Lysogene announced the termination of its license agreement with Sarepta for LYS-SAF302, a phase 2/3 asset for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA), effective as of July 11, 2022. This termination follows unsuccessful discussions for transferring back to Lysogene the responsibility for the global commercial supply of LYS-SAF302. The termination of the agreement will enable Lysogene to regain development and commercialization rights for LYS-SAF302 in the US and other non-EU territories as well as the responsibility for global commercial supply of LYS-SAF302, all previously granted to Sarepta.

Lysogene will be entitled to receive reimbursement for certain costs associated with the termination. LYS-SAF302 was granted Orphan Drug Designation in the US and European Union. In the US, it also has obtained Fast Track and Rare Pediatric Disease designations.

The global, open-label, single-arm, multi-center Ph2/3 clinical trial AAVance (NCT03612869) with LYS-SAF302 is fully enrolled and fully dosed, and all patients are being monitored per study protocol. The primary endpoint data readout of this registrational trial is expected mid-2022, as initially anticipated.