The aim of Lysogene’s IPO in February 2017 was to enable it to continue developing two drug candidates (LYS-SAF-302 and LYS-GM101) intended to treat two devastating and fatal central nervous system (CNS) diseases that start in infancy and cause the premature death of their sufferers. By directly attacking the causes of those diseases, our treatments have the potential to address widespread medical needs that are currently unmet.
Since our IPO, we have continued to make progress, reaching key milestones with our most advanced drug candidate, LYS-SAF302 for MPS IIIA. As a result, we will be starting the pivotal phase 2/3 trial for LYS-SAF302 in the second half of 2018. Our scientific board has also met with the European Medicines Agency to refine the development plan for our second drug candidate, LYS-GM101 for GM1 gangliosidosis.
We have expanded our programme portfolio, forming a partnership to develop AAV-based gene therapies aimed at treating Fragile X syndrome, the most common hereditary cause of learning disabilities and autism spectrum disorders. The Fragile X syndrome programme relies on Lysogene’s existing expertise in CNS diseases, and capitalises on the company’s clinical and manufacturing capabilities.
We are starting a new phase of our development through the recently announced partnership with Sarepta, a world leader in innovative treatments for genetic diseases, which marks a major step forward for Lysogene. We are confident that Sarepta is the ideal partner to market LYS-SAF302 in the United States and other countries outside of Europe. We are proud of this partnership, which represents a clear validation of our work, and we are looking forward to working closely with Sarepta in future. Our increased financial resources will enable us to maintain our commitment to developing a treatment for people suffering from MPS IIIA.
Finally, we have significantly strengthened our Board of Directors, welcoming Philippe Goupit, who has more than 30 years of experience in the pharmaceutical and biotech industry, along with Peter Lichtlen who joined us at the start of this year.
These changes show that Lysogene has entered a new phase of its development as we become a leading European player in gene therapy and leverage our recent agreement with Sarepta.
Thank you for your support and trust, and we aim to repay you with major progress and success in the coming years.
Founder and CEO