(Boursier.com) — Lysogen a Phase 3 biopharmaceutical company leveraging a gene therapy technology platform targeting central nervous system (CNS) diseases, presented updated clinical data from the ongoing Phase 2/3 AAVance clinical trial with gene therapy experimental LYS-SAF302 for the treatment of MPS IIIA (NCT03612869) at the American Society for Gene & Cell Therapy (ASGCT) 25th Annual Meeting in Washington, DC.
Preliminary results from the AAVance study evaluating LYS-SAF302 in patients with Sanfilippo syndrome type A (MPS IIIA) followed for at least two years showed improvement, stabilization or slowing of age decline from cognitive development (DA) in about half of patients. In particular, in the 6 patients recruited before the age of 30 months, a lasting increase or stabilization of cognitive, language and motor skills according to BSID-III was observed in the 24 months following treatment. Two of these 6 patients, one of whom is over 4 years old, have already exceeded the highest cognitive AD observed in the natural history cohort (35 months) by several months. The 2 patients achieved 41 and 42 months of cognitive AD respectively during the 24 months post-treatment, with an increase of 25 and 17 months respectively compared to their study inclusion visit.
These results, which suggest that young patients respond more to gene therapy treatment, need to be corroborated and confirmed by definitive statistical analyses. They will also include secondary behavioral and imaging endpoints as well as data from the observational study conducted in children treated with LYS-SAF302 using videos and interviews with parents (also called the PROVide study [Patient Reported Outcome Videos]). Full results will be available in Q3 2022.
Given these encouraging efficacy signals and the general trend observed for stabilization and/or reduction in the total volume of lesions in the white matter at the injection sites from 12 months after treatment, and in l absence of apparent clinically significant consequences, the company anticipates further discussions with regulatory authorities to determine next steps.
Karen Aiach, Founder and CEO of Lysogene said, “We are pleased to share these promising efficacy results in patients treated with LYS-SAF302 after a 2-year follow-up period. These data show encouraging signals for effectiveness on the cognitive, language and motor development of young patients. We look forward to confirming and supporting these observations by analyzing all the data collected since the start of the study, including the secondary behavioral and imagery as well as the real-life observations assessed by the video study. After this full analysis, expected in Q3 of this year, we will have all the elements necessary to discuss the way forward with the regulatory authorities.” Karen Aiach added: “These promising results will support our fundraising effort, which remains our priority. expand its financial visibility.”
AAVance is a single-arm, open-label, multicenter study to evaluate the efficacy in children with MPS IIIA of a single intracerebral administration of the gene encoding N-sulfoglucosamine sulfohydrolase (SGSH), using adeno-associated virus as a vector recombinant AAVrh.10 (LYS-SAF302, olenasufligene relduparvovec).
MPS IIIA is due to the presence of mutations in the SGSH gene coding for an enzyme necessary for the catabolism of heparan sulphate. By delivering a functional copy of the SGSH gene directly into the brain, LYS-SAF302 could enable it to produce the missing enzyme.
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