Amicus Therapeutics (NASDAQ:FOLD) Announce Encouraging Preliminary AT-GTX-501 Gene Therapy Results In CNL6 Batten Disease

Amicus Therapeutics (NASDAQ:FOLD) has announced more positive preliminary data from the CLN6 Batten disease gene therapy program, AT-GTX-501.

Amicus presents AT-GTX-501 results

The released results were featured in a poster presentation at the virtual Joint 16th International Child Neurology Congress and the 49th Annual Child Neurology Society Meeting. The Abigail Wexner Research Institute (AWRI) at the National Children’s Hospital is carrying the ongoing Phase 1/2 clinical study. The study evaluates a single one-off intrathecal administration of the At-GTX-501 gene therapy for a type of late-infantile neuronal ceroid lipofuscinosis 6 disease called CLN6 Batten disease. Currently, there is no approved treatment for CLN6 Batten disease, which is a neurological condition that can take away a child’s cognitive and motor functions.

Preliminary safety results are available for 13 children having CLN Batten disease, while interim efficacy results are available for the first 12 children. These are the children who reached the 12-month time point and eight children that reached 24 months after the AAV-CLN6 gene therapy administration.

AT-GTX-501 well-tolerated in patients

AT-GTX-501 treatment was well tolerated in children with mild adverse events unrelated to the therapy. There was no observed pattern of adverse events related to CLN6 or AAV immunogenicity. Assessment of ambulation and speech showed significant effects in reducing the disease’s progression at 12 and 12 months.

Amicus chief development officer Jeff Castelli said they are pleased to share positive interim clinical data for the intrathecal AAV gene therapy with the CLN6 community. He added that data suggests that gene therapy has the potential of treating CLN6 batten disease in children. The rare condition can result in a progressive decline in motor and cognitive functions and sometimes results in early death.

The study’s principal investigator Emily de loos Reyes said they are pleased with the CLN6 clinical trial’s progress. Reyes also said the collection of natural history data will further inform the outcome for the AAV-CLN6 gene therapy study. She added that preliminary results indicate that AT-GTX-501 has the potential of slowing disease progression in CLN6 Batten disease patients.