PBGM01 / Passage Bio - LARVOL DELTA

Imagine-1: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=26 | Recruiting | Sponsor: Passage Bio, Inc. | Active, not recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Lysosomal Storage Diseases • Pediatrics • GLB1 • NEFL

Imagine-1: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=26 | Active, not recruiting | Sponsor: Passage Bio, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders • Lysosomal Storage Diseases • Pediatrics • GLB1 • NEFL

Imagine-1: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=26 | Recruiting | Sponsor: Passage Bio, Inc. | Active, not recruiting ➔ Recruiting | N=20 ➔ 26

Enrollment change • Enrollment open • Genetic Disorders • Lysosomal Storage Diseases • Pediatrics • GLB1 • NEFL

Passage Bio Reports First Quarter 2023 Financial Results and Provides Recent Business Highlights (GlobeNewswire) - "Anticipated Upcoming Milestones: Present initial safety and biomarker data from Cohort 4 (early infantile, high dose) in the Imagine-1 clinical trial for GM1 in mid-2023. Dose first patient at higher dose (Dose 3) in Imagine-1 clinical trial for GM1 in the second half of 2023. Present initial safety and biomarker data for Cohort 1 in upliFT-D clinical trial for FTD in the second half of 2023."

P1/2 data • Trial status • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders

Imagine-1: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=20 | Active, not recruiting | Sponsor: Passage Bio, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders • Lysosomal Storage Diseases • Pediatrics • GLB1 • NEFL

GM1 Treatment PBGM91 Shows Positive Interim Data in Phase 1/2, Additional Cohorts Planned (NeurologyLive) - P1/2 | N=20 | Imagine-1 (NCT04713475) | Sponsor: Passage Bio | "'Children with infantile GM1 experience a rapid decline in neurologic function and unfortunately there are no disease-modifying treatments available to them,' principal investigator for the Imagine-1 study,' Roberto Giugliani, MD, PhD, MSc...said in a statement. 'The disease is devastating to patients and their families, so I am encouraged by the early data with PBGM0 - that it was not only well-tolerated but also showed an increase in activity of the beta-galactosidase enzyme in both serum and CSF. I look forward to the continued clinical advancement of PBGM01 and learning its potential for GM1 patients.'"

Media quote • P1/2 data

Passage bio presents new interim clinical data for patients with GM1 gangliosidosis in Imagine-1 study at 2022 worldsymposium - "'After initially gaining early milestones, children with late onset GM1 typically plateau at 12-13 months of age developmentally before regressing. Seeing a child continuing to achieve new developmental milestones at 2 years of age is highly encouraging and atypical of this condition,' said Roberto Giugliani, MD, PhD...Imagine-1 principal investigator. 'In 35 years of seeing GM1 patients, I have also never seen a child regain the ability to walk after losing this skill. While still relatively early, these results are exciting, and I look forward to the continuation of this trial.'"

Media quote

Interim Safety, Biomarker, and Efficacy Data From Imagine-1: A Phase 1/2 Open-label, Multicenter Study to Assess the Safety, Tolerability, and Efficacy of a Single Dose, ICM Administration of PBGM01 in Subjects with Type I (Early Onset) and Type IIa (Late Onset) Infantile GM1 Gangliosidosis (GM1) (ASGCT 2022) - P1/2 | "Interim safety, BM, and developmental data from these initial pts support the effectiveness of PBGM01 as a disease-modifying treatment of infantile GM1."

Biomarker • Clinical • Late-breaking abstract • P1/2 data • CNS Disorders • Developmental Disorders • Genetic Disorders • Lysosomal Storage Diseases • GLB1

Imagine-1: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2; N=20; Recruiting; Sponsor: Passage Bio, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Pediatrics • MRI

Imagine-1: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2; N=20; Not yet recruiting; Sponsor: Passage Bio, Inc.

Clinical • New P1/2 trial • Pediatrics • MRI

Study of Safety, Tolerability, Efficacy and of PBGM01 in Pediatric Subjects with GM1 Gangliosidosis (clinicaltrialsregister.eu) - P1/2; N=28; Sponsor: Passage Bio, Inc.

Clinical • New P1/2 trial • Pediatrics