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Amicus Therapeutics Announces 'Positive' Clinical Data for CLN6 Batten Disease Gene Therapy

Clinical Data Highlights: Interim safety data are available for 13 children with CLN6 Batten disease. Interim efficacy data are available for the first 12 children reaching the 12-month timepoint and for eight children

· 10/12/2020 07:01

Clinical Data Highlights:

Interim safety data are available for 13 children with CLN6 Batten disease. Interim efficacy data are available for the first 12 children reaching the 12-month timepoint and for eight children up to 24 months, post-administration of the AAV-CLN6 gene therapy.

  • Safety (n=13): Treatment with AT-GTX-501 was generally well tolerated. The majority of adverse events (AEs) were mild and unrelated to treatment. No pattern of adverse events related to AAV or CLN6 immunogenicity was observed. Additional details are provided in the presentation.
  • Hamburg Motor & Language Aggregate Score (n=12): The Hamburg Motor & Language Score, an assessment of ambulation and speech, shows a meaningful effect in slowing disease progression at 12 and 24 months:
     
    • On a combined scale of 0 to 6, the mean rate of decline was 0.4 vs 1.2 points over 12 months in treated patients (n=12) vs subjects from the natural history cohort (n=16).
       
    • On a combined scale of 0 to 6, the mean rate of decline was 0.6 vs 2.4 points over 24 months in treated patients (n=8) vs subjects from the natural history cohort (n=16).
       
  • Natural History (n=16): Within the natural history cohort, two years from their first decline on the Hamburg Motor & Language Score 63% of subjects experienced an additional 2-point decline (10 out of 16), compared to only 13% of treated patients (1 out of 8).
     

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 Children'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.