Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, has announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type A (MPS IIIA). The clinical study was approved by the Australian Government Department of Health Therapeutic Goods Administration and the Company is conducting the Phase 1/2 clinical study at Adelaide Women’s and Children’s Hospital, Australia.
“MPS IIIA is a profound degenerative disorder of childhood, which manifests progressive neuromotor and cognitive decline with associated systemic complications and premature death, typically before adulthood. The reported clinical data has proved most encouraging with a reduction in central nervous system (CNS) heparan sulphate, a reduction in liver volume, and preliminary evidence of slowed neurocognitive decline,” indicated Nicholas Smith, M.D., Ph.D., Principal Investigator and Dept. Head, Neurology and Pediatric Neurologist at Women’s and Children’s Hospital. “With no approved treatments for this relentless disease, this investigational gene therapy approach, delivered as a single intravenous injection to treat the whole body, holds great potential for patients and their families.”
The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA. Per the design of the trial, subjects receive a single, intravenous injection of ABO-102 to deliver the AAV viral vector systematically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease, particularly the CNS. Subjects are evaluated at multiple time points over the initial six-months post-injection for safety assessments and initial signals of biopotency. The global clinical study is supported by a 25-subject MPS III Natural History Study, which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments.
“Abeona continues to demonstrate global leadership in advancing clinical and commercial development of promising gene therapies for children with Sanfilippo syndrome, and the additional clinical site in Australia will help advance global access to this potential treatment for families suffering from this devastating disease,” said Megan Donnell, Executive Director of The Sanfilippo Children’s Foundation.