Lead Program

IB1001 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. 

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).

Mechanism of Action
IB1000s are believed to have a unique transport mechanism which delivers the drug to target tissues (including across the blood-brain barrier to the central nervous system) 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
In June 2023, IntraBio announced the positive results of its Phase III pivotal trial IB1001-301: Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study.

IB1001-301 met the primary endpoint and secondary endpoints showing high statistical significance and demonstrating an improvement in neurological signs and symptoms, functioning, and quality of life in pediatric and adult patients on IB1001 vs placebo. IB1001 was safe and well-tolerated with a favorable safety profile consistent with previous clinical and pre-clinical studies [Bremova-Ertl et al. 2024, NEJM].

The complete announcement is available here.

In addition to IntraBio’s Phase III pivotal trial, IB1001-201 (NPC) & IB1001-202 (GM2) clinical trials showed that IB1001 demonstrated a statistically significant 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.

Regulatory History
IntraBio has been granted fourteen Orphan Drug Designations (US Food and Drug Administration)/ Orphan Medicinal Drug Designations (European Commission) for the IB1000 Series for the treatment of NPC, GM1 Gangliosidosis, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), Spinocerebellar Ataxias (of which there are over 40 known subtypes), Ataxia-Telangiectasia, Ataxia Oculomotor Apraxia type 4 (AOA4), as well as Multiple Systems Atrophy (MSA).

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.

In March 2024, U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). The application was granted Priority Review and was given a Prescription Drug User Fee Act (PDUFA) target action date of September 24th, 2024.

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