IB1000 Series

IB1000 Series

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:

  • N-Acetyl-L-Leucine is the active ingredient of N-Acetyl-DL-Leucine responsible for the long-term neuroprotective and disease-modifying effects;
  • After chronic administration of N-Acetyl-DL-Leucine or D-enantiomer, the D-enantiomer may accumulate, with possible unwanted harmful effects on cell function (Churchill, 2019).

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.

Clinical Development

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.

Regulatory History

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.

The Investigational New Drug Applications (IND) and Clinical Trial Applications have been accepted for all studies in all countries where they will be conducted, including the United States, United Kingdom, Germany, Spain, and Slovakia

Publications - IB1000 Series