IB1001 Pivotal NPC Results

World-Class Team


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 key secondary endpoints showing high statistical significance. The primary endpoint of the trial evaluated the impact of IB1001 on the Scale for the Assessment and Rating of Ataxia (SARA) compared to placebo after 12 weeks. Treatment with IB1001 demonstrated a statistically significant and clinically meaningful 1.37-point reduction of the SARA score compared to placebo (-1.97 on IB1001 vs. -0.60 on placebo; p<0.001). The trial also met its secondary endpoints, the modified Scale for the Assessment and Rating of Ataxia (mSARA) (-1.66 on IB1001 vs. on -0.67 placebo; p<0.001) and the Investigator’s Clinical Global Impression of Change (CGI-C) (-0.7 on IB1001 vs. +0.1 on placebo; p<0.001).

IB1001 showed a clinically meaningful improvement in symptoms, functioning, quality of life, and cognition in both pediatric and adult patients with NPC. Subgroup analyses showed that the improvement in neurological status was observed across all demographics of patients respective of age (which included a broad range, aged 5 to 67 years), age of disease onset, disease severity, etc.

IB1001 was safe and well-tolerated with a favourable safety profile consistent with previous clinical and pre-clinical studies.

The complete announcement is available here.


IntraBio is a biopharmaceutical company with a late-stage drug pipeline that includes novel treatments for rare and common neurological disorders, as well as ageing and healthspan.

Our clinical programs leverage the expertise of our scientific founders from the University of Oxford and University of Munich, the preeminent experts and pioneers in discovering and developing small molecule drugs that modulate lysosomal function and intracellular calcium signaling.

Our management team and business consultants have vast commercial and regulatory experience in drug product development, including all stages from small molecule manufacturing, clinical studies, and regulatory approvals in the USA and Europe.

With their successful track record of drug development and commercialization, IntraBio’s team translates research in the fields of lysosomal biology, autophagy, and neurology into orphan drugs and treatments that will significantly improve the lives of patients, their caregivers, and families.

Our Scientists, Management Team, Advisory Boards

IntraBio’s platform technology results from decades of research and over $350 million of investments from organizations worldwide. Our pipeline has broad applicability to be developed as novel treatments for rare (“orphan”) and common neurological disorders, as well as ageing/extending healthspan.

Our lead compound (IB1001) is characterized by a well-established safety and tolerability profile and has a unique potential to address the underlying neurological symptoms and manifestations across a spectrum of genetic and common disorders.

IB1001 is initially being developed for multiple 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).

Clinical Programs