IntraBio is developing it’s lead compound IB1001 (N-acetyl-L-leucine) for the treatment of rare and common neurological disorders.

Information on active clinical programs is listed below.

Niemann-Pick disease type C (NPC)

IntraBio has conducted two multinational clinical trials with IB1001 for NPC (positive Phase IIb and Phase III studies).

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

Most recently, IntraBio announced positive topline results from the pivotal, Phase III IB1001-301 clinical trial (NCT05163288), showing IB1001 significantly improved symptoms (motor and cognitive) functioning, quality of life, and cognition in pediatric and adult patients with Niemann-Pick disease Type C (NPC).

IB1001-301 ClinicalTrials.gov (NCT05163288)

IB1001-201 – Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

 IB1001-201 ClinicalTrials.gov (NCT03759639)

The IB1001-201 (NPC) clinical trial demonstrated a statistically significant and clinically meaningful improvement in both the primary and topline secondary endpoints for the treatment of pediatric and adult patients with NPC.

Publication: Bremova et al. 2021

GM2 Gangliosidosis

IB1001-202 – Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

IB1001-202 ClinicalTrials.gov (NCT03759665)

The IB1001-202 (GM2) clinical trial demonstrated a statistically significant and clinically meaningful improvement in both the primary and topline secondary endpoints for the treatment of pediatric and adult patients with GM2.

Publication: Martakis et al. 2023

Ataxia Telangiectasia

IB1001-203 Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

IB1001-203 ClinicalTrials.gov (NCT03759678)

IB1000s Series