Oxford BioMedica, a UK-based gene and cell therapy group, has published results from the RetinoStat (OXB-201) Phase I study in patients with advanced wet age-related macular degeneration (AMD) in the journal Human Gene Therapy.
The results were previously announced on May 6, 2016.
The company said that, according to key published findings in the associated peer-reviewed paper, RetinoStat demonstrated a favourable safety profile and led to robust, reproducible sustained expression of endostatin and angiostatin in the eye.
The Phase I study was primarily designed to evaluate the safety and tolerability of RetinoStat for the treatment of severe wet AMD following a single subretinal injection and represented the first time a lentiviral based vector had been administered to the human eye. Twenty-one subjects with highly fibrotic retinas who were refractory to anti-VEGF therapy following a prior responsive history were treated. As previously announced the results of the Phase I study indicated that RetinoStat met the primary endpoint of safety and tolerability. Importantly, therapeutic gene expression, measured in these patients as a secondary study endpoint, was found to be dose-dependent and maintained at the last measurement (2.5 years in 8 subjects and >4 years in two subjects).
John Dawson, the CEO of Oxford BioMedica, said “Like the ProSavin trial before it, the RetinoStat First-in-Man study was a clinical trial of ‘firsts’: the first ever trial to directly administer a lentiviral vector-based product to the eye, the first directly administered lentiviral vector trial in the USA, the first direct measurement of an ocular gene therapy transgene during a study and the first reporting of data showing direct demonstration of long-lasting expression of an ocular gene therapy in human subjects.
“These peer-reviewed published results further validate the ground-breaking utility of our LentiVector delivery platform for the treatment of chronic disease.”