FOLD - Amicus announces early-stage interim data for AT-GTX-502 in Batten disease

Amicus Therapeutics ([[FOLD]] -1.2%) announced what it called the ‘positive initial results’ for its gene therapy candidate, AT-GTX-502, currently undergoing a Phase 1/2 study in classic juvenile neuronal ceroid lipofuscinosis (“JNCL”), also known as CLN3 Batten disease.Per the safety data available for the first four children (n=4) up to 15 months post-administration AT-GTX-502, the experimental therapy was well-tolerated with mild or moderate adverse events unrelated to treatment.Preliminary efficacy data available for the low-dose cohort (n=3) indicate that the mean yearly rate of change in UBDRS (Unified Batten Disease Rating Scale) Physical Impairment scores were +0.07 over 12 months vs. +2.86 in untreated subjects from published natural history (n=82).“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,” commented Jeff Castelli, Chief Development Officer of Amicus.With Rare Pediatric Disease and Orphan Drug designations from the FDA,

For further details see:

Amicus announces early-stage interim data for AT-GTX-502 in Batten disease