Lysogene announces important updates on AAVance phase 2/3 clinical trial with LYS-SAF302 in MPS IIIA
- Category: DNA RNA and Cells
- Published on Friday, 05 June 2020 11:05
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PARIS, France I June 05, 2020 I Lysogene (Paris:LYS) (FR0013233475 – LYS), a Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that, following discussions with the U.S. Food and Drug Administration (FDA), a clinical hold was issued for the clinical trial AAVance (NCT03612869), a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A). To date, 19 out of 20 patients have been treated, and all patients remain in the study and are being followed per study protocol. The primary and secondary trial endpoints are based on the analyses of these 19 patients already enrolled and there is no anticipated impact on the current clinical trial timelines.
The clinical hold follows observations in some patients of localized findings on MRI images at the intracerebral injection sites. The localized nature of the findings suggests a potential connection to delivery. To date, no clinical symptoms have been observed that could be directly attributed to the observed MRI findings and all findings have been reported to competent authorities, ethics committees and the trial’s DSMB (Data Safety Monitoring Board). All trial participants continue to be closely monitored by their care teams. A path forward has been discussed with FDA, and Lysogene looks forward to gathering additional information to better understand the safety profile of LYS-SAF302.
“I would like to thank the families for their participation in the study” said Dr Sophie Olivier, Chief Medical Officer at Lysogene. “We consider patient safety the utmost priority and believe the clinical development of LYS-SAF302 can offer meaningful benefits to patients with this devastating disease. We will now focus on gathering additional information and continue working with FDA to determine the appropriate path forward.”
Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. www.lysogene.com.