Abeona treats first patient in second cohort of Phase 1/2 ABO-101 trial
Abeona Therapeutics announced that the company has completed dosing cohort 1 and treated the first patient in cohort 2 in an ongoing Phase 1/2 clinical trial evaluating ABO-101, its novel one-time gene therapy for Sanfilippo syndrome type B. The company expects to report interim data from the trial in the second half of 2019. "We believe that ABO-101 can help address the underlying lysosomal enzyme deficiency that ultimately results in progressive cell damage and neurodevelopmental and physical decline in children with MPS IIIB," said Joao Siffert, M.D., CEO. The Phase 1/2 trial is a two-year, open-label, dose-escalation, global clinical trial in patients diagnosed with MPS IIIB who are older than six months of age and have a minimum cognitive Development Quotient of 60 or above. The study is expected to include up to nine patients and is designed to evaluate two doses of ABO-101 with cohort 1 receiving 2 X 1013 vg/kg and cohort 2 receiving 5 X 1013 vg/kg. ABO-101 is delivered using NAV AAV9 technology via a one-time intravenous infusion. The primary endpoint of the study is to assess neurodevelopment and safety, with multiple secondary and exploratory endpoints including neurocognitive and behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid and plasma, biomarkers in CSF, plasma and urine, and brain and liver volume.