- First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses.
- Both studies are part of Azafaros’ mission to develop treatments for the unmet need of patients with rare lysosomal storage disorders
- The news follows the company’s recent successful €132M Series B financing, aimed at supporting the rapid development of nizubaglustat and the expansion of Azafaros’ pipeline to other indications
Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that the first patient has been dosed in the company’s pivotal, multicenter Phase 3 clinical program to evaluate the safety and efficacy of the company’s lead asset, nizubaglustat, in patients with Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses.
The initiation of the two Phase 3 studies (NCT07054515)represents a major milestone in Azafaros’ commitment to addressing the urgent unmet medical needs of children affected by these devastating neurodegenerative disorders.
The Phase 3 program consists of two studies targeting the late-infantile and juvenile-onset forms of NPC, and GM1/GM2 gangliosidoses. The studies aim to assess the potential of nizubaglustat to alter disease progression and improve functional outcomes in these patient populations.
Today’s news follows the recent, successful completion of an oversubscribed series B financing, raising €132M to support the acceleration of nizubaglustat and the expansion of the company’s pipeline to other indications.
“The dosing of the first patient in our Phase 3 program with nizubaglustat is a significant achievement for Azafaros and a huge step forward in our efforts to bring new, disease modifying treatments to patients with these seriously debilitating diseases,” said Stefano Portolano, Chief Executive Officer at Azafaros. “We are deeply grateful to the patients, families, clinicians, and advocacy groups who are partnering with us to advance this promising therapy.”
