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IB1001 (N-Acetyl-L-Leucine) is a calcium channel modulator. It is being evaluated in the phase III clinical trial studies for the treatment of Type C Niemann-Pick Disease.
Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Rare Diseases and Disorders Product Name: IB1001
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 26, 2024
Details:
IB1001 (n-acetyl-l-leucine) is a novel, orally administered therapy with a unique mechanism of action offering neuroprotective and symptomatic benefits for rare and common neurological disorders. it's being developed for NPC, GM1 and GM2 gangliosides, and inherited cerebellar ataxias.
Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Rare Diseases and Disorders Product Name: IB1001
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 29, 2023
Details:
IB1001-301 investigates N-Acetyl-L-Leucine (IB1001) for both the symptomatic and long-term neuroprotective effect of treatment of Niemann-Pick disease Type C (NPC), with the primary endpoint based on the Scale for the Assessment and Rating of Ataxia.
Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Rare Diseases and Disorders Product Name: IB1001
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 08, 2022
Details:
IB1001-301 investigates IB100 (N-acetyl-L-leucine) for both the symptomatic and long-term neuroprotective effect of treatment of Niemann-Pick disease Type C (NPC), with the primary endpoint based on the Scale for the Assessment and Rating of Ataxia.
Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Rare Diseases and Disorders Product Name: IB1001
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 22, 2022
Details:
IB1001 demonstrated a statistically significant and clinically meaningful change in both the primary and secondary endpoints.
Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: IB1001
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 19, 2020
Details:
IB1001 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.
Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: IB1001
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 30, 2020
Details:
IB1001 (N-acetyl-L-leucine) is currently being investigated for the symptomatic, and disease-modifying, neuroprotective treatment of NPC in a multinational clinical trial (IB1001-201).
Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 25, 2020
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