Azafaros Raises €132M in Series B to Advance Phase 3 LSD Therapies
Azafaros, a clinical-stage biotechnology company with a specialized focus on rare lysosomal storage disorders (LSDs), announced the successful completion of a €132 million ($142 million USD equivalent) Series B financing round. The round was significantly oversubscribed, reflecting strong investor confidence in the company’s innovative pipeline and promising lead drug candidate. The funding round was led by Jeito Capital, with Forbion Growth acting as co-lead. Additional participation came from new investors including Seroba and the Pictet Group, alongside continued support from existing backers Forbion Ventures, Schroders Capital, and BioGeneration Ventures (BGV).
The influx of capital will primarily be used to support the late-stage clinical development of nizubaglustat, Azafaros’s lead investigational therapy, which is poised to enter Phase 3 clinical trials later in 2025. The drug is being developed as a potential disease-modifying treatment for Niemann-Pick disease Type C (NPC) and GM1 and GM2 gangliosidoses, all of which are devastating and ultra-rare lysosomal storage disorders with limited therapeutic options.
A Leap Forward in Treating Rare Neurodegenerative Diseases
Niemann-Pick disease Type C and gangliosidoses are both progressive, inherited disorders that cause severe neurodegeneration, developmental delay, and, in many cases, premature death. Current treatments are either non-existent or only offer marginal symptomatic relief, leaving patients and families desperate for options that can alter the course of the disease.
Azafaros aims to change that narrative. Founded in 2018 by BioGeneration Ventures, the company leverages pioneering academic science developed at Leiden University and the Amsterdam University Medical Center (UMC). From its inception, Azafaros has positioned itself as a translational leader, bringing first-in-class small molecule therapies from early discovery through late-stage clinical development with the goal of reshaping the treatment landscape for underserved rare disease communities.
Its lead compound, nizubaglustat, is a dual-mechanism, orally available drug designed to cross the blood-brain barrier and modulate the pathways involved in glycosphingolipid metabolism. By addressing the underlying biochemical abnormalities central to lysosomal storage disorders, the therapy has the potential to deliver meaningful clinical outcomes, particularly for patients suffering from neurologically progressive conditions like NPC and gangliosidoses.
Accelerating Clinical Development and Pipeline Expansion
The completion of the Series B round provides Azafaros with the financial flexibility to execute an ambitious development strategy. In the near term, funds will be used to initiate Phase 3 pivotal trials for nizubaglustat in both Niemann-Pick Type C and the GM1/GM2 gangliosidoses indications. The company has already gathered encouraging safety and efficacy data in early and mid-stage studies, and this next phase is expected to yield critical evidence to support potential regulatory submissions in key global markets, including the United States and Europe.
Moreover, the funding will support pipeline expansion, allowing Azafaros to explore additional therapeutic applications for its platform beyond NPC and gangliosidoses. The company is already evaluating other lysosomal storage disorders and related neurodegenerative conditions as potential targets for future clinical programs.
“Completing this Series B round is a transformational moment for Azafaros,” said Stefano Portolano, Chief Executive Officer of Azafaros. “It empowers us to move forward rapidly with the development of nizubaglustat and to further expand our pipeline. The enthusiasm and confidence shown by both new and existing investors highlight the strength of our scientific platform, the dedication of our team, and the critical unmet need we are addressing for patients affected by rare and often fatal neurological disorders.”
Strategic Investors Bolster Governance and Expertise
As part of the financing round, Azafaros is also strengthening its corporate governance and strategic oversight. Three highly experienced investors will join the Azafaros Board of Directors:
- Rachel Mears, Partner at Jeito Capital
- Julien Elric, Senior Principal at Jeito Capital
- Audrey Cacaly, Principal at Forbion Growth
These appointments reflect the deep commitment of the new investor syndicate to actively support Azafaros as it navigates the complex clinical and regulatory pathways required to bring transformative therapies to market.
“Azafaros has demonstrated exceptional execution capabilities, and nizubaglustat is now well positioned to enter Phase 3 development,” said Rachel Mears, Partner at Jeito Capital. “We are thrilled to be leading this round and to work closely with a team so deeply committed to improving outcomes for patients with rare, life-limiting diseases. This investment is entirely aligned with Jeito’s mission to accelerate innovation in life sciences with a focus on patient benefit.”
Audrey Cacaly of Forbion Growth echoed these sentiments, underscoring the alignment between Azafaros’s mission and Forbion’s investment philosophy. “Azafaros embodies the kind of cutting-edge, purpose-driven company we aim to support. With a first-in-class therapeutic like nizubaglustat, the company has the potential to redefine standards of care in these diseases, offering hope where very little has existed.”
A Company Built on Scientific Excellence and Rare Disease Expertise
Azafaros’s scientific foundation is rooted in its collaborations with leading academic institutions and a leadership team with deep expertise in the development and commercialization of rare disease therapies. The company’s executive team and board bring decades of experience from global biopharmaceutical companies, ensuring that Azafaros is not only advancing a promising therapeutic but is also well-positioned to navigate regulatory, manufacturing, and commercial challenges.
Lysosomal storage disorders (LSDs) comprise more than 70 distinct diseases, many of which are associated with severe neurological symptoms and early mortality. Although individually rare, these disorders collectively affect tens of thousands of individuals worldwide, most of whom have little to no access to disease-modifying treatments. Azafaros’s approach seeks to address the root causes of these conditions rather than simply treating the symptoms, a distinction that sets the company apart from many others working in the rare disease space.
Hope for a Previously Untreatable Population
With the new funds in hand and Phase 3 studies on the horizon, Azafaros is entering a pivotal period in its growth trajectory. The company’s success could not only result in the first approved treatment for NPC and gangliosidoses that alters disease progression but could also validate its broader platform and pave the way for future treatments across a spectrum of rare neurodegenerative disorders.
“Our vision is to offer meaningful and lasting benefits to patients and families affected by rare diseases, where hope is often in short supply,” said Portolano. “We are energized by the support we’ve received from this exceptional syndicate of investors and excited about the path ahead. Our commitment to patients is at the core of everything we do, and this milestone brings us one step closer to fulfilling that mission.”
