Passage Bio presents additional interim data from Imagine-1 study

Passage Bio announced updated clinical data from Imagine-1, a Phase 1/2 study of PBGM01, a gene therapy for GM1 gangliosidosis, are being presented today at the 19th Annual WORLDSymposium. Imagine-1 is a global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna injection in four cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis. "We are pleased to share promising data from the first six patients in our Imagine-1 study that continue to demonstrate PBGM01 has exerted a biological effect in patients with infantile forms of GM1 gangliosidosis," said William Chou, M.D., chief executive officer of Passage Bio. "At this interim analysis, all treated patients showed stabilization of MRI severity scores, a potential marker of disease severity and progression measured by structural damage of the brain. Furthermore, patients exhibited decreases in urine levels of the beta-Gal substrate Dp5, an exploratory biomarker of peripheral beta-Gal activity. These data are supported by the favorable safety profile of PBGM01 and bolster our confidence in PBGM01 as a potential treatment option for GM1 patients. We look forward to continued advancement of our Imagine-1 study and sharing new data from Cohort 4 by mid-year."