Product Description
IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine that can restore neuronal function and protect against/delay disease progression in multiple neurological circuits of the brain. The mechanism of N-acetyl-L-leucine is multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death. (Sourced from: https://intrabio.com/2021/08/19/intrabio-reports-statistically-significant-and-clinically-meaningful-improvements-in-the-use-of-ib1001-for-treatment-of-gm2-gangliosidosis/)
Mechanisms of Action: FIX Analogue
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: IntraBio
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Brazil, Czech Republic, Georgia, Germany, Moldova, Netherlands, Slovakia, South Africa, Spain, Switzerland, Turkey, Ukraine, United Kingdom, United States
Active Clinical Trial Count:
Highest Development Phases
Phase 3: Aphasia, Primary Progressive|Ataxia Telangiectasia|Cerebellar Ataxia|Frontotemporal Dementia|Hemophilia A|Hemophilia B|Niemann-Pick Disease, Type A|Niemann-Pick Disease, Type C|Niemann-Pick Diseases|Pick Disease of the Brain|Telangiectasis
Phase 2: Gangliosidoses|Gangliosidosis, GM2|Sandhoff Disease|Tay-Sachs Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
---|---|---|---|---|---|
IB1001-303 | P3 |
Not yet recruiting |
Cerebellar Ataxia|Telangiectasis|Ataxia Telangiectasia |
2027-12-31 |
|
IB1001-301 | P3 |
Unknown Status |
Niemann-Pick Disease, Type C|Niemann-Pick Diseases |
2027-12-31 |
|
2021-005356-10 | P3 |
Active, not recruiting |
Niemann-Pick Disease, Type C|Niemann-Pick Diseases |
2025-01-25 |
|
IB1001-203 | P2 |
Recruiting |
Ataxia Telangiectasia|Cerebellar Ataxia|Telangiectasis |
2024-12-01 |
32% |