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

•  Lysogene remains focused on developing its pipeline led by LYS-SAF302, LYS-GM101, Fragile X and other high potential preclinical projects

PARIS–(BUSINESS WIRE)–Regulatory News:

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.”

About Lysogene

Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). 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. www.lysogene.com.

Forward Looking Statement

This press release may contain certain forward-looking statements, especially on the Company’s progress of its clinical trials and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Company’s control, (iii) clinical trial results, (iv) increased manufacturing costs, (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “objective”, “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Company’s regulatory filings with the French Autorité des Marchés Financiers, including in the 2020 universal registration document, registered with the French Markets Authorities on April 12, 2021, under number D.21-0296, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

Contacts

Stéphane Durant des Aulnois

Chief Financial Officer

[email protected]
+ 33 1 41 43 03 99