Jeito Capital, Forbion Growth, Seroba, Pictet Group and a syndicate of leading Dutch and Swiss existing investors including Forbion Ventures, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.
For further information: Azafaros B.V. Email: info@azafaros.com www.azafaros.com
