IntraBio Inc. is pleased to announce significant progress in the treatment of rare and neurodegenerative diseases with IB1000, a well-tolerated, modified amino acid that is administered orally. Most notably, IB1000 is showing promising effects in Phase 2 observational clinical studies in patients with Niemann-Pick Type C (NPC) disease, Late-Onset Tay Sachs (LOTS) disease, and different types of Cerebellar Ataxia (CA). A multinational, randomized, controlled Phase 2/3 clinical trial of IB1000 in 108 CA patients was recently completed, with the final results expected in 2018.
IntraBio is now planning multi-national randomized, controlled pivotal clinical trials to evaluate IB1000 as a therapeutic intervention in patients with NPC, LOTS/TS, and specific CA diseases. Our goal is to commence enrollment in these pivotal trials in the EU starting enrollment by early 2018 to be followed by recruitment in North America. In these trials, the safety and efficacy of IB1000 will be determined for each indication using validated assessment scales. IntraBio will provide full details of the clinical trials, including inclusion criteria, as soon as they are available.