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IntraBio Reports Statistically Significant and Clinically Meaningful Improvements in the Use of IB1001 For Treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease)

~ Multinational clinical trial is the first successful clinical trial for GM2 Gangliosidosis; favourable safety and efficacy data consistent with previously announced IB1001 clinical trial results for NPC ~

OXFORD, UK. August 19, 2021 -- Life Science Newswire -- IntraBio Inc 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 a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both the primary and secondary endpoints for pediatric 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, centralized raters (professors of neurology with expertise in movement and neurological disorders).

The trial 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.”

Professor 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.”

In a joint statement, Rick Karl, President of the Cure Tay-Sachs Foundation and Dan Lewi, Chief Executive Officer of the Cure Action for Tay-Sachs Foundation, commented: “This treatment is a major breakthrough for the GM2 Gangliosidosis community that includes Tay-Sachs and Sandhoff. It is the first drug to offer hope to the patients and families affected by these devastating diseases. They are progressive, life-threatening conditions with no approved medicinal treatments. There is an urgent need for this effective treatment to be approved and made available for patients in our community before the window of therapeutic opportunity is lost.”

The positive results of this IB1001-202 study are reinforced by the efficacy and safety profile of IB1001 already demonstrated in IntraBio’s successful IB1001-201 study for Niemann-Pick disease Type C (NPC). As is the case in the IB1001-202 clinical trial for GM2 Gangliosidosis, IB1001-201 was the first clinical trial to demonstrate statistical significance and a clinically meaningful effect in patients with NPC. These results provide further momentum for the broad clinical development program planned for IB1001 which will address high unmet medical needs for treatment of both rare and common neurological disorders. 

For the full media release with all supplementary information about IB1001 and the IB1001-202 Trial, please visit

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Last Updated: 24-Aug-2021