Lysogene announces the end of patient enrollment for the first pivotal study a prospective, observational…

A rapid recruitment : 23 patients in less than a year

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A scientific consensus on tools for assessing cognitive and behavioral (BSID-III, VABS-II) used

PARIS, France and CAMBRIDGE MA USA – may 30, 2017, 07: 00 CET – Lysogene (the “Company” FR0013233475 – LYS), a biopharmaceutical company pioneering and specializing in gene therapy targeting the rare disease of the central nervous system (CNS), announced today the completion of recruitment of patients in the framework of SAMOS [1], observational study international disease Sanfilippo type A – also known as Mucopolysaccharidosis Type IIIA (MPS IIIA).

SAMOS is particularly important because there is currently no biomarker is validated to follow the progression of MPS IIIA in the CNS, or to observe the effects on the CNS of the future proposed treatment. SAMOS has therefore been designed to evaluate the clinical progression of untreated patients with MPS IIIA. As agreed with the regulatory authorities, this international study was a multicenter, will serve as the “control group” in view of the pivotal study of Lysogene (Phase II/III) via gene therapy, which should begin in the first quarter of 2018.

“Lysogene has adopted a very pro-active approach and rigorous to improve the knowledge of MPS IIIA,” said Dr. Benedict Heron, interviewed at the Hospital Armand-Trousseau (AP-HP), Paris, France. “The efforts made by the Company to carry out this observational study will allow him to further develop his research and therapies, while useful to the whole scientific community. ”

Samantha Parker, Director of External Affairs of Lysogene, said : “The recruitment of 23 patients in 5 different countries took place quickly. The leaders of our clinical sites and the networks of associations of patients have shown their interest and their active participation in order to respond to medical needs as yet unmet in the MPS IIIA. ”
In designing SAMOS, Lysogene was the first panel of international experts in neurology and neuropsychology to the MPS IIIA. These experts considered that the main criterion to assess is the cognitive function. A criterion to be both rigorous and measurable scientifically, thanks to the tool that is the scale of the Bayley of infant development and the infant, 3rd edition (BSID-III). The scale Vineland on the adaptation of the behavior, 2nd edition (VABS-II) was defined as the second measurement tool is the most appropriate.

To go further in the validation of the clinical criteria of the MPS neurological, a consensus meeting between the international experts was held in London on 2 and 3 December 2016 by an academic committee in partnership with the UK Society for Mucopolysaccharide Diseases and the US National MPS Society, chaired by Elsa Shapiro [2]. Among all the instruments available, the BSID-III has definitely been recommended to measure the cognitive outcomes, while the VABS-II has been recommended to measure the evolution of adaptive behavior (based on the long version of the interview (van der Lee, 2017)). [3]

Lysogene has already obtained clinical data of the study SAMOS, in evaluating the age-cognitive in the first 15 patients included, ages 3 to 8 years, depending on the scale, BSID-III, which confirmed that the intellectual decline, progressive, hyperactivity, and behavior disorders in these patients.

Lysogene has also completed its clinical trial phase I/II as well as its follow-up study of 4 patients with MPS IIIA for 5 years without any treatment-related adverse effects or mode of administration. [4]

Lysogene target for the treatment of neurological symptoms of the disease MPS IIIA
MPS IIIA is a fatal disease of the CNS that require a targeted treatment. The product of gene therapy in development by Lysogene to the disease MPS IIIA is a vector rAAV serotype rh.10, including the gene encoding SGSH. Vivo gene therapy offers the possibility of a treatment at once, by inserting a healthy copy of the gene SGSH in the body, allowing the body to produce the missing enzyme, with the objective to slow down or even stop the progression of the disease. The candidate drug is delivered directly into the brain in a single procedure neurosurgical.

For more information about SAMOS: Id: NCT02746341.

About Lysogene
Lysogene is a biotechnology corporation clinical stage pioneer in the basic research and the clinical development of gene therapies using vectors derived from adeno-associated viruses to treat rare diseases and life-threatening CNS of the child, for which there is today, to the knowledge of the Company, no treatment. Since 2009, Lysogene has put in place a strong platform and a large network, with innovative products in the MPS-IIIA and in the gangliosidosis GM1. Each of the products developed by Lysogene, LYS-SAF302 and LYS-GM101, has orphan drug designations by the FDA and the EMA, and the designation of rare disease pediatric by the FDA.
Lysogene is listed on the regulated market of Euronext Paris of Euronext Paris (ISIN code : FR0013233475). More information :

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