Regulatory News:

Lysogene (Paris:LYS) (FR0013233475 – LYS), a leading clinical-stage biopharmaceutical company specializing in gene therapy technology applied to central nervous system diseases (CNS), today reports its cash position at December 31, 2017.

Cash position and revenues as of December 31, 2017

As of December 31, 2017, cash and cash equivalents amounted to €14.1 million1 compared to €17.3 million1 as of September 30, 2017.

As expected, Lysogene did not generate any revenues during the fourth quarter of 2017.

Key operational highlights since September 30, 2017

  • Since September 30, 2017, Lysogene participated in the annual congress of European Society of Gene and Cell Therapy (ESGCT) and in the World Orphan Drug Congress. The company also led its first Key Opinion Leader Meeting in New York, with the participation of Ronald G. Crystal, MD, Professor and Chairman of the Department of Genetic Medicine at Weill Cornell Medicine.
  • During the quarter, the company continued to progress on CMC and manufacturing for its two product candidates. Lysogene also finished its preclinical toxicology work in order to determine the optimal dose for its pivot clinical trial in MPS IIIA.
  • In December, the Clinical Advisory Board dedicated to MPS IIIA and GM1 Gangliosidosis met for the first time, gathering world-renowned international gene therapy and lysosomal disease experts. The key output of this meeting was the confirmation of the feasibility of Lysogene’s direct-to-CNS trial design and recruitment strategy in MPS IIIA.

About Lysogene

Lysogene is a leading, clinical stage biotechnology company, specializing in the basic research and clinical development of AAV gene therapy for CNS disorders with a high unmet medical need. Since 2009, Lysogene has established a solid platform and network, with lead products in MPS IIIA and GM1, to become a global leader in orphan CNS diseases. Lysogene has obtained ODD from the EMA and FDA and rare pediatric designation by the FDA for both its MPS IIIA and GM1 programs.

Lysogene is listed on the Euronext regulated market in Paris (ISIN code: FR0013233475). For more information, please visit www.lysogene.com.

1 Unaudited and not subject to approval by the board of directors