14 January 2022 - - France-based gene therapy platform Lysogene's (FR0013233475 LYS) (PAR: LYS) license agreement with Sarepta for LYS-SAF302, a phase 2/3 asset for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA), has been terminated, 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.

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system.

The company has built a unique capability to enable a delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA is ongoing.

An adaptive clinical trial in GM1 gangliosidosis is also ongoing. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.