Our Therapeutic Approach
A small molecule with the potential to become the first disease-modifying therapy for GM1 and GM2 gangliosidosis and Niemann-Pick Type C
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 AZ-3102 is to develop a disease-modifying therapy for GM1 and GM2 (gangliosidoses) and Niemann-Pick disease type C to improve the lives of these patients.
Nizubaglustat can be swallowed 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 that can be effective regardless of whether the patient is afflicted by GM1/ GM2 gangliosidoses, or NPC.
An essential stage of all drug development is human trials
Our Phase I first-in-human study with AZ-3102
Our Phase I Study in healthy volunteers assessed the safety and tolerability of AZ-3102, with specific measurements on drug levels and biomarkers in plasma and cerebrospinal fluid (CSF). The study design served the future development of AZ-3102 in neurological lysosomal storage disorders, including GM1 and GM2 gangliosidosis, and Niemann-Pick Type C.
Our Natural History Study:
a prospective longitudinal study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 gangliosidoses, PRONTO.
To gain more insight in the natural development of the disease, our clinical team designed PRONTO (PROspective Neurological Disease TrajectOry Study), which is a prospective Natural History study.
By enrolling children with late infantile and juvenile onset of GM1 and GM2 gangliosidosis, we aim to understand how the disease progresses, with a focus on how it affects neurological signs and symptoms, and to identify if some patients have different traits that could explain and predict how their disease will progress. In this way we build an external control dataset and develop a model of disease progression. This study will support the future development of a clinical trial investigating the potential of our lead therapeutic candidate, AZ-3102, in this patient group.
PRONTO is designed as a multinational study in up to 6 countries, with at least 75 participants between the age of 2 to 20 years with neurological disease onset after their 1st birthday. Enrolment was initiated in 2021, with follow-ups continuing until 2025.
By joining this study, you can contribute to the gathering of important data that will be highly valuable for the development of future treatments. If you want to learn more about PRONTO and how to enrol, please contact your physician or click here for the published clinical trial information: clinicaltrials.gov
For any questions and specific information on our PRONTO study, please send an email to firstname.lastname@example.org.
Our Phase II Study RAINBOW
RAndomised, double-blINd, placeBo-controlled, multicenter, 12-week study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of Oral AZ-3102 in patients With GM2 gangliosidosis or Niemann-Pick Type C (NPC) diseases.
This study aims to assess the correct doses of AZ-3102 to treat GM2 and NPC diseases by evaluating the clearance of AZ-3102 from the body and the effect of two different doses in a small number of patients with those diseases.
RAINBOW is designed as a short, multinational study in up to two countries, with 12 participants (six per disease) between the ages of 12 and 20 years, with late-infantile or juvenile onsets. Enrolment will be initiated in early 2023 with a duration study time of three months. We are currently selecting sites in Brazil and in the USA. Click here for our Q & A on the RAINBOW study for more information. For specific questions or information, please send an email to email@example.com.