Lysogene completes Enrollment in SAMOS

Lysogene has completed the enrollment in its “Sanfilippo A Multi-national Observational Study” (SAMOS). Sanfilippo A is also known as Mucopolysaccharidosis Type IIIA (MPS IIIA).

SAMOS is particularly important as there is currently no validated biomarker in MPS IIIA that reflects CNS disease progression and response to future therapy. SAMOS has therefore been designed to evaluate clinical change in untreated MPS IIIA patients. As agreed with the regulatory authorities, this international multi-center study is to function as a non-concurrent control group for the upcoming Lysogene Phase II/III pivotal gene therapy trial, scheduled to start during the first quarter of 2018.

Armand-Trousseau Hospital, APHP France, neuro-pediatrician, Dr. Benedicte Heron said that Lysogene has taken a very proactive and rigorous approach to gaining a better understanding of MPS IIIA. The company’s efforts in running this observational study will support its own plans for future research and therapy development while also aiding the scientific community as a whole.

Lysogene Chief Patient Access Officer, Samantha Parker said that Enrollment of the 23 children from 5 countries has been rapid, reflecting the strong interest from the key opinion leaders running their clinical sites and the network of patient associations to address the significant unmet needs in MPS IIIA.

In designing SAMOS, Lysogene established the first international neurologist and neuropsychologist MPS IIIA expert group. This group determined that the most scientifically rigorous and relevant primary endpoint was cognitive assessment using the Bayley Scales of Infant and Toddler Development, 3rd edition (BSID-III). The Vineland Adaptive Behavior Scale, 2nd edition (VABS-II) was determined to be the most appropriate as a secondary endpoint measure.

Lysogene has successfully completed a Phase I/II trial and 5-year follow-up study of four MPS IIIA patients with no adverse events related to the treatment. Lysogene’s gene therapy candidate for MPS IIIA is a 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 in one 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 neural cells.