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Word from the CEO

Karen aiach
Karen Aiach, Founder and CEO

Dear Sir/Madam,

We are very pleased to announce the success of our initial public offering on Euronext Paris, which constitues a step that is a key milestone in the development of our company. We would like to take this opportunity to thank our historical shareholders as well as new partners for the trust they have placed in us and their decision to accompany Lysogene in the development of an innovative technology in gene therapy for the treatment of rare and deadly central nervous system diseases.

Lysogene is a clinical-stage biotechnology company that specialises in gene therapy for rare genetic pediatric neurological diseases, that are devastating, fatal, and for which to our best knowledge, there is currently no treatment. Driven by my desire as a parent to find a treatment for my daughter’s illness, I co-founded Lysogene in 2009 with Professor Olivier Danos, an internationally recognised pioneer in the field of gene therapy for neurodegenerative diseases. Since its creation, Lysogene has put the needs of patients and families at the heart of its mission. Our aim is to develop and provide patients with innovative, safe medicines capable of correcting the gene defect resulting in a better quality of life for the child and their family. By achieving this, we aim to reduce the social and financial impact involved in caring for people suffering from these diseases.

Our most advanced drug candidate, LYS-SAF302, targets mucopolysaccharidosis Type IIIA (MPS IIIA). MPS IIIA is a severely disabling pediatric lysosomal storage disorder that causes the child’s premature death. There is currently no marketed treatment that can stabilise or slow the deterioration in the clinical condition of patients caused by the disease.

We have successfully completed a Phase I/II trial that produced good results in terms of tolerability and safety. Though the results are good, the program needed to be improved to make the treatment more effective. As a result, we developed a second-generation product, LYS-SAF302, for which we are planning to start a pivotal trial in the first quarter of 2018. LYS-SAF302 has Orphan Drug Designations from the European and US healthcare regulators, which i) validate its medical potential and ii) mean that LYS-SAF302 benefits from specific regulatory support measures. LYS-SAF302 has also been granted a Rare Pediatric Disease Designation (RPDD) by the FDA, which gives it further specific advantages.

We have also developed a second product candidate (LYS-GM101) for GM1 gangliosidosis (or Landing disease), another severely disabling pediatric disease that also causes the premature death of children. As with MPS IIIA, there is currently no treatment that can halt the progress of this disease. In February 2015, Lysogene initiated a research collaboration with the University of Massachusetts Medical School and the University of Auburn, Alabama, with the aim of starting clinical trials on GM1 gangliosidosis in the near future. LYS-GM101 has also been granted an RPDD by the FDA.

In time, our strategy is to capitalise on our renowned expertise and know-how to replicate our innovative therapeutic approach for other rare and fatal CNS diseases and thus broaden our portfolio of product candidates.

Lysogene's team has driven cutting-edge drug development in two initial programs, working closely with networks of patient associations, reference centers, opinion leaders and experts from the scientific and academic community, with the ambition of establishing itself as a global leader in orphan and rare CNS diseases.

Today, we are delighted that you are joining us, through our IPO, as we reach another milestone in Lysogene's development. We have already come a long way, but there is still a lot to do. We are involved in a race against time, because treatment must happen very early to give patients any hope of radically changing the course of their lives and those of their friends and family.

Karen Aiach
Founder and CEO