Lead Program

IB1001 is an orally administered, modified amino acid (N-Acetyl-L-Leucine). It’s safety and tolerability profile has been studied extensively in clinical trials.
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), GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and Ataxia-Telangiectasia. This development is based on existing pre-clinical and clinical data demonstrating safety and efficacy in these three disorders. 

Mechanism of Action
IB1001 is a modified amino acid (N-acetyl-L-leucine) that targets hallmark patterns of neurological dysfunction and degeneration. IB1001 uses monocarboxylate transporters to efficiently cross the blood-brain barrier and reach the central nervous system, ensuring delivery to all tissues.
Inside cells, IB1001 enters enzyme-controlled pathways that correct metabolic dysfunction and has been shown to normalize mitochondrial function (improving ATP production/reducing oxidative stress) and lysosomal function (resulting in a reduction of substrate accumulation). It enhances cerebral glucose metabolism in the cerebellum, leading to improved cerebellar activity.
IB1001 reinvigorates cellular health, protects against peripheral cell dysfunction, reduces cell death, and prevents chronic neurodegeneration.9 IB1001 targets key neurological dysfunctions, including mitochondrial and lysosomal dysfunction, cellular signaling defects, and neuroinflammation.

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 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. [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.

Further details on IntraBio’s clinical trials with IB1001 are available here.

Regulatory History

IntraBio has been granted fifteen 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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