Passage Bio Stock Jumps After Gene Therapy Study In Lysosomal Storage Disorder

  • Passage Bio Inc PASG announced new interim safety, biomarker, and clinical development results from cohorts 1-3 in the Imagine-1 Phase 1/2 study of AAVhu68 gene therapy PBGM01 for GM1 Gangliosidosis (GM1). 
  • GM1 is a rare, fatal lysosomal storage disease in which mutations in the GLB1 gene result in the deficient activity of the enzyme beta-galactosidase (β-Gal), an enzyme that helps break down fats and sugars.
  • PBGM01 administration resulted in dose-dependent increases in CSF β-Gal activity, with both patients who received the high dose (Cohort 2, late infantile) exhibiting increases in enzyme activity well above baseline.
  • PBGM01 administration also resulted in dose-dependent decreases in CSF GM1 ganglioside levels.
  • Patients with milder development delay at dosing showed a higher response to PBGM01 treatment.
  • Patients 1 (late infantile, low dose) and 5 (early infantile, low dose), who both demonstrated modest developmental delay at baseline, showed increases in overall developmental age.
  • On the safety front, no treatment-related serious adverse events (SAEs) were observed. All treatment-related adverse events (AEs) were mild to moderate in severity.
  • Passage has completed dosing of PBGM01 in the dose-escalation portion of the study and will evaluate dosing further patients at the current high dose and/or a higher dose as data from cohorts 1-4 mature. 
  • Data from Cohort 4 is anticipated by mid-2023.
  • Price Action: PASG shares are up 6.14% at $1.21 on the last check Wednesday.
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