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. In compassionate-use clinical trials, IB1000s have demonstrated statistically significant efficacy for a broad spectrum of lysosomal storage disorders (LSD) and rare and common neurodegenerative diseases.
Based on the pre-clinical studies with the compounds N-Acetyl-DL-Leucine, N-Acetyl-D-Leucine, and N-Acetyl-L-Leucine, it has been identified that:
Based on these findings, IntraBio is prioritizing the development of N-Acetyl-L-Leucine (IB1001).
Given the extremely high, unmet medical need, IB1001 is initially being developed for three orphan indications where there are currently no FDA approved therapies: Niemann-Pick Disease Type C (NPC), GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and inherited Cerebellar Ataxias (CA). These multinational clinical trials are active and ongoing.
Mechanism of Action
IB1000s are believed to have an effect on the normalization of neuronal membrane potential and intracellular ion regulation via calcium channels and have been observed to have symptomatic and neuroprotective properties and disease-modifying potential.
Several publications have explored the mechanism of action of IB1000 in different model systems, listed in the Publication section of our website, along with the published observational clinical study outcomes.
Most recently, results from IB1001-201 clinical trial have demonstrated that IB1001 (N-acetyl-L-leucine) 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. Multinational clinical trials with IB1001 for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) and Ataxia-Telangiectasia are 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 18 indications, including Traumatic Brain Injury (TBI) Lewy Body Dementia (LBD), Restless Leg Syndrome (RLS), and Amyotrophic Lateral Sclerosis (ALS), all of which of have high-unmet medical needs.
In compassionate use studies, IB1000s have demonstrated statistically significant improvement in key, clinically-validated neurological scales in patients with NPC, Tay-Sachs disease, and inherited Cerebellar Ataxias (as well as additional LSDs and neurodegenerative diseases). These findings have been significantly supported in additional in vitro and in vivo studies, and are described in multiple published peer-review papers.
IntraBio has been granted eight Orphan Drug Designations (US Food and Drug Administration)/ Orphan Medicinal Drug Designations (European Commission) for IB1000s. for the treatment of NPC, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), Spinocerebellar Ataxias (of which there are over 40 known subtypes) as well as Ataxia-Telangiectasia.
IntraBio has also been granted two Rare Pediatric Disease Designation for IB1000s by the FDA for the treatment of NPC and GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). 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 GM2Gangliosidosis.Publications - IB1000 Series