IntraBio today announced positive data from its multinational clinical trial of IB1001 (N-acetyl-L-leucine) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease).
IB1001 demonstrated statistically significant and clinically meaningful improvements in symptoms, functioning, and quality of life in both the primary and secondary endpoints for paediatric and adult patients with GM2 Gangliosidosis.
The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), which was assessed by blinded, centralised raters (professors of neurology with expertise in movement and neurological disorders).
It also met secondary endpoints including the scale for the assessment and rating of ataxia (SARA), the modified disability rating scale (mDRS), the investigators’, caregivers’, and patients’ clinical global impression of change (CGI-C) assessment.
IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events.
“The results of this study are hugely important for the GM2 community,” said Dr Susanne Schneider, Principal Investigator and Professor of Neurology from Ludwig Maximilian University of Munich. “IB1001 is the first drug to demonstrate a statistically significant and a clinically meaningful effect for the treatment of GM2 Gangliosidosis. IB1001 has a very compelling safety profile, easy oral administration [sachet mixed with water], affirming its very favourable risk/benefit profile as a treatment for this devastating disease.”
Prof Antony Galione, FRS, FMedSci, Statutory Professor of Pharmacology, University of Oxford commented: “GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease) is a devastating disease that has never had any available treatment. We are very excited that IB1001 is the first drug that is effective for this disorder and will improve the lives of so many patients and their families. Given what is known about IB1001’s mechanism, and its multiple successful clinical trials, we will continue to investigate this drug for other rare genetic neurological diseases and for more common neurodegenerative diseases prevalent in society with large unmet medical needs.”
