Amicus Therapeutics Announces Positive Initial Clinical Data for CLN3 Batten Disease Gene Therapy at the 17th Annual WORLDSymposium™ 2021
February 08, 2021 at 14:30 PM EST
Initial Data Suggest Early Signs of Disease Stabilization in Children with Fatal Neurologic Disease
Plan to Submit IND for Next Clinical Study in 2H2021
PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced positive initial results from its first in-human study of its CLN3 Batten disease gene therapy program, AT-GTX-502. The results are featured in a virtual poster presentation at the 17th Annual WORLDSymposium™ 2021, being held February 8-12, 2021. The poster is also available in the Events and Presentations section of the Amicus Therapeutics corporate website.
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 administration of AT-GTX-502 gene therapy for classic juvenile neuronal ceroid lipofuscinosis (JNCL), also known as CLN3 Batten disease. With no approved treatments, CLN3 Batten disease is a fatal neurologic disease that leads to blindness, motor impairment, learning difficulties, epilepsy and, ultimately, premature death.
Primary outcome measures are determined using the physical impairment subscale of the Unified Batten Disease Rating Scale (UBDRS), a clinical rating instrument developed specifically to assess disease progression in children with verified JNCL and includes evaluations of motor, behavioral, seizure and functional capabilities. UBDRS separately scores measures of vision, motor, speech, tone and abnormal movement over time. Higher scores indicate greater physical impairment.
Clinical Data Highlights:
Jeff Castelli, Ph.D., Chief Development Officer of Amicus Therapeutics, stated, “We are pleased to share this first set of clinical data for our intrathecal AAV gene therapy for CLN3 Batten disease and the second clinical program in our Batten portfolio. Preliminary results from this analysis suggest early signs of disease stabilization and has the potential to slow the neurological disease progression in children with CLN3 Batten disease. We are encouraged by the data and hope to make a meaningful impact for individuals living with CLN3 Batten disease, an ultra-rare, devastating neurodegenerative disease with no approved treatments.”
Emily de los Reyes, M.D., Ph.D., Principal Investigator at Nationwide Children’s and Professor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine is leading the CLN3 clinical trial at AWRI.
Regulatory interactions for AT-GTX-502 are ongoing and the Company expects to provide feedback on the clinical path forward later this year.
Amicus has exclusive rights under a license to the CLN3 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. Affected children may also experience vision loss, personality changes, behavioral problems, learning impairment and seizures. Children living with Batten disease typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden and die prematurely.
About Amicus Therapeutics