Netherlands-based biotechnology company Azafaros has completed patient recruitmentfor its Phase III study assessing nizubaglustat for GM1/GM2 gangliosidoses.
A part of the company’s NAVIGATE programme, the trial aims to assess the effects of nizubaglustat, an orally administered small molecule, on neurological manifestations and disease progression in these rare conditions.
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Top line results from this trial are expected in early 2028.
The NAVIGATE programme consists of two separate randomised, placebo-controlled, multi-centre Phase III trials.
The GM1/GM2 gangliosidoses study enrolled at least 75 patients across 25 clinical sites in 13 countries, targeting individuals with late-infantile and juvenile-onset forms of the diseases.
Over an 18-month treatment period, patients received either nizubaglustat or placebo. All participants remain eligible to enter an open-label extension period with continued nizubaglustat treatment.
Recruitment is still ongoing in a parallel NAVIGATE Phase III study evaluating nizubaglustat in Niemann-Pick type C disease (NPC), where 72 patients are being enrolled.
Both trials are taking place at centres in India, Latin America, North America, Pakistan and selected European countries.
Azafaros CEO Stefano Portolano said: “Completing enrolment in our Phase III GM1/GM2 study is a significant step toward potentially bringing nizubaglustat to patients and families living with these devastating neurodegenerative diseases. We are deeply grateful to the patients, caregivers, investigators and advocacy groups who have made this milestone possible.
“As we advance the GM1/GM2 study toward data readout and regulatory submission, we continue to enrol in our Phase III NPC study and remain focused on our goal of addressing the significant unmet medical needs faced by these rare disease communities.”
Nizubaglustat is being investigated for its dual mode of action targeting neurological manifestations in lysosomal storage disorders.
It has obtained several regulatory designations in the US and Europe, including rare paediatric disease, orphan drug and fast track designations from the US Food and Drug Administration (FDA).
The therapy has also received orphan medicinal product designation from the European Medicines Agency (EMA), and Innovation Passport from the UK Medicines and Healthcare Products Regulatory Agency (MHRA).
