Passage Bio Announces Promising Interim Clinical Data from First Eight Patients with GM1 Gangliosidosis in Imagine-1 Study

• Interim safety data up to 28 months showed Dose 1 and 2 of PBGM01 were well tolerated and had a favorable safety and immunological profile
  
• Dose 2 resulted in substantial improvements in key CSF biomarkers and was able to achieve normal levels of CSF β-Gal activity and GM1 gangliosides, similar to healthy controls
• Dose 2 biomarker responses demonstrated durability up to 12 months after treatment
• Imagine-1 study participants showed initial evidence of improved survival relative to natural history data
• Treated first patient at highest dose level, Dose 3, in July; initial safety and biomarker data from Dose 3 expected by mid-2024
• Management to host a conference call and webcast today at 8:30 a.m. ET

PHILADELPHIA, Aug. 07, 2023 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders, today announced new interim safety, biomarker, and survival data from cohorts 1-4 in the Imagine-1 clinical study. Imagine-1 is a Phase 1/2, global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna (ICM) injection in six cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis (GM1). GM1 is a rare, fatal lysosomal storage disease in which mutations in the GLB1 gene result in very low activity of the enzyme beta-galactosidase (β-Gal).

"We are highly encouraged by the compelling data emerging from our Imagine-1 study, with results underscoring the potential of PBGM01 to be a transformative therapy for GM1 patients,” said William Chou, M.D., president and chief executive officer of Passage Bio. “Updated data from the first eight treated patients show that PGBM01 continues to have a favorable safety profile, is well tolerated and shows initial evidence of improved survival relative to what is predicted by the natural history of the disease. Furthermore, Dose 2 of PBGM01 has shown the ability to achieve healthy control levels of β-Gal activity and GM1 gangliosides in CSF, with durability up to 12 months after treatment.”