We successfully completed our IPO on Euronext Paris in February, raising in about EUR24.6 million to finance our most important research and development projects. I would like to take this opportunity, on behalf of everyone at Lysogene, to welcome all new shareholders and extend our thanks to those pre-IPO shareholders that have supported us through this journey. We enter 2017 as a well-capitalized public company, focused on advancing our pipeline of novel adeno associated vector (AAV) gene therapies for severe, CNS diseases with unmet medical need.
Today we have two programs in our pipeline for mucopolysaccharidosis Type IIIA (MPS IIIA) and GM1 gangliosidosis (GM1). Both MPS IIIA and GM1 are classified as neuropathic (i.e. affecting the central nervous system) Lysosomal Storage Disorders, a group of approximately 50 rare inherited metabolic disorders that result from defects in lysosomal function. Simply put, both disorders prevent normal development of the central nervous system, resulting in premature death in children, presenting an urgent need for effective therapeutic intervention. However there is reason for hope as both diseases can be traced back to relatively simple genetic mutations (i.e. errors in the child’s genetic material), which makes them particularly good candidates for gene therapy, because the genetic correction of a small subset of neural cells should be sufficient to target large regions of the central nervous system.
We are very enthusiastic about our most advanced drug candidate, LYS-SAF302, which is nearing pivotal clinical testing for the treatment of MPS IIIA. We are currently completing preclinical work required by regulators and plan to begin the pivotal trials in the U.S. and Europe in the first quarter of 2018. MPS IIIA is a severe, progressive disorder caused by a mutation in the SGSH gene and resulting in severe neurological symptoms such as progressive dementia, aggressive behavior, hyperactivity, and an inability to sleep for more than a few hours at a time. The goal of LYS-SAF302 treatment is to correct the gene defect and restore long-term expression of the missing enzyme in the brain.
We also are encouraged by progress on our GM1 preclinical program. GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene and resulting in the progressive destruction of neural cells in the brain and spinal cord. Our lead candidate, LYS-GM101, has been designed to replace this defective gene in the cells of GM1 patients, in order to allow for production of the functional lysosomal acid beta-galactosidase (bgal) enzyme. LYS-GM101 was recently granted Orphan Drug Designation by both the FDA and EMA as well as Rare Pediatric Disease Designation by the FDA. Our goal is to enter the clinic in 2018.
We are singularly focused on driving forward the rigorous development of our lead products. And, going forward, our strategy is to continue to leverage the use of our technology, expertise and know-how to replicate this innovative therapeutic approach for other rare CNS diseases and thus broaden our portfolio of product candidates.
In order to do this, Lysogene has assembled a dedicated and talented team with all the necessary competencies and experience. In 2016 we made key additions to the leadership team and Board of Directors. Dr. Kimberly Gannon was appointed Chief Scientific Officer and Dr. Mark Plavsic, Chief Technical Officer. Both are based in our growing U.S. headquarters in Cambridge, Mass. We also appointed David Schilansky, currently CFO of the successfully allergy company DBV, to our Board of Directors. These accomplished and successful executives bring a wealth of world class experience in R&D, manufacturing and finance. We look forward to the opportunities and responsibilities of being a publicly listed company. We thank you for your support and trust and look forward to sharing our progress and success with you in the coming years.