London, UK, June 10, 2024: Focalis Bio, a biotechnology company focused on research and development of cutting-edge gene therapies to treat focal refractory epilepsy, today announced the completion of canine biodistribution studies with AAV9 intraparenchymal gene therapy, supporting the planned submission of a clinical trial application for the lead program EPY201.
The toxicology and biodistribution studies have confirmed the tolerability and optimal, highly localized biodistribution of AAV9 intraparenchymal gene therapy in the brain of treated animals. The data from the studies will complement the data already generated in the preclinical mouse efficacy, tolerability and GLP toxicology studies with EPY201. The totality of the data will be included in the planned submission of a first-in-human, Phase 1/2a clinical study with EPY201.
"The canine GLP biodistribution studies with AAV9 have confirmed the highly localized distribution of AAV9 in the target focus in the brain," said Dimitri Kullmann, chief scientific officer and scientific founder of the company. “We have made fantastic progress towards a clinical trial application for EPY201. The mouse efficacy and tolerability studies with EPY201 are complete and have confirmed the tolerability and potent antiseizure effect of EPY201. Furthermore, the GLP toxicology mouse study with EPY201 is ongoing and the in-life phase will complete this month”.
Nicolas Koebel, chief executive officer, commented, “The team has done an impressive job in delivering a robust nonclinical data package that will support the submission of a clinical trial application for the lead program EPY201. We now look forward to initiating a groundbreaking first-in-human study with EPY201, which will be another milestone for the company.”
