PARIS, France I July 6, 2016 I Lysogene, a leading, gene therapy clinical-stage biotechnology company, today announces the enrolment of the first patient in its Sanfilippo A multi-national observational study (SAMOS); Sanfilippo A is also known as Mucopolysaccharidosis Type IIIA (MPS IIIA). This is a prospective natural history study, so no experimental drug will be given. Patients will receive study-related care. Recruitment is planned before the start of Lysogene’s gene therapy (LYS-SAF302) trial. The information gained from this study will enable optimal trial design, greater understanding of disease progression and better predictions of future therapeutic effects.

The protocol for this observational study is the result of an international multi-stakeholder collaboration to reach agreement on the cognitive and related assessments of Sanfilippo Type A. “This study is critical for comparability of clinical endpoints with the future gene therapy trial and will complement existing published data,” stated Soraya Bekkali, Lysogene’s Chief Medical Officer.

“The neuropsychology-focused group organized by Lysogene brought together renowned experts in the field and afforded a much needed occasion to consolidate experience,” said Jan Pieter Marchal, Research Psychologist at AMC Medical Research BV at the University of Amsterdam. “It was a milestone for participants who rarely meet in such early stages of protocol design.”

“Lysogene is proud to collaborate with MPS PACT (Patient Access to Clinical Trials) for travel arrangements for families to clinical study sites,” said Samantha Parker, Lysogene’s Chief Health Policy and Patient Access Officer. “They will provide a safe and independent logistical resource to families of children recruited to the study.”

Lysogene conducted a phase I/II clinical trial, in 2011-2012, with its first generation gene therapy formulation LYS-SAF301. Currently, Lysogene is planning a clinical trial with its next generation formulation LYS-SAF302. LYS-SAF-302 has the potential to replace the defective gene in the cells, of Sanfilippo Type A patients, which will allow for the production of the enzyme and prevent the progressive nature of the neurological damage. Individuals included in the observational study will be provided the opportunity to enroll in subsequent trials if they satisfy the specified enrolment criteria. However, participating in the observational study does not guarantee a place in the gene therapy clinical trial.

About Mucopolysaccharidosis Type A (also known as Sanfilippo A):

MPS IIIA is a lysosomal disease caused by an autosomal recessive defect of the SGSH gene and affecting approximately 1:100,000 live births. MPS IIIA presents in early childhood, causing progressive neurodegeneration associated with intractable behavioral problems and developmental regression. Life span is shortened, and there is currently no treatment.

About Lysogene

Lysogene is a clinical stage biotechnology company pioneering in the basic research and clinical development of AAV gene therapy for CNS disorders with a high unmet medical need. Since 2009, Lysogene has established a unique platform and network, with lead products in Sanfilippo A and GM1 Gangliosidosis, to become a global leader in orphan CNS diseases.

SOURCE: Lysogene