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Sio Gene Therapies' GM1 Gangliosidosis Gene Therapy Candidate Shows Substrate Reduction In CSF

Sio Gene Therapies' GM1 Gangliosidosis Gene Therapy Candidate Shows Substrate Reduction In CSF
  • Sio Gene Therapies Inc (NASDAQ: SIOX) has announced new biomarker data from Phase 1/2 study of AXO-AAV-GM1, an adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis.
  • This new data will be presented at the American Society of Gene & Cell Therapy (ASGCT) today.
  • Today's cerebrospinal fluid (CSF) data, demonstrating a biomarker response in the CNS, provides the first indication that intravenous administration of AXO-AAV-GM1 may treat both the systemic and neurological manifestations of the disease.
  • The data represents the first direct evidence in humans that intravenously administered AXO-AAV-GM1 gene therapy exerts a measurable biochemical effect on GM1 ganglioside accumulation.
  • Key findings from the new biomarker analysis:
    • 18-49% reductions from baseline in the GM1 ganglioside (accumulated substrate) were observed in CSF of 4 out of 5 children in the low-dose cohort (1.5x1013 vg/kg) at six months.
    • 3 out of 5 children demonstrated CSF GM1 ganglioside levels less than 100 ng/mL at the 6-month follow-up visit.
    • One child, whose disease was the most advanced at baseline and worsened on certain clinical parameters, exhibited an increase in CSF GM1 ganglioside of 19% from baseline at six months.
  • Upcoming Catalysts:
    • 12-month data from the low-dose cohort is expected in the second half of 2021.
    • 12-month data from the first two children dosed in the high-dose cohort ( 4.5x1013 vg/kg) in Q1 2022.
  • AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an AAV vector to restore β-galactosidase enzyme activity to treat GM1 gangliosidosis.
  • Price Action: SIOX shares are trading 0.39% higher at $2.56 during the premarket session on the last check Thursday.

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