Intrabio research update – November 2018
We are pleased to share the below from Intrabio and their research into a potential treatment for Tay-Sachs and Sandhoff disease
IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for rare (“orphan”) and common neurodegenerative diseases, announced that the European Commission has granted Orphan Medicinal Drug Designation for its lead compound series (IB1000s) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), a rare lysosomal storage disorder that is characterized by rapid, progressive neurodegeneration and premature death.
GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and is caused by mutations in the HEXA gene, which disrupts the activity of the enzyme beta-hexosaminidase A, preventing the enzyme from breaking down GM2 gangliosides. As a result, GM2 gangliosides accumulate to toxic levels, particularly in neurons in the brain and spinal cord, leading to cell death and resulting in the signs and symptoms of Tay-Sachs and Sandhoff disease. There is nothing medically available for the treatment of GM2 Gangliosidosis at this time.
“This is extremely exciting news for the Tay-Sachs and Sandhoff community,” said Dan Lewi, Chief Executive of the Cure & Action for Tay-Sachs Foundation. “Intrabio has put these rare diseases back on the map and the success of IB1000s gives a glimmer of hope that we are quickly moving towards a successful symptomatic treatment for these devastating conditions.”
This orphan designation provides a number of regulatory benefits to IB1000s, such as a waiver of the marketing application fee/single marketing application made to the European Commission, which, when approved, is valid in all EU member states (as well as Iceland, Norway, and Lichtenstein), as well as 10 year exclusivity in Europe from the date of marketing authorization.
IntraBio is currently in the process of applying for multi-national, multi-center clinical trials with its lead asset (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease), Niemann-Pick disease Type C (NPC) and inherited Cerebellar Ataxias (CA).
IntraBio, with its collaborators, has evaluated the effect of IB1000s in compassionate use studies in over 175 patients, forming the scientific basis for IB1000s to be further investigated for the treatment of 18 indications, including neurodegenerative diseases and lysosomal storage disorders. Future opportunities to further develop the IB1000s series in additional indications include Lewy Body Dementia, Restless Leg Syndrome, ALS, and Multiple Sclerosis, all of which of have high unmet medical needs