Abeona's Global Sanfilippo Syndrome Trial Gains Ground In Spain

Abeona enrolls first subject in Spain in ongoing phase 1/2 clinical trial in MPS IIIA, also known as Sanfilippo syndrome.

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, has enrolled the first patient in its ABO-102 Phase 1/2 clinical trial in Spain in the fight against Mucopolysaccharidosis type III (MPS IIIA), more commonly known as Sanfilippo syndrome. The patient was enrolled at the Hospital Clinico Universitario of Santiago de Compostela.

Abeona has established a local subsidiary to manage clinical trial and regulatory development efforts in Europe.

Sanfilippo syndrome is a progressive disorder that primarily affects the brain and spinal cord, along with other body systems, according to the National Institutes of Health (NIH). People with MPS III generally do not display any features of the condition at birth, but they begin to show signs and symptoms of the disorder during early childhood. Affected children often initially have delayed speech and behavior problems.

NIH says those afflicted may become restless, destructive, anxious, or aggressive, and some display features of autism spectrum disorder, which is a condition characterized by difficulty with social interactions and communication. In later stages, people with MPS III may develop seizures and movement disorders.

“Developing a local company presence in Spain allows us to exercise closer supervision and further advance Abeona’s gene therapy programs in Europe, as well as advance our relationships with the patient community,” Juan Ruiz, M.D., Ph.D., chief medical officer of Abeona Therapeutics, said in a statement.

In the clinical trial subjects receive a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease.

Subjects are evaluated at multiple time points post-injection for safety assessments and signals of biopotency and clinical activity, which to date have demonstrated that ABO-102 successfully reaches target tissues throughout the body, including the central nervous system.

A total of nine subjects have been infused in the ongoing global MPS IIIA dose-escalation clinical trial, which has been well-tolerated through more than 2,000 cumulative follow-up days. Safety and efficacy data has been reviewed and enrollment in Cohort 3 continues at all three active clinical sites (U.S., Spain, and Australia).

(Source: Abeona Therapeutics Inc.)