Novel GM2 gangliosidosis treatment improves symptoms, functioning, QOL
IntraBio Inc. announced positive clinical trial results for IB1001, a pipeline drug intended to treat GM2 gangliosidosis.
The multinational clinical trial met its primary endpoint, clinical impression of change in severity (CI-CS), as well as its secondary endpoints, which included patient clinical global impression of change (CGI-C) assessment, according to a press release from the company.
“IB1001 is the first drug to demonstrate a statistically significant and clinically meaningful effect for the treatment of GM2 gangliosidosis,” Susanne Schneider, DrMed, principal investigator and professor of neurology at Ludwig Maximilian University of Munich in Germany, said in the release. “IB1001 has a very compelling safety profile, easy oral administration [sachet mixed with water], affirming its very favorable risk:benefit profile as a treatment for this devastating disease.”
The trial took place over multiple 6-week periods followed by an extension phase of 1 year, according to the release. Patients with GM2 gangliosidosis aged 6 years and older across the U.S., the United Kingdom and the European Union received treatment with IB1001 to examine its neuroprotective, disease-modifying effects. According to the release, there were no drug-related serious adverse events, with IB1001 being well tolerated by patients.
“This treatment is a major breakthrough for the GM2 gangliosidosis community that includes Tay-Sachs and Sandhoff,” Rick Karl, president of the Cure Tay-Sachs Foundation, and Dan Lewi, CEO of the Cure & Action for Tay-Sachs Foundation, said in the release. “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,” they continued. “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.”
IntraBio is conducting a parallel trial for IB1001 for the treatment of ataxia-telangiectasia.