A small molecule with the potential to become the first disease-modifying therapy for GM1 and GM2 gangliosidoses and Niemann-Pick type C disease
In recent years, researchers have discovered several enzymes that may serve as potential targets for the treatment of neurodegenerative lysosomal storage disorders.
Our lead candidate, nizubaglustat, was discovered by a group of scientists in the Netherlands (read more on Our Story and Mission) and belongs to the drug class of small molecules. With their ability to pass through cell membranes and the blood-brain barrier, these small molecules offer the opportunity to reach intracellular targets, including in the brain and spinal cord (central nervous system).
Nizubaglustat is designed to reach the central nervous system and to change the metabolism of certain fats or lipids. Through this mechanism, nizubaglustat has the potential to reduce the harmful accumulation of “waste” lipids and to reduce the impact of the impaired lysosome on cell function. Our initial goal with nizubaglustat is to develop a disease-modifying therapy for GM1 and GM2 gangliosidoses and Niemann-Pick type C disease in order to improve the lives of these patients, and the compound is currently being tested in Phase 3 studies in these indications.
Nizubaglustat can be administered in tablet form and therefore offers the possibility of convenient administration and treatment at home, which would significantly preserve the life quality of patients and their families. Nizubaglustat has the potential to provide the first disease-modifying therapy that can be effective regardless of whether the patient is afflicted by GM1/ GM2 gangliosidoses or NPC. The compound has been tested in a number of clinical trials to date.
An essential stage of all drug development is human trials
As a clinical stage biotech company, we are in the process of researching nizubaglustat. We have completed both a Phase I and a Phase II study, while running a Natural History Study alongside.