IntraBio Inc. is pleased to announce that the Extension Phase for the IB1001-202 Clinical Trial has been accepted in the United States, as well as in European countries in which the trial is being conducted, including Germany, Spain, and the UK.

IB1001-202 is a multinational, clinical trial that investigates IB1001 (N-acetyl-L-leucine) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff). The acceptance of the Extension Phases provides US patients enrolled in the current trial the opportunity to continue treatment with IB1001 if the principal investigator determines it is in the patient’s best interest.

The Extension Phase aims to provide further evidence of the symptomatic benefit of IB1001 and demonstrate the disease-modifying, neuroprotective effect of treatment. The potential benefit of long- term treatment has previously been observed in compassionate use studies and is supported by in vitro and in vivo data.

GM2 Gangliosidoses is a rare, debilitating, inherited lysosomal storage disorder that predominately affects pediatric patients. The disease begins in early childhood and is chronic and progressive in nature and severely impacts quality of life. The average age of death for GM2 Gangliosidosis patients is approximately 6.6 years. There are currently no approved treatments for GM2 Gangliosidosis.

In addition to Clinical Study IB1001-202, IntraBio is running parallel multinational clinical trials involving IB1001: IB1001-201 for the treatment of Niemann-Pick disease type C (NPC), and IB1001-203 for the treatment of Ataxia-Telangiectasia (A-T). To investigate both the symptomatic and neuroprotective, disease-modifying effects of treatment, IB1001 is assessed during two treatment sequences: a 6-week parent study, and a one-year extension phase. The parent studies of IB1001-201 and IB1001-202 are expected to be complete Q2 2020. Extension Studies are being conducted for all trials.