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Passage Bio Announces Positive Interim Clinical Data from First Six Patients with GM1 Gangliosidosis in Imagine-1 Study
Interim safety data up to 20 months showed the low and high dose of PBGM01 were well tolerated and had a favorable safety profileIntra-cisterna magna
About this update from Passage Bio, Inc.
[{"type":"text","content":"Interim safety data up to 20 months showed the low and high dose of PBGM01 were well tolerated and had a favorable safety profileIntra-cisterna magna administration of PBGM01 resulted in dose-dependent increases in CSF β-Gal activity and decreases in CSF GM1 gangliosidesPBGM01 administration resulted in a meaningful improvement across developmental areas in a subset of patients, as assessed by investigators and caretakersCompleted dosing of all patients in dose-ascending portion of study; initial safety and biomarker data from Cohort 4 expected by mid-2023Management to host a conference call and webcast today at 8:30 a.m. ET PHILADELPHIA, Dec. 14, 2022 (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 clinical development results from cohorts 1-3 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 four 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). The interim data include six treated patients from the first three cohorts. Cohort 1 (late infantile, low dose), Cohort 2 (late infantile, high dose) and Cohort 3 (early infantile, low dose) each consisted of two patients. Cohort 4 (early infantile, high dose) patients have been dosed and data is expected by mid-2023. “We are excited to share interim data from this first six patients in our Imagine-1 study, which further reinforce our confidence in PBGM01 as a promising treatment option for GM1 gangliosidosis,” said William Chou, M.D., chief executive officer of Passage Bio. “The goals of this Phase 1/2 study are to establish the safety profile for PBGM01, determine the optimal dose for therapeutic effect, and gain understanding of how PBGM01 can benefit patients across infantile GM1 populations. We are encouraged by emerging trends in the data and look forward to receiving results from Cohort 4 to further inform our program and support interactions with regulatory authorities...