IntraBio research update – January 2018

We are pleased to share the below from Intrabio and their research into a potential treatment for Tay-Sachs and Sandhoff disease.

Below, I am pleased to share highlights of the significant progress we have made over the last quarter of 2017, which have prepared IntraBio to conduct multi-national (EU and US), placebo-controlled, clinical trials in early 2018 that will evaluate its leading drug candidate in the treatment of Niemann-Pick Type C, Tay-Sachs disease, and Cerebellar Ataxia subtypes.

Regulatory Progress: Multi-National Clinical Trials

To prepare the best clinical-trial application, IntraBio has been seeking feedback on its trial designs from national regulators in the EU. Their responses have been extremely positive, suggesting only minor amendments to our trial protocols which we agree with. Additionally, IntraBio has requested a Pre-Investigation New Drug (PIND) meeting with the US (FDA) to discuss our proposed clinical trials. Following this meeting, we plan to submit an IND application and concurrently apply for Fast-Track designation to expedite the regulatory review and approval processes.

Orphan Medicinal Drug Designations


The European Commission recently granted Orphan Medicinal Drug Designation to IB1000 for the treatment of GM2 Gangliosides (Tay Sachs and Sandhoff Diseases). A public announcement by the European Medicines Agency (EMA) will be made shortly through the Community Register of Orphan Medicinal Products. This is the second Orphan Medicinal Drug Designation granted to IB1000 in the EU, in addition to its previous designation granted for the treatment of Niemann-Pick Diseases.


IntraBio has also applied for Orphan Drug Designation for IB1000 in the treatment of Niemann-Pick Diseases with the Federal Drug Administration (FDA) and will soon be filing an Orphan Drug application for GM2 Gangliosides. Due to the close collaboration between the FDA and EMA on matters relating to orphan designation, we hope to receive these approvals in early 2018.

Clinical Programs

IntraBio’s leading drug candidate IB1000 is being trialed in 14 additional indications and has been observed to have clinical benefits for each disorder. Notably, IB1000 has been observed to have significant efficacy in the treatment of rare lysosomal storage disorders as well as common neurodegenerative diseases. In 2018, IntraBio will continue to develop these clinical programs and identify new indications to be further investigated.

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