IntraBio Investigational New Drug Application Approved by the FDA for the Treatment of Tay-Sachs and Sandhoff Disease
IntraBio Inc., a late-stage biopharmaceutical company, today announced that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) Application for Clinical Trial IB1001-202 involving its lead compound (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).
The IND approval of IB1001-202 allows IntraBio to move forward with the trial at U.S. clinical sites. In addition to the U.S. centers, IntraBio intends to commence the study in European countries, including the United Kingdom, Germany, and Spain.
''The FDA's approval of our IND is a significant milestone for both patients and IntraBio,'' said Mallory Factor, Chairman of IntraBio Inc. ''Given the extremely debilitating nature of these diseases and the lack of any approved US drug for these conditions, we are committed to moving forward rapidly to bring our novel therapeutic treatment to patients in need.''
GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) is a rare, fatal condition that predominantly affects pediatric patients. GM2 Gangliosidosis is a neuro-visceral, autosomal recessive, lysosomal storage disorder that results in progressive neurodegeneration in the brain and spinal cord, leading to a myriad of debilitating symptoms and inevitable premature death. There are no drugs available for the treatment of GM2 Gangliosidosis.
In addition to Clinical Study IB1001-202, IntraBio has applied for two additional multinational clinical trials involving IB1001 for the treatment of Niemann-Pick disease Type C (NPC) and Ataxia-Telangiectasia (A-T). Enrollment in all three studies is expected to begin in Q2 2019.
IntraBio Inc. is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for common and rare neurodegenerative diseases. IntraBio's clinical programs leverage the expertise in lysosomal function and intracellular calcium signaling of its scientific founders and advisors from the University of Oxford, the University of Munich and other premier universities and institutions worldwide.
IntraBio's management team and consultants have vast commercial experience and a successful track record of drug development in the USA and Europe. IntraBio's team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases to significantly improve the lives of patients and their families.
IntraBio Inc. is a US corporation with its principal laboratories and offices in Oxford, United Kingdom.