LYS-SAF302 is a viral vector that carries a healthy SGSH gene. This gene gives instructions to produce an enzyme called sulfamidase, which is defective in Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA).
This gene therapy is a one-time treatment that allows the body to make the missing enzyme, which slows or halts disease progression.
Lysogene’s gene therapy is delivered directly to the central nervous system (CNS) during a neurosurgical procedure.
After positive preclinical studies on effectiveness and supporting toxicology for an investigational new drug (IND) application, the company is now preparing its pivotal Phase 2/3 single arm clinical trial in children with MPS IIIA, to begin in the second half of 2018.
In January, Lysogene received a positive opinion from the Pediatric Committee (PDCO) of the European Medicines Agency (EMA) for LYS-SAF302. The important regulatory milestone confirmed the design of the proposed Phase 2/3 clinical trial in children with MPS IIIA.
The start of the pivotal Phase 2/3 trial was first planned to start right after EMA’s PDCO positive opinion. However, due to manufacturing delays, the company had to postpone the launch to the second half of 2018.
Despite this delay, all plans to activate eight clinical sites in the U.S. and Europe for this trial are now back on track and will ensure maximum patient access to investigate Lysogene’s investigational gene therapy.
Consequently, Lysogene is eligible for an additional two-year marketing exclusivity extension in addition to the 10-year marketing exclusivity based on EMA’s orphan drug designation for the drug candidate.
The company also announced that enrollment in an International Pivotal Observational Study (SAMOS) was completed, serving as a standard for comparison in the Phase 2/3 clinical trial, as agreed with U.S. and European regulatory authorities. All patients in this study will complete their 12-month visit by the end of the first half of 2018.
“We enter 2018 with significant momentum to achieve key clinical development and regulatory milestones across our portfolio of gene therapy products for neurological diseases,” Karen Aiach, founder and CEO of Lysogene, said in a press release. “This year, we expect to initiate our pivotal clinical trial in Mucopolysaccharidosis type IIIA (MPS IIIA) in the U.S. and Europe and pursue IND enabling studies for our GM1 Gangliosidosis (GM1) product.”