Amicus Therapeutics Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 49th Annual Meeting of the Child Neurology Society
CRANBURY, N.J., Oct. 12, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive interim results from its CLN6 Batten disease gene therapy program, AT-GTX-501. The results are featured in a virtual poster presentation at the Joint 16th International Child Neurology Congress and 49th Annual Child Neurology Society Meeting being held October 12-23, 2020. The presentation is also available in the Events and Presentations section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/events-and-presentations.
The Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital is conducting the ongoing Phase 1/2 clinical study of a single one-time intrathecal administration of AT-GTX-501 gene therapy for variant late-infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease, also known as CLN6 Batten disease. With no approved treatments, CLN6 Batten disease is a fatal neurologic disease that rapidly robs children of their ability to walk, speak, think, and see.
Clinical Data Highlights:
Jeff Castelli, Ph.D., Chief Development Officer of Amicus Therapeutics, stated, “We are pleased to share these positive interim clinical data for our intrathecal AAV gene therapy with the CLN6 community. The data continues to suggest that our gene therapy has the potential to be a treatment option for children living with CLN6 Batten disease, an ultra-rare, debilitating condition that leads to progressive declines in cognitive and motor function, and often results in death early in life.”
Emily de los Reyes, M.D., Ph.D., Principal Investigator of the CLN6 clinical trial at AWRI at Nationwide Chldren’s and Professor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine, stated, “I remain pleased with the progress of this trial as well as our collection of natural history data to further inform the results for the AAV-CLN6 gene therapy. The interim results show that this investigational gene therapy has the potential to slow the neurological disease progression in children with CLN6 Batten disease.”
Regulatory interactions for AT-GTX-501 are ongoing and the Company expects to provide feedback on the path forward in 2021.
Amicus has exclusive rights to the CLN6 gene therapy program developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital.
About Batten Disease
Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden and die prematurely.
About Amicus Therapeutics