Benzinga | Jan 13, 2022 11:43AM EST

Lysogene Announced Termination of License Agreement With Sarepta for LYS-SAF302 Program, Regaining Global Commercial Rights

Lysogene (FR0013233475 LYS) (PARIS:LYS), a gene therapy platform company targeting central nervous system (CNS) diseases, today 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.

Karen Aiach, Chairman and Chief Executive Officer of Lysogene commented: "I wanted to thank Sarepta for helping us take the LYS-SAF302 program forward over the past years. With the data readout expected in a few months time, we believe more than ever that LYS-SAF302 is an important treatment option for patients suffering from MPSIIIA, and we remain fully committed to the MPSIIIA community."

Karen Aiach added: "The company keeps focusing its resources on its strategic objective of becoming a leading gene therapy technological platform targeting CNS diseases. Our priority remains to execute on our existing pipeline with the completion of the LYS-SAF302 pivotal trial, the respective completion and initiation of the recruitment of the safety and efficacy cohorts of the LYS-GM101 clinical trial for the treatment of GM1 gangliosidosis and move forward our pre-clinical programs in Fragile X and nGaucher/Parkinson. We are also constantly looking for opportunities to further expand our pipeline with new CNS gene therapy assets."