Amicus Therapeutics, Inc. (NASDAQ:FOLD) initiated the phase 1/2 clinical trial to find the effectiveness and safety of AAV9-CLN3 (adeno associated virus serotype 9 AAV9-CLN3) in children suffering from CLN3 Batten Disease. The company has not observed any serious side effects during the administration of the drug in the first month.

Life-Threatening CLN3 batten disease

The life-threatening CLN3 batten disease usually begins in the early childhood. If it is not treated in time, the disease could lead to the premature death of the child. Amicus is conducting the trial of the investigational gene therapy to treat children suffering from CLN3 batten disease. Neuronal ceroid lipofuscinoses (NCL), the inherited nervous system disorder, affects over 5,000 children.

CMO of Amicus, Jay Barth is pleased with the one-month outcome of the investigational therapy administered on CLN3 batten disease affected the child. No proven therapy exists in the market to treat children with CLN3 batten disease. The company is trying to fill this unmet medical need with innovative gene therapy and save the lives of thousands of children. The initial phase ½ study provided encouraging safety profile for its intrathecal AAV delivery platform.

The principal investigator at the National Children’s Hospital, Emily C. De Los Reyes, MD, said the phase ½ clinical study involving AAV9-CLN3 to treat CLN3 batten disease is an important milestone for Amicus. The demonstrated proof of concept in the clinical study and validation of intrathecal AAV delivery platform at National Children’s Hospital shows a promise to cure the nervous system disorder in children. She is looking forward to onboarding more children for the ongoing study and to match the findings with the historical data available in CLN3 batten disease.

Enrolling of children for phase 1/2 clinical trial

Amicus is onboarding children, who are in the age group of 3 and ten years and diagnosed with CLN3 batten disease, for clinical study in two groups. In the first group, Amicus will administer a lower dose of AAV9-CLN3 on children. After evaluating the outcome of the children in the first group, it will administer a higher dosage on children in the second group. The current study will last for three years. The company will share the clinical data at the future scientific congress.

Amicus promoted Daphne Quimi to CFO with effective from January 1, 2019. She will fill the gap of Chip Baird.