PARIS--(BUSINESS WIRE)--Regulatory News:
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.
Forward Looking Statement
This press release may contain certain forward-looking statements, especially on the Company’s progress of its phase 2-3 clinical trial and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Company’s control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “objective”, “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Company’s regulatory filings with the French Autorité des Marchés Financiers, including in the 2019 registration document (Document de référence), registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.
This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.