Clinical Trials

Date: 2018-09-05

Type of information: Clinical trial authorisation

phase: 2-3

Announcement: clinical trial authorization

Company: Lysogene (France)

Product: LYS-SAF302

Action mechanism:

gene therapy. LYS-SAF302 is an rAAV vector serotype rh.10 carrying the gene coding for SGSH. This in vivo gene therapy offers the possibility of a one-time treatment by inserting a healthy copy of the SGSH gene and allowing the body to start making the missing enzyme, therefore slowing or halting disease progression. Lysogene’s gene therapy is delivered directly to the CNS during a neurosurgical procedure. By delivering the missing SGSH gene, Lysogene believes MPS IIIA patients will be provided a permanent source of functional enzyme in the brain that reverses phenotypic abnormalities of CNS cells.
Additionally, LYS-SAF302 has received Orphan Drug Designation from the FDA and EMA as well as Rare Pediatric Disease Designation from the FDA.

Disease: mucopolysaccharidosis type IIIA (Sanfilippo B syndrome)

Therapeutic area: Rare diseases - Genetic diseases

Country: France, Germany, The Netherlands, USA

Trial details:

MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients. (NCT03612869)

Latest news:

• On September 5, 2018, Lysogene announced that the FDA has granted an Investigational New Drug (IND) clearance to proceed in the U.S. with its international, Phase 2-3 (AAVance) clinical trial of LYS-SAF302 for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA). Lysogene has also submitted Clinical Trial Applications (CTA) in Europe. LYS-SAF302 Pediatric Investigation Plan (PIP) received approval from the PDCO earlier this year.
Lysogene is currently looking for funding opportunities in order to extend its cash runway and launch the MPS IIIA clinical trial, notably through a strategic partnership or transaction, or potential sale of one or several products.
• On February 6, 2018, Lysogene announced that the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) adopted a positive opinion regarding the company’s Paediatric Investigation Plan (PIP) for LYS-SAF302 in patients with mucopolysaccharidosis type IIIA (MPS IIIA). Early PIP approval for LYS-SAF302 confirms the appropriate design of Lysogene’s phase 3, single arm clinical trial in children with MPS IIIA. Additionally, the positive PIP opinion makes Lysogene eligible for an additional two-year marketing exclusivity extension on top of the ten-year marketing exclusivity based on the EMA Orphan Drug Designation.