World-Class Team


Company Highlights

IntraBio is a biopharmaceutical company with a late-stage drug pipeline that includes novel treatments for genetic and neurodegenerative diseases.

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, Managment 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 and common neurological disorders, lysosomal storage disorders (LSDs), and inflammatory diseases.

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 three orphan indications where there are currently no FDA approved therapies: Niemann-Pick Disease Type C (NPC), GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and inherited Cerebellar Ataxias (CA).

Clinical Programs

IntraBio is currently prioritizing the development of its late-stage lead drug candidate (IB1001)  which has the potential for near-term approval in multiple indications.

Broad clinical applicability and efficacy

  • Three pivotal trials are ongoing with lead asset across 13 multinational clinical trial sites in the United States, United Kingdom, and Europe
  • Initially being developed for three orphan indications with currently no FDA-approved therapies: Niemann-Pick Disease Type C, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and inherited Cerebellar Ataxias

Novel Mechanism of Action with Broad Therapeutic Potential

  • Normalization of neuronal membrane potential and intracellular ion regulation via calcium channels
  • Symptomatic and neuroprotective properties and disease-modifying potential
  • Unique ability to address underlying neurological symptoms across a spectrum of lysosomal storage disorders and neurodegenerative disorders

Significant and Well-Established Proof-of-Concept and Safety Data

  • Established safety profile with minimal side effects and good tolerability profile
  • Demonstrated statistically significant improvement in key clinically-validated neurological scores in 19 different indications
  • Long-term efficacy with improvements in motor and cognitive function

Accelerated Clinical Development with Clear Regulatory Pathway

  • Consensus with regulators in US and Europe on protocols for multinational clinical trial
  • Enrollment aimed for late 2018 with potential approval by 2019

Robust Intellectual Property and Orphan Market Exclusivity

  • Broad, multi-faceted intellectual portfolio protecting IB1000s until 2037
  • FDA and EMA Orphan drug designation covering Niemann-Pick type C, Tay-Sachs disease, and Spinocerebellar Ataxias (currently over 40 known subtypes), and Ataxia-Telangiectasia
  • Designated for pediatric priority voucher for Niemann-Pick type C and Tay-Sachs disease
  • 10-year NCE exclusivity in the EU and 5-year NCE exclusivity in the US