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Passage Bio Presents New Interim Clinical Data for Patients with GM1 Gangliosidosis in Imagine-1 Study at 2022 WORLDSymposium
Both children with late infantile GM1 gangliosidosis in Cohort 1 of the Imagine-1 clinical trial showed developmental improvement in assessments by study
About this update from Passage Bio, Inc.
[{"type":"text","content":"Both children with late infantile GM1 gangliosidosis in Cohort 1 of the Imagine-1 clinical trial showed developmental improvement in assessments by study investigators and caregiversInterim safety data at three and nine months in Imagine-1 showed low dose PBGM01 well tolerated with no serious adverse events reported and no evidence of dorsal root ganglion toxicityFirst patients dosed in Cohort 2 (late infantile with high dose PBGM01) and Cohort 3 (early infantile with low dose PBGM01); anticipate reporting initial biomarker and safety data in 2H2022Management to host conference call and webcast on Friday, February 11 at 1 p.m. ET PHILADELPHIA, Feb. 11, 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 presented updated clinical data from Imagine-1, a Phase 1/2 study of PBGM01, a gene therapy for GM1 gangliosidosis (GM1). The platform presentation at the 18th Annual WORLDSymposium included clinical assessments of development milestones for Cohort 1 (two patients with late infantile GM1 who were administered a low dose of PBGM01). Additionally, the presentation reviewed safety and biomarker data first announced in December 2021. Life expectancy for children with GM1, a rare lysosomal storage disorder caused by mutations in the GLB1 gene, ranges from 2 to 10 years. Currently there are no approved disease-modifying treatments for the disorder. David Weinstein, M.D. M.M.Sc., senior vice president, Clinical Development, Passage Bio, presented interim data showing meaningful improvement in development milestones and no reported serious adverse events or complications related to intra-cisterna magna delivery. Development milestones were evaluated using two standardized, norm-referenced scales – the Bayley III, a formal assessment tool used by trained healthcare providers, and the Vineland II, a scale for parent or caregiver assessment. “The interim biomarker and safety data reported in December for these first two patients were positive indicators that PBGM01 is exerting a biological effect, and it is also important to see improvement in developmental milestones for these children,” said Eliseo Salinas, M.D., MSc., chief research and development officer, Passage Bio. “We are very encouraged...