IntraBio’s lead drug series, IB1000s, are a set of orally administered, modified amino acids (N-Acetyl-Leucine) characterized by a well-established safety and tolerability profile.  IB1000s have demonstrated statistically significant efficacy for a broad spectrum of lysosomal storage disorders (LSD) and rare and common neurodegenerative and ageing disorders.

Given the extremely high, unmet medical need, IB1001 is initially being developed for orphan indications where there are currently no FDA-approved therapies: Niemann-Pick Disease Type C (NPC), GM1 & GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and inherited Cerebellar Ataxias (CA). These multinational clinical trials are active and ongoing.

Mechanism of Action

IB1000s have a unique transport mechanism which delivers the drug to target tissues (including neuronal) and results in the normalization of lysosomal and mitochondrial function. Knock-on effects include a reduction in neuroinflammation and the normalization of neuronal membrane potential and intracellular ion regulation via calcium channels.

Multiple publications have explored the mechanism of action of IB1000 in different model systems, listed in the Publication section of our website.

Clinical Development

Most recently, results from IB1001-201 (NPC) & IB1001-202 (GM2) clinical trials have demonstrated that IB1001  demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC & GM2 A multinational clinical trial with IB1001 for Ataxia-Telangiectasia is ongoing.

To date, IntraBio, with its collaborators, has evaluated the effect of IB1000s in compassionate-use studies in over 100 patients. These studies form the scientific basis for IB1000s to be further investigated for the treatment of 20 indications, including Traumatic Brain Injury (TBI) Lewy Body Dementia (LBD), Restless Legs Syndrome (RLS),  and Amyotrophic Lateral Sclerosis (ALS), all of which of have high-unmet medical needs.

Regulatory History

IntraBio has been granted tweleve Orphan Drug Designations (US Food and Drug Administration)/ Orphan Medicinal Drug Designations (European Commission) for IB1000s. for the treatment of NPC, GM1 Gangliosidosis, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), Spinocerebellar Ataxias (of which there are over 40 known subtypes) as well as Ataxia-Telangiectasia.

IntraBio has been granted three Rare Pediatric Disease Designations for IB1000s by the FDA for the treatment of NPC, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and Ataxia-Telangiectasia. These Rare Pediatric Disease Designation makes IB1000s eligible for, and expedites the request of, a Rare Pediatric Disease Priority Review Voucher (PRV) granted at the time of marketing approval.

IntraBio has also been granted Fast Track Designation for IB1001 by the FDA for NPC and GM2 Gangliosidosis.

Publications - IB1000 Series