Passage Bio, Inc. · NASDAQ:PASG
Passage Bio — American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting (May 2022)
17 slides from Passage Bio's conference presentation, May 18, 2022
Slide 1: Interim Safety, Biomarker, and Efficacy Data from Imagine-1
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
Slide 2: Notice
2 Notice • This presentation is about a research study aimed at assessing the safety and efficacy of an investigational treatment called PBGM01. ▪ Use of PBGM01 is not approved by any of the regulatory agencies for the treatment of GM1. ▪ The safety and efficacy of PBGM01 for the treatment of GM1 ha
Slide 3: GM1 Gangliosidosis
3 GM1 Gangliosidosis • GLB1 gene mutation results in deficient β - Gal enzyme activity leading to intracellular ganglioside accumulation 1 • GM1 gangliosidosis is characterized by progressive neurological deterioration, progressive brain atrophy, cardiac and skeletal abnormalities 2 • Estimated inci
Slide 4: Clinical Course of Infantile GM1
4 Clinical Course of Infantile GM1 GM1 Gangliosidosis is a Continuum Disease Severity Residual Enzyme Activity 1,3 - 6 Imagine - 1 Trial will include Type I (Early Infantile) and Type IIa (Late Infantile) patients 7 Negligible ~1 - 5% ~3 – 10% Type I (Early Infantile) 1 • Onset <6 months 1 - 5 • Hyp
Slide 5: Imagine-1: Global Phase 1/2 Trial with PBGM01
5 Imagine - 1: Global Phase 1/2 Trial with PBGM01 Initial safety and biomarker data from Cohorts 2 & 3 expected in 2H 2022 Trial Design Phase 1/2, multi - center , open - label, dose escalation and confirmatory study Route of Administration Intra - cisterna magna (ICM) Vector AAVhu68 Duration Two ye
Slide 6: Cohort 1: Baseline Patient Characteristics
6 Cohort 1: Baseline Patient Characteristics Patient 1 Patient 2 Diagnosis Late Onset Late Onset Dosing Cohort Low dose Low dose Gender Male Male Age at onset of symptoms (months) 14 12 Age at diagnosis (months) 2 30 Age at gene transfer (months) 15 31 DBS β - Gal activity * (nmol/mL/h) 0.0 0.2 Geno
Slide 7: Cohort 1 Safety & Tolerability
7 Cohort 1 Safety & Tolerability No SAEs have occurred in either patient Reported AEs were mild - to - moderate; all moderate AEs resolved without intervention and were deemed non - treatment related No complications related to ICM administration were reported No changes in ALT or GGT occurred in ei
Slide 8: Patient 1: Vineland-II
8 Patient 1: Vineland - II Sustained improvement documented in all developmental areas through 12 - month assessments Parameter Baseline 3 months 6 months 9 months 12 months Patient Age 14 17 20 24 26 Motor Gross motor 13 14 21 22 21 Fine motor 12 14 16 19 22 Language Receptive language 12 14 25 34
Slide 9: Developmental Gains for Patient 1 (Vineland-II)
9 Developmental Gains for Patient 1 (Vineland - II) Baseline 12 - month Visit Gross motor Taking a few steps without support Observed jumping Balancing on 1 foot Climbing Stairs Fine motor Sometimes able to squeeze toy Opening door and locks Pours liquid without spilling Stacking 4 blocks Receptive
Slide 10: Patient 1: Bayley-III
10 Patient 1: Bayley - III Sustained improvement also directly observed in all developmental areas Parameter Baseline 3 months 6 months 12 months Patient Age 14 17 20 26 Motor Gross motor 13 14 15 15 Fine motor 11 16 16 17 Language Receptive language 10 16 16 18 Expressive language 10 14 15 19 Cogni
Slide 11: Patient 2: Vineland-II
11 Patient 2: Vineland - II Continued improvement despite having severe developmental delay at baseline and prior history of regression Parameter Baseline 3 months 6 months Patient Age 31.6 34.5 37.6 Motor Gross motor 11 12 13 Fine motor 11 12 12 Language Receptive language 14 13 20 Expressive langu
Slide 12: Developmental Gains for Patient 2 (Vineland-II)
12 Developmental Gains for Patient 2 (Vineland - II) Baseline 6 - month Visit Gross motor Stand with support for 5 seconds Pulls to standing position Standing without support for 1 - 3 minutes Walks without support 2 - 3 steps Throws ball Fine motor Sometimes able to squeeze toy Transfers object fro
Slide 13: Patient 2: Bayley-III
13 Patient 2: Bayley - III Overall improvement observed for motor, language, and cognitive domains Parameter Baseline 3 months 6 months Patient Age 31.6 35 37.6 Motor Gross motor 10 12 11 Fine motor 7 9 7 Language Receptive language 4 9 11 Expressive language 7 7 6 Cognitive Cognition 8 9 13 Overall
Slide 14: Cohort 1 Volumetric MRI Data
14 0 200 400 600 800 1000 Whole brain Cerebral cortex Cerebral white matter Cerebellum cortex Cerebellar white matter Baseline Month 6 Month 12 ↑ 15% ↑ 15% Patient 1: Change from Baseline at Month 12 (Age at gene transfer: 15 months) Patient 2: Change from Baseline at Month 6 (Age at gene transfer:
Slide 15: Cohort 1 Interim Biomarker Data
15 Cohort 1 Interim Biomarker Data *Baseline reflects the average of two samples collected within 48 - hours of dosing. † Based on preliminary data from University of Pennsylvania’s ODC NHS study (NCT04041102). b - Gal= b - galactosidase; CSF=cerebrospinal fluid; NHS=natural history study. NHS patie
Slide 16: Key Takeaways from Cohort 1 Interim Data
16 Key Takeaways from Cohort 1 Interim Data Low dose PBGM01 was well - tolerated with demonstration of functional transgene expression and developmental improvement AEs =adverse events; ICM= intra - cisterna magna .. SAFETY Positive safety profile and well - tolerated with no serious AEs and no comp
Slide 17: Acknowledgements
17 Acknowledgements Thank you to the patients, families, caregivers for their participation in this study. Thank you to the coordinators and site study staff who are instrumental in making this work happen. Co - authors Caroline A Hastings, UCSF Benioff Children’s Hospital Debra - Lynn Day - Salvato
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