Lysogene Announces first European patient treated in AAVance, Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)

Regulatory News:

Lysogene (FR0013233475 – LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system (CNS) diseases announced today, the first European patient has been dosed in AAVance, a global Phase 2-3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). Lysogene and Sarepta Therapeutics, its commercialization partner for LYS-SAF302, announced the treatment of the first patient in this clinical trial in the US earlier this year.

AAVance is a single-arm trial evaluating the safety and effectiveness of a one-time delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene. MPS IIIA is caused by mutations in the SGSH gene, which is involved in producing an enzyme necessary for the breakdown and disposal of long chain sugar molecules. The goal of the therapy is to show improved or stabilized neurodevelopmental status of MPS IIIA patients by allowing the brain cells to secrete the missing enzyme. In the clinical trial, the objective is to enroll 20 patients at eight sites in the U.S. and Europe. More information can be found on www.clinicaltrials.gov (NCT03612869).

“The first European patient dosed in the AAVance trial is an important step in addressing this relentlessly progressing neurodegenerative disease,” said Karen Aiach, Founder and Chief Executive Officer of Lysogene. “We are encouraged by the preliminary observations in AAVance to date, and are excited to bring this investigational therapy to patients in Europe.”

“MPS IIIA is a lethal neurological disease with debilitating symptoms for which there is currently no approved treatment,” said Prof Frits Wijburg, Academic Medical Center, University of Amsterdam and principal investigator for AAVance. “It is a great motivation to know that the work we are doing has the potential to make a life-changing difference for the children affected by this disease.”

“We are very pleased to have successfully administered LYS-SAF302 to the first European patient and mark this as a milestone for the field in advancing a potential one-time treatment for patients with MPS IIIA,” said Prof Michel Zerah, Hôpital Necker Enfants Malades in Paris.

About MPS IIIA

MPS IIIA is a rare inherited neurodegenerative lysosomal storage disorder affecting approximately 1 in 100,000 newborns. Inherited in an autosomal recessive pattern, it is characterized by intractable behavioral problems and developmental regression resulting in early death. It is caused by mutations in the SGSH gene, which encodes an enzyme called Heparan-N-sulfamidase necessary for heparan sulfate (HS) recycling in cells. The disrupted lysosomal degradation and resulting storage of HS and glycolipids such as gangliosides leads to severe neurodegeneration. There are currently no treatment options for patients.